Hem philia Canadian Hemophilia Society TODAYServing the Bleeding Disorders Community SPRING 2007 www.hemophilia.ca VOL 42 NO 1

YOUTH FILE OPENED page 13

RESEARCH TWINNING WITH TUNISIA page 23 SUMMIT page 5

OUR STORIES page 14

GLOBAL PERSPECTIVE • MEDICAL NEWS • CHAPTER SPOTLIGHT • OUR STORIES • THE BLOOD FACTOR 2 HEMOPHILIA TODAY SPRING 2007

Hem philia Canadian Hemophilia Society TODAYServing the Bleeding Disorders Community

Spring 2007 • VOL 42 • NO 1

Hemophilia Today 625 President Kennedy Avenue, Suite 505 Montreal, Quebec H3A 1K2 www.hemophilia.ca Phone: 514 848-0503 Fax: 514 848-9661 Toll-free: 1 800 668-2686 [email protected] We would like to thank the following companies, corporate foundations and employee fund programs for their generous support.Our way of recognizing them Hemophilia Today is the official publication of the Canadian Hemophilia Society (CHS) and appears three times yearly. for their generosity is through our National Corporate Giving Program. The Canadian Hemophilia Society strives to improve the health and quality of life for all people with inherited bleeding disorders and to find a cure. Its vision is a world free from the pain and suffering of inherited bleeding disorders. The purpose of Hemophilia Today is to inform the hemophilia and bleeding disorders community about current news and relevant issues. Publications and speakers may freely use the information contained herein, provided a PLATINUM credit line including the volume number of the issue is given. Opinions expressed are those of the writers and do not BAYER necessarily reflect the views of the CHS. The CHS consults medical professionals before distributing any medical information. However, the CHS does not practice medicine and in no circumstances recommends GOLD particular treatments for specific individuals. In all cases, it is recommended that individuals consult a physician before BAXTER pursuing any course of treatment. Brand names of treatment products are provided for CSL BEHRING information only. They are not an endorsement of a particular product or company by the writers or editors. NOVO NORDISK WYETH

EDITOR François Laroche SILVER

PRESIDENT DEWDNEY FAMILY CHARITABLE FOUNDATION Eric Stolte

EDITORIAL COMMITTEE BRONZE Hélène Bourgaize DELTA HOTEL WINNIPEG Clare Cecchini François Laroche DELUCA’S SPECIALTY FOODS David Page Chantal Raymond EDELSTEIN DIVERSIFIED CO. LTD. Patricia Stewart FONDATION DE BIENFAISANCE T.A. GERMAIN (LA)

CONTRIBUTING WRITERS JOHN BROUWER FOUNDATION (THE) Sarah Bradshaw JOHN DEERE FOUNDATION OF CANADA Clare Cecchini Dianna Cunning LILLIAN AND DON WRIGHT FOUNDATION (THE) François Laroche David Page MANITOBA LIQUOR CONTROL COMMISSION Andrea Pritchard, RN, PhD(c) PRICEWATERHOUSE COOPERS Jeff Rice Nisa Renault, BSc, PhD(c) SCHERING CANADA Eric Stolte SCOTIABANK Emil Wijnker TORONTO STAR FRESH AIR FUND PRODUCTION COORDINATOR David Page THE WINNIPEG FREE PRESS

PRODUCTION ASSISTANT AND FRENCH VERSION COORDINATOR Chantal Raymond

GRAPHIC DESIGN We would also like to thank our numerous additional donors – individuals, Paul Rosenbaum corporations and foundations – who each year express their confidence in us by making substantial supporting donations. TRANSLATORS Roy Keys Line Ladouceur Working together with the corporate sector in Canada helps the Canadian Marie Préfontaine Hemophilia Society (CHS) accomplish its mission and vision by extending our reach and reinforcing our messages. This enables us to… ▪ Offer national programs of training, education and awareness. ▪ Support research into hemophilia and other bleeding disorders. ▪ Produce educational publications, periodicals (such as Hemophilia Today) and keep our Web site www.hemophilia.ca up to date. ▪ Interact with stakeholders in the health care field to promote the www.hemophilia.ca well being of all our families. HEMOPHILIA TODAY editorial pages SPRING 2007 3

IN THIS ISSUE WORD SPRING 2007 VOL 42 FROM THE François Laroche NO 1 EDITOR

WORD FROM THE EDITOR ...... 3 PRESIDENT’S MESSAGE ...... 4 ou’ve probably heard it said that it’s easier to reach the top than to stay there. COMMUNITY NEWS Y This is often true, at least in professional sport. Some teams are winners thanks to CHS Research Summit: an alignment of the stars that brings them luck, helping them garner top honours Charting the way forward...... 5 one year, only to become nothing more than a good team the next year, barely able CHS approves funding for 7 research to qualify for the playoffs. It’s also like this for our organization. projects in 2007 ...... 6 Over the past 50 years or more, through the hard work of volunteers and dedi- Notice of Settlement: Pre-86/Post-90 Hepatitis C ...... 7 cated health care providers, the CHS has managed to create a state-of-the-art care What’s new on the CHS Web site? ...... 8 system for the treatment of hemophilia and other bleeding disorders. Thanks to the unrelenting work of a handful of these stalwarts, we have access to the highest qual- Rendez-vous Québec ...... 9 ity care that is the envy of people with bleeding disorders all over the world. For all Chapter spotlight ...... 9 practical purposes, we have reached the top. Upcoming events ...... 12 In the coming years, however, we will face a big challenge. Now that we’ve reached the top, we have to stay there. And a number of signs I’ve picked up here YOUTH FILE...... 13 and there as the country’s blood system is being reformed tell me that it’s going to OUR STORIES ...... 14 be a tough fight. MEDICAL NEWS In Quebec, the process of decentralizing budgets for blood and blood products Hep C press review...... 15 from Héma-Québec to designated hospitals already poses a threat to the integrity of Exploring rural health professionals’ needs in our care. Of course, the main advantage of this new approach is that it makes hos- providing hemophilia care ...... 16 pitals more accountable when using blood and blood products—which were being supplied to them for free—as opposed to substitutes—for which they have had to THE BLOOD FACTOR pay until now. Shifting the budgets to hospitals does, however, bring with it a num- Letter on Hemophilia Inhibitor Genetics Study ..18 ber of real or possible drawbacks for people with bleeding disorders. First of all, it Blood factor news ...... 18 creates an additional line item—and a major one—in hospital budgets. Two line vCJD risk from plasma-derived factor VIII items, actually, since fractionated products are separate from fresh components. “is extremely low but may not be zero”...... 19 Any hospital administrator with an iota of sense will eventually discover that the Factor VIII and IX concentrates licensed/ cost of fractionated products (including factor concentrates) is very high, and try to distributed in Canada...... 20 cut back—to the detriment of users of these products. The decentralization will THE FEMALE FACTOR also create a rift between the prescriber of the products—who practices in the hemophilia treatment centre (HTC)—and the distributor—who works in the hos- Symptomatic carriers: Can women and girls really express hemophilia? ...... 22 pital or is located at the regional blood bank. It would obviously be tempting for doctors in remote regions to control prescriptions for products that are budgeted THE GLOBAL PERSPECTIVE and distributed in their hospitals, with the inevitable consequences: improperly pre- Quebec Chapter – Tunisia twinning visit ...... 23 scribed drugs, budget overruns, and eventually, loss of the expertise currently exist- ing in the HTCs. But another threat also lurks… Now that factor concentrates produced by genet- ic engineering no longer contain human biological products (2nd generation prod- ucts, like Kogenate® FS and Niastate® contain albumin in trace amounts, while 3rd generation products like Advate® and Benefix® contain none), it would be tempting for provincial health officials to reclassify them as pharmaceutical products. As a result, these would no longer be paid for from the blood products budget, but would be covered by the users’ public, group or private insurance plans—with all the exasperation that would entail for someone with a chronic condition like hemo- philia. The possibility of discontinuing tracking of 3rd generation products was recently brought before the Comité d’hémovigilance du Québec. After the CHS’s position was presented and discussed, the idea of discontinuing product tracking was set aside for the time being. But it is only a matter of time before it’s back on the table, either in Quebec or somewhere else in Canada. We must remain vigilant if we don’t want the quality care for which we’ve fought these many years to gradually wither away. We certainly don’t want to return Steve and Mike Myers, p.14 to the dark ages of the 1950s. 4 HEMOPHILIA TODAY editorial pages SPRING 2007

Dear readers, PRESIDENT’S In the course of our subscription address update for Hemophilia MESSAGE Today, we were pleased and touched by the many appreciative ERIC STOLTE comments you took the time to send us. Below are some of the messages received. In addition to your written words, many of Dream our dream you were extremely generous with a total of more than $8,000 in donations! From all of us at the CHS and the Hemophilia “If you lose hope, somehow you lose the vitality that keeps life Today editorial team, thank you for this extraordinary support. moving, you lose that courage to be, that quality that helps you go on in spite of it all. And so today I still have a dream.” These words of Dr. Martin Luther King, Jr. were penned in the book, The Trumpet READERS’ COMMENTS… of Conscience, not long before his death. When I think of hope in our ▪ context, I think of cure. That cure will come from research. Thank you for the many printed issues of Hemophilia Today that you have sent me over the years. I have now subscribed to We, like Dr. King, have a dream. We dream of a world free from the email version. the pain and suffering of bleeding disorders. We strive to improve Let me congratulate you on your newsletter. It is beautifully the health and quality of life for all people with bleeding disorders done. As a former newsletter writer, I know how expensive it is and to find a cure. to carry out this activity. I also know that it is appreciated by The Hemophilia Research Million Dollar Club was created to readers. I have greatly enjoyed having the Canadian perspec- help ensure that cure is found. The effort to raise the endowment tive, and, tidbits of news I might otherwise not encounter. Let that has now become a $1.8 Million Dollar Club is probably the me wish you every success! most successful fundraising initiative ever undertaken by the CHS. Carol K. Kasper, MD This demonstrates the power of hope in a cure. Emerita Professor of Medicine, University of Southern California, There is an ancient Hebrew proverb that says, “Hope deferred Orthopaedic Hospital makes the heart sick, but desire fulfilled is a tree of life.” A modern ▪ Thank you for this wonderful publication. Although I am pro- translation of that same proverb states, “Unrelenting disappoint- environment, the paper issue is very useful for our meetings ment leaves you heartsick, but a sudden good break can turn life with the parents. (free translation) around.” My son recently turned thirty years of age. (I can’t be that Yolaine Houle, SW old, can I?) I remember hearing soon after his birth that a cure was Sainte-Justine Hospital, Montreal, QC only fifteen or twenty years away. Then, many times since, I have ▪ I am a fan of Hemophilia Today. heard the same prediction. Hope deferred. Dr. S. Kaiser Ali But we have had some “good breaks”. Recombinant products pro- Saskatoon, SK vide safety, efficacy and reliable supply. On the near horizon are ▪ products with longer half-lives. Gene therapy holds the promise of Thanks for a very informative magazine. increased factor levels for longer periods of time. All these advances Mr. Chris Slocombe Colchester, Essex, England mean a betterer quality of life. The recent CHS Research Summit, held in February in Toronto, ▪ Congratulations – publication very informative and VERY was attended by physicians, nurses, physiotherapists, social workers, WELL done. Keep up the GREAT work. CHS Board and staff members, past and current researchers, repre- Dr. Ron and Leni George sentatives from Canadian Blood Services, Héma-Québec, the U.S. Calgary, AB National Hemophilia Foundation, the five pharmaceutical compa- ▪ On behalf of the GREEK HAEMOPHILIA SOCIETY (GHS), nies in the Canadian factor concentrate market and the President of I would like to thank the Canadian Hemophilia Society for the Canadian Institutes for Health Research, Dr. Alan Bernstein. It sending us Hemophilia Today. All these years that we have been was a wonderful success. Since 1990 the CHS has channeled four receiving your publication we have shared the information million dollars into peer-reviewed research yet has never stopped to given in every issue. Please be generous and continue to send it analyse that investment. Through presentations and discussion there to us. We wish you every success in all your activities. emerged a strong sense that the CHS must give leadership in this Jane Pittadaki area. Our CHS Medical and Scientific Advisory Committee will now Executive Board Member, Chief editor of the Newsletter review the input and insights from the Summit in order to deter- “OUR NEWS” of GHS mine our best path forward. My heartfelt thanks to the organizing ▪ Many thanks to Hemophilia Today and to the Canadian committee of Bruce Ritchie, Tom Alloway and Craig Upshaw and Hemophilia Society for the years I have enjoyed your excellent the excellent staff support provided by David Page and Wendy Wong publication. in making this a landmark meeting. Dr. Campbell W. McMillan I’m also excited about the opportunity that Rendez-vous Québec Chapel Hill, NC, USA (May 24-27 in Quebec City) presents for us. Excellent consumer workshops along with interesting medical and scientific presenta- tions will make an event of great value. Visit our Web site, ERRATUM In the Fall 2006 issue of Hemophilia Today, p. 31, the article Where do you find www.hemophilia.ca, for more information. I look forward to seeing 25 wheelchairs? was written by Sandra Squire, BSc, PT in the Vancouver, BC you in Quebec City where, together, we can continue to hold on to Adult Hemophilia Program at St. Paul's Hospital. We apologize to Ms. Squire hope and dream our dream! for this unfortunate mistake. HEMOPHILIA TODAY community news SPRING 2007 5

disorders research. The COMMUNITY ilies cope. Research is also at responsibility for refining the top of the list of priorities NEWS these ideas and formulating for our donors. Finding a cure specific recommendations was is front and centre in our mis- given to the CHS Medical and sion statement. Attracting Scientific Advisory bright young clinicians and Committee. researchers is key to the future The proceedings of the of care for bleeding disorders. Research Summit, including summaries Therefore the research program and the CHS Research Summit: of all presentations and discussions, are results of this Summit are critical to the available on the CHS Web site Charting the way CHS and the bleeding disorder community. www.hemophilia.ca. forward The CHS received a very enthusiastic The CHS thanks those who agreed to response to its invitation to attend the speak at the Summit. We are grateful to David Page Research Summit. The attendees repre- all participants who volunteered their time CHS Director of Programs and Public Affairs sented a wide variety of perspectives: to advise us on our research programs. • the care providers: physi- The Research Summit n February 2, 2007, the Canadian cians, nurses, physiothera- would not have been pos- Hemophilia Society brought pists and social workers Research, sible without the generous together the Canadian bleeding • past and current O a cure and support of our Presenting disorder research community in what was researchers who have been Sponsor, Bayer, and our called the Research Summit. awarded CHS grants improved Supporting Sponsor, The Summit grew out of a desire to • research peer review com- quality of life. Baxter. ensure that the CHS is getting the maxi- mittees without whom we mum benefit from its research programs. could not operate our pro- While the CHS has invested 4 million dol- grams lars in research since 1989, it had never • Canadian Institutes of Health Research stopped to analyze how we are running • Canadian Blood Services and Héma- our programs. Can we be more effective? Québec Dr. Alan More efficient? Are there barriers to • our counterpart in the U.S., the Bernstein, applying to CHS for grants that we can National Hemophilia Foundation President, address? Is the focus of our research the Canadian • our industry partners Institutes of proper one? Are there partnerships we • members of the CHS Board of Health should be pursuing? Are there other Directors. Research, addressing the opportunities we should consider? Many said they were excited by the CHS Research When the CHS asks its members, What meeting and had never attended one like Summit. is our primary mission? Almost all reply, it before. Many ideas were put forward as Research, a cure and improved quality of to how the CHS can maximize the bene- life. Hope, through research, for a future fits of its research programs, find match- free of the pain and suffering of inherited ing funds and work even more closely bleeding disorders is what helps many fam- with other groups interested in bleeding

Participants at the Research Summit. Seated : Bruce Ritchie, MD; Lori Laudenbach, RN; Manuel Carcao, MD; David Page; Fred Ofosu, MD; Gonzalo Hortelano, MD; Greig Blamey, PT; Pam Hilliard, PT; Victor Blanchette, MD; David Lillicrap, MD; Maha Othman, MD. Standing: Lawrence Jardine, MD; Steve Green; Mark Blostein, MD, PhD; Wenda Greer, PhD; Tom Alloway, PhD; Ya-Gang Xie, MD; Anne Myerson; Pam Wilton, RN; Arlene C. Y. Ali, PhD; Jeff Bakker; Gordana Atanackovic, MD; Craig Upshaw; Eric Stolte; John K. Wu, MD; Patricia McCusker, MD; Dana Devine, PhD; Heinz Neuhaus; Paula James, MD; Connie Alvarez-Haines; Réal Lemieux, PhD; Anthony Chan, MD; Maureen Brownlow, SW; Jeff Rice. Missing from photo: Wendy Wong, Pauline Major. 6 HEMOPHILIA TODAY community news SPRING 2007

CHS approves funding for 7 research projects in 2007

The Canadian Hemophilia Society is pleased to announce that seven research grants have been approved by the CHS Grants Review Committee and will receive funding in 2007. Complete reports on this research will be published in the Fall 2007 issue of Hemophilia Today.

Canadian Hemophilia Society Research Grants Care Until Cure Research Program Supporting research towards improving the quality of life for The Care Until Cure Research Program was established in the persons with hemophilia and finding a cure have been goals of year 2000 in collaboration with Wyeth Canada. Wyeth Canada is the Canadian Hemophilia Society (CHS) since it was founded in engaged in the discovery, development, and commercialization of 1953. Since 1990, through funds provided by the Hemophilia human pharmaceuticals through recombinant DNA and other Research Million Dollar Club and the CHS, the Society provides technologies. basic scientific research grants and studentships aimed at This program allows Canadian investigators to conduct developing treatments for hemophilia and finding a cure. research on various medical and psychosocial aspects of bleeding Canadian Hemophilia Society Research Grants disorders. Grants are given for clinical research, including outcome evaluation, in fields relevant to improving the quality of life of per- Wenda L. Greer, PhD, FCCMG sons with hemophilia, persons with von Willebrand disease or Professor other inherited bleeding disorders, persons with related conditions Department of Pathology, Dalhousie University such as HIV or hepatitis C as well as carriers of an inherited bleed- ing disorder Halifax, Nova Scotia Project Title: The Role of X-inactivation in the expression of hemophilia A in women (Second year funding) JoAnn K. Nilson Physiotherapist Dr. Gonzalo Hortelano Saskatchewan Bleeding Disorders Program Associate Professor Saskatoon, Saskatchewan Department of Pathology, McMaster University Project Title: Creating meaningful messages for Hamilton, Ontario individuals with mild hemophilia Project Title: Gene therapy of hemophilia A (Second year funding) (Second year funding) Dr. Paula James, FRCPC Dr. Maha Othman Assistant Professor Adjunct Assistant Professor Queen’s University Department of Pathology and Molecular Medicine Kingston, Ontario Queen’s University Project Title: The prevalence of symptomatic Kingston, Ontario pediatric VWD Project Title: Platelet type von Willebrand disease: (First year funding) An underdiagnosed cause of excessive mucocutaneous bleeding? (First year funding) Dr. Rochelle Winikoff, FRCP Staff Hematologist Dr. William P.Sheffield CHU Ste-Justine Associate Professor Montreal, Quebec Department of Pathology and Molecular Medicine Project Title: Non-steroidal anti-inflammatory drugs McMaster University and menorrhagia revisited Hamilton, Ontario (First year funding) Project Title: Factor VII(a) clearance behaviour (First year funding) HEMOPHILIA TODAY community news SPRING 2007 7

Notice of Settlement Pre-86/Post-90 Hepatitis C Class Actions

he Government of Canada has agreed to settle several To read a complete version of the Settlement Agreement, go to: Class Action lawsuits commenced on behalf of people www.reko.ca/html/hepc_settleagreement.pdf Twho became infected with the hepatitis C virus (HCV) For more information: from receiving blood and blood products in Canada prior to Residents of British Columbia: www.kleinlyons.com 1986 and after July 1, 1990. The proposed Settlement is not yet Residents of Alberta: www.marshallattorneys.com final and must be approved by the Courts in British Columbia, Residents of Quebec: www.lauzonbelanger.qc.ca Alberta, Ontario and Quebec. Public hearings to approve the Residents of Ontario, Saskatchewan, Manitoba, the Atlantic settlement were held in each of those provinces during the Provinces, the Territories, and residents residing outside of months of February and March. Class Members and their fami- Canada: www.reko.ca/html/hepatitisc.html lies had the opportunity to give their views on the fairness of the Settlement to the Courts. If the Courts approve the For updates check the CHS Web site at: www.hemophilia.ca Settlement, further notices will be mailed to Class Members, posted on the Internet and published in newspapers explaining how Class Members may apply for compensation under the Settlement or choose to be excluded from the Settlement. To settle the lawsuits, the Government of Canada has agreed to establish a compensation fund in the amount of $962 mil- lion. People primarily or secondarily infected with HCV as a result of blood received in Canada during the period prior to More work to be done on MPTAP indexation January 1, 1986 and the period from July 2, 1990 to September 28, 1998 may apply for compensation under the Settlement. Across Canada, four provinces have already indexed Estates of infected Class Members who have died, family mem- to the cost of living those benefits individuals bers of infected Class Members, and the dependants of infect- receive as part of the Multi-provincial/territorial ed Class Members whose death was caused by their infec- Assistance Program (MPTAP). They are: Manitoba, tion with HCV may also apply for compensation. Ontario, Prince Edward Island, and Newfoundland Under the terms of the Settlement, the & Labrador (dark grey). The provinces of British Government does not admit liability. Columbia, Alberta, Saskatchewan, Quebec and New Approved claimants will receive lump Brunswick (red) have yet to index benefits provided sum compensation based on their age, to Canadians who became infected with HIV current disease level and the probability through tainted blood. Nova Scotia follows a of disease progression in the future. different program (light grey). Compensation is also available for peo- ple who have lost income or house- hold services as a result of their infec- tion with HCV, for estates of infect- ed Class Members who have died, and for dependants of claimants whose death was caused by their infection with HCV. If the Settlement British is approved by the Courts, Class Columbia Alberta Saskatchewan Manitoba

Members should be able to apply to Newfoundland receive compensation from the Settlement Ontario & Labrador Quebec in the first half of 2007. At the Settlement approval hearings, Class Prince Edward Island Counsel requested that their legal fees of approxi- New Nova Scotia Brunswick mately $37.29 million (plus GST and PST, where applicable) be approved by the Courts. The Government of Canada has agreed to add that amount to the settlement Representatives from the CHS, who are involved fund, as well as an amount for the administration of the settle- with Chapters in those provinces where benefits ment, so that no legal fees or administration expenses will be have not yet been indexed, continue to press deducted from the $962 million negotiated for Class Member their governments to act. compensation. 8 HEMOPHILIA TODAY community news SPRING 2007

NOTICE What’s new on the CHS Web site? ANNUAL GENERAL MEETING

The Annual General Meeting of the Canadian Hemophilia Society will convene as follows:

Saturday, May 26, 2007 - 8:30 a.m. at the Delta Québec Hotel, Quebec City

1. To receive the report of the Nominating Committee. Rendez-vous Québec – Find out about the Canadian 2. To acknowledge the Designated Directors of each Hemophilia Society Annual General Meeting and Medical Chapter. and Scientific Symposium that will take place in Quebec City, May 24-27, 2007. 3. To nominate candidates for the Director-at-Large www.hemophilia.ca/en positions on the CHS Board for 2007-2008. 4. To receive the audited financial statements of the Red, White & You event – Learn more about this outstanding Canadian Hemophilia Society for the year ended fundraising and awareness program intended to educate December 31, 2006. people about inherited bleeding disorders while generating needed revenue to carry out our mission. 5. To appoint an auditor for the ensuing year. www.hemophilia.ca/en 6. To approve the recommendation from the Board of Directors to amend the CHS by-laws to allow the Chair or a Co-Chair of the National Youth Committee to sit on the National Board of Directors as an ex-officio member.

That Section IV, Article 4.03 (c) of by-law no. 1 be amended as follows: A new expanded Web site section for Hemophilia Ontario The ex-officio directors for each term shall be the and its regions with news of recent and upcoming activities immediate Past President of the Corporation, the www.hemophilia.ca/en/8.1 Chairman of the Medical and Scientific Advisory Committee and the Chair or a Co-Chair of the National A full report on the CHS Research Summit, held February 2, Youth Committee to be selected by the National Youth 2007 in Toronto Committee. Each ex-officio director shall commence his www.hemophilia.ca/en term as director of the Corporation at the end of the annual general meeting of members of the Corporation The new CHS Case for Support and shall continue until the election by the Board of a www.hemophilia.ca/en/1.8 new President of the Corporation, a new Chairman of the Medical and Scientific Advisory Committee or a new A new section describing the international work of the CHS Chair or a Co-Chair of the National Youth Committee as and its Chapters applicable. For purposes of clarity, the ex-officio directors www.hemophilia.ca/en/11.6 shall not be entitled to vote to elect directors-at-large but shall be entitled to vote on all other issues of the board. If Our Stories – The “Bruise Brothers” qualified, an ex-officio director shall be eligible for re- www.hemophilia.ca/en/2.9 appointment. Regular updates to the home page highlighting “Upcoming 7. To transact such other business as may properly come events” in the bleeding disorders community. before this Annual General Meeting of the members of www.hemophilia.ca/en the Canadian Hemophilia Society.

James Kreppner Secretary www.hemophilia.ca HEMOPHILIA TODAY community news SPRING 2007 9

Rendez-vous Québec CHAPTER MAY 24-27, 2007 SPOTLIGHT

Nova Scotia Chapter

CHS Annual General Meeting Nova Scotia Chapter and Medical & Scientific Symposium to be in 2007 pumpkin race! and Consumer Workshops DELTA QUEBEC HOTEL, QUEBEC CITY Dianna Cunning SCHEDULE OF EVENTS am very excited to announce that this year the Nova Scotia THURSDAY, MAY 24, 2007 IChapter will be entering the Windsor/West Hants Pumpkin 8:30 - 20:30 New Team Workshop Festival. Over 40,000 people attended in 2006. This is a large 16:00 - 22:00 Registration media event. People from all over the world come to see this FRIDAY, MAY 25, 2007 regatta. The Farmer’s Almanac comes annually from Atlanta, Georgia with a media crew. The CBS Sunday Morning Early 8:00 - 9:00 Registration Show and even Martha Stewart were scheduled to come last year 9:00 - 17:00 CHS Medical & Scientific Symposium (simultaneous translation) but immigration and legalities prevented it. This event is huge. 17:00 - 18:30 Reception – Exhibit Area Numerous articles and photos have been in publications world 19:00 - 21:00 Youth Session wide. CBS provides full coverage of the festival along with cor- 19:30 - 20:30 Board Orientation porate sponsors. Nova Scotia Chapter will be a corporate spon- sor. Three Metro Halifax radio stations do a complete 2-week SATURDAY MAY 26, 2007 promotion prior to the pumpkin festival. There are also 3000 8:30 - 17:00 AHCDC Annual Meeting brochures in which we will be named. The media attention that CANHC Meeting this event and its corporate sponsors receive is enormous. CPHC Meeting We will receive a massive pumpkin for the regatta, carve it Social Work Group Meeting out and paint it with the Red, White and You theme. Wim Peters 8:30 - 9:30 CHS Annual General Meeting 9:30 - 9:45 Break has agreed to paddle the pumpkin across the Windsor River 9:30 - 12:00 Twinning Workshop with about 40 other competitors. (for those responsible for current We will have a float in the parade with a Nova Scotia Chapter WFH/CHS organizational twinning) banner. We will pass out candies and brochures about hemo- 9:30 - 11:00 Volunteer Recruitment Workshop philia and bleeding disorders. On the back of the float will be 11:00 - 12:00 Fundraising Workshop our pumpkin, Wim, chapter members and children. I truly feel 12:00 - 13:00 Lunch that we will get excellent exposure for our Nova Scotia Chapter 13:00 - 17:00 CHS Board of Directors’ Meeting and the CHS, and greater awareness of bleeding disorders. Our 13:00 - 17:00 All About Carriers Workshop or participation will promote education about bleeding disorders 13:00 - 15:00 Navigating the ER Workshop and help to raise donations. This will be the most coverage our 15:00 - 15:15 Break Nova Scotia Chapter has ever received. Let’s all be there in 15:15 - 17:00 Hepatitis C Information Session October to celebrate... together! 19:00 CHS Banquet

SUNDAY, MAY 27, 2007 We will receive a 8:30 - 14:00 CHS Board of Directors’ Meeting massive pumpkin for (continuation) the regatta, carve it 8:30 - 12:00 AHCDC Meeting (continuation) out and paint it with Medical Meetings (if required) the Red, White and 9:00 - 12:00 “Hands-on” Fundraising Workshop You theme. Wim 12:00 Lunch Peters has agreed to paddle the pumpkin across the Windsor River with about 40 PRINCIPAL SPONSORS other competitors. 10 HEMOPHILIA TODAY community news SPRING 2007

Quebec Chapter Hemophilia Ontario

he CHSQ’s spotlight activity, the annual members family Hemophilia Ontario has moved! Tweekend, was held on March 16 to 18 at Auberge Matawinie in St-Michel-des-Saints, and once again this year s of April 2, 2007 you can find Hemophilia Ontario at a was a resounding success. More than 180 people attended. Anew address. We’ve moved up in the world, to the eighth The program featured a variety of workshops that offered floor! Our new address is: something for everyone (coffee klatches for youngsters or par- Hemophilia Ontario ents, workshops on physiotherapy, blood product safety and 45 Charles Street East, Suite 802 supply, and even relaxation). One of our young members Toronto, ON M4Y 1S2 organized a fund-raising activity, the Factor Auction, as part of If you are in Toronto, please drop by and see our new digs! a school project. The event was a lot of fun, and managed to bring in a total of $912 for the organization. New Staff The organization’s Annual General Meeting, the focal point of the weekend, was held on Saturday afternoon. A number of Hemophilia Ontario has more than a few new employees to announce. presentations on results for the year 2006 and possible new Aaron Cantor is the new Executive Director for Hemophilia Ontario. paths of development for the CHSQ were made by volunteers Justine Casserly is working as Administrative Associate for and staff. The presence of three delegates from the Tunisian Hemophilia Ontario and TCOR. Andrea Lajdecki is our new Regional Service Coordinator for TCOR. Hemophilia Association provided an opportunity to update Terri-Lee Higgins is our new Regional Service Coordinator filling the members on our twinning project and raise awareness of the maternity leave in SWOR. Welcome to all the new staff in Ontario! quality of services provided in Canada, as well as the impor- tance of international cooperation projects between hemophilia associations. In his presentation, David Page explained how The staff of Hemophilia Ontario/TCOR: (from left to vital cooperation between CHS and provincial chapters is, as right) Administration and he reviewed a number of joint projects currently underway. Finance Coordinator In short, the 2007 weekend was enjoyable, useful, a great Andrée Delisle, HIV/HCV Program Manager Sarah forum for discussion and exchanging information, and Crymble, Executive Director spawned many promising ideas Aaron Cantor, Regional Service Coordinator (TCOR) for fundraising, programs, and Nigel Small – Front Row: the future of our organization. Regional Service Coordinator (TCOR) Andrea Mission accomplished! Lajdecki, Administrative Associate Justine Casserly.

The Lifetime Member Award was presented to François Laroche by Winter Wanakita 2007 Patricia Stewart for his outstanding contribution Sarah Bradshaw, to the organization’s advancement. Member of TCOR, National Youth representative On February 2-4, 2007, eleven brave young guys and girls set off to Camp Wanakita on Koshlong Lake in Haliburton, Ontario. For six of the young men, it was their first time on this winter trip; for five, it was their first time ever on a Hemophilia Ontario Youth (HOY) trip. We did everything from cross-country skiing, to snowshoeing, to our annual broomball game. This year we challenged an all-girls school from Oshawa. After a tough fight, Team Hemo prevailed, win- ning 2-0. We can’t forget about those who braved the high ropes in the middle of winter and the same group who lived through the almost six-hour drive home in the storm after hav- ing our chicken lunch... leave it to the boys to be boys! We’d like to welcome MJ, Trent, James, Greg and Korey to the group of select few who are brave enough to come up North with us in February. François Laroche (far right) presented the delegates from the Tunisian Hemophilia Special thanks go out to Julia Sek and Dane Pederson for Association with “dreamcatchers” as a memento of their visit to Quebec. Left to right: Taoufik Raissi, Secretary-general; Professor Raouf Hafsia, Vice-president; and taking the time to join us on our trip and planning everything Islem Nafti, President. so wonderfully for us. HEMOPHILIA TODAY community news SPRING 2007 11

Hemophilia Ontario – Winter Youth Trip

Manitoba Chapter Hemophilia Saskatchewan 2007 Bayer Healthcare’s Gala Dinner of Hemophilia Saskatchewan AGM Culinary Inspirations from Around the World aturday, March 10, 2007 was a busy day for Hemophilia he 2007 Manitoba Chapter Gala held on Saturday March 3, SSaskatchewan. The Annual General Meeting brought every- T2007 was a huge success! Our 230 guests enjoyed a one up to date with the business side of the organization. sumptuous meal prepared by 5 of Winnipeg’s best Chefs. Twelve members were elected to the board. Thank you to the committee who worked so hard for 8 months Following the AGM, there was good fellowship over a to ensure that the evening was perfect. Wendy Wong, the catered roast beef dinner. Canadian Hemophilia Society’s National Director of Resource After dinner, Darla Walz gave a presentation, Live Well with Development, came from Toronto to observe and was a huge Chronic Conditions, showing how you can control how you live help! This year we raised over $25,000. The 2007 Gala with a chronic condition. Committee is working to create a manual for the event and A wonderful skit by Colleen Beuhler and Janice Kelly from hope to share the information with other Chapters that might the Saskatchewan Bleeding Disorders Program illustrated how like to try this fundraiser. action plans can be very beneficial to the patients who attend clinic. In conjunction with the AGM there was a very successful cookie dough sale.

Alberta Chapter

ince our change in governance several years ago, the regions Shave strived to collaborate and create a single strategic direc- tion for the province, develop a synergistic approach to dealing 2007 Gala Head Table, left to right: Shane Keilback; Christine Keilback, 2007 with the health authorities, as well as expand our support and Gala Chairperson; Wendy Wong, CHS National; Guest MC Jon Ljungberg from CityTV Breakfast Television; Christine Ljungberg; CHS-MC President Tony Tavares; education programs into small rural communities outside of from Platinum Sponsor Bayer Healthcare, Tom Hsu; and Joanne Ukno, Business Calgary and Edmonton. This collaboration started under the Relations Manager, Bayer Healthcare, Pharmaceutical Division. direction of Craig Upshaw and Tony Niksic, the regional presi- dents, but has an increased focus with the expansion to include Clara Penner representing the Medicine Hat/Lethbridge region, and with the recently elected executive in the southern region. In November, during the Southern Region’s Annual General Meeting and Christmas gathering, a new executive was elected consisting of Darlene Gates (President), Stacey Johnston (VP), Crystal Verbeek (Treasurer) and Susan Anderson (Secretary). As part of our reaching out to all communities in the province, the Northern Region extended an invitation to all members of the Alberta Chapter to attend the annual “beach party” at West Edmonton Mall. This very popular event brings families together to share experiences and connect in a warm environment during Back row, left to right: Bruce Cameron, Shane Keilback, Cory Prestayko, John the harsh February Alberta winter. Families attended the beach Rogasky. Front row, left to right: Lynda Regan, Katherine Cameron, Christine Keilback, Wendy Wong, Donna Rogasky, Colleen Cario. 12 HEMOPHILIA TODAY community news SPRING 2007

UPCOMING EVENTS NOVA SCOTIA CHAPTER JULY 1-15, 2007, Camp Wanakita 2007 – As we ramp up MAY 2007 – Chapter Fundraising and Maritime Family for another year at camp, we are feeling prepared and Weekend Planning Committee Meetings. Royal Artillery ready to make this the best year yet! We have put in place Park Officer’s Mess, Halifax, Nova Scotia. a new registration process, post camp committee meeting and Camp Committee. We are ready to do this right! MAY 25, 2007 – Deadline for articles to be included in We are looking forward to providing a camp experience the June issue of the Nova Scotia Chapter newsletter. for 55 campers with inherited bleeding disorders. Hemophilia Ontario is excited about this new opportunity JULY 28-29, 2007 – Maritimes Family Weekend 2007 to and look forward to a great partnership. be held at Saint Mary’s University, Halifax, Nova Scotia. If your child is attending camp or you are a past camper and want to volunteer, please let us know. We need people JULY 2007 – Commencement of NS Chapter Quarterly with camping experience for feedback to make our camp Executive meetings. Cambridge Military Library, Royal program focused and most of all FUN! Artillery Park, Halifax, Nova Scotia. If you require any information about Hemophilia Ontario’s Camp Wanakita, please contact Justine Casserly, AUGUST 2007 – Maritimes Adventure Camp 2007 to be 1 888 838-8846. held at Scotian Glen, Nova Scotia. AUGUST 15-19, 2007, Summer Youth Trip – The next scheduled HOY trip is a Canoe Trip. We hope to see you QUEBEC CHAPTER this summer for our annual summer trip. Don’t delay in contacting Sarah Crymble (Hemophilia Ontario) or your MAY 5, 2007 – Bowl-a-thon (Quebec City) local Regional Service Coordinator for more information, and keep your eyes open for the flyers! Summer is just MAY 6, 2007 – Bowl-a-thon (Montreal) around the corner!

MAY 12, 2007 – Strategic planning day at the Estrimont Suites & Spa. HEMOPHILIA SASKATCHEWAN

JUNE 2, 2007 JUNE 1-3, 2007 – Just the Gals weekend at the Estrimont – Regina BBQ Suites & Spa. JUNE 9, 2007 – Saskatoon BBQ AUGUST 5-10, 2007 – 2007 Summer Camp at Camp Portneuf, located in St-Raymond-de-Portneuf. JULY 21-22, 2007 – Guys Getaway

SUMMER 2007 – Youth camping weekend The youth committee of the CHSQ is preparing its annual ALBERTA CHAPTER event. This year, young people aged between 15 and 28 are invited to go on a camping trip. JUNE 2007 – Northern Region: Family Picnic and Education event SEPTEMBER 15, 2007 – Deadline to send in applications for student bursaries. JUNE 30, 2007 – Southern Region: Zoo, Picnic and Education event

HEMOPHILIA ONTARIO SUMMER/FALL 2007 – Southern Region: Alberta Children’s Hospital new clinic open house and Passport to There are always many exciting developments in the works Fitness Educational event at Hemophilia Ontario and the regional offices. Plans are being made for a tri-regional Just the Guys event, a provin- SEPTEMBER 7-9, 2007 – Alberta Chapter: Annual cial educational summit for volunteer development, as well General Meeting and Family Retreat, Goldeye as the Toronto Marathon. All of these events will be held in the fall. The staff is working hard to make these events a Keep an eye out for Alberta Chapter announcements with success! registration and confirmation of event dates. HEMOPHILIA TODAY youth file SPRING 2007 13

EMIL WIJNKER Our first All-Canada event is going to tact your local youth representative or YOUTH be a Youth Forum at Rendez-vous Québec your Chapter’s or Region’s Board of where we will be sharing our plan of Directors. FILE action for 2007. Youth will have the If you want to get involved, want to opportunity to meet others from all help plan/organize an event, or just want SARAH CO-CHAIRS across Canada, network, attend a Medical some information about CHS’s new youth BRADSHAW Symposium on bleeding disorders and initiative, feel free to contact your local celebrate with the CHS-NYC as we offi- youth representative at the following cially launch our all-new Web page! If you addresses! They will be excited to hear Calling want to attend Rendez-vous Québec, con- from you! all youth! MEMBERS OF THE NATIONAL YOUTH COMMITTEE Crystal Verbeek (Alberta) [email protected] Emil Wijnker Sheri Jevne (Alberta) [email protected] n the fall of 2006, the Canadian Jesse Katzman-Beimuts (Saskatchewan) [email protected] Hemophilia Society identified the Silvana Moran (Manitoba) [email protected] need for a committee to address the I Jaime Villeneuve (Ontario – Ottawa/OEOR) [email protected] issue of youth involvement in the organi- zation, and so the National Youth Adam Del Gobbo (Ontario – Toronto/TCOR) [email protected] Committee was born! The CHS-NYC Sarah Bradshaw (Co-Chair) (Ontario – Toronto/TCOR) [email protected] consists of very excited and energetic Greg Stutz (Ontario – Hamilton/CWOR) [email protected] individuals who represent almost all regions of Canada who are working Emil Wijnker (Co-Chair) (Ontario – London/SWOR) [email protected] together “to support, promote and David Pouliot (Québec) [email protected] address youth involvement within the Martin Kulczyk (Québec) [email protected] organization.” We are currently working on acquiring a representative from British Patrick Syriani (Québec) [email protected] Columbia to complete our coast-to-coast Robert Maye (Newfoundland) [email protected] committee! The Committee is co-chaired Meech Kean (New Brunswick) [email protected] by Sarah Bradshaw and me and is sup- ported by Hélène Bourgaize, CHS Katie Hines (Nova Scotia) [email protected] Director of Volunteer Development and Human Resources, and Julia Sek, Ontario Hemophilia Provincial Coordinator. At our first meeting held in Montréal last February, the committee identified the various roadblocks in our various communities to youth involvement. We are currently working towards overcom- ing them and engaging Canadian youth with bleeding disorders in order to sup- port each other and develop amazing local, provincial and national youth pro- gramming. We recognized that some regions already had very developed youth programming and some regions wanted to do this, but needed some help getting the ball rolling. That is one of our main goals and we have begun working togeth- er on an unprecedented national level towards this end! Keep your eyes and ears open for awesome youth-oriented pro- grams coming to a Chapter/Region near Members of the newly created National Youth Committee met for the first time in Montreal on you! February 10 and 11, 2007. 14 HEMOPHILIA TODAY our stories SPRING 2007

As Steve was proud to point out, “He asked Mike and I to autograph a poster for the wall of his club.” UR In 2005 they recorded their CD Straight From the Woods O with the legendary delta drummer Sam Carr of the Jelly Roll STORIES Kings. In 2007 they will be returning to the delta to record another session for their upcoming CD release. The brothers have factor VIII levels of 20 to 30% and, since they have mild hemophilia, are not on home infusion. They The “Bruise Brothers” receive treatment as needed at the Hemophilia Treatment Centre in Ottawa and in emergency situations rely on their play the blues FactorFirst cards. When asked if hemophilia has affected their lives as musicians, Mike mentioned that he suffers from arthri- tis in his hands and has had to adapt the picking style he uses when playing the guitar. He also recalled the time that Steve bit Clare Cecchini, CHS Program Coordinator his tongue minutes before a show and it was bleeding badly during the performance. Mike joked, “It’s lucky that he doesn’t do the vocals!” t has often been said that to play the blues you have to have Steve and Mike are both married and Mike has a ten-year- lived the blues, or at least overcome challenges or hard times old son. Like many musicians, the brothers work by day and at some point in life. In the case of Steve and Mike Myers, play the blues by night. Mike is involved in a family-run pollu- I tion abatement environmental business and Steve recently com- blues musicians from Ottawa, Ontario, their experiences growing up with mild hemophilia in the 1960s and 70s led them to their pleted a degree in criminology at Carleton University. They own special style of Mississippi hill-country juke joint blues. plan on retiring within ten to fifteen years, which would give The Myers brothers were diagnosed at a young age with mild them the opportunity to realize their dream of going on the hemophilia when they almost bled to death after having their road for several months a year to perform the blues. tonsils removed. There was no history in the family. After the Members of the Ottawa and Eastern Ontario Region diagnosis, they were discouraged from participating in active (OEOR) of Hemophilia Ontario had a unique opportunity to sports like hockey and picked up instruments instead of baseball hear the Myers Brothers, or as they sometime refer to them- bats. selves, the Bruise Brothers, perform on March 24, 2007 at the “Without realizing it at the time, this was probably our first Region’s Annual General Meeting and Family Weekend. In past real experience with the blues,” Steve remembers. years, Steve and Mike volunteered on the OEOR Board and Even though the boys could not play hockey or go roller- attended several meetings and retreats, but this was the first skating, their father kept them busy doing outdoor activities like time that they had performed for the CHS. camping, canoeing and fishing and never allowed them to use To find out more about the Myers Brothers Band, please visit their hemophilia as an excuse. their website at www.quazimojoblues.com. Living through the tainted blood tragedy in the 1980s and seeing the impact on the bleeding disorders community was another challenge. “Mike and I count our blessings for having made it through these tough times and our personal experiences are reflected in our blues.” Music was always a big part of their lives while growing up in Ottawa and the Gatineau region. Their mother’s French Canadian family was musical and many of their uncles and cousins played instruments, mostly old-time music and country & western. Their first contact with blues music occurred in the early seventies when they saw BB King performing on CBC tele- vision. As they began to explore playing blues music they were influenced by musicians like Johnny Winter, John Lee Hooker and Muddy Waters. In the early 1990s, the TV documentary Deep Blues/A Musical Pilgrimage to the Crossroads had a profound impact on their musical direction. They began a new musical and spiritual jour- ney, and developed their own style of “hypnotic trance-blues and rockin’ boogie”. By the late nineties they were travelling to the Mississippi delta on yearly pilgrimages and performing in “juke joints” in places like Memphis and other delta towns, including a club owned by Morgan Freeman. Mike Myers, seated on left; Steve Myers, standing on right. HEMOPHILIA TODAY medical news SPRING 2007 15

Occult HCV infection a milder disease For HIV positive and HIV negative than chronic HCV transplant recipients alike, however, HCV is associated with poorer outcomes, since Journal of Viral Hepatitis 14(1): 36-40. HCV nearly always infects the new liver MEDICAL Jan. 2007 – Occult or “hidden” HCV graft. NEWS infection is a term used to describe indi- Researchers compared outcomes for 9 viduals who have HCV RNA in their liv- co-infected liver transplant patients and ers, but no detectable HCV RNA or anti- the HIV negative HCV-infected patients Hep C press revue HCV antibodies in their blood. who received transplants before and after Spanish researchers previously deter- each co-infected patient. Immuno- Jeff Rice, mined that occult HCV was sometimes suppressive regimens (used to prevent CHS Hepatitis C Program Coordinator present in patients with persistently organ rejection) consisted of tacrolimus abnormal liver function of unknown with steroids or mycophenolate mofetil. Effectiveness of complementary and cause. Investigators sought to compare the Results indicate that acute cellular alternative medicine in hepatology characteristics of 68 patients with occult rejection occurred in 44% of the HIV- HCV infection and 69 untreated patients HCV co-infected patients and 22% of the Clinical Gastroenterology and with typical chronic HCV (detectable HIV negative patients. 3-year actuarial Hepatology. January 13, 2007 – Use of HCV RNA and anti-HCV antibodies in patient survival rates were 87.5% for the complementary and alternative medicines the blood), matched for sex, age, body co-infected group and 93.7% for the HIV (CAM) is increasing among individuals mass index and duration of abnormal negative group. Acute HCV re-infection with liver disease, with the most popular liver function tests. occurred earlier (2.3 vs 4.3 months) and CAM currently in use being herbal thera- The results indicate that AST and ALT was more cholestatic (mean bilirubin 10.8 pies. The single most commonly used levels were higher in patients with chronic vs 1.6 mg/dL) in the co-infected patients. herbal agent is silymarin, a mixture of the HCV than in those with occult HCV. 8 co-infected patients (100%) and 9 HIV active ingredients of the milk thistle plant. Chronic HCV patients also had higher negative patients (64.3%) received antivi- In animal models, many of the frequently levels of gammaglobulin, iron, and alpha- ral treatment with pegylated interferon used CAM agents have shown anti- fetoprotein, a marker associated with plus ribavirin. 1 co-infected patient (20%) inflammatory and antifibrotic effects. hepatocellular carcinoma. In contrast, and 1 HIV negative patient (11.1%) Although many human studies have cholesterol and triglyceride levels were achieved sustained virological response. shown improvements in subjective symp- significantly higher in patients with occult The authors wrote, “Short- to mid- toms (well being) and liver biochemistry, HCV infection. The rate of necro-inflam- term results of liver transplantation in “there are no convincing data to suggest a matory activity and fibrosis was higher HIV-HCV co-infected patients were excel- definite histologic and/or virologic among chronic HCV patients.The mean lent and similar to those of non-HIV - improvement with most of these agents.” percentage of infected hepatocytes (liver infected patients.” “Poorly designed studies, heteroge- cells) was higher in chronic HCV patients neous patient populations, lack of stan- than in those with occult HCV. dardized preparations, and poorly defined The authors wrote, “Occult HCV infec- Shorter, simplified treatment option non-objective end points may partly tion is a milder disease than chronic HCV, may encourage more patients to seek explain the conflicting reports in the liter- and this could be related to the signifi- treatment ature,” write the authors. cantly lower number of infected hepato- It has been well documented that liver cytes observed in occult HCV.” BASEL, Switzerland, March 6 – Some toxicity and/or unwanted drug interac- hepatitis C patients with difficult-to- tions may occur form the use of many treat HCV genotype 1 who respond herbal medications (e.g. St. John’s Wort Liver transplantation in HIV-HCV co- quickly to treatment with a combination adverse interaction with HIV protease infected patients: A case-control study of pegylated interferon alfa-2a plus rib- inhibitors). Physicians are urged to talk to avirin can benefit from a shorter and their patients about their potential use of Transplantation 83(3): 354-358. Feb. simplified course of therapy. Following a CAM in order to help avoid use of any 15, 2007 – Traditionally, people living European Union (EU) decision, a subset toxic agents or those that might cause with HIV were not considered suitable of patients with genotypes 1 and 4 HCV deleterious drug interactions. candidates for liver transplantation, but in who receive rapid viral response can now “Only well-designed, randomized, con- the era of highly active antiretroviral ther- receive a shortened, 24-week duration of trolled trials will be able to ascertain apy (HAART), studies have shown that treatment. whether CAM has any role in the man- HIV positive individuals with well-con- The EU approval is based on data agement of patients with acute or chronic trolled virus and relatively preserved from two pivotal clinical trials, with liver diseases. Until such time, the use of immune function can have post-trans- results showing that among patients CAM cannot be recommended as a thera- plant outcomes nearly as good as those of who achieved a rapid viral response py for patients with liver disease.” HIV negative individuals. continued on p. 16 16 HEMOPHILIA TODAY medical news SPRING 2007

continued from p. 15 happens, potentially the child. By contrast, the 3,800 reported cases (undetectable viral load at week 4) in the The risk of transmitting the virus from outside California in which couples with first month of treatment, up to 93% of an infected man to uninfected female an HIV-positive man have used reproduc- patients with genotype 1 HCV and with a through intercourse depends on the tive assistance hasn’t turned up a single low pre-treatment viral load, and 83% of amount of virus in the blood stream. It instance of transmission to the female patients with genotype 4 were cured fol- generally ranges from 1 in 1,000 to 1 in partner, said Cohan, an assistant professor lowing only 24 weeks of therapy – a simi- 8,000 per act of intercourse, Cohan said, in the University of California-San lar cure rate to that seen following 48 but those odds multiply the longer it takes Francisco’s Department of Obstetrics, weeks of therapy. to get pregnant. Gynecology and Reproductive Sciences. “This is excellent news for patients with hepatitis C,” said Dr. Peter Ferenci, Professor of the Department of Internal Exploring rural health professionals needs in Medicine IV, Gastroenterology and Hepatology at the University of Vienna. providing hemophilia care “This means that patients can find out within one month of starting therapy if they have an excellent chance of being cured and can benefit from a shortened …the urgent nature treatment duration.” of bleeding and the relative rarity of Bill would let men with HIV use hemophilia presents reproduction techniques an obvious challenge SACRAMENTO, March 6 – California for those healthcare State Senator Carole Migden (Democrat, providers practicing San Francisco) has introduced legislation that would allow couples to use repro- in geographically ductive services when the man has HIV. Andrea Pritchard, RN, PhD(c); isolated rural Before a couple could try artificial Faculty – Mount Royal College, Calgary, insemination or in vitro fertilization, the Alberta Nurse Coordinator, communities. man’s semen would first be treated by Calgary HTC, 1993-2005 sperm washing to minimize the chance [email protected] of transmitting HIV. Migden said current law discriminates Rural hemophilia care lenges that exist in the rural health care against HIV-positive men and that her setting (MacLeod, 1999). Given the nature legislation, Senate Bill 443, would ccording to the Public Health of bleeding disorders and coordination of “ensure equal reproductive rights for Agency of Canada (2003), “rural specialty care in a rural setting, this issue women,” regardless of their partner’s A Canada occupies … around 95% was identified as a priority to explore and HIV status. of Canada’s territory… [and] about 30% address. In particular, the question arose One HIV-positive man who lives in of Canada’s population live in rural and if rural providers had adequate prepara- the Sacramento area said he and his wife remote areas of the country. In rural tion and support for safe and effective have risked trying to conceive on their Canada, as in urban Canada, good health hemophilia care of HTC families. own, to no avail. Their last hope, he said, is a major resource for social, economic, is in vitro fertilization, but California law community and personal development.” Rural hemophilia study precludes them from trying. According to Taylor (1999) and UBC “She cannot get pregnant without (2001), Canadians living with bleeding This topic was formally studied as my reproductive assistance, but because I disorders identified difficulties associated Masters thesis research project with the have HIV there is no way for them to use with accessing rural healthcare services assistance of my classmate and rural my sperm. It’s unfair.’’ with required expertise. Furthermore, the health expert, Kari Simonson, and faculty California is one of two states where urgent nature of bleeding and the relative advisor, Dr. Kathleen Oberle; this article is couples with an HIV-positive male rarity of hemophilia presents an obvious dedicated in memory to Dr. Marlene can’t take advantage of so-called assist- challenge for those healthcare providers Reimer, Acting Dean-Department of ed reproduction services. Many of practicing in geographically isolated rural Nursing at the University of Calgary who those couples try to conceive instead communities. This is compounded by the was also an instrumental part of our through intercourse, increasing the risk fact that urban-based HTC providers may research team. Our study was recently of infecting the woman and, if that not be fully aware of the unique chal- published in October 2006 by the HEMOPHILIA TODAY medical news SPRING 2007 17

Australian Journal of Rural Health in an our rationale. We are still competent the upcoming Alberta provincial confer- effort to share hemophilia related infor- but different.” ence for registered nurses, physicians and mation to an audience that has wide- 2. Subjective knowledge – “The actual pharmacists. I hope that sharing these spread international rural readership. The care plan starts with the basics of hemo- findings will help interdisciplinary health following summary has been adapted philia care and objectives of assessment. care professionals learn more about from that article (Pritchard, Reimer, Then as the staff get to know the family bleeding disorders and collaborative part- Simonson & Oberle, 2006). they start to fine tune the care plan.” nerships in safe and effective care. Our study had two phases: focus 3. Team roles – “Our staff take care of groups (phase I) and telephone inter- many patients at once… hemophilia Acknowledgment views (phase II). We invited participation just happens in this larger picture. This by 20 interdisciplinary healthcare profes- needs to be respected: what we can do Sincere appreciation goes to Bayer for its sionals or ‘providers’ who worked in rural and what we are faced with.” generous support of this study through an communities that had at least one family 4. Objective knowledge – “We need drugs unrestricted grant. I would also like to living with hemophilia. These rural used, dosages, facts about hemophilia, give heartfelt thanks to the Calgary HTC providers were from five different hospi- what to watch for when they come in, families and staff – I miss you all very tals within southern Alberta and south- quick reference tools, guidelines and much and you have inspired me to con- eastern British Columbia, and included protocols. We need specific information tinue my doctoral studies in bleeding dis- nursing, medicine, lab technology, social about hemophilia care… We don’t need order care. work and physiotherapy. Learning and pages, just basics.” resource needs, capacities and related 5. Partnerships – “The hemophilia clinic * C-STOP is an acronym for barriers, and facilitators to rural hemo- is like the hub of the wheel with the Communication, Subjective knowledge, philia care were identified as 5 main spokes going out to everyone involved. Team roles, Objective knowledge, themes represented in our “C-STOP”* It’s not a quick fix – it comes as a whole Partnerships. model: package.” 1. Communication network - Facilitate References multidirectional, connection of fami- Using findings in practice lies, rural providers and specialty clinic 1. MacLeod M. “We’re it.” Issues and reali- staff. We used study findings to develop rural ties in rural nursing practice. W. Ramp, J. 2. Subjective knowledge - Provide readily resources at the Calgary HTC including Kulig, I. Townshend & V. McGowan (Ed.,) available individual care plans for fami- an interdisciplinary education day. This Health in rural settings: contexts for ly, rural providers and specialty clinic event focused on rural healthcare action (165-178). Lethbridge: University staff. provider education for nurses, physicians, of Lethbridge, 1999. 3. Team roles - Establish clarity of respon- lab technologists, and physiotherapists sibilities for family, rural providers and from hospitals throughout southern 2. Pritchard, A.M. (2004). Learning and specialty clinic staff. Alberta and southeastern British resource needs and capacities of rural 4. Objective knowledge - Make available Columbia, including 34 onsite partici- hemophilia health care providers. Masters succinct resources such as protocols, pants, and telehealth links to 5 rural hos- Thesis, University of Calgary. practice guidelines and standards. pitals. Presentations given by HTC, CHS 3. Pritchard, A.M., Reimer, M.A., 5. Partnerships - Recognize shared expert- and CBS representatives included topics Simonson, K. & Oberle, K. (2006). ise, affiliation, and accountability such as emergency care, factor replace- Learning and resource needs and capacities amongst families, rural providers and ment therapy, care of muscles and joints, assessment of rural hemophilia healthcare specialty clinic staff (Pritchard, Reimer, and care of the person and family. Pre- providers. Australian Journal of Rural Simonson & Oberle, 2006, p. 188). and post-test surveys revealed that partic- Health,14(5), 184 -8. ipants had an increased overall readiness 4. Public Health Agency of Canada Rural healthcare providers identified these to provide rural hemophilia care regard- (2003). Rural Health. five key elements as requirements for safe ing all 5 key elements of the C-STOP and effective rural hemophilia care. model. 5. Taylor G. (1999). The emerging needs of Comments by study participants persons living with haemophilia and revealed their direct insights (Pritchard, Now what? HIV/AIDS in British Columbia. 2004, p. 58-61): Haemophilia, 5(1), 1-8. 1. Communication - Rural providers… As discussed with CHS program planners, 6. University of British Columbia, The “need to know what the family study findings and C-STOP model may Institute of Health Promotion Research knows… need to be as informed… support the implementation of the (2001). Psychosocial needs assessment of when we don’t know these things it FactorFirst program. I look forward to persons with hemophilia and von causes discomfort and difficulty… continuing my work with the CHS on Willebrand’s disease (with or without sometimes we use a different solution their commendable initiative. Also, I will hepatitis C). Montreal: The Canadian than at the specialty clinic but we have be presenting this study in May 2007 at Hemophilia Society. 18 HEMOPHILIA TODAY the blood factor SPRING 2007

THE CHS supports promotion of inhibitor study BLOOD ACTOR An article in the Fall 2006 issue of Hemophilia Today described the Hemophilia F Inhibitor Genetics Study. David Page, CHS Director of Programs and Public Affairs In April 2007 the CHS Blood Safety and Supply Committee Co-chairs, Michael King and Bill Mindell, wrote to Dr. Manuel Carcao, President of the Association of Hemophilia Clinic Directors of Canada, to encourage maximum participation In the news in the study. That letter is re-printed below.

CSL Behring and Bayer extend agreement on Helixate FS through 2017

KING OF PRUSSIA, Pa., February 1, 2007 – CSL Behring (formerly ZLB Behring) has announced an agreement with Bayer HealthCare to continue the world-wide supply and distribution of Helixate®FS for the treatment of hemo- philia A. Helixate FS is identical to Kogenate®FS except for packaging and dis- tribution. Both are manufactured by Bayer, and both are approved for use by Health Canada. Under the contract exten- sion, Bayer will continue to supply Helixate FS to CSL Behring through 2017. More than six billion units of Kogenate FS and Helixate FS have been infused over the last nearly two decades.

Wyeth latest company to initiate research into longer-lasting factor concentrates

MADISON, N.J., February 5, 2007 – Wyeth Pharmaceuticals has become the latest manufacturer of clotting factor con- centrates to announce research into longer-lasting products. Over the last two years, both Bayer and Baxter have initiat- ed research into products with a goal of reducing the frequency of prophylactic or on-demand therapy for hemophilia. Wyeth and Nautilus Biotech in Evry, France, have announced the signing of a research collaboration and license agree- ment. The goal is to develop novel recom- binant factor IX proteins with an extend- ed half-life for the treatment of hemo- philia B. The technology makes minimal and specific changes to amino acid sequences in order to slow the breakdown of the protein in the body. HEMOPHILIA TODAY the blood factor SPRING 2007 19

Bloodbot installed in BBPSP lab vCJD risk from plasma-derived factor VIII EDMONTON, February 15, 2007 – “is extremely low, but may not be zero” In February, a unique robotic device was installed in the Blood Borne Pathogens David Page, Surveillance Project (BBPSP) laboratory at the University of Alberta. Researchers are CHS Director of Programs and Public Affairs using the bloodbot to increase the number of blood samples from people with bleed- n December 2006, the U.S. Food and Drug Administration (FDA) released its risk ing disorders that can be stored. assessment for transmission of variant Creutzfeldt-Jakob disease (vCJD) by plasma- The BBPSP consists of a secure collec- Iderived factor VIII products. Results of the risk assessment model suggest that the tion of blood samples collected through risk of vCJD infection from plasma-derived FVIII currently manufactured from Canadian comprehensive bleeding disor- American plasma… “is extremely low, but may not be zero.” der clinics, with a link to clinical data from The FDA risk assessment finds that the risk for users of U.S. plasma-derived concen- the Canadian Hemophilia Assessment and trates is between 1 in 105,000 and 1 in 9,400,000 per patient per year. Resource Management System (CHARMS) The FDA documents note that donor deferral criteria in place since 1999 have database. The collection or archive of reduced the risk of blood donation by people exposed to vCJD through ingestion of blood samples is made available to labora- infected beef. As well, manufacturing processes for human plasma-derived FVIII prod- tories looking for known and emerging ucts likely reduce the quantity of vCJD agent, if present, but the level of reduction blood-borne pathogens, as well as labs through manufacturing steps is not precisely known. that look for the mutation causing the The risk analysis concerns only plasma-derived factor VIII products manufactured bleeding disorder, and other genetic from American plasma since the introduction of donor deferral criteria for visitors to changes that affect the bleeding tendency. and residents of the U.K. and France. It does not attempt to calculate risk from prod- The collection undergoes regular audits and oversight to assure that it functions in ucts manufactured previously. a transparent and responsible manner. See There has been no known case of transmission of vCJD the Summer 2005 issue of Hemophilia by plasma products anywhere in the world. This includes in “There are a Today (Vol 40, No 2) for more informa- the U.K. where hundreds of thousands of bovines were number of uncer- tion on the project. stricken with Mad Cow Disease in the 1980s and more than tainties in the “We’re very pleased with how the 150 people have died from its human form, vCJD. Four robot is working,” said Dr. Bruce Ritchie, cases of vCJD, however, have been linked to blood transfu- risk assessment professor in the University of Alberta sion in the U.K. These people contracted the disease after models and it is Division of Hematology and the director transfusion of red blood cells from donors who were later important to con- of the BBPSP. “Where we could only diagnosed with vCJD. tinue to follow process about 25 samples a day, with the In a statement issued to the FDA, World Federation of developments robot we’re now able to do more than 50 Hemophilia President, Mark Skinner, noted, “There are a samples, and we hope to increase this to number of uncertainties in the risk assessment models and closely as more more than 100 in the near future. One of it is important to continue to follow developments closely as is learned about the main things this machine does is more is learned about issues such as prevalence of vCJD, issues such as eliminate the possibility of human error clearance of prions, or other factors.” in some minute, repetitive work.” prevalence of He went on to say, “The WFH continues to view both vCJD, clearance The robot does the tedious work of sepa- recombinant and plasma-derived products as important rating the components of the blood samples, treatment options for the global bleeding disorders commu- of prions, or registering them according to a barcode nity. The potential remains for adverse events including other factors.” reading, and then preparing them to be inhibitor development or unknown pathogen risk, thus frozen until researchers can analyze them. continuous learning and communication are required.” Dr. Ritchie hopes the increased produc- Canadians with hemophilia A use recombinant factor VIII. These products are not tion will allow the BBPSP program to expand its scope and accept blood samples considered to be at risk. A very small number of Canadians, however, have used plas- from more people in Canada who require ma-derived factor VIII over the last several years. These products were all manufac- frequent blood transfusions, such as tha- tured from American plasma. In addition, those people with von Willebrand disease lassemia and sickle cell anemia patients. who use clotting factor concentrates with factor VIII and von Willebrand factor use The BBPSP laboratory is sponsored by products manufactured from American plasma. The risks cited by the FDA apply to the Public Health Agency of Canada and these products. the Association of Hemophilia Clinic No risk assessment has been completed for plasma-derived factor IX; however, Directors of Canada. Researchers at the because infectious vCJD prions are concentrated in cryoprecipitate and not in the frac- University of Alberta teamed up with tion of plasma used to manufacture factor IX, the risk is thought to be lower. Baxter to commission a company in For more information, including the complete FDA risk analysis, see the CHS Web California to create the robot. site (www.hemophilia.ca/en/4.2). 20 HEMOPHILIA TODAY the blood factor SPRING 2007

Factor VIII and IX concentrates licensed/distributed in Canada

The products available to treat hemophilia A, hemophilia B, and von Willebrand disease in Canada are in constant evolution. New ones are introduced; older ones are replaced. Hemophilia Today has produced this chart to provide some basic information on the recombinant and plasma-derived factor concentrates currently licensed and/or distributed in Canada by Canadian Blood Services and Héma-Québec. For more complete information, see the product monographs that accompany the products. Most of these monographs are available via the Internet.

In the next issue of Hemophilia Today, we will publish a summary of the factor concentrates available to treat some of the rare factor deficiencies.

Recombinant factor VIII

Product Generation* Albumin Albumin Viral Vial size Storage Availability Comments in cell as stabilizer inactivation culture

Recombinate 1 Yes Yes None 250, 500, 2-8°C, Licensed, Full-length Manufactured (bovine 1000 IUs room currently being factor VIII and distributed albumin) temperature removed from molecule, by Baxter for up to Canadian no von 6 months market Willebrand factor, human albumin added as stabilizer

Kogenate FS 2 Yes No Solvent 250, 500, 2-8°C, Licensed, Full-length Manufactured (human (stabilized detergent: 1000 IUs room distributed in factor VIII and distributed albumin) with sucrose) TNBP/ temperature all provinces molecule, by Bayer Polysorbate for up to no von 80 3 months Willebrand factor

Helixate FS 2 Yes No Solvent 250, 500,2-8°C, Licensed, Identical to Manufactured (human (stabilized detergent: 1000 IUs room not currently Kogenate FS, by Bayer albumin) with sucrose) TNBP/ temperature distributed Full-length Distributed by Polysorbate for up to in Canada factor VIII CSL Behring 80 3 months molecule, no von Willebrand factor

ReFacto 2 Yes No Solvent 250, 500, 2-8°C, Licensed, B-domain Manufactured (human (stabilized detergent: 1000, room not currently deleted and distributed albumin) with sucrose) TNBP/ 2000 IUs temperature distributed factor VIII by Wyeth Triton X 100 for up to in Canada molecule, 3 months no von Willebrand factor

Advate 3 No No Solvent 250, 500, 2-8°C, Licensed, Full-length Manufactured (stabilized detergent: 1000, 1500, room distributed in factor VIII and distributed with sucrose) Polysorbate 2000 IUs temperature all provinces molecule, by Baxter 80 for up to no von 6 months Willebrand factor

* Generation 1: Human or animal albumin as both nutrient in cell culture and as stabilizer in final bottle. Generation 2: Human albumin as nutrient in cell culture but not as stabilizer in final bottle. Generation 3: No human or animal albumin either as nutrient in cell culture or as stabilizer in final bottle. HEMOPHILIA TODAY the blood factor SPRING 2007 21

Plasma-derived factor VIII with/without von Willebrand factor

Product Plasma source Fractionation Viral inactivation Vial size Storage Availability Comments

Hemofil M USA: volunteer, Monoclonal TNBP/Triton 250, 500, 2-8°C, Licensed, Albumin Manufactured remunerated antibody X 100 1000 IUs room provided on added as by Baxter plasmapheresis affinity and ion temperature a case- stabilizer, donors exchange for up to by-case basis no von chromatography 6 months Willebrand factor

Humate P USA: volunteer, Multiple Pasteurization - 250 IUs of FVIII 2-8°C, Licensed, Albumin Manufactured remunerated precipitation at 60°C, (600 IUs of room distributed in added as by CSL plasmapheresis 10 hours Ristocetin co-factor) temperature all provinces stabilizer, Behring donors - 500 IUs of FVIII for up to contains von (1200 IUs of 6 months Willebrand Ristocetin co-factor) factor - 1000 IUs of FVIII (2400 IUs of Ristocetin co-factor)

Wilate USA: volunteer, Size exclusion Solvent - 450 IUs of FVIII 2-8°C Licensed, Albumin Manufactured remunerated high pressure detergent (400 IUs of VWF) not currently added as by plasmapheresis liquid & dry heat - 900 IUs of FVIII available in stabilizer, Octapharma donors chromatography (100°C, 120 (800 IUs of VWF) Canada contains von minutes) Willebrand factor

Recombinant factor IX

Product Generation* Albumin Albumin Viral Vial size Storage Availability Comments in cell culture as stabilizer inactivation

Benefix 3 No No Nano-filtration & 250, 500, 2-8°C, Licensed, Manufactured (stabilized solvent detergent 1000 IUs room distributed and distributed with sucrose) (Polysorbate 80) temperature in all by Wyeth for up to provinces 6 months

* Generation 1: Human or animal albumin as both nutrient in cell culture and as stabilizer in final bottle. Generation 2: Human albumin as nutrient in cell culture but not as stabilizer in final bottle. Generation 3: No human or animal albumin either as nutrient in cell culture or as stabilizer in final bottle.

Plasma-derived factor IX

Product Plasma source Fractionation Viral inactivation Vial size Storage Availability Comments

Immunine USA: volunteer, Ion exchange Polysorbate 80; 250, 500, 2-8°C, Licensed, Albumin Manufactured remunerated and hydrophobic Vapour heat, 1000 IUs room distributed in added as by Baxter plasmapheresis interaction 60°C, 10 hr @ temperature all provinces stabilizer donors chromatography 190 mbar; for up to then 80°C, 3 months 1 hr @ 375 mbar

Mononine USA: volunteer, Immunoaffinity Sodium 500, 1000 2-8°C, Licensed, Albumin Manufactured remunerated chromatography thiocyanate & IUs room currently added as by CSLBehring plasmapheresis ultrafiltration temperature available stabilizer donors for up to in Canada 1 month on a case- by-case basis 22 HEMOPHILIA TODAY the female factor SPRING 2007

(including the Canadian Hemophilia normal factor VIII activities. Others have

THE female Society, CIHR, CDHA, IWK, Killam), our turned off mostly normal Xs and have FACTOR research team in Dr. Wenda Greer’s lab low factor VIII activities and symptoms of (Pathology Department, CDHA and hemophilia. So, the hemophilia symptoms Patricia Stewart Dalhousie University, Halifax, Nova in the carriers in Olivia’s family can be Scotia) has been able to answer a few explained by unbalanced, or skewed, X- Symptomatic carriers: questions. Females sometimes express X- chromosome inactivation. That’s one linked recessive conditions (like hemophilia question answered. However, our findings Can women and girls really A, hemophilia B, Duchenne Muscular pose a new problem to solve: why do express hemophilia? Dystrophy, colour-blindness) because of Olivia and many of her female relatives unbalanced X-chromosome inactivation. have skewed X-chromosome inactivation? Nisa Renault BSc, PhD candidate, The “choice” of which X gets turned off in It is possible that Olivia has skewed X- Dalhousie University each cell has typically been viewed as a chromosome inactivation by chance. Halifax, Nova Scotia random process, like flipping a coin. However, it is unlikely that many women Heads – the X-chromosome inherited in the same small family would all have In the Spring 2006 issue of Hemophilia from your father gets turned off, tails – the skewed X-chromosome inactivation by Today, JoAnn told the story of her daugh- X from your mother gets turned off. This chance (a probability of less than 5% in ter, Olivia, who has severe hemophilia A. process is thought to occur near the 8-cell our case). We are exploring the possibility Her symptoms were unexpected because, stage of development. So, each embryo that the choice of which X-chromosome typically, only males express hemophilia flips a coin 8 times to determine A. Most females who inherit one altered which of the two X-chromo- hemophilia A gene are asymptomatic car- somes in each cell will be inacti- riers. This is the usual pattern because of vated. Most get a balanced ratio the genetics of hemophilia A. of 4 heads to 4 tails, but some Hemophilia A is caused by insufficient get 3 heads to 5 tails or even 8 activity of the factor VIII protein that is heads to 0 tails. An unbalanced needed for blood clotting. Changes in the ratio of heads to tails (or father’s structure of the factor VIII protein can X off to mother’s X off) is only a affect its activity and lead to hemophilia concern if one of those Xs has Olivia Craig A. The DNA encoding the factor VIII an altered gene. gene is found on the X-chromosome. We were able to show that, in hemo- to inactivate may not be random, but Females have 2 X-chromosomes, and philia carriers in Olivia’s family, the more rather is genetically influenced. males have one X- and one Y- chromo- cells that contained a functional copy of We have several lines of evidence sup- some in each cell. The amount of genetic the factor VIII gene, the higher the factor porting this idea. First, the same skewed material on one X-chromosome is just the VIII blood clotting activity. The fewer the X-chromosome inactivation was observed right amount needed for each cell, so each cells activating the normal factor VIII in different types of cells from each family cell with 2 X-chromosomes needs to inac- gene, the lower the factor VIII activity and member. Also, high resolution studies tivate one of them. Genes on the inacti- the more severe the hemophilia symp- revealed no X-chromosome structural vated chromosome are not available for toms. For example, in Olivia’s sister, half abnormalities that can sometimes cause use. X-chromosome inactivation is a nor- of her cells express the normal factor VIII X-chromosome inactivation skewing. mal and necessary process of develop- protein, and half are expressing the Lastly, our review of published studies ment. Genes on the X-chromosome are altered factor VIII protein. Consequently, indicates that X-chromosome inactivation called X-linked genes, and conditions she has half the normal factor VIII activi- skewing may be more common in the resulting from alterations in those genes ty. Fortunately, half the normal factor VIII general population than can be explained are called X-linked conditions. For males activity is sufficient to protect her from by random models. We are currently in to express X-linked conditions like hemo- clinical symptoms of hemophilia. In pursuit of a possible gene influencing the philia, they only need to inherit one Olivia, however, most of her cells are choice of which X-chromosome to inacti- altered copy of a gene. Because females expressing the altered factor VIII gene vate. Such a gene may be influencing X- have two copies of X-linked genes, they (92%). To continue the analogy, 7 out of chromosome inactivation in Olivia’s fami- typically need to inherit one altered copy 8 coins came up tails! Consequently, she ly. If we can find it, we will learn a great from each parent. Because Olivia only has very low factor VIII activity (<2% deal about X-chromosome inactivation, inherited one altered factor VIII gene, she normal) and severe symptoms. Many and could potentially use this information should be an asymptomatic carrier. So women in Olivia’s family have unbalanced to predict the severity of many X-linked what happened? That’s where we come in. ratios (at least 6 of 13 have ratios exceed- conditions, like hemophilia, in female car- Thanks to the enthusiastic participa- ing 70 to 30). Some of these family mem- riers. Eventually, this information could tion of Olivia and her extended family, bers have turned off mostly Xs with lead to ways of relieving or preventing and funding from several institutions altered factor VIII protein and have nearly symptoms in carriers. HEMOPHILIA TODAY the global perspective SPRING 2007 23

On Friday, the day after our arrival, there was a medical sympo- sium entitled: Tunisia-Quebec Hemophilia Day. Under Honorary Patron Her Excellency Mrs. Leila Ben

GLOBALTHE Ali, wife of the President of the PERSPECTIVE Republic of Tunisia, the symposium was officially opened by Mrs. Naziha Cheikh, Secretary of State in the Quebec Chapter - Tunisia Tunisian Ministry of Public Health, and Mr. Bruno Picard, Canadian twinning: January 2007 visit Ambassador to Tunisia. The presence of these two dignitaries, and above An example of synovitis, caused by frequent François Laroche, President of the CHSQ all the fact that the wife of President hemarthroses, in the knee of a young Tunisian with hemophilia. Ben Ali had agreed to act as he partnership between the Honorary Patron for the event, drew dentists, and representatives of the blood Quebec Chapter (CHSQ) and the a large number of reporters. product supplier (Pasteur Institute), TTunisian Hemophilia Association The main goals of the symposium were mainly from the four major cities in (ATH) produced some memorable as follows: Tunisia (Tunis, Sousse, Sfax, and Gafsa), moments from January 18 to 25 during • Raise public awareness about hemophilia in addition to representatives of the two the second CHSQ visit to Tunisia. The through the media associations. At the end of the day, the Quebec delegation was made up of the • Increase the number of cases of hemo- participants said they were delighted with following members: Dr. Georges-Étienne philia recorded by raising health profes- the wealth of information and quality of Rivard, Director of the Hemophilia sionals’ awareness about hemophilia the discussions. From the opening talks to Treatment Centre (HTC) at Sainte-Justine • Improve care and treatment provided to the closing reception, everything went Hospital in Montreal; Nichan Zourikian, hemophiliacs through knowledge very smoothly, all to the credit of the physiotherapist attached to the HTC at acquired from the talks, especially those Organizing Committee. The mere fact of Sainte-Justine Hospital; David Page, Vice given by hematologists securing the patronage of Mrs. Ben Ali President, Communications and Public • Improve care provided to hemophiliacs was a great accomplishment by the ATH. Policy with the World Federation of through the workshop for physiothera- Even though the following day, Hemophilia (WFH); Patricia Stewart, pists. Saturday, was the Chair of the International Projects A total of sixty physicians (hematolo- Muslim New Year At the end of Committee at the CHSQ; and François gists, orthopedists, general practitioners, and a national Laroche, then Vice President of the etc.) took part, along with laboratory holiday, we were the day, the CHSQ. technicians, biologists, physiotherapists, still received at participants Aziza Othmana said they were Hospital by the delighted with Head of the the wealth of Clinicial and Biological information Hematology and quality Department, of the Dr. Balkis Meddeb. discussions. We were able to visit the clinical and biological haematology facilities, the new haematol- ogy treatment centre (in preparation), and meet with the hospital director. David Page gave a talk on the various WFH programs, such as the Global Alliance for Progress. In the afternoon, François Laroche gave a talk on strategic considerations in an organization, and Patricia Stewart followed with another The opening of the Tunisia-Quebec Hemophilia Day, with representatives of the Tunisian Hemophilia talk on basic principles of fundraising. Association, the Tunisian Ministry of Public Health, the Canadian Hemophilia Society (Quebec Chapter) and the Canadian Embassy. continued on p. 24 24 HEMOPHILIA TODAY the global perspective SPRING 2007

continued from p. 23 Othmana Hospital will make a On Monday, January 22, we had two tremendous contribution activities on the program for which we had toward this goal. The involve- to divide the delegation in two. François ment of a number of hematol- and David accompanied several doctors ogists together with volunteers and member of the ATH to meet the who are dedicated to the cause Minister of Public Health, Dr. Rihad gives the organization a lot of Kechrid. After explaining the situation of credibility with government people with hemo- officials. In the ATH there are philia in Tunisia people with remarkable lobby- Our general and discussing with ing skills, and the organization impression of each of the mem- is gradually discovering the this visit is bers of the delega- almost limitless possibilities extremely posi- tion, the Minister Three members of the Tunisian Hemophilia Association brave the this can open up. tive. We once Montreal winter cold in March 2007. Left to right: Professor made a commit- Raouf Hafsia, Vice-president; Islem Nafti, President; and Taoufik At present, 270 people with again noted a ment that Raissi, Secretary-general. hemophilia have been regis- strong will to “Tunisians with tered in Tunisia, out of a pop- improve living hemophilia should sion in the morning for Tunisian physiother- ulation of 10 million (therefore out of a conditions for have access to the apists, led by Nichan Zourikian. The after- potential total of 1000 people with hemo- Tunisians with best treatment avail- noon was devoted to case studies with young philia). Of these 270 people with hemo- hemophilia on able and adequate people with hemophilia who had come to philia, 130 (or 48%) have the severe form the part of both comprehensive care consult the experts with their parents. of the disease. Since our last visit in 2005, health profes- by the State.” This Our general impression of this visit is Tunisia’s consumption of factor VIII has sionals and ATH commitment was extremely positive. We once again noted a increased from 0.15 International Units volunteers. certainly one of the strong will to improve living conditions (IU) per capita of total population to a high points of our for Tunisians with hemophilia on the little more than 0.3 IU/capita. Factor VIII visit, and it is very encouraging for the future part of both health professionals and ATH concentrate has of care to those with hemophilia in the coun- volunteers. The impending opening of an tripled from 0.1 try. Physiotherapy was the other item on the official hemophilia treatment centre in IU/capita to 0.3 Since our last day’s agenda, with a theoretical training ses- the Hematology Department of Aziza IU/ capita (largely visit in 2005, due to a better ten- Tunisia’s con- dering process sumption of resulting in lower factor VIII has prices), whereas the standard for increased adequate treatment from 0.15 of hemophilia, International according to the Units (IU) per WFH, is 1.0 capita of total IU/capita. Consumption of population to factor VIII in a little more Canada is 5.0 than 0.3 IU/capita. IU/capita. The CHSQ is very enthusiastic about the care that can be provided to Tunisians with hemo- philia in the future. A very promising soli- darity has emerged between the two organizations. Tunisians have a reputation The Quebec, French and Tunisian delegations during the visit to the Aziza Othmana Hospital in Tunis. (Standing): Dr. Georges-Étienne Rivard (hematologist, Montreal), Dr. Sondes Mseddi (hematologist, Sfax), as a warm and welcoming people, a repu- Dr. Balkis Meddeb (hematologist, Tunis), Mr. François Laroche (President of CHSQ), Mr. David Page (Vice- tation they well deserve. The hospitality president of WFH), Dr. Viviane Guérin (hematologist, Bordeaux), M. Hédi Moulahi (Directeur-general of the hospital), Dr. Aïcha Hafsia (Honorary President of ATH), Dr. Ségolène Clayssens (hematologist, we received throughout our long stay was Toulouse), Mr. Taoufik Raissi (Secretary-general of ATH), Mr. Hamma Amdouni (member of the Board of superb, and the CHSQ hopes to be able to ATH) et Ms. Nejia Grichi (member of the Board of ATH). (Kneeling): Dr. Emna Gouider-Belhadjali (hema- tologist, Tunis), Ms. Kaouther Zahra (physiotherapist, Tunis), Ms. Patricia Stewart (Chair of the extend its partnership with the ATH for International Projects Committee of CHSQ) et Mr. Habib Chouikha (member of the Board of ATH). several more years.