March 2003 Lifeline Cooley’s Anemia Foundation L e a d i n g t h e F i g h t A g a i n s t T h a l a s s e m i a CDC & CAF Launch Thalassemia Program by Jeanne Devine and Craig Butler Responding to patient concerns comprehensive thalassemia treatment The CDC has also provided significant centers to identify currently underserved funding for a related initiative for Prevention about the possible transmission people, promote blood safety, encourage of Complications of Thalassemia. This of illnesses through blood healthy behaviors, and provide information project involves a cooperative agreement and training to medical professionals. between the CDC and comprehensive transfusions, the Cooley’s thalassemia treatment centers across the Anemia Foundation (CAF) U.S. aimed at preventing complications for individuals with thalassemia. CAF will work successfully appealed to the with these thalassemia treatment centers to U.S. Congress to increase achieve common goals of the two projects. access to prevention services Key to the success of both programs is the gathering of complete, accurate information for people with thalassemia. from the thalassemia population in the U.S. For that reason, Ms. Sawh requests that all Congress in turn awarded the Centers for individuals with thalassemia update their Disease Control and Prevention (CDC) contact information with CAF. This will $2.2 million to initiate a new five-year enable CAF to keep them informed about program to ensure that people with developments with the CDC program. thalassemia are identified and have access to appropriate information, treatment and Another goal of the program, providing Under the terms of the agreement, the prevention services. information and training to medical CDC will provide first-year funding to CAF professionals, has long been a primary CAF has entered into a cooperative in the amount of $172,650. CAF has hired concern for CAF. Most medical agreement with the CDC to help develop an Outreach Coordinator to help implement professionals do not possess an in-depth and implement a major component of this the program, under the supervision of knowledge about the various complications project, the Thalassemia Prevention National Executive Director Jayne Restivo of thalassemia and/or the most current Education and Outreach Program. The and Patient Services Director Radhika treatments. Supplying CAF with updated Foundation will work with the CDC and Sawh (see story, page 7). (article cont’d page 3) contents
Legislative News 3 • Interview with Dr. Claude Lenfant
Philanthropy News 4 • CAF Set to Benefit from Wellspent.Org • CAF Receives Support from New York • Gala 2003
• Meeting in Chicago • Oral Chelator • Blood Donations Needed • In Memoriam Patient News 6 • Outreach Coordinator • Patient Services: There for You • TAG Conference • Iron Content in Heart • Thalassemia Clinical Research Network • ASH Convention Medical News 8 • CME Seminar • Blood Safety • Stefano Rivella • Non Invasive Liver Measurement
International News 12 • TIF Holds First Associations Meeting • Reactions to TIF Associations Workshop • All You Need is the Wish
• Chapter Updates • Chapters Help Finance Medical Technology • Chapter Contact Numbers Chapter News 14 • New Chapter Development Manager • Tribute to the Founders of CAF President’s Message
I am amazed by the strides we have made in And these are just the high points! the past year or two, though I should probably There are countless other initiatives underway have anticipated it based on our history. We that will improve the quality of life for our are, after all, “The Mouse That Roared.” patients and their families. Our executive Thanks to the diligent work of dozens of director, Jayne Restivo, has put together a people for dozens of years, we are recognized staff that understands this completely and as a very influential group in Washington D.C., uses this as a mantra to guide them through and the results have been phenomenal. Frank Somma their day-to-day activities. Their caring attitude Currently we are looking forward to clinical and motivation to help our patients is apparent trials on deferiprone and Desferal in our in everything they do. Please rely on them to clinical network (a 12.5 million dollar help you manage your care; get you the latest investment by the NIH). We have a blood medical information or insurance help; locate a safety program through the CDC (a two hard-to-find needle; get you a replacement million dollar annual investment by congress). pump; interface with the billing department of The Maternal and Child Health Bureau has your hospital, etc. The list goes on and on. pledged half a million annually in order to Much of what I am mentioning here is on our continue helping financially challenged patients website, www.cooleysanemia.org Please visit handle pregnancies in a healthy and informed it often to stay abreast of the latest goings on manner. at YOUR foundation. We have had over 100 patients’ livers May God bless you all. evaluated by SQUID, and are currently setting up the same availability to measure heart iron Frank Somma with MRI (see Medical News).
National Executive Director’s Message
The gift of blood is a gift of life. A single pint even more severe shortages grows more and of donated blood can help up to four people. more likely. As CAF’s National Executive Director, I spend Even without these factors, the need for a great deal of time talking to people about blood grows by 11% each year, while blood, because of its importance to the donations increase by only 8%. And thalassemia community. I constantly nationwide, less than 5% of those eligible to encourage people I meet to give blood, not donate blood do so. just because our patients need it but because Jayne Restivo For a person with thalassemia, who may need everyone needs it. a transfusion as often as every two weeks, Our nation’s supply of available blood is never those numbers are frightening. as large as we would like, but it tends to be If you are one of the 95% who has not particularly low during the winter months. donated blood this year, I urge you to call This has been especially true this winter, 1-888-USBLOOD or 1-800-GIVELIFE today. when we have seen much blood quarantined For people with thalassemia, it truly is the gift due to possible infection, a drop in donations of life. due to harsh weather and the removal of imported blood from Europe. With the Jayne Restivo prospect of war looming, the possibility of
statement of purpose
The Cooley’s Anemia Foundation, incorporated in 1954, is the only national, EXECUTIVE COMMITTEE nonprofit health organization dedicated to serving patients afflicted with various Frank Somma President forms of thalassemia, most notably the major form of this genetic blood disease, Cammie Brandofino 1st Vice President Cooley’s anemia. Terri DiFilippo Treasurer Ralph Colasanti Secretary the foundation’s mission Nunzio Cazzetta Joseph Giammalvo Amy Celento-Stamateris Diane Kamble • Advancing the treatment and cure for this fatal blood disease. Peter Chieco Richard Mancino • Enhancing the quality of life of patients. Dr. Alan Cohen, MD Edward Martella • Educating the medical profession, thalassemia trait carriers and the public Robert Ficarra Concetta Paradiso Diane Germann about thalassemia/Cooley’s anemia.
Jayne Restivo National Executive Director CAF : 129-09 26th Avenue, Suite 203, Flushing, NY 11354 Craig Butler Lifeline Editor (800) 522-7222 (718) 321-CURE (2873) (718) 321-3340 FAX www.cooleysanemia.org l e g i s l a t i v e n e w s CDC (cont’d from front page) contact information will enable us to connect each primary care physician with a comprehensive thalassemia treatment center. This will be an excellent resource Interview with Dr. Claude Lenfant for physicians that will allow them to communicate with many different specialists who are familiar with the issues faced by people with thalassemia, thereby Dr. Claude Lenfant was appointed Director ensuring that their patients receive the of the National Heart, Lung and Blood most effective care. Institute (NHLBI), which provides funding The CDC program will also encourage for the Thalassemia Clinical Research healthy behaviors that reduce or prevent Network, on July 6, 1982. Born in Paris, complications of thalassemia and promote Dr. Lenfant received his B.S. degree from blood safety. The first blood safety issue the University of Rennes and his M.D. from that will be addressed by the CDC will be the University of Paris. He was a transmission of the West Nile Virus. This postdoctoral fellow at both the University issue is of particular interest to the of Buffalo and Columbia University, and a thalassemia community due to the large member of the faculty of the University of number of transfusions involved in Washington, Seattle. His association with treatment of the disorder. the NHLBI (then known as the NHLI) began in 1970. Dr. Lenfant is the recipient of numerous awards, including the Surgeon General’s Exemplary Award and the Dr Claude Lenfant American Society of Hematology’s name of the game! I think one of the most Outstanding Service Award. He graciously striking changes that I have witnessed is took time out of his busy schedule to our increased ability to make the benefits of answer the following questions for CAF. science available to the public, patients and their families, and health professionals— You have been associated with a wide though I should note that this remains our range of organizations and projects. Are greatest challenge. The other change I there particular projects or affiliations that have seen is the increasing complexity of you would like to mention? scientific research, so that investigations I have taken special pleasure in projects require a mix of different areas of expertise that strive to improve the care of patients, and the ability to coordinate that expertise both here and abroad. For example, as in an effective team effort. Sally Crudder president of the World Hypertension What are some of the accomplishments of League I work with over 80 international Sally Crudder, the CDC’s Project Officer for the NHLBI during your directorship which societies on efforts to advance global the program, states that “the CDC you feel are of particular significance? hypertension prevention and control. I partnership with CAF will provide an We just celebrated the 30th anniversary of opportunity to improve the health status of have also found it very rewarding to be involved in the implementation of the the Institute’s mandate for research in patients with thalassemia by providing diseases of the blood and blood resources. access to specialized healthcare services. Global Initiative for Asthma, a worldwide network of individuals and organizations Over this time, it has been gratifying to see This initiative is critical to preventing the life expectancy of persons with sickle complications of thalassemia through that disseminates information about the care of patients with asthma, and the US cell disease and thalassemia more than assessment, surveillance, outreach, double and our blood supply become the education, consultation and management.” COPD coalition of professional, government, academic, and patient safest in the world. Moreover, many forms CAF Executive Director Jayne Restivo says organizations dedicated to increasing of cardiovascular and lung diseases now that “CAF is honored and excited to work awareness of chronic obstructive have treatments where before there were with the CDC in developing the Thalassemia pulmonary disease and improving its none, and our pipeline of potential therapies Prevention Education and Outreach prevention, diagnosis, and care. continues to grow. Also, through the Program. We are enormously pleased that efforts of the Institute and its many partner the federal government through the CDC You have been associated with NHLBI organizations, we have initiated and recognizes the needs of people with since 1970, and have been Director since fostered national education efforts that thalassemia and is responding in a positive 1982. How have you seen the Institute reach out not only to individual patients, change in t and forceful manner.” hat time? their families, and health professionals, but Science is an ever-evolving quest for also to the communities in which they live To supply updated information, contact improved knowledge, so change is the and work. (article cont’d page 4) Jeanne Devine at (800) 522-7222, ext. 211. (cont’d from page 3) thalassemia community most identifies NHLBI, but there are other programs (such as those that deal with bone marrow CAF Receives transplants, gene therapy, blood borne illnesses, etc.) which have an impact on Support from When did you first become aware of individuals with thalassemia. Would you thalassemia? like to discuss some of the work that the New York I first learned about thalassemia in medical NHLBI is doing in such relevant areas? school, and this disorder has taught us a At the NHLBI we have solicited the input of great deal about hemoglobin, genetics, iron a cross-section of outside experts and overload, transfusion medicine, and chronic CAF has received a generous grant of organizations to help develop and disease in general. Over the years, I have $125,000 from the New York State implement planning efforts for many had a personal interest in hemoglobin and Department of Health for the current fiscal diseases such as thalassemia and for its variations as I was very interested in the year. This money will be used to support broader, crosscutting areas as well. This way hemoglobin carries oxygen from the CAF’s general operating expenses. has led to identification of new research lungs and delivers it to the tissues of the directions--including areas such as The grant is a result of actions taken within body. Of course, having the opportunity to genetics, genomics, proteomics, and stem the state Senate and Assembly as part of meet and know many patients and their cell biology and transplantation--that are of their budget process. CAF has been the families has only intensified my interest. great potential benefit to the thalassemia recipient of New York state funding for many The Thalassemia Clinical Research community. Recent accomplishments have years and is thrilled that so many of New Network (TCRN) is of enormous included crafting a strategic plan for a gene- York’s elected officials are willing to provide importance to thalassemia patients and the based cure of the hemoglobinopathies; assistance in our fight against thalassemia. physicians who treat them. What role did developing a pipeline of potential therapies Chief among these elected officials are the you play in the establishment of the TCRN? through grants, contracts, and small How do you see the role of NHLBI in the business grants; and establishing constant ongoing activities of the TCRN? communication with other groups that can We at the NHLBI pioneered the clinical bring expertise and resources to the efforts research network model, which provides a to treat and ultimately cure this disease. rapid and cost-effective way of answering As you know, the thalassemia community is important questions about patient therapy. relatively small but passionate. Do you The TCRN gives the community of have any advice for this community? investigators who work with thalassemia I have always been impressed with the patients an infrastructure and opportunity thalassemia community and the to evaluate therapies for the various forms perseverance and dedication that it brings of the disease. The NHLBI’s role is to to its work. I believe that the community ensure that the network functions should continue to build partnerships with efficiently and answers important clinical groups that represent and speak for questions in a timely manner. RoAnn Destito patients and their families or that can work What do you see as the TCRN’s goal or with the community to achieve common members of the New York Conference of mission? goals. “Leveraging” resources is a Italian-American State Legislators (NYCIASL), which includes Assemblywoman There is an urgent need to evaluate new powerful tool for maximizing a group’s RoAnn Destito, an ardent CAF supporter. and existing therapeutic approaches for potential impact. Although I cannot determine what topics the community people who suffer from thalassemia, and to “As the Vice-President of the NYCIASL, I should address, I encourage it to continue disseminate the findings to health-care am very concerned about thalassemia and to establish and foster an ongoing dialogue professionals, patients, and families. The the devastating effects this terrible affliction with others such as the public interest mission of the TCRN is to accelerate has upon those young people and their organizations whose representatives meet clinical research and thereby meet this families that have this genetic disease,” annually with the NHLBI (see our website pressing need. Assemblywoman Destito says. “We in the at: http://www.nhlbi.nih.gov/ New York State Legislature are proud of our What do you hope the TCRN can public/pinterest.htm). record of support, yet we want to see the accomplish in both the short term and the day come when Cooley’s anemia is a distant long term? Is there anything else you’d like our patients to know about NHLBI? memory of a bygone era. All of our I hope the network can establish and I would like the patients to know that the collective efforts, especially those of CAF, complete a number of clinical trials that will are dedicated to this important goal.” enhance the lives of people with NHLBI exists to serve patients and public thalassemia and provide improved health. Without your support, wisdom, and CAF thanks Assemblywoman Destito, therapeutic options for them and for those confidence in the scientific endeavor, we Senator Serphin Maltese and all the who care for them. could not continue. I have enjoyed working members of NYCIASL and the state with the thalassemia community and look Legislature for their continued support. The TCRN is the project with which the forward to continuing to do so in the future. p h i l a n t h r o p y n e w s CAF Set to Benefit from Wellspent.Org Gala 2003: A Carnevale
The Cooley's Anemia Foundation has has appeared in public service entered into an arrangement with announcements promoting awareness of Wellspent.org, an online store that lets you thalassemia and of CAF. donate to Cooley's Anemia Foundation John Sialiano recently joined the Board of simply by shopping. Wellspent.org is an CAF, after having lent his support to online superstore with thousands of tools, various CAF events. He was a guest of electronics, kitchen/housewares, honor at the 2002 CAF Gala, and home/garden, outdoor and computer participated as CAF’s special guest at the products - all sold at discount prices. 2001 and 2002 Columbus Day Parades. John has helped raise awareness of Each time you make a purchase through thalassemia through his popular morning Wellspent.org, you can choose Cooley's radio show and has also helped raise CAF’s Anemia Foundation as the non-profit cause profile with various funders. you want to support. A portion of the Gianni and Chazz Palmintieri
Gianna and Chazz Palminteri hosted the 2003 CAF Spring Gala on March 4, at New York City's famous Tavern on the Green restaurant. Celebrated actor and performer Joe Piscopo was selected as guest of money you pay is then donated to us. The honor for this festive celebration, and John donation is in your name and is even tax- Sialiano (well known to radio audiences as deductible. WKTU's "Goumba Johnny") served as Honorary Chairperson of the Gala So next time you go to buy a gift for a Committee. friend, loved one, or yourself, go to Built around the theme of "Carnevale," the Wellspent.org, click to support Cooley's 2003 Gala was an exceptionally lively Anemia Foundation and start shopping. event. The 300 people who attended were And please let others who might have an treated to a convivial cocktail hour, a interest know about this arrangement. delicious sit down dinner and a marvelous
band whose music inspired plenty of Joe Piscopo For more information, please contact Jo energetic dancing. Many guests Ann Barbella at (800) 522-7222, ext. 202. Joe Piscopo shot to fame in the early participated in both the live and silent 1980s through his appearances on auctions, bidding on tempting prizes such Saturday Night Live, where his characters as a cruise to Paris, a chance to be a walk- and celebrity impersonations (such as on in Chazz Palminteri’s upcoming film and Frank Sinatra) were widely acclaimed. He a stunning Breitling watch. has since spread his talents into motion Gala sponsors included the Order Sons of pictures, night clubs and Broadway and has Italy in America (OSIA), Generoso Pope devoted his time to a range of worthy Foundation, Novartis Oncology, Con Edison causes. and the Durst Organization. “The Cooley’s Anemia Foundation is CAF Board member Gianna Palminteri and delighted that Mr. Piscopo is willing to join National CAF Spokesperson Chazz us in the fight against thalassemia,” says For up-to-the-minute news Palminteri have a long history of supporting CAF National Executive Director Jayne about thalassemia-related CAF and the fight for thalassemia. They Restivo. “With people the calibre of Joe have hosted two previous Gala dinners, Piscopo, Gianna and Chazz Palminteri, and issues, visit our website, both of which were enormous successes. John Sialiano involved, this year’s Gala www.cooleysanemia.org A well respected actor and writer, Chazz was destined to be a huge success.” p a t i e n t n e w s
who all seemed to want to get involved and Report on TAG Meeting in Chicago support each other,” he said. He especially commended Dr. Alexis Thompson and the nurse practitioners from CCMH for “taking time out of their lives to get involved with these patients, most of whom aren’t treated at CCMH.” Ralph thanked the TAG Board for its efforts in organizing the event and extended his gratitude to everyone that attended, encouraging them to stay involved with CAF and TAG. “The energy that you On October 19, about 30 thalassemia Ralph said. “The patient base in the area is demonstrated boosted my morale,” he patients and family members from Illinois, very strong and Bruce feels that a CAF said. “I hope that you can continue to grow Ohio and Indiana, as well as staff from Chapter would benefit the patients of the a CAF Chapter into all we talked about it Chicago Children’s Memorial Hospital area, as well as expand CAF’s presence.” could be. (CCMH), gathered in Chicago to exchange The TAG Board made this outreach effort “And if there are other patients out there information and discuss the possible to the Chicago area for several reasons. who can get a group of patients and family formation of a new CAF chapter. TAG “We had heard stories from Bruce about together for a day of outreach, please feel Board member Bruce Rod led the efforts to how isolated the patients there felt,” Ralph free to contact CAF and let us know. You organize the meeting, which also included explained. “He and Todd were instrumental are not alone in this; there is a whole TAG President Ralph Colasanti and TAG in getting TAG to Chicago. Coming from foundation to support you.” Board members Cathie Jo Langon, Todd the area, Bruce could relate to what type of Becker and Diane Kamble. Since the initial meeting, CAF Chapter questions the patients had, and Todd knew Development Manager Jennifer Fleming “The primary purpose of the meeting was a few patients from the area as well. We has met twice with the patients and families to show support to patients in the area and think that Chicago is a good location to in the Chicago area and reports that the to increase awareness of TAG and CAF,” service patients from the midwest.” development of the chapter is progressing Ralph was very encouraged by the level of smoothly. If all goes well, CAF will soon be As you may know, an oral chelator enthusiasm present in the meeting. “We welcoming a new chapter into the family.
(der feriprone, or L1) has been developed had some dynamic and energetic people which is currently being used (in a specifically restricted manner) in other o parts of the world but which has not yet Blood Donations Desperately Needed beet n approved for use in the U.S. However, there is a possibility that The past few months have been some of USBLOOD) or the American Red Cross defa eriprone may be available to some the worst on record in terms of blood (800-GIVE-LIFE). U.Sl . patients under very strict donations. A combination of factors, We also encourage you to consider setting circumstances through the Treatment including brutally cold weather that
e up a blood drive in your neighborhood It IND program of the Food and Drug discourages people from venturing out to sounds complicated, but it’s actually fairly Administration (FDA). blood collection centers and the removal of easy – and would have a significant impact.
h imported blood from Europe due to new If you think that deferiprone should be Call CAF Chapter Development Manager federal restrictions, has resulted in severe considered as a treatment option for you Jennifer Fleming (800-522-7222, ext. 208) at this time, discuss this with your shortages throughout the U.S. The C to learn how to plan a blood drive. physician. If you both agree that this is possibility of a prolonged armed conflict an appropriate course of action, your threatens to worsen the situation, as some docl tor will need to contact the FDA and supplies would be diverted for use in treating wounded soldiers. In Memoriam Apotex, the manufacturer of the drug, We regretfully report the loss of Cooley’s a and request that deferiprone be made An adequate blood supply is of great anemia patients available for you under the Treatment r importance to people with thalassemia, due IND. (For more information, ask your to the frequent transfusions that form the Victoria Angelone doctor to call CAF Patient Services, core of their treatment. CAF urges all who Azeem Sherali (800) 522-7222.) O are capable of donating blood to do so. To and extend our sympathies to their friends find a blood collection site near you, please and family. call America’s Blood Centers (888- CAF Hires Outreach Coordinator Announcement of TAG Conference which thalassemia is most common. Jeanne received her undergraduate degree The Thalassemia Action Group (TAG) will from Cornell University, majoring in hold its annual Patient/Family Conference Genetics and Development and minoring in from April 4 - April 6, 2003 in Anaheim, American Indian Studies. She has a California. The theme of this year's Master’s in Genetic Counseling from Mount Conference will be "The Liver." Sinai School of Medicine. The annual Patient/Family Conference is Prior to joining CAF, Jeanne was a prenatal, one of the most important and eagerly pediatric and clinical genetic counselor at awaited of TAG's activities. "This is a Winthrop University Hospital in Mineola, wonderful chance for people with Long Island. “I left clinical work to join CAF thalassemia and their families to get because this project is on a larger scale together and exchange information and Jeanne Devine than one-on-one counseling,” Jeanne says. experiences," says TAG President Ralph CAF is pleased to announce that Jeanne “I feel I will be able to help more people Colsanti. Of special value is the Devine has joined its staff in the newly- through a nationwide education program of presentation of new scientific and medical created position of Outreach Coordinator. this scope.” information of importance to people with thalassemia. Jeanne will coordinate initiatives developed “On a more personal level,” she adds, “I under CAF’s recent cooperative agreement was interested in coming to CAF because with the Centers for Disease Control and of family history. My mother’s family is Prevention (CDC)’s Thalassemia Prevention affected by two different inherited anemias, and Education Outreach project. Among which are slightly different from beta these initiatives is a collaboration with thalassemia in hereditability and disease federally funded Thalassemia Treatment course but similar in respect to lack of Centers in the establishment of a national information in the general population and surveillance project to monitor blood safety much of the physician population. So I feel among the thalassemia community; I have a handle on what a lot of people with dissemination of information about thalassemia and their doctors go through in thalassemia-related complications; and that regard.” The Conference will be held at outreach efforts to individuals with Disneyland's Paradise Pier Hotel, (714-520- CAF is delighted to have Jeanne on its thalassemia and to communities among 5005, fax: 714-520-6079). Conference team. participants may book rooms either in that Hotel or the nearby Candy Cane Inn (800- CAF Patient Services: There for You 345-7057 or 714-774-5284, fax: 714-772- 5462). TAG has secured a rate of $129.00 per room per night at the Paradise Pier and Having problems with your pump? waiting for the new service to kick in and of $87.00 per room per night at the Candy Confused by new insurance issues? also helped him solve a problem involving the proper functioning of his pump. Cane Inn. Participants should make In need of someone to talk to you about reservations by March 5 and tell the hotel compliance issues? “I was glad that things worked out so that they are with the Thalassemia Action well,” Melissa says. “This is really what G CAF Patient Services is there for you. roup. If anyone has problems making Patient Services is about. With short term reservations with either hotel, please Here’s one example of how Patient Services problems, like obtaining needles or contact Dawn Adler at (510) 531-0940. can help you. Recently, an individual called Desferal, we can provide a quick response. Patient Services Coordinator Melissa Chin. With more complicated problems, like In addition to making hotel reservations, pa His homecare company had gone out of insurance issues, we help empower rticipants need to register for the business, and his insurance had patients by giving them the assistance and Conference itself. For complete automatically switched him to another information they need to understand and registration information, please contact Jesal Kapasi company; however, he was not pleased with take control of the situation.” at [email protected], his new company. Melissa listened to his Diane Kamble at (914) 237-0381 between The CAF Patient Services staff is anxious story, gathered the facts and then told him 9:00 a.m. and 9:00 p.m. (Eastern time) or to help. Call (800) 522-7222, ext. 205 Melissa Chin at the steps that he needed to take to get a the CAF offices at (800) (Patient Services Director Radhika Sawh) homecare company that meets his needs in 522-7222, ext. 206. Questions pertaining or ext. 206 (Melissa Chin) with your a more satisfactory manner. In addition, to matters other than registration can be questions. sent to TAG@c Melissa was able to obtain a supply of ooleysanemia.org. butterfly needles for him while he was m e d i c a l n e w s
placed on a padded table and special T2* Measures electrical leads will be placed on their chest Thalassemia to monitor their heart rate. The patient will Iron Content also wear a headset with a microphone so Clinical he/she can communicate with the in Heart technician. The table will be slowly moved Research by Radhika Sawh into the MRI machine, so that the patient’s entire body will be inside the tunnel. (The Network Update Individuals with thalassemia major and MRI machine is shaped like a large donut intermedia are at risk of developing with a deep tunnel.) During the MRI scan, complications due to iron overload. Iron the patient must lie very still and listen for The Thalassemia Clinical Research related heart problems are the most directions to hold his/her breath for short Network (TCRN) is a collaborative network dangerous complications of iron overload. durations of time for measurement of the five leading thalassemia treatment purposes. The MRI machine will make loud As part of their comprehensive care, centers in North America. Funded by the clanking and banging noises while the patients should undergo cardiac evaluation National Heart, Lung and Blood Institute, measurements are being gathered. The tests in order to determine if there are any the purpose of the TCRN is to conduct headset the patient wears will protect their heart function abnormalities that have clinical trials to evaluate current and future ears from the loud sounds. The test is developed due to iron overload. However, therapies for the treatment of thalassemia. completely non-invasive and painless. until recently there was no method to As of December 2002, 528 patients from specifically measure the amount of iron The T2* technology can also be used to the five main clinical centers and a number deposited in the heart. assess heart function and measure the size of satellite centers have been enrolled in and performance of the right ventricle. Now, thanks to a new technology called the TCRN registry. This database contains “T2*,” the degree of iron overload in the T2* testing is available for all patients with valuable medical information related to heart can be determined. a diagnosis of thalassemia major or thalassemia and its complications and intermedia who are 7 years or older. treatment. Patients included in the registry T2* is a time measurement that reflects iron Because the testing is part of a research may be eligible to participate in new and content in tissue. The T2* measurement is study, testing will be done free-of-charge. innovative research studies to benefit obtained from images taken on a standard For safety reasons, patients who have themselves and all thalassemia patients. Magnetic Resonance Image (MRI) machine. pacemakers are not eligible for T2* The MRI machine uses a strong magnet and The members of the TCRN have been busy measurements, but Port-a-Cath’s, other radio waves to image body issues. The developing many protocols that they hope intravenous catheters, and surgical clips MRI machine detects differences in will benefit thalassemia patients. A cross- are safe and do not affect the image. magnetic properties of the body and sectional study on low bone mass among Additionally, because the T2* must be done converts these differences into pictures that patients with thalassemia has begun, and in a closed MRI machine, patients who physicians can use to diagnose various two treatment protocols have been suffer from claustrophobia are also diseases. Iron overload causes changes in approved and will begin in the near future: ineligible for testing. the magnetic properties of tissues, which one for patients with low bone mass and causes the T2* values to become smaller. If you are a thalassemia patient or a parent one to determine the safety and The T2* measurement of the heart is of a child with thalassemia and are effectiveness of current treatment for important because it has been found that interested in having the T2* measurement, hepatitis C. Another protocol currently patients with a cardiac T2* value less than first get permission from your hematologist being developed will involve a comparison 20 milliseconds are at higher risk for heart and then email [email protected] of the effectiveness of deferiprone and problems related to iron overload than with the name of the patient, age, Desferal in removing cardiac iron deposits. patients with T2* value greater than 20 diagnosis, contact information and dates In addition, three papers from the TCRN milliseconds. available to have testing done in Los registry were presented at this year’s Angeles. Interested parties can also call T2* is not routinely measured on cardiac American Society of Hematology (ASH) Melissa Chin, CAF Patient Services MRI exams because it requires special convention. Coordinator, at 800-522-7222 x 206. Keep imaging and analysis programs to interpret in mind that patients and their families must CAF congratulates the TCRN on its the results. However, this technology is make their own travel and lodging progress and looks forward to more now available for all interested thalassemia arrangements. exciting developments in the near future. major and intermedia patients as part of a research study by cardiologist John Wood At present, CHLA is the only medical at Children’s Hospital Los Angeles (CHLA). center in the US offering T2* measurements; however,future sites for T2* testing involves a 45 minute scan in a T2* measurements are currently being closed MRI machine. The patient will be developed on the East Coast. ASH Facilitates Information Exchange
Representatives from CAF, including “My experience at ASH was very seeing worldwide medical advances,” Bob National President Frank Somma and enlightening,” says Mr. Somma. says. “And by distributing so much National Executive Director Jayne Restivo, “For example, combination therapy wonderful material, we are also introducing were among the 17,000 attendees at the involving Desferal and deferiprone CAF and TIF to new doctors entering the 44th annual American Society of was a big topic of conversation, field from domestic and foreign countries.” which makes me feel hopeful about the Hematology (ASH) convention, held Mr. Somma agrees with Mr. Ficarra, possibility of such a therapy. I was also December 7-10, 2002 in Philadelphia. praising the exchange of ideas with doctors glad to see that information about cardiac The largest gathering of medical from the international community. “Quality iron evaluation using T2* was being broadly professionals with an interest in blood of life is a big theme internationally,” he shared.” disorders, the convention provides a unique states. “Focusing on the unique needs opportunity for CAF to exchange For Bob Ficarra, a Board member of both that prolonged life has brought to people information with these doctors. CAF and the Thalassaemia International with thalassemia is a new challenge that Federation (TIF), the presence and the European community, especially, seems “There’s a very valuable give-and-take,” availability of many doctors from other to be focusing on.” Ms. Restivo confirms. “When we have all countries is a tremendous asset. of these medical professionals gathered in The 2003 ASH Convention will be held one place, we’re able to talk about our “I think that the introduction of Frank December 6-9 in San Diego. concerns in a more efficient and effective Somma and Peter Chieco (CAF V.P. for manner. At the same time, CAF receives Medical Information) to the major feedback and important new information.” thalassemia doctors from so many other countries has helped them tremendously in
CAF and Weill Schedule Second The agenda of the CME is as follows: Welcome: Introduction and Overview - Medical Seminar Patricia J. Giardina, MD Transfusion Therapy for Thalassemia Major and Intermedia - Alan Cohen, MD Following the successful 2002 seminar, May 1. The upcoming ASPHO meeting will Iron Metabolism, Transfusional Iron CAF and the Weill Medical College of also focus on issues related to thalassemia Overload and Chelation Therapy - Cornell University have scheduled another intermedia. The CME course will Robert W. Grady, PhD Continuing Medical Education (CME) complement the ASPHO presentations. course for Wednesday, April 30, 2003. Comprehensive Medical Care - The goal of the CME is to provide an Entitled “2nd Annual Thalassemia Update: Elliott Vichinsky, MD update on current advances in the Current Management and Future Cardiac Complications in Thalassemia - diagnosis and treatment of thalassemia, as Therapies,” this CME is made possible John Wood, MD well as to discuss novel strategies and through a generous grant from Novartis future therapies. Its target audience is Endocrine Complications in Thalassemia - Pharmaceuticals to CAF. hematology specialists, nurses and other Maria Vogiatzi, MD Dr. Patricia J. Giardina, the director of the allied health professionals who specialize in Curative Approaches: BMT - New York Presbyterian Hospital / Weill the treatment and diagnosis of thalassemia Farid Boulad, MD Medical College of Cornell University’s in the adult and pediatric community. Curative Approaches: Gene Therapy - Comprehensive Thalassemia Center, along Physician attendees can earn Category 1 Michel Sadelain,MD, PhD with CAF National Executive Director, credits for participation in the course which Psychosocial Issues in Thalassemia - Jayne Restivo and CAF Patient Services can be applied towards the AMA Susan M. Carson, RN, MSN, CPNP Director Radhika Sawh, have organized the Physicians Recognition Award. upcoming CME course, which precedes the Closing Remarks - Patricia J. Giardina, MD For more information, or to learn how to annual American Society of Pediatric enroll in this CME course, please contact Hematology/Oncology (ASPHO) Radhika Sawh at (800) 522-7222 ext. 205. convention being held in Seattle beginning m e d i c a l n e w s ( c o n t ’ d ) Blood Safety Panel Report By Gargi Pahuja
The National Advisory Committee for Blood Safety and Availability met in January to Interview with discuss “Prioritizing Decisions in Transfusion Medicine.” The Committee Stefano Rivella discussed the relative threats to the nation’s blood supply and how to best prioritize Stefano Rivella is a former recipient of a them. Experts presented evidence that CAF Medical Fellowship who has been showed that the risks of infection by HIV, involved in several exciting and important hepatitis C and hepatitis B had become very research projects related to thalassemia. small as compared to the threat of bacterial While working with the highly esteemed Dr. contamination, Transfusion Associated Lung Michel Sadelain at New York’s Memorial Injury and transfusion processing errors. As Sloan-Kettering Cancer Center, Dr. Rivella a result, the committee recommended that played a key role in the development of a spending and resources increase to focus project that cured thalassemia intermedia in on understanding and preventing these a mouse model. Moreover, in the same threats. laboratory, he was the lead author of a new There was also a presentation on West Nile project that has cured a new adult mouse Virus (WNV) and transfusion transmission model affected by thalassemia major. Now of the same. The Food and Drug Assistant Professor of Genetic Medicine in Administration (FDA) has challenged the Pediatrics at New York Presbyterian’s biotech industry to come up with a Children’s Blood Foundation (CBF) diagnostic test for WNV by the end of the Laboratories, Dr. Rivella is fortunate to be Former CAF Fellow Dr Stefano Rivella year. Roche Molecular Diagnostics® and working closely with Dr. Patricia Giardina, Chiron Inc. ® are both in the process of one of the leading lights in the thalassemia cells, platelets and red cells. At this point, developing a Nucleic Acid Test for WNV. field and Director of New York we have to make sure that the new beta This type of test can detect viral infection in Presbyterian’s Thalassemia Program. Dr. globin gene “expresses” itself – that is, the body prior to the body developing a Rivella is committed to using his experience actively produces high levels of the ß-globin recognized immune response, thereby and knowledge to better understand this protein, and only in the red cells. And, of reducing the window period significantly. disease and to find a definitive cure for course, we want it to continue expressing Both companies hope to submit applications patients affected by ß-thalassemia. itself over time. to the FDA for clinical trials by the summer. Dr. Rivella is enthusiastic about his work, To introduce the gene, we use what is Since last August there have been 17 cases and is drawn to thalassemia research called a vector. A vector is derived from a of suspected transfusion transmission of because of a genuine concern for the well- virus, because viruses are very good at WNV. All recipients of contaminated blood being of individuals with thalassemia. CAF infecting cells, but in this case, the showed symptoms within four weeks of interviewed Dr. Rivella in January. pathogenic part of the virus has been removed. We then take the vector, fill it transfusion. All reported cases occurred in Can you tell us a little about the project with the therapeutic gene and with other areas with heavy human activity and that cured thalassemia intermedia in a DNA sequences to help regulate it, and mosquito exposure. The CDC also mouse model? emphasized that transmission seemed to introduce it into the mouse. To make an occur only during the time of peak mosquito Certainly. First, you need to understand analogy, the ß-globin gene can be activity (August-September). something about how gene therapy works, considered the “hardware” and the DNA especially in relation to thalassemia. In ß- that regulates its expression the Quantifying the risk of infection by WNV thalassemia, generally, the patients have “software.” is difficult. Overall in the U. S. the estimated unhealthy beta globin genes that do not This all makes it sound very simple, but this risk is 1 per 10,000 donations, but in produce enough hemoglobin. What we are actually was very complicated. The reason areas of heavy human activity the risk is as trying to do is to add healthy beta globin that our team was so successful is that we high as 15 per 10,000. The CDC plans to genes. To do this, we have to find a way of were using a totally new vector, one that develop a surveillance system that is introducing the healthy genes into the body. sensitive to the fluctuation of the presence was not only more effective at transferring of mosquitoes seasonally and We also have to make sure the healthy the gene but also able to transfer much geographically in the country. gene is transferred exactly into the right larger DNA sequences than previous In understanding the pattern, the CDC cells, in this case the hematopoietic stem vectors. This was important because it hopes to develop a plan to combat future cells of the bone marrow. These allowed us to add a large amount of cases of transfusion transmission. hematopoietic stem cells have the property “software DNA” that could tell the gene all to generate all the blood cells, i.e. white the things it needed to do – express the gene only in the red cells, at high levels, and so on. Non-Invasive Liver Measurement And the new vector worked wonderfully. in California by Radhika Sawh After its introduction, the hemoglobin levels of the mice went from between 7 and 9 grams per deciliter to between 11 and 13 After much anticipation, the California Oakland, CA. The facility also houses a grams per deciliter, and essentially once a Ferritometer® has finally become a reality. bone densitometry machine, allowing patient reaches 11, he is cured. Using technology similar to the SQUID patients at risk for osteoporosis to have (Superconducting QUantum Interference this test performed during the same visit. Then this team, consisting of Dr. Sadelain, Device) machine at Columbia-New York Testing of liver iron through the Dr. Chad May and yourself, next turned its Presbyterian, the Ferritometer® provides Ferritometer® is free of charge for all attention to thalassemia major? accurate non-invasive measurement of the thalassemia patients, in part due to iron content of the liver. That’s right. And the first challenge was significant funding provided by CAF. that there were no mice with ß-thalassemia In individuals with thalassemia, the liver is a Appointments are available on Mondays major in existence. In a human, there are very important site for storage of iron. and Fridays. Please note that because of three different kinds of hemoglobin that are Thus, it is valuable for doctors to monitor the sensitivity of the Ferritometer®and the found during different stages of the liver iron content in order to prescribe nature of the measurements, those development – embryonic, fetal and adult appropriate chelation therapy. In the past, patients with pacemakers, artificial joints, hemoglobin. The fetal hemoglobin (which liver biopsy has been the standard for dental braces, metal surgical staples/clips does not contain the beta globin gene) measurement of liver iron. Studies have or indwelling catheters with metallic lingers in the body of the newborn for six shown that magnetic measurements of liver components are NOT eligible for months before the adult hemoglobin (which iron using SQUID technology in patients Ferritometer® testing. contains the mutated beta globin gene in with iron overload are equivalent to If you have thalassemia or are a parent of a patients affected by ß-thalassemia) takes measurements using liver biopsy samples. child with thalassemia and wish to have over. In mice, there is no fetal hemoglobin, The Ferritometer®, like the SQUID, testing through the Ferritometer®, get and the switch between embryonic and involves no needles. The procedure is permission from your hematologist and adult hemoglobin takes place in utero, with completely painless and takes then contact Eileen Scott at (800) 522- the result that the mouse does not live past approximately 40 minutes to complete. birth. 7222 x 207. The Ferritometer® is housed in the So our first challenge was to create mice HEDCO Health Sciences Building of the with thalassemia major, in order to see if Children’s Hospital & Research Center in our treatment would be effective in them. And to do this, we essentially performed a bone marrow transplant in reverse, SQUID in New York replacing normal multipotential stem cells CAF is now booking new dates for with cells that did not have the mouse beta non-invasive SQUID testing of liver iron in New York. globin gene. We analyzed these mice to Please contact Eileen Scott at (800) 522-7222, x 207 to schedule an appointment. make sure that they displayed all the features of thalassemia - they could not 12, of course, is excellent, while the 5 is I know this is hard to answer, but in the make healthy red blood cells, they had compatible with life but is still below very best case, assuming that everything – splenomegaly, were iron overloaded, they standard. So the question is why, when funding, research, testing, etc. – somehow became very weak, and so on. they all started making hemoglobin, did fell into place, and that the results of future some make more or less than others? studies didn’t result in any setbacks, when We then divided the mice into two groups, There may be any number of reasons for do you think this therapy could be made one of which was treated with the new the variability, and what we need to do now available to people with thalassemia? vector, one of which was not. All of those is to find a way to reduce the variability and that were not treated died within 8-10 I think it could happen as early as several get a consistently high level of result. weeks of extreme anemia, while all that years, but again, that would depend on the were treated lived and began producing We also have the opportunity, now that a very best case scenario, and doesn’t new hemoglobin, which electrophoresis mouse model of thalassemia major exists, include factors (such as possible changes demonstrated was coming from the new to study the biology of the disease – iron in government restrictions) that could affect vector. overload, bone formation, etc.– and use the pace of our work. But I do think these that information to improve our studies show a tremendous amount of Now, the level of hemoglobin varied understanding and treatment of the potential and do promise that we are on the between 5 and 12 grams per deciliter. The disease. path to a cure for people with thalassemia. i n t e r n a t i o n a l n e w s
TIF Holds First Associations Meeting
The Thalassaemia International Federation Thalassaemia Association, the UK There was a wonderful exchange of ideas, (TIF) held its first Thalassemia Associations Thalassaemia Society, the Panhellenic with the result that we all have a better Workshop on September 26-27, 2002 in Thalassaemia Federation, Thalassaemics understanding of what each of us is doing, Cyprus. Over 250 participants, India and the Iranian thalassemia program. as well as what needs to be done. And representing 77 associations from 26 whether an association was struggling or The concluding day of deliberations countries, attended the workshop, the was established didn’t matter – there was featured presentations from a number of focus of which was how to establish and an equal exchange from all parties.” newer organizations, many based in the support strong, effective thalassemia developing world. Participants from Ms. Adler believes that the effects of the associations. The workshop tried to Thailand, Argentina, the People's Republic meeting could be felt on a personal as well provide participants with important general of China, Egypt, Hong Kong, Australia, as an organizational level. She relates how information on thalassemia, while leaving India, Indonesia, Brazil, Palestine, Italy, she met a patient from Singapore who plenty of time for individual associations to Lebanon, Malaysia, Pakistan, Iran, rarely did Desferal and had a negative present and discuss their experiences. Bangladesh, Singapore, Taiwan, the UK and outlook on life. “Even though she was in The event kicked off with a welcome from Canada painted a vivid picture of the love with someone and was engaged to be the President of the Cyprus Thalassaemia challenges they faced in trying to secure married, she never felt the importance of Association, Stelios Kreouzos; TIF appropriate treatment and support for taking care of herself Chairman Panos Englezos; Dr. Victor thalassemia patients and their families. and acting like she had Boulyjenkov of the World Health her whole life ahead of Organization (WHO) ; and the Cyprus her.” Minister of Health, Frixos Savvides. The first day heard presentations on a wide range of topics,
including the epidemiology of thalassemia, “What impressed me so much,” said Ms. But Ms. Adler says this patient’s attitude the role of WHO in the fight against the Restivo, “was that despite the difficulties changed after speaking with other patients disease, current policies of clinical care, many of these people face – ignorance at the workshop. “She used her pump blood safety, and the ideal structure of a about the disease, prejudice, financial every day and couldn’t wait to marry her thalassemia reference centre. Specialist problems, unavailability of medicine and fiancé. She was finally looking forward to speakers included Dr. Antonio Piga and Dr. resources – despite all this, they are living instead of dying.” Androulla Eleftheriou. They were followed determined to keep on pushing and by a group of speakers – including CAF fighting. They may be overwhelmed at The first Associations Workshop brought to National Executive Director Jayne Restivo times and sometimes may want to give up, fruition a long-term goal of the Federation, and CAF members/TIF USA but that’s why meetings like this are marking a major step forward in TIF’s effort representatives Bob Ficarra and Dawn important – to give them the to promote and support thalassemia Adler – who shared their experiences on encouragement that they need to keep associations around the world. CAF ways to organize, fund and promote a going.” congratulates TIF on this wonderful event national thalassemia association. and looks forward to participating in the TIF USA Representative Dawn Adler, next one. The first day of the workshop ended with whose speech focused on CAF’s presentations from some of the longest- Thalassemia Action Group (TAG), felt that Special thanks to TIF for providing established organizations – the Cyprus the Association meeting was “amazing. information used in this article. Reactions to From a Patient’s Point of View: TIF Associations All You Need is the Wish Workshop (While attending meetings and conferences, CAF members encounter many wonderful Bob Ficarra, CAF Board Member and TIF individuals with thalassemia from around the USA Representative: “TIF’s first world. They come from different Associations workshop was a wonderful backgrounds, but they all share a firm success. A plan was adopted to expand commitment to fighting thalassemia. Below and formalize the procedure, so that similar is a profile of one of these individuals.) meetings would be held on alternate years “My dad is very strong and really made a between the parent/patient conferences. difference. When he was told I had The Steagall Family The goal will be to formally help teach thalassemia, he decided he would cry only we need to raise funds to implement associations how to organize, expand and at my funeral. Until then, he would devote improvements. There are many challenges, strengthen their associations.” himself to searching for the best treatment but there is much more good will to win!” Bob Azariah, Honorary Secretary, available from anywhere in the world.” Merula met her husband, Denis, while in Thalassaemia Association of Malaysia: “I So says Merula Steagall, a 36-year-old college and told him in stages about her and Jasmin Chong Abdullah, a parent, Brazilian woman who is president of thalassemia. “He accepted all of this, attended the meeting in Cyprus, and were ABRASTA, the Brazilian Association of seeing how naturally I handled these able to meet with delegates from various Thalassemia. Attractive, self-assured and ‘problems.’” countries and exchange notes on what was giving, Merula credits her parents with being done at each place. We gathered a Denis and Merula are the birth parents of instilling in her a determination to live a full lot of information, including pricing and nine-year-old Daniel and five-year-old Dina life despite thalassemia. availability of Desferal and L1, services and the adoptive parents of three-year-old provided for thalassemics at each hospital “I always received a lot of love from my Roberto. “Before getting married, I and stem cell and umbilical cord cell parents,” she says, “but they also taught traveled to the U.S. on business and research. We also received and me responsibility, especially with regard to decided to get information about older exchanged offers to help each other my treatment. As a result, I always wanted patients, pregnancies and so on. I was whenever and wherever possible.” to maintain the best treatment, because I lucky to meet wonderful Sophie Sioshillos, knew I needed to be well to do all the things who introduced me to all the wonderful Ms. Amal R. Daoud, Thalassemia Patients’ that I wanted to do. I loved parties, friends, patients and friends of TAG. I also met a Friends Society, Palestine: “It was a great boy friends, sports; but I knew that to enjoy fantastic doctor, Fernando Tricta, in my opportunity to learn about other them, I needed to have discipline in my country, who never said no to any of my organizations, their activities, their treatment.” wishes but still gave me the best advice.” struggles and successes, as well as to strengthen the relationship with TIF and Merula began chelating when she was four Merula needed no special care during her learn about different ways that cooperation years old, taking Desferal through injection. pregnancies. She interrupted Desferal in this field could happen. For us, it was Her father obtained a pump for her from therapy before the confirmation of the also an opportunity to meet associations London when she was nine or ten; she now pregnancies in the cases of both Daniel and from the Arab world, who mainly have uses a microjet pump five or six days a Dina and experienced no complications similar problems and issues of concern in week. L1 was recently approved for use in during or after the pregnancies. our countries; it was a time to strengthen Brazil, so she will soon start a new therapy In between Daniel and Dina, Merula was relations and start networking on ways to (four days of L1 and three days of pregnant again, but the child developed help on the regional level as well as the Desferal.) anecephalia and did not survive. When she national level. But the most significant fact When she was growing up, Merula’s heard about a baby in need of adoption, she is how important it is to empower the parents had to import Desferal from said, “Don’t worry, this baby is mine,” and patients and volunteers in the associations Greece. Now the government pays for immediately adopted him. “I knew another for them to take active part in decision Desferal for patients, although about half of baby was waiting for me, it just took time making and strengthening the associations ABRASTA’s 485 patients lack new pumps. for us to find each other.” so as to work better for fellow “But all of us at ABRASTA are working hard thalassemics and their families. A believer in positive attitudes, Merula says, to change that and make sure that all “Thalassemia is no limitation to doing “It was a great time to learn from each Brazilian centers have the best treatment anything. Life is a wonderful, endless other and meet new friends. We hope that options available, and in the shortest time – construction. this event will be held again!” thalassemia patients have no time to lose! We have many advantages in Brazil: “You can be anything you wish. All you wonderful people, wonderful doctors. Now need is the wish.” c h a p t e r n e w s
California Chapter Queens Chapter undertook either the 62-mile ride, the 12-mile ride or the four-mile walk. The Queens Chapter held a Valentine’s Dance The California chapter has hired Melissa “I’m very pleased with the turnout,” says Hank on Saturday, February 8, at Jericho Terrace in Tamburo as the new chapter coordinator. A Cerruti, Event Chair. “Once again the people of Jericho, NY. Tickets were $65.00, and 240 recent college graduate, with a B.S. degree in Suffolk came through for Cooley’s anemia.” The people attended the evening, which also included psychology, Melissa plans to attend graduate event raised approximately $50,000. school to pursue a PhD in social psychology dinner, entertainment, raffles and prize drawings. next fall. Currently, along with working as the Atlantis, Applebee’s Restaurant and Adam The first Annual Queens Chapter Golf Classic chapter coordinator, she is working with a Kaufman of Northwestern Mutual Financial was held on September 23 at the North Hills professor at the University of California, Network were the Corporate Sponsors, each Country Club in Manhasset. Twenty-five Riverside. Melissa has a strong desire and generously donating $10,000 in cash or commitment to make a positive impact on the services. Applebee’s donation included California chapter, due to her connection to wonderful food, and Atlantis supplied four thalassemia (her fiancé has thalassemia major). complimentary tickets for each participant. Phil Melissa is excited to have this opportunity and is and Kitty Lagana were individual donors at the anxious to work hard for the chapter. $10,000 level. Pine Barrens Printing was the Platinum Sponsor, while Gold Sponsors included The chapter is sponsoring the 2003 Thalassemia Stop & Shop, Going Sign Company, Jake and Conference/Meeting in San Diego on June 28. Carol Watral, and Tom Watral and Dennis Vita. The aim of the conference is to raise funds and Silver Sponsors were Starbuck’s Coffee and awareness. The chapter hopes to build a Outback Portable Toilet Inc. stronger community among the thalassemia patients and parents in California and the The 2003 Bike-A-Thon will again be held in surrounding states. The conference will be a September. A 30-mile bike ride will be added to daylong event consisting of presentations by the 12-mile ride and the 62-mile ride. Once doctors and patient/parent networking, as well Queens Chapter Golfers again Starbucks and Krispy Kreme will serve as a day at Sea World. coffee and donuts and Applebee’s will serve foursomes participated in the outing, which was lunch. A Kiddie Korner will be set up with a slide For more information, contact Melissa Tamburo followed by a cocktail hour and a delicious and trampoline, and a clown will be available to at (866) 922-9314 or dinner. City Wide Demolition and Rubbish paint faces. The day will end with raffle prizes [email protected]. Removal served as host for the event, and and a great magician. Online registration begins tickets were $350 ($150 for cocktail party and in April at www.cooleysmilesforsmiles.com. Long Island Chapter dinner only). The Suffolk chapter held its first Halloween Party To help during this period of severe blood The annual Dinner Dance is scheduled for April on Friday, October 25, 2002, at the Marriott shortages, the Long Island Chapter held a blood 18 at Russo's on the Bay (Howard Beach), Hotel in Melville. The costumes were great, and drive on January 26, 2003, from 9:00 a.m. to starting at 6:30 p.m. This year's honoree is Billy the crowd partied until 2:00 a.m., with singing, 2:30 p.m. at the school cafeteria at St. Joseph's Jelley, Director (retired), Bureau of Cleaning & dancing and prizes. Collection, Department of Sanitation. Tickets Church. Suffolk’s annual Beef-O-Rama was held at are $85.00 and include a seven-course meal and Pace's Steak House in Port Jefferson on Bobby Valentine was the honoree at the Long a night of dancing. RSVP to John Mancino January 21. The restaurant was exclusively ours Island chapter’s annual Gift of Life Ball on (718-334-9080) or Paul Tucci (516-322-3373) by for the night. Drinks, cocktail hour and a steak November 16, which was held at Leonard’s of March 15. Great Neck. Tickets were $175 and included a dinner were featured. A select 100 guests were invited at a cost of $175 each. Raffle tickets cocktail reception, dinner and dancing. This Staten Island Chapter year’s dinner journal was dedicated in memory of were sold for the entire evening, and everyone had a great time. the late Vincent J. Cuti, Jr. Over 250 people attended the Staten Island chapter's fifteenth annual "Let Your Heart Suffolk kicked Super Bowl Sunday off at Cafe La New Jersey Chapter Dance," held on February 14 at LiGreci's Staaten Strada in Hauppauge. Again, the restaurant was Restaurant. The $75.00 ticket price included a closed to patrons. Drinks, a cocktail hour and The New Jersey chapter held its first "Night at full sit down dinner, a top shelf open bar and buffet dinner were served at a cost of $250 per the Races" at the Brooklake Country Club in both a deejay and a band for great dance music. person. Raffles were sold during the game. Florham Park, NJ on November 15. It was a This was a first for the chapter, but it was so For the twelfth year, the Staten Island chapter significant success for the chapter, with over 100 successful that it will become an annual event. attending, raising $7,000. In addition to the presented a magnificent holiday light display, racing, a highlight was the fabulous buffet which ran from November 30, 2002 to January Last year Suffolk had a wine tasting party at prepared by Chef Barry D'Onofrio, previously 5, 2003. Sponsored by Casale Jewelers and Monica Bella's Restaurant in Melville. Vinifera from Windows On The World. The chapter Barone Steel, the light display has become a Wine Imports served 16 different wines and would like to thank its friends at the greatly-anticipated Staten Island tradition. Over discussed the features of each. A limited Westchester-Rockland chapter for helping with 100,000 lights were used in the decorations, and number of guests were invited at a cost of $350 its first “races” event. Next year’s “Night at children were treated to such favorites as per person. This year's date has not been the Races’ is scheduled for Friday, November Barney, Baby Bop, Rudolph, the Power Rangers, scheduled, but it will be in May. Winnie-the-Pooh and, of course, Santa. 14, at Brooklake, and the chapter expects to Arthur Pace of Pace's Steak House in double its attendance. Hauppauge and Port Jefferson will be honored Suffolk Chapter The chapter’s Annual Golf Classic was held on as this year's "Man of the Year." Arthur has been a dedicated supporter of Cooley's anemia September 23 at Minisceongo Golf Club in More than 400 people turned out for the Suffolk for more than 20 years, and the chapter expects Pomona, New York. There were 55 players Cooley’s Anemia Foundation chapter’s first Bike- over 50 people to be on his Tribute Committee. participating. It was a perfect afternoon for golf. A-Thon on Saturday, September 21. Although The dinner dance will be held on Friday, June 20, The 2003 Golf Outing will be Tuesday, May 27 at many came simply to enjoy the variety of family 2003, at Leonard's of Great Neck. Between Minisceongo. Call 973-893-2241 for details. activities, such as children’s slides, face painting 400 and 500 guests are expected. Call (631) and refreshments, approximately 150 people 863-0532 for details. The annual golf outing will be held in September at Cherry Creek Golf Course; a date has not yet been selected. Chapters Help Finance Medical
Westchester-Rockland Chapter Technology for Patients
The Westchester-Rockland chapter will hold a Several CAF chapters have made commitment to the health and wellbeing of Night at the Races fundraiser on March 14, 2003, at the VIP Club in New Rochelle. Tickets significant contributions toward CAF’s thalassemia patients across the country." funding of the innovative “SQUID” are $50 each and include beer, wine and a buffet In a joint statement, Cammie Brandofino, dinner. Some 300 people attended last year's technology now being used to painlessly President of the Staten Island chapter, and event, which featured betting on videotaped measure liver iron in thalassemia patients Peter Chieco, President of the Westchester- horse races, betting tables, and a raffle. For (see story on page 11). more information, please call (914) 232-1808 or Rockland chapter, said, “People with email [email protected]. The Long Island chapter has given $50,000, Cooley’s anemia face many difficulties. We The New York Country Club in Hempstead was and the Staten Island and Westchester- are delighted that an alternative to painful the site of the chapter’s annual Golf Outing, held Rockland chapters have each donated liver biopsies is now available for them and September 10, 2002. The event attracted 120 $30,000 to help underwrite this exciting proud to help CAF in its efforts to improve players, including “Goumba Johnny” Sialiano of program. the quality of life for thalassemia patients.” WKTU. Tickets were $250 and included a delicious post-game dinner. The “SQUID” (Superconducting Quantum "Our patients have waited a long time for Interference Device) technology measures something like SQUID," said former Long magnetic fields to determine iron content in Island chapter President Dominick the liver. The process is an alternative to a Guglielmo. "The Long Island chapter is TRIBUTE painful and more invasive liver biopsy. proud to be able to make a significant contribution toward this important Due to the efforts of the national Cooley’s TO THE FOUNDERS OF CAF technology." Anemia Foundation (CAF), thalassemia patients have had access to the SQUID Brandofino, Chieco and Guglielmo also The Suffolk chapter honored four people technology at Columbia Presbyterian acknowledged the invaluable support of the who have played key roles in the life of Medical Center in Manhattan for the last National Institute of Diabetes and Digestive CAF with a “Tribute to the Founders” on several months. CAF has also recently and Kidney Diseases (NIDDK). A division October 13. Over 250 people feted Nunzio entered into a similar arrangement with the of the National Institutes of Health, NIDDK and Rose Cazzetta, Theresa Ficarra and Children’s Hospital Oakland for the use of a was instrumental in the development of the Connie Paradiso, each of whom continues Ferritometer®, which also measures liver SQUID technology. to play an influential role in guiding CAF. iron by means of technology similar to that CAF’s chapters are the lifeblood of the When CAF was founded in 1954, its of the SQUID machine. organization. They provide immediate meetings were held in a small office behind CAF President Frank Somma applauded the contact with thalassemia patients on a local Frank and Theresa Ficarra’s butcher shop. actions of these three chapters. "This level, raise valuable funds and spread Ed and Connie Paradiso joined soon after, generosity is greatly appreciated by both awareness about thalassemia among and Rose and Nunzio Cazzetta became a CAF and the many patients who will benefit members of their local communities. For part of CAF life in the early 1960’s. from their largesse," he said. "Once again, more information about CAF chapters, “They were parents whose love for their CAF's chapters are demonstrating their contact CAF at (800) 522-7222. children was so all consuming that they HAD to act,” said CAF National Executive I want to help in the fight against thalassemia. Director Jayne Restivo. “Our patients live longer, fuller lives today because of the Enclosed is my contribution of work of these brave pioneers.” ____ $25 ____ $50 ____ $100 ____ $250 Other $ ______CAF President Frank Somma praised the founders in a letter read aloud at the event, Name stating that “without these four wonderful, ______caring and determined individuals, there Address wouldn’t be a CAF at all.” ______
Nunzio Cazzetta expressed the philosophy City /State /Zip of the founders by quoting Stephen Grellet: ______"I expect to pass through this world but Please make all checks payable to the Cooley's Anemia Foundation. once. Any good thing, therefore, that I can do, let me do it now, for I shall not pass this Mastercard ____ Visa _____ Card# ______exp. ______way again." Mail to: Cooley's Anemia Foundation, 129-09 26th Ave #203, Flushing, NY11354
All contributions are tax-deductible. Buffalo – Dennis Locurto s r 716-834-8903 CAF Welcomes e California – Carmen DiLorenzo b 866-922-9314 Chapter
m Capital Area – Emanuel Katsaros u 410-633-1309 Development
N Connecticut – Peter Steiwer
t 914-232-1808 Manager c Long Island – Joseph Giammalvo a 631-271-8577 The Cooley’s Anemia Foundation (CAF) is t pleased to announce that Jennifer Fleming n Massachusetts – Rudi Viscomi has joined its staff as the new National o 617-332-5952 Chapter Development Manager. A Jennifer Fleming C New Jersey – Matt Stamateris graduate of Hofstra University with a “I am excited to be part of an organization
r 973-893-2241 degree in communications, Jennifer comes that takes proactive roles in both furthering e Queens – Frank Zito t to CAF from the National Alliance for research and enhancing the lives of its 718-746-7677 p Autism Research, where she was a members,” Jennifer states when asked a Rochester – Peter Paradiso Chapter Manager. She brings to CAF what attracted her to become a part of CAF.
h 716-248-3385 experience in both chapter development
C Anyone who needs information about a CAF Staten Island – Cammie Brandofino and fund raising. 718-761-3580 chapter near them, or who would like to Suffolk – Edward Martella “One of my primary goals will be to help take part in creating a new chapter, is 631-863-0532 existing chapters raise awareness about encouraged to contact Jennifer at (800) thalassemia in their local communities, as 522-7222, ext. 208. Westchester-Rockland – Peter Chieco well as increase the amount of dollars 914-232-1808 CAF is delighted to have a person of obtained through fund raising efforts,” Texas – Mateen Shah Jennifer’s caliber leading our efforts to Jennifer says. With the help of CAF staff 214-324-6147 increase community awareness and raise and volunteers, she also aims to develop funds for vital medical research and new chapters in areas where there is an improved patient care. interest and need.
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