FALL 2008 NUMBER 59

THE NATIONAL NEWSLETTER OF THE IMMUNE DEFICIENCY FOUNDATION Diagnosis— How Long Does It Take?

Immune Deficiency Since 1995, IDF has taken the lead in The first reaction many have to this news— Foundation Mission: conducting national surveys of patients with “How can it be getting worse with all the primary immunodeficiencies. Our survey awareness, education and new medical The national patient data has been used effectively to advocate advances?” may not be appropriate. In organization dedicated with government for legislative and addition, the average age of the patients in regulatory remedies for our patients, has the 2007 survey was almost 39 years, to improving the been cited and used by medical professionals compared to 34 years in the 2002 survey. diagnosis, treatment to outline patient treatment and diagnosis These results do not necessarily indicate that and quality of life of experiences, as well as by industry and it is taking patients longer to become persons with primary government to understand the demand for diagnosed. It could mean that recognition of immunodeficiency and efficacy of immunoglobulin replacement these diseases has actually improved and, diseases through therapy. IDF survey data has been that some adults with “mystery” illnesses indispensable in furthering the have finally received a definitive diagnosis. advocacy, education understanding of these diseases by better and research. defining the health status, treatment The 2007 Patient Survey also saw an increase patterns and psychosocial experiences of our in the proportion of patients reporting patients. Common Variable Immodeficiency Disease Diagnosis Survey ...... 1-2 (CVID) as their diagnosis. These findings Health Insurance Coverage One of the most quoted IDF survey findings would be consistent with longer times to For College Students ...... 3 is from the IDF 2002 National Patient diagnosis, as those with CVID consistently Medicare IVIG Access Act ...... 3 Survey —9.2 years is the average time from report longer times from symptom onset to Diagnosis Interviews ...... 4-6 the onset of symptoms to diagnosis for diagnosis than those who report other patients with primary immunodeficiency. specific primary immunodeficiency disease New Plasma Donation Website ...... 6 This discovery has stunned many— “How diagnoses. (Continued on next page) IDF Family Retreats ...... 7 could diagnosis take this long?” and Industry News ...... 8 outraged others—“Something must be 2002 & 2007 IDF National Patient Surveys SCID International Conference ...... 9 done!” Nonetheless, over the years, the 9.2 Average Time to Diagnosis From Volunteers Across the Country ..... 10 years average has become universally Sympton Onset With Gratitude ...... 11 acknowledged and accepted. Monthly Giving Program ...... 11 However, new data from the IDF 2007 2009 National Conference ...... 12 National Patient Survey has provided us with some interesting results. It would appear as though the average time to diagnosis for patients with primary immunodeficiency disease has increased from 9.2 years to 12.4 years. Since the second survey excluded patients who were included in the 2002 survey, the true average for all patients would actually be between 9.2 and This newsletter is sponsored by 12.4 years. an educational grant from Talecris Biotherapeutics. Diagnosis—How Long Does It Take? Reasons (Continued from page 1) to be Time to Diagnosis After Sympton Onset 30% 2002 2007 Thankful 25% 25% 24%

20% 20% 20% 17% 16% 15% 15% We are thankful 13% 12% 10% 10% 10% for our donors. 8% 6% 5% 5%

In this season of 0% Less than 1 - 2 3 - 5 6 - 9 10 - 14 15- 19 20 + giving, won’t you 1 Year years years years years years years Q9. At what age (in years) was that person first diagnosed with a primary immunodeficiency disease? please support the Q8. At what age (in years) did these repeated, serious unusual infection begin? These longer times to diagnose are often As the chart below indicates, those who periods of repeated infections for patients. were diagnosed in fewer than 9 years had Immune Deficiency Crippling infections unfortunately can the best chance of avoiding life-long lead to permanent impairments or loss of impairments and enjoying a higher Foundation? function. Survey respondents were asked quality of life. Although immunoglobulin if they experienced any permanent therapy can reduce the frequency and impairments or loss of function in such severity of infections in patients with areas as the lungs, digestive system, in antibody deficiencies, it cannot erase or fix their vision, hearing or even if they had existing permanent impairments. IDF Advocate mobility or neurological issues before they Editor were diagnosed. Christine M. Belser Number of Permanent Functional Impairments Before Diagnosis Medical Editor Rebecca H. Buckley, MD Five or more 30.1 Publication Manager Adam Freestone Four 19.4 The Immune Deficiency Foundation publishes IDF Advocate three times Three a year. To obtain a free 19.1 subscription, please contact: Two 18.2 IDF Advocate Immune Deficiency Foundation 40 West Chesapeake Avenue One 13.6 Suite 308 Towson, Maryland 21204 None 8.6

Toll-Free 800.296.4433 0510 15 20 25 30 35 Direct 410.321.6647 Fax 410.321.9165 Number of Years Before Diagnosis E-mail [email protected] Q. By the time of initial diagnosis as immune deficient, had the patient suffered any permanent Web Site www.primaryimmune.org impairment or loss of the following.

Copyright © 2008 by the Immune Deficiency Foundation. The Immune Deficiency The one thing IDF patient data does indicate is that the time to diagnosis for patients with Foundation is a publicly supported, tax-exempt primary immunodeficiencies is not improving. As the following personal stories organization as described under Section 501 (C) (3) of the Internal Revenue Code of 1954, demonstrate, it is critically important for patients to receive timely and accurate diagnosis. as amended. Early diagnosis and proper treatment give patients the best possible opportunity to live healthy, productive lives.

2 | FALL 2008 CMS to Remove Preadministration Fee Our and Reduce Reimbursement for IVIG Congressional Supporters he Centers for Medicare and Since June, countless patients, physicians Medicaid Services (CMS) and family members participated during the SENATE announced new rules that public comment period following the Senator Lamar Alexander (R-TN) T eliminate the preadministrative announcement of the proposed rules by Senator Sam Brownback (R-KS) fee for IVIG infused in the physician’s office using the IDF Action Alert to send letters Senator Elizabeth Dole (R-NC) and the hospital outpatient department for directly to CMS and to encourage their Senator John Ensign (R-NV) Medicare beneficiaries. The legislators to do the same. IDF is extremely Senator John Kerry (D-MA) preadministration fee was introduced by grateful for the outpouring of support from Senator Debbie Stabenow (D-MI) CMS in 2006 to assist physicians in locating the community on this issue, and for the Senator Ron Wyden (D-OR) and obtaining IVIG. The new rules also letters sent to CMS by concerned Senators further reduce reimbursement in the and Representatives. HOUSE OF hospital outpatient department for all REPRESENTATIVES Medicare Part B drugs, including IVIG. Both Please do not become discouraged, but Representative Kevin Brady (R-TX) rules will begin in January 2009. continue your commitment to this issue and Representative Jo Ann Emerson (R-MO) join us in the coming year as we carry on our Representative Phil English (R-PA) IDF is deeply disappointed with this efforts on Capitol Hill. We urge you to alert Representative Mike Ferguson (R-NJ) Representative Jim Gerlach (R-PA) decision. It is our belief that eliminating the IDF to any problems you experience Representative Gene Green (D-TX) preadministration fee and reducing obtaining your prescribed product in the Representative Wally Herger (R-CA) reimbursement for Medicare beneficiaries in setting that is best suited to your needs, and Representative Maurice Hinchey (D-NY) the hospital outpatient department will it is especially important to report any Representative Steve Israel (D-NY) exacerbate the current access and Medicare related access issues. We also Representative Sam Johnson (R-TX) reimbursement problem. request that you take a few minutes to Representative Michael McNulty (D-NY) complete any IDF surveys you receive. This Representative Grace Napolitano (D-CA) Representative Ron Paul (R-TX) Citing a 2007 report by the Office of data is critical in demonstrating the Inspector General, “Intravenous Immune Representative Joe Pitts (R-PA) problems our patients are facing. Representative David Price (D-NC) Globulin: Medicare Payment and Representative Jim Ramstad (R-MN) Availability,” as well as IDF survey data from As we prepare for the 111th Congress, IDF is Representative Lucille Roybal-Allard (D-CA) 2006, 2007 and 2008, IDF responded to reviewing our legislative strategies. We Representative Chris Smith (R-NJ) CMS by requesting that the remain hopeful that lawmakers will keep in Representative Hilda Solis (D-CA) preadministration fee be maintained in both mind the challenges facing our community Representative Ellen Tauscher (D-CA) settings, and reimbursement in the hospital and work with us to ensure access to all Representative Tim Walberg (R-MI) outpatient department not be reduced. treatment options, and quality care for all of Representative Robert Wexler (D-FL) our patients.

LAW ENSURES and well-being of college students with coverage. Ultimately, Michelle lost her primary immunodeficiency disease, battle with cancer. HEALTH INSURANCE passed through Congress on September COVERAGE FOR 25, 2008. Michelle’s Law was signed Keeping the struggle of the Morse into law in the state of New Hampshire family and other families throughout COLLEGE STUDENTS by Governor John Lynch on June 22, the country in mind, IDF would like to 2006, and on October 9, 2008, after thank all of you who used the IDF NATIONWIDE being signed by President Bush, it Action Alert to contact their Senators became law nationwide. and Representatives in support of this You spoke and Congress listened! Over the legislation, and, of course, Senator course of five days, more than 700 people Michelle’s Law is in honor of New Sununu and Congressman Hodes for used the IDF Action Alert to rally in support Hampshire student Michelle Morse championing such an important of Michelle’s Law, a bill that provides a 12- who was diagnosed with colon cancer initiative. It is our hope that students month extension for critically ill or injured her senior year of college. She was in the future will never have to decide college students to retain coverage under forced to remain a full-time student between their health and their their parents’ health insurance. This piece of while undergoing chemotherapy to education. legislation, crucial in protecting the health retain her parents’ health insurance

ADVOCATE | 3 DIAGNOSIS INTERVIEWS by J. Doug Gill

According to a past Immune Deficiency 20 years, and took the time to document The basic defect in X-Linked Agamma - Foundation survey of patients, the aver - nearly every experience. John chronicled globulinemia is a failure of B-lymphocyte age time from the onset of symptoms to more than 100 doctor visits, a thousand precursors to mature into B-lymphocytes diagnosis is 9.2 years. Some patients, blood tests, and six major surgeries, in - and ultimately plasma cells. however, are diagnosed rather quickly. cluding the removal of his tonsils, ade - Matt was too young to remember the re - For others, finding the source of illness noids, uvula and appendix. current illnesses that had his parents fre - stretches on interminably. He has suffered from recurring bouts of quently calling upon doctors. “That nine year average looks pretty good pneumonia, lung infections, nausea, diar - “Constant infections,” is how Matt de - to me,” says John Armes, a 58-year-old rhea and indigestion. Some medical pro - scribes his first three years of life, “and CVID patient from Florida. “It took 28 fessionals referred him to psychiatrists, quite a few cases of pneumonia.” years to diagnose me and all because no suggesting that perhaps his illnesses were one bothered to give me a relevant blood of the psychological variety. He was also Matt, however, still feels his situation was test.” diagnosed with chronic fatigue syndrome one of fortune, thanks to a mother who and sleep apnea – which led to yet an - made her living as a biologist and a fa - CVID – or Common Variable Immune other surgery for a deviated septum. ther who worked in the field of chem - Deficiency – is a disorder characterized istry. by low levels of serum immunoglobulins “I’ve learned that you don’t live with (antibodies) and an increased susceptibil - CVID,” John tells me, “You fight with it. “One of my mother’s brothers was also a ity to infections. CVID is a relatively com - And you better be ready to fight – fight doctor,” Matt explains, “My family had mon form of immunodeficiency, and the for everything you need to survive; every - quite the support network, and their un - clinical course varies from patient to pa - thing you need to have a good quality of derstanding of the medical field – espe - tient. life.” cially the research side – was of great benefit. It made navigating the health John doesn’t place all the blame on the care system easier than it is for most peo - myriad of doctors he encountered on ple facing a similar situation.” what, at various intervals, seemed like every two months. He is also quick to point out that living with XLA has not prevented him from “Look, what you have to remember is living a normal life, but he does admit to that this was so long ago – and I grew up a bit of naivety about how the public at in a small town in Illinois – so going to large – and even the medical community the doctor back then was something - perceives those with primary immune folks didn’t do at the first sign of the deficiencies. sniffles. Besides, he was one of those old country doctors who treated everything “It shocks me that the medical field isn’t with either penicillin or paragoric.” better versed in – or even more aware of – primary immune deficiency diseases. I Armes does - think I’ve long been under the mistaken n’t recall his Matt Roth and his daughter Jane assumption that immunology study is early visits much more prevalent. It’s such a big part to that ‘old Matt Roth, a 41-year-old XLA patient of my life that I assume the rest of the country from Colorado, also cites respiratory world is just as in tune.” doctor,’ damage as a result of not being diag - but he nosed in a timely manner. Scar tissue on Ohio resident Mary Rath feels a connec - does re - his lungs and chronic bronchitis are the tion to Matt Roth’s parents; she, too, is a member legacy of his elongated diagnosis. medical professional and credits that ca - the reer path with helping during the diagno - Matt was not quite four when he was di - process sis of her son’s disease. agnosed with X-Linked Agammaglobu - that linemia. XLA is caused by a defect in the played “Brian was healthy for the first three BTK gene that resides on the X chromo - out over months of his life,” Mary relates, “but some and thus is only expressed in men. the next from that point on, it was doctor visits John Armes

4 | FALL 2008 mainly because each time we’d go see yet son, Jerry, was tested at birth for what another doctor they would ask me for doctors had determined was responsible his medical history. After repeatedly lay - for their passing. ing out this enormous list, I thought it Jerry was diagnosed with Severe Com - might be easier if I had it all written bined Immune Deficiency (SCID), the down.” most serious form of primary immunod - Fungal and bacterial are two terms quite eficiency disease. SCID, embedded on familiar to Massachusetts resident Bar - the public consciousness through the bara Meade, the difference being the re - case of David Vetter (“the boy in the sults were visible and not hidden within. bubble”), is a primary immunodeficiency Mary and Brian Rath in which there is combined absence of T- Barbara’s son, Joe, was diagnosed with lymphocyte and B-lymphocyte function. and antibiotics. And I’m not exaggerat - CGD when he was two. Chronic Granu - ing when I tell you that he took antibi - lomatous Disease (CGD) is a genetically Yvette’s first son, who died in 1972, oc - otics about 360 days a year.” determined disease characterized by an cupied a hospital room across the hall inability of the body’s phagocytic cells to from David. Brian, her son, was diagnosed with make hydrogen peroxide and other oxi - Hyper IgM Syndrome, but it was not “They knew right away with my first dants needed to kill certain microorgan - until after a serious case of pneumonia son, and instantly put him in a germ-free isms. As a that almost cost him his environment. But he was only with us result, pa - life. for seven months.” tients with Patients with the Hyper CGD have Yvette is even more forthcoming with IgM Syndrome have an in - an in - the circumstances surrounding the death ability to switch their anti - creased of her second child. body (immunoglobulin) suscepti - production from IgM to bility to “A lot of it was denial,” she says without IgG, IgA, and IgE, result - infections hesitation. “Number one, I was con - ing in decreased levels of caused by vinced that there was no way I would IgG and IgA and normal certain lose another baby, and to distance myself or elevated levels of IgM. bacteria from that loss, I didn’t go back to the The most common form is and doctor who cared for my first child. inherited as an X-chromo - fungi. Number two, at every single milestone de some linked trait and af - d Barbara Mea check-up for newborns, my new doctor Joe an “As an infant, Joe fects only boys. told me that my second child was per - suffered from one skin infection after fectly healthy.” “He was literally at death’s door,” Mary another,” Barbara shares. “He would de - remembers, “and had it not been for a velop various rashes and boils and at the Jerry’s story is nothing short of remark - hematologist who kept insisting that same time, he was running almost con - able. At birth, he was placed in a germ- normally healthy people do not get stant fevers that the doctors couldn’t ex - free environment; at three-and-a-half, he pneumocystis, I hate to think of the out - plain.” had his bone marrow transplant. come.” (Continued on next page) “He had developed large lymph nodes Pneumocystis is an infection caused by a on his neck,” Barbara clarifies, “and the microscopic fungus that lives in the doctors had the symptom needed to lungs of people and usually causes no make the diagnosis.” symptoms in healthy people, but can “Even thought it took two years to diag - cause pneumonia in infants who have nose Joe,” she continues, “I – luckily – conditions that affect the immune sys - don’t believe it resulted in additional ad - tem. Once the fungus in the lung was verse effects. You usually hear about found, diagnosis came quickly. CGD being diagnosed much later in life Mary documented each time her son got – I think that’s when the irreparable sick. damage is more prevalent.”

“Brian had so many illnesses,” she con - Sadly, some diagnoses of the quick, accu - fides, “that I started keeping track of rate variety happen as a result of the what he had and when. I kept a journal, death of a preceding sibling. Yvette Shorten lost two boys before her third Jerry and Yvette Shorten

ADVOCATE | 5 DIAGNOSIS INTERVIEWS (Continued from page 5)

Now 22, he works as a corrections officer in Texas. And Beyond their connection to immune deficiency, these five not hidden away in an administrative office, either, he extraordinary people share a couple of other common walks the front lines of the general population. threads.

“Oooh… I wasn’t exactly pleased to hear that news,” Yvette All would like to see medical schools worldwide expand laughs. “And I told him about it, too! Jerry waited for me their curriculum and offer more than a cursory glance at to finish my lecture and then made a point to use my own the immune system. words against me.” And all agree that arming yourself with volumes of But she couldn’t argue with his response. Throughout knowledge about the disease - even though that puts ad - his life, Yvette would constantly tell Jerry that he was spe - ditional pressure on the patient and their family – is the cial, and that he was indeed part of a greater plan. first step toward feeling like you’re not in this alone.

“He reminded me that I’ve always preached about the power of prayer and faith,” Yvette continues, “and told me he was just living his life the way I had taught him – the way God had planned.”

New Web Site www.donatingplasma.org

Plasma donors looking for information about eligibility, how plasma is used and locations of source plasma collection centers now have a new, one-stop resource to guide them. On September 17, 2008, the Plasma Protein Therapeutics Association (PPTA) launched www.DonatingPlasma.org, a Web site dedicated to raising awareness and providing information about source plasma donation and how it is used to produce therapies that save and improve lives. Debunking myths associated with plasma donation and providing testimonials on the importance of plasma from patients, donors and physicians will allow individuals, the media, local policymakers and the general public to become better informed about how plasma is integral to a global medical chain.

“There are often many misconceptions about plasma and how it is donated,” said Marcia Boyle, President & Founder of IDF. “This site will help take the mystery out of the process and hopefully increase the number of donors, Macey Holleman, daughter of IDF volunteer Ursula Holleman, which is always good for our primary immunodeficiency visited the Biomat plasma center in Macon, Georgia. Melvin community.” Young, Manager, gave her a complete tour of the facility and ex - plained every step of the plasma donation process. Macey was able Visitors can use a zip code search to find a plasma to speak to several donors and explain how their plasma donation collection center near them in the U.S., or search a country helped her. Several donors commented that they were glad to list for centers in Europe. An FAQs section will answer the know how much their time and donation helped her. Thanks most commonly asked questions about plasma donation Macey—we are proud to have you represent IDF! and the differences between blood donations for transfusion medicine in local hospitals and plasma donations used to produce therapies for rare, chronic diseases and disorders like primary immunodeficiency diseases, hemophilia and alpha-1 antitrypsin deficiency.

6 | FALL 2008 IDF Family Retreats—Spotlight on Primary Immunodeficiency

This summer, 200 adults and 80 youth from 21 states came their feet up to enjoy the beautiful settings with a barbecue from throughout the country to join IDF to put the Spotlight followed by a family friendly outdoor movie and snacks. on Primary Immunodeficiency at the IDF Family Retreats. The Gold Canyon Golf Resort in Gold Canyon, Arizona in June and IDF would like to extend special recognition to Gail Moore and the Hueston Woods Resort and Conference Center in College her dedicated team of helpers who make these family retreats Corner, Ohio in September provided beautiful backdrops for successful year after year, since 1997. The 2008 IDF Family patients, family members, healthcare professionals and Retreats were sponsored, in part, through educational grants members of industry to share knowledge, experiences and many from the American Academy of Allergy, Asthma, & laughs. Immunology, Baxter Healthcare Corporation, CSL Behring, Grifols, IgG America/ASD Healthcare, Octapharma and Talecris The Family Retreat weekends featured presentations for adults Biotherapeutics. on a wide range of topics affecting patients with primary immunodeficiency diseases, as well as a fun-filled schedule of activities for youth attendees throughout the day. Saturday night provided an opportunity for everyone to relax and put

“I love the family retreats. My 13 year old is very “My son now understands his condition. Thank you for happy while I am in classes. If she was unhappy I would educating him.” be unhappy.” “I had the most wonderful time. Everyone, and I mean “Thank you for having our family to this retreat. I feel everyone, has gone above and beyond.” I have started on a new journey.” “Beautiful setting, great family atmosphere!”

A Special Thanks to our Presenters!

Melvin Berger MD, PhD Kim Duff, RN, BSN Robert Hostoffer, Jr., DO Richard Schiff, MD Cori Daines, MD George Gwinn, MD Kim Jones Jennifer Seda, MD Michael Daines, MD Leslie Harman, MA, MFT Judi Miller John Seymour, PhD, LMFT Maggi Dodds, CPNP Terry Harville, MD, PhD Jennifer Puck, MD Lynne Szott, RN David Hauswirth, MD

ADVOCATE | 7 INDUSTRY NEWS Octapharma Agrees to Acquire 33 Talecris Biotherapeutics Helps Patients Receive Gamunex Plasma Centers Throughout the U.S. During Lapses in Private Insurance Coverage from International BioResources People with PI require IGIV on a regular basis and without regular treatments, Octapharma announced that it will acquire 33 plasma which can be disrupted in the often complex and challenging health insurance collection centers from International BioResources environment, PI patients can suffer from serious infections and illnesses. For (IBR), the largest independent source plasma collection this reason, Talecris developed the Gamunex Connexions Certificate Program, company in the United States. IBR will develop and to help ensure that patients can temporarily continue to receive their therapy staff the plasma centers with over 800 employees. The during times of uncertainty. plasma centers will be located predominantly in the East and Midwest. To qualify for the Gamunex Connexions Certificate Program, a patient must have third-party, private health insurance as their primary health insurance and Plasma is the source of numerous proteins used for be using Gamunex regularly for PI or ITP. Full details about the program are drug development and therapeutic available by calling 1-888-263-8243. purposes. Octapharma’s core business is the Excerpted from Talecris Biotherapeutics News Release October 15, 2008. development, production and sale of high quality, virus safe plasma derivatives, including IVIG. In the U.S., Grifols Breaks Ground on New IVIG Facility Octapharma’s IVIG product, octagam ®, is used to treat disorders of the immune system, and Octapharma’s Grifols announced that its new facility is anticipated to be operational in 2013 Albumin (Human) 5% is indicated for the restoration and represents an expansion of Grifols’ existing manufacturing capabilities in and maintenance of circulating blood volume. Los Angeles. Grifols’ new production facility will almost double the company’s IVIG production capacity and will utilize the same proprietary Excerpted from Octapharma’s News Release August 5, 2008. technologies and process flow designs employed at the company’s existing IVIG production facility in Barcelona, Spain. Talecris Enters into Definitive Merger Agreement with CSL Building this additional production capacity represents one more step in Grifols’ multi-year global growth plan to meet increasing patient need for Talecris Biotherapeutics, Inc. announced that it has lifesaving plasma therapies. Other elements of the long-range investment plan signed a definitive merger agreement with CSL under include expanding plasma collection capacity and additional production and which CSL has agreed to acquire Talecris for $3.1 billion testing facilities. in cash. This amount includes net debt, which as of June Excerpted from Grifols News Release, October 6, 2008. 30, 2008 was approximately $1.2 billion, implying an equity value as of that date of about $1.9 billion. Data Published in Journal Grifols Launches PediGri ® On Line for its US Plasma The combination of Talecris and CSL will result in: of Clinical Immunology Show Privigen™ Therapies - a combined company with one of the broadest portfolios in the plasma-derived therapeutics industry Demonstrates Grifols launched in the US, its with key products in each plasma therapeutic area; Effectiveness and proprietary PediGri ® On Line system that gives healthcare providers access to - one of the most robust and efficient plasma collection Tolerability in Patients quality and safety information about the capabilities in the industry, to better assure supply of with Primary plasma used in the production of Grifols’ plasma for manufacturing essential therapies; Immunodeficiencies therapies. - expanded and integrated manufacturing with greater New L-Proline Stabilized Immune efficiency and improved ability to supply therapies; Through a web portal at Globulin Intravenous (Human) 10 www.pedigrionline.net, registered - an enhanced R&D pipeline; Percent Liquid can be stored at healthcare providers (including room temperature, offering physicians, nurses and pharmacists) can - operating efficiencies that will facilitate further convenience to healthcare access lot specific information about the investment in R&D, quality, compliance and plasma professionals. individual plasma sources that collection; and contributed to each product vial. The Data published online in the - improved and more reliable supply of therapies to Grifols PediGri ® system provides full current edition of Journal of patients and customers through efficiencies, avoidance traceability from donation to final Clinical Immunology demonstrates of capacity constraints and more assured plasma supply. product. that Privigen TM , the new liquid Closing of the transaction is subject to receipt of certain immune globulin intravenous Grifols’ PediGri ® has been available in regulatory approvals, as well as other customary (human) 10 percent product from Europe for nearly a decade and will now conditions. If the necessary approvals are not obtained CSL Behring, is effective and well- be made available for all Grifols’ plasma within one year, either CSL or Talecris will have the right tolerated in patients with Primary therapies marketed and sold in the US. to terminate the transaction. Immune Deficiencies (PI). Excerpted from Grifols News Release, Excerpted from Talecris Biotherapeutics News Release Excerpted from CSL Behring News August 6th, 2008. August 12, 2008. Release September 25, 2008.

8 | FALL 2008 CLOSING DOORS ON SCID, OPENING HOPE ON TREATMENT

By: Heather Smith, IDF Volunteer and SCID Conference 2008 Coordinator weekend was an incredibly special time, providing hope, support, and, invaluable information to all who attended.

Many thanks to our excellent presenters who not only came prepared to share the most current information, but also stayed for the weekend and made themselves available to families for extended conversations. Our thanks to: Barb Ballard, Dr. Mei Baker, Dr. Rebecca Buckley, Dr. Fabio Heather Smith congratulates 14-year-old Daniel Fratto, winner Candotti, Dr. Morton Cowan, Joie Davis, Cheryl Deep, Carol Ann of the logo contest. His drawing was chosen to be the logo for Demaret, Dr. Joanna Fanos, Dr. Alan Flake, Dr. Terry Harville, Dr. Harry this years conference t-shirt. He did an excellent job! Malech, Lindsay Middelton, and most importantly, Dr. Jennifer Puck.

This past July, the SCID (Severe Combined Immune A special thanks to IDF for their generous contribution toward this Deficiency) Community hosted an international conference event. Without the help of our wonderful sponsors, this important for families who are affected or have been affected by SCID. educational event The conference was held in the beautiful Blue Ridge could not have Mountains of Virginia, at Wintergreen Resort and Spa. This been possible. is the first such event where families with ALL forms of SCID We thank you were invited to attend. I am happy to say we had over 140 and look forward people in attendance, with families coming from as far away to collaborating as California and New Zealand! The event provided a again for future wonderful opportunity for patients, families, children, conferences. physicians and industry to come together and learn more Please check out about this devastating disease, and the quality of life issues the IDF SCID that go along with successful treatment. The adult Initiative section educational sessions offered new, up-to-date information of the IDF Web from top experts in the field. The children’s programs were site for future packed with fun-filled activities for all ages, which Marcia Boyle, Barb Ballard, Carol Ann Demaret and conference emphasized teamwork, cooperation, and acceptance. The Katherine Canion at the Survivor themed dinner at the information. SCID Conference Many thanks…

The Petrelli and Walsh families “put a face on primary immunodeficiency disease” and thanked the donors and staff for their role in providing lifesaving plasma to our patient community at BioLife Plasma Services in DeKalb, Illinois.

ADVOCATE | 9 VOLUNTEERS ACROSS the COUNTRY

Susan Branch hosted “A Little Texas in Rhode Island” barbecue to benefit IDF on August 29, 2008. The successful event included a silent auction, raffle, and live music, and raised nearly $5,000. Susan, shown on the right, was assisted by IDF volunteer Betty Gordon. Thanks to Susan, Betty, and all of the other wonderful volunteers for their outstanding efforts!

These young ladies participated in the IDF Kid’s Club at the IDF Family Conference Day in St. Paul, Minnesota in July.

Allissa Rastad staffed the information table at a blood drive in honor of her brother Alec. Her mother, Towma Rastad, an IDF volunteer in Massachusetts, worked with the Red Cross to coordinate the event that did double duty as a fundraiser and helped spread awareness about primary immunodeficiency diseases and IDF.

In September, the IDF Family Conference Day in Shrewsbury, Massachusetts provided wonderful opportuni - ties for youth to con - nect through games and activities in the IDF Kid’s Club and the Teen Track Program.

10 | FALL 2008 WITH GRATITUDE

Gifts In Honor Of Gifts In Memory Of Clara Frith Josephine Rosemary Papa Joyce Barranco Mandy Abfalter Suzanne Knight Don Quicksilver Laura Bekier David Carey Marv Mareck Marsha Ralich Dr. Mary Ellen Conley Margaret Evans Kristin Anna Martin Carla Richards Noelle Drews Amy Fisher Betty McNeil Addison Trillo Brenden Denver Alli, Dan & Jamie Fillery These donations help IDF to improve the diagnosis and treatment of patients with primary immunodeficiency diseases through advocacy, education and research. If you would like to Joshua Osborn make a donation, please go on our Web site, www.primaryimmune.org and click the “please Joyce Shapiro donate today” picture in the top right corner. You can also contact us in any of the following ways: Aidan Walsh Phone: 800.296.4433 or 410.321.6647 Bill York E-mail: [email protected] Dina Farrell Mail: IDF, 40 W. Chesapeake Avenue, Suite 308, Towson, MD 21204

Small Gifts Add Up

That’s why IDF has created a new “Monthly Giving” Program. At your request, IDF can automatically charge your credit card each month. Imagine what a difference you can make to IDF by giving monthly. For example, if you contribute $25 each month, at the end of the year your total contribution to IDF will be $300! You can receive points or miles on your credit card, and you don’t have to remember to send a check. Gifts to IDF are tax-deductible, and IDF will be very grateful! All you need to do is fill out this form and send it to IDF! ¡ YOUR GIFT

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ADVOCATE | 11 IMMUNE DEFICIENCY FOUNDATION BOARD OF TRUSTEES Marcia Boyle, Chair and President Barbara Ballard, Secretary Douglas R. Bell John Boyle, PhD Rebecca H. Buckley, MD, Chair Medical Advisory Committee Lisa Codespoti Carol Ann Demaret Steve Fietek Terry Halper Robert LeBien John Seymour, PhD, LMFT, Vice Chair Jerry Winkelstein, MD

Non-Profit Org. U.S. Postage PAID Southern MD Permit No. 305 40 West Chesapeake Avenue Suite 308 Towson, MD 21204