As the Executive Director of the Diamond Blackfan Anemia Foundation (DBAF), it is both my professional responsibility and personal passion to fulfill the DBAF’s Mission Statement:
The mission of the Diamond Blackfan Anemia Foundation is to advance research initiatives that promote a better understanding, therapeutic strategies and a cure for this rare bone marrow failure syndrome. We are dedicated to providing patient advocacy, support and education services to individuals, families and medical professionals resulting in improvements in the diagnosis, clinical treatment and management of the disorder, while enhancing the quality of life of affected families worldwide.
We are grateful to the many Diamond Blackfan Anemia (DBA) patients and families who have partnered with us to support our efforts. A recent article in People Magazine, “Four Brothers, One Deadly Disease,” written by reporter, Caitlin Keating, featured one of the many extraordinary families whose lives have been touched by Diamond Blackfan Anemia. We acknowledge that the article has brought attention to the rare, and occasionally life-limiting blood disorder and suspect that the author of this piece was well-intentioned. The family’s love for their sons is both palpable and heartwarming. Their personal journey and their dedication to recruiting bone marrow donors are applauded and appreciated.
Of deep concern to the DBAF, several of our DBA families, and numerous clinicians and researchers is the outdated or inferred misinformation reported in the article. Jeffrey Lipton, MD, Ph.D., Director-Hematology/Oncology and Stem Cell Transplantation, Cohen Children’s Medical Center of New York; Professor-The Center for Autoimmune Musculoskeletal and Hematopoietic Diseases, Feinstein Institute for Medical Research; Professor of Pediatrics and Molecular Medicine, Zucker School of Medicine at Hofstra/Northwell; Co-Director-Diamond Blackfan Anemia Registry (DBAR) stated, “Extrapolating from one family’s experience can be very misleading. The life expectancy for any patient with Diamond Blackfan Anemia depends upon myriad factors – so what is true for this family cannot, and should not, be interpreted as true for all. While some affected individuals have a markedly shortened life expectancy, the majority will live well past 30 years of age.”
Untreated iron overload resulting from chronic blood transfusions does threaten our patients’ health with complications from iron accumulation and can result in early death. However, the vast majority of patients do respond to iron chelating agents that can significantly reduce the iron burden and toxicity to major organs. Many adult patients are able to manage transfusional iron overload and live well beyond their third decade without the need for a stem cell transplant. Additional misstatements cited in the article includes information regarding the number of worldwide cases, the inferred inheritance pattern of DBA, and the omission of the potential risks and limitations of treatment options, including stem cell transplantation. There is no cure for all aspects of Diamond Blackfan Anemia.
The DBAF and our families are encouraged by the advances that have been made and we hold hope for the future. We are proud of our families’ tireless fundraising efforts that provide much needed funding for numerous research projects. We are motivated by the research being conducted and the worldwide collaborations that have been established to facilitate international exchange and foster progress in many areas of DBA research. At the recent 15th Diamond Blackfan Anemia International Consensus Conference, hosted by the DBAF in March 2018, a Senior Investigator at the National Human Genome Research Institute, David Bodine, Ph.D. stated, “We have Diamond Blackfan Anemia surrounded.” Research projects, along with current clinical trials, are aiming to provide a better understanding of DBA, effective surveillance and management of DBA and treatment related complications, novel approaches to less toxic treatments, and promising advances toward successful therapeutic cures. Indeed, there has been remarkable progress and there is hope!
Furthermore, the DBAF is proud to support Diamond Blackfan Anemia patients and families and be a resource for factual, up-to-date information. We understand that the inaccuracies in this poorly researched article have caused unnecessary anxiety and we welcome you to reach out to us. Please feel free to contact me at firstname.lastname@example.org. Additional information can be found on the Diamond Blackfan Anemia Registry’s website at www.DBAR.org.
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Dawn M. Baumgardner
Diamond Blackfan Anemia Foundation