Thalassemia Management Checklists: Quick Reference Guides to Reduce Disparities in the Care of Patients with Transfusion-Dependent Thalassemia

Abstract Background: The prevalence of thalassemia in the US is rising due to migration, new births, and improved survival. Advances in monitoring and treatment have significantly reduced morbidity and mortality in transfusion-dependent thalassemia (TDT), the most severe form of this inherited disease. Thalassemia Treatment Centers (TTCs) utilize a comprehensive care model to provide expert-recommended and evidence-informed treatment, but a majority of the patients with TDT are not managed at such centers owing to long travel distance and lack of insurance portability. The resulting lack of access to specialized care increases the risk of complications and reduces health-related quality of life. To address this challenge, a national project was launched to develop Thalassemia Management Checklists (TMCs), a set of quick reference guides that provide decision support to physicians for blood transfusion, iron overload and chelation therapy. Methods: Three TTC's (New York, NY, Philadelphia, PA, and Oakland, CA) collaborated on the development of the following TMCs: (1) Guidelines for Managing Transfusion therapy for Thalassemia, (2) Monitoring of Iron Overload, and (3) Monitoring Deferasirox Therapy. A comprehensive review of literature including over 600 published studies and case reports, as well as the existing expert guidelines was conducted. Utilizing relevant references, the clinical guidelines were developed and a consensus on content and design of the Checklists was achieved. Subsequently, feedback obtained from national experts and patients with thalassemia was incorporated into the final Checklists. Results: Each Checklist was divided into three sections and formatted as a quick reference guide. Part 1 was a summary table having essential information printed on one side of letter-sized paper. For transfusion therapy, the table contained actions to be triggered by the pre-transfusion hemoglobin level. For iron overload, the optimal and elevated liver and cardiac iron concentration were defined along with the frequency of iron measurement using MRI. For monitoring of deferasirox, the monitoring guidelines for adverse effects and the response to abnormal laboratory tests were presented. Part 2 consisted of a literature review and rationale for the recommendations presented in the table, which was printed on the opposite face of the page. Part 3 was a bibliography of publications cited in the literature review that was made available online with a link provided in the TMC. The final product was three separate pages each covering a single topic, allowing easy access to the summary information while displaying detailed information on demand. The TMCs were distributed as printed copies to hematologists and can be downloaded from thalassemia-related websites. Discussion: The TTC's involved with this effort recognized that physicians providing care to only a few patients with TDT within general hematology (or more commonly oncology) -focused practices are far more likely to consult a desktop quick reference guide instead of a textbook, journal or handbook of comprehensive guidelines. TMCs are expected to cover most of the routine management of TDT while encouraging consultation with TTC's for complex decisions. TMCs will form the backbone of the first national attempt to standardize the management of TDT and reduce disparities in access to and quality of care. Over the next 3 years, the adoption of TMCs and their impact on patient care will be formally evaluated in selected regions. Patient access to TMCs through online publication will increase knowledge and promote self-advocacy. We are grateful to Craig Butler and Cooley's Anemia Foundation for coordinating this project. This project is/was supported by the Health Resources and Services Administration (HRSA) of the U.S. Department of Health and Human Services (HHS) under grant number U1AMC28548: Cooperative Agreements to Support Comprehensive Medical Care for Thalassemia with no funds from non-governmental sources. This information or content and conclusions are those of the author and should not be construed as the official position or policy of, nor should any endorsements be inferred by HRSA, HHS or the U.S. Government. Disclosures Lal: Insight Magnetics: Research Funding; La Jolla Pharmaceutical Company: Consultancy, Research Funding; Novartis: Research Funding; Bluebird Bio: Research Funding; Terumo Corporation: Research Funding; Celgene Corporation: Research Funding. Sheth: Terumo Corporation: Research Funding; Novartis: Research Funding; La Jolla Pharmaceutical Company: Research Funding; Celgene Corporation: Consultancy, Research Funding; Bluebird Bio: Consultancy. Kwiatkowski: Novartis: Research Funding; Apopharma: Research Funding; bluebird bio: Consultancy, Honoraria, Research Funding; Terumo: Research Funding; Agios Pharmaceuticals: Consultancy, Research Funding.