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New developments in the management of sickle cell disease

Alexis A. Thompson

Abstract

Sickle cell disease (SCD) is frequently called the first molecular disease, and scientific discoveries in the hemoglobinopathies have served as guideposts for advancing many fields of study and medical breakthroughs. Today, it is clear that SCD is far more complex a condition than initially assumed scientifically, and the multisystem clinical manifestations have proved challenging for both patients and clinicians. Although more of the underlying pathophysiology of SCD has been elucidated, progress in medical management has not kept apace. The state of SCD, focusing on the role of the American Society of Hematology (ASH), is discussed in this presentation. ASH has a multifaceted agenda to enhance research and improve patient outcomes in SCD in the United States and globally. The ASH Research Priorities for SCD and sickle cell trait highlight many opportunities for the greatest impact. ASH has also taken a leadership role in calling for increased attention by other national and international organizations in its ASH SCD: A Call to Action. More recently, ASH has supported the launch of a SCD coalition to coordinate efforts by ASH and other stakeholders to conquer SCD and improve outcomes through research and accessible high-quality care.

Correspondence: Alexis A. Thompson, Ann & Robert H. Lurie Children's Hospital of Chicago/Northwestern University, 225 E Chicago Ave, Box 30, Chicago, IL 60611; e-mail: a-thompson{at}northwestern.edu.

Footnotes

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  • The complete text of this Blood Advances Talk is available as a data supplement.

  • Contribution: A.A.T. wrote the manuscript and is the speaker in the audio version of this Blood Advances Talk.

  • Conflict-of-interest disclosure: A.A.T. receives research funding from the following companies: Amgen, Baxalta, bluebird bio, Celgene, Mast, Novartis. She has been a consultant for ApoPharma, Novartis, and bluebird bio. She does not participate in speaker's bureaus, has no owner equity, and receives no royalties from any entity.