denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Hematology Disease Topics & Pathways:
Sickle Cell Disease, Biological therapies, Diversity, Equity, and Inclusion (DEI) , Hemoglobinopathies, Diseases, Gene Therapy, Therapies
Description:
Sickle cell disease (SCD) can lead to debilitating morbidity and premature mortality. Over the past decade, 3 new drugs have been FDA-approved for treating children and adults with SCD, and many others are actively being studied. Similarly, novel curative approaches are increasingly becoming available. Unfortunately, a paucity of data exists regarding whether outcomes are improving in individuals with SCD.
Dr. Ken Ataga will discuss disease-modifying therapies that are currently FDA-approved and some of the most promising drugs that are underway.
Dr. Adetola Kassim will describe the most recent outcomes of haploidentical hematopoietic cell transplantation compared to more traditional approaches. He will also compare the pros and cons of various haploidentical platforms that are currently available.
Dr. John Tisdale will discuss the encouraging results of gene therapy and gene editing trials for patients with SCD and the remaining challenges.
Dr. Ken Ataga will discuss disease-modifying therapies that are currently FDA-approved and some of the most promising drugs that are underway.
Dr. Adetola Kassim will describe the most recent outcomes of haploidentical hematopoietic cell transplantation compared to more traditional approaches. He will also compare the pros and cons of various haploidentical platforms that are currently available.
Dr. John Tisdale will discuss the encouraging results of gene therapy and gene editing trials for patients with SCD and the remaining challenges.