For decades, treatment options for patients suffering from chronic, severe manifestations of sickle cell disease have been limited to hydroxyurea therapy and chronic red blood cell transfusions. Recently however, an explosion of targeted agents and curative stem cell therapies have emerged that hold the promise of transforming the paradigm of how we manage sickle cell disease. In this lecture, we will provide an overview for general practitioners and hematologists in which we review limitations of current therapies for sickle cell disease, pivotal data leading to the emergence of novel therapies, and approaches to deciding which therapies are optimal for individual patients.
- Understand the approach to current long-term disease-modifying therapies for patients with SCD
- Review the evolution of allogeneic stem cell transplantation for patients with SCD
- Review current clinical trial approaches for stem cell-based gene therapy for patients with SCD, and how these approaches compare with allogeneic stem cell transplantation.
- Discuss the referral process for stem cell transplantation for patients with SCD
This seminar focuses on hematology and was delivered at a virtual event titled, Curative and Emerging Therapies for Sickle Cell Disease, on January 15, 2021.
Additional CHOP Resources:
- Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)
- Upcoming Healthcare Professionals Events
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