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
Contact: Shelly Norbury, (856) 397-5588, email@example.com
ACCME Accreditation Statement: Children’s Hospital of Philadelphia is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians. Approval of AMA PRA Category 1 Credit(s) for this lecture is pending. If approved, physicians can claim CME credit commensurate with the extent of their participation in the activity for free.