Operationalizing Delivery of Gene Therapy for Sickle Cell Disease

Speaker(s)

• 

  Stella Chou, MD (she/her/hers)

  Chief, Division of Transfusion Medicine
  Children's Hospital of Philadelphia, University of Pennsylvania
  Philadelphia, Pennsylvania, United States

  Disclosure(s): Pfizer, Inc. Common Stock: Consultant/Advisory Board (Ongoing)

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• 

  Yan Zheng, MD/PhD (she/her/hers)

  Associate Member
  St. Jude Children's Research Hospital
  Memphis, Tennessee, United States

  Disclosure(s): No financial relationships to disclose

  PDF
• JK

  Janet Kwiatkowski, MD, MSCE

  Director
  Thalassemia Center
  CHOP

  Disclosure(s): Agios Pharmaceuticals: Consultant/Advisory Board (Ongoing), Grant/Research Support (Ongoing); Bluebird Bio: Consultant/Advisory Board (Ongoing), Grant/Research Support (Ongoing); Celgene/Bristol Myers Squibb: Consultant/Advisory Board (Terminated); Chiesi: Consultant/Advisory Board (Ongoing); Editas Medicine: Grant/Research Support (Ongoing); Imara: Consultant/Advisory Board (Terminated), Grant/Research Support (Terminated); NovoNordisk/Forma: Consultant/Advisory Board (Ongoing), Grant/Research Support (Ongoing); Pfizer: Consultant/Advisory Board (Terminated); Regeneron: Consultant/Advisory Board (Terminated); Sangamo Therapeutics: Grant/Research Support (Terminated); Vertex Pharmaceuticals: Consultant/Advisory Board (Ongoing), Grant/Research Support (Ongoing)

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Program Chair(s)

• RM

  Ryan Metcalf, MD

  Section Chief, Transfusion Medicine
  ARUP Labs and Utah School of Medicine
  Salt Lake City, Utah, United States

  Disclosure(s): Werfen/Instrumentation Laboratory: Consultant/Advisory Board (Ongoing)

  PDF

This session focuses on new FDA-approved gene therapies for sickle cell disease (SCD) and the critical aspects of coordinating care for gene therapy, including an approach to transfusion support and maximizing stem cell collections. With recent advancements in gene therapy offering a cure for SCD, effective coordination among multidisciplinary healthcare teams is essential to ensure optimal patient outcomes. Barriers to offering gene therapy for SCD may include alloimmunization to multiple antigens and an inability to collect sufficient autologous hematopoietic stem cells. The session will delve into the details of patient selection, pre-treatment assessments, treatment protocols, and coordination of complex care. Participants will have the opportunity to engage in discussions, case presentations, and a Q&A session with experts in the field of sickle cell disease, gene therapy and transfusion support. The session aims to share best practices and practical strategies for integrating gene therapy into the care plan for patients with SCD at both adult and pediatric institutions.

CABP CE Eligible