Webinar - Achieving Equitable Access to BMT and Gene Therapy for Patients with Sickle Cell Disease

Live Webinar

Wednesday, February 12, 2025

3:00-4:00pm CT

Description: 

Bone marrow transplant (BMT) has over the decades offered a curative option for sickle cell disease (SCD), a multisystem disorder that leads to end-organ damage, decreased quality of life and survival. However, less than 20% of patients have a suitable HLA matched sibling donor and concerns about toxicity have limited its use. The recent FDA approval of two gene therapy (GT) products, which offer transformative effects on disease amelioration and quality of life, have added to the armamentarium of treatment options for patients. The availability of these therapies has not necessarily led to equitable access for patients. Several factors limit access to these therapies including the cost of these therapies, geographic accessibility of GT/BMT centers, concerns about toxicity and lack of data about the long-term durability of the newer gene therapy products. Healthcare providers and patients/families are challenged in decision-making about which curative/transformative therapy to select given the lack of data showing comparative effectiveness of the newer therapies compared to BMT. This webinar will address these challenges in the US while offering suggestions to ensure equitable access for patients living with SCD.

Pediatric hematologists serve as trusted sources of information for treatment options for patients with sickle cell disease and are gatekeepers for access to these therapies. Thus, it is important for pediatric hematologists to be knowledgeable about these therapies including the challenges faced by patients/families as they consider these therapies, and to have insights into their own biases, to facilitate decision-making about these therapies that is patient/family centered and offers equitable access to these therapies.

Learning Objectives:

1. Describe the current landscape in the US for access to BMT, gene therapy and editing techniques for SCD.

2. Describe patient and healthcare provider perspectives about gene therapy for SCD.

3. Describe the challenges related to achieving equitable access these therapies and suggestions for improving access.

Speakers

• Maa-Ohui Quarmyne, MBChB MS (moderator) - Phoenix Children's Hospital
• Lakshmanan Krishnamurti, MD - Yale School of Medicine
• Nitya Bakshi, MBBS MS - Yale School of Medicine
• Elizabeth Stenger, MD – Children’s Healthcare of Atlanta

To access the webinar live at the date/time above, once registered, log into your ASPHO My Library account with your ASPHO username and password, and click on the video icon: - "Access the Webinar". 

To access the webinar on-demand, once registered, log into your ASPHO My Library account with your ASPHO username and password, and click on the video icon: - "Access the Webinar".