Françoise Bernaudin (Intercommunal Hospital of Créteil, Créteil, France) joins touchHAEMATOLOGY to discuss the main challenges in implementing alloSCT as a treatment for sickle cell anaemia in children, particularly with significant disparities in access to curative treatments like bone marrow transplantation in third-world countries. While newborn screening identifies around 600 cases annually in regions like the USA and Europe, the lack of sufficient transplant units limits access to life-saving procedures. In Africa, where early mortality rates are alarmingly high, progress is evident with successful transplants in Nigeria and Tanzania, however, the numbers remain low. Despite advancements like hydroxyurea, curative options are crucial. Expanding transplant units and leveraging Africa’s high sibling donor match rates could offer cost-effective, life-saving solutions for affected children globally.
The Late-Breaking Abstract, ‘Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities’ (https://ash.confex.com/ash/2024/webprogram/Paper212967.html) was presented at the 66th ASH Annual Meeting & Exposition on 7–10 December 2024 in San Diego, CA, USA.
Question: What do you see as the main challenges in implementing alloSCT as a treatment for sickle cell anemia in children, and how could these challenges be overcome?
Click here to view more from Francois Bernaudin on the DREPAGREFFE-1 trial: Comparing stem cell transplantation and standard-of-care in paediatric sickle cell anaemia
Disclosures: Françoise Bernaudin is a consultant for Bluebirdbio, Vertex, GBT/Pfizer and Terumo; she has received grant/research support from, and is on the Advisory Board for, GBT/Pfizer, and has received honoraria/honorarium from AddMedica and Terumo.
This content has been developed independently by Touch Medical Media for touchHAEMATOLOGY. It is not affiliated with the American Society of Hematology (ASH). Views expressed are the speaker’s own and do not necessarily reflect the views of Touch Medical Media.
Cite: Bernaudin F. Bridging the gap in paediatric sickle cell care: Expanding transplant access globally. touchHAEMATOLOGY. 22 January, 2025.
