Focus of the Report: The focus of this report is on allogeneic hematopoietic stem cell transplantation (HSCT) utilizing alternative human leukocyte antigen (HLA)-matched donor sources in pediatric patients with sickle cell disease (SCD) without an HLA-matched sibling donor (MSD).
Technology Description: Allogeneic HSCT is the most successful curative treatment for SCD, but its use is limited primarily due to the lack of matched donors and concerns for transplant-related toxicities, including death. The ideal transplantation source for HSCT is with an HLA- MSD. However, use of alternative donor sources including haploidentical donor relatives (HID), HLA-matched unrelated donors (MUD), and cord blood transplant (CBT) is being increasingly reported in the peer-reviewed literature.
Controversy: Allogeneic HSCT is the most successful potentially curative treatment for SCD, but its use is limited because of the lack of donors, concerns for transplant-related toxicities (including death), and preferences of the patient and family. Additional controversy may arise once gene editing strategies are available as an alternative potentially curative treatment outside of a research setting for SCD patients who lack an HLA-MSD. Questions remain regarding the optimal conditioning regimen, graft-versus-host disease (GvHD) prophylaxis, and timing of the transplant for achieving improved efficacy and safety. The potential benefits of allogeneic HSCT for a young patient experiencing or at risk of serious complications due to SCD must be weighed against the risks of the procedure, particularly if there is no MSD.
Key Questions:
Is MUD or HID HSCT effective in treating patients with SCD?
How does MUD or HID HSCT compare with other treatments for SCD?
Is MUD or HID HSCT safe in patients with SCD?
Have definitive patient selection criteria been identified for MUD or HID HSCT in patients with SCD?
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