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:
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Is MUD or HID HSCT effective in treating patients with SCD?
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How does MUD or HID HSCT compare with other treatments for SCD?
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Is MUD or HID HSCT safe in patients with SCD?
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Have definitive patient selection criteria been identified for MUD or HID HSCT in patients with SCD?
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