Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases - PubMed
3 hours ago
- #rare diseases
- #haploidentical
- #stem cell transplantation
- Haploidentical stem cell transplantation (haplo-HSCT) expands donor availability using partially matched family donors.
- Transplant platforms like TCRαβ+/CD19+ depletion, Beijing protocol, and Baltimore protocol show survival outcomes comparable to matched sibling donor transplantation.
- Haplo-HSCT is increasingly used for rare hematological, metabolic, and immunological disorders, where donor shortages and delayed treatment worsen prognosis.
- Gene therapy alternatives face limitations in cost, accessibility, and safety, making haplo-HSCT a crucial option.
- The review compares outcomes across transplant platforms, discusses disease-specific adaptations, and suggests future directions to improve safety and applicability.