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• W4384408377 abstract "Children with acquired hypocellular bone marrow failures of unknown cause (AHBMF) are usually diagnosed either as severe aplastic anaemia (SAA) or refractory cytopenia of Childhood (RCC). Patients with AHBMF who lack a matched donor and failed or relapsed after immunosuppressive therapy need alternative therapies. Haploidentical stem cell transplant (HAPLO) offers a curative treatment for these patients. To probe that HAPLO can be a successful therapy in paediatric patients with AHBMF and to assess therapy-related toxicity A multicentre retrospective review of AHBMF treated with HAPLO. Eleven paediatric patients (SAA 9, RCC 2) were transplanted with HAPLO with different strategies of lymphodepletion (five post-transplant cyclophosphamide, two α/β CD19 depletion, and four CD45 RA depletion). Most patients (10/11) had failed to respond or relapsed after immunosuppressive therapy. The conditioning regimen was reduced intensity in SAA and myeloablative in RCC. Patients with SAA received low-dose radiotherapy as part of the conditioning regimen. All patients engrafted. Viral reactivation was common (8/11). Acute GVHD ≥ II was seen in 5 patients. Chronic GVHD was diagnosed in 4 of the long-term survivors. Transplant-associated microangiopathy was a frequent complication in SAA patients and was related to a worse outcome. Two patients died of transplant-related complications. Overall survival was 81%, with a median follow-up of 36 months. HAPLO was a successful salvage curative treatment for paediatric patients with AHBMF, but with significant toxicities that must be addressed. Transplant-associated microangiopathy was the most critical complication." @default.
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• W4384408377 date "2023-10-01" @default.
• W4384408377 modified "2023-10-17" @default.
• W4384408377 title "Haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Patients with Acquired Hypocellular Bone Marrow Failure" @default.
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• W4384408377 doi "https://doi.org/10.1016/j.jtct.2023.07.011" @default.
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