ArticleAnnals of hematology2026
Haploidentical allogeneic haematopoietic stem cell transplantation for paroxysmal nocturnal haemoglobinuria: a retrospective analysis.
Article in Annals of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
objectiveTo evaluate the efficacy and prognostic factors of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) for paroxysmal nocturnal haemoglobinuria (PNH).
methodsWe retrospectively analyzed 36 PNH patients (2 classic PNH, 34 AA-PNH syndrome) undergoing haplo-HSCT (G-CSF/ATG-based protocol) from June 2013 to December 2024, with BU/CY/ATG (n = 28) or BU/CYlow/FLU/ATG (n = 8) conditioning and uniform GVHD prophylaxis.
resultsThe overall myeloid engraftment rate was 100% (median + 12 d) and platelet engraftment rate 95.5% (median + 13 d). Competing risk analysis showed 42.7% acute GVHD (26.7% grade II-IV, 5.3% grade III-IV); 27.3% chronic GVHD (15.2% mild, 12.1% moderate-severe) among 33 evaluable patients. With a median follow-up of 57 months, the 5-year overall survival rate was 88.6 ± 5.4%. All 32 monitored patients achieved PNH clone clearance (median 3 months), 33 had normalized blood counts, and 3 died of infection, organ hemorrhage or pulmonary embolism.
conclusionsHaplo-HSCT yields favorable outcomes and is a curative therapy for PNH, serving as a valuable option for severe bone marrow failure patients, with G-CSF/ATG-based haplo-HSCT as a promising alternative strategy.
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