Haploidentical allogeneic haematopoietic stem cell transplantation for paroxysmal nocturnal haemoglobinuria: a retrospective analysis.

OBJECTIVE

To evaluate the efficacy and prognostic factors of haploidentical haematopoietic stem cell transplantation (haplo-HSCT) for paroxysmal nocturnal haemoglobinuria (PNH).

METHODS

We 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.

RESULTS

The 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.

CONCLUSIONS

Haplo-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.