Unmanipulated haploidentical donor and matched unrelated donor hematopoietic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria: a single-center study

We analyzed the outcomes of 32 patients with paroxysmal nocturnal hemoglobinuria (PNH) who underwent either a haploidentical donor (HID) or a matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT). Seventeen patients received an HSCT from an HID and 15 patients received an HSCT from an MUD. The median follow-up time of the surviving patients was 36 months (range: 12-96 months). No significant differences were observed in the 3-year overall survival (OS) between the HID and MUD cohorts (74.1%+/- 11.4% vs. 93.3%+/- 6.4%, respectively, p=.222) or in the 3-year failure-free survival (68.8%+/- 11.8% vs. 86.7%+/- 8.8%, respectively, p=.307). Treatment-related mortality occurred in five patients. A univariate analysis of risk factors revealed platelet engraftment failure negatively impacted OS and FFS. We conclude that HID and MUD-HSCT are feasible and can be effective options for those PNH patients with concomitant bone marrow failure, recurrent life-threatening thrombosis, and uncontrollable hemolysis.

Automated summary

A study on 32 PNH patients undergoing HID or MUD-HSCT showed no significant differences in 3-year OS and FFS between the two groups, but treatment-related mortality had a negative impact on outcomes. HID and MUD-HSCT are feasible and effective options for PNH patients with certain complications.