What is the efficacy of stem cell transplantation for the treatment of paroxysmal nocturnal hemoglobinuria (PNH)?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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An analysis by the International PNH Interest Group reviewed data from 67 patients from single centers and from teo registry studies, with special emphasis in eliminating duplication in patient reporting. [22] Results included the following:

  • Of the seven patients transplanted from a twin syngeneic donor, the four who had no conditioning therapy either failed to engraft or relapsed after transplantation, indicating that a marrow ablative conditioning is necessary before syngeneic transplantation.

  • In 47 of 67 patients, a human leukocyte antigen (HLA)-identical sibling was used as the donor, 1 from a haploidentical family member and 12 from an unrelated donor (matched unrelated donor [MUD]).

  • In the only single-center study providing a Kaplan-Meier analysis, overall survival at 5 years was 58 +/- 13%. This is less favorable than the survival estimate of approximately 75% generated by combining the data from the other reports.

  • Investigation is currently in progress regarding whether reduced-intensity conditioning can improve the outcome.

A retrospective study of 21 PNH patients who underwent HSCT after previous treatment with eculizumab found that HSCT was associated with almost 30% mortality, mainly due to infections and acute graft-versus-host disease (GvHD). Syngeneic HSCT transplants were well tolerated. The authors suggested that these results may call into question the role of HSCT for patients with classic PNH who continue to require transfusions despite eculizumab, in absence of a syngeneic donor. [62]

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