What is the role of hematopoietic stem cell transplantation in 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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Answer

The International Bone Marrow Transplant Registry (IBMTR) reported a 2-year survival probability of 56% in 48 recipients of HLA-identical sibling transplants between 1978 and 1995. [61] Data using nonmyeloablative conditioning and haploidentical donors was similar to the identical donors, indicating some form of graft-versus-PNH effects. Now that an effective, nontransplant therapy is available, the use of allogeneic HSCT to treat PNH has decreased.

Before the introduction of eculizumab, PNH patients with severe symptoms from classic PNH and patients with AA/PNH with peripheral cytopenias meeting criteria for severe aplastic anemia were considered good candidates for allogeneic bone marrow transplantation, especially if a matched sibling donor was available.

With eculizumab for PNH, the indications for allogeneic HSCT in this setting have changed. First, HSCT should not be offered as initial therapy for most patients with classic PNH, given the high transplant-related mortality, especially when using unrelated or mismatched donors. Exceptions are PNH patients in countries where eculizumab is not available. HSCT is also a reasonable option for patients who do not have a good response to eculizumab therapy. Second, aplastic anemia/PNH patients continue to be reasonable candidates for HSCT if they have life-threatening cytopenias. [29]


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