Hemopoietic Stem Cell Transplantation (HSCT) for Paroxysmal Nocturnal Hemoglobinuria (PNH) and Acquired Severe Aplastic Anemia (SAA): a report from the SAA Working Party of the European Blood and Marrow Transplant group (EBMT)

  • Andrea Bacigalupo
  • Rosi Oneto
  • Barbara Bruno
  • Hubert Schrezenmeier on behalf of the working party on severe aplastic anemia (WPSAA) of the European Blood and Marrow Transplantation group (EBMT)
Conference paper


Background and Objective. Severe aplastic anemia (SAA) and paroxysmal nocturnal haemoglobinuria (PNH) share many clinical and possibly pathogenetic features Allogeneic hemopoietic stem cell transplantatin (HSCT) is a curative treatment for both disorders. In the present study we compare the outcome of PNH and SAA patients allografted in Europe in the past 3 decades.

Patients. The patient population consisted of 2678 patients with severe aplastic anemia (SAA) and of 90 patients with PNH and reported to the EBMT. The donor was an HLA identical sibling in 2117 SAA and 74 PNH patients respectively, an identical twin in 45 and 0, a mismatched family of matched unrelated donor in 514 and 882 for PNH (P>0.0001) and the median age of patients 18 and 29 respectvely (p>0.0001). Radiation was used in the conditioning regimen in 19% and 21% of patients (p=0.5); the source of stem cells was bone marrow in 92% and 86% respectively (p=0.04).

Results. The overall actuarial survival for HLA identical sibling HSCT 10 years is 62% in SAA vs 58% in PNH (p=0.09). Patients with PNH had more acute (p>0.002) and more chronic GvHD (p=0.04) as compared to SAA patients, also when adjusted for age and excluding radiation based conditioning. There is a strong overall effect of patient age, interval diagnosis-HSCT and year of HSCT on outcome: older age is a significant negative predictor in both SAA and PNH, whereas year of HSCT and duration of the disease appears to be significant only in SAA.

The small number of PNH patients undergoing an alternative donor transplant (n=16) did relatively well with 12 patients surviving. Conclusion. This study confirms the feasibility of HSCt in patients with PNH. When compard to the SAA patients, there are differences in patient characteristics, disease duration and outcome of the transplant. Patients receiving a non radiation based conditioning and bone marrow as the source of stem cells, seem to be doing better


Conditioning Regimen Graft Versus Host Disease Paroxysmal Nocturnal Haemoglobinuria Unrelated Donor Severe Aplastic Anemia 
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Copyright information

© Springer Japan 2003

Authors and Affiliations

  • Andrea Bacigalupo
    • 1
  • Rosi Oneto
    • 1
  • Barbara Bruno
    • 1
  • Hubert Schrezenmeier on behalf of the working party on severe aplastic anemia (WPSAA) of the European Blood and Marrow Transplantation group (EBMT)
  1. 1.Dipartimento di Ematologia e OncologiaOspedale San MartinoGenovaItaly

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