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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)

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Paroxysmal Nocturnal Hemoglobinuria and Related Disorders

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Abstract

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

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References

  1. Parker CJ. Molecular basis of Paroxysmal Nocturnal Hemoglobinuria. Stem Cells 1996; 14: 396–411.

    Article  PubMed  CAS  Google Scholar 

  2. Rosse WF Ware RE. The molecular basis of paroxysmal nocturnal haemoglobinuria. Blood 1995; 86: 3277–86

    PubMed  CAS  Google Scholar 

  3. Schrezenmeier H, Hertenstein B, Wagner B, et al. A pathogenetic link between aplastic anemia and paroxysmal noctumal hemoglobinuria is suggested by a high frequency of aplastic anemia patients with a deficiency of phosphatidylinositol glycan anchored proteins. Exp Hematol 1995; 23: 81–7.

    PubMed  CAS  Google Scholar 

  4. Griscelli-Bennaucer A, Gluckman E, Scrobohaci ML, et al. Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria: Search for a Pathogenetic Link. Blood 1995; 85:1354–63.

    Google Scholar 

  5. Luzzatto L, Bessler M, Rotoli B. Somatic mutations in Paroxysmal Nocturnal Hemoglobinuria: a Blessing in Disguise?, Cell 1997; 88:1–4.

    Article  PubMed  CAS  Google Scholar 

  6. Spath-Schwalbe E, Schrezenmeier H, Heimpel SH. Paroxysmal nocturnal hemoglobinuria. Clinical experiences with 40 patients at one center over 25 years. Dtsch Med Wochenschr 1995; 28: 1027–33.

    Article  Google Scholar 

  7. Socié G, Mary J, de Gramont A, et al. Paroxysmal noctumal haemoglobinuria: longterm follow-up and prognostic factors. Lancet 1996; 348: 573–8.

    Article  PubMed  Google Scholar 

  8. Dacie JV, Lewis M. Paroxysmal nocturnal hemoglobinuria: clinical manifestation, haematology, and nature of the disease. Series Haematol 1972; 3: 3–23.

    Google Scholar 

  9. Hillmen P, Lewis SM, Bessler M, Luzzato L, et al. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995; 9: 1253–8.

    Article  Google Scholar 

  10. Fujioka S, Takayoshi T. Prognostic features of paroxysmal nocturnal hemoglobinuria in Japan. Acta Haematol JPN 1989; 52:1386–94.

    CAS  Google Scholar 

  11. Packman CH. Pathogenesis and management of paroxysmal nocturnal haemoglobinuria. Blood Rev 1998; 12(1): 1–11.

    Article  PubMed  CAS  Google Scholar 

  12. Van Kamp H, Van Imhoff, de Wolf JT, et al. The effect of cyclosporine on haematological parameters in patients with paxysmal nocturnal haemoglobinuria. Br J Haematol 1995; 89(1): 79–82.

    PubMed  Google Scholar 

  13. Stoppa AM, Vey N, Sainty D, et al. Correction of aplastic anaemia complicating paroxysmal nocturnal haemoglobinuria: absence of eradication of the PNH clone and dependence of response on cyclosporin A administration. Br J Haematol 1996; 93(1): 42–4.

    Article  PubMed  CAS  Google Scholar 

  14. Schubert J, Scholz C, Geissler RG, et al. G-CSF and cyclosporin induce an increase of normal cells in hypoplastic paroxysmal nocturnal hemoglobinuria. Ann Hematol 1997; 74: 225–30.

    Article  PubMed  CAS  Google Scholar 

  15. Paquette RL, Yoshimura R, Veiseh C, et al. Clinical characteristics predict response to anthithymocyte globulin in paroxysmal nocturnal haemoglobinuria. Br J Haematol 1997; 96: 92–7.

    Article  PubMed  CAS  Google Scholar 

  16. Szer J, Deeg HJ, Witherspoon RP, et al. Long-term survival after marrow transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia. Ann Intern med 1984; 101: 193–5.

    PubMed  CAS  Google Scholar 

  17. Antin JH, Ginsburg D, Smith BR, et al. Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria: eradication of the PNH clone and documentation of complete lymphohematopoietic engraftment. Blood 1985; 66:1247–50.

    PubMed  CAS  Google Scholar 

  18. Kolb HJ, Holler E, Bender-Gotze, Walther U. Myeloablative conditioning for marrow transplantation in myelodysplastic syndromes and paroxysmal nocturnal haemoglobinuria. Bone Marrow Transplant 1989; 4: 29–34.

    PubMed  CAS  Google Scholar 

  19. Kawahara K, Witherspoon SP, Storb R. Marrow transplantation for paroxysmal nocturnal hemoglobinuria. Am J Hematol 1992; 39: 283–8.

    Article  PubMed  CAS  Google Scholar 

  20. Bemba M, Guardiola P, Garderet et al. Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria. Br J Haematol 1999, 105: 366–8.

    Article  PubMed  CAS  Google Scholar 

  21. Saso R, Marsh J, Cevreska L et al. Bone marrow transplants for paroxysmal nocturnal haemoglobinuria., Br J Haematol 1999, 104: 392–6

    Article  PubMed  CAS  Google Scholar 

  22. Locasciulli A. Hepatitis viruses infections in bone marrow transplantation. Bone Marrow Transplant 1996; 18 Suppl 2: 115–6.

    PubMed  Google Scholar 

  23. Araten DJ, Nafa K, Pakdeesuwan K, Luzzatto L. Clonal populations of hematopoietic cells with paroxysmal nocturnal hemoglobinuria genotype and phenotype are present in normal individuals, Proc Natl Acad Sci USA 1999, 27: 5209–14

    Article  Google Scholar 

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Authors and Affiliations

  1. Dipartimento di Ematologia e Oncologia, Ospedale San Martino, Genova, Italy

    Andrea Bacigalupo, Rosi Oneto & Barbara Bruno

Authors
  1. Andrea Bacigalupo
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  2. Rosi Oneto
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  3. Barbara Bruno
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Hubert Schrezenmeier on behalf of the working party on severe aplastic anemia (WPSAA) of the European Blood and Marrow Transplantation group (EBMT)

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Editors and Affiliations

  1. Division of Hematology, Internal Medicine Showa University Fujigaoka Hospital, 1-30 Fujigaoka, Aoba-ku, Yokohama, 227-8501, Japan

    Mitsuhiro Omine M.D. (Professor) (Professor)

  2. Department of Immunoregulation, Research Institute for Microbial Diseases Osaka University, 3-1 Yamadaoka, Suita, Osaka, 565-0871, Japan

    Taroh Kinoshita Ph.D. (Professor) (Professor)

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© 2003 Springer Japan

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Bacigalupo, A., Oneto, R., Bruno, B., Hubert Schrezenmeier on behalf of the working party on severe aplastic anemia (WPSAA) of the European Blood and Marrow Transplantation group (EBMT). (2003). 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). In: Omine, M., Kinoshita, T. (eds) Paroxysmal Nocturnal Hemoglobinuria and Related Disorders. Springer, Tokyo. https://doi.org/10.1007/978-4-431-67867-0_18

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  • DOI: https://doi.org/10.1007/978-4-431-67867-0_18

  • Publisher Name: Springer, Tokyo

  • Print ISBN: 978-4-431-68004-8

  • Online ISBN: 978-4-431-67867-0

  • eBook Packages: Springer Book Archive

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