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Stem cell transplantation for aplastic anemia and myelodysplastic syndrome

Bone Marrow Transplantation volume 35, pages S13–S16 (2005)Cite this article

Summary:

Stem cell transplantation (SCT) from a histocompatible sibling is treatment of choice for severe aplastic anemia. Survival rates have been reported to be as high as 90% for children. Immunosuppressive therapy (IST) is employed in patients who are not candidates for SCT due to donor unavailability. The addition of cyclosporin A to antilymphocyte globulin has improved the response rate to 70–80%, and survival at 5 years among responders is about 90%. In all, 30% of patients treated by IST suffer from relapse, but long-term prognosis does not appear to be affected by this complication. Juvenile myelomonocytic leukemia (JMML) shares both myelodysplastic and myeloproliferative features. Survival (10-year) of patients with JMML without SCT is only 6%. Children with JMML should be transplanted early in the course of their disease. Conditioning regimen composed of three alkylating agents, busulfan, cyclophosphamide and melphalan has been favored by the EWOG-MDS and EBMT-Pediatric WP in the second half of the 1990s. SCT using this conditioning regimen is capable of curing approximately 50% of patients with JMML. More than 70% of patients with refractory cytopenia and more than 50% of children with advanced MDS are cured of by the early performed allogeneic SCT.

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Authors and Affiliations

  1. Department of Pediatric Hematology and Oncology, University Hospital Motol, Prague, Czech Republic

    J Starý

  2. Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy

    F Locatelli

  3. Division of Pediatr Hematol/Oncol, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Germany

    C M Niemeyer

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  1. J Starý
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  2. F Locatelli
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  3. C M Niemeyer
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on behalf of the European Working Group on Myelodysplastic Syndrome (EWOG-MDS) and Pediatric Diseases Working Party of the EBMT

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Correspondence to J Starý.

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Starý, J., Locatelli, F., Niemeyer, C. et al. Stem cell transplantation for aplastic anemia and myelodysplastic syndrome. Bone Marrow Transplant 35 (Suppl 1), S13–S16 (2005). https://doi.org/10.1038/sj.bmt.1704836

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