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.

Starý, J., Locatelli, F., Niemeyer, C. M., Stem cell transplantation for aplastic anemia and myelodysplastic syndrome, <<BONE MARROW TRANSPLANTATION>>, 2005; 35 Suppl 1 (S1): 13-16. [doi:10.1038/sj.bmt.1704836] [https://hdl.handle.net/10807/259996]

Stem cell transplantation for aplastic anemia and myelodysplastic syndrome

Locatelli, Franco;
2005

Abstract

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.
2005
Inglese
Starý, J., Locatelli, F., Niemeyer, C. M., Stem cell transplantation for aplastic anemia and myelodysplastic syndrome, <<BONE MARROW TRANSPLANTATION>>, 2005; 35 Suppl 1 (S1): 13-16. [doi:10.1038/sj.bmt.1704836] [https://hdl.handle.net/10807/259996]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/259996
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