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 Nature Publishing Group.

Stem cell transplantation for aplastic anemia and myelodysplastic syndrome / Stary, J.; Locatelli, F.; Niemeyer, C. M.. - In: BONE MARROW TRANSPLANTATION. - ISSN 0268-3369. - 35:(2005), pp. S13-S16. [10.1038/sj.bmt.1704836]

Stem cell transplantation for aplastic anemia and myelodysplastic syndrome

Locatelli F.;
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 Nature Publishing Group.
2005
Allogeneic stem cell transplantation; Children; Juvenile myelomonocytic leukemia; Myelodysplastic syndrome; Severe aplastic anemia
01 Pubblicazione su rivista::01a Articolo in rivista
Stem cell transplantation for aplastic anemia and myelodysplastic syndrome / Stary, J.; Locatelli, F.; Niemeyer, C. M.. - In: BONE MARROW TRANSPLANTATION. - ISSN 0268-3369. - 35:(2005), pp. S13-S16. [10.1038/sj.bmt.1704836]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1479906
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