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.
| Original language | English |
|---|---|
| Journal | Bone Marrow Transplantation |
| Volume | 35 |
| Issue number | SUPPL. 1 |
| DOIs | |
| Publication status | Published - Mar 2005 |
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Keywords
- Allogeneic stem cell transplantation
- Children
- Juvenile myelomonocytic leukemia
- Myelodysplastic syndrome
- Severe aplastic anemia
ASJC Scopus subject areas
- Hematology
- Transplantation
Cite this
Stem cell transplantation for aplastic anemia and myelodysplastic syndrome. / Starý, Jan; Locatelli, F.; Niemeyer, C. M.
In: Bone Marrow Transplantation, Vol. 35, No. SUPPL. 1, 03.2005.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Stem cell transplantation for aplastic anemia and myelodysplastic syndrome
AU - Starý, Jan
AU - Locatelli, F.
AU - Niemeyer, C. M.
PY - 2005/3
Y1 - 2005/3
N2 - 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.
AB - 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.
KW - Allogeneic stem cell transplantation
KW - Children
KW - Juvenile myelomonocytic leukemia
KW - Myelodysplastic syndrome
KW - Severe aplastic anemia
UR - http://www.scopus.com/inward/record.url?scp=18144380380&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=18144380380&partnerID=8YFLogxK
U2 - 10.1038/sj.bmt.1704836
DO - 10.1038/sj.bmt.1704836
M3 - Article
VL - 35
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
SN - 0268-3369
IS - SUPPL. 1
ER -