Stem cell transplantation for aplastic anemia and myelodysplastic syndrome

Jan Starý, F. Locatelli, C. M. Niemeyer

Research output: Contribution to journalArticle

27 Citations (Scopus)

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 languageEnglish
JournalBone Marrow Transplantation
Volume35
Issue numberSUPPL. 1
DOIs
Publication statusPublished - Mar 2005

Fingerprint

Juvenile Myelomonocytic Leukemia
Aplastic Anemia
Myelodysplastic Syndromes
Stem Cell Transplantation
Immunosuppressive Agents
Transplantation Conditioning
Busulfan
Melphalan
Antilymphocyte Serum
Survival
Alkylating Agents
Cyclophosphamide
Cyclosporine
Siblings
Therapeutics
Survival Rate
Tissue Donors
Pediatrics
Recurrence

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 journalArticle

@article{89c395b7677b46d19d4fe0b9142b5b45,
title = "Stem cell transplantation for aplastic anemia and myelodysplastic syndrome",
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.",
keywords = "Allogeneic stem cell transplantation, Children, Juvenile myelomonocytic leukemia, Myelodysplastic syndrome, Severe aplastic anemia",
author = "Jan Star{\'y} and F. Locatelli and Niemeyer, {C. M.}",
year = "2005",
month = "3",
doi = "10.1038/sj.bmt.1704836",
language = "English",
volume = "35",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Nature Publishing Group",
number = "SUPPL. 1",

}

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 -