title: Allogeneic bone marrow transplantation for chronic myelomonocytic leukemia in childhood: a report from the European Working Group on Myelodysplastic Syndrome in Childhood.
abstract: OBJECTIVE
  To evaluate the role of allogeneic bone marrow transplantation (BMT) in children with chronic myelomonocytic leukemia (CMML).
  
  
  METHODS
  Forty-three children with CMML given BMT and reported to the European Working Group on Myelodysplastic Syndrome in Childhood (EWOG-MDS) data base were evaluated. In 25 cases, the donor was a human leukocyte antigen (HLA)-identical or a one-antigen-disparate relative, in four cases a mismatched family donor, and in 14 a matched unrelated donor (MUD). Conditioning regimens consisted of total-body irradiation (TBI) and chemotherapy in 22 patients, whereas busulfan (Bu) with other cytotoxic drugs was used in the remaining patients.
  
  
  RESULTS
  Six of 43 patients (14%), five of whom received transplants from alternative donors, failed to engraft. There was a significant difference in the incidences of chronic graft-versus-host disease (GVHD) between children transplanted from compatible/one-antigen-mismatched relatives and from alternative donors (23% and 87%, respectively; P < .005). Probabilities of transplant-related mortality for children given BMT from HLA-identical/one-antigen-disparate relatives or from MUD/ mismatched relatives were 9% and 46%, respectively. The probability of relapse for the entire group was 58%, whereas the 5-year event-free survival (EFS) rate was 31%. The EFS rate for children given BMT from an HLA-identical sibling or one-antigen-disparate relative was 38%. In this latter group, patients who received Bu had a better EFS compared with those given TBI (62% v 11%, P < .01).
  
  
  CONCLUSIONS
  Children with CMML and an HLA-compatible relative should be transplanted as early as possible. Improvement of donor selection, GVHD prophylaxis, and supportive care are needed to ameliorate results of BMT from alternative donors.
authors.affiliation: Department of Pediatrics, University of Pavia, Instituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Italy. tmoped@ipv36.unipv.it
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: C
authors.initials: C
authors.lastname: Niemeyer
authors.forename: E
authors.initials: E
authors.lastname: Angelucci
authors.forename: C
authors.initials: C
authors.lastname: Bender-Götze
authors.forename: S
authors.initials: S
authors.lastname: Burdach
authors.forename: W
authors.initials: W
authors.lastname: Ebell
authors.forename: W
authors.initials: W
authors.lastname: Friedrich
authors.forename: H
authors.initials: H
authors.lastname: Hasle
authors.forename: J
authors.initials: J
authors.lastname: Hermann
authors.forename: N
authors.initials: N
authors.lastname: Jacobsen
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.forename: B
authors.initials: B
authors.lastname: Kremens
authors.forename: G
authors.initials: G
authors.lastname: Mann
authors.forename: A
authors.initials: A
authors.lastname: Pession
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: H J
authors.initials: HJ
authors.lastname: Schmid
authors.forename: J
authors.initials: J
authors.lastname: Stary
authors.forename: M
authors.initials: M
authors.lastname: Suttorp
authors.forename: C
authors.initials: C
authors.lastname: Uderzo
authors.forename: E T
authors.initials: ET
authors.lastname: van't Veer-Korthof
authors.forename: J
authors.initials: J
authors.lastname: Vossen
authors.forename: M
authors.initials: M
authors.lastname: Zecca
authors.forename: M
authors.initials: M
authors.lastname: Zimmermann
mesh_terms: D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006680:HLA Antigens; D006801:Humans; D007223:Infant; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000628:therapy*; D008297:Male; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens