title: Unrelated-donor bone marrow transplantation for Philadelphia chromosome-positive chronic myelogenous leukemia in children: experience of eight European Countries. The EBMT Paediatric Diseases Working Party.
abstract: From September 1988 to December 1995 forty-four children (age < 17 years) with Ph1 Chronic Myelogenous Leukemia (CML) received unrelated donor marrow transplantation in 8 European Countries. Thirty-three evaluable children were typed by serological testing on HLA-A, B, and DR loci. Thirty of them were further DR subtyped by DNA techniques. Twenty-four pairs were 6 antigen matched. Seven were mismatched at 1 locus (2 pairs at A and B loci respectively and 3 at DR locus). Two out of 30 pairs evaluated by molecular biology had one antigen mismatched at DRB1 locus. Thirty-two (96%) out of 33 evaluable children reached a sustained granulocyte count higher than 0.5 x 10(9)/l at a median of 21 (range 14-88) days after transplantation. The remaining child failed to engraft. Two children developed secondary graft failure. A platelet count greater than 50 x 10(9)/l sustained for at least seven consecutive days without transfusion support was reached at a median of 25 (range: 20-144) days by 24 out of 33 evaluable children and 9 children never recovered to above 50 x 10(9)/l. Twenty-one out of 33 evaluable children developed grade I (n = 7), grade II (n = 8), grade III (n = 2) or grade IV (n = 4) acute GvHD (63%). Seven of the 30 evaluable children surviving more than 100 days developed chronic GvHD (20%) which was limited in 4 cases and extensive in 3. Relapse occurred in 3 of the 44 (7%) children at 2 to 24 months (median 14). Twenty-four month relapse rate was 14%. Seventeen out of 44 children (38%) died of transplant related mortality (TRM), 4 (9%) of secondary tumor, 4 (9%) of infections, 3 (7%) of organ failure, 1 (2%) of interstitial pneumonia, 5 (11%) of unknown causes. Actuarial TRM was 61% for children grafted before December 1991 and 33% for children grafted after January 1992 (p = .01). EFS was 49.7%; it was 65% for children receiving more than 3.5 x 10(9)/Kg MNC.
authors.affiliation: Bone Marrow Transplant Unit, Istituto Giannina Gaslini, Genova, Italy.
authors.forename: G
authors.initials: G
authors.lastname: Dini
authors.forename: R
authors.initials: R
authors.lastname: Rondelli
authors.forename: M
authors.initials: M
authors.lastname: Miano
authors.forename: J
authors.initials: J
authors.lastname: Vossen
authors.forename: E
authors.initials: E
authors.lastname: Gluckman
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: P
authors.initials: P
authors.lastname: Bordigoni
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: R
authors.initials: R
authors.lastname: Miniero
authors.forename: P
authors.initials: P
authors.lastname: Ljungman
authors.forename: U
authors.initials: U
authors.lastname: Saarinen
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.forename: J
authors.initials: J
authors.lastname: Ortega
authors.forename: E
authors.initials: E
authors.lastname: Lanino
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005060:Europe; D005260:Female; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000628:therapy*; D008297:Male; D014184:Transplantation, Homologous; D016896:Treatment Outcome