Outcome of 125 children with chronic myelogenous leukemia who received transplants from unrelated donors: the Japan Marrow Donor Program.
Because of a small number of patients, only a few studies have addressed the outcome of bone marrow transplantation (BMT) in children with Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML), who receive graft from a volunteer-unrelated donor (VUD), especially after practical application of imatinib mesylate. The outcomes of BMT from a VUD in 125 children with Ph+ CML were retrospectively reviewed. Patients were identified through the Japan Marrow Donor Program as having undergone BMT between 1993 and 2005 and were aged 1-19 years at the time of transplant (median age, 14 years). The probabilities of 5-year overall survival (OS) and leukemia-free survival (LFS) were 59.3% and 55.5%, respectively. Multivariate analysis identified the following unfavorable survival factors: infused total nucleated cell dose<314 x 10(6) /kg (relative risk [RR]=2.43; 95% confidence interval [CI]=1.33-4.44; P=.004), advanced phase (RR=2.43; 95% CI=1.37-4.31; P=.004), and no major cytogenetic response (MCyR) at the time of BMT (RR=6.55; 95% CI=1.98-21.6; P=.002). Of the 17 patients treated with imatinib, 15 (88%) achieved MCyR at the time of BMT, and this group had an excellent 5-year OS of 81.9%. Disease phase, infused total nucleated cell dose, and cytogenetic response were independent risk factors for survival of unrelated BMT. These findings provide important information for assessing the indications for and improving outcome in unrelated BMT for the treatment of pediatric CML.
Muramatsu
Hideki
H
Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Kojima
Seiji
S
Yoshimi
Ayami
A
Atsuta
Yoshiko
Y
Kato
Koji
K
Nagatoshi
Yoshihisa
Y
Inoue
Masami
M
Koike
Kazutoshi
K
Kawase
Takakazu
T
Ito
Masaki
M
Kurosawa
Hidemitsu
H
Tanizawa
Akihiko
A
Tono
Chikako
C
Hamamoto
Kazuko
K
Hotta
Noriko
N
Watanabe
Akihiro
A
Morishima
Yasuo
Y
Kawa
Keisei
K
Shimada
Hiroyuki
H
D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001549:Benzamides; D001853:Bone Marrow; D016026:Bone Marrow Transplantation / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D046148:Donor Selection; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality; D006801:Humans; D000068877:Imatinib Mesylate; D007223:Infant; D007564:Japan; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000628:therapy*; D008297:Male; D010879:Piperazines / Q000627:therapeutic use; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D011743:Pyrimidines / Q000627:therapeutic use; D012042:Registries; D012074:Remission Induction; D012189:Retrospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
D000970:Antineoplastic Agents; D001549:Benzamides; D010879:Piperazines; D047428:Protein Kinase Inhibitors; D011743:Pyrimidines; D000068877:Imatinib Mesylate