1. Outcome of 125 Children with Chronic Myelogenous Leukemia Who Received Transplants from Unrelated Donors: The Japan Marrow Donor Program
- Author
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Takakazu Kawase, Yoshihisa Nagatoshi, Hiroyuki Shimada, Chikako Tono, Kazutoshi Koike, Noriko Hotta, Akihiro Watanabe, Hidemitsu Kurosawa, Yasuo Morishima, Masami Inoue, Keisei Kawa, Masaki Ito, Seiji Kojima, Koji Kato, Kazuko Hamamoto, Yoshiko Atsuta, Akihiko Tanizawa, Hideki Muramatsu, and Ayami Yoshimi
- Subjects
Male ,Transplantation Conditioning ,medicine.medical_treatment ,Hematopoietic stem cell transplantation ,Piperazines ,Japan ,Bone Marrow ,hemic and lymphatic diseases ,Medicine ,Registries ,Child ,Children ,Remission Induction ,Hematopoietic Stem Cell Transplantation ,Stem cell transplantation ,Hematology ,Treatment Outcome ,surgical procedures, operative ,medicine.anatomical_structure ,Child, Preschool ,Benzamides ,Imatinib Mesylate ,Female ,Chronic myelogenous leukemia ,medicine.medical_specialty ,Unrelated donor ,Adolescent ,Bone marrow transplantation ,Antineoplastic Agents ,Donor Selection ,Young Adult ,Leukemia, Myelogenous, Chronic, BCR-ABL Positive ,Internal medicine ,Humans ,Transplantation, Homologous ,Protein Kinase Inhibitors ,Survival analysis ,Japan Marrow Donor Program ,Retrospective Studies ,Transplantation ,business.industry ,Donor selection ,Infant ,Retrospective cohort study ,medicine.disease ,Survival Analysis ,Surgery ,Pyrimidines ,Imatinib mesylate ,Bone marrow ,business - Abstract
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 dose314 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.
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