Comparison of Outcomes for Pediatric Patients With Acute Myeloid Leukemia in Remission and Undergoing Allogeneic Hematopoietic Cell Transplantation With Myeloablative Conditioning Regimens Based on Either Intravenous Busulfan or Total Body Irradiation: A Report From the Japanese Society for Hematopoietic Cell Transplantation.
Pediatric patients with acute myeloid leukemia (AML) mainly receive myeloablative conditioning regimens based on busulfan (BU) or total body irradiation (TBI) before allogeneic hematopoietic cell transplantation (allo-HCT); however, the optimal conditioning regimen remains unclear. To identify which of these regimens is better for pediatric patients, we performed a retrospective analysis of nationwide registration data collected in Japan between 2006 and 2011 to assess the outcomes of patients receiving these regimens before a first allo-HCT. Myeloablative conditioning regimens based on i.v. BU (i.v. BU-MAC) (n = 69) or TBI (TBI-MAC) (n = 151) were compared in pediatric AML patients in first or second complete remission (CR1/CR2). The incidences of sinusoid obstruction syndrome, acute and chronic graft-versus-host disease, and early nonrelapse mortality (NRM) before day 100 were similar for both conditioning groups; however, the incidence of bacterial infection during the acute period was higher in the TBI-MAC group (P = .008). Both groups showed a similar incidence of NRM, and there was no significant difference in the incidence of relapse between the groups. Univariate and multivariate analyses revealed no significant differences in the 2-year relapse-free survival rates for the i.v. BU-MAC and TBI-MAC groups in the CR1/CR2 setting (71% versus 67%, P = .36; hazard ratio, .73; 95% CI, .43 to 1.24, respectively). TBI-MAC was no better than i.v. BU-MAC for pediatric AML patients in remission. Although this retrospective registry-based analysis has several limitations, i.v. BU-MAC warrants further evaluation in a prospective trial.
Department of Pediatrics and Blood and Marrow Transplantation, Matsushita Memorial Hospital, Moriguchi, Japan; Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto, Japan. Electronic address: ishidah@koto.kpu-m.ac.jp.
Hiroyuki
H
Ishida
Department of Pediatrics and Cell Therapy and Transplantation Medicine, University of Tokyo, Tokyo, Japan.
Motohiro
M
Kato
Department of Pediatrics, Fujita Health University School of Medicine, Aichi, Japan.
Kazuko
K
Kudo
Department of Pediatrics, Shiga University, Graduate School of Medicine, Otsu, Japan.
Takashi
T
Taga
Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.
Daisuke
D
Tomizawa
Department of Pediatrics, Osaka University, Graduate School of Medicine, Suita, Japan.
Takako
T
Miyamura
Devision of Hemato-Oncology/Regenerative Medicine, Kanagawa Children's Medical Center, Yokohama, Japan.
Hiroaki
H
Goto
Department of Pediatrics, National Kyushu Cancer Center, Fukuoka, Japan.
Jiro
J
Inagaki
Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Katsuyoshi
K
Koh
Department of Pediatrics, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.
Kiminori
K
Terui
Department of Pediatrics, Niigata Cancer Center Hospital, Niigata, Japan.
Atsushi
A
Ogawa
Department of Pediatrics, Kagoshima University Medical and Dental Hospital, Kagoshima, Japan.
Yoshifumi
Y
Kawano
Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, Osaka, Japan.
Masami
M
Inoue
Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, Osaka, Japan.
Akihisa
A
Sawada
Department of Hematology/Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Koji
K
Kato
Japanese Data Center for Hematopoietic Cell Transplantation and Department of Healthcare Administration, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Yoshiko
Y
Atsuta
Department of Hematopoietic Stem Cell Transplantation, National Cancer Center, Tokyo, Japan.
Takuya
T
Yamashita
Human Health Sciences, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Souichi
S
Adachi
D000293:Adolescent; D002066:Busulfan / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007275:Injections, Intravenous; D007564:Japan; D015470:Leukemia, Myeloid, Acute / Q000276:immunology / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D012008:Recurrence; D012074:Remission Induction; D012189:Retrospective Studies; D012955:Societies, Medical; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014916:Whole-Body Irradiation
D019653:Myeloablative Agonists; D002066:Busulfan
Acute myeloid leukemia; Busulfan; Children; Hematopoietic stem cell transplantation; Total body irradiation