Reduced-toxicity myeloablative conditioning consisting of 8-Gy total body irradiation, cyclophosphamide and fludarabine for pediatric hematological malignancies.
Conventional myeloablative conditioning (MAC) regimens often cause severe regimen-related toxicity (RRT). Furthermore, many patients suffer from poor quality of life in accordance with the increase in long-term survivors. We therefore devised a reduced-toxicity myeloablative conditioning (RTMAC) regimen consisting of 8-Gy total body irradiation (TBI), fludarabine (FLU) and cyclophosphamide (CY) for pediatric hematological malignancies. A retrospective single-center analysis was performed on patients with leukemia or myelodysplastic syndrome (MDS), aged ≤20 years, who had received an 8-Gy TBI/FLU/CY RTMAC regimen followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT). Thirty-one patients underwent first allo-HSCT after an RTMAC regimen. The diagnoses were acute lymphoblastic leukemia (n = 11), acute myeloid leukemia (n = 13), MDS (n = 4), juvenile myelomonocytic leukemia (n = 1) and acute leukemias of ambiguous lineage (n = 2). While 3 patients showed early hematological relapse, the remaining 28 patients achieved engraftments. None of the patients developed grade 4 or 5 toxicities during the study period. The 5-year overall survival and relapse-free survival were 80% [95% confidence interval: CI, 61-91%] and 71% [95% CI, 52-84%], respectively. Our RTMAC regimen would be less toxic and offers a high probability of survival for children with hematological malignancies.
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Koichi
K
Hirabayashi
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Yozo
Y
Nakazawa
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Kazuo
K
Sakashita
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Takashi
T
Kurata
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Shoji
S
Saito
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Kentaro
K
Yoshikawa
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Miyuki
M
Tanaka
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Ryu
R
Yanagisawa
Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan.
Kenichi
K
Koike
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D005720:Gamma Rays / Q000627:therapeutic use*; D006086:Graft vs Host Disease / Q000401:mortality / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007938:Leukemia / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011788:Quality of Life; D012008:Recurrence; D012189:Retrospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014916:Whole-Body Irradiation; D055815:Young Adult
D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine