title: Allogeneic haematopoietic stem cell transplantation for infant acute lymphoblastic leukaemia with KMT2A (MLL) rearrangements: a retrospective study from the paediatric acute lymphoblastic leukaemia working group of the Japan Society for Haematopoietic Cell Transplantation.
abstract: Allogeneic haematopoietic stem cell transplantation (HSCT) is still considered to play an important role as a consolidation therapy for high-risk infants with acute lymphoblastic leukaemia (ALL). Here, we retrospectively analysed outcomes of HSCT in infants with ALL based on nationwide registry data of the Japan Society for Haematopoietic Cell Transplantation. A total of 132 allogeneic HSCT for infant ALL with KMT2A (MLL) gene rearrangements, which were performed in first complete remission (CR1), were analysed. The 5-year overall survival rate after transplantation was 67·4 ± 4·5%). Although recent HSCT (after 2004) had a trend toward better survival, no statistical correlation was observed between outcomes and each factor, including age at diagnosis, initial leucocyte count, cytogenetics, donor types or conditioning of HSCT. Myeloablative conditioning with total body irradiation did not provide a better survival (60·7 ± 9·2%) over that with busulfan (BU; 67·8 ± 5·7%). Two of the 28 patients treated with irradiation, but none of the 90 BU-treated patients, developed a secondary malignant neoplasm. In conclusion, allogeneic HSCT using BU was a valuable option for infant ALL with KMT2A rearrangements in CR1.
authors.affiliation: Department of Paediatrics, The University of Tokyo, Tokyo, Japan; Department of Cell Therapy and Transplantation Medicine, The University of Tokyo, Tokyo, Japan.
authors.forename: Motohiro
authors.initials: M
authors.lastname: Kato
authors.forename: Daiichiro
authors.initials: D
authors.lastname: Hasegawa
authors.forename: Katsuyoshi
authors.initials: K
authors.lastname: Koh
authors.forename: Keisuke
authors.initials: K
authors.lastname: Kato
authors.forename: Junko
authors.initials: J
authors.lastname: Takita
authors.forename: Jiro
authors.initials: J
authors.lastname: Inagaki
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Goto
authors.forename: Souichi
authors.initials: S
authors.lastname: Adachi
authors.forename: Akira
authors.initials: A
authors.lastname: Hayakawa
authors.forename: Yasufumi
authors.initials: Y
authors.lastname: Takeshita
authors.forename: Akihisa
authors.initials: A
authors.lastname: Sawada
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Atsuta
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
mesh_terms: D064591:Allografts; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D036101:Cord Blood Stem Cell Transplantation / Q000706:statistics & numerical data; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000706:statistics & numerical data; D011495:Histone-Lysine N-Methyltransferase; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D019520:Living Donors; D019653:Myeloablative Agonists / Q000627:therapeutic use; D051788:Myeloid-Lymphoid Leukemia Protein / Q000235:genetics; D016609:Neoplasms, Second Primary / Q000453:epidemiology / Q000209:etiology; D015514:Oncogene Proteins, Fusion / Q000235:genetics; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy / Q000235:genetics / Q000628:therapy*; D016016:Proportional Hazards Models; D012042:Registries / Q000706:statistics & numerical data; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014916:Whole-Body Irradiation
chemical_list: C497422:KMT2A protein, human; D019653:Myeloablative Agonists; D015514:Oncogene Proteins, Fusion; D051788:Myeloid-Lymphoid Leukemia Protein; D011495:Histone-Lysine N-Methyltransferase; D002066:Busulfan
keywords: acute lymphoblastic leukaemia; busulfan; infant; stem cell transplantation; total body irradiation