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Allogeneic hematopoietic stem cell transplantation for the treatment of BCR-ABL1-negative atypical chronic myeloid leukemia and chronic neutrophil leukemia: A retrospective nationwide study in Japan.

Abstract
Atypical chronic myeloid leukemia (aCML) and chronic neutrophilic leukemia (CNL) are rare BCR-ABL1 fusion gene-negative myeloid neoplasms with a predominance of neutrophils. Since no standard therapeutic strategy currently exists for these diseases, we retrospectively evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for aCML and CNL. Data from 14 aCML and 5 CNL patients as their diagnoses were collected using a nationwide survey. Allo-HSCT was performed between 2003 and 2014. Preconditioning regimens included myeloablative (n = 15), reduced-intensity (n = 3), and non-myeloablative (n = 1) regimens. Transplanted stem cells were obtained from HLA-matched related donors (n = 5) and alternative donors (n = 14). Neutrophil engraftment was successfully achieved in 17 patients. One-year overall survival rates (OS) were 54.4% (95% confidence interval [CI], 24.8 to 76.7%) and 40.0% (95% CI, 5.2 to 75.3%) in patients with aCML and CNL, respectively. Among aCML patients, 1-year OS were 76.2% (95% CI, 33.2 to 93.5%) and 20.0% (95% CI, 0.8 to 58.2%) in patients with <5% myeloblasts (n = 9) and ≥5% myeloblasts (n = 5) in peripheral blood before allo-HSCT, respectively. These results suggest that allo-HSCT achieves long-term survival in patients with aCML and CNL. Better pre-transplant management is required to improve the outcomes of aCML patients with ≥5% blasts in peripheral blood.
AuthorsHidehiro Itonaga, Shuichi Ota, Takashi Ikeda, Hirohumi Taji, Itsuto Amano, Yuichi Hasegawa, Tatsuo Ichinohe, Takahiro Fukuda, Yoshiko Atsuta, Akihiko Tanizawa, Takeshi Kondo, Yasushi Miyazaki
JournalLeukemia research (Leuk Res) Vol. 75 Pg. 50-57 (12 2018) ISSN: 1873-5835 [Electronic] England
PMID30458320 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
CopyrightCopyright © 2018 Elsevier Ltd. All rights reserved.
Topics
  • Adolescent
  • Adult
  • Aged
  • Child
  • Disease-Free Survival
  • Female
  • Hematopoietic Stem Cell Transplantation (methods)
  • Humans
  • Japan
  • Kaplan-Meier Estimate
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative (mortality, pathology, therapy)
  • Leukemia, Neutrophilic, Chronic (mortality, pathology, therapy)
  • Male
  • Middle Aged
  • Retrospective Studies
  • Transplantation, Homologous
  • Young Adult

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