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Bone Marrow Transplant. 2019 Feb 4. doi: 10.1038/s41409-019-0461-0. [Epub ahead of print]

Hematopoietic stem-cell transplantation in children with refractory acute myeloid leukemia.

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Department of Pediatrics, Kagoshima University Graduate School of Medical and Dental Sciences, 8-35-1 Sakuragaoka, Kagoshima, 890-8544, Japan.
Department of Pediatrics, Fujita Health University Hospital, 1-98 Dengakugakubo Kutsukake-cho, Toyoake-city, Aichi, 470-1192, Japan.
Department of Pediatrics, Tokyo Metropolitan Cancer and Infectious Disease Center Komagome Hospital, 3-18-22 Honkomagome, Bunkyo-ku, Tokyo, 113-8677, Japan.
Children's Cancer Center, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo, 157-8535, Japan.
Department of Pediatrics, Shiga University of Medical Science Hospital, Seta Tsukinowa-cho, Otsu, Shiga, 520-2192, Japan.
Division of Hematology/Oncology, Kanagawa Children's Medical Center, 2-138-4 Mutsukawa, Minami-ku, Yokohama, Kanagawa, 232-8555, Japan.
Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, 4-1-1 Kitakaname, Hiratsuka, Kanagawa, 259-1292, Japan.
Department of Pediatrics, Shinshu University School of Medicine, 3-1-1 Asahi, Matsumoto, Nagano, 390-8621, Japan.
Department of Hematology/Oncology, Saitama Children's Medical Center, 1-2 Shintoshin, Chuo-ku, Saitama, 330-8777, Japan.
Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, 3-35 Michishita-cho, Namamura-ku, Nagoya, Aichi, 453-0046, Japan.
Department of Hematology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, 2-2-2 Toranomon, Minato-ku, Tokyo, 105-8470, Japan.
Department of Hematology and Oncology, Shizuoka Children's Hospital, 860 Urushiyama, Aoi-ku, Shizuoka, 420-8660, Japan.
Department of Pediatrics, Kyushu University Hospital, 3-1-1, Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan.
Department of Hematology/Oncology, Osaka Women's and Children's Hospital, 840 Murodo-cho, Izumi, Osaka, 594-1101, Japan.
Department of Healthcare Administration, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 466-8550, Japan.
Japanese Data Center for Hematopoietic Cell Transplantation, 1-1-20 Daikominami, Higashi-ku, Nagoya, Aichi, 461-0047, Japan.
Department of Pediatrics, Kyoto City Hospital, 1-2 Mibuhigashitakadacho, Nakagyo Ward, Kyoto, 604-8845, Japan.


Allogeneic hematopoietic stem cell transplantation (HSCT) can be used to treat children with refractory acute myeloid leukemia (AML). This retrospective analysis aimed to describe the outcomes and risk factors in such children. Data were collected through the nation-wide registry program in Japan. A total of 417 AML (median age: 13 years) patients 20 years or younger at HSCT, between January 2001 and December 2015, were included. A total of 314 patients died, and the median follow-up duration of the survivors was 1052 days. The most frequent cause of death was leukemia progression (58%). The 3-year overall survival (OS) rate was 23% (95% confidence interval [CI]: 19-28%). Chronic GVHD was associated with improved 3-year OS (47%, 95% CI, 36-57%, hazard ratio: 0.603, pā€‰=ā€‰0.001). Low performance status, presence of more than 25% of marrow blasts, presence of blasts in the blood at transplantation, FAB (other than M1 or M2), male donors, and number of transplantations ā‰„ā€‰2 were adverse pre-HSCT variables. Patients with 0, 1-2, 3-4, 5, and 6-7 pre-HSCT variables had 3-year OS rates of 52%, 32%, 19%, 8, and 0%, respectively. Our findings may help experts decide if HSCT should be performed in such cases.


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