title: HLA haploidentical hematopoietic cell transplantation using clofarabine and busulfan for refractory pediatric hematological malignancy.
abstract: Haploidentical hematopoietic cell transplantation (HCT) conditioning with clofarabine and target area under the blood concentration-time curve (AUC)-based busulfan adjustment was performed in three patients with refractory pediatric leukemia. The target AUC for two patients who had already received multiple transplantations was 3600 and 4000 μmol min/L, and that for the patient with Down's syndrome was 3000 μmol min/L. Regimen-related toxicity was well tolerated in all cases. All three maintained cytological remission throughout the follow-up period (between 31 and 167 weeks). Thus, haploidentical HCT conditioning with clofarabine and target AUC-based busulfan adjustment may be a preferable option for children with recurrent or refractory pediatric leukemia.
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan. m.takagi.ped@tmd.ac.jp.
authors.forename: Masatoshi
authors.identifier: http://orcid.org/0000-0002-7580-9184
authors.initials: M
authors.lastname: Takagi
authors.affiliation: Department of Hospital Pharmacy, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan.
authors.forename: Yasuyoshi
authors.initials: Y
authors.lastname: Ishiwata
authors.affiliation: Department of Pediatric Oncology, National Cancer Research Center, Tsukiji 5-1-1, Chuo-ku, Tokyo, Japan.
authors.forename: Yuki
authors.initials: Y
authors.lastname: Aoki
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Satoshi
authors.initials: S
authors.lastname: Miyamoto
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Akihiro
authors.initials: A
authors.lastname: Hoshino
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Kazuaki
authors.initials: K
authors.lastname: Matsumoto
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Akira
authors.initials: A
authors.lastname: Nishimura
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Mari
authors.initials: M
authors.lastname: Tanaka
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Masakatsu
authors.initials: M
authors.lastname: Yanagimachi
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Noriko
authors.initials: N
authors.lastname: Mitsuiki
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Kohsuke
authors.initials: K
authors.lastname: Imai
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Kanegane
authors.affiliation: Department of Transfusion Medicine, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan.
authors.forename: Michiko
authors.initials: M
authors.lastname: Kajiwara
authors.affiliation: Department of Hospital Pharmacy, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan.
authors.forename: Kanako
authors.initials: K
authors.lastname: Takikawa
authors.affiliation: Department of Hospital Pharmacy, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan.
authors.forename: Tsukasa
authors.initials: T
authors.lastname: Mae
authors.affiliation: Department of Pediatrics and Adolescent Medicine, Juntendo University, School of Medicine, Yushima 2-1-1, Bunkyo-ku, Tokyo, Japan.
authors.forename: Osamu
authors.initials: O
authors.lastname: Tomita
authors.affiliation: Department of Pediatrics and Adolescent Medicine, Juntendo University, School of Medicine, Yushima 2-1-1, Bunkyo-ku, Tokyo, Japan.
authors.forename: Junya
authors.initials: J
authors.lastname: Fujimura
authors.affiliation: Department of Pharmacokinetics and Pharmacodynamics, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan.
authors.forename: Masato
authors.initials: M
authors.lastname: Yasuhara
authors.affiliation: Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Okura 2-10-1, Setagaya-ku, Tokyo, Japan.
authors.forename: Daisuke
authors.initials: D
authors.lastname: Tomizawa
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Shuki
authors.initials: S
authors.lastname: Mizutani
authors.affiliation: Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University (TMDU), Yushima 1-5-45, Bukyo-ku, Tokyo, 113-8510, Japan.
authors.forename: Tomohiro
authors.initials: T
authors.lastname: Morio
mesh_terms: D000227:Adenine Nucleotides / Q000008:administration & dosage*; D001087:Arabinonucleosides / Q000008:administration & dosage*; D002066:Busulfan / Q000008:administration & dosage*; D002648:Child; D000077866:Clofarabine; D005260:Female; D005500:Follow-Up Studies; D006680:HLA Antigens / Q000235:genetics*; D006238:Haploidy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007938:Leukemia / Q000628:therapy*; D008297:Male; D012008:Recurrence; D012074:Remission Induction; D013997:Time Factors; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D000227:Adenine Nucleotides; D001087:Arabinonucleosides; D006680:HLA Antigens; D000077866:Clofarabine; D002066:Busulfan
keywords: Busulfan; Clofarabine; Haploidentical hematopoietic cell transplantation; Pediatric refractory leukemia