title: Outcome of pediatric patients with Langerhans cell histiocytosis treated with 2 chlorodeoxyadenosine: a nationwide survey in Japan.
abstract: The aim of this study was to assess the outcome of treatment with 2-chlorodeoxyadenosine (2-CdA) in pediatric patients with Langerhans cell histiocytosis (LCH) in Japan. We retrospectively identified 17 pediatric LCH patients treated with 2-CdA. All patients were refractory or reactivated cases who had been initially treated according to the JLSG-02 protocol of the Japan LCH study group. At initiation of 2-CdA therapy, 4 patients had primary refractory multisystem (MS) disease with risk organ (RO) involvement (MS+), 9 patients had reactivated MS disease [5 MS+ and 4 without RO involvement (MS-)], and the remaining 4 patients had refractory/reactivated multifocal bone disease (MFB). Treatment with 2-CdA (4-9 mg/m(2)/day) was administered on 2-5 consecutive days and repeated every 3-4 weeks for a period that ranged from 2 to 12 months. Four primary refractory patients were treated with 2-CdA combined with high dose of cytarabine. In MS+ patients, response to treatment was observed in 5 of the 9 patients. In MS-/MFB patients, 5 of the 8 patients showed response to treatment. In the patients who were primary refractory or had reactivation during initial chemotherapy, 4 of 10 patients showed good response. On the other hand, in the patients having reactivation while off therapy, 6 of 7 patients showed good response. These findings suggest that 2-CdA is effective for reactivated LCH while off therapy.
authors.affiliation: Department of Pediatrics, Kyoto Prefectural University of Medicine, Kamigyo-ku, Kyoto, Japan. imamura@koto.kpu-m.ac.jp
authors.forename: Toshihiko
authors.initials: T
authors.lastname: Imamura
authors.forename: Takashi
authors.initials: T
authors.lastname: Sato
authors.forename: Yoko
authors.initials: Y
authors.lastname: Shiota
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Kanegane
authors.forename: Kazuko
authors.initials: K
authors.lastname: Kudo
authors.forename: Shinichirou
authors.initials: S
authors.lastname: Nakagawa
authors.forename: Hisaya
authors.initials: H
authors.lastname: Nakadate
authors.forename: Hisamichi
authors.initials: H
authors.lastname: Tauchi
authors.forename: Junji
authors.initials: J
authors.lastname: Kamizono
authors.forename: Akira
authors.initials: A
authors.lastname: Morimoto
mesh_terms: D015762:2-Chloroadenosine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage* / Q000009:adverse effects; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage / Q000009:adverse effects; D003625:Data Collection; D003839:Deoxyadenosines / Q000008:administration & dosage* / Q000009:adverse effects; D019008:Drug Resistance, Neoplasm; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007564:Japan; D008297:Male; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D000964:Antimetabolites, Antineoplastic; D003839:Deoxyadenosines; D003561:Cytarabine; D015762:2-Chloroadenosine; C055994:2-chloro-3'-deoxyadenosine