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Nationwide survey of bisphosphonate therapy for children with reactivated Langerhans cell histiocytosis in Japan
Author(s) -
Morimoto Akira,
Shioda Yoko,
Imamura Toshihiko,
Kanegane Hirokazu,
Sato Takashi,
Kudo Kazuko,
Nakagawa Shinichiro,
Nakadate Hisaya,
Tauchi Hisamichi,
Hama Asahito,
Yasui Masahiro,
Nagatoshi Yoshihisa,
Kinoshita Akitoshi,
Miyaji Ryosuke,
Anan Tadashi,
Yabe Miharu,
Kamizono Junji
Publication year - 2011
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.22703
Subject(s) - medicine , langerhans cell histiocytosis , histiocytosis , pediatrics , disease , surgery
Background Several studies have suggested that Langerhans cell histiocytosis (LCH) is responsive to treatment with bisphosphonates (BPs). However the efficacy and safety of BPs therapy for childhood LCH is unknown. Procedure Data on children with LCH who had received BPs therapy were collected retrospectively from hospitals participating in the Japanese Pediatric Leukemia/Lymphoma Study Group. Results Twenty‐one children with histologically proven LCH were identified. Of these, the case histories of 16 children who had been treated with pamidronate (PAM) for disease reactivation were analyzed in detail. The median post‐PAM therapy follow‐up period was 2.8 years (range: 0.9–9.3 years). The median age at commencement of PAM therapy was 9.4 years (range: 2.3–15.0 years). All children had one or more bone lesions but none had risk organ (RO) involvement. In the majority of the children, six courses of PAM were administered at a dose of 1.0 mg/kg/course at 4‐week intervals. In 12 of the 16 children, all active lesions including lesions of the skin (n = 3) and soft tissues (n = 3) resolved. Of these children, eight children had no active disease for a median of 3.3 years post‐PAM therapy (range: 1.8–9.3 years). Progression‐free survival (PFS) was 56.3 ± 12.4% at 3 years. PFS was significantly higher in children with a first reactivation compared with children experiencing a second or subsequent reactivation. Conclusions PAM may be an effective treatment for reactivated LCH with bone lesions. A prospective trial of the efficacy of PAM in recurrent pediatric LCH is warranted. Pediatr Blood Cancer. 2010;56:110–115. © 2010 Wiley‐Liss, Inc.