Department of Pediatrics, Jichi Medical University School of Medicine, Shimotsuke, Japan.
Pediatr Blood Cancer. 2011 Jan;56(1):110-5. doi: 10.1002/pbc.22703.
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.
Data on children with LCH who had received BPs therapy were collected retrospectively from hospitals participating in the Japanese Pediatric Leukemia/Lymphoma Study Group.
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.
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.
几项研究表明朗格汉斯细胞组织细胞增生症（LCH）对双膦酸盐（BPs）治疗有反应。然而，BPs 治疗儿童 LCH 的疗效和安全性尚不清楚。
从参与日本儿科白血病/淋巴瘤研究组的医院中回顾性地收集了接受 BPs 治疗的 LCH 患儿的数据。
共确定了 21 例经组织学证实的 LCH 患儿。其中，详细分析了 16 例因疾病复发而接受帕米膦酸盐（PAM）治疗的患儿的病史。PAM 治疗后中位随访时间为 2.8 年（范围：0.9-9.3 年）。PAM 治疗开始时的中位年龄为 9.4 岁（范围：2.3-15.0 岁）。所有患儿均有一个或多个骨病变，但均无风险器官（RO）受累。在大多数患儿中，每 4 周给予 1.0mg/kg/疗程的 PAM，共 6 个疗程。在 16 例患儿中，12 例患儿的所有活动性病变（包括皮肤病变[3 例]和软组织病变[3 例]）均得到缓解。在这些患儿中，8 例患儿在 PAM 治疗后中位 3.3 年（范围：1.8-9.3 年）无活动性疾病。3 年无进展生存率（PFS）为 56.3±12.4%。首次复发患儿的 PFS 明显高于二次或多次复发患儿。
PAM 可能是治疗有骨病变的复发性 LCH 的有效方法。需要进行前瞻性试验以评估 PAM 在复发性儿科 LCH 中的疗效。
