Outcome of pediatric patients with Langerhans cell histiocytosis treated with 2 chlorodeoxyadenosine: a nationwide survey in Japan
Outcome of pediatric patients with Langerhans cell histiocytosis treated with 2 chlorodeoxyadenosine: a nationwide survey in Japan
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DOI：
10.1007/s12185-010-0558-0
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发表时间：
2010-05-01
影响因子：
2.1
通讯作者：
Morimoto, Akira
中科院分区：
文献类型：
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作者：
Imamura, Toshihiko;Sato, Takashi;Morimoto, Akira
本研究的目的是评估2-氯脱氧腺苷（2-CdA）治疗日本儿童朗格汉斯细胞组织细胞增生症（LCH）的结果。我们回顾性地确定了17例接受2-CdA治疗的儿童LCH患者。所有患者均为根据日本LCH研究组的JLSG-02方案进行初始治疗的难治性或再激活病例。在开始2-CdA治疗时，4例患者患有原发性难治性多系统（MS）疾病伴危险器官（RO）受累（MS+），9例患者患有再活化MS疾病[5例MS+和4例无RO受累（MS-）]，其余4例患者患有难治性/再活化多灶性骨病（MFB）。连续2-5天给予2-CdA（4-9 mg/m2/天）治疗，每3-4周重复一次，持续2 - 12个月。4例原发性难治性患者接受2-CdA联合大剂量阿糖胞苷治疗。在MS+患者中，在9例患者中的5例中观察到治疗应答。在MS-/MFB患者中，8例患者中有5例对治疗有反应。在原发难治性或初次化疗期间复发的患者中，10例患者中有4例显示良好反应。另一方面，在停止治疗时重新激活的患者中，7例患者中有6例表现出良好的反应。这些结果表明，2-CdA是有效的再激活LCH，而关闭治疗。
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.