克拉屈滨治疗伴有尿崩儿童朗格汉斯细胞组织细胞增多症疗效观察

目的：探讨克拉屈滨（Cladribine，2-CdA）对于常规治疗无效的伴有尿崩儿童朗格汉斯细胞组织细胞增多症（Langerhans cell histiocytosis，LCH）疗效。方法：7例伴有尿崩症状的LCH患儿予2~4个疗程2-CdA5 mg·（m²·d）^-1×5 d]+阿糖胞苷Ara-c，1 000 mg·（m²·d）^-1×5 d]治疗，观察临床症状体征及辅助检查变化情况，并监测不良反应。结果：6例患儿治疗后临床症状缓解达非活动性疾病状态，1例患儿最终因肝功能衰竭死亡。7例患儿无严重不良反应导致治疗中断。结论：2-CdA对于常规治疗无效的伴有尿崩儿童LCH疗效好，为难治复发的LCH提供治疗新思路。

Observation of children with Langerhans cell histiocytosis accompanying diabetes insipidus treated by cladribine

OBJECTIVE To explore the efficacy of cladribine(2-CdA)in the treatment of Langerhans cell histiocytosis(LCH)in children with diabetes insipidus unresponsive to conventional treatment.METHODS Two to 4 courses of 2-CdA5 mg·(m2·d)-1×5 d]+ cytarabineAra-c, 1 000 mg·(m2·d)-1×5 d]treatment were applied to seven LCH children with concurrent diabetes insipidus.The clinical symptoms, signs and auxiliary examinations were recorded and adverse reactions monitored.RESULTS After treatment, all clinical symptoms reverted to the status of non-active disease.One case died of hepatic failure.No treatment was interrupted due to serious side effect.CONCLUSION 2-CdA is curative for LCH children with concurrent diabetes insipidus unresponsive to conventional treatment.And it provides a new therapeutic idea for refractorily recurrent LCH.