联合阿糖胞苷治疗难治性朗格汉斯细胞组织细胞增生症临床分析

Clinical analysis of combined chemotherapy with cytarabine for refractory Langerhans cell histiocytosis

目的评估联合阿糖胞苷(Ara-C)治疗儿童难治性朗格汉斯细胞组织细胞增生症(LCH)的疗效。方法回顾分析确诊难治性LCH且采用联合Ara-C化疗患儿的临床资料,评估疗效。结果共11例难治性LCH患儿,男性6例、女性5例,中位诊断年龄22个月(5~157个月),中位随访时间32个月(17~75个月)。所有患儿均为多系统LCH(MS-LCH)。经一线治疗,9例患儿疾病进展(8例为骨骼进展),2例病情稳定。所有患儿经联合Ara-C化疗,总体好转率91%(1例痊愈,9例好转,1例处于稳定状态),总体生存率100%。10例中枢神经系统(CNS)受累患儿,9例首诊时伴CNS肿块影,治疗后5例CNS肿块完全消退。2例患儿有后遗症,均为尿崩症。结论联合Ara-C化疗治疗难治性LCH的反应率高,对伴CNS受累者,亦有较好疗效。

Objective To evaluate the efficacy of combined chemotherapy with cytarabine(Ara-C)for children with refractory Langerhans cell histiocytosis(LCH).Methods The clinical data of patients with refractory LCH in Children's Hospital of Chongqing Medical University were retrospectively analyzed to evaluate the curative effect.Results A total of 11 patients with refractory LCH were included,with a male-to-female ratio of 6 to 5,the median diagnostic age was 22 months(5-157 months),with median follow-up duration of 32 months(17-75 months).All of the patients were MS-LCH.After first-line treatment,9 patients had disease progression(8 patients had skeletal progression),and 2 patients had stable disease.All patients were treated with combined Ara-C chemotherapy.The overall response rate was 91%(1 patient was cured,9 patients were improved,and 1 patient was in a stable state),the overall survival rate was 100%.For patients with central nervous system(CNS)involvement(n=10),9 patients were accompanied by CNS mass at the first diagnosis,and 5 of the 9 patients had a complete regression of the CNS mass after the treatment.And two patients had diabetes insipidus as sequelae.Conclusion Ara-C combined with chemotherapy for refractory LCH had a high response rate,and it also had a better effect for patients with CNS involvement.