克拉屈滨治疗毛细胞白血病24例临床分析

Clinical analysis of 24 patients of hairy cell leukemia treated by cladribine

目的探讨克拉屈滨治疗毛细胞白血病（HCL）的疗效。方法回顾性分析2006年11月至2017年10月应用克拉屈滨治疗的24例HCL患者的临床资料，对其疗效和不良反应进行分析。结果①24例患者中，男22例，女2例，诊断时中位年龄49．5（33～76）岁；脾大20例（轻度脾大4例，中度脾大4例，巨脾12例），淋巴结肿大3例，已行脾切除术者1例；全血细胞减少5例，2系减少15例，1系减少4例；流式细胞术检测骨髓中位HCL细胞比例为21．79％（0．69％～68．96％）；一代或二代测序技术检On,0BRAF突变阳性率75．00％（15／20）。②24例患者中，20例接受克拉屈滨单药治疗（单疗程19例，2个疗程1例），4例接受克拉屈滨联合利妥昔单抗治疗（单疗程3例，2个疗程1例）。克拉屈滨治疗后6～12个月进行疗效评估，除外5例随访时间〈6个月者，19例患者中完全缓解（CR）者9例，不确定的完全缓解（CRu）者9例，PR患者1例，CR／CRu率为94．7％，总体反应率为100．0％。③24例患者均于克拉屈滨治疗后发生2～4级血液学不良反应，主要为3—4级中性粒细胞减少（66．67％）和3～4级血小板减少（29．2％），所有不良反应经对症处理可控或自行恢复。④中位随访15（3—133）个月，无患者发生疾病进展、复发及死亡，中位总生存和无进展生存时间均为未达到。结论克拉屈滨治疗HCL反应率高，不良反应可控，预后好。

Objective To investigate the curative effect of hairy cell leukemia by clatabine. Methods The clinical data of 24 patients with hairy cell leukemia treated by cladribine from November 2006 to October 2017 were analyzed retrospectively, then the curative effect and adverse drug reaction were analyzed. Results ① A total of 24 patients including 22 male and 2 female, and the median age was 49.5 years （range 33 to 76） at diagnosis. There were 20 patients with of splenomegaly （4 patients with mild splenomegaly, 4 moderate splenomegaly, and 12 massive splenomegaly）, 3 patients with enlargement of lymph nodes, and I patients who had undergone sptenectomy. Five patients were pancytopenia, 15 were cytopenia in 2 lineages, and 4 patients were cytopenia only in one lineage. The median ratio of HCL cells detected by flow cytometry in bone marrow was 21.79% （0.69%-68.96%）. BRAF mutation was detected in 15 patients by first generation or next generation sequencing technology. ②Among 24 patients, 20 were treated with cladribine alone （one course in 19 patients, 2 courses in I patient）, and 4 patients were treated with cladribine combined with rituximab （one course in 3 patients, 2 courses in 1 patient）. Excepting 5 patients whose follow-up time was not reaching 6 months, 19 patients were evaluated for efficacy in 6-12 months after treatment： 9 patients obtained CR, 9 obtained unconfirmed CR （Cru）, the other 1 obtained PR, the CR/CRu rate was 94.7%, the overall response rate （ORR） was 100.0%.③ All the 24 patients appeared 2-4 grade hematological adverse reactions after cladribine treatment, which were mainly grade 3/4neutropenia （66.67%） and grade 3/4 thrombocytopenia （29.2%）. All the adverse reactions were controlled or recovered spontaneously. ④fter the median follow-up time of 15 （3-133） months, no progression, recurrence or death occurred in the patients. Both median OS and PFS were not reached. Conclusion This study suggests that treatment of HCL with cladribine has high response rate, controllable adverse reactions and the good prognosis.