毛细胞白血病临床特征及生存预后分析

Analysis of clinical and laboratory characteristics and survival with hairy cell leukemia

目的探讨我国毛细胞白血病（HCL）患者的临床表现与实验室特点、对治疗反应、生存及预后情况。方法回顾性总结1990年8月至2012年3月中国医学科学院北京协和医学院血液学研究所收治的30例HCL患者临床资料，并进行随访。结果经典型HCL患者22例（73．3％），变异型HCL患者8例（26．7％）。脾大是HCL最常见的体征。变异型患者白细胞计数均增高，全血细胞减少在经典型患者占36．4％（8／22）。5例行遗传学检查的变异型患者中3例染色体核型异常。12例患者骨髓电镜检查示3例核糖体．板层复合物阳性。在经典型患者，核苷类似物为主的化疗方案完全缓解（ca）率明显优于其他治疗方案（3／4比1／18，P=0．012）。中位随访27（1—142）个月，无失访，11例进展，6例死亡。中位总生存（OS）时间未达到，预计1、3、6年OS率分别为84％、78％、58％。中位无进展生存时间（PFS）为（63±24）个月，预计1、2、5年PFS率分别为86％、72％、44％。变异型患者中位PFS明显短于经典型患者[（23±3）比（78±12）个月，P=0．014]。单因素分析显示初诊发热、贫血为经典型患者预后不良因素（P=0．038、0．000），而核苷类似物使用与否不影响其疾病进展。结论我国HCL患者全血细胞减少者较低，电镜核糖体．板层复合物阳性率低。经典型患者使用核苷类似物可提高CR率，但不改善PFS。变异型HCL比例高于国外，常伴遗传学异常，且变异型患者PFS明显短于经典型，预后较差。

Objective To explore the clinical and laboratory characteristics and survival of Chinese patients with hairy cell leukemia （HCL）. Methods A total of 30 HCL patients from August 1990 to March 2012 were retrospectively analyzed. Results There were 22 cases with classical HCL （HCL-C） and 8 with variant HCL （HCL-V）. Splenomegaly was the most common physical finding. Leukocytosis was found in all cases of HCL-V. But pancytopenia only accounted for 36. 4% （8/22） in HCL-C. And 3/5 of HCL-V had abnormal chromosome karyotypes. Ribosomal-lamellae complexes （RLC） -were found only in about 3/12 of HCL cases. Chemotherapy regimens including purine nucleoside analogues achieved a better complete remission （CR） rate than other regimens （3/4 vs 1/18, P = O. 012 ） in HCL-C. The median follow-up period was 27 （ 1 - 142） months. There was no follow-up loss. Eleven cases progressed and 6 died. The median overall survival （OS） was not reached. And the 1, 3, 6-year OS rates were 84% ,78% and 58% respectively. The median progression-free survival （PFS） was （63 ＋ 24） months and the 1, 2, 5-year PFS rates were 86%, 72% and 44% respectively. The median PFS of HCL-V was significant shorter than HCL- C （（23 ＋3） vs （78 _＋12） months, P=0. O14）. In HCL-C group, fever （P=0.038） and anemia （P= O. 000） at diagnosis were poor prognostic factors. But purine nucleoside analogues made no significant difference in PFS. Condusiorm Pancytopenia is infrequent in Chinese HCL patients. And classical RLC is rare under electron microscope. Purine nucleoside analogues may achieve a better CR rate, but fail to improve PFS rate. As compared with HCL-C, HCL-V is common with genetic abnormalities and has a worse prognosis with a shorter PFS.