不同血小板水平原发性骨髓纤维化患者的临床特征及预后研究

A study of clinical characteristics and prognosis of primary myelofibrosis patients with thrombocytopenia in varied degrees

目的探讨不同血小板水平原发性骨髓纤维化(PMF)患者的临床特征及其预后。方法对1990年5月1日至2017年4月30日在中国医学科学院血液病医院就诊的1 305例PMF患者进行回顾性分析。结果1 305例PMF患者中,重度血小板减少(PLT<50×10^9/L)、轻度血小板减少[PLT(50~99)×10^9/L]、无血小板减少患者(PLT≥100×10^9/L)分别为320例(24.5%)、198例(15.2%)和787例(60.3%)。在血小板计数越低的组别中,HGB<100 g/L患者比例、WBC<4×10^9/L患者比例和外周血原始细胞≥3%患者比例、异常染色体核型患者比例和预后不良染色体核型患者比例越高(P<0.001,P<0.001,P=0.004,P<0.001,P<0.001),JAK2V617F突变阳性率越低(P<0.001)。血小板计数与脾肿大、HGB、WBC呈正相关(P值均<0.001,相关系数分别为0.131、0.445、0.156),与体质性症状和外周血原始细胞比例呈负相关(P值分别为0.009、0.045,相关系数为-0.096、-0.056)。重度血小板减少、轻度血小板减少、无血小板减少组患者中位生存时间分别为32、67、89个月(P<0.001)。血小板减少程度分组[HR=1.693(95%CI 1.320~2.173),P<0.001]和动态国际预后积分系统(DIPSS)预后分级[HR=2.051(95%CI 1.511~2.784),P<0.001]均为PMF患者独立不良预后因素。结论血小板计数越低的PMF患者出现贫血、白细胞减少和外周血原始细胞比例增高的比例越高、临床表现越重、预后越差,临床上应采取更加积极的治疗措施。

Objective To evaluate clinical characteristics and prognosis of primary myelofibrosis (PMF) patients with thrombocytopenia in varied degrees.Methods Clinical features and survival data of 1 305 Chinese patients with PMF were retrospectively analyzed.The prognostic value of thrombocytopenia in patients with PMF was evaluated.Results 320 subjects (47%) presented severe thrombocytopenia (PLT<50×10^9/L),198 ones (15.2%) mild thrombocytopenia [PLT (50-99)×10^9/L] and 787 ones (60.3%) without thrombocytopenia (PLT ≥ 100×10^9/L).The more severe the thrombocytopenia,the higher the proportions of HGB<100 g/L,WBC<4×10^9/L,circulating blasts ≥ 3%,abnormal karyotype and unfavourable cytogenetics (P<0.001,P<0.001,P=0.004,P<0.001 and P<0.001,respectively) were observed in this cohort of patients.The more severe the thrombocytopenia,the lower the proportion of JAK2V617F positive (P<0.001) was also noticed.Platelet count was positively correlated with splenomegaly,HGB and WBC (P<0.001,correlation coefficients were 0.131,0.445 and 0.156,respectively).Platelet count was negative correlated with constitutional symptoms and circulating blasts (P=0.009,P=0.045,respectively;correlation coefficients were -0.096 and -0.056,respectively).The median survival of patients with severe thrombocytopenia,mild thrombocytopenia and without thrombocytopenia were 32,67 and 89 months,respectively (P<0.001).Multivariate analysis identified thrombocytopenia in varied degrees (HR=1.693,95%CI 1.320-2.173,P<0.001) and Dynamic Internation Prognostic Scoring System(DIPSS) prognostic model (HR=2.051,95%CI 1.511-2.784,P<0.001) as independent risk factors for survival.Conclusion PMF patients with severe thrombocytopenia frequently displayed anemia,leucopenia,circulating blasts and short survival,so active treatment measures should be taken especially in these patients.