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.
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。.
Keywords: Clinical characteristic; Primary myelofibrosis; Prognosis; Thrombocytopenia.