56例45岁以下原发性骨髓纤维化患者的临床特点和预后分析

Clinical features and prognostic factors in primary myelofibrosis patients under 45 years old

目的研究45岁以下原发性骨髓纤维化(PMF)患者的临床特点和影响生存期的预后因素。方法分析56例年龄≤45岁的PMF患者的临床特征和实验室参数,确定影响生存期的预后因素。单因素分析用Logrank检验,多因素分析用COX回归模型。结果56例患者中,男27例,女29例,84%患者有贫血,66%患者Hb<100g/L,32%患者有低热、盗汗和消瘦等全身症状。中位生存期为69(11～127)个月。单因素分析显示外周血Hb<100g/L、BPC<100×109/L、WBC<10×109/L、有全身症状及从发病到诊断间隔时间≤6个月是影响患者生存期的危险因素。多因素分析显示Hb<100g/L和有全身症状与生存期短显著相关。以是否同时具有2项不良指标为标准将患者分为高危组和低危组。高危组患者生存期(16个月)明显短于低危组患者(88个月)(P<0.01)。用此2项指标预测生存期<3年的敏感性、特异性和阳性预测值分别为67%、100%和100%。结论对45岁以下PMF患者,Hb<100g/L和有全身症状是生存期短的预后不良因素,用此2项指标可将患者分为高危组和低危组,高危组患者生存期短于3年。

Objective To investigate the clinical and hematological features and identify the prognostic factors associated with short-term survival in primary myelofibrosis (PMF) patients under 45 years old. Methods The clinical manifestations, laboratory parameters, and survival were retrospectively analyzed in 56 PMF patients under 45 years old. Univariate analysis of prognostic factors was performed using Logrank test and multivariate analysis using COX model. Results Of the 56 patients, 27 were males and 29 females. The range of age was from 20 to 45 years (median 38 years). 84% of the patients were anemic and 66% Hb<100 g/L. 32% of the patients had constitutional symptoms including fever, night sweats and weight loss. The median survival was 69 months (95%CI 11~127). By univariate analysis, Hb<100 g/L, platelet count<100×10 9/L, WBC<10×10 9/L, constitutional symptoms and the duration from first signs to diagnosis ≤6 months were associated with poor prognosis. By multivariate analysis, only Hb<100 g/L and constitutional symptoms were independently associated with short survival. With these two adverse prognostic factors, the patients could be separated into a high risk and a low risk groups, and the median survivals were 16 and 88 months, respectively (P<0.001). Using these two factors to predict the less than 3-years survival for individual patient, the sensitivity, specificity and positive predictive value were 67%, 100% and 100%, respectively. Conclusion Hb<100 g/L and constitutional symptoms in PMF patients under 45 years old were significantly associated with short survival and poor prognosis. These two prognostic factors enabled to separate patients into a high and a low risk groups. The survival of high-risk patients was less than three years.