69例血栓性血小板减少性紫癜患者的临床特点及疗效分析

Clinical characteristics and outcome of 69 patients with thrombotic thrombocytopenic purpura

回顾性分析2017年10月至2020年10月郑州大学第一附属医院血栓性血小板减少性紫癜(TTP)患者的临床特点、诊治及转归。收集69例成人TTP患者的临床资料,其中男19例、女50例,中位年龄42(18~79)岁。64例可进行PLASMIC评分的患者,53例(82.8%)患者PLASMIC评分为6~7分。21例在治疗前检测血管性血友病因子裂解酶(ADAMTS13)活性,17例ADAMTS13活性<5%,3例为5%~10%,>10%的患者仅1例。69例患者均行血浆置换(PEX)和/或新鲜冰冻血浆输注(PI)治疗,其中43例加用糖皮质激素治疗,另有20例在PEX/PI+糖皮质激素治疗基础上联合利妥昔单抗和/或免疫抑制剂。69例患者中位随访时间12(1~57)个月,治疗后缓解率69.6%,复发率11.6%,2年总生存(OS)率为69.6%±5.5%。对可能影响OS的因素进行单因素及多因素分析,结果显示复发难治是影响OS的独立危险因素,26例复发难治患者2年OS率显著低于43例非复发难治者(41.5%±9.8%比83.7%±5.6%,P<0.001)。复发难治患者预后极差,需及早加用利妥昔单抗和/或免疫抑制剂等治疗以改善生存。

To analyze the clinical characteristics,diagnosis,treatment and outcome of patients with thrombotic thrombocytopenic purpura(TTP).The clinical data of 69 adult patients with TTP were retrospectively analyzed.There were 19 males and 50 females with a median age of 42(18-79)years.PLASMIC score 6-7 was recognized in 82.8%(53/64)patients.The activity of von Willebrand factor-cleaving protease(ADAMTS13),which was detected in 21 patients before treatment,was less than 5%in 17 patients and 5%-10%in 3 patients.All 69 patients were treated with plasma exchange(PEX)and/or fresh frozen plasma infusion(PI),43 of whom were also given glucocorticoid.In addition to PEX/PI and glucocorticoid,rituximab and/or immunosuppressants were administrated in 20 patients.The median follow-up time was 12(1-57)months.The remission rate was 69.6%,while the relapse rate was 11.6%.The 2-year overall survival(OS)rate was 69.6%±5.5%.The univariate and multivariate analysis showed that relapsed/refractory disease was an independent risk factor for OS.The 2-year OS rate of relapsed/refractory patients was significantly lower than that of the rest patients(41.5%±9.8%vs.83.7%±5.6%,P<0.001).Regarding the unfavorable prognosis in relapsed/refractory patients,rituximab and/or immunosuppressants are strongly recommended for sake of improving the overall survival.