血栓性血小板减少性紫癜的临床特点及预后

Clinical Features and Prognosis of Thrombotic Thrombocytopenic Purpura

目的探讨血栓性血小板减少性紫癜(thrombotic thrombocytopenic purpura,TTP)的临床特点及预后因素。方法回顾性分析北京协和医院2012至2017年60例TTP患者的临床表现、实验室检查、诊断、治疗及转归,采用Logistic单因素及多因素回归分析危险因素对预后的影响,采用K多个独立样本检验及Mann-Whitney U检验分析不同治疗方案对预后的影响。结果 60例患者中,男性17例(28.3%,17/60),女性43例(71.7%,43/60),平均年龄(41±15)岁。28例患者(46.7%,28/60)表现为发热、微血管病性溶血性贫血、血小板降低三联征,23例(38.3%,23/60)表现为发热、微血管病性溶血性贫血、血小板降低、肾功能不全、神经系统异常五联征,其余9例(15.0%,9/60)无典型三联征/五联征表现。28例患者接受血管性血友病因子裂解蛋白酶13(a disintegrin and metalloproteinase with a thrombospondin type 1motif,member 13,ADAMTS13)活性检测,其中23例ADAMTS13活性<10%,阳性率为82.1%(23/28);20例患者检测了ADAMTS13抑制物,阳性率为90.0%(18/20);20例同时行ADAMTS13活性及其抑制物检测,二者均为阳性者占90.0%(18/20)。根据是否有血源、患者经济条件等不同情况,采用血浆置换、糖皮质激素、利妥昔单抗、免疫抑制剂、注射用免疫球蛋白等不同治疗方案,42例患者(70.0%,42/60)病情缓解,18例(30.0%,18/60)死亡。在血浆置换及激素治疗的基础上加用免疫抑制治疗,可将缓解率由57.1%提高至85.2%(P=0.032)。未发现导致患者死亡率增高的危险因素。结论 TTP多表现为三联征或五联征,ADAMTS13活性降低及抑制物检测阳性率高,免疫抑制治疗可改善预后。

Objective The aim of this study was to explore the clinical characteristics and prognosis of thrombotic thrombocytopenic purpura(TTP).Methods Clinical manifestations,laboratory examinations,diagnosis,therapeutic m ethods,and prognosis of60TTP patients diagnosed between2012and2017in Peking Union Medical College Hospital were retrospectively analyzed.Risk factors of prognosis were analyzed by single factor and multivariate Logistic regression analysis,and the influence of therapeutic strategieson prognosis was analyzed by K ruska l-W allis test and M ann-W hitney U test.Results Among60TTP p a tien ts,17were males(28.3%,17/60)and43were females(71.7%,43/60),aged(41±15)years o ld.Twenty-eight patients(46.7%,28/60)had T riad Syndrome including fe v e r,m icroangiopathic hem olytic anem ia,and throm bocytopenia;23patients(38.3%,23/60)had Quinary Syndrome in clud in g fe v e r,m icroangiopathic hem olytic anem ia,throm bocytopenia,renal in s u ffic ie n c y,and neurological symptoms;and the other9patients(15.0%,9/60)have neither the typical T riad Syndrome nor the Quinary Syndrome.Twenty-eight patients received the measurement for A D A M T S13a ctivity and23(82.1%,23/28)was<10%among w hom;A D A M T S13in h ib ito r was tested in20patients w ith18(90.0%,18/20)of them being po sitive;20patients received these both tests and the double positive rate was90%(18/20).D ifferent treatm ents,w hich included plasma exchange,glucocorticoids,ritu x-im a b,immunosuppresors,and intravenous im m unoglobulin,were given based on patients'fina ncial situation and blood fo u n ta in;42patients(70.0%,42/60)were relieved and18patients died(30.0%,18/60).The combined immunosuppressive therapy on the basis o f plasma exchange and glucocorticoids im proved the remission rate from57.1%to85.2%(P=0.032).No ris k factor was found between the dead group and the remission group.Conclusions Most of TTP patients manifest the T riad Syndrome or the Q uinary Syndrome.The decline of A D A M T S13a ctivity and its in h ib ito r have high positive rates in T T P.Immunosuppressive therapy can im prove the prognosis.