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摘要:
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 1 motif, 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。 -
关键词:
- 血栓性血小板减少性紫癜 /
- 血浆置换 /
- 糖皮质激素 /
- 免疫抑制治疗
Abstract: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 methods, and prognosis of 60 TTP patients diagnosed between 2012 and 2017 in 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 Kruskal-Wallis test and Mann-Whitney U test. Results Among 60 TTP patients, 17 were males (28.3%, 17/60) and 43 were females (71.7%, 43/60), aged (41±15) years old. Twenty-eight patients (46.7%, 28/60) had Triad Syndrome including fever, microangiopathic hemolytic anemia, and thrombocytopenia; 23 patients (38.3%, 23/60) had Quinary Syndrome including fever, microangiopathic hemolytic anemia, thrombocy-topenia, renal insufficiency, and neurological symptoms; and the other 9 patients (15.0%, 9/60) have neither the typical Triad Syndrome nor the Quinary Syndrome. Twenty-eight patients received the measurement for ADAMTS13 activity and 23 (82.1%, 23/28) was < 10% among whom; ADAMTS13 inhibitor was tested in 20 patients with 18 (90.0%, 18/20) of them being positive; 20 patients received these both tests and the double positive rate was 90% (18/20). Different treatments, which included plasma exchange, glucocorticoids, rituximab, immunosuppresors, and intravenous immunoglobulin, were given based on patients' financial situation and blood fountain; 42 patients (70.0%, 42/60) were relieved and 18 patients died (30.0%, 18/60). The combined immunosuppressive therapy on the basis of plasma exchange and glucocorticoids improved the remission rate from 57.1% to 85.2% (P=0.032). No risk factor was found between the dead group and the remission group. Conclusions Most of TTP patients manifest the Triad Syndrome or the Quinary Syndrome. The decline of ADAMTS13 activity and its inhibitor have high positive rates in TTP. Immunosuppressive therapy can improve the prognosis. -
表 1 60例TTP患者危险因素的单因素分析
危险因素 例数(n) 发生率(%) OP值 95% CI Wald值 P值 男性 13 21.67 1.409 0.426~4.666 0.315 0.575 年龄＞50岁 21 35.00 3.523 1.108~11.198 4.554 0.033 住院时间＞30 d 17 28.33 0.096 0.012~0.789 4.752 0.029 肾功能不全 35 58.33 1.179 0.382~3.640 0.082 0.775 神经系统症状 51 85.00 1.600 0.298~8.577 0.301 0.583 获得性病因 32 53.33 1.571 0.511~4.837 0.621 0.431 血小板＜10×109/L 25 41.67 1.625 0.533~4.952 0.729 0.393 总胆红素＞44 μmol/L 21 35.00 1.273 0.405~3.997 0.171 0.680 间接胆红素＞25 μmol/L 23 38.33 1.471 0.484~4.465 0.463 0.496 乳酸脱氢酶＞1000 U/L 23 38.33 1.500 0.441~5.100 0.422 0.516 肾脏替代治疗 10 16.67 1.000 0.227~4.400 0 1 
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表 2 联用免疫抑制治疗对患者转归的影响
治疗方案 例数(n) 缓解(n) 死亡(n) 缓解率(%) 未联用组(方案Ⅰ) 21 12 9 57.1 联用组(方案Ⅱ) 27 23 4 85.2 P值 0.032
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表 3 采用血浆置换对患者转归的影响
治疗方案 例数(n) 缓解(n) 死亡(n) 缓解率(%) 血浆置换组 50 37 13 74.0 非血浆置换组 10 6 4 26.0 P值 0.374
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