Department of Rheumatology and Immunology, Drum Tower Hospital, Clinical College of Nanjing Medical University, Nanjing, 210008, People's Republic of China.
Clin Rheumatol. 2018 Dec;37(12):3337-3344. doi: 10.1007/s10067-018-4312-y. Epub 2018 Oct 2.
To analyze the clinical characteristics of severe thrombocytopenia in patients with various connective tissue diseases (CTDs), one hundred thirty-one consecutive CTD patients with blood platelet count less than 20 × 10/L on admission, which was ascribed to the nature of diseases, during January 2011 to June 2015 in our department were enrolled and checked for their survival status in September 2015. The patients were categorized based on background diseases or therapeutic effects, and compared with clinical features, treatment strategies, and long-term outcomes among the groups. Cumulative survival rates were estimated using Kaplan-Meier analysis. Of the patients, 88.5% were female. The most frequently seen background diseases were primary Sjögren's syndrome (pSS) (53.4%) and systemic lupus erythematosus (SLE) (40.5%). Age on admission for SLE patients (36.7 ± 14.1 years) was much younger than that for other patients (44.4 ± 15.4 years for pSS and 46 ± 16.1 years for other CTDs, p < 0.05). Ninety-six cases accompanied with various bleeding symptoms, which were more common in pSS patients than in SLE patients (80.0% vs. 64.2%, p < 0.05). Glucocorticoids and/or intravenous immunoglobulin were applied as initial therapy with an overall response rate of 36.6%. For patients failed to respond, immunosuppressive drugs were added and the other 22.8% benefited from the treatment. Compared to those ineffective to the aforementioned drugs, patients with therapeutic effects had significantly high immunoglobulin G levels. Twenty patients with refractory diseases accepted mesenchymal stem cell transplantation (MSCT) with a total effective rate of 65.0%. Eleven patients died after the follow-up for a mean time of 27.7 months, of which 7 were associated with hemorrhage. There was no difference in the survival rate among different background diseases. However, compared with those who did not gain remission, patients achieved partial or complete remission had better cumulative survival rates (p < 0.01). In conclusion, among various CTDs, severe thrombocytopenia often occurs in patients with SLE or pSS. Early response to the treatments, but not the background disease, is an important predictor of long-term prognosis. For patients with refractory thrombocytopenia, MSCT may provide an alternative therapeutic strategy.
为了分析各种结缔组织病（CTD）患者严重血小板减少症的临床特征，我们回顾性分析了 2011 年 1 月至 2015 年 6 月期间本科 131 例入院时血小板计数＜20×10/L 的 CTD 患者的临床资料，这些患者的血小板减少均归因于疾病本身。并于 2015 年 9 月对患者的生存状况进行了随访。根据基础疾病或治疗效果对患者进行分组，并比较各组间的临床特征、治疗策略和长期预后。采用 Kaplan-Meier 分析估计累积生存率。患者中 88.5%为女性。最常见的基础疾病为原发性干燥综合征（pSS）（53.4%）和系统性红斑狼疮（SLE）（40.5%）。SLE 患者的入院年龄（36.7±14.1 岁）明显低于其他患者（pSS 为 44.4±15.4 岁，其他 CTD 为 46±16.1 岁，p＜0.05）。96 例伴有各种出血症状，pSS 患者比 SLE 患者更常见（80.0%比 64.2%，p＜0.05）。糖皮质激素和/或静脉注射免疫球蛋白作为初始治疗，总有效率为 36.6%。对于治疗无效的患者，加用免疫抑制剂，其余 22.8%的患者受益于治疗。与上述药物无效的患者相比，治疗有效的患者免疫球蛋白 G 水平明显升高。20 例难治性疾病患者接受间充质干细胞移植（MSCT），总有效率为 65.0%。11 例患者在随访 27.7 个月后死亡，其中 7 例与出血有关。不同基础疾病的生存率无差异。然而，与未缓解的患者相比，部分或完全缓解的患者具有更好的累积生存率（p＜0.01）。总之，在各种 CTD 中，严重血小板减少症常发生于 SLE 或 pSS 患者。早期对治疗的反应，而不是基础疾病，是长期预后的重要预测因素。对于难治性血小板减少症患者，MSCT 可能提供一种替代治疗策略。
