Severe thrombocytopenia in connective tissue diseases: a single-center review of 131 cases

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 × 10⁹/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.