Primary immune thrombocytopenia (ITP) is a bleeding disorder commonly encountered in clinical practice. The International Working Group (IWG) on ITP has published several landmark papers on terminology, definitions, outcome criteria, bleeding assessment, diagnosis, and management of ITP. The Chinese consensus reports for diagnosis and management of adult ITP have been updated to the 4th edition. Based on current consensus positions and new emerging clinical evidence, the thrombosis and hemostasis group of the Chinese Society of Hematology issued Chinese guidelines for management of adult ITP, which aim to provide evidence-based recommendations for clinical decision making.

Background Hetrombopag, a novel thrombopoietin receptor agonist, has been found in phase I studies to increase platelet counts and reduce bleeding risks in adults with immune thrombocytopenia (ITP). This phase III study aimed to evaluate the efficacy and safety of hetrombopag in ITP patients. Methods Patients who had not responded to or had relapsed after previous treatment were treated with an initial dosage of once-daily 2.5 or 5 mg hetrombopag (defined as the HETROM-2.5 or HETROM-5 group) or with matching placebo in a randomized, double-blind, 10-week treatment period. Patients who received placebo and completed 10 weeks of treatment switched to receive eltrombopag, and patients treated with hetrombopag in the double-blind period continued hetrombopag during the following open-label 14-week treatment. The primary endpoint was the proportion of responders (defined as those achieving a platelet count of ≥ 50 × 109/L) after 8 weeks of treatment. Results The primary endpoint was achieved by significantly more patients in the HETROM-2.5 (58.9%; odds ratio [OR] 25.97, 95% confidence interval [CI] 9.83–68.63; p < 0.0001) and HETROM-5 (64.3%; OR 32.81, 95% CI 12.39–86.87; p < 0.0001) group than in the Placebo group (5.9%). Hetrombopag was also superior to placebo in achieving a platelet response and in reducing the bleeding risk and use of rescue therapy throughout 8 weeks of treatment. The durable platelet response to hetrombopag was maintained throughout 24 weeks. The most common adverse events were upper respiratory tract infection (42.2%), urinary tract infection (17.1%), immune thrombocytopenic purpura (17.1%) and hematuria (15%) with 24-week hetrombopag treatment. Conclusions In ITP patients, hetrombopag is efficacious and well tolerated with a manageable safety profile. Trial registration Clinical trials.gov NCT03222843, registered July 19, 2017, retrospectively registered.

Immune thrombocytopenia (ITP), often diagnosed in the elderly, is a hematologic disorder induced by autoimmune mechanism. In this retrospective study, we evaluated the clinical features, the risk of bleeding, and the response to treatment in 525 elderly ITP patients (age ≥60 years) diagnosed at our center from 1980 to 2009. There were more females at 60-74 years of age (P = 0.044). The median duration of follow-up was 27 months (range 1-253 months). Ten patients developed thrombosis during treatment of ITP. At diagnosis, 461 patients (87.8 %) had signs of bleeding. The risk of severe bleeding was associated with both platelet count (P < 0.001; odds ratio (OR), 0.973) and age (P = 0.025; OR, 1.039). The cutoff points in the platelet count at which bleeding and severe bleeding would begin to appear were 29.5 × 10(9) and 21.5 × 10(9)/L, respectively. Sixteen of 144 patients (11.1 %) who did not receive any treatment achieved remission spontaneously. The total response rate to treatment was 62.4 % (166/266). The median time to remission was 7 days, and combined use of intravenous immunoglobulin and steroids took effect faster than use of steroids alone (P = 0.001). Fifty-two patients (31.3 %) relapsed during follow-up. Of the 27 patients who died during follow-up, seven deaths were directly attributed to ITP. In conclusion, the response rate has been improved since the last 10 years. ITP is also a self-limited disease to some extent in the elderly, but easy to relapse. This review represents the largest collection of elderly ITP patients in China in a single center.

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