Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder, in which rituximab (RTX) induces the best long-term effect among recommended second-line treatments. Nevertheless, the optimal regimen of RTX remains unclear. We herein conducted a prospective, multicenter, open-label, randomized controlled trial to compare the efficacy and safety of RTX at two different dosage regimens in patients with corticosteroid-resistant or relapsed ITP. Recruited patients were randomly assigned (1:1) to receive either RTX at a repeated low dose (100 mg weekly for 4 weeks, LD-RTX) or at a single dose (375 mg/m², S-RTX). Overall response was achieved in 64.3% of patients who received LD-RTX versus 67.4% of those receiving S-RTX (p = .759). The complete response (CR) rate was 23.8% after LD-RTX and 28.3% after S-RTX (p = .635). In health-related quality of life, S-RTX improved patients' psychological status, quality of life, social activities, and work compared with LD-RTX. Furthermore, S-RTX significantly reduced physician visits without compromising efficacy. Our findings demonstrate that a S-RTX is comparable to LD-RTX in effectiveness and safety for treatment of corticosteroid-resistant or relapsed ITP. The single-dosage regimen optimizes the use of medical resources, improves the cost-effectiveness of RTX, and represents a promising and more convenient replacement for LD-RTX in ITP. This study has been completed and is registered with ClinicalTrials.gov, number NCT03258866.

中文翻译：

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单剂量与低剂量利妥昔单抗治疗皮质类固醇耐药或复发性 ITP：一项多中心、随机、对照研究

原发性免疫性血小板减少症 (ITP) 是一种自身免疫性出血性疾病，其中利妥昔单抗 (RTX) 在推荐的二线治疗中可诱导最佳的长期效果。然而，RTX 的最佳方案仍不清楚。我们在此开展了一项前瞻性、多中心、开放标签、随机对照试验，以比较 RTX 在两种不同剂量方案中对皮质类固醇耐药或复发性 ITP 患者的疗效和安全性。招募的患者被随机分配 (1:1) 接受重复低剂量 RTX（每周 100 mg，持续 4 周，LD-RTX）或单剂量（375 mg/m ²，S-RTX）。接受 LD-RTX 的患者有 64.3% 获得了总体反应，而接受 S-RTX 的患者为 67.4%（p = .759)。LD-RTX 后的完全缓解 (CR) 率为 23.8%，S-RTX 后为 28.3% ( p  = .635)。在与健康相关的生活质量方面，与 LD-RTX 相比，S-RTX 改善了患者的心理状态、生活质量、社会活动和工作。此外，S-RTX 在不影响疗效的情况下显着减少了就诊次数。我们的研究结果表明，在治疗皮质类固醇耐药或复发性 ITP 的有效性和安全性方面，S-RTX 与 LD-RTX 相当。单剂量方案优化了医疗资源的使用，提高了 RTX 的成本效益，是 ITP 中 LD-RTX 的一种有希望且更方便的替代品。该研究已完成并在 ClinicalTrials.gov 注册，编号为 NCT03258866。