Larue Marion, Moulis Guillaume, Rueter Manuela, Audia Sylvain, Comont Thibault, Terriou Louis, Viallard Jean-François, Pan-Petesch Brigitte, Royer Bruno, Bonnotte Bernard, Galicier Lionel, Lambotte Olivier, Lefrere François, Cheze Stéphane, Ebbo Mikael, Duong Tu-Ahn, Boutin Emmanuelle, Languille Laetitia, Mahevas Matthieu, Godeau Bertrand, Canoui-Poitrine Florence, Michel Marc
Assistance Publique Hôpitaux de Paris, Service de Médecine interne, Hôpital Henri-Mondor, Université Paris-Est Créteil, Créteil, France.
Service de Médecine Interne, Centre Hospitalier Universitaire de Toulouse, Toulouse, France.
Blood Adv. 2025 Apr 22;9(8):1976-1983. doi: 10.1182/bloodadvances.2024014939.
To assess efficacy and safety of dapsone in adult immune thrombocytopenia (ITP), a multicenter randomized controlled trial (RCT) and a real-world cohort study were performed. Participants were adults with primary ITP, transient response to corticosteroids with/without intravenous immunoglobulin, and a platelet count of ≤30 × 109/L (or ≤50 × 109/L with bleeding). Patients in the RCT were randomized in arm A (prednisone × 3 weeks + dapsone for 12 months) or arm B (prednisone alone). The observational study involved dapsone initiation at 100 mg/d with standard follow-up. The primary end point was the response rate (platelet count of >30 × 109/L and ≥2× baseline level) at 52 weeks, with the response rate at 24 weeks and adverse events as secondary end points. The RCT enrolled 93 patients (54.8% female), with median age of 48.5 years (46 years in arm A; and 47 years in arm B). In the intention-to-treat analysis, 78.3% of patients in arm A discontinued dapsone after a median of 4.6 weeks because of adverse events (66.7%) or lack of efficacy (33.3%). The response rate at week 52 was 21.7% (95% confidence interval [CI], 10.9-36.4) in arm A vs 8.5% (95% CI, 2.7-18.6) in arm B (P = .17). The observational study, which was conducted after the end of the RCT, included 46 patients (52.2% female), median age of 50.7 years. Adverse events occurred in 30.4%, leading to discontinuation of dapsone in 23.9%, and 13.6% (95% CI 5.2-27.4) met the primary efficacy end point. Results from both studies showed an unfavorable risk-benefit ratio for the use of dapsone in adult primary ITP and suggest that, whenever available, second-line options should be used. This trial was registered at www.ClinicalTrials.gov as #NCT02627417 and #NCT02877706.
为评估氨苯砜治疗成人免疫性血小板减少症（ITP）的疗效和安全性，开展了一项多中心随机对照试验（RCT）和一项真实世界队列研究。参与者为患有原发性ITP、对皮质类固醇有/无静脉注射免疫球蛋白的短暂反应且血小板计数≤30×10⁹/L（或有出血时≤50×10⁹/L）的成年人。RCT中的患者被随机分为A组（泼尼松×3周 + 氨苯砜治疗12个月）或B组（仅用泼尼松）。观察性研究包括以100mg/d起始使用氨苯砜并进行标准随访。主要终点是52周时的缓解率（血小板计数>30×10⁹/L且≥基线水平的2倍），次要终点是24周时的缓解率和不良事件。RCT纳入了93例患者（54.8%为女性），中位年龄为48.5岁（A组46岁；B组47岁）。在意向性分析中，A组78.3%的患者在中位4.6周后因不良事件（66.7%）或缺乏疗效（33.3%）停用了氨苯砜。A组52周时的缓解率为21.7%（95%置信区间[CI]，10.9 - 36.4），B组为8.5%（95%CI，2.7 - 18.6）（P = 0.17）。在RCT结束后进行的观察性研究纳入了46例患者（52.2%为女性），中位年龄为50.7岁。30.4%的患者发生了不良事件，23.9%的患者因此停用氨苯砜，13.6%（95%CI 5.2 - 27.4）达到了主要疗效终点。两项研究的结果均显示氨苯砜用于成人原发性ITP的风险效益比不佳，并表明只要有二线治疗方案，就应使用二线方案。该试验已在www.ClinicalTrials.gov上注册，注册号为#NCT02627417和#NCT02877706。
