A Randomized and Double-Blind Controlled Trial Evaluating the Safety and Efficacy of Rituximab for Warm Auto-Immune Hemolytic Anemia in Adults (the RAIHA study)

This phase 3 multicentre randomized double-blind and placebo-controlled trial aimed to compare the efficacy and safety of rituximab (RTX) to placebo for treating newly diagnosed warm autoimmune hemolytic anemia (wAIHA) in adults receiving prednisone. Adults with a confirmed diagnosis of wAIHA who previously received corticosteroids for less than 6 weeks could be included. At inclusion, all patients received prednisone at a daily dose of 1 mg/kg for 2 weeks, then tapered according to a pre-defined recommended reduction scheme. Besides prednisone, eligible patients received 2 infusions of RTX or placebo at a fixed dose of 1,000 mg 2 weeks apart. The primary endpoint was overall response rate (complete response [CR] + partial response [PR]) in an intent-to-treat (ITT) analysis at 1 year. A total of 32 patients (17 females [53%], mean age at inclusion 71±16 years) were enrolled and randomized. In all, 27 patients were followed for at least 1 year and their data were evaluable for response. With an ITT analysis, the overall response rate at 1 year was 75% [95%CI: 47.6-92.7] with 11 CR and 1 PR with RTX versus 31% [11.0-58.7] (5 CR) with placebo (p=0.032). At 2 years, 10/16 patients with RTX versus 3/16 with placebo still showed CR (p=0.011). Overall, 8 severe infections occurred during follow-up, 6 with placebo and 2 with RTX (p=0.39). At 2 years, 6 patients with placebo had died, but none with RTX (p=0.017). Compared to placebo, RTX combined with prednisone may be effective and safe for treating newly-diagnosed wAIHA in adults. This article is protected by copyright. All rights reserved.