Objective: To explore the efficacy and safety of ibrutinib treatment for relapsed/refractory (R/R) primary autoimmune hemolytic anemia (AIHA) . Methods: Two cases of primary AIHA with relapse events were refractory to glucocorticoid, anti-CD20 monoclonal antibody, immunosuppressive drugs, and splenectomy (case 1 only) . Ibrutinib treatment was administered at an initial dose of 280 mg/d (420 mg/d for case 1 from the 3rd to 8th week) . Results: Both patients achieved transfusion independence and HGB>20 g/L above baseline after 2 weeks (partial response) . For case 1, HGB concentration restored to 113 g/L but with incomplete hemolysis recovery after 10 weeks; HGB reached the level of 118 g/L, also with incomplete hemolysis recovery, after 6 weeks in case 2. They all acquired complete response with incomplete hemolysis recovery (CRi) . The responses sustained 14 weeks and 10 weeks after follow-up at 16 weeks and 12 weeks, respectively. During the treatment, hematologic and nonhematologic toxicity is mild and acceptable. Conclusion: Ibrutinib alone is effective for the 2 R/R primary AIHA cases. We need further clinical trial to identify its efficacy and safety.
目的： 探索伊布替尼治疗复发/难治原发性自身免疫性溶血性贫血（AIHA）的疗效及安全性。 方法： 2例原发性AIHA患者多次复发，1例有脾切除史，经糖皮质激素、利妥昔单抗及多种免疫抑制药物治疗无效，诊断为复发/难治原发性AIHA。给予伊布替尼起始剂量280 mg/d（例1第3~8周加量到420 mg/d）治疗，观察疗效及安全性。 结果： 例1治疗2周后脱离输血，HGB增长>20 g/L，获得部分疗效；10周后HGB 113 g/L伴有代偿性溶血实验室改变，获得完全缓解伴代偿性溶血状态（CRi）。例2治疗2周后脱离输血，HGB增长>20 g/L，获得部分疗效；6周后HGB 118 g/L伴有代偿性溶血实验室改变，获得CRi。2例患者分别随访16周和12周，已维持疗效14周和10周，未复发。伊布替尼治疗期间2例患者血液学不良反应轻，非血液学不良反应轻微。 结论： 伊布替尼单药治疗复发/难治原发性AIHA有初步疗效，尚需规范的临床研究试验进一步验证其疗效及安全性。.
Keywords: Autoimmune hemolytic anemia; Ibrutinib; Relapsed/refractory.