伊布替尼治疗复发/难治原发性自身免疫性溶血性贫血二例——探索性研究

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.