The Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia

A Phase II, Multicenter, Open-label Trial to Determine the Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic

This is an open-label, single-arm study to evaluate the safety and efficacy of Ibrutinib in subjects with refractory/relapsed autoimmune hemolytic anemia.

Inclusion Criteria:

• ECOG ≤ 3
• Age from 6 to 70.
• Diagnosed with WAIHA or MAIHA.
• Meets the criteria of relapsed / refractory AIHA.
• Meets the criteria of DAT-negative AIHA without any other inherited or acquired hemolytic diseases, and previously treated effectively with glucocorticoids and rituximab.
• Signed informed consent.
• Organs in good function.

Exclusion Criteria:

• Nursing woman
• Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, etc.
• Diagnosed with cold agglutinin disease or cold agglutinin syndrome or paroxysmal cold hemoglobinuria.
• Secondary AIHA caused by drugs or infection.
• Received rituximab in 8 weeks before enrollment.
• Previously received treatment with BTK inhibitor.
• Previously received organ or stem cell transplantation.
• Have a history of thrombosis or organ infarction.
• Diagnosed with an active stage of connective tissue disease.
• Have a history of lymphoproliferative tumors or any other malignant tumors.
• Have other inherited or acquired hemolytic diseases.
• Received low-molecular-weight heparin or warfarin within 1 week before enrollment or during the Ibrutinib treatment.
• Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment or during the Ibrutinib treatment.
• Have a history of mental illness.
• Inability to understand or to follow study procedures.