a multicenter prospective study of iptacopan in the treatment of refractory/relapsed autoimmune hemolytic anemia (aiha)

Conditions: AIHA - Warm Autoimmune Hemolytic Anemia; AIHA - Cold Autoimmune Hemolytic Anemia

Summary: to evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed aiha.


Eligibility criteria:
inclusion criteria: 1. voluntarily signed an informed consent form (icf); 2. males or females aged 18 or older; 3. physical status score \[eastern cooperative oncology group (ecog) score\] ≤2; 4. confirmed diagnosis of primary aiha or secondary autoimmune disease (except rheumatoid arthritis and systemic lupus erythematosus) with underlying disease in a stable state; 5. poor response to at least previous glucocorticoid therapy, including ineffective (defined as failure to achieve stabilization of hb levels at 100 g/l or erythrocyte hematocrit \<30% despite at least 4 weeks of treatment with previously recommended doses), or glucocorticoid-dependent (defined as maintenance of equal doses of prednisone exceeding 15 mg/d), or relapsed (defined as treatment that is effective and then again has an hb of \<100 g/l or an erythrocyte hematocrit of \<30%), or otherwise contraindicated. 30%), or otherwise contraindicated or intolerant to glucocorticoid therapy; 6. hemoglobin (hb) \<100 g/l before drug administration; 7. positive direct anti-human globulin test (dat) (iga, igm or igg+, with or without c3+). 8. combination of one anti-aiha therapy \[glucocorticoids only (≤15 mg prednisone equivalent), immunosuppressants (azathioprine, cyclosporine, and merti-macrolide only)\] is permitted in this study, provided that the dose has been stable for at least 28 days prior to enrollment; 9. laboratory tests meet the following criteria (no treatment for the abnormality of the index within 2 weeks prior to blood collection, or no long-acting g-csf treatment within 2 weeks) 1. neutrophil count \>1.5×109/l and platelet \>30×109/l; 2. alt and ast ≤ 2 × uln; 3. serum creatinine concentration ≤ 2 × uln and creatinine clearance ≥ 50ml/min; 10. no active infection; no pregnancy or lactation; 11. caiha patients presenting with skin cyanosis and thrombosis; 12. written evidence of neisseria meningitidis and streptococcus pneumoniae vaccinations within 2 years or, if none, antibiotic prophylaxis until 2 weeks after completion of vaccination. exclusion criteria: 1. presence of secondary aiha outside the inclusion criteria; 2. hb \<100 g/l due to non-aiha factors; and 3. infections requiring systemic therapy; 4. those with a past history of malignancy (except cured basal cell carcinoma of the skin or cervical carcinoma in situ); 5. with history of vital organ transplantation or hematopoietic stem cell/bone marrow transplantation; 6. those who have undergone splenectomy within 24 weeks prior to enrollment; 7. those who have had major surgery within four weeks prior to enrollment or who require major elective surgery during the study period; 8. history of severe cardiovascular disease \[e.g., class iii/iv congestive heart failure, arrhythmia or angina requiring medication, unstable angina, coronary stenting, angioplasty or coronary artery bypass grafting, or corrected q-t interval (qtcf) ≥ 90 mmhg\] 9. patients with medically uncontrolled hypertension (systolic blood pressure ≥140 mmhg or diastolic blood pressure ≥90 mmhg); or comorbid portal hypertension; 10. patients with severe gastrointestinal disorders such as dysphagia, active gastric ulcers, etc., who are unable to take drugs orally or have impaired absorption of oral drugs; 11. human immunodeficiency virus (hiv) infection 12. uncontrolled or active hbv infection \[hepatitis b surface antigen (hbsag) or hepatitis b core antibody (hbcab) positive patients, need to confirm hepatitis b virus deoxyribonucleic acid (hbv dna) positive\]; or hepatitis c \[patients with hepatitis c virus ribonucleic acid (hcv rna) positive patients\]; or cirrhosis of the liver; 13. those who had received herbal treatment within one week prior to enrollment that interfered with the assessment of efficacy; 14. patients with severe psychological or psychiatric abnormalities; 15. alcohol or drug abusers; 16. female patients who are pregnant or breastfeeding; 17. patients who, in the opinion of the investigator, are not suitable for participation in this study.