Sirolimus in the Treatment of Refractory/Relapsed Warm Autoimmune Hemolytic Anemia (AIHA)： a Phase 2 Prospective Trial

Sirolimus-1

Autoimmune hemolytic anemia (AIHA) is a rare and heterogeneous disorder characterized by the destruction of red blood cells through warm or cold antibodies. Glucocorticoid (combined with rituximab) is the first-line treatment. However, the recurrence rate is very high and some patients may not respond to steroids. Second-line therapies include cyclosporine A (CsA), cyclophosphamide, rituximab, azathioprine, and even splenectomy. Our previous study of sirolimus in refractory/relapsed AIHA and ES found an effective rate of 80%. Therefore, the investigators plan to explore the efficacy and safety of sirolimus in the treatment of refractory/relapsed wAIHA.

2023-06-24

2024-12-01

2025-12-01

Recruiting

Early phase I

22

Inclusion Criteria: 1. Age ≥18 years old. 2. Diagnosed as primary warm autoimmune hemolytic anemia or Evans syndrome (primary or secondary). There is no treatment indication of other systemic involvement in the original disease if secondary. 3. No response to glucocorticoid therapy or recurrence. 4. Baseline liver (ALT, AST) was less than 2 times the normal value. 5. No active infection; Not pregnant or breastfeeding. 6. Agree to sign the consent form. Exclusion Criteria: 1. Patients with connective tissue disease or other organs involvement 2. Infection or bleeding that cannot be controlled by standard treatment. 3. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension. 4. Progressed uncontrolled malignant tumors and lymphoma 5. Cirrhosis or portal hypertension. 6. Pregnant or breastfeeding.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 22, 'type': 'ESTIMATED'}}

2023-08-21