Sirolimus in the Treatment of Refractory/Relapsed wAIHA
Sirolimus in the Treatment of Refractory/Relapsed Warm Autoimmune Hemolytic Anemia (AIHA)： a Phase 2 Prospective Trial
Peking Union Medical College Hospital
22 participants
Jun 24, 2023
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria6
- Age ≥18 years old.
- 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.
- No response to glucocorticoid therapy or recurrence.
- Baseline liver (ALT, AST) was less than 2 times the normal value.
- No active infection; Not pregnant or breastfeeding.
- Agree to sign the consent form.
Exclusion Criteria6
- Patients with connective tissue disease or other organs involvement
- Infection or bleeding that cannot be controlled by standard treatment.
- Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- Progressed uncontrolled malignant tumors and lymphoma
- Cirrhosis or portal hypertension.
- Pregnant or breastfeeding.
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Interventions
Oral administration, 1-3 mg/d, sirolimus plasma concentration: 4-15 ng/mL
Locations(1)
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NCT05925023