Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Last updated: August 6, 2025
Sponsor: Peking Union Medical College Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hemoglobinuria, Paroxysmal

Anemia

Treatment

Sirolimus

Clinical Study ID

NCT05925023
Sirolimus-1
  • Ages 18-90
  • All Genders

Study 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 Criteria

Inclusion

Inclusion Criteria:

  1. Age ≥18 years old.

  2. Diagnosed as primary warm autoimmune hemolytic anemia or Evans syndrome (primary orsecondary). There is no treatment indication of other systemic involvement in theoriginal 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

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.

Study Design

Total Participants: 22
Treatment Group(s): 1
Primary Treatment: Sirolimus
Phase:
Study Start date:
June 24, 2023
Estimated Completion Date:
December 31, 2025

Study Description

Based on the optimal autoantibody-RBC reactivity temperatures, AIHA is classified into warm type, cold type, and mixed type. AIHA can be further classified into primary or secondary in nature. 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. The refractory/relapsed wAIHA patients have increased cardiovascular events, increased opportunities for infections, decreased quality of life, and even death. A prospective multi-institutional trial in autoimmune cytopenia found that 8 of 10 patients with AIHA and Evans syndrome respond to sirolimus. Our previous study of sirolimus in refractory/relapsed AIHA and ES also found an effective rate of approximately 80%. Since sirolimus is cheap and accessible, our findings may reduce the economic burden of patients and be a guide on the selection of second-line treatment drugs in refractory/relapsed wAIHA and Evans syndrome.

Connect with a study center

  • Peking Union Medical College Hospital

    Beijing, Beijing 100730
    China

    Active - Recruiting

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