Using sirolimus to treat refractory warm autoimmune hemolytic anemia
Sirolimus in the Treatment of Refractory/Relapsed Warm Autoimmune Hemolytic Anemia (AIHA)： a Phase 2 Prospective Trial
This study is testing if sirolimus can help adults with tough cases of warm autoimmune hemolytic anemia who haven't improved with other treatments.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 22 (estimated) |
| Ages | 18 Years to 90 Years |
| Sex | All |
| Sponsor | Peking Union Medical College Hospital Academic / other |
| Drugs / interventions | rituximab, cyclophosphamide |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Trial ID | NCT05925023 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the efficacy and safety of sirolimus in patients with refractory or relapsed warm autoimmune hemolytic anemia (wAIHA) and Evans syndrome. The study builds on previous findings that indicated an 80% effective rate of sirolimus in similar patient populations. Participants will be adults aged 18 and older who have not responded to glucocorticoid therapy or have experienced a recurrence of their condition. The trial aims to provide a cost-effective treatment option that could improve patient outcomes and reduce the economic burden associated with this rare disorder.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with primary warm autoimmune hemolytic anemia or Evans syndrome who have not responded to glucocorticoid therapy.
Not a fit: Patients with active infections, connective tissue diseases, or other organ involvement may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients suffering from refractory wAIHA by providing an effective alternative to current therapies.
How similar studies have performed: Previous studies have shown promising results with sirolimus in similar patient populations, indicating a potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Beijing, Beijing Municipality
- Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.