Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · Jun 21, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called sirolimus to treat a condition known as warm autoimmune hemolytic anemia (wAIHA), which is when the body’s immune system mistakenly destroys its own red blood cells. The standard treatment often involves steroids, but many patients either do not respond to these treatments or see their symptoms come back. The researchers previously found that sirolimus was effective for many patients with similar conditions, so they want to see if it can also help those with wAIHA who have not responded well to other treatments.

To participate in this trial, you must be at least 18 years old and have been diagnosed with wAIHA or a related condition. You also should not have responded to steroid treatment or should have had your symptoms return after treatment. Participants will be monitored closely throughout the study to ensure their safety and to see how well sirolimus works. Moreover, those who are pregnant, breastfeeding, or have certain infections or health issues may not be eligible. If you are considering joining, you'll need to sign a consent form to confirm your participation.

Gender

ALL

Eligibility 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.