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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Not Applicable
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 90
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
China
Peking Union Medical College Hospital
RECRUITING
Beijing
Time Frame
Start Date: 2023-06-24
Estimated Completion Date: 2025-12
Participants
Target number of participants: 22
Treatments
Experimental: Sirolimus on refractory/relapsed wAIHA
A prospective research of the sirolimus efficiency on refractory/relapsed primary wAIHA patients. Sirolimus dosage: 1-3 mg/d with plasma concentration 4-15ng/mL. Medication time should last at least 6 months. After reaching the optimal response, responders continue to use sirolimus for 1 year, and then gradually reduce the dosage.
Sponsors
Leads: Peking Union Medical College Hospital

This content was sourced from clinicaltrials.gov