Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-24

Study Completion Date

2025-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Plasma Exchange Combination of Immunosuppressive Regimens for Auto-immune Hepatitis

NCT02874586

Hepatitis, Autoimmune

UNKNOWN NA

Study on Pharmacokinetics of Meperizumab Injection and NUCALA® in Healthy Male Volunteers

NCT05353179

Granulomatosis With Polyangiitis Hemophagocytic Syndrome

UNKNOWN EARLY_PHASE1

Establishment of an Early Warning Screening System for Hemophagocytic Lymphohistiocytosis a Multi-center, Prospective Study

NCT06614998

Hemophagocytic Syndrome

RECRUITING

Rituximab in Refractory Primary Membranous Nephropathy

NCT03880643

Glomerulonephritis, Membranous

COMPLETED

A Trial on the Recurrence Free Survival Rate of Type I AIP With High-risk of Recurrence

NCT06468709

Type 1 AIP

RECRUITING PHASE4

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Warm Autoimmune Hemolytic Anemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

Oral administration, 1-3 mg/d, sirolimus plasma concentration: 4-15 ng/mL

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Sirolimus

Oral administration, 1-3 mg/d, sirolimus plasma concentration: 4-15 ng/mL

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No