This study is a prospective, multicenter, randomized controlled trial. A total of 216 adult patients with newly diagnosed wAIHA were planned to be included and randomly assigned in a 1:1 ratio to the experimental group (glucocorticoid combined with sirolimus) or the control group (glucocorticoid monotherapy). The initial dose of sirolimus in the experimental group was 1mg/d, adjusted according to the blood drug concentration. The target concentration was 4-12ng/mL, and the treatment course was 6 months. Both groups of hormones were gradually reduced according to the standard protocol. All patients were followed up for 24 months, and the differences between the two groups at endpoints such as the hormone-free sustained response rate at the 12th month were compared.
Age ≥18 years old, gender not limited;
Diagnosed as newly diagnosed wAIHA and requiring systemic immunosuppressive therapy (those with short-term hormone exposure of ≤2 weeks before enrollment can be included)
After a comprehensive rheumatology and immunology assessment, there is no evidence of clinical organ involvement in SLE or other connective tissue diseases (only serological abnormalities can be included).
Positive Coombs test (IgG type, IgG+C3d type, or only C3d type with a condensed agglutinin titer of 1:64);
Pregnant or lactating patients;
Clinically confirmed SLE or other definite connective tissue diseases;
Secondary AIHA secondary to lymphoproliferative diseases, other hematological malignancies, solid tumors, infections, and drugs;
Cold-resistant type or hybrid type AIHA;
Parallel
Glucocorticoids
DrugGlucocorticoids combined with sirolimus
The 12-month sustained remission rate
Defined as the proportion of patients who simultaneously met the following conditions at the 12th month after randomization: Discontinue glucocorticoids (or only use prednisone ≤5 mg/d or an equivalent dose for maintenance); * Has not received any follow-up treatment for wAIHA; * Still in complete remission (CR) or partial remission (PR) status.
Safety
1. The incidence of adverse events (AE), serious adverse events (SAE), and grade ≥3 AE; 2. Adverse events of particular concern: infections (especially opportunistic infections), metabolic disorders (new-onset diabetes, dyslipidemia), bleeding events, hormone-related adverse events (Cushing's syndrome, hypertension, osteoporosis, etc.); 3. The proportion of patients who discontinued medication, reduced dosage or interrupted treatment due to adverse events
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Chen Miao
Lead sponsor
Peking Union Medical College Hospital
Sponsor institution
This trial is not recruiting at the moment. You can still explore other options:
