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.
This study was a randomized controlled study. Two groups of patients were randomly assigned in a 1:1 ratio to receive the following treatment regimens: 1. Glucocorticoid monotherapy group Prednisone at a dose of 1-2 mg/kg/d (or an equivalent dose of methylprednisolone) is taken orally for 2-3 weeks, and then the dosage is reduced regularly. The total course of treatment is approximately 3-6 months. The reduction plan was formulated by the researchers based on clinical practice. 2. Glucocorticoid combined with sirolimus group Glucocorticoids: The usage, dosage and reduction regimen are the same as those in the monotherapy group. \| Sirolimus: Starting dose 1 mg/d, orally, once a day. After reaching a steady state 7 to 14 days of medication, monitor the trough concentration. The target trough concentration range is 4 to 12 ng/mL. Adjust the dosage based on the results of blood drug concentration monitoring. The total course of treatment for sirolimus is 6 months. If grade ≥3 related adverse events or trough concentration \> occur; 12 ng/mL. Consider reducing the dosage or suspending the administration
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
216
Glucocorticoids combined with sirolimus
Peking union medical college hospital
Beijing, Shuangfuyuan, NO I., China
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.
Time frame: 12 months
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
Time frame: 1,3,6,12 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.