A Multicenter, Prospective, Randomized Controlled Study Comparing Glucocorticoid Combined With Sirolimus With Monotherapy of Glucocorticoid in the Treatment of Newly Diagnosed Mild Autoimmune Hemolytic Anemia

Summary

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.

Timeline

Start

2026-04-01

Primary completion

2028-04-01

Completion

2028-04-01

Publications

• Background 1.Birgens H, Frederiksen H, Hasselbalch HC, et al. A phase III randomized trial comparing glucocorticoid monotherapy versus glucocorticoid and rituximab in patients with autoimmune haemolytic anaemia. Br J Haematol. 2013;163(2):244-252.https://doi.org/10.1111/bjh.12541. 2.Kuter DJ. Warm autoimmune hemolytic anemia and the best treatment strategies. Hematology Am Soc Hematol Educ Program. 2022;2022(1):393-402.https://doi.org/10.1182/hematology.2022000405. 3.Murakhovskaya I, Crivera C, Leon A,et al. Healthcare resource utilization of patients with warm autoimmune hemolytic anemia initiating first line therapy of oral corticosteroids with or without rituximab. Ann Hematol. 2024;103(2):521-533.https://doi.org/10.1007/s00277-023-05613-8. 4.Berentsen S. Treatment of autoimmune hemolytic anemia: novel and investigational approaches. Minerva Med. 2025;116(2):145-158.https://doi.org/10.23736/S0026-4806.25.09617-X. 5.Ciudad M, Ouandji S, Lamarthée B, et al. Regulatory T-cell dysfunctions are associated with increase in tumor necrosis factor α in autoimmune hemolytic anemia and participate in polarization. Haematologica. 2023;108(11):3012-3025. https://doi.org/10.3324/haematol.2023.282859. 6.中华医学会血液学分会红细胞疾病（贫血）学组. 中国成人自身免疫性溶血性贫血诊疗指南（2023年版）[J]. 中华血液学杂志,2023,44(01)：12-18. DOI:10.3760/cma.j.issn.0253-2727.2023.01.003

Drugs

Indications