Sirolimus for refractory/relapsed warm autoimmune hemolytic anemia and Evans syndrome: a prospective study.
Blood AdvSep 12, 2026
Clinical ResearchHematology
Qi Wang, Ziwei Liu, Chen Yang et al.
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In 30 seconds
This prospective, single-center clinical trial evaluated sirolimus in 78 adults with refractory or relapsed warm autoimmune hemolytic anemia (wAIHA) and Evans syndrome (ES). The study found a 6-month overall response rate of 80.7% and a complete response rate of 51.3%. Sirolimus demonstrated durable responses and a favorable safety profile over a median follow-up of 13.9 months.
Key findings
- The overall response rate at 6 months was 80.7%, with 81.0% for wAIHA and 80.0% for ES.
- The complete response rate at 6 months was 51.3%, with 52.4% for wAIHA and 46.7% for ES.
- Sustained responses at 12 months showed an overall response rate of 78.2% and a complete response rate of 50.0%.
- Treatment-emergent adverse events occurred in 28.2% of patients, primarily mild and reversible.
Why it matters
The study highlights sirolimus as a potentially effective treatment for adults with refractory or relapsed wAIHA and ES, offering a steroid-sparing option that may improve patient outcomes in this challenging population.