CTLA4-related primary immune dysregulatory disorders

Tailored immunomodulation using abatacept and sirolimus improves disease control and quality of life in LRBA deficiency and CTLA-4 insufficiency. Early diagnosis, multidisciplinary care, and timely HSCT in high-risk patients are critical for optimal outcomes. Ongoing research into gene therapy and artificial intelligence-driven care pathways offers hope for durable, personalized treatment strategies.