The following is a summary of “Therapeutic modalities and clinical outcomes in a large cohort with LRBA deficiency and CTLA4 insufficiency,” published in the December 2023 issue of Allergy & Immunology by Taghizade, et al.
LPS-responsive beige-like anchor (LRBA) deficiency (LRBA–/–) and cytotoxic T-lymphocyte–associated antigen-4 (CTLA4) insufficiency (CTLA4+/–) are diseases sharing recurrent infections and autoimmunity. Optimal treatment strategies remain uncertain. For a study, researchers sought to compare the efficacy and long-term outcomes of immunosuppressants, CTLA4-immunoglobulin (abatacept), and hematopoietic stem cell transplantation (HSCT) in a 98-patient single-country multicenter cohort with a median 5-year follow-up.
Ninety-eight patients (63 LRBA–/–, 35 CTLA4+/–) were monitored every 6 months for clinical manifestations and treatment response.
LRBA–/– patients experienced a more severe disease course, requiring more immunosuppressants, abatacept, and HSCT. Among the 58 patients receiving abatacept, 79.3% achieved sustained complete control without severe side effects. In contrast, 72.1% of those primarily treated with immunosuppressants showed partial or no disease control, often needing additional therapies. Patients with partial or no response to abatacept had longer disease activity, more organ involvement, and poorer outcomes. HSCT in 14 LRBA–/– patients resulted in 64.2% achieving complete remission, with 21.3% still requiring immunosuppressants post-transplantation. HSCT and abatacept therapy exhibited similar survival probabilities.
Abatacept demonstrated superior long-term disease control compared to immunosuppressants, particularly when initiated early. It represented a safe and effective alternative to transplantation, offering a valuable therapeutic option for LRBA–/– and CTLA4+/– patients.
Reference: jacionline.org/article/S0091-6749(23)01017-5/fulltext