Acute graft-versus-host disease (aGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT) for inborn errors of immunity (IEI).
We retrospectively analyzed all pediatric patients who underwent allogeneic HSCT for IEI at the Pediatric Hematology Department, Bone Marrow Transplantation Center, Tunis, between January 2018 and March 2023. GVHD prophylaxis consisted of cyclosporine with short-course methotrexate for HLA-matched transplants, and post-transplant cyclophosphamide with mycophenolate mofetil and cyclosporine for haploidentical transplants. aGVHD was graded according to modified Glucksberg criteria.
37 children (median age 12 months) were included: 23 with combined immunodeficiencies, 7 with congenital phagocyte defects, 4 with immune dysregulation, and 2 with osteopetrosis. 10 patients had active infection at transplantation. Donors were haploidentical in 20 and genoidentical in 17; 26 cases involved sex mismatch. Conditioning was myeloablative in 35 patients and reduced-intensity in 2. Serotherapy with anti-lymphocyte globulin was given in 12 cases. Median CD34+ cell dose was 6.1 × 106/kg. Four patients had primary graft failure. Among evaluable patients, grade II–IV aGVHD occurred in 12 (36.6%), including 2 grade III–IV cases. Incidence was 37.5% in haploidentical and 35.2% in genoidentical transplants. 10 patients responded to corticosteroids; one required ruxolitinib. No clinical or transplant-related factors were significantly associated with aGVHD. Overall survival was 50% in patients with grade II–IV aGVHD versus 100% in those without (p = 0.4).
aGVHD remains a frequent complication after HSCT for IEI, affecting one-third of patients, with substantial impact on survival. Early recognition and prompt management are essential, and new strategies to prevent and treat severe aGVHD are needed in this high-risk population.7.
