The Treatment of Steroid-Refractory Severe Gastrointestinal Acute Graft-Versus-Host Disease in Children after Allogeneic Hematopoietic Stem Cell Transplantation: A Single-Centre Experience

Acute graft-versus-host disease is a common complication of allogeneic hematopoietic stem cell transplantation and is a major cause of morbidity and mortality. Systemic steroid therapy is the first-line treatment for acute graft-versus-host disease, although about half of patients will become refractory to treatment. We aimed to evaluate treatment options by reviewing available alternatives for patients with steroid-refractory acute graft-versus-host disease by comparing data from recently published studies. We retrospectively studied the safety and efficacy of treatment in 22 children with steroid‐dependent/refractory acute GVHD between the years 2010 and 2023. Seven (31.8%) out of 22 patients with acute graft-versus-host disease were still alive. The seven surviving patients have been followed for an average of 1141 (±403) days. 15 non-responders with grade III/IV acute graft-versus-host disease died from causes associated with acute graft-versus-host disease and/or other conditions. Among 15 patients who died, the number of patients who received all three treatments, MSC, ECP and infliximab, was 5 (33%). The most common cause of death was infection (8 cases in 15 patients, 53.3%). Other causes of death were gastrointestinal hemorrhages (n=5, 33.3%), and intracranial hemorrhages (n=2, 13.3%). When factors causing morbidity were evaluated, it was observed that three patients developed posterior reversible encephalopathy syndrome, two patients developed hepatic veno-occlusive disease, and one patient developed hypertension. The estimated probability of survival after 1 year was 31.8%, and the median survival was 655 days. We believe that it would be crucial to show the safety and efficacy of novel treatments in comprehensive, randomized clinical trials.