Introduction
Allogeneic hematopoietic cell transplantation is used to treat a variety of malignant and non-malignant hematologic disorders. (1,2) In patients with NMD, rejection, treatment-related mortality, and GVHD are the main obstacles to favorable outcomes.
Graft-versus-host disease is an immunologically mediated process involving donor immune cell responses against major or minor histocompatibility antigens of the recipient. Acute GVHD is driven by donor’s lymphocytes and inflammatory cytokine cascade, while chronic GVHD involves inflammation, immune dysregulation and fibrosis. (3,4) In children with malignant disorders, graft-versus-host effect may correlate with graft-versus-leukemia effect and contribute to reduction of relapse; (5,6) however, there is no benefit of GVHD in patients with NMD, and any degree of GVHD in this population is considered an undesirable iatrogenic effect. Understanding the incidence and risk factors for GVHD in children with NMD is in important step in developing strategies for its prevention.
In this report we describe the incidence of acute and chronic GVHD in a contemporary cohort of children receiving HCT for NMD, describe survival outcomes based on GVHD grades, and identify risk factors for aGVHD grade III/IV, which correlated with inferior survival.