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.