Treatment of Acute Graft-vs-Host Disease

Abstract

Over the last two decades, advances in our understanding of the pathophysiology of acute GVHD (aGVHD) [1, 2] have not yet translated into significant changes in upfront management. Corticosteroids have remained the cornerstone of aGVHD therapy for the last several decades, and still, failure to respond to steroids remains the primary predictor of poor overall outcome. Several issues limit the usefulness of most retrospective reports of therapeutic intervention for aGVHD. First, we must acknowledge that the majority of series using the “temporal onset” definition of acute and chronic GvHD actually included a mix of patients with either late onset acute or early chronic GvHD, confounding their interpretation and extrapolation to recent efforts to define these syndromes on the basis of clinical features regardless of timing of onset. Second is the lack of prognostic tools to identify cohorts who may need more (vs less) aggressive induction. Third, we also must acknowledge the significant variability in clinical practice between transplant physicians, often well-founded in their efforts to individualize therapy on the basis of specific organ involvement, severity of symptoms, etc. Rather than surrendering to the potentially insurmountable task of establishing a single and perhaps suboptimal standard of care for the upfront treatment of aGVHD, advances in the field and improved patient outcomes will require modern and well-designed clinical trials supported throughout the transplant community.