Treatment Options in Steroid-Refractory Acute Graft-Versus-Host Disease Following Hematopoietic Stem Cell Transplantation

Objective: To evaluate the treatment options in steroid-refractory acute graft-versus-host disease (GVHD) following hematopoietic stem cell transplantation. Data Sources: Literature was obtained by searching MEDLINE (1966–May 2007) and EMBASE (1980–May 2007). Study Selection and Data Extraction: All pertinent clinical trials, retrospective studies, case reports, and compassionate use studies were identified and evaluated for safety and efficacy of the pharmacologic agents. Data Synthesis: Steroid-refractory acute GVHD is associated with high rates of morbidity and mortality. Although various pharmacologic agents have been studied in the treatment of steroid-refractory acute GVHD, no treatments have been established as a salvage therapy. Preliminary data on different pharmacologic agents have been identified and evaluated for their efficacy and tolerability in the treatment of steroid-refractory acute GVHD. The effects of the pharmacologic agents varied significantly among patients: severity of the disease, involvement of different organs, and the patient's age seem to be the major factors that affect an individual's response to drug therapy. In addition, the treatments are further challenged by the high incidence of potentially fatal opportunistic infections that occur during the therapy. Conclusions: Selection of pharmacologic agents for the treatment of steroid-refractory acute GVHD should be based on the target organs, adverse drug reactions, and economic factors. Further studies with larger sample sizes are warranted to better understand the roles of these agents in the treatment of steroid-refractory acute GVHD.