Quality of life associated with sirolimus for prevention of graft-versus-host disease: results from a randomized trial.

Several studies have examined sirolimus-based immune suppression for the prevention of graft-versus-host disease after allogeneic hematopoietic cell transplantation, but little is known regarding its effects on quality of life. The current study reports on changes in quality of life to Day 360 in a randomized phase II trial of sirolimus and tacrolimus versus methotrexate and tacrolimus. Quality of life was assessed prior to transplant and on Days 30, 90, 180, 270, and 360 with the Functional Assessment of Cancer Therapy - Bone Marrow Transplant Trial Outcome Index. Random effects models examined the effects of study arm on change in Trial Outcome Index scores from Day 30 to 360, controlling for base-line Trial Outcome Index. The sirolimus/tacrolimus arm (n=37) showed less improvement in Trial Outcome Index scores over time compared to the methotrexate/tacrolimus arm (n=34) (P=0.02). Patients receiving sirolimus and tacrolimus were more likely to endorse nausea and a lack of energy over time (PS≤0.01). These data suggest that sirolimus-based immune suppression is associated with less improvement in quality of life in the first year post-transplant compared to methotrexate/tacrolimus. Quality of life differences may be due to increased fatigue and nausea in patients treated with sirolimus. These findings should be considered in the clinical management of patients treated with sirolimus. (Clinicaltrials.gov identifier:00803010).