Results from two randomized trials have shown that oral beclomethasone dipropionate (BDP) is effective for treatment of acute gastrointestinal graft-versus-
host disease (GVHD). Here, we report results of a double-blind, randomized
placebo-controlled phase II study designed to test the
hypothesis that acute GVHD could be prevented by administration of oral BDP, beginning before
hematopoietic cell transplantation (HCT) and continuing until day 75 after HCT. Study
drug (BDP or
placebo) was administered as 1 mg immediate-release formulation plus 1 mg delayed-release formulation orally four times daily. According to the primary endpoint, systemic
glucocorticoid treatment for GVHD was given to 60 of the 92 participants (65%) in the BDP arm, versus 31 of 46 participants (67%) in the
placebo arm. The secondary
efficacy endpoints showed no statistically significant differences between the two arms. The proportion of participants who took at least 90% of the prescribed study
drug during the first 4 weeks after HCT was 54% overall. Lower severity of
mucositis strongly correlated with higher adherence to the schedule of study
drug administration. Inconsistent adherence related to
mucositis during
recovery after myeloablative conditioning may have obscured a beneficial
therapeutic effect in the current study.