A randomised trial of topical polaprezinc to prevent oral mucositis in patients undergoing haematopoietic stem cell transplantation (ToPaZ study).

ABSTRACT

PURPOSE: Oral mucositis (OM) is a common complication in haematopoietic stem cell transplantation (HSCT). Polaprezinc, an anti-ulcer drug, has been shown to be effective to prevent OM in several studies when administered topically and systemically. This study aimed to evaluate the effectiveness of topical polaprezinc in patients undergoing HSCT. METHODS: This was an open-label randomised clinical trial comparing polaprezinc and sodium bicarbonate mouthwashes for the prevention of severe OM in HSCT patients. Adult patients who received conditioning regimens at moderate to high risk of developing OM were included. The primary endpoint was the incidence of severe (WHO grades 3-4) OM. The secondary endpoints included duration of grades 3-4 OM, incidence and duration of grades 2-4 OM, patient-reported pain and functional limitations. RESULTS: In total, 108 patients (55 test arm and 53 control arm) were randomised. There was no difference in the incidence of grades 3 to 4 OM (35% test arm versus 36% control arm). The secondary endpoints were not significantly different. In both arms, patients reported more throat pain compared to mouth pain. CONCLUSIONS: Topical polaprezinc had no effect in the prevention of OM in HSCT patients. Further research is required to evaluate the effects of systemic polaprezinc. The OM assessment tool needs to be reviewed as throat mucositis was a main issue in this study. TRIAL REGISTRATION ACTRN12320001188921 (Date Registered 10th November 2020).