Double-blind, placebo-controlled cross-over study of oral pilocarpine for the prevention of chemotherapy-induced oral mucositis in adult patients with cancer

This study aimed to investigate the effect of oral pilocarpine (OP) in reducing the incidence of chemotherapy-induced oral mucositis. 32 adult cancer patients completed a total of 82 courses of chemotherapy in which either OP or placebo was given prophylactically in a double-blind cross-over design to prevent mucositis. Mucositis was documented in 20 out of 41 courses in which patients were given placebo, whereas mucositis was documented in only six out of 41 courses when patients were given OP (P<0.005). OP treatment was found to significantly reduce the mucositis score when assessed by the method of Donnelly and colleagues (Donnelly JP, Muus P, Schattenberg A, De Witte T, Horrevorts A, De Pauw BE. Bone Marrow Transplant 1992, 9, 409–413). Using this score, all patients scored a total of 52 when they were given the placebo versus eleven when they were treated with OP (P<0.001). A similar reduction in mucositis score was noticed using the World Health Organization (WHO) mucositis score; the total patient score was 25 for the placebo-treated group versus 6 for the OP group (P<0.001). We therefore conclude that oral pilocarpine is highly effective in the prevention of oral mucositis when given prophylactically to adult patients receiving a variety of cancer chemotherapy regimens.