Lockhart, P.B., Brennan, M.T., Kent, M.L., Packman, C.H., Norton, H.J., Fox, P.C., & Frenette, G. (2005). Randomized controlled trial of pilocarpine hydrochloride for the moderation of oral mucositis during autologous blood stem cell transplantation. Bone Marrow Transplantation, 35, 713–720.
Patients were given a 5-mg pilocarpine tablet or placebo starting the day before the conditioning regimen, every 4 hours, for a total of 4 tablets per day. Patients continued taking tablets until absolute neutrophil count (ANC) was greater than 500 for 48 hours, discharge from hospital, or mucosal recovery.
This was a prospective, double-blind, randomized, placebo-controlled trial. Patients were stratified by diagnosis and randomized by a computer-generated numbering scheme.
Despite prior small trials that showed a benefit, this study clearly did not. The intervention was not effective.
A standardized scoring system is needed.