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.

• The study reported on 36 patients with 20 receiving pilocarpine and 16 receiving placebo.
• Patients were undergoing autologous stem cell transplant (SCT) with multiple (not all hemotologic) malignancies.

This was a prospective, double-blind, randomized, placebo-controlled trial. Patients were stratified by diagnosis and randomized by a computer-generated numbering scheme.

• The World Health Organization (WHO) mucositis scale and CMC outcome measures toxicity criteria were used.
• Numerous outcomes were measured.

• No statistically significant differences were found in the incidence, severity, or duration of mucositis.
• Pain at rest and with swallowing and the use of systemic narcotics were not significantly different.
   

Despite prior small trials that showed a benefit, this study clearly did not.  The intervention was not effective.

A standardized scoring system is needed.