Efficacy and Safety of Eflornithine (CPP-1X)/Sulindac Combination Therapy Versus each as Monotherapy in Patients with Familial Adenomatous Polyposis (FAP): Design and Rationale of a Randomized, Double-blind, Phase III Trial | oneFAPvoice

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Efficacy and Safety of Eflornithine (CPP-1X)/Sulindac Combination Therapy Versus each as Monotherapy in Patients with Familial Adenomatous Polyposis (FAP): Design and Rationale of a Randomized, Double-blind, Phase III Trial

key information

source: BMC Gastroenterology

year: 2016

authors: Burke CA, Dekker E, Samadder NJ, Stoffel E, Cohen A

summary/abstract:

Molecular studies suggest inhibition of colorectal mucosal polyamines (PAs) may be a promising approach to prevent colorectal cancer (CRC). Inhibition of ornithine decarboxylase (ODC) using low-dose eflornithine (DFMO, CPP-1X), combined with maximal PA export using low-dose sulindac, results in greatly reduced levels of normal mucosal PAs. In a clinical trial, this combination (compared with placebo) reduced the 3-year incidence of subsequent high-risk adenomas by >90 %. Familial Adenomatous Polyposis (FAP) is characterized by marked up-regulation of ODC in normal intestinal epithelial and adenoma tissue, and therefore PA reduction might be a potential strategy to control progression of FAP-related intestinal polyposis. CPP FAP-310, a randomized, double-blind, Phase III trial was designed to examine the safety and efficacy of sulindac and DFMO (alone or in combination) for preventing a clinically relevant FAP-related progression event in individuals with FAP.

organisation: Cleveland Clinic, Academic Medical Center Amsterdam, Huntsman Cancer Institute, Michigan Health System, Cancer Prevention Pharmaceuticals

DOI: 10.1186/s12876-016-0494-4

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