Familial adenomatous polyposis (FAP) is a dominantly inherited condition characterized by the formation of numerous adenomatous polyps in the epithelium of the large intestine. If left untreated, these benign polyps can progress into colon cancer. Eflornithine and sulindac are two drugs used to stop the progression of FAP, but whether these drugs work better alone or in combination with each other is not clear. This study aims to evaluate the safety and efficacy of eflornithine-sulindac combination therapy for FAP.
This randomized trial included a total of 171 patients with FAP stratified on the basis of the anatomical site with the highest polyp burden and surgical status. The patients were randomly assigned in a 1:1:1 ratio to receive 750 mg of eflornithine (n=57), 150 mg of sulindac (n=58), or both (n=56) once daily for up to 48 months. The primary outcome of the study was disease progression.
Disease progression occurred in 18 (32%) patients in the combination therapy group, 22 (38%) in the sulindac group, and 23 940%) patients in the eflornithine group. The incidence of adverse and severe events was similar across all three treatment groups.
The research concluded that in patients with FAP, the use of eflornithine-sulindac combination therapy did not lower the risk of FAP progression, as compared with the use of individual drugs.
Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa1916063
