A sulindac-erlotinib combination reduced the burden and number of duodenal polyps in persons with familial adenomatous polyposis (FAP), a study published in JAMA has shown.1

Persons with FAP, an inherited disorder, are at significantly increased risk for duodenal polyps and cancer. Neoplasia in the duodenum is difficult to manage, and chemoprevention has not been effective. Therefore, researchers sought to determine if a combination therapy using sulindac and erlotinib would produce regression of duodenal adenoma in patients with FAP.

For the study, researchers assigned 92 patients with FAP to receive sulindac 150 mg twice daily and erlotinib 75 mg daily (n = 46) or placebo (n = 46) for 6 months.2 Primary outcome was a change in total polyp burden at 6 months; secondary outcomes included change in total duodenal polyp count, change in duodenal polyp burden or count stratified by genotype and initial polyp burden, and percentage of change from baseline in duodenal polyp burden.

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Over 6 months of treatment, median duodenal polyp burden in the sulindac-erlotinib group decreased from 29.0 mm to 19.5 mm (median change, −8.5 mm), whereas polyp burden increased from 23.0 mm to 31.0 mm (median change, 8.0 mm) in the placebo group.

Median duodenal polyp count decreased from 13.5 to 10.0 (median change, −2.8) in the sulindac-erlotinib group, but increased from 10.5 to 17.0 (median change, 4.3) in the placebo group. However, participants receiving the treatment more commonly experienced grade 1 and 2 adverse events.

Eighty-seven percent of participants experienced an acne-like rash, but this effect was seen in only 20% of participants receiving placebo (P < .001). Grade 3 adverse events were seen in 2 participants: oral mucositis in 1 participant in the treatment group and abdominal pain reported by 1 participant in the placebo group.

Although lower duodenal polyp burden and count was achieved after 6 months of treatment with sulindac-erlotinib combination, adverse events may limit the use of the regimen used in the study. “Further research is necessary to evaluate these preliminary findings in a larger study population with longer follow-up to determine whether the observed effects will result in improved clinical outcomes,” the researchers concluded.


1. Drug combination reduces polyps for patients with high risk for colorectal cancer [news release]. EurekAlert! Web site. http://www.eurekalert.org/pub_releases/2016-03/tjnj-dcr031716.php. Published March 22, 2016. Accessed March 29, 2016.

2. Samadder NJ, Neklason DW, Boucher KM, et al. Effect of sulindac and erlotinib vs placebo on duodenal neoplasia in familial adenomatous polyposis: a randomized clinical trial. JAMA. 2016;315(12):1266-1275. doi:10.1001/jama.2016.2522.