Chemoprevention in Patients with Peutz-Jeghers Syndrome: Lessons Learned.

ABSTRACT:

Lessons learned

Motivating patients to enroll in chemopreventive studies is challenging.Chemoprevention with toxic drugs is not feasible.

Background

LKB1 mutations are the underlying genetic abnormality causing Peutz-Jeghers syndrome (PJS) and are a potential target for everolimus. In this phase II study, the efficacy of everolimus on polyp and tumor growth in PJS patients was investigated.

Methods

Adult patients with a proven LKB1 mutation and who were suitable for everolimus treatment were included in two different PJS cohorts: (a) patients with unresectable malignancies and (b) patients with high-risk polyps. Treatment in both groups was oral everolimus, 10 mg daily. Response rates were primary endpoints for both cohorts.

Results

Between October 2011 and Apr

SUBMITTER: de Brabander J 

PROVIDER: S-EPMC5896716 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature