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Chemoprevention in Patients with Peutz-Jeghers Syndrome: Lessons Learned

Justin de Brabander1, Ferry A L M Eskens2, Susanne E Korsse3

  • 1University of Amsterdam, Amsterdam, The Netherlands.

The Oncologist
|January 27, 2018
PubMed
Abstract

Insights

Enrollment challenges and severe toxicity in a phase II study question the feasibility of everolimus for chemoprevention in Peutz-Jeghers syndrome (PJS) patients with LKB1 mutations.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Peutz-Jeghers syndrome (PJS) is linked to LKB1 mutations.
  • LKB1 mutations present a potential therapeutic target for everolimus.

Purpose of the Study:

  • To investigate the efficacy of everolimus in managing polyp and tumor growth in PJS patients with LKB1 mutations.

Main Methods:

  • A phase II study enrolled adult PJS patients with LKB1 mutations.
  • Two cohorts were established: unresectable malignancies and high-risk polyps.
  • Patients received oral everolimus (10 mg daily); response rates were primary endpoints.

Main Results:

  • Only two patients enrolled between 2011 and 2016.
  • One patient experienced disease progression; the other had severe toxicity and discontinued treatment.
  • The trial was aborted due to insufficient patient accrual.

Conclusions:

  • Accrual difficulties prevented conclusions on everolimus efficacy in PJS.
  • The study raises concerns about the feasibility of everolimus for PJS chemoprevention.

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