Evidence map›Paper›PMID 29371475›Full record

Trial reportThe oncologist2018

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

Justin de Brabander, Ferry A L M Eskens, Susanne E Korsse, Evelien Dekker, Pieter Dewint, Monique E van Leerdam, Susanne van Eeden, Heinz-Josef Klümpen

Open access · bronzeAbstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The oncologist, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.3field-weighted citation impact, top 21% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 26 citations in OpenAlex.

  1. Sirolimus for Colon Polyposis in PTEN Hamartoma Tumor Syndrome.Clinical and translational gastroenterology · 2025
    Trial
  2. Review
  3. Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Review
  9. Hereditary Polyposis Syndromes.Current treatment options in gastroenterology · 2019
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 6 institutions in 2 countries.

Justin de BrabanderUniversity of Amsterdam, Amsterdam, The Netherlands.
Ferry A L M EskensDepartment of Medical Oncology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands.
Susanne E KorsseDepartment of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, The Netherlands.
Evelien DekkerDepartment of Gastroenterology and Hepatology, Cancer Center Amsterdam, Academic Medical Center, Amsterdam, The Netherlands.
Pieter DewintDepartment of Gastroenterology and Hepatology, Maria Middelares Hospital, Gent, Belgium.
Monique E van LeerdamDepartment of Gastroenterology, Netherlands Cancer Institute, Amsterdam, The Netherlands.
Susanne van EedenDepartment of Pathology, Cancer Center Amsterdam, Academic Medical Center, Amsterdam, The Netherlands.
Heinz-Josef KlümpenDepartment of Medical Oncology, Cancer Center Amsterdam, Academic Medical Center, Amsterdam, The Netherlands h.klumpen@amc.uva.nl.
Academic Medical Center · NLAZ Maria Middelares · BEErasmus MC Cancer Institute · NLLeiden University Medical Center · NLThe Netherlands Cancer Institute · NLUniversity of Amsterdam · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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

backgroundLKB1 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.

methodsAdult 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.

resultsBetween October 2011 and April 2016, only two patients were enrolled, one in each cohort. A 49-year-old patient with advanced pancreatic cancer in cohort 1 was progressive after 2 months. A 52-year-old male patient in cohort 2 experienced severe toxicity and refused treatment after 4 months, even though endoscopy suggested stabilization of polyps. Adverse events included dental inflammations, mucositis, and rash. In 2016, the trial was aborted for lack of accrual, despite extensive accrual efforts in an area where PJS is highly prevalent and care is highly centralized.

conclusionDue to accrual problems, no conclusions can be drawn about the value of everolimus in PJS treatment, questioning the feasibility of this agent for chemoprevention.

Indexed as

AMP-Activated Protein Kinase KinasesAntineoplastic AgentsChemopreventionEverolimusHumansIntestinal PolypsMaleMiddle AgedMutationPancreatic NeoplasmsPeutz-Jeghers SyndromeProtein Serine-Threonine KinasesAMP-Activated Protein Kinase KinasesAntineoplastic AgentsEverolimusProtein Serine-Threonine KinasesSTK11 protein, human

Identifiers

PMID29371475
PMCPMC5896716
OpenAlexW2784681437

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.