Evidence map›Paper›PMID 38351684›Full record

ArticleCancer research and treatment2024

Global Expanded Access Program for Pemigatinib in Patients with Previously Treated Locally Advanced or Metastatic Cholangiocarcinoma and Fibroblast Growth Factor Receptor Gene Alterations.

Anouk Lindley, Gerald Prager, Michael Bitzer, Timothy C Burn, Christine F Lihou, Elisabeth Croft

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cancer research and treatment, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Erdafitinib in Patients with FGFR-Altered Advanced or Metastatic Cholangiocarcinoma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026
    Trial
  3. 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

6 authors.

Anouk LindleyIncyte Corporation, Wilmington, DE, USA.
Gerald PragerMedical University of Vienna, Department of Internal Medicine I, Comprehensive Cancer Center Vienna, Vienna, Austria.
Michael BitzerDepartment of Internal Medicine I, Eberhard-Karls University, Tübingen, Germany.
Timothy C BurnIncyte Corporation, Wilmington, DE, USA.
Christine F LihouIncyte Corporation, Wilmington, DE, USA.
Elisabeth CroftIncyte Corporation, Wilmington, DE, USA.

Funding

Incyte Corporation
6 · The paper itself

Abstract

purposePemigatinib is a fibroblast growth factor receptor-2 (FGFR2) inhibitor approved for use in patients with previously treated cholangiocarcinoma (CCA) and FGFR2 fusions or rearrangements. This ongoing global Expanded Access Program (EAP) allows physicians in regions where pemigatinib is not commercially available to request pemigatinib for patients with locally advanced or metastatic CCA who, in the physician's opinion, could benefit from pemigatinib treatment. MATERIALS AND

methodsEighty-nine patients from Europe, North America, and Israel were treated from January 2020 through September 2021.

resultsPatients had FGFR gene fusions (68.5%), rearrangements (12.4%), translocations (5.6%), amplifications (2.2%), and other alterations (11.2%). Median duration of treatment in the EAP was 4.0 months (range, 0.1 to 13.6 months). The most frequently reported adverse event (AE) was hyperphosphatemia (22.5%); the most common serious AE was cholangitis (3.4%). Treatment discontinuation was associated with reports of AEs for seven patients (7.9%). AEs associated with pemigatinib were consistent with those observed in clinical trials.

conclusionEfficacy was not assessed in this EAP. However, some patients remained on treatment for up to a year, suggesting that they observed a benefit from treatment. Patients with CCA should undergo molecular testing to identify those who could benefit from targeted treatments such as pemigatinib.

Indexed as

Bile Duct NeoplasmsCholangiocarcinomaReceptor, Fibroblast Growth Factor, Type 2AdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedMorpholinesProtein Kinase InhibitorsPyrimidinesPyrrolesFGFR2 protein, humanMorpholinespemigatinibProtein Kinase InhibitorsPyrimidinesPyrrolesReceptor, Fibroblast Growth Factor, Type 2CholangiocarcinomaExpanded Access ProgramFGFR2 fusionsFGFR2 rearrangementsPemigatinib

Identifiers

PMID38351684
PMCPMC11261204

What OpenQuestion holds

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Registered trials

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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.