Evidence map›Paper›PMID 31755953›Full record

ArticleJAMA network open2019

Estimation of Percentage of Patients With Fibroblast Growth Factor Receptor Alterations Eligible for Off-label Use of Erdafitinib.

Lelia Maria de Almeida Carvalho, Sandra de Oliveira Sapori Avelar, Alyson Haslam, Jennifer Gill, Vinay Prasad

Open access · goldAbstract read
In one paragraph

Article in JAMA network open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
0.8field-weighted citation impact, top 28% 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

11 citing papers in PubMed, 21 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Article
  4. Review
  5. Article
  6. Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Article
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

5 authors at 2 institutions in 2 countries.

Lelia Maria de Almeida CarvalhoHospital das Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Sandra de Oliveira Sapori AvelarHospital das Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Alyson HaslamKnight Cancer Institute, Division of Hematology Oncology, Oregon Health and Science University, Portland.
Jennifer GillKnight Cancer Institute, Division of Hematology Oncology, Oregon Health and Science University, Portland.
Vinay PrasadKnight Cancer Institute, Division of Hematology Oncology, Oregon Health and Science University, Portland.
Oregon Health & Science University · USHospital das Clínicas da Universidade Federal de Minas Gerais · BR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Importance: When a novel drug is granted accelerated approval, both its on-label and off-label uses must be taken into account. Objectives: To estimate the potential upper bound of off-label use of erdafitinib to treat advanced cancer with fibroblast growth factor receptor gene (FGFR) alterations, compare it to the upper bound of on-label use in urothelial cancer, and to review studies that may support off-label use. Design, Setting, and Participants: This cross-sectional study used frequency data on FGFR alterations by cancer type and the estimated number of deaths from all cancers for 2019 in the United States. Mortality statistics were used as surrogates for patients with advanced cancer. Analysis was conducted in May 2019. Exposure: Percentage of patients with an FGFR2 or FGFR3 alteration. Main Outcomes and Measures: Estimated number of patients with advanced cancer expressing an FGFR2 or FGFR3 alteration eligible for off-label use of erdafitinib by cancer type; number of studies investigating FGFR-targeting drugs for patients with cancer; and number of ongoing clinical trials on erdafitinib by cancer type. Results: A total of 15 cancer types had reported FGFR alterations. Of 455 440 estimated patients who died of cancer in 2019, 17 019 (3.7%) were estimated to have FGFR2 or FGFR3 alterations. Of these patients, 12 955 (76.1%) could be eligible for off-label treatment with erdafitinib. A total of 29 completed studies evaluated FGFR-targeting drugs in 11 cancer types, and 10 ongoing studies are studying erdafitinib for different oncological indications. Conclusions and Relevance: This study indicates that the potential for off-label use of FGFR inhibitors such as erdafitinib spans a number of cancer types and a large patient population. Systematic trials exploring off-label uses may be desirable for drugs that target clear, identifiable molecular alterations because this may be more efficient than off-label use in identifying clinical scenarios where the agent has activity.

Indexed as

Antineoplastic AgentsGenetic VariationHumansNeoplasmsOff-Label UsePyrazolesQuinoxalinesReceptor, Fibroblast Growth Factor, Type 2Receptor, Fibroblast Growth Factor, Type 3Urologic NeoplasmsAntineoplastic AgentserdafitinibFGFR2 protein, humanFGFR3 protein, humanPyrazolesQuinoxalinesReceptor, Fibroblast Growth Factor, Type 2Receptor, Fibroblast Growth Factor, Type 3

Identifiers

PMID31755953
PMCPMC6902826
OpenAlexW2990195494

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.