Evidence map›Paper›PMID 40616288›Full record

Observational studyFuture oncology (London, England)2025

Real-world experience of patients newly initiated on pexidartinib for tenosynovial giant cell tumor.

Dong Dai, Kristen Tecson, Irene Pan, Xin Ye, William Tap

Abstract readObservational Study
In one paragraph

Observational study in Future oncology (London, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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

1 citing paper in PubMed.

  1. 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

5 authors.

Dong DaiGlobal Health Economics and Outcomes Research, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA.ORCID 0000-0002-0231-1422
Kristen TecsonGlobal Health Economics and Outcomes Research, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA.
Irene PanEpidemiology and Real-World Evidence, United BioSource LLC, King of Prussia, PA, USA.
Xin YeGlobal Health Economics and Outcomes Research, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA.
William TapSarcoma Medical Oncology Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
NCI NIH HHS P30 CA008748
6 · The paper itself

Abstract

backgroundTenosynovial giant-cell tumor (TGCT) is a rare condition characterized by pain, stiffness, and limited range of motion. Pexidartinib is the first regulatory-approved systemic therapy for adult patients with symptomatic TGCT not amenable for surgery. This study evaluated symptom improvement in patients who newly initiated pexidartinib.

methodsA longitudinal, observational study collected data on patient-reported outcomes, demographics, disease history, pexidartinib dosing, and clinicians' perspective.

resultsIdentified from the TURALIO Risk Evaluation and Mitigation Strategy (REMS) program, 80 patients who newly initiated pexidartinib between April and October 2022 received a study invitation, and 14 completed the baseline assessment; mean (SD) age was 39.8 (13.60) years, 50.0% were female, and most common tumor sites were in the lower extremities (71.4%). By the end of study follow-up (May 2023), 11 patients completed some PRO assessments at follow-up, and median time on pexidartinib was 7.5 months. Results showed a trend of reduction in pain and stiffness and improvement in physical functioning. Overall, 80% (8/10) endorsed moderately high treatment satisfaction with pexidartinib treatment.

conclusionsFindings from this study provided real-world evidence of an association between pexidartinib initialization and symptom improvements among patients with symptomatic TGCTs from the patients' and clinicians' perspective.

Indexed as

AminopyridinesGiant Cell Tumor of Tendon SheathPyrrolesAdultFemaleHumansLongitudinal StudiesMaleMiddle AgedPatient Reported Outcome MeasuresTreatment OutcomeAminopyridinespexidartinibPyrrolespatient-reported outcomesPexidartinibsymptom changetenosynovial giant-cell tumortreatment effectivenesstreatment satisfaction

Identifiers

PMID40616288
PMCPMC12330255

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.