Evidence map›Paper›PMID 33197285›Full record

ArticleCancer2021

Long-term outcomes of pexidartinib in tenosynovial giant cell tumors.

Hans Gelderblom, Andrew J Wagner, William D Tap, Emanuela Palmerini, Zev A Wainberg, Jayesh Desai, John H Healey, Michiel A J van de Sande, Nicholas M Bernthal, Eric L Staals and 6 more

2 registry-linked trialsOpen access · hybridAbstract read
In one paragraph

Article in Cancer, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 35 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
35citing papers in PubMed, 1 pooled it
5.8field-weighted citation impact, top 3% 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.

NCT01004861 phase1completednot on this map

A Phase 1 Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of PLX3397 in Patients With Advanced, Incurable, Solid Tumors in Which the Target Kinases Are Linked to Disease Pathophysiology

TypeinterventionalSponsorDaiichi SankyoRan2009 to 2018Enrolled132ConditionsSolid TumorArmsPLX3397
NCT02371369 phase3completednot on this map

A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath

TypeinterventionalSponsorDaiichi SankyoRan2015 to 2021Enrolled120ConditionsPigmented Villonodular Synovitis, Giant Cell Tumors of the Tendon Sheath, Tenosynovial Giant Cell TumorArmsPexidartinib, Placebo
3 · Its place in the literature

Who cites it

35 citing papers in PubMed, 1 synthesis or guideline pooled it, 65 citations in OpenAlex.

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  18. CYP3A Mediates an Unusual C(spAngewandte Chemie (International ed. in English) · 2024
    Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

16 authors at 10 institutions in 4 countries.

Hans GelderblomLeiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0001-9270-8636
Andrew J WagnerDana-Farber Cancer Institute, Boston, Massachusetts.ORCID 0000-0002-4384-9448
William D TapMemorial Sloan Kettering Cancer Center, New York, New York.
Emanuela PalmeriniIRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.ORCID 0000-0003-3406-6705
Zev A WainbergDavid Geffen School of Medicine, University of California Los Angeles, Santa Monica, California.
Jayesh DesaiPeter MacCallum Cancer Centre, Melbourne, Victoria, Australia.
John H HealeyMemorial Sloan Kettering Cancer Center, New York, New York.ORCID 0000-0002-0802-1186
Michiel A J van de SandeLeiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0002-9156-7656
Nicholas M BernthalDavid Geffen School of Medicine, University of California Los Angeles, Santa Monica, California.
Eric L StaalsIRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Charles G PeterfySpire Sciences, Inc, Boca Raton, Florida.
Anna Maria FrezzaFondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.ORCID 0000-0003-2335-7224
Henry H HsuPlexxikon, Berkeley, California.
Qiang WangDaiichi Sankyo, Inc, Basking Ridge, New Jersey.
Dale E ShusterDaiichi Sankyo, Inc, Basking Ridge, New Jersey.
Silvia StacchiottiFondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.ORCID 0000-0002-1742-8666
Daiichi Sankyo (United States) · USFondazione IRCCS Istituto Nazionale dei Tumori · ITIstituto Ortopedico Rizzoli · ITLeiden University Medical Center · NLMemorial Sloan Kettering Cancer Center · USUniversity of California, Los Angeles · USBoca Raton Regional Hospital · USHarvard University · USPeter MacCallum Cancer Centre · AUPlex Pharmaceuticals (United States) · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
Daiichi SankyoNCI NIH HHS P30 CA008748
6 · The paper itself

Abstract

backgroundThe objective of this study was to report on the long-term effects of pexidartinib on tenosynovial giant cell tumor (TGCT).

methodsThis was a pooled analysis encompassing 3 pexidartinib-treated TGCT cohorts: 1) a phase 1 extension study (NCT01004861; 1000 mg/d; n = 39), 2) ENLIVEN patients randomized to pexidartinib (1000 mg/d for 2 weeks and then 800 mg/d; n = 61), and 3) ENLIVEN crossover patients (NCT02371369; 800 mg/d; n = 30). Eligible patients were 18 years old or older and had a histologically confirmed TGCT that was unresectable and symptomatic. Efficacy endpoints included the best overall response (complete or partial response) and the duration of response (DOR) by the Response Evaluation Criteria in Solid Tumors (RECIST) and the tumor volume score (TVS). The safety assessment included the frequency of treatment-emergent adverse events (TEAEs) and hepatic laboratory abnormalities (aminotransferase elevations and mixed/cholestatic hepatotoxicity). The data cutoff was May 31, 2019.

resultsOne hundred thirty patients with TGCT received pexidartinib (median treatment duration, 19 months; range, 1 to 76+ months); 54 (42%) remained on treatment at the end of the analysis (26 months after initial data cut of March 2017). The RECIST overall response rate (ORR) was 60%; the TVS ORR was 65%. The median times to response were 3.4 (RECIST) and 2.8 months (TVS), with 48 of the responding patients (62%) achieving a RECIST partial response by 6 months and with 72 (92%) doing so by 18 months. The median DOR was reached for TVS (46.8 months). Reported TEAEs were mostly low-grade, with hair color changes being most frequent (75%). Most liver abnormalities (92%) were aminotransferase elevations; 4 patients (3%) experienced mixed/cholestatic hepatotoxicity (all within the first 2 months of treatment), which was reversible in all cases (recovery spanned 1-7 months).

conclusionsThis study demonstrates the prolonged efficacy and tolerability of long-term pexidartinib treatment for TGCT.

Indexed as

AdultAgedAged, 80 and overAminopyridinesFemaleGiant Cell Tumor of Tendon SheathHumansMaleMiddle AgedPyrrolesYoung AdultAminopyridinespexidartinibPyrrolesefficacylong termpexidartinibpooled analysissafetytenosynovial giant cell tumor (TGCT)tumor response

Identifiers

PMID33197285
PMCPMC7946703
OpenAlexW3099482482

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.