Evidence map›Paper›PMID 40349140›Full record

Observational studyThe oncologist2025

Evaluation of the effectiveness and safety of avapritinib in real-world Spanish cases with gastrointestinal stromal tumor and D842V-PDGFRA mutation.

Isaac Nuñez Hernández, Cristina Gómez Palmero, Juan Ramón Delgado, Ana Nuño, Maria Ángeles Sala González, Ana González Ageitos, Héctor Aguilar, Pablo Ayala de Miguel, Elizabeth Condori, Roberto Díaz Beveridge and 5 more

Abstract readMulticenter StudyObservational Study
In one paragraph

Observational study in The oncologist, 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

15 authors.

Isaac Nuñez HernándezHospital Universitario Nuestra Señora de Candelaria, Santa Cruz de Tenerife, Ctra. Gral. del Rosario, 145, 38010, Spain.
Cristina Gómez PalmeroHospital Universitario de Toledo, Toledo, Av. del Río Guadiana, 45007, Spain.
Juan Ramón DelgadoHospital Universitario Virgen de las Nieves (HUVN), Av. de las Fuerzas Armadas, 2, Beiro, 18014 Granada, Spain.
Ana NuñoHospital Obispo Polanco, Av. Ruiz Jarabo, s/n, 44002, Teruel, Spain.
Maria Ángeles Sala GonzálezHospital Universitario Basurto, Montevideo Etorb., 18, Basurtu-Zorrotza, 48013, Bilbao, Spain.
Ana González AgeitosHospital Universitario de Toledo, Toledo, Av. del Río Guadiana, 45007, Spain.
Héctor AguilarFundación Instituto Valenciano de Oncología, Carrer del Professor Beltrán Báguena, 8, Campanar, 46009, Valencia, Spain.
Pablo Ayala de MiguelComplejo Hospitalario Universitario de Cáceres, Av. de la Universidad, 75, Norte, 10004 Cáceres, Spain.
Elizabeth CondoriHospital Santa Bárbara de Soria, Pº de Santa Bárbara, s/n, 42005, Soria, Spain.
Roberto Díaz BeveridgeHospital Universitari i Politècnic La Fe, Avinguda de Fernando Abril Martorell, 106, Quatre Carreres, 46026, Valencia, Spain.
Jerónimo Martínez GarcíaHCU Virgen de la Arrixaca, Ctra. Madrid-Cartagena, s/n, 30120 El Palmar, Murcia, Spain.
Gloria MarquinaDepartment of Medical Oncology, Hospital Clínico San Carlos, Department of Medicine, School of Medicine, Universidad Complutense de Madrid (UCM), Instituto de Investigación Sanitaria (IdISSC), Calle del Prof Martín Lagos, S/N, Moncloa - Aravaca, 28040, Madrid, Spain.
Vanesa Varela-PoseUniversity Hospital of Santiago de Compostela (SERGAS), Rúa da Choupana, s/n, 15706, Santiago de Compostela, Spain.
Joel Veas RodríguezHospital Arnau de Vilanova de Lleida, Av. Alcalde Rovira Roure, 80, 25198, Lleida, Spain.
César SerranoHospital Universitario Vall d'Hebron, Pg. de la Vall d'Hebron, 119, Horta-Guinardó, 08035, Barcelona, Spain.ORCID 0000-0003-1416-8739

Funding

Asociación Española Contra el Cáncer AECC CLSEN20004SERRBlueprint Medicines
6 · The paper itself

Abstract

introductionGastrointestinal stromal tumors (GISTs) are the most common sarcoma subtype. Patients with unresectable or metastatic GISTs harboring the D842V mutation in the PDGFRA gene have a poor prognosis due to intrinsic resistance to imatinib and all other approved tyrosine kinase inhibitors. Avapritinib, targeting this mutation, is the first agent approved for patients with unresectable or metastatic GIST that have the PDGFRA D842V mutation. This study assesses the effectiveness and safety of avapritinib in real-world clinical scenarios involving Spanish patients with this mutation. MATERIALS AND

methodsThe AVARWE study is a descriptive, retrospective, multicenter observational study of 21 patients treated with avapritinib across 13 Spanish centers from June 9, 2023, to December 18, 2023. Data collected included patient demographics, disease characteristics, treatment history, and response rates based on RECIST criteria. The main outcomes, progression-free survival (PFS) and overall survival (OS), were measured, with safety assessed through adverse events documentation according to CTCAE criteria.

resultsMedian PFS 35.6 was months and median OS was 42.2 months, with survival rates at 1, 5, and 3 years demonstrating avapritinib effectiveness. The objective response rate was 76.2% for partial response and 4.8% for complete response. Avapritinib enabled surgical intervention in previously unresectable cases and was generally well-tolerated, with manageable adverse events.

conclusionAvapritinib extends PFS and OS among patients with PDGFRA D842V-mutant GIST in real-world practice, mirroring pivotal trial outcomes. Its substantial activity supports its use as a first-line therapy for this subgroup. The manageable safety profile reinforces avapritinib viability for routine use. Given the rarity of these cases, it is advised to consult sarcoma-expert units.

Indexed as

Gastrointestinal NeoplasmsGastrointestinal Stromal TumorsMutationProtein Kinase InhibitorsPyrazolesPyrrolesReceptor, Platelet-Derived Growth Factor alphaAdultAgedAged, 80 and overAvapritinibFemaleHumansMaleMiddle AgedRetrospective StudiesAvapritinibProtein Kinase InhibitorsPyrazolesPyrrolesReceptor, Platelet-Derived Growth Factor alphaTriazinesavapritinibD842V mutationgastrointestinal stromal tumorGISToutcomesPDGFRAtreatment

Identifiers

PMID40349140
PMCPMC12065942

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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.