Evidence map›Paper›PMID 39921935›Full record

ArticleESMO open2025

High expression level of ERBB2 and efficacy of trastuzumab deruxtecan in desmoplastic small round cell tumour: a monocentric case series report.

M Brahmi, H Vanacker, A Dufresne, V Isnardi, M Dupont, A Meurgey, M Karanian, P Meeus, M-P Sunyach, F Tirode and 1 more

Abstract readCase Reports
In one paragraph

Article in ESMO open, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Article
  3. 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

11 authors.

M BrahmiCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France; Cancer Research Center of Lyon, INSERM U1052-CNRS UMR5286, Lyon, France. Electronic address: mehdi.brahmi@lyon.unicancer.fr.
H VanackerCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France; Cancer Research Center of Lyon, INSERM U1052-CNRS UMR5286, Lyon, France.
A DufresneCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
V IsnardiCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
M DupontCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
A MeurgeyCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
M KaranianCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France; Cancer Research Center of Lyon, INSERM U1052-CNRS UMR5286, Lyon, France.
P MeeusCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
M-P SunyachCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France.
F TirodeCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France; Cancer Research Center of Lyon, INSERM U1052-CNRS UMR5286, Lyon, France.
J-Y BlayCentre Léon Bérard & Université Claude Bernard Lyon I, Lyon, France; Cancer Research Center of Lyon, INSERM U1052-CNRS UMR5286, Lyon, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDesmoplastic small round cell tumours (DSRCTs) represent an ultra-rare subtype of soft tissue sarcoma characterized by a recurrent EWSR1::WT1 oncogenic translocation. Considered as an extremely aggressive cancer, the prognosis remains poor with a median overall survival not exceeding 24-36 months and a 5-year survival <10%. PATIENTS AND

methodsWe analysed ERBB2/human epidermal growth factor receptor 2 (HER2) expression levels in a series of 13 DSRCT patients, using whole-exome RNA sequencing on formalin-fixed paraffin-embedded samples from a local biopathological database. In addition, a retrospective case series describes the clinical outcome of three successive DSRCT patients treated with trastuzumab deruxtecan (T-DXd).

resultsThe gene expression analysis demonstrated a consistent high RNA expression level of ERBB2 in DSRCT, with elevated levels [>5 log2(transcripts per million + 1)] across all samples of the cohort and the expression level was the highest compared with all other sarcoma subtypes. In addition to these results, T-DXd showed a marked activity in all three DSRCT patients who presented with metastatic disease refractory to previous standard chemotherapy. So far, the treatment has been overall well tolerated and is currently pursued in the three patients (duration of response >3 months for all three), which warrants additional investigation.

conclusionsThis case series presents a major information, suggesting that HER2 is a therapeutic target in DSRCT and T-DXd might represent a novel therapeutic option. Those results require to be rapidly shared with the scientific community and confirmed in a prospective clinical trial in this context of very poor prognosis disease and urgent unmet need.

Indexed as

Antineoplastic Agents, ImmunologicalDesmoplastic Small Round Cell TumorErb-b2 Receptor Tyrosine KinasesTrastuzumabAdultAgedCamptothecinFemaleHumansImmunoconjugatesMaleMiddle AgedPrognosisRetrospective StudiesYoung AdultAntineoplastic Agents, ImmunologicalCamptothecinERBB2 protein, humanErb-b2 Receptor Tyrosine KinasesImmunoconjugatesTrastuzumabtrastuzumab deruxtecanantibody–drug conjugatedesmoplastic small round cell tumourshuman epidermal growth factor receptor 2trastuzumab deruxtecan

Identifiers

PMID39921935
PMCPMC11850745

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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.