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. 2025 Jun:9:e2400387.
doi: 10.1200/PO-24-00387. Epub 2025 Jun 11.

Real-World Outcomes of Molecular Tumor Board Treatment Recommendations

Federico Nichetti  1   2 , Marta Brambilla  1 , Matteo Duca  1 , Alberta Piccolo  3 , Daniela Miliziano  1 , Chiara Cavalli  1 , Francesca Marra  3 , Paolo Ambrosini  1 , Caterina Zanella  1 , Claudio Vernieri  1   4 , Daniele Lorenzini  3 , Elena Colombo  1 , Silvia Damian  1 , Paolo Baili  5 , Claudia Proto  1 , Adele Busico  3 , Elena Conca  3 , Iolanda Capone  3 , Siranoush Manoukian  6 , Elena Tamborini  7 , Federica Perrone  7 , Monica Niger  1 , Andrea Vingiani  3   8 , Luca Agnelli  1   3 , Filippo de Braud  1   8 , Giancarlo Pruneri  3   8
Affiliations

Real-World Outcomes of Molecular Tumor Board Treatment Recommendations

Federico Nichetti et al. JCO Precis Oncol. 2025 Jun.

Abstract

Purpose: The Molecular Tumor Board (MTB) is an important tool for the selection of molecularly targeted agents (MTAs), but its clinical utility is unconfirmed in real-world practice.

Materials and methods: We explored the clinical relevance of MTAs in patients discussed by the MTB of the National Cancer Institute of Milan. Patients were stratified into eligible (ie, at least one actionable molecular alteration identified) and treated with MTA (group A), eligible but not treated (group B), or not eligible (group C). Cases for whom a European Society for Medical Oncology Scale for Clinical Actionability of Molecular Targets IA tier target was identified were excluded from this analysis and managed as per clinical practice. The study end points were overall survival (OS), progression-free survival (PFS), and PFSratio, defined as PFS on MTA/PFS on the last previous treatment in group A.

Results: As of November 2022, 1,813 cases were discussed, of which 458 (25.2%) were potentially eligible to MTA and 117 (6.4%) started treatment. The median PFS on MTA was 12.0 months and the median PFSratio in patients receiving MTA as >1 line of treatment was 2.71, with a benefit rate of 64%. OS was significantly longer in group A than in B and C metastatic patients (adjusted hazard ratio, 2.21 and 1.71 in group B and C, respectively; P < .001).

Conclusion: Efforts should be pursued to offer MTB-driven therapies, as eligible patients who did not receive MTA showed a significantly poorer prognosis.

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Conflict of interest statement

The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/po/author-center.

Open Payments is a public database containing information reported by companies about payments made to US-licensed physicians (Open Payments).

Giancarlo Pruneri

Honoraria: Novartis, Roche, Genomic Health, AstraZeneca, Menarini

Consulting or Advisory Role: ADS Biotec

Research Funding: Roche, Roche Molecular Diagnostics

No other potential conflicts of interest were reported.

Figures

FIG 1.
FIG 1.
(A, B) Workflow and patient accrual. (A) Study flow chart. (B) Tumor entity subcohorts. ESCAT, ESMO Scale for Clinical Actionability of Molecular Targets; ESMO, European Society for Medical Oncology; INT, Fondazione IRCCS Istituto Nazionale dei Tumori; MTA, molecularly targeted agent; MTB, Molecular Tumor Board.
FIG 2.
FIG 2.
(A-D) Outcomes of patients treated with MTA. (A) Kaplan-Meier plot of progression-free survival of MTA-treated patients. (B) Alluvial plot of best response according to targeted molecular alteration and ESCAT tiers; each case is colored and grouped according to best response. (C) Kaplan-Meier plot of PFSratio of MTA-treated patients. (D) Continuous survival plot showing the impact of PFSratio on overall survival, after adjustment for covariates in multivariable analysis. CR, complete response; ESCAT, ESMO Scale for Clinical Actionability of Molecular Targets; ESMO, European Society for Medical Oncology; MTA, molecularly targeted agent; PFS, progression-free survival; PD, progressive disease; PR, partial response; SD, stable disease; TMB, tumor mutational burden.
FIG 3.
FIG 3.
(A, B) Overall survival in MTB-discussed patients with metastatic cancer. (A) Kaplan-Meier plot of overall survival in patients with metastatic cancer. (B) Confounder-adjusted Kaplan-Meier plot of overall survival in patients with metastatic cancer. MTB, Molecular Tumor Board.
See this image and copyright information in PMC

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