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.