Molecular Tumor Board of the University Medical Center Groningen (UMCG-MTB): outcome of patients with rare or complex mutational profiles receiving MTB-advised targeted therapy.

Autor: de Jager VD; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Plomp P; Department of Respiratory Medicine, Isala Hospital, Zwolle, the Netherlands., Paats MS; Department of Pulmonary Medicine, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, the Netherlands., van Helvert S; Department of Pathology, Radboud University Medical Center, Nijmegen, the Netherlands., Elst AT; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., van den Berg A; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Dubbink HJ; Department of Pathology, Erasmus MC Cancer Institute, University Medical Center Rotterdam, Rotterdam, the Netherlands., van Geffen WH; Department of Pulmonary Diseases, Medical Center Leeuwarden, Leeuwarden, the Netherlands., Zhang L; Structural Biology in Drug Design, Groningen Research Institute of Pharmacy, University of Groningen, Groningen, the Netherlands., Hendriks LEL; Department of Pulmonary Diseases, GROW - School for Oncology and Reproduction, Maastricht University Medical Center, Maastricht, the Netherlands., Hiltermann TJN; Department of Pulmonary Diseases and Tuberculosis, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Hiddinga BI; Department of Pulmonary Diseases and Tuberculosis, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Hijmering-Kappelle LBM; Department of Pulmonary Diseases and Tuberculosis, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Jalving M; Department of Medical Oncology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Kluiver J; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Koopman B; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., van Kruchten M; Department of Medical Oncology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., van der Logt EMJ; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands; Department of Molecular Diagnostics, Pathology Friesland, Leeuwarden, the Netherlands., Piet B; Department of Pulmonary Diseases, Radboud University Medical Center, Nijmegen, the Netherlands., van Putten J; Department of Pulmonary Diseases, Martini Hospital, Groningen, the Netherlands., Reitsma BH; Department of Pulmonology, Nij Smellinghe, Drachten, the Netherlands., Rutgers SR; Department of Pulmonology, Treant Hospital Group, Scheper Hospital, Emmen, the Netherlands., de Vries M; Department of Pulmonology, Tjongerschans, Heerenveen, the Netherlands., Stigt JA; Department of Respiratory Medicine, Isala Hospital, Zwolle, the Netherlands., Groves MR; Structural Biology in Drug Design, Groningen Research Institute of Pharmacy, University of Groningen, Groningen, the Netherlands., Timens W; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Willems SM; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., van Kempen LC; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands., Schuuring E; Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands. Electronic address: e.schuuring@umcg.nl., van der Wekken AJ; Department of Pulmonary Diseases and Tuberculosis, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands. Electronic address: a.j.van.der.wekken@umcg.nl.
Jazyk: angličtina
Zdroj: ESMO open [ESMO Open] 2024 Nov 04; Vol. 9 (11), pp. 103966. Date of Electronic Publication: 2024 Nov 04.
DOI: 10.1016/j.esmoop.2024.103966
Abstrakt: Purpose: Molecular tumor boards (MTBs) are considered beneficial for treatment decision making for patients with cancer with uncommon, rare, or complex mutational profiles. The lack of international MTB guidelines results in significant variation in practices and recommendations. Therefore, periodic follow-up is necessary to assess and govern MTB functioning. The objective of this study was to determine the effectiveness of MTB treatment recommendations for patients with rare and complex mutational profiles as implemented in the MTB of the University Medical Center Groningen (UMCG-MTB) in 2019-2020.
Patients and Methods: A retrospective follow-up study was carried out to determine the clinical outcome of patients with uncommon or rare (combinations of) molecular aberrations for whom targeted therapy was recommended as the next line of treatment by the UMCG-MTB in 2019 and 2020.
Results: The UMCG-MTB recommended targeted therapy as the next line of treatment in 132 of 327 patients: 37 in clinical trials, 67 in the on-label setting, and 28 in the off-label setting. For on- and off-label treatment recommendations, congruence of recommended and received treatment was 85% in patients with available follow-up (67/79). Treatment with on-label therapy resulted in a response rate of 50% (21/42), a median progression-free survival (PFS) of 6.3 months [interquartile range (IQR) 2.9-14.9 months], and median overall survival (OS) of 15.8 months (IQR 6.4-34.2 months). Treatment with off-label therapy resulted in a response rate of 53% (8/15), a median PFS of 5.1 months (IQR 1.9-7.3 months), and a median OS of 17.7 months (IQR 5.1-23.7 months).
Conclusion: Treatment with MTB-recommended next-line targeted therapy for patients with often heavily pretreated cancer with rare and complex mutational profiles resulted in positive overall responses in over half of patients. Off-label use of targeted therapies, for which there is sufficient rationale as determined by an MTB, is an effective treatment strategy. This study underlines the relevance of discussing patients with rare and complex mutational profiles in an MTB.
(Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.)
Databáze: MEDLINE