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

V. D. de Jager; P. Plomp; M. S. Paats; S. van Helvert; A. ter Elst; A. van den Berg; H. J. Dubbink; W. H. van Geffen; L. Zhang; L. E.L. Hendriks; T. J.N. Hiltermann; B. I. Hiddinga; L. B.M. Hijmering-Kappelle; M. Jalving; J. Kluiver; B. Koopman; M. van Kruchten; E. M.J. van der Logt; B. Piet; J. van Putten; B. H. Reitsma; S. R. Rutgers; M. de Vries; J. A. Stigt; M. R. Groves; W. Timens; S. M. Willems; L. C. van Kempen; E. Schuuring; A. J. van der Wekken

doi:10.1016/j.esmoop.2024.103966

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

V. D. de Jager
, P. Plomp
, M. S. Paats
, S. van Helvert
, A. ter Elst
, A. van den Berg
, H. J. Dubbink
, W. H. van Geffen
, L. Zhang
, L. E.L. Hendriks
, T. J.N. Hiltermann
, B. I. Hiddinga
, L. B.M. Hijmering-Kappelle
, M. Jalving
, J. Kluiver
, B. Koopman
, M. van Kruchten
, E. M.J. van der Logt
, B. Piet
, J. van Putten

B. H. Reitsma, S. R. Rutgers, M. de Vries, J. A. Stigt, M. R. Groves, W. Timens, S. M. Willems, L. C. van Kempen, E. Schuuring^*, A. J. van der Wekken^*

^*Corresponding author for this work

University of Groningen
University Medical Centre Groningen
Isala Clinics
Radboud University Medical Center
Medical Centre Leeuwarden
Maastricht University
GROW - School for Oncology and Reproduction
Department of Molecular Diagnostics
Martini Ziekenhuis
Department of Pulmonology
Scheper Hospital
Tjongerschans Ziekenhuis

Research output: Contribution to journal › Article › Academic › peer-review

6 Citations (Web of Science)

53 Downloads (Pure)

Abstract

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.

Original language	English
Article number	103966
Journal	ESMO Open
Volume	9
Issue number	11
DOIs	https://doi.org/10.1016/j.esmoop.2024.103966
Publication status	Published - Nov 2024

Bibliographical note

Publisher Copyright:
© 2024 The Authors

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

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de Jager, V. D., Plomp, P., Paats, M. S., van Helvert, S., Elst, A. T., van den Berg, A., Dubbink, H. J., van Geffen, W. H., Zhang, L., Hendriks, L. E. L., Hiltermann, T. J. N., Hiddinga, B. I., Hijmering-Kappelle, L. B. M., Jalving, M., Kluiver, J., Koopman, B., van Kruchten, M., van der Logt, E. M. J., Piet, B., ... van der Wekken, A. J. (2024). 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. ESMO Open, 9(11), Article 103966. https://doi.org/10.1016/j.esmoop.2024.103966

@article{501ea7209e054db98f7891401901d574,

title = "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",

abstract = "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.",

author = "\{de Jager\}, \{V. D.\} and P. Plomp and Paats, \{M. S.\} and \{van Helvert\}, S. and Elst, \{A. ter\} and \{van den Berg\}, A. and Dubbink, \{H. J.\} and \{van Geffen\}, \{W. H.\} and L. Zhang and Hendriks, \{L. E.L.\} and Hiltermann, \{T. J.N.\} and Hiddinga, \{B. I.\} and Hijmering-Kappelle, \{L. B.M.\} and M. Jalving and J. Kluiver and B. Koopman and \{van Kruchten\}, M. and \{van der Logt\}, \{E. M.J.\} and B. Piet and \{van Putten\}, J. and Reitsma, \{B. H.\} and Rutgers, \{S. R.\} and \{de Vries\}, M. and Stigt, \{J. A.\} and Groves, \{M. R.\} and W. Timens and Willems, \{S. M.\} and \{van Kempen\}, \{L. C.\} and E. Schuuring and \{van der Wekken\}, \{A. J.\}",

note = "Publisher Copyright: {\textcopyright} 2024 The Authors",

year = "2024",

month = nov,

doi = "10.1016/j.esmoop.2024.103966",

language = "English",

volume = "9",

journal = "ESMO Open",

issn = "2059-7029",

publisher = "Elsevier",

number = "11",

}

de Jager, VD, Plomp, P, Paats, MS, van Helvert, S, Elst, AT, van den Berg, A, Dubbink, HJ, van Geffen, WH, Zhang, L, Hendriks, LEL, Hiltermann, TJN, Hiddinga, BI, Hijmering-Kappelle, LBM, Jalving, M, Kluiver, J, Koopman, B, van Kruchten, M, van der Logt, EMJ, Piet, B, van Putten, J, Reitsma, BH, Rutgers, SR, de Vries, M, Stigt, JA, Groves, MR, Timens, W, Willems, SM, van Kempen, LC, Schuuring, E & van der Wekken, AJ 2024, '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', ESMO Open, vol. 9, no. 11, 103966. https://doi.org/10.1016/j.esmoop.2024.103966

TY - JOUR

T1 - Molecular Tumor Board of the University Medical Center Groningen (UMCG-MTB)

T2 - outcome of patients with rare or complex mutational profiles receiving MTB-advised targeted therapy

AU - de Jager, V. D.

AU - Plomp, P.

AU - Paats, M. S.

AU - van Helvert, S.

AU - Elst, A. ter

AU - van den Berg, A.

AU - Dubbink, H. J.

AU - van Geffen, W. H.

AU - Zhang, L.

AU - Hendriks, L. E.L.

AU - Hiltermann, T. J.N.

AU - Hiddinga, B. I.

AU - Hijmering-Kappelle, L. B.M.

AU - Jalving, M.

AU - Kluiver, J.

AU - Koopman, B.

AU - van Kruchten, M.

AU - van der Logt, E. M.J.

AU - Piet, B.

AU - van Putten, J.

AU - Reitsma, B. H.

AU - Rutgers, S. R.

AU - de Vries, M.

AU - Stigt, J. A.

AU - Groves, M. R.

AU - Timens, W.

AU - Willems, S. M.

AU - van Kempen, L. C.

AU - Schuuring, E.

AU - van der Wekken, A. J.

PY - 2024/11

Y1 - 2024/11

N2 - 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.

AB - 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.

UR - https://www.scopus.com/pages/publications/85208075446

U2 - 10.1016/j.esmoop.2024.103966

DO - 10.1016/j.esmoop.2024.103966

M3 - Article

C2 - 39500140

AN - SCOPUS:85208075446

SN - 2059-7029

VL - 9

JO - ESMO Open

JF - ESMO Open

IS - 11

M1 - 103966

ER -

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

Abstract

Bibliographical note

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