格罗宁根大学医学中心分子肿瘤委员会（UMCG-MTB）：接受 MTB 建议的靶向治疗的罕见或复杂突变患者的治疗结果

IF 7.1 2区医学 Q1 ONCOLOGY

ESMO Open Pub Date : 2024-11-01 DOI:10.1016/j.esmoop.2024.103966

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 , A.J. van der Wekken

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引用次数: 0

摘要

目的分子肿瘤委员会（MTB）被认为有利于对具有不常见、罕见或复杂突变特征的癌症患者做出治疗决策。由于缺乏国际性的MTB指南，因此在实践和建议方面存在很大差异。因此，有必要进行定期随访，以评估和管理 MTB 的功能。本研究的目的是确定格罗宁根大学医学中心（UMCG-MTB）MTB在2019-2020年针对罕见和复杂突变特征患者实施的治疗建议的有效性。患者和方法进行了一项回顾性随访研究，以确定不常见或罕见（组合）分子畸变患者的临床结果，UMCG-MTB在2019年和2020年建议将靶向治疗作为下一步治疗方案。结果UMCG-MTB建议将靶向治疗作为327名患者中132名患者的下一步治疗方案：结果UMCG-MTB推荐327名患者中的132名患者接受靶向治疗，其中37名患者接受了临床试验，67名患者接受了标签内治疗，28名患者接受了标签外治疗。就标示内和标示外治疗建议而言，在可进行随访的患者中，建议治疗与接受治疗的一致性为 85%（67/79）。标签内治疗的反应率为 50%（21/42），中位无进展生存期（PFS）为 6.3 个月（四分位距（IQR）为 2.9-14.9 个月），中位总生存期（OS）为 15.8 个月（IQR 为 6.4-34.2 个月）。标示外治疗的反应率为53%（8/15），中位PFS为5.1个月（IQR为1.9-7.3个月），中位OS为17.7个月（IQR为5.1-23.7个月）。根据 MTB 确定的充分理由，标签外使用靶向疗法是一种有效的治疗策略。这项研究强调了在 MTB 中讨论具有罕见和复杂突变特征的患者的意义。

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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

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.

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来源期刊

ESMO Open Medicine-Oncology

CiteScore

11.70

自引率

2.70%

发文量

255

审稿时长

10 weeks

期刊介绍： ESMO Open is the online-only, open access journal of the European Society for Medical Oncology (ESMO). It is a peer-reviewed publication dedicated to sharing high-quality medical research and educational materials from various fields of oncology. The journal specifically focuses on showcasing innovative clinical and translational cancer research. ESMO Open aims to publish a wide range of research articles covering all aspects of oncology, including experimental studies, translational research, diagnostic advancements, and therapeutic approaches. The content of the journal includes original research articles, insightful reviews, thought-provoking editorials, and correspondence. Moreover, the journal warmly welcomes the submission of phase I trials and meta-analyses. It also showcases reviews from significant ESMO conferences and meetings, as well as publishes important position statements on behalf of ESMO. Overall, ESMO Open offers a platform for scientists, clinicians, and researchers in the field of oncology to share their valuable insights and contribute to advancing the understanding and treatment of cancer. The journal serves as a source of up-to-date information and fosters collaboration within the oncology community.