Boscolo Bielo L, Crimini E, Repetto M, Barberis M, Battaiotto E, Katrini J, Martino E, Gaudio G, Lombardi M, Zanzottera C, Aurilio G, Belli C, Zhan Y, Fuorivia V, Marsicano R M, Etessami J D, Zagami P, Marra A, Trapani D, Taurelli Salimbeni B, Criscitiello C, Ciardiello D, Lavinia B, Gervaso L, Cella C A, Spada F, Zampino M G, Fusco N, Fazio N, Guerini Rocco E, Curigliano G
Early Drug Development for Innovative Therapies, European Institute of Oncology IRCCS, Milan, Italy; Department of Oncology and Hemato-Oncology, University of Milano, Milan, Italy.
Early Drug Development Service, Memorial Sloan-Kettering Cancer Center, New York, USA.
ESMO Open. 2025 Apr;10(4):104510. doi: 10.1016/j.esmoop.2025.104510. Epub 2025 Mar 19.
Comprehensive genomic profiling (CGP) is being increasingly adopted in clinical practice to guide the use of molecularly guided treatment options (MGTOs). To optimize the integration of MGTOs in routine cancer care, molecular tumour boards (MTBs) have been established. Limited data are available to address the clinical value of implementing MTBs to inform treatment decision making in patients with gastrointestinal (GI) cancers.
We retrospectively retrieved medical records from patients with advanced GI cancers discussed at the European Institute of Oncology's MTB between August 2019 and December 2024. We evaluated clinical outcomes resulting from applying MGTOs in cancer care according to MTB recommendations, describing real-world progression-free (rwPFS) and overall survival (OS), and used the growth modulation index (GMI) (ratio of PFS to PFS) to quantify the effectiveness of MTB's recommended cancer treatment in extending PFS.
Among 192 patients with GI cancers discussed at MTB, 139 (72.3%) received an MTB treatment recommendation. For patients with available follow-up data (n = 82), 31 patients (41.4%, 17.7% overall) received MGTOs, while 51 patients received standard treatments. Patients receiving MGTOs exhibited a longer rwPFS compared with cases receiving standard therapies [5.35 versus 3.55 months, hazard ratio (HR) 0.62, 95% confidence interval (CI) 0.36-1.08, P = 0.08] and with unmatched cases showing actionable biomarkers but not treated with targeted agents (n = 31) (rwPFS 5.35 versus 2.40 months, HR 0.49, 95% CI 0.27-0.90, P = 0.02). The use of MGTOs resulted in a GMI of 1.12 (interquartile range 0.68-2.36). The European Society for Medical Oncology (ESMO) Scale for Clinical Actionability of molecular Targets (ESCAT) tier I-III treatments resulted in a restricted mean PFS gain of 4.87 months compared with standard therapies (95% CI 1.02-8.72 months, P = 0.01). No OS difference was observed between patients receiving MGTOs and standard treatments (P = 0.89).
Our results suggest that MTB-informed clinical decision making could provide valuable clinical benefits and expanded therapeutic options in patients affected by advanced GI cancers.
综合基因组分析(CGP)在临床实践中越来越多地被用于指导分子导向治疗方案(MGTO)的使用。为了优化MGTO在常规癌症治疗中的整合,已设立了分子肿瘤学委员会(MTB)。关于实施MTB以指导胃肠道(GI)癌症患者治疗决策的临床价值,现有数据有限。
我们回顾性检索了2019年8月至2024年12月期间在欧洲肿瘤研究所MTB讨论的晚期GI癌症患者的病历。我们根据MTB的建议评估了在癌症治疗中应用MGTO所产生的临床结果,描述了真实世界的无进展生存期(rwPFS)和总生存期(OS),并使用生长调节指数(GMI)(PFS与PFS的比值)来量化MTB推荐的癌症治疗在延长PFS方面的有效性。
在MTB讨论的192例GI癌症患者中,139例(72.3%)接受了MTB的治疗建议。对于有可用随访数据的患者(n = 82),31例患者(41.4%,总体为17.7%)接受了MGTO,而51例患者接受了标准治疗。与接受标准治疗的患者相比,接受MGTO的患者表现出更长的rwPFS[5.35个月对3.55个月,风险比(HR)0.62,95%置信区间(CI)0.36 - 1.08,P = 0.08],并且与显示可操作生物标志物但未接受靶向药物治疗的非匹配患者(n = 31)相比也是如此(rwPFS 5.35个月对2.40个月,HR 0.49,95%CI 0.27 - 0.90,P = 0.02)。MGTO的使用导致GMI为1.12(四分位间距0.68 - 2.36)。与标准治疗相比,欧洲医学肿瘤学会(ESMO)分子靶点临床可操作性量表(ESCAT)I - III级治疗导致受限平均PFS增加4.87个月(95%CI 1.02 - 8.72个月,P = 0.01)。接受MGTO和标准治疗的患者之间未观察到OS差异(P = 0.89)。
我们的结果表明,基于MTB的临床决策可为晚期GI癌症患者提供有价值的临床益处和更多的治疗选择。