由于缺乏标准化疗法和临床试验，罕见/超罕见肿瘤的治疗需求尚未得到满足。我们开发了分子肿瘤委员会 (MTB)，这是一个多学科团队，整合分子分析，为晚期癌症提供个性化的多合一治疗方法。本研究评估了 112 名患有罕见/超罕见肿瘤的患者，这些患者曾就诊于 MTB，并且可以评估临床治疗结果。总体而言，46/112 名患者 (41%) 接受了肿瘤分子改变与所给予药物高度匹配的治疗方案（通过高匹配分数 (≥50%) 反映出来）。匹配评分高的患者与低匹配评分的患者相比，无进展生存期 (p = 0.005) 和总生存期 (p = 0.047) 显着更长，临床获益率更高（疾病稳定≥6 个月、部分缓解或完全缓解）（ 54% 与 32% p = 0.027）。 MTB 促进了药物与肿瘤分子改变的个性化 N-of-One 匹配，这与罕见/超罕见癌症患者临床结果的改善相关。© 2024 作者。

Treatment of rare/ultra-rare tumors is an unmet need due to a lack of standardized therapies and clinical trials. We developed the Molecular Tumor Board (MTB), a multidisciplinary team that integrates molecular profiling to generate personalized, N-of-One treatments for advanced cancers. This study evaluates 112 patients with rare/ultra-rare tumors who presented to the MTB and were evaluable for clinical therapeutic outcome. Overall, 46/112 patients (41%) received a treatment regimen with a high degree of matching between tumor molecular alterations and drugs given (reflected by a high Matching Score (≥50%)). Patients with a high versus low Matching Score experienced significantly longer progression-free survival (p = 0.005) and overall survival (p = 0.047), and higher rates of clinical benefit (stable disease ≥6 months, partial response, or complete response) (54% vs. 32% p = 0.027). The MTB facilitated personalized N-of-One matching of drugs to tumor molecular alterations, which was associated with improved clinical outcomes in patients with rare/ultra-rare cancers.© 2024 The Author(s).