Louie Bryan H, Kato Shumei, Lim Jordan S, Kim Ki Hwan, Lim Hyo Jeong, Okamura Ryosuke, Lee Suzanna, Kim Lisa, Sicklick Jason K, Lippman Scott M, Kurzrock Razelle
Center for Personalized Cancer Therapy and Division of Hematology and Oncology, Department of Medicine, UC San Diego Moores Cancer Center, La Jolla, CA, USA.
Division of Hematology and Medical Oncology, Department of Internal Medicine, Seoul National University Boramae Medical Center, Seoul, Republic of Korea.
iScience. 2024 Jul 5;27(8):110465. doi: 10.1016/j.isci.2024.110465. eCollection 2024 Aug 16.
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 ( = 0.005) and overall survival ( = 0.047), and higher rates of clinical benefit (stable disease ≥6 months, partial response, or complete response) (54% vs. 32% = 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.
由于缺乏标准化治疗方法和临床试验，罕见/超罕见肿瘤的治疗需求尚未得到满足。我们组建了分子肿瘤委员会（MTB），这是一个多学科团队，整合分子分析以生成针对晚期癌症的个性化单病例治疗方案。本研究评估了112例就诊于MTB且可评估临床治疗结果的罕见/超罕见肿瘤患者。总体而言，46/112例患者（41%）接受了肿瘤分子改变与所用药物高度匹配的治疗方案（以高匹配分数（≥50%）反映）。匹配分数高与低的患者无进展生存期（P = 0.005）和总生存期（P = 0.047）显著更长，临床获益率（疾病稳定≥6个月、部分缓解或完全缓解）更高（54%对32%，P = 0.027）。MTB促进了药物与肿瘤分子改变的个性化单病例匹配，这与罕见/超罕见癌症患者临床结局改善相关。
