Gupta Diviya, Kurzrock Razelle, Lee Suzanna, Okamura Ryosuke, Lim Hyo Jeong, Kim Ki Hwan, Sicklick Jason K, Kato Shumei
School of Medicine, University of California San Diego, La Jolla, CA, USA.
Center for Personalized Cancer Therapy, UC San Diego Moores Cancer Center, La Jolla, CA, USA.
NPJ Precis Oncol. 2022 Mar 28;6(1):18. doi: 10.1038/s41698-022-00259-7.
Though advanced cancers generally display complex molecular portfolios, there is a subset of patients whose malignancies possess only one genomic alteration or alterations in one oncogenic pathway. We assess how N-of-One therapeutic strategies impact outcomes in these patients. From 12/2012 to 9/2018, 429 therapy-evaluable patients with diverse treatment-refractory cancers were presented at Molecular Tumor Boards at Moores Cancer Center at UC San Diego. The clinical benefit rate, defined by RECIST1.1, was assessed for patients with solid tumors who underwent next-generation sequencing (NGS) profiling revealing one genomic or pathway alteration, subsequently managed with N-of-One therapies. Nine of 429 patients (2.1%) met evaluation criteria. Using matched therapy indicated by NGS, the clinical benefit rate (stable disease ≥ 6 months/partial/complete response) was 66.7%. Median progression-free survival was 11.3 months (95% CI: 3.4-not evaluable). Thus, a small subset of diverse cancers has single pathway alterations on NGS testing. These patients may benefit from customized therapeutic matching.
尽管晚期癌症通常表现出复杂的分子特征，但有一部分患者的恶性肿瘤仅存在一种基因组改变或一条致癌途径的改变。我们评估了单病例治疗策略对这些患者治疗结果的影响。2012年12月至2018年9月期间，429例可评估治疗的患有各种难治性癌症的患者在加州大学圣地亚哥分校穆尔斯癌症中心的分子肿瘤委员会接受了评估。对于接受下一代测序（NGS）分析显示一种基因组或途径改变、随后采用单病例治疗的实体瘤患者，评估了根据RECIST1.1定义的临床获益率。429例患者中有9例（2.1%）符合评估标准。使用NGS指示的匹配治疗，临床获益率（疾病稳定≥6个月/部分/完全缓解）为66.7%。无进展生存期的中位数为11.3个月（95%CI：3.4-不可评估）。因此，一小部分不同类型的癌症在NGS检测中存在单一途径改变。这些患者可能从定制的治疗匹配中获益。
