Del Vecchio Michele, Chiarion Sileni Vanna, Quaglino Pietro, Rinaldi Gaetana, Minisini Alessandro, Troiani Teresa, Consoli Francesca, Sponghini Andrea, Banzi Maria, Morelli Maria Francesca, Palleschi Dario, Rossi Ernesto, Marconcini Riccardo, Depenni Roberta, Carnevale-Schianca Fabrizio, Marcon Ilaria, Queirolo Paola
Fondazione IRCCS Istituto Nazionale dei Tumori, 20133 Milan, Italy.
Veneto Institute of Oncology IOV-IRCCS, 35128 Padova, Italy.
Cancers (Basel). 2023 Mar 26;15(7):1980. doi: 10.3390/cancers15071980.
In patients with B-RAF-mutated cutaneous melanoma, targeted therapies are the treatment of choice to achieve a rapid response. In this multicentric, prospective, observational study, patients with B-RAF-mutated cutaneous melanoma who were treated with dabrafenib and trametinib were categorized in two cohorts (cohort A: limited disease (n = 104) and cohort B: bulky disease (n = 97)) according to lactate dehydrogenase levels. The primary endpoint was the progression pattern; the secondary endpoints were overall survival (OS), progression-free survival (PFS), and safety data. From baseline to time of progression, there was a progression from nodal to other sites of disease in cohort A and from skin and nodal to other sites in cohort B. In both the cohorts, the number of involved organs and metastases at each location decreased. The median OS was 32.4 months (95% CI: 20.1 months (not estimable)) for cohort A, and 10.5 months (95% CI: 8.3-14.4 months) for cohort B; median PFS was 12.4 months (95% CI: 10.9-17.0 months) for cohort A, and 8.1 months (95% CI: 6.3-9.4 months) for cohort B. No new safety signals were reported. This study describes the patterns of first-line treatment progression with dabrafenib and trametinib in Italian clinical practice. The effectiveness and safety data were consistent with previous trials and extended to a real-world heterogeneous population.
在B-RAF突变的皮肤黑色素瘤患者中，靶向治疗是实现快速反应的首选治疗方法。在这项多中心、前瞻性、观察性研究中，接受达拉非尼和曲美替尼治疗的B-RAF突变皮肤黑色素瘤患者根据乳酸脱氢酶水平被分为两个队列（队列A：局限性疾病（n = 104）和队列B：广泛性疾病（n = 97））。主要终点是疾病进展模式；次要终点是总生存期（OS）、无进展生存期（PFS）和安全性数据。从基线到疾病进展时，队列A中疾病从淋巴结转移到其他部位，队列B中疾病从皮肤和淋巴结转移到其他部位。在两个队列中，每个部位受累器官和转移灶的数量均减少。队列A的中位OS为32.4个月（95%CI：20.1个月（不可估计）），队列B为10.5个月（95%CI：8.3 - 14.4个月）；队列A的中位PFS为12.4个月（95%CI：10.9 - 17.0个月），队列B为8.1个月（95%CI：6.3 - 9.4个月）。未报告新的安全信号。本研究描述了意大利临床实践中达拉非尼和曲美替尼一线治疗的疾病进展模式。有效性和安全性数据与先前试验一致，并扩展到了真实世界的异质性人群。
