文章：

美国选择性RET抑制剂塞尔帕替尼的真实世界疗效：基于两项数据库的描述性回顾性研究结果

Real-World Outcomes of SelectiveRETInhibitor Selpercatinib in the United States: Descriptive, Retrospective Findings from Two Databases

原文发布日期：15 November 2024

DOI: 10.3390/cancers16223835

类型: Article

开放获取: 是

英文摘要：

Objectives: This study described real-world patient characteristics and outcomes among selpercatinib-treated patients in the United States, using the Flatiron Health electronic health record-derived deidentified database (FHD) for advanced/metastatic non-small cell lung cancer (a/mNSCLC) and Optum’s de-identified Clinformatics®Data Mart Database (CDM). Methods: Patients initiating selpercatinib treatment between 08MAY2020 and 30JUN2023 were included. We evaluated real-world time to selpercatinib treatment discontinuation or death (rwTTDd) and time to next treatment or death (rwTTNTd) using Kaplan–Meier analyses. Medication possession ratio (MPR) was estimated as a measure of medication adherence in CDM patients. Results: In a/mNSCLC patients from the FHD (N = 68), the median rwTTDd and rwTTNTd were 22.4 [95%CI: 13.3–NR] and 21.0 [95%CI: 11.6–NR] months, respectively. In CDM, these durations were 12.1 [95%CI: 9.6–NR] and 16.2 [95%CI: 9.6–NR] months for lung cancer (n = 43), while these were not reached for thyroid cancer (n = 24) patients. The median MPR was 0.98 [IQR: 0.84–1.00] among all patients in the CDM (N = 75), with 77.3% of patients adhering (MPR ≥ 0.80) to selpercatinib. Conclusions: Real-world outcomes in this older and frailer patient cohort align with phase 3 trial results, further supporting selpercatinib as the standard of care for patients withRET-altered cancers. Early testing for the detection ofRETalterations remains essential.

摘要翻译： 

目的：本研究利用Flatiron Health电子健康记录去标识化数据库（FHD）中晚期/转移性非小细胞肺癌（a/mNSCLC）数据及Optum去标识化Clinformatics®数据市场数据库（CDM），描述美国接受塞尔帕替尼治疗患者的真实世界特征与结局。方法：纳入2020年5月8日至2023年6月30日期间开始塞尔帕替尼治疗的患者。采用Kaplan-Meier分析法评估真实世界治疗终止或死亡时间（rwTTDd）及至下次治疗或死亡时间（rwTTNTd）。通过药物持有率（MPR）评估CDM患者的用药依从性。结果：在FHD的a/mNSCLC患者（N=68）中，中位rwTTDd与rwTTNTd分别为22.4个月[95%CI: 13.3–未达到]和21.0个月[95%CI: 11.6–未达到]。CDM数据库中肺癌患者（n=43）的对应时间分别为12.1个月[95%CI: 9.6–未达到]和16.2个月[95%CI: 9.6–未达到]，甲状腺癌患者（n=24）的相关时间尚未达到。CDM全体患者（N=75）的中位MPR为0.98[IQR: 0.84–1.00]，77.3%的患者对塞尔帕替尼保持依从性（MPR≥0.80）。结论：在这类年龄更大、健康状况更脆弱的患者群体中观察到的真实世界结局与III期临床试验结果一致，进一步支持塞尔帕替尼作为RET基因变异癌症的标准治疗方案。早期检测RET基因变异仍然至关重要。

原文链接：

Real-World Outcomes of SelectiveRETInhibitor Selpercatinib in the United States: Descriptive, Retrospective Findings from Two Databases