晚期/转移性甲状腺髓样癌患者的诊断特点、治疗模式和临床结果

IF 16.4 1区 化学 Q1 CHEMISTRY, MULTIDISCIPLINARY
Rohan Parikh, Lisa M Hess, Elizabeth Esterberg, Naleen Raj Bhandari, James A Kaye
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引用次数: 5

摘要

背景:甲状腺髓样癌(MTC)约占甲状腺癌新发病例的1.6%。本研究的目的是描述美国现实环境中晚期/转移性MTC患者的患者特征、生物标志物检测、治疗模式和临床结果,并确定这些患者护理中的潜在差距。方法:选定的肿瘤学家回顾性分析年龄≥12岁诊断为晚期MTC的患者的病历。患者必须在2013年1月至2018年12月期间接受≥1条晚期/转移性MTC的全身治疗才有资格。对患者特征、生物标志物检测和治疗模式进行了描述性总结;使用Kaplan-Meier法估计无进展生存期(PFS)和总生存期(OS)。结果:本研究纳入的203例患者的平均(SD)年龄为52.2(10.4)岁;从一线治疗开始的平均随访时间(SD)为24.5(16.0)个月。大多数患者(82.8%)最初诊断为IVA、IVB或IVC期疾病。在所有患者中,有121例(59.6%)进行了RET突变检测,其中37.2%为RET突变型MTC。28例患者报告ret突变型;最常见的突变是M918T(64.3%)和C634R(32.1%)。在203例患者中,75.9%的患者仅接受了一种晚期疾病的全身治疗,36%的患者在数据提取时仍在接受一线治疗。卡博赞替尼(30.0%)、万德替尼(30.0%)、索拉非尼(17.2%)和lenvatinib(4.9%)是最常见的一线治疗药物。在49名接受二线治疗的患者中,大多数接受了卡博赞替尼(22.4%)、万德替尼(20.4%)、lenvatinib(12.2%)或舒尼替尼(12.2%)。一线和二线治疗开始后的中位PFS(95%可信区间[CI])分别为26.6个月(20.8-60.8)和15.3个月(6.6-不可估计[NE])。一线和二线治疗开始后的中位OS分别为63.8个月(46.3个ne)和22.4个月(12.4个ne)。结论:对于晚期/转移性MTC的治疗,在一线和二线治疗中没有观察到系统性药物排序的特异性偏好。考虑到最近批准选择性RET抑制剂用于RET突变型MTC患者,未来的研究应该调查这些患者的治疗模式如何演变。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Diagnostic characteristics, treatment patterns, and clinical outcomes for patients with advanced/metastatic medullary thyroid cancer.

Diagnostic characteristics, treatment patterns, and clinical outcomes for patients with advanced/metastatic medullary thyroid cancer.

Diagnostic characteristics, treatment patterns, and clinical outcomes for patients with advanced/metastatic medullary thyroid cancer.

Diagnostic characteristics, treatment patterns, and clinical outcomes for patients with advanced/metastatic medullary thyroid cancer.

Background: Medullary thyroid cancer (MTC) accounts for approximately 1.6% of new cases of thyroid cancer. The objective of this study was to describe patient characteristics, biomarker testing, treatment patterns, and clinical outcomes among patients with advanced/metastatic MTC in a real-world setting in the United States and to identify potential gaps in the care of these patients.

Methods: Selected oncologists retrospectively reviewed medical records of patients aged ≥ 12 years diagnosed with advanced MTC. Patients must have initiated ≥ 1 line of systemic treatment for advanced/metastatic MTC between January 2013-December 2018 to be eligible. Patient characteristics, biomarker testing, and treatment patterns were summarized descriptively; progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan-Meier method.

Results: The 203 patients included in this study had a mean (SD) age of 52.2 (10.4) years; mean (SD) duration of follow-up from start of first-line treatment was 24.5 (16.0) months. Most patients (82.8%) were initially diagnosed with stage IVA, IVB, or IVC disease. Among all patients, 121 (59.6%) had testing for RET mutations, of whom 37.2% had RET-mutant MTC. The RET-mutation type was reported for 28 patients; the most common mutations reported were M918T (64.3%) and C634R (32.1%). Of the 203 patients, 75.9% received only one line of systemic treatment for advanced disease, and 36% were still undergoing first-line therapy at the time of data extraction. Cabozantinib (30.0%), vandetanib (30.0%), sorafenib (17.2%), and lenvatinib (4.9%) were the most common first-line treatments. Among 49 patients who received second-line treatment, most received cabozantinib (22.4%), vandetanib (20.4%), lenvatinib (12.2%), or sunitinib (12.2%). Median PFS (95% confidence interval [CI]) from start of first- and second-line treatments was 26.6 months (20.8-60.8) and 15.3 months (6.6-not estimable [NE]), respectively. Median OS from initiation of first- and second-line treatment was 63.8 months (46.3-NE) and 22.4 months (12.4-NE), respectively.

Conclusions: For the treatment of advanced/metastatic MTC, no specific preference of sequencing systemic agents was observed in the first- and second-line settings. Considering the recent approval of selective RET inhibitors for patients with RET-mutant MTC, future research should investigate how treatment patterns evolve for these patients.

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来源期刊
Accounts of Chemical Research
Accounts of Chemical Research 化学-化学综合
CiteScore
31.40
自引率
1.10%
发文量
312
审稿时长
2 months
期刊介绍: Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.
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