Real-world clinical profile, RET mutation testing, treatments, and PROs for MTC in Europe.

IF 3.5 2区 医学 Q2 ENDOCRINOLOGY & METABOLISM European Thyroid Journal Pub Date : 2024-01-01 DOI:10.1530/ETJ-23-0172
Grace Segall, Ravinder Singh, Min-Hua Jen, Isaac Sanderson, Alex Rider, Katie Lewis, Urpo Kiiskinen
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Abstract

Objective: This study aimed to describe real-world patient and physician characteristics, rearranged during transfection (RET) mutation testing and results, treatment patterns, and patient-reported outcomes (PROs) in advanced or metastatic medullary thyroid cancer (aMTC) across five populous European countries.

Methods: Cross-sectional physician and patient surveys were used to collect quantitative and qualitative data in France, Germany, Italy, Spain, and the UK from July to December 2020, prior to the introduction of selective RET inhibitors in Europe. Physicians completed patient record forms and a survey about their specialty and practice site. Patients were asked to provide PRO data using four validated instruments, including the EuroQol 5 Dimension (EQ-5D) questionnaire.

Results: The physician-reported sample included 275 patients with aMTC, including 79 patients with RET mutation-positive disease; median age was 60 and 56 years, respectively. Overall, 75% were tested for RET mutation (35% germline only, 21% somatic only, 44% both). Common physician-cited barriers to RET mutation testing included high cost, difficulty accessing latest tests, and time delay for results. First-line systemic therapy (most commonly vandetanib or cabozantinib) was prescribed for 69% of patients overall and 82% of the RET mutation-positive subgroup. Second-line therapy was prescribed for 12% of patients who received first-line therapy; most patients remained on first-line therapy at data capture. PROs revealed substantial disease/treatment burden.

Conclusions: Patients with aMTC report substantial disease/treatment burden. Outcomes could be improved by identifying patients eligible for treatment with selective RET inhibitors through more optimal RET mutation testing.

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欧洲 MTC 的真实临床概况、RET 基因突变检测、治疗方法和 PROs。
研究目的本研究旨在描述五个人口众多的欧洲国家晚期或转移性甲状腺髓样癌 (aMTC) 患者和医生的真实特征、转染期间重排(RET)突变检测和结果、治疗模式以及患者报告结果(PROs):方法:在欧洲引入选择性 RET 抑制剂之前,于 2020 年 7 月至 12 月在法国、德国、意大利、西班牙和英国进行了横断面医生和患者调查,以收集定量和定性数据。医生填写了患者记录表,并对其专业和执业地点进行了调查。患者被要求使用四种经过验证的工具提供PRO数据,包括EuroQol 5 Dimension (EQ-5D)问卷:医生报告的样本包括 275 名 aMTC 患者,其中包括 79 名 RET 基因突变阳性患者;中位年龄分别为 60 岁和 56 岁。总体而言,75%的患者接受了RET基因突变检测(35%仅为种系突变,21%仅为体细胞突变,44%两者均有)。医生普遍认为进行 RET 基因突变检测的障碍包括费用高昂、难以获得最新检测结果以及检测结果的时间延迟。69%的总体患者和82%的RET突变阳性亚组患者接受了一线系统治疗(最常见的是凡德他尼或卡博替尼)。接受一线治疗的患者中有 12% 接受了二线治疗;数据采集时,大多数患者仍在接受一线治疗。PROs显示了巨大的疾病/治疗负担:结论:MTC 患者的疾病/治疗负担很重。通过更优化的 RET 基因突变检测,确定符合选择性 RET 抑制剂治疗条件的患者,可以改善治疗效果。
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来源期刊
European Thyroid Journal
European Thyroid Journal Medicine-Endocrinology, Diabetes and Metabolism
CiteScore
6.70
自引率
2.10%
发文量
156
期刊介绍: The ''European Thyroid Journal'' publishes papers reporting original research in basic, translational and clinical thyroidology. Original contributions cover all aspects of the field, from molecular and cellular biology to immunology and biochemistry, from physiology to pathology, and from pediatric to adult thyroid diseases with a special focus on thyroid cancer. Readers also benefit from reviews by noted experts, which highlight especially active areas of current research. The journal will further publish formal guidelines in the field, produced and endorsed by the European Thyroid Association.
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