Andrew G Gianoukakis, Jennifer H. Choe, Daniel W. Bowles, Marcia S. Brose, Lori Wirth, Taofeek Owonikoko, Svetlana Babajanyan, Francis P. Worden
{"title":"RAI 难治性分化型甲状腺癌的实际应用模式和结果","authors":"Andrew G Gianoukakis, Jennifer H. Choe, Daniel W. Bowles, Marcia S. Brose, Lori Wirth, Taofeek Owonikoko, Svetlana Babajanyan, Francis P. Worden","doi":"10.1530/etj-23-0039","DOIUrl":null,"url":null,"abstract":"<p>Background: The optimal timing for initiating multi-kinase inhibitors (MKIs) in patients with radioactive iodine-refractory (RAI-R) differentiated thyroid cancer (DTC) remains unclear. Thus, we evaluated the real-world practice patterns and outcomes in asymptomatic patients with progressive RAI-R DTC (≥1 lesion ≥1 cm in diameter) in the United States (US) and outside the US (non-US). </p><p>Methods: In this prospective, non-interventional, open-label study, eligible patients were chosen by treating physicians to receive MKI therapy (Cohort 1) or undergo active surveillance (Cohort 2) at study entry. Cohort 2 patients were allowed to transition to MKI therapy later. The primary endpoint was time to symptomatic progression (TTSP) from study entry. Data were compared descriptively. When endpoints were inestimable, 36-month rates were calculated.\n</p><p>Results: Among 647 patients, 478 underwent active surveillance (Cohort 2) and 169 received MKI treatment (Cohort 1). Patients underwent surveillance at a higher rate in the US (92.6%) versus the non-US (66.9%). Half of US and non-US patients who qualified for MKI treatment had initial American Thyroid Association (ATA) low-to-intermediate-risk disease. Among Cohort 2, the 36-month TTSP rates from study entry were 65.6% and 66.5% in the US and non-US, respectively. Cohort 2 patients treated later demonstrated 36-month TTSP rates of 30.8% and 55.8% in the US and non-US, respectively.\n</p><p>Conclusions: Active surveillance is a viable option for asymptomatic patients with progressive RAI-R DTC. However, early intervention with MKI therapy may be more suitable for others. Further research is needed to identify patients who are optimal for active surveillance. </p><p>Registration: NCT02303444</p>","PeriodicalId":12159,"journal":{"name":"European Thyroid Journal","volume":"43 1","pages":""},"PeriodicalIF":3.5000,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Real-World Practice Patterns and Outcomes for RAI-Refractory Differentiated Thyroid Cancer\",\"authors\":\"Andrew G Gianoukakis, Jennifer H. Choe, Daniel W. Bowles, Marcia S. Brose, Lori Wirth, Taofeek Owonikoko, Svetlana Babajanyan, Francis P. Worden\",\"doi\":\"10.1530/etj-23-0039\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Background: The optimal timing for initiating multi-kinase inhibitors (MKIs) in patients with radioactive iodine-refractory (RAI-R) differentiated thyroid cancer (DTC) remains unclear. Thus, we evaluated the real-world practice patterns and outcomes in asymptomatic patients with progressive RAI-R DTC (≥1 lesion ≥1 cm in diameter) in the United States (US) and outside the US (non-US). </p><p>Methods: In this prospective, non-interventional, open-label study, eligible patients were chosen by treating physicians to receive MKI therapy (Cohort 1) or undergo active surveillance (Cohort 2) at study entry. Cohort 2 patients were allowed to transition to MKI therapy later. The primary endpoint was time to symptomatic progression (TTSP) from study entry. Data were compared descriptively. When endpoints were inestimable, 36-month rates were calculated.\\n</p><p>Results: Among 647 patients, 478 underwent active surveillance (Cohort 2) and 169 received MKI treatment (Cohort 1). Patients underwent surveillance at a higher rate in the US (92.6%) versus the non-US (66.9%). Half of US and non-US patients who qualified for MKI treatment had initial American Thyroid Association (ATA) low-to-intermediate-risk disease. Among Cohort 2, the 36-month TTSP rates from study entry were 65.6% and 66.5% in the US and non-US, respectively. Cohort 2 patients treated later demonstrated 36-month TTSP rates of 30.8% and 55.8% in the US and non-US, respectively.\\n</p><p>Conclusions: Active surveillance is a viable option for asymptomatic patients with progressive RAI-R DTC. However, early intervention with MKI therapy may be more suitable for others. Further research is needed to identify patients who are optimal for active surveillance. </p><p>Registration: NCT02303444</p>\",\"PeriodicalId\":12159,\"journal\":{\"name\":\"European Thyroid Journal\",\"volume\":\"43 1\",\"pages\":\"\"},\"PeriodicalIF\":3.5000,\"publicationDate\":\"2023-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Thyroid Journal\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1530/etj-23-0039\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"ENDOCRINOLOGY & METABOLISM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Thyroid Journal","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1530/etj-23-0039","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
Real-World Practice Patterns and Outcomes for RAI-Refractory Differentiated Thyroid Cancer
Background: The optimal timing for initiating multi-kinase inhibitors (MKIs) in patients with radioactive iodine-refractory (RAI-R) differentiated thyroid cancer (DTC) remains unclear. Thus, we evaluated the real-world practice patterns and outcomes in asymptomatic patients with progressive RAI-R DTC (≥1 lesion ≥1 cm in diameter) in the United States (US) and outside the US (non-US).
Methods: In this prospective, non-interventional, open-label study, eligible patients were chosen by treating physicians to receive MKI therapy (Cohort 1) or undergo active surveillance (Cohort 2) at study entry. Cohort 2 patients were allowed to transition to MKI therapy later. The primary endpoint was time to symptomatic progression (TTSP) from study entry. Data were compared descriptively. When endpoints were inestimable, 36-month rates were calculated.
Results: Among 647 patients, 478 underwent active surveillance (Cohort 2) and 169 received MKI treatment (Cohort 1). Patients underwent surveillance at a higher rate in the US (92.6%) versus the non-US (66.9%). Half of US and non-US patients who qualified for MKI treatment had initial American Thyroid Association (ATA) low-to-intermediate-risk disease. Among Cohort 2, the 36-month TTSP rates from study entry were 65.6% and 66.5% in the US and non-US, respectively. Cohort 2 patients treated later demonstrated 36-month TTSP rates of 30.8% and 55.8% in the US and non-US, respectively.
Conclusions: Active surveillance is a viable option for asymptomatic patients with progressive RAI-R DTC. However, early intervention with MKI therapy may be more suitable for others. Further research is needed to identify patients who are optimal for active surveillance.
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.