Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe
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...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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Bioscientifica
2024-02-01
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| Series: | European Thyroid Journal |
| Subjects: | |
| Online Access: | https://etj.bioscientifica.com/view/journals/etj/13/1/ETJ-23-0172.xml |
| _version_ | 1797301274662666240 |
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| author | Grace Segall Ravinder Singh Min-Hua Jen Isaac Sanderson Alex Rider Katie Lewis Urpo Kiiskinen |
| author_facet | Grace Segall Ravinder Singh Min-Hua Jen Isaac Sanderson Alex Rider Katie Lewis Urpo Kiiskinen |
| author_sort | Grace Segall |
| collection | DOAJ |
| description | 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 and 44% both). Common physician-cited barriers to RET mutation testing included high cost, difficulty accessing the 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 a substantial disease/treatment burden.
Conclusion: Patients with aMTC report a substantial disease/treatment burden. Outcomes could be improved by identifying patients eligible for treatment with selective RET inhibitors through more optimal RET mutation testing. |
| first_indexed | 2024-03-07T23:19:13Z |
| format | Article |
| id | doaj.art-a890d240d3ca40ab92c23f209b1819cc |
| institution | Directory Open Access Journal |
| issn | 2235-0802 |
| language | English |
| last_indexed | 2024-03-07T23:19:13Z |
| publishDate | 2024-02-01 |
| publisher | Bioscientifica |
| record_format | Article |
| series | European Thyroid Journal |
| spelling | doaj.art-a890d240d3ca40ab92c23f209b1819cc2024-02-21T09:14:47ZengBioscientificaEuropean Thyroid Journal2235-08022024-02-01131112https://doi.org/10.1530/ETJ-23-0172Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in EuropeGrace Segall0Ravinder Singh1Min-Hua Jen2Isaac Sanderson3Alex Rider4Katie Lewis5Urpo Kiiskinen6Eli Lilly and Company, Indianapolis, IN, USAEli Lilly and Company, Indianapolis, IN, USAEli Lilly and Company, Indianapolis, IN, USAAdelphi Real World, Bollington, UKAdelphi Real World, Bollington, UKAdelphi Real World, Bollington, UKEli Lilly and Company, Indianapolis, IN, USAObjective: 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 and 44% both). Common physician-cited barriers to RET mutation testing included high cost, difficulty accessing the 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 a substantial disease/treatment burden. Conclusion: Patients with aMTC report a substantial disease/treatment burden. Outcomes could be improved by identifying patients eligible for treatment with selective RET inhibitors through more optimal RET mutation testing.https://etj.bioscientifica.com/view/journals/etj/13/1/ETJ-23-0172.xmlmedullary thyroid cancerret mutationreal-world datatreatment patternspatient-reported outcomeseurope |
| spellingShingle | Grace Segall Ravinder Singh Min-Hua Jen Isaac Sanderson Alex Rider Katie Lewis Urpo Kiiskinen Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe European Thyroid Journal medullary thyroid cancer ret mutation real-world data treatment patterns patient-reported outcomes europe |
| title | Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe |
| title_full | Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe |
| title_fullStr | Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe |
| title_full_unstemmed | Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe |
| title_short | Real-world clinical profile, RET mutation testing, treatments and patient-related outcomes for medullary thyroid cancer in Europe |
| title_sort | real world clinical profile ret mutation testing treatments and patient related outcomes for medullary thyroid cancer in europe |
| topic | medullary thyroid cancer ret mutation real-world data treatment patterns patient-reported outcomes europe |
| url | https://etj.bioscientifica.com/view/journals/etj/13/1/ETJ-23-0172.xml |
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