Long-term Survivorship Challenges of Advanced/Metastatic GIST Patients Responding to Tyrosine Kinase Inhibitor Treatment: an Observational Study (EORTC-1944)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 330
- 试验地点
- 1
- 主要终点
- Health-Related Quality of Life assessed using the Item List.
研究概览
简要总结
Gastrointestinal stromal tumours (GISTs) are rare malignancies arising along the gastrointestinal tract. Tyrosine kinase inhibitors (TKIs) have substantially improved survival for patients with unresectable or metastatic GIST. As a result, an increasing number of patients live with advanced disease under chronic TKI therapy, highlighting the need to understand long-term survivorship, including health-related quality of life (HRQoL), treatment burden, and clinical outcomes.
This international, multicentre observational study includes two components: a retrospective cross-sectional study to evaluate the relevance and applicability of patient-reported outcome instruments, and a prospective cohort to document long-term clinical outcomes, HRQoL, treatment patterns, and survivorship challenges.
Participants will include adults with advanced or metastatic GIST who have received TKI therapy for at least 2 years. Data will be collected from medical records and through validated patient-reported outcome questionnaires at baseline and annually for up to 10 years. This study aims to provide insight into HRQoL problems, treatment discontinuation, coping strategies, and the impact of financial toxicity among long-term survivors of GIST.
详细描述
Gastrointestinal stromal tumour (GIST) is a rare malignancy for which long-term treatment with tyrosine kinase inhibitors (TKIs), particularly imatinib, has substantially improved survival. As a result, an increasing number of patients live with advanced or metastatic disease under chronic TKI therapy, highlighting the need to better understand long-term survivorship, including health-related quality of life (HRQoL), treatment burden, and clinical outcomes.
This study is an international, multicentre observational study consisting of two components:
- a retrospective cross-sectional study and
- a prospective observational cohort (registry).
Study design and population
The retrospective component includes patients with unresectable or metastatic GIST receiving long-term TKI treatment (≥5 years). This component aims to evaluate the relevance and applicability of patient-reported outcome (PRO) instruments and to identify GIST-specific survivorship issues.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18 years or older (no upper age limit);
- •Advanced or metastatic GIST, diagnosis of GIST must be histologically proven;
- •Treated with TKIs (eg. sunitinib, regorafenib, avapritinib, ripretinib) for at least 2 years; TKI treatment ongoing; interruptions up to 3 months are allowed;
- •Able to read and answer questionnaires;
- •Able to provide informed consent.
排除标准
- •Patients receiving TKI in an adjuvant treatment setting.
- •Any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before inclusion the study.
- •Patients who are too ill (death is imminent), as determined by the referring health care professional.
结局指标
主要结局
Health-Related Quality of Life assessed using the Item List.
时间窗: Baseline and at all scheduled follow-up assessments (every year for 10 years)
* Outcome measure: HRQoL Item List * Measurement instrument: EORTC-developed item list (Item list developed in phase 1 of this study). List consists of HRQoL issues specifically relevant to GIST patients receiving long-term TKI treatment. * Patients will be asked to rate the HRQoL issues on relevance (4 point Likert scale (1) not relevant - (4) very relevant), to prioritize the 10 most important issues, and to indicate relevant issues missing from this list. * Analysis: prevalence, severity, and longitudinal course of HRQoL issues
次要结局
- Health-Related Quality of Life measured by EORTC QLQ-C30(Baseline and scheduled follow-up assessments (every year for 10 years))
- Survivorship issues measured by EORTC QLQ-SURV100(Baseline and scheduled follow-up assessments (every year for 10 years))
- Health status measured by EQ-5D-5L(Baseline and at all scheduled follow-up assessments (every year for 10 years))
- Overall Survival (OS)(From diagnosis to death or last follow-up (follow-up will be 10 years))
- Progression-Free Survival (PFS)(From start of TKI treatment to progression, death, or last follow-up (follow-up is 10 years))
- Tumour response assessed by RECIST(At each radiologic assessment, annually up to 10 years.)
- Reasons for discontinuation of tyrosine kinase inhibitor (TKI) treatment(Annually up to 10 years.)
- Coping style measured by Threatening Medical Situations Inventory (TMSI)(Baseline and annually up to 10 years.)
- Illness cognitions measured by Illness Cognition Questionnaire (ICQ)(Baseline and annually up to 10 years.)
- Financial toxicity measured by Financial Index of Toxicity (FIT)(Baseline, annually up to 10 years.)
- Work productivity loss measured by Productivity Cost Questionnaire(Baseline, annually up to 10 years.)
- Healthcare utilization and informal care use measured by Medical Consumption Questionnaire(Baseline, annually up to 10 years.)
- Tyrosine Kinase Inhibitor (TKI) Plasma Concentration(Baseline and annually up to 10 years.)