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.
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;
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.
Advanced/metastatic GIST patients responding to tyrosine kinase inhibitor treatment Exclusion criteria: * Any psychiatric condition or cognitive impairment that would hamper completion of self-reported questionnaires. * Patients who are too ill, as determined by the referring health care professional. Part B - Prospective observational cohort Potentially, we estimate to be able to include 330 patients. According to the sample size calculation we need a minimum of 228 patients. Inclusion criteri
Cohort
Prospective
Not listed
Health-Related Quality of Life assessed using the Item List.
* 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
Health-Related Quality of Life measured by EORTC QLQ-C30
The EORTC QLQ-C30 is a ) is a validated, 30-item HRQoL questionnaire consisting of five functional scales (physical, role, cognitive, emotional and social), a global quality of life scale, three symptom scales (fatigue, pain, nausea and vomiting) and a number of single items assessing common symptoms (dyspnea, loss of appetite, sleep disturbance, constipation and diarrhea) and perceived financial impact of the disease. Items are scored on a four-point Likert scale (1 = not at all, 2 = a little, 3 = quite a bit, 4 = very much). After linear transformation, all scales and single item measures range in score from 0-100. A higher score on the functional scales and global QoL means better functioning and HRQoL, whereas a higher score on the symptom scales means more complaints. To determine cut-off for impaired HRQoL, the thresholds for clinical importance of Giesinger et al will be used.
Survivorship issues measured by EORTC QLQ-SURV100
The EORTC QLQ-SURV100 assesses survivorship-related issues in cancer patients. The QLQ-SURV100 is a 100 item HRQoL questionnaire consisting of 13 functional scales, 9 symptom scales, a Symptom Checklist of Chronic Side Effects of Treatment, and 12 single items. Items are scored on a four-point Likert scale (1 = not at all, 2 = a little, 3 = quite a bit, 4 = very much). After linear transformation, all scales and single item measures range in score from 0-100.
Health status measured by EQ-5D-5L
The EQ-5D-5L measures health status in two parts: a descriptive part and the EuroQol Visual Analogue Scale (EQ-VAS). The descriptive part assesses health in five generic dimensions (mobility, self-care, usual activity, pain and discomfort, and anxiety and/or depression), each of which has five levels of response (no problems till extreme problems/unable to). Each health state can potentially be assigned a summary index score based on societal preference weights for the health state. These weights, sometimes referred to as 'utilities', are often used to compute QALYs for use in health economic analyses. Health state index scores range from 0 (equivalent to dead) to 1 (equivalent of perfect health), with higher scores indicating higher health utility. The EQ-VAS records the patient's self-rated overall health on a vertical visual analogue scale. Consists of 1 item, range 0-100 from "Worst Possible" to "Best Possible" health, higher scores represent better health.
Overall Survival (OS)
Definition: Time from diagnosis of unresectable or metastatic disease to death from any cause. Censoring: Patients alive at analysis → censored at last known alive date
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The Netherlands Cancer Institute
Lead sponsor
European Organisation for Research and Treatment of Cancer - EORTC
Collaborator
