Long-term Survivorship Challenges of Advanced/Metastatic GIST Patients Responding to Tyrosine Kinase Inhibitor Treatment: an Observational Study

NCT07522762 | Recruiting

• 2 other identifiers: IRBd22-158004-2020
• Study Type: observational
• Target: 330
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Gastrointenstinal Stromal Tumor (GIST)

Keywords

Gastrointestinal Stromal Tumor (GIST); Tyrosine Kinase Inhibitor; Imatinib; Health-related quality of life; Metastatic disease

Outcome Measures

Primary Outcomes (1)

• 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

  Baseline and at all scheduled follow-up assessments (every year for 10 years)

Secondary Outcomes (13)

• 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)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• Patients receiving TKI in an adjuvant treatment setting.
• Patients who are too ill (death is imminent), as determined by the referring health care professional.

Sponsors & Collaborators

• The Netherlands Cancer Institute: lead
• European Organisation for Research and Treatment of Cancer - EORTC: collaborator

Study Sites (1)

Antoni van Leeuwenhoek

Amsterdam, North Holland, 1066 CX, Netherlands

RECRUITING

MeSH Terms

Conditions

Gastrointestinal Stromal Tumors; Neoplasm Metastasis

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Winette V van der Graaf, Prof. dr.

  Antoni van Leeuwenhoek

  PRINCIPAL INVESTIGATOR

• Olga Husson, Dr., Associate professor

  Antoni van Leeuwenhoek

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tessa van Amerongen, MSc, Drs.

CONTACT

+31 020 512 9111; t.v.amerongen@nki.nl

Olga Husson, Dr., Associate professor

CONTACT

+31 020 512 9111; o.husson@nki.nl

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2026
• First Posted: April 13, 2026
• Study Start: May 21, 2025
• Primary Completion (Estimated): May 21, 2027
• Study Completion (Estimated): May 21, 2037
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

NL (1)