Long-term survivorship challenges of advanced/metastatic GIST patients responding to Imatinib treatment: An observational study

Gastrointestinal stromal tumours (GISTs) represent a rare family of diseases arising anywhere along the gastrointestinal tract. The mainstay of therapy for patients with localized GIST is surgical resection, which is curative in about half of patients. The development of targeted agents known as tyrosine kinase inhibitors (TKIs) has significantly improved the prognosis of non-operable advanced/metastatic GIST patients. Today, 10-15 % of TKI treated patients with advanced or metastatic GIST are still responding after 10 years of treatment. Because of the extended survival and the increasing group of patients with GIST who live with an advanced stage of a rare cancer with a more or less chronic perspective, it becomes more relevant to assess treatment effectiveness both in terms of objective outcomes (e.g. survival) and in terms of subjective patient reported outcomes including health-related quality of life (HRQOL) to determine the net clinical benefit of GIST treatment. Studies specifically investigating the experiences of the long-term survivors with metastatic GIST who are using TKI for at least 5 years are lacking.

The study aims at setting up a prospective database of patients who have been on TKI treatment for at least 5 years and documenting the follow-up treatment. This registry will provide a unique opportunity to collect information to describe the HRQOL survivorship challenges these patients are facing.

This study consist of 3 parts.
Part 1. Develop an item list with HRQOL issues of relevance for GIST patients with advanced/metastatic disease on TKI.
A search of the literature was performed to identify all relevant HRQOL issues for GIST patients. Interviews were conducted with GIST patients on long term (>5 years) TKI treatment (N=15) and health care professionals (HCPs) (N=10).

Part 2. Retrospective observational cohort – Test the item list and applicability of the EORTC QLQ-C30 among GIST patients with metastatic disease on TKI. Patients with advanced or metastasized GIST who have been treated with TKI for five years or longer will be included in the study.

Part 3. Prospective observational cohort (2-year recruitment) – Determine the prevalence of HRQOL problems, risk factors for HRQOL problems and the course of HRQOL problems over time among GIST patients with metastatic disease on TKI. Patients with advanced or metastasized GIST who have been treated with first and second line TKI for at least 2 years will be included in the study.

Data collection will be done online via Castor
Part 2: once after registration.
Part 3: every year, and in case of disease progression and/or treatment switch. Follow-up: 10 years

• Literature review to identify all relevant HRQOL issues for GIST patients completed.
• Interviews with GIST patients on long term (>5 years) TKI treatment (N=15) and health care professionals (HCPs) (N=10) completed.
• The item list is finalized and ready to be applied to part 2 and 3 of the study, which are being set up in Castor.
• Obtained ethical approval for part 2 and 3 of the study.
• Findings from the literature review and the qualitative interviews with GIST patients and health care professionals have been published.

October 2024: setting up and starting recruitment of patients for phase 2

November 2024 – November 2025: recruitment of patients for phase 3 and analyses for phase 2

This study specifically investigates the experiences of the long-term survivors with advanced or metastatic GIST who are using tyrosine kinase inhibitors (TKI) for a long duration.