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.
First, a search of the literature will be performed to identify all relevant HRQoL issues for GIST patients. Second, interviews will be conducted with GIST patients on long term (>5 years) TKI treatment (N=15) and health care professionals (HCPs) (N=10). This complete list of HRQoL issues will be consolidated into a comprehensive list of issues. Thereafter, this new list of HRQoL issues will be presented to GIST patients (N=40). Patients will be asked to rate the HRQoL issues on relevance and to indicate relevant missing issues. Data will be analysed according to the EORTC module development guidelines to come to a final list of issues of relevance for GIST patients.
Work in progress
Part 2. Retrospective observational cohort – Pilot test the item list and applicability of the EORTC QLQ-C30 among GIST patients with metastatic disease on TKI.
Inclusion criteria: patients with metastasized GIST who have been treated with TKI for ten years or longer.
– Clinical data (sociodemographics, tumour characteristics, treatment characteristics, medical check-ups and consultations, important adverse events, response, relapse, survival, second tumours).
– Tumour tissue (through SPECTA): KIT, PDGFR, BRAF, KRAS mutation, SDH analysis for translational research.
– The Item List created in part 1, together with the EORTC QLQ-C30.
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.
Inclusion criteria: patients with metastasized GIST who respond to TKI. Two groups will be formed: (1) long-term responders (> 5 years); (2) short-term responders.
Data collection: patients will be asked to complete the EORTC QLQ-C30 (if feasible based on results part 2 study) and final Item List created in part 2 every year via the CHES system.
Conducted the literature search, currently reading the full text and collecting data. Started the patient interviews.
September 2021: finish part 1, ethics part 2.
October 2021 – January 2022: recruitment part 2 and ethics part 3.
February 2022 – January 2024: recruitment part 3 and final analyses.
This study specifically investigates the experiences of the long-term survivors with advanced or metastatic GIST who are using tyrosine kinase inhibitors (TKI) for at least 5 years.