PhD defence D. (Deborah) van de Wal

Below is a brief summary of the dissertation: 

This dissertation discusses the results of several studies performed in patients with gastrointestinal stromal tumor (GIST). Every year, around 400 people in the Netherlands are diagnosed with GIST. One in five GIST patients already has metastases at diagnosis, and in a third of the patients the GIST recurs after surgery or metastases develop over time.

Before the introduction of tyrosine kinase inhibitors (TKIs), the median overall survival of patients with metastatic GIST was poor, but imatinib has been the game changer as its introduction led to an impressive median overall survival benefit of over five years. Since that time new TKIs, including sunitinib, regorafenib, ripretinib and avapritinib have been approved.

All these developments make it important to evaluate treatments not only through objective clinical outcomes, such as survival, but also from the patient's perspective by including patient-reported outcomes. Patient-reported outcomes can be collected through questionnaires, and most often encompass symptoms, side effects, functioning, or other domains of health-related quality of life (HRQoL). HRQoL is a multidimensional concept that includes the patient’s perception of the impact of the disease and its treatment on physical, psychological, and social functioning.

In the various studies included in this dissertation, we provided insight into (rare) side effects of TKIs and the HRQoL of patients with or after GIST, the challenges they may experience physically, psychologically and socially, and how these challenges may affect their HRQoL.