OBJECTIVES: This systematic literature review aimed to identify treatment recommendations based on clinical practice guidelines (CPGs) and to understand real-world patterns of care for metastatic gastrointestinal stromal tumours (mGISTs).

METHODS: We searched Embase® and MEDLINE® to identify relevant post-2012 English language publications providing CPGs and real-world treatment patterns in patients with mGISTs.

RESULTS: Of the 1032 records screened, 24 studies met the inclusion criteria (treatment patterns: 18 studies and CPGs: 6 studies). Two guidelines were identified for Europe, two for Spain and one each for Germany and France. To treat mGISTs all CPGs have a standard recommendation of imatinib as first-line treatment followed by sunitinib as second-line and regorafenib as third line. European Society for Medical Oncology guidelines prefer avapritinib at first line for patients with imatinib-non-sensitive mutation. In the first-line setting, guidelines recommend continuing imatinib indefinitely until disease progression or intolerance. This avoids rapid tumour progression and highlights the importance of patient compliance to therapy. Real-world treatment patterns also show that imatinib is the first-line treatment of choice (86.1–99.2%). However, for further lines of therapy there is some discord against treatment guidelines with sunitinib (administered in 17.6% [France]–100% [Netherlands] patients) and regorafenib (10.5%–76% patients) not always being physicians’ second- and third-line treatment of choice. Strikingly, imatinib was also given as second-line choice (France 100%; Italy 26.8%) as well as third- to sixth-line choice in 9%–53% of patients. Other therapeutic options for subsequent lines of therapy included avapritinib, cabozantinib and nilotinib.

CONCLUSIONS: European CPGs recommend imatinib, sunitinib and regorafenib as first- second- and third-line treatment options for mGISTs. There is concordance between both real-word treatment patterns and CPGs’ recommendations for first-line treatment (i.e. imatinib). However, in real-world settings recommendations for subsequent lines are not always adhered to. Further research is needed to address these differences.