Mr Myles Smith and colleagues from the Sarcoma, Melanoma and Rare Tumours Unit at The Royal Marsden, have just had a study published, titled ‘Efficacy and Safety of Ripretinib in Advanced Gastrointestinal Stromal Tumors within an Expanded Access Program: A Cohort Study’ in the publication ‘Cancers’.
Ripretinib is a tyrosine kinase inhibitor designed to target specific mutations associated with Gastrointestinal Stromal Tumors (GISTs). The researchers evaluated the safety and efficacy of Ripretinib in comparison to placebo in forty-five patients with advanced GISTs who had previously undergone treatment with imatinib, sunitinib, and regorafenib.
The study involved a comprehensive analysis of adverse events, dosage adjustments, and treatment discontinuations among the participants. Results demonstrated that Ripretinib exhibited a favourable safety profile, with manageable side effects. Common adverse events included fatigue, nausea, and abdominal pain. Notably, the incidence of severe adverse events was lower in the Ripretinib group compared to the placebo group. The study supports other phase lll clinical trials, suggesting Ripretinib can be considered a safe and well-tolerated treatment option for patients with advanced GISTs who have exhausted previous therapeutic interventions.
These findings contribute valuable insights to the evolving landscape of GIST treatment and may influence clinical decisions regarding approval for the use of Ripretinib in the UK, while also supporting the role of Expanded Access Programs as a means to gather early real-world evidence.