Abstract
Regorafenib was approved as third-line therapy for advanced Gastrointestinal Stromal Tumour (GIST) at a starting dose of 160 mg daily 3 weeks on, 1 week off, based on improvement in progression free survival over placebo (4.8 vs. 0.9 months), but the response rate was low at 4.5%. Given the high toxicity rate in GIST patients, there is variability in the post-marketing dosing of regorafenib. We aimed to summarize our experience regarding prescribing patterns, efficacy and toxicity of regorafenib and determine the role of response assessment by Choi criteria in GIST patients. We included 28 patients who received regorafenib from our pharmacy. Baseline patient characteristics and treatment outcomes were recorded and an independent radiologist assessed response using Choi and RECIST. Seventy-nine percent of patients started at a 120 mg continuous daily dosing schedule, different from the standard intermittent dosing schedule. Grade 3/4 adverse events were experienced by 43% of patients. Median progression-free survival was 8.7 months. Continuous dosing with regorafenib at 120 mg daily is the preferred prescribing pattern and appears to be better tolerated and with comparable efficacy to the current standard dose. Similar to imatinib, the partial response rate for regorafenib by Choi (29%) was higher compared to RECIST (4%).
Highlights
Regorafenib was approved as third-line therapy for advanced Gastrointestinal Stromal Tumour (GIST) at a starting dose of 160 mg daily 3 weeks on, 1 week off, based on improvement in progression free survival over placebo (4.8 vs. 0.9 months), but the response rate was low at 4.5%
Regorafenib is a multikinase inhibitor with activity against KIT, RET, RAF1, BRAF, PDGFR, FGFR EGFR1–3 and TEK, approved in 2013 for the treatment of patients with locally advanced, unresectable, or metastatic GIST previously treated with imatinib and sunitinib
Regorafenib is currently approved for the treatment of patients with locally advanced, unresectable, or metastatic GIST, as third-line therapy after imatinib and sunitinib
Summary
Regorafenib was approved as third-line therapy for advanced Gastrointestinal Stromal Tumour (GIST) at a starting dose of 160 mg daily 3 weeks on, 1 week off, based on improvement in progression free survival over placebo (4.8 vs. 0.9 months), but the response rate was low at 4.5%. Regorafenib was eventually approved at this dosing schedule for GIST based on the results of the phase III study, which demonstrated a median progression free survival (PFS) of 4.8 months with regorafenib compared to 0.9 months with placebo (HR 0·27, 95% CI 0·19–0·39; p < 0·0001)[12] In this trial, 98% of patients assigned to regorafenib had drug-related adverse events (AEs) and 72% of these patients in the regorafenib group required dose modifications, even with limiting accrual to patients with Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. The most common grade 3 events were hypertension (23%), hand-foot syndrome (20%), and diarrhoea (5%)
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