1. Using futibatinib to dainty patients with FGFR mutated intrahepatic cholangiocarcinoma resulted successful a 42% nonsubjective effect complaint (partial oregon complete), with nary differences successful the effectiveness of attraction crossed assorted FGFR2 fusions oregon mutations.
2. Most communal people 3 oregon higher treatment-related adverse events included hyperphosphatemia (30%), elevated AST levels (7%), stomatitis (6%), and fatigue (6%).
Evidence Rating Level: 2 (Good)
Study Rundown: Fibroblast maturation origin receptor 2 (FGFR2) fusions/rearrangements tin hap successful up to 14% of patients with intrahepatic cholangiocarcinoma. This insubstantial investigates futibatinib, an FGFR1-4 inhibitor which irreversibly binds to the FGFR kinase, for patients with confirmed FGFR mutated intrahepatic cholangiocarcinoma who failed a erstwhile enactment of systemic therapy successful the precocious signifier setting. The superior endpoint was nonsubjective effect complaint (ORR) and secondary endpoints included duration of effect (DoR), illness control, progression-free endurance (PFS), wide endurance (OS), safety, and prime of life. This survey recovered that ORR was 42%, median DoR was 9.7 months, and 83% of patients had immoderate illness control. The median PFS was 9.0 months and the median OS was 21.7 months. There were nary differences successful the effectiveness of attraction crossed assorted FGFR2 fusions oregon mutations. The astir communal people 3 oregon higher treatment-related adverse events included hyperphosphatemia (30%), elevated AST levels (7%), stomatitis (6%), and fatigue (6%). Quality of beingness was preserved passim the study. The strengths of this survey included the magnitude of follow-up clip arsenic good arsenic its elaborate genomic profiling analysis. The limitations of this survey included the fig of patients and the single-arm methodology. Overall, this insubstantial recovered a measurable payment successful utilizing futibatinib to dainty patients with FGFR mutated intrahepatic cholangiocarcinoma successful this pre-treated population, and further probe is warranted.
Click to work the survey successful NEJM
Relevant Reading: Phase I, first-in-human survey of futibatinib, a highly selective, irreversible FGFR1–4 inhibitor successful patients with precocious coagulated tumors
In-Depth [prospective cohort]: This open-label, single-group, multinational signifier 2 survey recruited 103 patients who had unresectable oregon metastatic intrahepatic cholangiocarcinoma with an FGFR2 fusion-positive oregon rearrangement who failed astatine slightest 1 enactment of systemic therapy. The median follow-up was 17.1 months and the median duration of attraction was 9.1 months. The ORR was 42% (95%CI, 32 to 52), with 72% of those having a effect that lasted astatine slightest 6 months, and 14% having responses lasting astatine slightest 12 months. The median DoR was 9.7 months (95%CI 7.6 to 17.0) and 83% of patients (95%CI, 74 to 89) had illness control. For those patients with a response, the median clip to respond was 2.5 months. The median PFS was 9.0 months (95%CI, 6.9 to 13.1) and the median OS was 21.7 months (95%CI, 14.5 to not reached). On an extended follow-up, 8 months station the archetypal analysis, effect and endurance rates were recovered to beryllium similar. The survey recovered that the effectiveness of attraction successful patients did not beryllium connected the beingness of circumstantial FGFR2 fusions oregon mutations. With regards to safety, the astir communal people 3 oregon higher treatment-related adverse lawsuit included hyperphosphatemia (30%), elevated AST levels (7%), stomatitis (6%), and fatigue (6%). These and different adverse events led to dose interruptions successful 50% of patients and dose reductions successful 54% of patients. Patient-reported outcomes were evaluated done EORTC QLQ-C30 and EQ-5D and passim attraction scores remained unchangeable oregon improved successful astir patients. Overall, this survey recovered immoderate measurable benefits successful utilizing futibatinib to dainty patients with FGFR mutated intrahepatic cholangiocarcinoma.
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