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Ref Type Journal Article
PMID (39138436)
Authors Park JO, Feng YH, Su WC, Oh DY, Keam B, Shen L, Kim SW, Liu X, Liao H, Qing M, Zhang C, Qian J, Tang X, Li P, Triantos S, Sweiti H
Title Erdafitinib in Asian patients with advanced solid tumors: an open-label, single-arm, phase IIa trial.
Journal Vol Issue Date Pages Journal Short Title
BMC cancer 24 1 2024 Aug 13 1006 BMC Cancer
URL
Abstract Text FGFR genomic aberrations occur in approximately 5-10% of human cancers. Erdafitinib has previously demonstrated efficacy and safety in FGFR-altered advanced solid tumors, such as gliomas, thoracic, gastrointestinal, gynecological, and other rare cancers. However, its efficacy and safety in Asian patients remain largely unknown. We conducted a multicenter, open-label, single-arm phase IIa study of erdafitinib to evaluate its efficacy in Asian patients with FGFR-altered advanced cholangiocarcinoma, non-small cell lung cancer (NSCLC), and esophageal cancer.Patients with pathologically/cytologically confirmed, advanced, or refractory tumors who met molecular and study eligibility criteria received oral erdafitinib 8 mg once daily with an option for pharmacodynamically guided up-titration to 9 mg on a 28-day cycle, except for four NSCLC patients who received erdafitinib 10 mg (7 days on/7 days off) as they were recruited before the protocol amendment. The primary endpoint was investigator-assessed objective response rate per RECIST v1.1. Secondary endpoints included progression-free survival, duration of response, disease control rate, overall survival, safety, and pharmacokinetics.Thirty-five patients (cholangiocarcinoma: 22; NSCLC: 12; esophageal cancer: 1) were enrolled. At data cutoff (November 19, 2021), the objective response rate for patients with cholangiocarcinoma was 40.9% (95% CI, 20.7-63.6); the median progression-free survival was 5.6 months (95% CI, 3.6-12.7) and median overall survival was 40.2 months (95% CI, 12.4-not estimable). No patient with RET/FGFR-altered NSCLC achieved objective response and the disease control rate was 25.0% (95% CI, 5.5-57.2%), with three patients with stable disease. The single patient with esophageal cancer achieved partial response. All patients experienced treatment-emergent adverse events, and grade ≥ 3 treatment-emergent adverse events were reported in 22 (62.9%) patients. Hyperphosphatemia was the most frequently reported treatment-emergent adverse event (all-grade, 85.7%).Erdafitinib demonstrated efficacy in a population of Asian patients in selected advanced solid tumors, particularly in those with advanced FGFR-altered cholangiocarcinoma. Treatment was tolerable with no new safety signals.This trial is registered with ClinicalTrials.gov (NCT02699606); study registration (first posted): 04/03/2016.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR2 rearrange cholangiocarcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial, Balversa (erdafitinib) treatment resulted in a confirmed objective response rate (ORR) of 40.9% (9/22, 1 complete, 8 partial responses), a median progression-free survival of 5.6 months, and median overall survival of 25.8 months in patients with cholangiocarcinoma harboring an FGFR rearrangement or FGFR short variant, with an ORR of 57.1% (8/14) with FGFR rearrangement and 12.5% (1/8) with FGFR short variant (PMID: 39138436; NCT02699606). 39138436
FGFR1 rearrange cholangiocarcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial, Balversa (erdafitinib) treatment resulted in a confirmed objective response rate (ORR) of 40.9% (9/22, 1 complete, 8 partial responses), a median progression-free survival of 5.6 months, and median overall survival of 25.8 months in patients with cholangiocarcinoma harboring an FGFR rearrangement or FGFR short variant, with an ORR of 57.1% (8/14) with FGFR rearrangement and 12.5% (1/8) with FGFR short variant (PMID: 39138436; NCT02699606). 39138436
FGFR3 rearrange cholangiocarcinoma predicted - sensitive Erdafitinib Case Reports/Case Series Actionable In a Phase II trial, Balversa (erdafitinib) treatment resulted in a confirmed objective response rate (ORR) of 40.9% (9/22, 1 complete, 8 partial responses), a median progression-free survival of 5.6 months, and median overall survival of 25.8 months in patients with cholangiocarcinoma harboring an FGFR rearrangement or FGFR short variant, with an ORR of 57.1% (8/14) with FGFR rearrangement and 12.5% (1/8) with FGFR short variant (PMID: 39138436; NCT02699606). 39138436