FGFR2 fusion

P1, N=160, Recruiting, SystImmune Inc. | N=120 --> 160

Malignant effusion CBs show favourable intra-patient biomarker consistency over time, supporting their feasibility for longitudinal monitoring. Effusion-based testing shows potential value for potentially identifying candidates for FGFR2b-targeted therapies, pending further clinical validation.

Existing literature primarily identifies such tumors in the distal extremities and temporomandibular joint; however, the case reported herein is the first documented occurrence in the perineum of a female. This finding suggests that the spectrum of CCMN may extend beyond the traditionally recognized locations of the distal extremities and temporomandibular joint.

The integration of NGS and the subsequent use of matched targeted therapy significantly improved survival outcomes in selected patients with advanced BTC, highlighting the importance of precision medicine strategies.

In contrast to the pheochromocytoma, the BDA showed no evidence of a second TP53 alteration that might suggest that TP53 had played a role in its pathogenesis. This case highlights the rare presentation of pheochromocytoma in LFS and provides a molecular hypothesis of how this tumor may have developed.

Only one patient died 16 months after surgery due to an unrelated traffic accident.

Pathological subtypes of CCA exhibit distinct clinical outcomes and molecular characteristics. Classification based on pathological subtype provides a useful framework for understanding the clinical and molecular heterogeneity of CCA.

Early termination limited the ability to draw definitive conclusions on the efficacy of infigratinib as first-line treatment of FGFR2-rearranged CCA. This study illustrates the challenges of powering confirmatory studies in biomarker-selected subpopulations of rare tumors and highlights the need for regulatory collaboration to develop pragmatic frameworks for assessing novel therapies in ultra-rare malignancies.

Parallel translational work using cfDNA-based liquid biopsy has mapped a spectrum of secondary kinase-domain mutations that underlie acquired resistance, informing the development of next-generation FGFR2-selective inhibitors (eg, lirafugratinib) and combination strategies with EGFR/ERBB blockade. Collectively, these data underscore the need for comprehensive molecular profiling and innovative umbrella trial designs to optimise targeted therapy in this rare, biologically heterogeneous malignancy.

It covers their mechanisms in driving cancer, their potential as biomarkers to predict treatment response, and the emerging challenges and opportunities for FGFR-targeted therapy. Ultimately, a deeper understanding of FGFR rearrangements is critical for advancing precision oncology and improving patient benefit.

Retrospective data indicate that gemcitabine plus platinum-based chemotherapy achieves the most consistent efficacy among conventional regimens, with median overall survival (OS) of 10-16 months...For clinical settings where comprehensive genomic profiling is not feasible, we discuss a pragmatic surrogate-based approach using imaging characteristics and serum tumor markers to guide initial treatment selection. We also address post-progression treatment considerations, including phenotype-based regimen switching and the role of re-biopsy.

P2, N=63, Completed, Eisai Co., Ltd. | Active, not recruiting --> Completed | Trial completion date: Mar 2026 --> Nov 2025 | Trial primary completion date: Mar 2026 --> Nov 2025