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Cited by 365

The DOI matches its Crossref record; 34 of the 34 references it cites were found in a registry or an index.

Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma

Open access
Mazzaferro, Vincenzo, El-Rayes, Bassel F., Droz dit Busset, Michele, Cotsoglou, Christian, Harris, William P., Damjanov, Nevena, Masi, Gianluca, Rimassa, Lorenza, Personeni, Nicola, Braiteh, Fadi, Zagonel, Vittorina, Papadopoulos, Kyriakos P., Hall, Terence, Wang, Yunxia, Schwartz, Brian, Kazakin, Julia, Bhoori, Sherrie, de Braud, Filippo, Shaib, Walid L.·2019·British Journal of Cancer
DOI 10.1038/s41416-018-0334-0
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Abstract

BACKGROUND: Next-generation sequencing has identified actionable genetic aberrations in intrahepatic cholangiocarcinomas (iCCA), including the fibroblast growth factor receptor 2 (FGFR2) fusions. Derazantinib (ARQ 087), an orally bioavailable, multi-kinase inhibitor with potent pan-FGFR activity, has shown preliminary therapeutic activity against FGFR2 fusion-positive iCCA. METHODS: This multicentre, phase 1/2, open-label study enrolled adult patients with unresectable iCCA with FGFR2 fusion, who progressed, were intolerant or not eligible to first-line chemotherapy (NCT01752920). Subjects received derazantinib in continuous daily doses. Tumour response was assessed according to RECIST 1.1 every 8 weeks. RESULTS: Twenty-nine patients (18 women/11 men; median age, 58.7 years), 2 treatment-naive and 27 who progressed after at least one prior systemic therapy, were enrolled. Overall response rate was 20.7%, disease control rate was 82.8%. Estimated median progression-free survival was 5.7 months (95% CI: 4.04-9.2 months). Treatment-related adverse events (AE) were observed in 27 patients (93.1%, all grades), including asthenia/fatigue (69.0%), eye toxicity (41.4%), and hyperphosphatemia (75.9%). Grade ≥ 3 AEs occurred in 8 patients (27.6%). CONCLUSION: Derazantinib demonstrated encouraging anti-tumour activity and a manageable safety profile in patients with advanced, unresectable iCCA with FGFR2 fusion who progressed after chemotherapy. A pivotal trial of derazantinib in iCCA is ongoing (NCT03230318).

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Metadata

Authors
Mazzaferro, V., El-Rayes, B. F., Droz dit Busset, M., Cotsoglou, C., Harris, W. P., Damjanov, N., Masi, G., Rimassa, L., Personeni, N., Braiteh, F., Zagonel, V., Papadopoulos, K. P., Hall, T., Wang, Y., Schwartz, B., Kazakin, J., Bhoori, S., de Braud, F., & Shaib, W. L.
Venue
British Journal of Cancer
Publisher
Springer Science and Business Media LLC
Published
2018-11-13
Volume
120
Issue
2
Pages
165-171
ISSN
  • 0007-0920 (ISSN-L)
  • 1532-1827
DOI
10.1038/s41416-018-0334-0
OpenAlex
W2899924640
Cited by
365
References
34
FWCI
35.11
Type
journal article
Language
en
Open access
hybrid

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CoChat. (2026). Citation check: Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. https://app.cochat.ai/papers/10.1038/s41416-018-0334-0
The paper
Mazzaferro, V., El-Rayes, B. F., Droz dit Busset, M., Cotsoglou, C., Harris, W. P., Damjanov, N., Masi, G., Rimassa, L., Personeni, N., Braiteh, F., Zagonel, V., Papadopoulos, K. P., Hall, T., Wang, Y., Schwartz, B., Kazakin, J., Bhoori, S., de Braud, F., & Shaib, W. L. (2019). Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. British Journal of Cancer. https://doi.org/10.1038/s41416-018-0334-0

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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.

Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.