Pemigatinib for patients with previously treated, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements: A joint analysis of the French PEMI-BIL and Italian PEMI-REAL cohort studies.

Autor: Parisi A; Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy. Electronic address: alexparis@hotmail.it., Delaunay B; Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy; Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France., Pinterpe G; Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy., Hollebecque A; Département d'Innovation Thérapeutique et Essais précoces (DITEP), Gustave Roussy, Villejuif Cedex, France., Blanc JF; Oncology Digestive Unit, Hôpital haut-Lévêque, CHU Bordeaux France, France., Bouattour M; Liver Oncology and Therapeutic Innovation Functional Unit, Beaujon Hospital APHP, Clichy, France., Assenat E; Medical oncology, ICM - Institut du Cancer de Montpellier, Montpellier Cedex, France., Ben Abdelghani M; Oncology Department, ICANS - Institut de Cancérologie Strasbourg Europe, Strasbourg, France., Sarabi M; Medical Oncology, Centre Léon Bérard, Lyon, GI Oncology Department, France; GI Oncology Department, Hôpital privé Jean Mermoz, Lyon, France., Niger M; Medical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Via Venezian 1, 20133 Milan, Italy., Vivaldi C; Department of Translational Research and New Technologies in Medicine and Surgery, University of Pisa, 56126 Pisa, Italy., Mandalà M; Unit of Medical Oncology, University of Perugia, Perugia, Italy., Palloni A; Medical Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy., Bensi M; Oncologia Medica, Comprehensive Cancer Center, Fondazione Policlinico Universitario Agostino Gemelli-IRCCS, Italy; Università Cattolica del Sacro Cuore, Roma, Italy., Garattini SK; Department of Oncology, Academic Hospital of Udine ASUFC, Piazzale Santa Maria della Misericordia 15, Udine, UD 33100, Italy., Tougeron D; Université de Poitiers, Department of Gastroenterology and Hepatology, Poitiers University Hospital, Poitiers, France., Combe P; Medical Oncology, CORT37, Pôle Santé Léonard de Vinci, Chambray-lès-Tours, France., Salati M; Division of Oncology, Department of Oncology and Hematology, University Hospital Modena, Modena Cancer Centre, Via del Pozzo 71, 41125 Modena, Italy; Clinical and Experimental Medicine, University of Modena and Reggio Emilia, Modena, Italy., Rimini M; Vita-Salute University San Raffaele, Milan, Italy; Department of Oncology, IRCCS San Raffaele Hospital, via Olgettina N. 60, Milan 20132, Italy., Cella CA; Division of Gastrointestinal Medical Oncology and Neuroendocrine Tumors, European Institute of Oncology, IEO IRCCS, Via Ripamonti 435, Milan, Italy., Tucci M; Department of Interdisciplinary Medicine, Oncology Unit, University of Bari 'Aldo Moro', P.za Giulio Cesare, 11, 70124, Bari, Italy., Diana A; UOC Oncologia - Ospedale del Mare, Naples., Mori E; Department of Medical Oncology, New Hospital of Prato S. Stefano, 59100 Prato, Italy., Longarini R; Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy., Artru P; GI Oncology Department, Hôpital privé Jean Mermoz, Lyon, France., Roth G; Univ. Grenoble Alpes / Hepato-Gastroenterology and Digestive Oncology Department, CHU Grenoble Alpes / Institute for Advanced Biosciences, CNRS UMR 5309-INSERM, U1209, France., Evesque L; Medical Oncology Department, Centre Antoine-Lacassagne, Nice, France., Vienne A; Oncology Department, CHU Sud Réunion, Saint Pierre, France., Turpin A; Medical Oncology Department, Hopital Claude Huriez, Lille, France., Hiret S; Oncology Department, ICO Institut de Cancerologie de l'Ouest René Gauducheau, Saint-Herblain, France., Bourgeois V; Digestive Oncology, Hopital duchenne Boulogne-sur-Mer, France., Herve C; Digestive Oncology, Groupe Hospitalier Mutualiste, Grenoble., Paulon R; Medical Oncology, Clinique du Sidobre, Castres, France., Stacoffe M; Medical Oncology, CHRU Hopitaux de Tours - Hopital Bretonneau, Tours Cedex, France., Malka D; Medical Oncology, Institut Mutualiste Montsouris, Paris, France., Neuzillet C; GI Oncology, Medical Oncology Department, Curie Institute, Paris, France., Edeline J; Medical Oncology Department, Centre Eugene - Marquis, Rennes, France., Lievre A; Department of Gastroenterology, CHU de Rennes - Hopital Pontchaillou, Rennes Cedex, France., Guimbaud R; Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France., Chapda MCP; MeDatas, CIC (Centre d'Investigation Clinique), CHU Toulouse, Toulouse, France., Rimassa L; Department of Biomedical Sciences, Humanitas University, 20072 Pieve Emanuele, Milan, Italy; Medical Oncology and Hematology Unit, Humanitas Cancer Center, IRCCS Humanitas Research Hospital, 20089 Rozzano, Milan, Italy., Giampieri R; Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy., Valle J; Cholangiocarcinoma Foundation, Salt Lake City, Utah, USA; Division of Cancer Sciences, University of Manchester, Manchester, UK., Berardi R; Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy., Fares N; Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France.
Jazyk: angličtina
Zdroj: European journal of cancer (Oxford, England : 1990) [Eur J Cancer] 2024 Mar; Vol. 200, pp. 113587. Date of Electronic Publication: 2024 Feb 06.
DOI: 10.1016/j.ejca.2024.113587
Abstrakt: Background: Pemigatinib is approved for patients with pretreated, locally advanced or metastatic CCA harboring FGFR2 rearrangements or fusions. We aim to assess the effectiveness and safety of pemigatinib in real-world setting.
Material and Methods: A joint analysis of two multicentre observational retrospective cohort studies independently conducted in France and Italy was performed. All consecutive FGFR2-positive patients affected by CCA and treated with pemigatinib as second- or further line of systemic treatment in clinical practice, within or outside the European Expanded Access Program, were included.
Results: Between July 2020 and September 2022, 72 patients were treated with pemigatinib in 14 Italian and 25 French Centres. Patients had a median age of 57 years, 76% were female, 81% had ECOG-PS 0-1, 99% had intrahepatic CCA, 74% had ≥ 2 metastatic sites, 67% had metastatic disease at diagnosis, while 38.8% received ≥ 2 previous lines of systemic treatment. At data cut-off analysis (April 2023), ORR and DCR were 45.8% and 84.7%, respectively. Median DoR was 7 months (IQR: 5.8-9.3). Over a median follow-up time of 19.5 months, median PFS and 1-year PFS rate were 8.7 months and 32.8%. Median OS and 1-year OS rate were 17.1 months and 60.6%. Fatigue (69.4%), ocular toxicity (68%), nail toxicities (61.1%), dermatologic toxicity (41.6%) hyperphosphataemia (55.6%), stomatitis (48.6%), and diarrhea (36.1%) were the most frequent, mainly G1-G2 AEs. Overall incidence of G3 AEs was 22.2%, while no patient experienced G4 AE. Dose reduction and temporary discontinuation were needed in 33.3% and 40.3% of cases, with 1 permanent discontinuation due to AEs.
Conclusions: These results confirm the effectiveness and safety of pemigatinib in a real-world setting.
Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.)
Databáze: MEDLINE
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