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

Alessandro Parisi; Blandine Delaunay; Giada Pinterpe; Antoine Hollebecque; Jean Frederic Blanc; Mohamed Bouattour; Eric Assenat; Meher Ben Abdelghani; Matthieu Sarabi; Monica Niger; Caterina Vivaldi; Mario Mandalà; Andrea Palloni; Maria Bensi; Silvio Ken Garattini; David Tougeron; Pierre Combe; Massimiliano Salati; Margherita Rimini; Chiara Alessandra Cella; Marco Tucci; Anna Diana; Elena Mori; Raffaella Longarini; Pascal Artru; Gael Roth; Ludovic Evesque; Agathe Vienne; Anthony Turpin; Sandrine Hiret; Vincent Bourgeois; Camille Herve; Rodolphe Paulon; Marion Stacoffe; David Malka; Cindy Neuzillet; Julien Edeline; Astrid Lievre; Rosine Guimbaud; Marie Christelle Pajiep Chapda; Lorenza Rimassa; Riccardo Giampieri; Juan Valle; Rossana Berardi; Nadim Fares

doi:10.1016/j.ejca.2024.113587

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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

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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

Alessandro Parisi, Blandine Delaunay, Giada Pinterpe, Antoine Hollebecque, Jean Frederic Blanc, Mohamed Bouattour, Eric Assenat, Meher Ben Abdelghani, Matthieu Sarabi, Monica Niger, …

European journal of cancer (1990), Vol.200, p.113587

03/2024

DOI: https://doi.org/10.1016/j.ejca.2024.113587

PMID: 38340384

Résumé

BTC

FGFR2 fusions/rearrangements

Intrahepatic cholangiocarcinoma

Real-world data

Systemic treatment

Targeted therapy

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. 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. 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. These results confirm the effectiveness and safety of pemigatinib in a real-world setting. •Effectiveness of pemigatinib in advanced FGFR2-positive CCA patients was investigated.•ORR was 45.8%, DCR was 84.7%, median PFS was 8.7 months, median OS was 17.1 months.•Effectiveness and safety of pemigatinib was confirmed in a real-world setting.

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Titre: 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
Créateurs - sans rôle: Alessandro Parisi - Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy
Blandine Delaunay - Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy
Giada Pinterpe - Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy
Antoine Hollebecque - Département d′Innovation Thérapeutique et Essais précoces (DITEP), Gustave Roussy, Villejuif Cedex, France
Jean Frederic Blanc - Oncology Digestive Unit, Hôpital haut-Lévêque, CHU Bordeaux France, France
Mohamed Bouattour - Liver Oncology and Therapeutic Innovation Functional Unit, Beaujon Hospital APHP, Clichy, France
Eric Assenat - Medical oncology, ICM - Institut du Cancer de Montpellier, Montpellier Cedex, France
Meher Ben Abdelghani - Institut de Cancérologie Strasbourg
Matthieu Sarabi - Centre Léon Bérard
Monica Niger - Fondazione IRCCS Istituto Nazionale dei Tumori
Caterina Vivaldi - University of Pisa
Mario Mandalà - University of Perugia
Andrea Palloni - Medical Oncology, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
Maria Bensi - Oncologia Medica, Comprehensive Cancer Center, Fondazione Policlinico Universitario Agostino Gemelli–IRCCS, Italy
Silvio Ken Garattini - Department of Oncology, Academic Hospital of Udine ASUFC, Piazzale Santa Maria della Misericordia 15, Udine, UD 33100, Italy
David Tougeron - Université de Poitiers, Department of Gastroenterology and Hepatology, Poitiers University Hospital, Poitiers, France
Pierre Combe - Medical Oncology, CORT37, Pôle Santé Léonard de Vinci, Chambray-lès-Tours, France
Massimiliano Salati - Division of Oncology, Department of Oncology and Hematology, University Hospital Modena, Modena Cancer Centre, Via del Pozzo 71, 41125 Modena, Italy
Margherita Rimini - Vita-Salute University San Raffaele, Milan, Italy
Chiara Alessandra Cella - Division of Gastrointestinal Medical Oncology and Neuroendocrine Tumors, European Institute of Oncology, IEO IRCCS, Via Ripamonti 435, Milan, Italy
Marco Tucci - Department of Interdisciplinary Medicine, Oncology Unit, University of Bari "Aldo Moro", P.za Giulio Cesare, 11, 70124, Bari, Italy
Anna Diana - UOC Oncologia - Ospedale del Mare, Naples
Elena Mori - Department of Medical Oncology, New Hospital of Prato S. Stefano, 59100 Prato, Italy
Raffaella Longarini - Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
Pascal Artru - GI Oncology Department, Hôpital privé Jean Mermoz, Lyon, France
Gael Roth - Inserm
Ludovic Evesque - Centre Antoine Lacassagne
Agathe Vienne - Oncology Department, CHU Sud Réunion, Saint Pierre, France
Anthony Turpin - Hôpital Claude Huriez
Sandrine Hiret - Oncology Department, ICO Institut de Cancerologie de l′Ouest René Gauducheau, Saint-Herblain, France
Vincent Bourgeois - Digestive Oncology, Hopital duchenne Boulogne-sur-Mer, France
Camille Herve - Digestive Oncology, Groupe Hospitalier Mutualiste, Grenoble
Rodolphe Paulon - Medical Oncology, Clinique du Sidobre, Castres, France
Marion Stacoffe - Medical Oncology, CHRU Hopitaux de Tours - Hopital Bretonneau, Tours Cedex, France
David Malka - Medical Oncology, Institut Mutualiste Montsouris, Paris, France
Cindy Neuzillet - GI Oncology, Medical Oncology Department, Curie Institute, Paris, France
Julien Edeline - Medical Oncology Department, Centre Eugene - Marquis, Rennes, France
Astrid Lievre - Centre Hospitalier Universitaire de Rennes
Rosine Guimbaud - Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France
Marie Christelle Pajiep Chapda - MeDatas, CIC (Centre d′Investigation Clinique), CHU Toulouse, Toulouse, France
Lorenza Rimassa - Humanitas University
Riccardo Giampieri - Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy
Juan Valle - Cholangiocarcinoma Foundation, Salt Lake City, Utah, USA
Rossana Berardi - Clinica Oncologica e Centro Regionale di Genetica Oncologica, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Via Conca 71, 60126 Ancona, Italy
Nadim Fares - Centre Hospitalier Universitaire de Toulouse
Détails de publication: European journal of cancer (1990), Vol.200, p.113587
Éditeur: Elsevier Ltd
Identifiants: 99139656109311
Unité académique: Institut de Génétique Moléculaire de Montpellier - IGMM
Langue: English
Type de ressource: Journal article