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Epidemiology,clinical picture and long-term outcomes of FIP1L1-PDGFRA-positive myeloid neoplasm with eosinophilia: Data from 151 patients |
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| Authors: | Julien Rohmer Amélie Couteau-Chardon Julie Trichereau Kewin Panel Cyrielle Gesquiere Raouf Ben Abdelali Audrey Bidet Jean-Sébastien Bladé Jean-Michel Cayuela Pascale Cony-Makhoul Vincent Cottin Eric Delabesse Mikaël Ebbo Olivier Fain Pascale Flandrin Lionel Galicier Catherine Godon Nathalie Grardel Aurélien Guffroy Mohamed Hamidou Mathilde Hunault Etienne Lengline Faustine Lhomme Ludovic Lhermitte Irène Machelart Laurent Mauvieux Catherine Mohr Marie-Joelle Mozicconacci Dina Naguib Franck E. Nicolini Jerome Rey Philippe Rousselot Suzanne Tavitian Louis Terriou Guillaume Lefèvre Claude Preudhomme Jean-Emmanuel Kahn Matthieu Groh CEREO GBMHM collaborators |
| Affiliation: | 1. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France;2. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Department of Intensive Care medicine, Centre Hospitalier Annecy Genevois, Saint-Julien-en-Genevois, France;3. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Clinical Research Department, Hôpital Foch, Suresnes, France;4. Pole Hématologie et Oncologie, Laboratoire CERBA, Saint-Ouen-l'Aumône, France;5. Laboratory of Hematology, CHU de Bordeaux, Pessac, France;6. Department of Oncology, Sainte-Anne Military Teaching Hospital, Toulon, France;7. Laboratory of Hematology, Saint-Louis Hospital, University of Paris, Paris, France;8. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Hematology Department, CH Annecy Genevois, Annecy, France;9. National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Lyon, France Hospices Civils de Lyon, UMR754, University Claude Bernard Lyon 1, Lyon, France;10. Laboratory of Hematology, Institut Universitaire du Cancer de Toulouse Oncopole, CHU de Toulouse, Toulouse, France;11. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Aix Marseille University, Department of Internal Medicine, Hôpital de la Timone, AP-HM, CNRS, INSERM, CIML, Marseille, France;12. Department of Internal Medicine, CHU Saint Antoine, Paris, France;13. Laboratory of Hematology, Hôpital Nord, CHU de Saint-Étienne, Saint-Étienne, France;14. Department of Clinical Immunology, Saint Louis hospital, Paris, France;15. Laboratoire de cytogénétique hématologique, CHU de Nantes, Nantes, France;16. Laboratory of Hematology, CHU de Lille, Lille, France;17. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Department of Clinical Immunology and Internal Medicine, National Reference Center for Systemic Autoimmune Diseases (RESO), Tertiary Center for Primary Immunodeficiency, Strasbourg University Hospital, Strasbourg, France;18. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Department of Internal Medicine, CHU de Nantes, Nantes, France;19. CRCINA CHU d'Angers, Angers, France;20. Department of Hematology, Saint Louis hospital, Paris, France;21. Department of Hematology, CHU Pontchaillou, Rennes, France;22. University of Paris, Institut National de Recherche Médicale U1151, Laboratory of Onco-Hematology, Hôpital Necker Enfants-Malades, Paris, France;23. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Department of Internal Medicine, CHU de Bordeaux, Bordeaux, France;24. Université de Strasbourg, INSERM U1113 Interface de Recherche Fondamentale et Appliquée en Cancérologie, Laboratoire d'hématologie du CHRU Strasbourg, Faculté de Médecine de Strasbourg, Strasbourg, France;25. Service d'Hématologie Oncologie, CHU, Groupe Hospitalier Sud Réunion, Saint Pierre, Reunion, France;26. Institut Paoli-Calmettes, Centre de Recherche en Cancérologie de Marseille, Biopathologie, Marseille, France;27. Laboratory of Hematology, CHU Caen, Caen, France;28. Department of Hematology, INSERM U 1052, CRCL, Centre Léon Bérard, Lyon, France;29. Department of Hematology, Institut Paoli-Calmettes, Marseille, France;30. Hematology Department, Versailles André Mignot Hospital, University Paris-Saclay, Le Chesnay, France;31. Service d'Hématologie, Institut Universitaire du Cancer de Toulouse-Oncopole, Centre Hospitalier Universitaire de Toulouse, Toulouse, France;32. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Université de Lille, CHU Lille, Département de Médecine Interne et Immunologie Clinique, Centre de Référence des Maladies Auto-immunes Systémiques Rares du Nord et Nord-Ouest de France (CeRAINO), Lille, France;33. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Université Paris-Saclay, Department of Internal Medicine, Ambroise Paré hospital, Boulogne Billancourt Cedex, France;34. National Reference Center for Hypereosinophilic syndromes (CEREO), Suresnes, France Department of Internal Medicine, Hôpital Foch, Suresnes, France |
| Abstract: | FIP1L1-PDGFRA-positive myeloid neoplasm with eosinophilia (F/P+ MN-eo) is a rare disease: robust epidemiological data are lacking and reported issues are scarce, of low sample-size and limited follow-up. Imatinib mesylate (IM) is highly efficient but no predictive factor of relapse after discontinuation has yet been identified. One hundred and fifty-one patients with F/P+ MN-eo (143 males; mean age at diagnosis 49 years; mean annual incidence: 0.18 case per million population) were included in this retrospective nationwide study involving all French laboratories who perform the search of F/P fusion gene (study period: 2003-2019). The main organs involved included the spleen (44%), skin (32%), lungs (30%), heart (19%) and central nervous system (9%). Serum vitamin B12 and tryptase levels were elevated in 74/79 (94%) and 45/57 (79%) patients, respectively, and none of the 31 patients initially treated with corticosteroids achieved complete hematologic remission. All 148 (98%) IM-treated patients achieved complete hematologic and molecular (when tested, n = 84) responses. Forty-six patients eventually discontinued IM, among whom 20 (57%) relapsed. In multivariate analysis, time to IM initiation (continuous HR: 1,01 [0.99-1,03]; P = .05) and duration of IM treatment (continuous HR: 0,97 [0,95-0,99]; P = .004) were independent factors of relapse after discontinuation of IM. After a mean follow-up of 80 (56) months, the 1, 5- and 10-year overall survival rates in IM-treated patients were 99%, 95% and 84% respectively. In F/P+ MN-eo, prompt initiation of IM and longer treatment durations may prevent relapses after discontinuation of IM. |
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