Epidemiology, clinical picture and long‐term outcomes of FIP1L1‐PDGFRA‐positive myeloid neoplasm with eosinophilia: Data from 151 patients. Issue 11 (19th September 2020)

• Record Type: Journal Article
• Authors: Rohmer, Julien; Couteau‐Chardon, Amélie; Trichereau, Julie; Panel, Kewin; Gesquiere, Cyrielle; Ben Abdelali, Raouf; Bidet, Audrey; Bladé, Jean‐Sébastien; Cayuela, Jean‐Michel; Cony‐Makhoul, Pascale; Cottin, Vincent; Delabesse, Eric; Ebbo, Mikaël; Fain, Olivier; Flandrin, Pascale; Galicier, Lionel; Godon, Catherine; Grardel, Nathalie; Guffroy, Aurélien; Hamidou, Mohamed; Hunault, Mathilde; Lengline, Etienne; Lhomme, Faustine; Lhermitte, Ludovic; Machelart, Irène; Mauvieux, Laurent; Mohr, Catherine; Mozicconacci, Marie‐Joelle; Naguib, Dina; Nicolini, Franck E.; Rey, Jerome; Rousselot, Philippe; Tavitian, Suzanne; Terriou, Louis; Lefèvre, Guillaume; Preudhomme, Claude; Kahn, Jean‐Emmanuel; Groh, Matthieu;
• Other Names: Ackermann Félix investigator.; Adiko Didier investigator.; Ahwij Nuri investigator.; Baruchel André investigator.; Beal Caroline investigator.; Bemba Maxime investigator.; Beylot Barry Marie investigator.; Beyne Rauzy Odile investigator.; Bielefeld Philip investigator.; Boisseau Mario investigator.; Bonmati Caroline investigator.; Bonnote Bernard investigator.; Borel Cécile investigator.; Bouredji Daoud investigator.; Brignier Anne investigator.; Brouillard Marc investigator.; Campos France investigator.; Carre Martin investigator.; Chalayer Emilie investigator.; Chomel Jean Claude investigator.; Coiteux Valérie investigator.; Contejean Adrien investigator.; Corby Anne investigator.; Darre Stéphane investigator.; Dubruille Viviane investigator.; Durel Cécile Audrey investigator.; El Yamani Abderrazak investigator.; Etancelin Pascaline investigator.; Etienne Nicolas investigator.; Evon Philippe investigator.; Gyan Emmanuel investigator.; Hachulla Eric investigator.; Hermet Marion investigator.; Huguet Françoise investigator.; Ianotto Jean Christophe investigator.; Inchiappa Lucas investigator.; Jdid Ibrahim investigator.; Jondeau Katayoun investigator.; Joubert Morgane investigator.; Legrand Fanny investigator.; Lejeune Caroline investigator.; Le Pendu Claire investigator.; Lidove Olivier investigator.; Lemal Richard investigator.; Limal Nicolas investigator.; Lopinet Elena investigator.; Maloisel Fréderic investigator.; Marfaing Anne investigator.; Marroun Ibrahim investigator.; Maurier François investigator.; Muller Emilie investigator.; Muron Thierry investigator.; Ojeda Mario investigator.; Paule Romain investigator.; Pignon Jean Michel investigator.; Rossi Cédric investigator.; Roumier Mathilde investigator.; Sene Damien investigator.; Sene Thomas investigator.; Simon Laurence investigator.; Slama Bohrane investigator.; Suarez Felipe investigator.; Tcherakian Colas investigator.; Torregrosa Jose Miguel investigator.; Toussaint Elise investigator.; Vatan Rémi investigator.; Visanica Sorin investigator.; Voilat Laurent investigator.; Zini Jean Marc investigator.;
• Is Part Of: American journal of hematology. Volume 95:Issue 11(2020:Nov.)
• Page Start: 1314
• Page End: 1323
• Publication Date: 2020-09-19
• Subjects: Hematology -- Periodicals; 616.15
• DOI: 10.1002/ajh.25945
• Languages: English
• ISSNs: 0361-8609

Abstract

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.