Epidemiology, clinical picture and long‐term outcomes of FIP1L1‐PDGFRA ‐positive myeloid neoplasm with eosinophilia: Data from 151 patients

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 Nicolini; Jérôme Rey; Philippe Rousselot; Suzanne Tavitian; Louis Terriou; Guillaume Lefèvre; Claude Preudhomme; Jean‐emmanuel Kahn; Matthieu Groh

doi:10.1002/ajh.25945

Journal articles

Epidemiology, clinical picture and long‐term outcomes of FIP1L1‐PDGFRA ‐positive myeloid neoplasm with eosinophilia: Data from 151 patients

Julien Rohmer ^{1, 2} Amélie Couteau‐chardon ^{1, 3} Julie Trichereau ^{1, 2} Kewin Panel ^{1, 2} Cyrielle Gesquiere ¹ Raouf Ben Abdelali ⁴ Audrey Bidet ⁵ Jean‐sébastien Bladé ⁶ Jean‐michel Cayuela ⁷ Pascale Cony‐makhoul ^{1, 8} Vincent Cottin ^{9, 10} Eric Delabesse ¹¹ Mikaël Ebbo ^{1, 12} Olivier Fain ¹³ Pascale Flandrin ¹⁴ Lionel Galicier ¹³ Catherine Godon ¹⁵ Nathalie Grardel ¹⁶ Aurélien Guffroy ^{1, 17} Mohamed Hamidou ^{1, 15} Mathilde Hunault ¹⁸ Etienne Lengline ⁷ Faustine Lhomme ¹⁹ Ludovic Lhermitte ²⁰ Irène Machelart ^{1, 5} Laurent Mauvieux ^{21, 22} Catherine Mohr ²³ Marie‐joelle Mozicconacci ²⁴ Dina Naguib ²⁵ Franck Nicolini ²⁶ Jérôme Rey ²⁴ Philippe Rousselot ²⁷ Suzanne Tavitian ¹¹ Louis Terriou ^{1, 28} Guillaume Lefèvre ^{1, 28} Claude Preudhomme ¹⁶ Jean‐emmanuel Kahn ¹ Matthieu Groh ^{29, 30}

1 CEREO - Centre de Référence National des Syndromes Hyperéosinophiliques

2 Hôpital Foch [Suresnes]

3 Centre Hospitalier Annecy-Genevois [Saint-Julien-en-Genevois]

4 Laboratoire CERBA [Saint Ouen l'Aumône]

5 CHU Bordeaux [Bordeaux]

6 Saint Anne Military Teaching Hospital [Toulon, France]

7 AP-HP - Hopital Saint-Louis [AP-HP]

8 CH Annecy Genevois

9 National Coordinating Reference Center for Rare Pulmonary Diseases

10 IVPC - Infections Virales et Pathologie Comparée - UMR 754

11 IUCT Oncopole - UMR 1037 - Institut Universitaire du Cancer de Toulouse - Oncopole

12 CIML - Centre d'Immunologie de Marseille - Luminy

13 CHU Saint-Antoine [AP-HP]

14 CHU de Saint-Etienne - Centre Hospitalier Universitaire de Saint-Etienne

15 CHU Nantes - Centre hospitalier universitaire de Nantes

16 CHU Lille

17 RESO - Centre National de Référence pour les Maladies Auto-immunes Rares [CHU Strasbourg]

18 CRCINA-ÉQUIPE 7 - Innate Immunity and Immunotherapy

CRCINA - Centre de Recherche en Cancérologie et Immunologie Nantes-Angers

19 CHU Pontchaillou [Rennes]

20 INEM - UM 111 (UMR 8253 / U1151) - Institut Necker Enfants-Malades

21 IRFAC - Inserm U1113 - Interface de Recherche Fondamentale et Appliquée en Cancérologie

22 CHU Strasbourg

23 CHU Sud Saint Pierre [Ile de la Réunion]

24 Institut Paoli-Calmettes

25 CHU Caen

26 UNICANCER/CRCL - Centre de Recherche en Cancérologie de Lyon

27 CHV - Centre Hospitalier de Versailles André Mignot

28 CeRAINO - Centre National de Référence des Maladies Auto-Immunes Systémiques Rares du Nord et Nord-Ouest de France

29 Hôpital Ambroise Paré [AP-HP]

30 Université Paris-Saclay

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.

Document type :

Journal articles

Domain :

Life Sciences [q-bio] / Cancer

Complete list of metadata

https://www.hal.inserm.fr/inserm-03496695
Contributor : Elizabeth Bernardo Connect in order to contact the contributor
Submitted on : Monday, December 20, 2021 - 2:54:36 PM
Last modification on : Tuesday, January 4, 2022 - 5:58:17 AM

Epidemiology, clinical picture and long‐term outcomes of FIP1L1‐PDGFRA ‐positive myeloid neoplasm with eosinophilia: Data from 151 patients

Identifiers

Collections

Citation

Export

Metrics

Epidemiology, clinical picture and long‐term outcomes of FIP1L1‐PDGFRA ‐positive myeloid neoplasm with eosinophilia: Data from 151 patients

Identifiers

Collections

Citation

Export

Share

Metrics