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 E 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 ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU 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
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
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 ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU 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
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
Julien Rohmer
- Function : Author
- PersonId : 806373
- ORCID : 0000-0002-3128-4187
Vincent Cottin
- Function : Author
- PersonId : 784269
- ORCID : 0000-0002-5591-0955
- IdRef : 166774731
Eric Delabesse
- Function : Author
- PersonId : 769021
- ORCID : 0000-0002-0928-0753
Pascale Flandrin
- Function : Author
- PersonId : 778880
- ORCID : 0000-0001-6258-7028
Lionel Galicier
- Function : Author
- PersonId : 762207
- ORCID : 0000-0002-0360-7620
Catherine Godon
- Function : Author
- PersonId : 785868
- ORCID : 0000-0002-0423-0546
Mathilde Hunault
- Function : Author
- PersonId : 752203
- IdHAL : mathilde-hunault-berger
- ORCID : 0000-0001-7777-5216
Ludovic Lhermitte
- Function : Author
- PersonId : 771345
- ORCID : 0000-0003-2498-0376
Laurent Mauvieux
- Function : Author
- PersonId : 760697
- ORCID : 0000-0002-9043-1845
- IdRef : 067407463
Philippe Rousselot
- Function : Author
- PersonId : 759734
- ORCID : 0000-0001-6798-7805
Claude Preudhomme
- Function : Author
- PersonId : 762197
- ORCID : 0000-0002-1267-9546
- IdRef : 07342255X
Matthieu Groh
- Function : Author
- PersonId : 774634
- ORCID : 0000-0003-1039-4835
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.