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