Epidemiology, clinical picture and long-term outcomes of FIP1L1-PDGFRA-positive myeloid neoplasm with eosinophilia: Data from 151 patients.
Adrenal Cortex Hormones
/ administration & dosage
Adult
Disease-Free Survival
Eosinophilia
/ blood
Female
France
/ epidemiology
Hematologic Neoplasms
/ blood
Humans
Incidence
Male
Middle Aged
Myeloproliferative Disorders
/ blood
Oncogene Proteins, Fusion
/ blood
Receptor, Platelet-Derived Growth Factor alpha
/ blood
Retrospective Studies
Survival Rate
Tryptases
/ blood
Vitamin B 12
/ blood
mRNA Cleavage and Polyadenylation Factors
/ blood
Journal
American journal of hematology
ISSN: 1096-8652
Titre abrégé: Am J Hematol
Pays: United States
ID NLM: 7610369
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
19
07
2020
accepted:
23
07
2020
pubmed:
29
7
2020
medline:
30
12
2020
entrez:
29
7
2020
Statut:
ppublish
Résumé
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.
Substances chimiques
Adrenal Cortex Hormones
0
Oncogene Proteins, Fusion
0
mRNA Cleavage and Polyadenylation Factors
0
FIP1L1-PDGFRA fusion protein, human
EC 2.7.10.1
Receptor, Platelet-Derived Growth Factor alpha
EC 2.7.10.1
Tryptases
EC 3.4.21.59
Vitamin B 12
P6YC3EG204
Types de publication
Clinical Trial
Journal Article
Multicenter Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
1314-1323Investigateurs
Félix Ackermann
(F)
Didier Adiko
(D)
Nuri Ahwij
(N)
André Baruchel
(A)
Caroline Beal
(C)
Maxime Bemba
(M)
Marie Beylot Barry
(M)
Odile Beyne Rauzy
(O)
Philip Bielefeld
(P)
Mario Boisseau
(M)
Caroline Bonmati
(C)
Bernard Bonnote
(B)
Cécile Borel
(C)
Daoud Bouredji
(D)
Anne Brignier
(A)
Marc Brouillard
(M)
France Campos
(F)
Martin Carre
(M)
Emilie Chalayer
(E)
Jean Claude Chomel
(JC)
Valérie Coiteux
(V)
Adrien Contejean
(A)
Anne Corby
(A)
Stéphane Darre
(S)
Viviane Dubruille
(V)
Cécile Audrey Durel
(CA)
Abderrazak El Yamani
(A)
Pascaline Etancelin
(P)
Nicolas Etienne
(N)
Philippe Evon
(P)
Emmanuel Gyan
(E)
Eric Hachulla
(E)
Marion Hermet
(M)
Françoise Huguet
(F)
Jean Christophe Ianotto
(JC)
Lucas Inchiappa
(L)
Ibrahim Jdid
(I)
Katayoun Jondeau
(K)
Morgane Joubert
(M)
Fanny Legrand
(F)
Caroline Lejeune
(C)
Claire Le Pendu
(C)
Olivier Lidove
(O)
Richard Lemal
(R)
Nicolas Limal
(N)
Elena Lopinet
(E)
Fréderic Maloisel
(F)
Anne Marfaing
(A)
Ibrahim Marroun
(I)
François Maurier
(F)
Emilie Muller
(E)
Thierry Muron
(T)
Mario Ojeda
(M)
Romain Paule
(R)
Jean Michel Pignon
(JM)
Cédric Rossi
(C)
Mathilde Roumier
(M)
Damien Sene
(D)
Thomas Sene
(T)
Laurence Simon
(L)
Bohrane Slama
(B)
Felipe Suarez
(F)
Colas Tcherakian
(C)
Jose Miguel Torregrosa
(JM)
Elise Toussaint
(E)
Rémi Vatan
(R)
Sorin Visanica
(S)
Laurent Voilat
(L)
Jean Marc Zini
(JM)
Informations de copyright
© 2020 Wiley Periodicals LLC.
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