title: High molecular response rate of polycythemia vera patients treated with pegylated interferon alpha-2a.
abstract: V617F JAK2 mutation is a reliable molecular marker of polycythemia vera (PV), potentially useful to monitor the effect of treatments in this disease. In a phase 2 study of pegylated (peg) IFN-alpha-2a in PV, we performed prospective sequential quantitative evaluation of the percentage of mutated JAK2 allele (%V617F) by real-time polymerase chain reaction (PCR). The %V617F decreased in 24 (89%) of 27 treated patients, from a mean of 49% to a mean of 27% (mean decrease of 44%; P < .001), and no evidence for a plateau was observed. In one patient, mutant JAK2 was no longer detectable after 12 months. In 3 patients homozygous for the mutation, reappearance of 50% of wild-type allele was observed during treatment. The results seem to confirm the hypothesis that IFN-alpha preferentially targets the malignant clone in PV and show that %V617F assessment using a quantitative method may provide the first tool to monitor minimal residual disease in PV. This trial was registered at www.clinicaltrials.gov as #NCT00241241.
authors.affiliation: AP-HP, Hôpital Avicenne and Paris 13 University, Service d'Hématologie Clinique, 125 rue de Stalingrad, 93000 Bobigny, France.
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.forename: Bruno
authors.initials: B
authors.lastname: Cassinat
authors.forename: Pascal
authors.initials: P
authors.lastname: Turlure
authors.forename: Nathalie
authors.initials: N
authors.lastname: Cambier
authors.forename: Murielle
authors.initials: M
authors.lastname: Roussel
authors.forename: Sylvia
authors.initials: S
authors.lastname: Bellucci
authors.forename: Marie-Laurence
authors.initials: ML
authors.lastname: Menot
authors.forename: Gerald
authors.initials: G
authors.lastname: Massonnet
authors.forename: Jean-Luc
authors.initials: JL
authors.lastname: Dutel
authors.forename: Kamel
authors.initials: K
authors.lastname: Ghomari
authors.forename: Philippe
authors.initials: P
authors.lastname: Rousselot
authors.forename: Marie-Jose
authors.initials: MJ
authors.lastname: Grange
authors.forename: Yasmina
authors.initials: Y
authors.lastname: Chait
authors.forename: William
authors.initials: W
authors.lastname: Vainchenker
authors.forename: Nathalie
authors.initials: N
authors.lastname: Parquet
authors.forename: Lina
authors.initials: L
authors.lastname: Abdelkader-Aljassem
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Bernard
authors.forename: Jean-Didier
authors.initials: JD
authors.lastname: Rain
authors.forename: Sylvie
authors.initials: S
authors.lastname: Chevret
authors.forename: Christine
authors.initials: C
authors.lastname: Chomienne
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D005819:Genetic Markers; D006801:Humans; D000077190:Interferon alpha-2; D016898:Interferon-alpha / Q000627:therapeutic use*; D053614:Janus Kinase 2; D008297:Male; D008875:Middle Aged; D017354:Point Mutation; D011087:Polycythemia Vera / Q000188:drug therapy* / Q000235:genetics*; D011092:Polyethylene Glycols / Q000627:therapeutic use*; D011505:Protein-Tyrosine Kinases / Q000235:genetics*; D011518:Proto-Oncogene Proteins / Q000235:genetics*; D011994:Recombinant Proteins; D013997:Time Factors
chemical_list: D005819:Genetic Markers; D000077190:Interferon alpha-2; D016898:Interferon-alpha; D011518:Proto-Oncogene Proteins; D011994:Recombinant Proteins; D011092:Polyethylene Glycols; D011505:Protein-Tyrosine Kinases; C507924:JAK2 protein, human; D053614:Janus Kinase 2; C100416:peginterferon alfa-2a