High molecular response rate of polycythemia vera patients treated with pegylated interferon alpha-2a.
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.
Kiladjian
Jean-Jacques
JJ
AP-HP, Hôpital Avicenne and Paris 13 University, Service d'Hématologie Clinique, 125 rue de Stalingrad, 93000 Bobigny, France.
Cassinat
Bruno
B
Turlure
Pascal
P
Cambier
Nathalie
N
Roussel
Murielle
M
Bellucci
Sylvia
S
Menot
Marie-Laurence
ML
Massonnet
Gerald
G
Dutel
Jean-Luc
JL
Ghomari
Kamel
K
Rousselot
Philippe
P
Grange
Marie-Jose
MJ
Chait
Yasmina
Y
Vainchenker
William
W
Parquet
Nathalie
N
Abdelkader-Aljassem
Lina
L
Bernard
Jean-François
JF
Rain
Jean-Didier
JD
Chevret
Sylvie
S
Chomienne
Christine
C
Fenaux
Pierre
P
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
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