High molecular response rate of polycythemia vera patients treated with pegylated interferon alpha-2a

Jean-Jacques Kiladjian, Bruno Cassinat, Pascal Turlure, Nathalie Cambier, Murielle Roussel, Sylvia Bellucci, Marie-Laurence Menot, Gerald Massonnet, Jean-Luc Dutel, Kamel Ghomari, Philippe Rousselot, Marie-Jose Grange, Yasmina Chait, William Vainchenker, Nathalie Parquet, Lina Abdelkader-Aljassem, Jean-François Bernard, Jean-Didier Rain, Sylvie Chevret, Christine Chomienne, Pierre Fenaux
Blood 2006 September 15, 108 (6): 2037-40
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 as #NCT00241241.

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