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 Blood, 15 September 2006, Vol. 108, No. 6, pp. 2037-2040.
Prepublished online as a Blood First Edition Paper on May 18, 2006; DOI 10.1182/blood-2006-03-009860.

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NEOPLASIA
Brief report

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, and Pierre Fenaux

From the AP-HP, Hôpital Avicenne, Service d'Hématologie Clinique, Bobigny, France; Paris 13 University, Paris, France; Assistance Publique–Hôpitaux de Paris (AP-HP), Hôpital Saint-Louis, Unité de Biologie Cellulaire, Paris, France; CHU Dupuytren, Service d'Hématologie, Limoges, France; Centre Hospitalier Universitaire (CHU) de Lille, Hopital Huriez, Service des Maladies du Sang, Lille, France; AP-HP, Hôpital Lariboisiere, Service d'Hématologie, Paris, France; CH de Beauvais, Service d'Hématologie, Beauvais, France; CH de Versailles, Service d'Hématologie, Versailles, France; AP-HP, Hôpital Bichat, Paris, France; CH de Montfermeil, Service d'Hématologie, Montfermeil, France; Institut National de la Santé et de la Recherche Médicale (INSERM) Unité 790 (U790), Institut Gustave Roussy, Villejuif, France; AP-HP, Hôpital Saint-Louis, Therapie Cellulaire, Paris, France; CH de Chartres, Service d'Hématologie, Chartres, France; and AP-HP, Hôpital Saint-Louis, Département de Biostatistique et Informatique Medicale (DBIM), Paris, France.

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 [ClinicalTrials.gov] .


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