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Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease.

Holly Geyer Robyn Scherber Heidi Kosiorek Amylou C Dueck Jean-Jacques Kiladjian 1, 2 Zhijian Xiao Stefanie Slot Sonja Zweegman Federico Sackmann Ana Kerguelen Fuentes Dolores Hernández-Maraver Konstanze Döhner Claire N Harrison Deepti Radia Pablo Muxi Carlos Besses Francisco Cervantes Peter L Johansson Bjorn Andreasson Alessandro Rambaldi 3 Tiziano Barbui 4 Karin Bonatz Andreas Reiter Francoise Boyer 5, 6 Gabriel Etienne 7 Jean-Christophe Ianotto 8, 9 Dana Ranta Lydia Roy Jean-Yves Cahn 10 Norman Maldonado Giovanni Barosi Maria L Ferrari 11 Robert Peter Gale Gunnar Birgegard 12 Zefeng Xu 13 Yue Zhang Xiujuan Sun Junqing Xu 14 Peihong Zhang 15 Peter a W Te Boekhorst Suzan Commandeur Harry Schouten Heike L Pahl Martin Griesshammer Frank Stegelmann Thomas Lehmann Zhenya Senyak Alessandro M Vannucchi Francesco Passamonti Jan Samuelsson Ruben A Mesa 
Abstract : Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated the effectiveness of JAK inhibitor therapy in patients with PV patients who have a history of prior hydroxyurea (HU) use (including resistance or intolerance), phlebotomy requirements, and palpable splenomegaly. We aimed to determine how these features contribute alone and in aggregate to the PV symptom burden. Through prospective evaluation of 1,334 patients with PV who had characterized symptom burden, we assessed patient demographics, laboratory data, and the presence of splenomegaly by disease feature (ie, known HU use, known phlebotomy requirements, splenomegaly). The presence of each feature in itself is associated with a moderately high symptom burden (MPN symptom assessment form [SAF] total symptom score [TSS] range, 27.7 to 29.2) that persists independent of PV risk category. In addition, symptoms incrementally increase in severity with the addition of other features. Patients with PV who had all three features (PV-HUPS) faced the highest total score (MPN-SAF TSS, 32.5) but had similar individual symptom scores to patients with known HU use (PV-HU), known phlebotomy (PV-P), and splenomegaly (PV-S). The results of this study suggest that patients with PV who have any one of the features in question (known HU use, known phlebotomy, or splenomegaly) have significant PV-associated symptoms. Furthermore, it demonstrates that many PV symptoms remain severe independent of the number of features present.
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Submitted on : Friday, February 19, 2016 - 4:11:17 PM
Last modification on : Thursday, September 1, 2022 - 4:03:20 AM

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Holly Geyer, Robyn Scherber, Heidi Kosiorek, Amylou C Dueck, Jean-Jacques Kiladjian, et al.. Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease.. Journal of Clinical Oncology, 2016, 34 (2), pp.151-9. ⟨10.1200/JCO.2015.62.9337⟩. ⟨hal-01276589⟩



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