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Title: Symptomatic profiles of 1334 polycythemia vera patients: implications of inadequately controlled disease
Author: Geyer, Holly L.
Scherber, Robyn
Kosiorek, Heidi
Dueck, Amylou C.
Kiladjian, Jean-Jacques
Xiao, Zhijian
Slot, Stefanie
Zweegman, Sonja
Sackmann, Federico
Kerguelen Fuentes, Ana
Hernández-Maraver, Dolores
Döhner, Konstanze
Harrison, Claire N.
Radia, Deepti
Muxi, Pablo
Besses, Carlos
Cervantes Requena, F.
Johansson, Peter L.
Andreasson, Bjorn
Rambaldi, Alessandro
Barbui, Tiziano
Bonatz, Karin
Reiter, Andreas
Boyer, Francoise
Etienne, Gabriel
Ianotto, Jean-Christophe
Ranta, Dana
Roy, Lydia
Cahn, Jean-Yves
Maldonado, Norman
Barosi, Giovanni
Ferrari, Maria L.
Gale, Robert Peter
Birgegard, Gunnar
Xu, Zefeng
Zhang, Yue
Sun, Xiujuan
Xu, Junqing
Zhang, Peihong
Te Boekhorst, Peter A.
Commandeur, Suzan
Schouten, Harry
Pahl, Heike L.
Griesshammer, Martin
Stegelmann, Frank
Lehmann, Thomas
Senyak, Zhenya
Vannucchi, Alessandro M.
Passamonti, Francesco
Samuelsson, Jan
Mesa, Ruben A.
Keywords: Hematologia
Medul·la òssia
Malalties hematològiques
Bone marrow
Hematologic diseases
Issue Date: 1-Jan-2016
Publisher: American Society of Clinical Oncology
Abstract: Purpose 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. Patients and Methods 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). Results 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). Conclusion 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.
Note: Reproducció del document publicat a:
It is part of: Journal of Clinical Oncology, 2016, vol. 34, num. 2, p. 151-159
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ISSN: 0732-183X
Appears in Collections:Articles publicats en revistes (Medicina)
Articles publicats en revistes (IDIBAPS: Institut d'investigacions Biomèdiques August Pi i Sunyer)

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