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J Clin Oncol. 2016 Jan 10;34(2):151-9. doi: 10.1200/JCO.2015.62.9337. Epub 2015 Nov 23.

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

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1
Holly Geyer, Robyn Scherber, Heidi Kosiorek, Amylou C. Dueck, and Ruben A. Mesa, Mayo Clinic, Scottsdale, AZ; Jean-Jacques Kiladjian, Hospital Saint-Louis, Paris; Francoise Boyer, Centre Hospitalier Universitaire, Angers; Gabriel Etienne, Institut Bergonie, Bordeaux; Jean-Christophe Ianotto, Centre Hospitalier Universitaire, Brest; Dana Ranta, Centre Hospitalier Universitaire, Grenoble; Lydia Roy, Centre Hospitalier Universitaire, Poitiers; Jean-Yves Cahn, Centre Hospitalier Universitaire, Grenoble, France; Zhijian Xiao, Zefeng Xu, Yue Zhang, Xiujuan Sun, Junqing Xu, and Peihong Zhang, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China; Stefanie Slot and Sonja Zweegman, VU University Medical Center, Amsterdam; Peter A.W. te Boekhorst, Erasmus Medical Center, Rotterdam; Suzan Commandeur, Leiden University Medical Center, Leiden; Harry Schouten, Maastricht University Medical Center, Maastricht, the Netherlands; Frederico Sackmann, Fundaleu, Buenos Aires, Argentina; Ana Kerguelen Fuentes and Dolores Hernández-Maraver, University Hospital La Paz, Madrid; Carlos Besses, Hospital del Mar; Francisco Cervantes, University of Barcelona, Barcelona, Spain; Konstanze Döhner and Frank Stegelmann, University Hospital of Ulm; Karin Bonatz and Andreas Reiter, Universitätsmedizin Mannheim, Mannheim; Heike L. Pahl, University Hospital Freiburg, Freiburg; Martin Griesshammer, Johannes Wesling Klinikum Minden, Germany; Claire N. Harrison and Deepti Radia, Guy's and St Thomas NHS Foundation Trust; Robert Peter Gale, Imperial College, London, United Kingdom; Pablo Muxi, Hospital Británico, Montevideo, Uruguay; Peter L. Johansson and Bjorn Andreasson, NU Hospital Organization, Uddevalla; Gunnar Birgegard, University Hospital, Uppsala; Jan Samuelsson, Stockholm South Hospital, Stockholm, Sweden; Alessandro Rambaldi, Tiziano Barbui, and Maria L. Ferrari, Ospedali Riuniti di Bergamo, Bergamo; Giovanni Barosi, IRCCS Policlinico S. Matteo Foundation; Franc

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.

PMID:
26598745
DOI:
10.1200/JCO.2015.62.9337
[Indexed for MEDLINE]
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