Symptomatic Profiles of Patients leWith Polycythemia Vera: Implications of Inadequately Controlled Disease

被引：50

作者：

Geyer, Holly ^{[1
]}

Scherber, Robyn ^{[1
]}

Kosiorek, Heidi ^{[1
]}

Dueck, Amylou C. ^{[1
]}

Kiladjian, Jean-Jacques ^{[2
]}

Xiao, Zhijian ^{[9
,10
]}

Slot, Stefanie ^{[11
]}

Zweegman, Sonja ^{[11
]}

Sackmann, Federico ^{[15
]}

Kerguelen Fuentes, Ana ^{[16
]}

Hernandez-Maraver, Dolores ^{[16
]}

Doehner, Konstanze ^{[19
]}

Harrison, Claire N. ^{[23
]}

Radia, Deepti ^{[23
]}

Muxi, Pablo ^{[25
]}

Besses, Carlos ^{[17
]}

Cervantes, Francisco ^{[18
]}

Johansson, Peter L. ^{[26
]}

Andreasson, Bjorn ^{[26
]}

Rambaldi, Alessandro ^{[29
]}

Barbui, Tiziano ^{[29
]}

Bonatz, Karin ^{[20
]}

Reiter, Andreas ^{[20
]}

Boyer, Francoise ^{[3
]}

Etienne, Gabriel ^{[4
]}

Ianotto, Jean-Christophe ^{[5
]}

Ranta, Dana ^{[6
]}

Roy, Lydia ^{[7
]}

Cahn, Jean-Yves ^{[8
]}

Maldonado, Norman ^{[33
]}

Barosi, Giovanni ^{[30
]}

Ferrari, Maria L. ^{[29
]}

Gale, Robert Peter ^{[24
]}

Birgegard, Gunnar ^{[27
]}

Xu, Zefeng ^{[9
,10
]}

Zhang, Yue ^{[9
,10
]}

Sun, Xiujuan ^{[9
,10
]}

Xu, Junqing ^{[9
,10
]}

Zhang, Peihong ^{[9
,10
]}

te Boekhorst, Peter A. W. ^{[12
]}

Commandeur, Suzan ^{[13
]}

Schouten, Harry ^{[14
]}

Pahl, Heike L. ^{[21
]}

Griesshammer, Martin ^{[22
]}

Stegelmann, Frank ^{[19
]}

Lehmann, Thomas ^{[34
]}

Senyak, Zhenya ^{[35
]}

Vannucchi, Alessandro M. ^{[32
]}

Passamonti, Francesco ^{[31
]}

Samuelsson, Jan ^{[28
]}

机构：

[1] Mayo Clin, Scottsdale, AZ USA

[2] Hosp St Louis, Paris, France

[3] Ctr Hosp Univ, Angers, France

[4] Inst Bergonie, Bordeaux, France

[5] Ctr Hosp Univ, Brest, France

[6] Ctr Hosp Univ, Grenoble, France

[7] Ctr Hosp Univ, Poitiers, France

[8] Ctr Hosp Univ, Grenoble, France

[9] Chinese Acad Med Sci, Tianjin, Peoples R China

[10] Peking Union Med Coll, Tianjin, Peoples R China

[11] Vrije Univ Amsterdam, Med Ctr, Amsterdam, Netherlands

[12] Erasmus MC, Rotterdam, Netherlands

[13] Leiden Univ, Med Ctr, Leiden, Netherlands

[14] Maastricht Univ, Med Ctr, NL-6200 MD Maastricht, Netherlands

[15] Fundaleu, Buenos Aires, DF, Argentina

[16] Univ Hosp La Paz, Madrid, Spain

[17] Hosp del Mar, Barcelona, Spain

[18] Univ Barcelona, Barcelona, Spain

[19] Univ Hosp Ulm, Ulm, Germany

[20] Univ Med Mannheim, Mannheim, Germany

[21] Univ Hosp Freiburg, Freiburg, Germany

[22] Johannes Wesling Klinikum Minden, Minden, Germany

[23] Guys & St Thomas NHS Fdn Trust, London, England

[24] Univ London Imperial Coll Sci Technol & Med, London, England

[25] Hosp Britan, Montevideo, Uruguay

[26] NU Hosp Org, Uddevalla, Sweden

[27] Univ Uppsala Hosp, Uppsala, Sweden

[28] Stockholm South Hosp, Stockholm, Sweden

[29] Osped Riuniti Bergamo, Bergamo, Italy

[30] IRCCS Policlin S Matteo Fdn, Milan, Italy

[31] Univ Pavia, Fdn IRCCS Policlin San Matteo, Via Palestro 3, I-27100 Pavia, Italy

[32] Osped Circolo Varese, Varese, Italy

[33] Univ Puerto Rico, Sch Med, San Juan, PR 00936 USA

[34] Univ Basel Hosp, CH-4031 Basel, Switzerland

[35] MPN Forum, Asheville, NC USA

来源：

JOURNAL OF CLINICAL ONCOLOGY | 2016年 / 34卷 / 02期

关键词：

BURDEN; CLASSIFICATION; HYDROXYUREA; INTOLERANCE; RESISTANCE; DIAGNOSIS; CRITERIA; TRIAL;

D O I：

10.1200/JCO.2015.62.9337

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Purpose Polycythemia vera (PV) is amyeloproliferative 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. (C) 2015 by American Society of Clinical Oncology

引用

页码：151 / +

页数：11

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