Essential Thrombocythemia (ET) and Polycythemia Vera (PV) Symptom Burden: Phenotypic Cluster Analysis Among an International Sample of 1,141 ET and PV Patients

Robyn M Emanuel, Amylou C Dueck, Holly Lynn Geyer, Jean-Jacques Kiladjian, Stefanie Slot, Sonja Zweegman, Peter te Boekhorst, Suzan Commandeur, Harry C Schouten, Federico Sackmann, Ana Kerguelen Fuentes, Dolores Hernandez, Heike L Pahl, Martin Griesshammer, Frank Stegelmann, Konstanze Doehner, Thomas Lehmann, Karin Bonatz, Andreas Reiter, Francoise BoyerGabriel Etienne, Jean-Christophe Ianotto, Dana Ranta, Lydia Roy, Jean-Yves Cahn, Claire N Harrison, Deepti H Radia, Pablo J Muxi, Norman I Maldonado, Carlos Besses, Francisco Cervantes, Peter Johansson, Tiziano Barbui, Giovanni Barosi, Alessandro M Vannucchi, Francesco Passamonti, Bjorn Andreasson, Maria L Ferarri, Alessandro Rambaldi, Gunnar Birgegard, Zhijian Xiao, Zefeng Xu, Yue Zhang, Xiujuan Sun, Junqing Xu, Peihong Zhang, Robert Peter Gale, Ayalew Tefferi, Ruben A Mesa

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Abstract 1726 BackgroundWe previously reported that symptom burden among persons with ET and PV can be severe and adversely affect QOL. The presence of severe symptoms is linked to poor prognosis. There is considerable inter-subject heterogeneity regarding which symptoms are present in which subjects. No studies have empirically evaluated whether disease characteristics can be grouped in related symptom clusters. Using our previously validated 18 item Myeloproliferative Neoplasm Assessment Form (MPN-SAF) (Blood 2011;118:401-408) given in conjunction with the 9 item Brief Fatigue Inventory (BFI) (Cancer 1999;85:1186-1196), we sought to evaluate symptom burden by means of cluster analysis. MethodsData was collected from an international cohort of subjects with MPNs including demographics, disease features and the completed BFI and MPN-SAF instruments. Surveyed symptoms included fatigue, early satiety, abdominal pain and discomfort, inactivity, headaches, concentration, dizziness, extremity tingling, insomnia, sexual difficulties, mood changes, cough, night sweats, pruritus, bone pain and fever on a 0 (absent) to 10 (worst-imaginable) scale. Development of symptom clusters was based on consideration of r-squared in hierarchical clustering using Ward linkage. Final cluster assignment was based on the nonhierarchical k-means method. Comparisons between symptom clusters were based on ANOVA and chi-squared tests. ResultsSubject Demographic and Disease CharacteristicsData from 1,141 subjects with PV (N=519) and ET (N=622) was prospectively collected (Chinese 236, French 305, German 45, Italian 114, Dutch 191, English 56, Spanish 109, Swedish 85. Age (mean 59, range, 26-87) and gender (54% F) were typical. Five clusters were selected (Figure 1). Frequencies of prior bleeding, spleen size, anemia, presence of any lab abnormality, language, gender, and MPN type varied significantly between clusters (P[IMG]/small/bld0211200860128.gif" ALT="Figure 1">[IMG]/small/bld0211200860129.gif" ALT="Figure 1"> Figure 1. Individual symptom means by cluster. DisclosuresKiladjian: Celgene: Research Funding; Novartis: Honoraria, Research Funding; Shire: Honoraria. Roy: Novartis, BMS: Speakers Bureau. Harrison: Novartis: Honoraria, Research Funding, Speakers Bureau; YM Bioscience: Consultancy, Honoraria; Sanofi Aventis: Honoraria; Shire: Honoraria, Research Funding. Vannucchi: Novartis: Membership on an entity's Board of Directors or advisory committees. Passamonti: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees. Mesa: Incyte: Research Funding; Lilly: Research Funding; Sanofi: Research Funding; NS Pharma: Research Funding; YM Bioscience: Research Funding.\n
Original languageEnglish
Pages (from-to)1726
Number of pages1
JournalBlood (ASH Annual Meeting Abstracts)
Issue number21
Publication statusPublished - 16 Nov 2012

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