The Myelofibrosis Symptom Burden (MF-SB): An International Phenotypic Cluster Analysis of 329 Patients

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

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Abstract 1731 BackgroundSymptom burden in primary, post-ET and post-PV myelofibrosis (MF) is frequently severe and correlates with a poor prognosis. However, symptom manifestations are heterogeneous with variable presence of specific symptoms, splenomegaly and cytopenias. We sought to identify the spectrum and features of MF symptomatic phenotypes by cluster analysis of prospectively gathered information on MF symptoms and disease features. MethodsData was collected among an international cohort of subjects with MF. Data included demographics, disease features and completion of the Brief Fatigue Inventory (BFI) and Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) (Blood 2011; 118:401-408). Surveyed symptoms addressed key disease features on a 0 (absent) to 10 (worst-imaginable) scale. Cluster development was based on consideration of r-squared in hierarchical clustering using Ward's 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 329 prospectively enrolled persons with MF was collected (Chinese 102, French 54, German 19, Italian 22, Dutch 45, English 51, Spanish 29, Swedish 7) including 223 PMF, 67 post-ET MF and 39 post-PV MF patients. Participants were of typical age (mean 59) and gender (47% F). Among all participants, four natural symptom clusters were identified (Figure 1). Among clusters, disease features including leukopenia, thrombocytopenia, and enlarged spleen varied significantly between clusters (P 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
Publication statusPublished - 2012

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