Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs

Allison H. Scotch, Heidi Kosiorek, Robyn Scherber, Amylou C. Dueck, Stefanie Slot, Sonja Zweegman, Peter A.W.te Boekhorst, Suzan Commandeur, Harry Schouten, Federico Sackmann, Ana Kerguelen Fuentes, Dolores Hernández-Maraver, Heike L. Pahl, Martin Griesshammer, Frank Stegelmann, Konstanze Döhner, Thomas Lehmann, Karin Bonatz, Andreas Reiter, Francoise BoyerGabriel Etienne, Jean Christophe Ianotto, Dana Ranta, Lydia Roy, Jean Yves Cahn, Claire N. Harrison, Deepti Radia, Pablo Muxi, Norman Maldonado, Carlos Besses, Francisco Cervantes, Peter L. Johansson, Tiziano Barbui, Giovanni Barosi, Alessandro M. Vannucchi, Chiara Paoli, Francesco Passamonti, Bjorn Andreasson, Maria L. Ferrari, Alessandro Rambaldi, Jan Samuelsson, Gunnar Birgegard, Zhijian Xiao, Zefeng Xu, Yue Zhang, Xiujuan Sun, Junqing Xu, Jean Jacques Kiladjian, Peihong Zhang, Robert Peter Gale, Ruben A. Mesa, Holly L. Geyer*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Myelofibrosis is a myeloproliferative neoplasm associated with progressive cytopenias and high symptom burden. MF patients with thrombocytopenia have poor prognosis but the presence of thrombocytopenia frequently precludes the use of JAK2 inhibitors. In this study, we assessed quality of life and symptom burden in 418 MF patients with (n = 89) and without (n = 329) thrombocytopenia using prospective data from the MPN-QOL study group database, including the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and Total Symptom Score (MPN10). Thrombocytopenia, defined as platelet count <100 × 109/L (moderate 51–100 × 109/L; severe ≤50 × 109/L), was associated with anemia (76% vs. 45%, p < 0.001), leukopenia (29% vs. 11%, p < 0.001), and need for red blood cell transfusion (35% vs. 19%, p = 0.002). Thrombocytopenic patients had more fatigue, early satiety, inactivity, dizziness, sad mood, cough, night sweats, itching, fever, and weight loss; total symptom scores were also higher (33 vs. 24, p < 0.001). Patients with severe thrombocytopenia were more likely to have anemia (86% vs. 67%, p = 0.04), leukopenia (40% vs. 20%, p = 0.04), and transfusion requirements (51% vs. 20%, p = 0.002) but few differences in symptoms when compared to patients with moderate thrombocytopenia. These results suggest that MF patients with thrombocytopenia experience greater symptomatic burden than MF patients without thrombocytopenia and may benefit from additional therapies.

Original languageEnglish
Pages (from-to)34-40
Number of pages7
JournalLeukemia Research
Volume63
DOIs
Publication statusPublished - 1 Dec 2017

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