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. 2017 Dec:63:34-40.
doi: 10.1016/j.leukres.2017.10.002. Epub 2017 Oct 14.

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

Allison H Scotch  1 , Heidi Kosiorek  2 , Robyn Scherber  3 , Amylou C Dueck  2 , Stefanie Slot  4 , Sonja Zweegman  4 , Peter A W Te Boekhorst  5 , Suzan Commandeur  6 , Harry Schouten  7 , Federico Sackmann  8 , Ana Kerguelen Fuentes  9 , Dolores Hernández-Maraver  9 , Heike L Pahl  10 , Martin Griesshammer  11 , Frank Stegelmann  12 , Konstanze Döhner  12 , Thomas Lehmann  13 , Karin Bonatz  14 , Andreas Reiter  14 , Francoise Boyer  15 , Gabriel Etienne  16 , Jean-Christophe Ianotto  17 , Dana Ranta  18 , Lydia Roy  19 , Jean-Yves Cahn  20 , Claire N Harrison  21 , Deepti Radia  21 , Pablo Muxi  22 , Norman Maldonado  23 , Carlos Besses  24 , Francisco Cervantes  25 , Peter L Johansson  26 , Tiziano Barbui  27 , Giovanni Barosi  28 , Alessandro M Vannucchi  29 , Chiara Paoli  29 , Francesco Passamonti  30 , Bjorn Andreasson  26 , Maria L Ferrari  31 , Alessandro Rambaldi  27 , Jan Samuelsson  32 , Gunnar Birgegard  33 , Zhijian Xiao  34 , Zefeng Xu  34 , Yue Zhang  34 , Xiujuan Sun  34 , Junqing Xu  34 , Jean-Jacques Kiladjian  35 , Peihong Zhang  36 , Robert Peter Gale  37 , Ruben A Mesa  38 , Holly L Geyer  39
Affiliations

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

Allison H Scotch et al. Leuk Res. 2017 Dec.

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.

Keywords: Myelofibrosis; Myeloproliferative neoplasm; Quality of life; Symptomatology; Thrombocytopenia.

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Figures

Fig. 1.
Fig. 1.
Thrombocytopenia versus no thrombocytopenia by individual MPN-SAF score.
Fig. 2.
Fig. 2.
Thrombocytopenia versus no thrombocytopenia by individual MPN-SAF symptom prevalence.
Fig. 3.
Fig. 3.
Moderate versus severe thrombocytopenia by individual MPN-SAF Score.
Fig. 4.
Fig. 4.
Moderate versus severe thrombocytopenia by individual MPN-SAF symptom frequency.
See this image and copyright information in PMC

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