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

Publication

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

Journal Paper/Review - Oct 14, 2017

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

Citation

Scotch A, Kosiorek H, Scherber R, Dueck A, Slot S, Zweegman S, Boekhorst P, Commandeur S, Schouten H, Sackmann F, Fuentes A, Hernández-Maraver D, Pahl H, Griesshammer M, Stegelmann F, Döhner K, Lehmann T, Bonatz K, Reiter A, Boyer F, Etienne G, Ianotto J, Ranta D, Roy L, Cahn J, Harrison C, Radia D, Muxi P, Maldonado N, Besses C, Cervantes F, Johansson P, Barbui T, Barosi G, Vannucchi A, Paoli C, Passamonti F, Andreasson B, Ferrari M, Rambaldi A, Samuelsson J, Birgegard G, Xiao Z, Xu Z, Zhang Y, Sun X, Xu J, Kiladjian J, Zhang P, Gale R, Mesa R, Geyer H. Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs. Leuk Res 2017; 63:34-40.

Type

Journal Paper/Review (English)

Journal

Leuk Res 2017; 63

Publication Date

Oct 14, 2017

Issn Electronic

1873-5835

Pages

34-40

Brief description/objective

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 <100×10/L (moderate 51-100×10/L; severe ≤50×10/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.