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 Boyer; Gabriel 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

doi:https://doi.org/10.1016/j.leukres.2017.10.002

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

Research output: Contribution to journal › Article › Academic › peer-review

13 Citations (Scopus)

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 language	English
Pages (from-to)	34-40
Number of pages	7
Journal	Leukemia research
Volume	63
DOIs	https://doi.org/10.1016/j.leukres.2017.10.002
Publication status	Published - 1 Dec 2017

Keywords

Myelofibrosis
Myeloproliferative neoplasm
Quality of life
Symptomatology
Thrombocytopenia

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Scotch, A. H., Kosiorek, H., Scherber, R., Dueck, A. C., Slot, S., Zweegman, S., Boekhorst, P. A. W. T., Commandeur, S., Schouten, H., Sackmann, F., Fuentes, A. K., Hernández-Maraver, D., Pahl, H. L., Griesshammer, M., Stegelmann, F., Döhner, K., Lehmann, T., Bonatz, K., Reiter, A., ... Geyer, H. L. (2017). Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs. Leukemia research, 63, 34-40. https://doi.org/10.1016/j.leukres.2017.10.002

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title = "Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs",

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",

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

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doi = "https://doi.org/10.1016/j.leukres.2017.10.002",

language = "English",

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pages = "34--40",

journal = "Leukemia research",

issn = "0145-2126",

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Scotch, AH, Kosiorek, H, Scherber, R, Dueck, AC, Slot, S , Zweegman, S, Boekhorst, PAWT, Commandeur, S, Schouten, H, Sackmann, F, Fuentes, AK, Hernández-Maraver, D, Pahl, HL, Griesshammer, M, Stegelmann, F, Döhner, K, Lehmann, T, Bonatz, K, Reiter, A, Boyer, F, Etienne, G, Ianotto, JC, Ranta, D, Roy, L, Cahn, JY, Harrison, CN, Radia, D, Muxi, P, Maldonado, N, Besses, C, Cervantes, F, Johansson, PL, Barbui, T, Barosi, G, Vannucchi, AM, Paoli, C, Passamonti, F, Andreasson, B, Ferrari, ML, Rambaldi, A, Samuelsson, J, Birgegard, G, Xiao, Z, Xu, Z, Zhang, Y, Sun, X, Xu, J, Kiladjian, JJ, Zhang, P, Gale, RP, Mesa, RA & Geyer, HL 2017, 'Symptom burden profile in myelofibrosis patients with thrombocytopenia: Lessons and unmet needs', Leukemia research, vol. 63, pp. 34-40. https://doi.org/10.1016/j.leukres.2017.10.002

TY - JOUR

T1 - Symptom burden profile in myelofibrosis patients with thrombocytopenia

T2 - Lessons and unmet needs

AU - Scotch, Allison H.

AU - Kosiorek, Heidi

AU - Scherber, Robyn

AU - Dueck, Amylou C.

AU - Slot, Stefanie

AU - Zweegman, Sonja

AU - Boekhorst, Peter A.W.te

AU - Commandeur, Suzan

AU - Schouten, Harry

AU - Sackmann, Federico

AU - Fuentes, Ana Kerguelen

AU - Hernández-Maraver, Dolores

AU - Pahl, Heike L.

AU - Griesshammer, Martin

AU - Stegelmann, Frank

AU - Döhner, Konstanze

AU - Lehmann, Thomas

AU - Bonatz, Karin

AU - Reiter, Andreas

AU - Boyer, Francoise

AU - Etienne, Gabriel

AU - Ianotto, Jean Christophe

AU - Ranta, Dana

AU - Roy, Lydia

AU - Cahn, Jean Yves

AU - Harrison, Claire N.

AU - Radia, Deepti

AU - Muxi, Pablo

AU - Maldonado, Norman

AU - Besses, Carlos

AU - Cervantes, Francisco

AU - Johansson, Peter L.

AU - Barbui, Tiziano

AU - Barosi, Giovanni

AU - Vannucchi, Alessandro M.

AU - Paoli, Chiara

AU - Passamonti, Francesco

AU - Andreasson, Bjorn

AU - Ferrari, Maria L.

AU - Rambaldi, Alessandro

AU - Samuelsson, Jan

AU - Birgegard, Gunnar

AU - Xiao, Zhijian

AU - Xu, Zefeng

AU - Zhang, Yue

AU - Sun, Xiujuan

AU - Xu, Junqing

AU - Kiladjian, Jean Jacques

AU - Zhang, Peihong

AU - Gale, Robert Peter

AU - Mesa, Ruben A.

AU - Geyer, Holly L.

PY - 2017/12/1

Y1 - 2017/12/1

N2 - 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.

AB - 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.

KW - Myelofibrosis

KW - Myeloproliferative neoplasm

KW - Quality of life

KW - Symptomatology

KW - Thrombocytopenia

UR - http://www.scopus.com/inward/record.url?scp=85032433607&partnerID=8YFLogxK

U2 - https://doi.org/10.1016/j.leukres.2017.10.002

DO - https://doi.org/10.1016/j.leukres.2017.10.002

M3 - Article

C2 - 29096334

SN - 0145-2126

VL - 63

SP - 34

EP - 40

JO - Leukemia research

JF - Leukemia research

ER -

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

Abstract

Keywords

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