Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: Prospective International Assessment of an Abbreviated Symptom Burden Scoring System Among Patients With MPNs

被引:344
作者
Emanuel, Robyn M.
Dueck, Amylou C.
Geyer, Holly L.
Kiladjian, Jean-Jacques [2 ]
Slot, Stefanie [9 ]
Zweegman, Sonja [9 ]
te Boekhorst, Peter A. W. [10 ]
Commandeur, Suzan [11 ]
Schouten, Harry C. [12 ]
Sackmann, Federico [13 ]
Kerguelen Fuentes, Ana [14 ]
Hernandez-Maraver, Dolores [14 ]
Pahl, Heike L. [17 ]
Griesshammer, Martin [18 ]
Stegelmann, Frank [19 ]
Doehner, Konstanze [19 ]
Lehmann, Thomas [21 ]
Bonatz, Karin [20 ]
Reiter, Andreas [20 ]
Boyer, Francoise [3 ]
Etienne, Gabriel [4 ]
Ianotto, Jean-Christophe [5 ]
Ranta, Dana [6 ]
Roy, Lydia [8 ]
Cahn, Jean-Yves [7 ]
Harrison, Claire N. [22 ]
Radia, Deepti [22 ]
Muxi, Pablo [23 ]
Maldonado, Norman [24 ]
Besses, Carlos [16 ]
Cervantes, Francisco [15 ]
Johansson, Peter L. [25 ]
Barbui, Tiziano [28 ]
Barosi, Giovanni [29 ]
Vannucchi, Alessandro M. [30 ]
Passamonti, Francesco [29 ]
Andreasson, Bjorn [25 ]
Ferarri, Maria L. [28 ]
Rambaldi, Alessandro [28 ]
Samuelsson, Jan [26 ]
Birgegard, Gunnar [27 ]
Tefferi, Ayalew [31 ]
Mesa, Ruben A. [1 ]
机构
[1] Mayo Clin Scottsdale, Div Hematol & Oncol, Scottsdale, AZ 85259 USA
[2] Hop St Louis, Paris, France
[3] CHU Angers, Angers, France
[4] Inst Bergonie, Bordeaux, France
[5] CHU Brest, F-29285 Brest, France
[6] Hosp Univ, Nancy, France
[7] CHU Grenoble, F-38043 Grenoble, France
[8] Ctr Hosp Univ, Poitiers, France
[9] Vrije Univ Univ Med Ctr, Amsterdam, Netherlands
[10] Erasmus MC, Rotterdam, Netherlands
[11] Leiden Univ Med Ctr, Leiden, Netherlands
[12] Maastricht Univ Med Ctr, Maastricht, Netherlands
[13] Fundaleu, Buenos Aires, DF, Argentina
[14] Univ Hosp La Paz, Madrid, Spain
[15] Univ Barcelona, Hosp Clin, Inst Invest Biomed August Pi i Sunyer, Barcelona, Spain
[16] Hosp del Mar, Barcelona, Spain
[17] Univ Hosp Freiburg, Freiburg, Germany
[18] Johannes Wesling Klinikum Minden, Minden, Germany
[19] Univ Hosp Ulm, Ulm, Germany
[20] Univ Med Mannheim, Mannheim, Germany
[21] Univ Basel Hosp, Basel, Switzerland
[22] Guys & St Thomas Natl Hlth Serv Fdn Trust, London, England
[23] Hosp Britan, Montevideo, Uruguay
[24] Univ Puerto Rico Sch Med, San Juan, PR USA
[25] NU Hosp Org, Uddevalla, Sweden
[26] Stockholm S Hosp, Stockholm, Sweden
[27] Uppsala Univ, Uppsala, Sweden
[28] Osped Riuniti Bergamo, I-24100 Bergamo, Italy
[29] Univ Pavia, Fdn Ist Ricovero & Cura Carattere Sci Policlin Sa, I-27100 Pavia, Italy
[30] Univ Florence, Florence, Italy
[31] Mayo Clin, Rochester, MN USA
关键词
QUALITY-OF-LIFE; MYELOFIBROSIS; TRIAL; INVENTORY; FATIGUE;
D O I
10.1200/JCO.2012.42.3863
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Purpose Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies. We aimed to assess the utility of an abbreviated symptom score for the most pertinent and representative MPN symptoms for subsequent serial use in assessing response to therapy. Patients and Methods The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (MPN-SAF TSS) was calculated as the mean score for 10 items from two previously validated scoring systems. Questions focus on fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers. Results MPN-SAF TSS was calculable for 1,408 of 1,433 patients with MPNs who had a mean score of 21.2 (standard deviation [SD], 16.3). MPN-SAF TSS results significantly differed among MPN disease subtypes (P < .001), with a mean of 18.7 (SD, 15.3), 21.8 (SD, 16.3), and 25.3 (SD, 17.2) for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis, respectively. The MPN-SAF TSS strongly correlated with overall quality of life (QOL; r = 0.59; P < .001) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) functional scales (all P < .001 and absolute r >= 0.50 except social functioning r = 0.48). No significant trends were present when comparing therapy subgroups. The MPN-SAF TSS had excellent internal consistency (Cronbach's alpha = .83). Factor analysis identified a single underlying construct, indicating that the MPN-SAF TSS is an appropriate, unified scoring method. Conclusion The MPN-SAF TSS is a concise, valid, and accurate assessment of MPN symptom burden with demonstrated clinical utility in the largest prospective MPN symptom study to date. This new prospective scoring method may be used to assess MPN symptom burden in both clinical practice and trial settings.
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页码:4098 / 4103
页数:6
相关论文
共 10 条
[1]   THE EUROPEAN-ORGANIZATION-FOR-RESEARCH-AND-TREATMENT-OF-CANCER QLQ-C30 - A QUALITY-OF-LIFE INSTRUMENT FOR USE IN INTERNATIONAL CLINICAL-TRIALS IN ONCOLOGY [J].
AARONSON, NK ;
AHMEDZAI, S ;
BERGMAN, B ;
BULLINGER, M ;
CULL, A ;
DUEZ, NJ ;
FILIBERTI, A ;
FLECHTNER, H ;
FLEISHMAN, SB ;
DEHAES, JCJM ;
KAASA, S ;
KLEE, M ;
OSOBA, D ;
RAZAVI, D ;
ROFE, PB ;
SCHRAUB, S ;
SNEEUW, K ;
SULLIVAN, M ;
TAKEDA, F .
JOURNAL OF THE NATIONAL CANCER INSTITUTE, 1993, 85 (05) :365-376
[2]  
Mendoza TR, 1999, CANCER-AM CANCER SOC, V85, P1186, DOI 10.1002/(SICI)1097-0142(19990301)85:5<1186::AID-CNCR24>3.0.CO
[3]  
2-N
[4]   The burden of fatigue and quality of life in myeloproliferative disorders (MPDs) - An international internet-based survey of 1179 MPD patients [J].
Mesa, Ruben A. ;
Niblack, Joyce ;
Wadleigh, Martha ;
Verstovsek, Srdan ;
Camoriano, John ;
Barnes, Sunni ;
Tan, Angelina D. ;
Atherton, Pamela J. ;
Sloan, Jeff A. ;
Tefferi, Ayalew .
CANCER, 2007, 109 (01) :68-76
[5]   Evaluating the Serial Use of the Myelofibrosis Symptom Assessment Form for Measuring Symptomatic Improvement Performance in 87 Myelofibrosis Patients on a JAK1 and JAK2 Inhibitor (INCB018424) Clinical Trial [J].
Mesa, Ruben A. ;
Kantarjian, Hagop ;
Tefferi, Ayalew ;
Dueck, Amylou ;
Levy, Richard ;
Vaddi, Kris ;
Erickson-Viitanen, Susan ;
Thomas, Deborah A. ;
Cortes, Jorge ;
Borthakur, Gautam ;
Pardanani, Animesh D. ;
Estrov, Zeev ;
Verstovsek, Srdan .
CANCER, 2011, 117 (21) :4869-4877
[6]   The Myelofibrosis Symptom Assessment Form (MFSAF): An evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis [J].
Mesa, Ruben A. ;
Schwager, Susan ;
Radia, Deepti ;
Cheville, Andrea ;
Hussein, Kebede ;
Niblack, Joyce ;
Pardanani, Animesh D. ;
Steensma, David P. ;
Litzow, Mark R. ;
Rivera, Candido E. ;
Camoriano, John ;
Verstovsek, Srdan ;
Sloan, Jeffrey ;
Harrison, Claire ;
Kantarjian, Hagop ;
Tefferi, Ayalew .
LEUKEMIA RESEARCH, 2009, 33 (09) :1199-1203
[7]   Safety and Efficacy of TG101348, a Selective JAK2 Inhibitor, in Myelofibrosis [J].
Pardanani, Animesh ;
Gotlib, Jason R. ;
Jamieson, Catriona ;
Cortes, Jorge E. ;
Talpaz, Moshe ;
Stone, Richard M. ;
Silverman, Michael H. ;
Gilliland, D. Gary ;
Shorr, Jolene ;
Tefferi, Ayalew .
JOURNAL OF CLINICAL ONCOLOGY, 2011, 29 (07) :789-796
[8]   The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): International Prospective Validation and Reliability Trial in 402 patients [J].
Scherber, Robyn ;
Dueck, Amylou C. ;
Johansson, Peter ;
Barbui, Tiziano ;
Barosi, Giovanni ;
Vannucchi, Alessandro M. ;
Passamonti, Francesco ;
Andreasson, Bjorn ;
Ferarri, Maria L. ;
Rambaldi, Alessandro ;
Samuelsson, Jan ;
Birgegard, Gunnar ;
Tefferi, Ayalew ;
Harrison, Claire N. ;
Radia, Deepti ;
Mesa, Ruben A. .
BLOOD, 2011, 118 (02) :401-408
[9]   A Double-Blind, Placebo-Controlled Trial of Ruxolitinib for Myelofibrosis [J].
Verstovsek, Srdan ;
Mesa, Ruben A. ;
Gotlib, Jason ;
Levy, Richard S. ;
Gupta, Vikas ;
DiPersio, John F. ;
Catalano, John V. ;
Deininger, Michael ;
Miller, Carole ;
Silver, Richard T. ;
Talpaz, Moshe ;
Winton, Elliott F. ;
Harvey, Jimmie H., Jr. ;
Arcasoy, Murat O. ;
Hexner, Elizabeth ;
Lyons, Roger M. ;
Paquette, Ronald ;
Raza, Azra ;
Vaddi, Kris ;
Erickson-Viitanen, Susan ;
Koumenis, Iphigenia L. ;
Sun, William ;
Sandor, Victor ;
Kantarjian, Hagop M. .
NEW ENGLAND JOURNAL OF MEDICINE, 2012, 366 (09) :799-807
[10]   Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: Report of the ISPOR Task Force for Translation and Cultural Adaptation [J].
Wild, D ;
Grove, A ;
Martin, M ;
Eremenco, S ;
McElroy, S ;
Verjee-Lorenz, A ;
Erikson, P .
VALUE IN HEALTH, 2005, 8 (02) :94-104