Patient-Reported Outcomes Enhance the Survival Prediction of Traditional Disease Risk Classifications: An International Study in Patients With Myelodysplastic Syndromes

被引:35
作者
Efficace, Fabio [1 ]
Cottone, Francesco [1 ]
Abel, Gregory [2 ]
Niscola, Pasquale [3 ]
Gaidano, Gianluca [4 ]
Bonnetain, Franck [5 ,6 ]
Anota, Amelie [5 ,6 ]
Caocci, Giovanni [7 ]
Cronin, Angel [2 ]
Fianchi, Luana [8 ]
Breccia, Massimo [9 ]
Stauder, Reinhard [10 ]
Platzbecker, Uwe [11 ]
Palumbo, Giuseppe A. [12 ]
Luppi, Mario [13 ]
Invernizzi, Rosangela [14 ]
Bergamaschi, Micaela [15 ]
Borin, Lorenza [16 ]
Di Tucci, Anna Angela [17 ]
Zhang, Huiyong [18 ]
Sprangers, Mirjam [19 ]
Vignetti, Marco [1 ]
Mandelli, Franco [1 ]
机构
[1] Italian Grp Adult Hematol Dis GIMEMA, Data Ctr & Hlth Outcomes Res Unit, Rome, Italy
[2] Harvard Med Sch, Dana Farber Canc Inst, Div Populat Sci, Boston, MA USA
[3] St Eugenio Hosp, Hematol Unit, Rome, Italy
[4] Univ Piemonte Orientale, Div Hematol, Dept Translat Med, Novara, Italy
[5] Univ Hosp Besancon, Methodol & Qual Life Oncol Unit, Besancon, France
[6] Univ Franche Comte, Platform Qual Life & Canc, INSERM 1098, Besancon, France
[7] Univ Cagliari, Dept Med Sci, Cagliari, Italy
[8] Univ Cattolica Sacro Cuore, Inst Hematol, Rome, Italy
[9] Sapienza Univ, Div Hematol, Dept Cellular Biotechnol & Hematol, Rome, Italy
[10] Innsbruck Med Univ, Dept Internal Med Hematol & Oncol 5, Innsbruck, Austria
[11] Univ Hosp Dresden Carl Gustav Carus, Dept Med 1, Dresden, Germany
[12] AOU Policlin V Emanuele, UO Ematol, Catania, Italy
[13] Univ Modena, Dept Hematol, Modena, Italy
[14] Univ Pavia, San Matteo IRCCS Policlin Fdn, Dept Internal Med, Pavia, Italy
[15] San Martino Clin Hematol Clin, Genoa, Italy
[16] San Gerardo Hosp, Dept Hematol, Monza, Italy
[17] Armando Businco Hosp, Hematol & Bone Marrow Transplantat Unit, Cagliari, Italy
[18] Liaoning Univ Tradit Chinese Med, Affiliated Hosp, Dept Hematol, Shenyang, Liaoning, Peoples R China
[19] Univ Amsterdam, Dept Med Psychol, Acad Med Ctr, Amsterdam, Netherlands
关键词
fatigue; myelodysplastic syndromes; patient-reported outcomes; quality of life; survival; QUALITY-OF-LIFE; PROGNOSTIC VALUE; SCORING SYSTEM; FATIGUE; DECISION; MODELS; CARE;
D O I
10.1002/cncr.31193
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
BACKGROUND: Current prognostic systems for myelodysplastic syndromes (MDS) are based on clinical, pathologic, and laboratory indicators. The objective of the current study was to develop a new patient-centered prognostic index for patients with advanced MDS by including self-reported fatigue severity into a well-established clinical risk classification: the International Prognostic Scoring System (IPSS). METHODS: A total of 469 patients with advanced (ie, IPSS intermediate-2 or high-risk) MDS were analyzed. Untreated patients (280 patients) were recruited into an international prospective cohort observational study to create the index. The index then was applied to an independent cohort including pretreated patients with MDS from the Dana-Farber Cancer Institute in Boston, Massachusetts (189 patients). At baseline, patients completed the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). RESULTS: A new prognostic index was developed: the FA-IPSS(h), in which FA stands for fatigue and h for higher-risk. This new risk classification enabled the authors to distinguish 3 subgroups of patients with distinct survival outcomes (ie, risk-1, risk-2, and risk-3). Patients classified as FA-IPSS(h) risk-1 had a median overall survival (OS) of 23 months (95% confidence interval [95% CI], 19-29 months), whereas those with risk-2 had a median OS of 16 months (95% CI, 12-17 months) and those with risk-3 had a median OS of 10 months (95% CI, 4-13 months). The predictive accuracy of this new index was higher than that of the IPSS alone in both the development cohort as well as in the independent cohort including pretreated patients. CONCLUSIONS: The FA-IPSS(h) is a novel patient-centered prognostic index that includes patients' self-reported fatigue severity. The authors believe its use might enhance physicians' ability to predict survival more accurately in patients with advanced MDS. (C) 2017 American Cancer Society.
引用
收藏
页码:1251 / 1259
页数:9
相关论文
共 34 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]   Myelodysplastic syndromes [J].
Ades, Lionel ;
Itzykson, Raphael ;
Fenaux, Pierre .
LANCET, 2014, 383 (9936) :2239-2252
[3]   BOOTSTRAP INVESTIGATION OF THE STABILITY OF A COX REGRESSION-MODEL [J].
ALTMAN, DG ;
ANDERSEN, PK .
STATISTICS IN MEDICINE, 1989, 8 (07) :771-783
[4]   Symptom Monitoring With Patient-Reported Outcomes During Routine Cancer Treatment: A Randomized Controlled Trial [J].
Basch, Ethan ;
Deal, Allison M. ;
Kris, Mark G. ;
Scher, Howard I. ;
Hudis, Clifford A. ;
Sabbatini, Paul ;
Rogak, Lauren ;
Bennett, Antonia V. ;
Dueck, Amylou C. ;
Atkinson, Thomas M. ;
Chou, Joanne F. ;
Dulko, Dorothy ;
Sit, Laura ;
Barz, Allison ;
Novotny, Paul ;
Fruscione, Michael ;
Sloan, Jeff A. ;
Schrag, Deborah .
JOURNAL OF CLINICAL ONCOLOGY, 2016, 34 (06) :557-+
[5]   Adverse Symptom Event Reporting by Patients vs Clinicians: Relationships With Clinical Outcomes [J].
Basch, Ethan ;
Jia, Xiaoyu ;
Heller, Glenn ;
Barz, Allison ;
Sit, Laura ;
Fruscione, Michael ;
Appawu, Mark ;
Iasonos, Alexia ;
Atkinson, Thomas ;
Goldfarb, Shari ;
Culkin, Ann ;
Kris, Mark G. ;
Schrag, Deborah .
JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE, 2009, 101 (23) :1624-1632
[6]   Prognostic models in myelodysplastic syndromes [J].
Bejar, Rafael .
HEMATOLOGY-AMERICAN SOCIETY OF HEMATOLOGY EDUCATION PROGRAM, 2013, :504-510
[7]   Health-related quality of life and symptom assessment in patients with myelodysplastic syndromes [J].
Caocci, Giovanni ;
La Nasa, Giorgio ;
Efficace, Fabio .
EXPERT REVIEW OF HEMATOLOGY, 2009, 2 (01) :69-80
[8]   Assessing calibration of prognostic risk scores [J].
Crowson, Cynthia S. ;
Atkinson, Elizabeth J. ;
Therneau, Terry M. .
STATISTICAL METHODS IN MEDICAL RESEARCH, 2016, 25 (04) :1692-1706
[9]  
Deschler B, 2006, HAEMATOLOGICA, V91, P1513
[10]   Descriptive and prognostic value of patient-reported outcomes: The bortezomib experience in relapsed and refractory multiple myeloma [J].
Dubois, D ;
Dhawan, R ;
van de Velde, H ;
Esseltine, D ;
Gupta, S ;
Viala, M ;
de la Loge, C .
JOURNAL OF CLINICAL ONCOLOGY, 2006, 24 (06) :976-982