Patient-reported outcomes enhance the survival prediction of traditional disease risk classifications: An international study in patients with myelodysplastic syndromes

Fabio Efficace; Francesco Cottone; Gregory Abel; Pasquale Niscola; Gianluca Gaidano; Franck Bonnetain; Amelie Anota; Giovanni Caocci; Angel Cronin; Luana Fianchi; Massimo Breccia; Reinhard Stauder; Uwe Platzbecker; Giuseppe A Palumbo; Mario Luppi; Rosangela Invernizzi; Micaela Bergamaschi; Lorenza Borin; Anna Angela Di Tucci; Huiyong Zhang; Mirjam Sprangers; Marco Vignetti; Franco Mandelli

doi:10.1002/cncr.31193

Patient-reported outcomes enhance the survival prediction of traditional disease risk classifications: An international study in patients with myelodysplastic syndromes

Cancer. 2018 Mar 15;124(6):1251-1259. doi: 10.1002/cncr.31193. Epub 2017 Dec 12.

Authors

Fabio Efficace¹, Francesco Cottone¹, Gregory Abel², Pasquale Niscola³, Gianluca Gaidano⁴, Franck Bonnetain^{5

6}, Amelie Anota^{5

6}, Giovanni Caocci⁷, Angel Cronin², Luana Fianchi⁸, Massimo Breccia⁹, Reinhard Stauder¹⁰, Uwe Platzbecker¹¹, Giuseppe A Palumbo¹², Mario Luppi¹³, Rosangela Invernizzi¹⁴, Micaela Bergamaschi¹⁵, Lorenza Borin¹⁶, Anna Angela Di Tucci¹⁷, Huiyong Zhang¹⁸, Mirjam Sprangers¹⁹, Marco Vignetti¹, Franco Mandelli¹

Affiliations

¹ Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
² Division of Population Sciences, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts.
³ Hematology Unit, Sant'Eugenio Hospital, Rome, Italy.
⁴ Division of Hematology, Department of Translational Medicine, University of Eastern Piedmont, Novara, Italy.
⁵ Methodology and Quality of Life in Oncology Unit, University Hospital of Besançon, Besançon, France.
⁶ Platform Quality of Life and Cancer, INSERM 1098, University of Franche-Comté, Besançon, France.
⁷ Department of Medical Sciences, University of Cagliari, Cagliari, Italy.
⁸ Institute of Hematology, Catholic University of the Sacred Heart, Rome, Italy.
⁹ Division of Hematology, Department of Cellular Biotechnologies and Hematology, Sapienza University, Rome, Italy.
¹⁰ Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria.
¹¹ Department of Medicine I, University Hospital Dresden Carl Gustav Carus, Dresden, Germany.
¹² UO Ematologia, AOU Policlinico-V Emanuele, Catania, Italy.
¹³ Department of Hematology, University of Modena, Modena, Italy.
¹⁴ Department of Internal Medicine, University of Pavia, San Matteo IRCCS Policlinic Foundation, Pavia, Italy.
¹⁵ San Martino Clinical Hematology Clinic, Genova, Italy.
¹⁶ Department of Hematology, San Gerardo Hospital, Monza, Italy.
¹⁷ Hematology and Bone Marrow Transplantation Unit, Armando Businco Hospital, Cagliari, Italy.
¹⁸ Department of Hematology, Affiliated Hospital of Liaoning University of Traditional Chinese Medicine, Shenyang, China.
¹⁹ Department of Medical Psychology, Academic Medical Center/University of Amsterdam, Amsterdam, the Netherlands.

PMID: 29231969
DOI: 10.1002/cncr.31193

Abstract

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. Cancer 2018;124:1251-9. © 2017 American Cancer Society.

Keywords: fatigue; myelodysplastic syndromes; patient-reported outcomes; quality of life; survival.

Publication types

Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Aged
Aged, 80 and over
Combined Modality Therapy
Female
Follow-Up Studies
Humans
International Agencies
Male
Middle Aged
Myelodysplastic Syndromes / mortality*
Myelodysplastic Syndromes / pathology
Myelodysplastic Syndromes / therapy
Patient Reported Outcome Measures*
Prognosis
Prospective Studies
Quality of Life*
Risk Factors
Self Report*
Survival Rate