Fabio Efficace1, Fausto Castagnetti2, Bruno Martino3, Massimo Breccia4, Mariella D'Adda5, Emanuele Angelucci6, Fabio Stagno7, Francesco Cottone1, Alessandra Malato8, Elena Trabacchi9, Silvana Franca Capalbo10, Marco Gobbi11, Giuseppe Visani12, Marzia Salvucci13, Isabella Capodanno14, Patrizia Tosi15, Mario Tiribelli16, Anna Rita Scortechini17, Luciano Levato18, Elena Maino19, Gianni Binotto20, Gabriele Gugliotta2, Marco Vignetti1, Michele Baccarani2, Gianantonio Rosti2. 1. Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases, Rome, Italy. 2. L. and A. Seràgnoli Institute of Hematology, Department of Experimental, Diagnostic, and Specialty Medicine, S. Orsola-Malpighi University Hospital, University of Bologna, Bologna, Italy. 3. Hematology Unit, Bianchi-Melacrino-Morelli Azienda Ospedaliera, Reggio Calabria, Italy. 4. Department of Cellular Biotechnologies and Hematology, Sapienza University of Rome, Rome, Italy. 5. Hematology Unit, Spedali Civili Azienda Ospedaliera, Brescia, Italy. 6. Ospedale Policlinico San Martino, Genoa, Italy. 7. Department of Hematology, University of Catania, Catania, Italy. 8. Department of Hematology, Hospital Cervello, Palermo, Italy. 9. Hematology and Bone Marrow Transplantation Unit, Department of Hematology and Oncology, G. da Saliceto Hospital, Piacenza, Italy. 10. Hematology Unit, Azienda Ospedaliero-Universitaria Ospedali Riuniti, Foggia, Italy. 11. Clinical Hematology, Ospedale Policlinico S. Martino, Istituto di Ricovero e Cura a Carattere Scientifico, Genoa, Italy. 12. Hematology and Stem Cell Transplantation Unit, Azienda Ospedaliera Ospedali Riuniti Marche Nord, Pesaro, Italy. 13. Hematology Unit, Santa Maria delle Croci Hospital, Ravenna, Italy. 14. Hematology Unit, Arcispedale Santa Maria Nuova, Istituto di Ricovero e Cura a Carattere Scientifico, Reggio Emilia, Italy. 15. Hematology Unit, Infermi Hospital Rimini, Rimini, Italy. 16. Division of Hematology and Bone Marrow Transplantation, Department of Experimental and Clinical Medical Sciences, Azienda Ospedaliero-Universitaria di Udine, Udine, Italy. 17. Clinical Hematology Laboratory, Department of Molecular and Clinical Sciences, Polytechnic University of Marche, Ancona, Italy. 18. Hematology Unit, Pugliese-Ciaccio Hospital, Catanzaro, Italy. 19. Hematology Unit, Dell'Angelo Hospital, Venezia-Mestre, Italy. 20. Hematology and Clinical Immunology, Department of Medicine, Padua School of Medicine, Padua, Italy.
Abstract
BACKGROUND: Although a wealth of efficacy and safety data is available for many tyrosine kinase inhibitors used in chronic myeloid leukemia (CML), there is a dearth of information on their impact on patients' health-related quality of life (HRQOL). The primary objective of this study was to evaluate HRQOL and fatigue outcomes in patients with CML receiving first-line therapy with nilotinib. METHODS: This was a multicenter, prospective study enrolling 130 patients with chronic-phase CML. HRQOL and fatigue were evaluated with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and its validated Fatigue module at the baseline and then at 3, 6, 12, 18, and 24 months. The primary prespecified HRQOL endpoints defined in the study protocol for longitudinal analysis were the Physical Functioning, Social Functioning, Role Functioning, and Fatigue scales. The remaining scales were investigated on an exploratory basis. RESULTS: The rate of baseline compliance with the HRQOL assessment was 95.4% (124 of 130), and the rate of overall compliance with HRQOL forms was 91%. Among the 4 prespecified primary HRQOL endpoints, statistically significant improvements over time were found for Physical Functioning (P = .013), Role Functioning (P = .004), and Fatigue (P < .001). Clinically meaningful improvements were found already 3 months after the treatment start. The baseline patient self-reported fatigue severity was an independent predictive factor for the achievement of a major molecular response with an odds ratio of 0.960 (95% confidence interval, 0.934-0.988; P = .005). CONCLUSIONS: For most patients, HRQOL improvements with nilotinib occur during the early phase of therapy and are maintained over time. Also, a more systematic HRQOL evaluation during the diagnostic workup of CML may help to predict clinical outcomes. Cancer 2018;124:2228-37.
BACKGROUND: Although a wealth of efficacy and safety data is available for many tyrosine kinase inhibitors used in chronic myeloid leukemia (CML), there is a dearth of information on their impact on patients' health-related quality of life (HRQOL). The primary objective of this study was to evaluate HRQOL and fatigue outcomes in patients with CML receiving first-line therapy with nilotinib. METHODS: This was a multicenter, prospective study enrolling 130 patients with chronic-phase CML. HRQOL and fatigue were evaluated with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and its validated Fatigue module at the baseline and then at 3, 6, 12, 18, and 24 months. The primary prespecified HRQOL endpoints defined in the study protocol for longitudinal analysis were the Physical Functioning, Social Functioning, Role Functioning, and Fatigue scales. The remaining scales were investigated on an exploratory basis. RESULTS: The rate of baseline compliance with the HRQOL assessment was 95.4% (124 of 130), and the rate of overall compliance with HRQOL forms was 91%. Among the 4 prespecified primary HRQOL endpoints, statistically significant improvements over time were found for Physical Functioning (P = .013), Role Functioning (P = .004), and Fatigue (P < .001). Clinically meaningful improvements were found already 3 months after the treatment start. The baseline patient self-reported fatigue severity was an independent predictive factor for the achievement of a major molecular response with an odds ratio of 0.960 (95% confidence interval, 0.934-0.988; P = .005). CONCLUSIONS: For most patients, HRQOL improvements with nilotinib occur during the early phase of therapy and are maintained over time. Also, a more systematic HRQOL evaluation during the diagnostic workup of CML may help to predict clinical outcomes. Cancer 2018;124:2228-37.
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