Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib

Fabio Efficace, Fausto Castagnetti, Bruno Martino, Massimo Breccia, Mariella D'Adda, Emanuele Angelucci, Fabio Stagno, Francesco Cottone, Alessandra Malato, Elena Trabacchi, Silvana Franca Capalbo, Marco Gobbi, Giuseppe Visani, Marzia Salvucci, Isabella Capodanno, Patrizia Tosi, Mario Tiribelli, Anna Rita Scortechini, Luciano Levato, Elena MainoGianni Binotto, Gabriele Gugliotta, Marco Vignetti, Michele Baccarani, Gianantonio Rosti

Research output: Contribution to journalArticle

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. © 2018 American Cancer Society.

Original languageEnglish
Pages (from-to)2228-2237
Number of pages10
JournalCancer
Volume124
Issue number10
DOIs
Publication statusPublished - May 15 2018

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Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Quality of Life
Fatigue
Therapeutics
4-methyl-N-(3-(4-methylimidazol-1-yl)-5-(trifluoromethyl)phenyl)-3-((4-pyridin-3-ylpyrimidin-2-yl)amino)benzamide
Leukemia, Myeloid, Chronic Phase
Quality Improvement
Secondary Prevention
Protein-Tyrosine Kinases
Multicenter Studies
Longitudinal Studies
Neoplasms
Odds Ratio
Organizations
Prospective Studies
Confidence Intervals
Safety

Cite this

Efficace, F., Castagnetti, F., Martino, B., Breccia, M., D'Adda, M., Angelucci, E., ... Rosti, G. (2018). Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib. Cancer, 124(10), 2228-2237. https://doi.org/10.1002/cncr.31323

Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib. / Efficace, Fabio; Castagnetti, Fausto; Martino, Bruno; Breccia, Massimo; D'Adda, Mariella; Angelucci, Emanuele; Stagno, Fabio; Cottone, Francesco; Malato, Alessandra; Trabacchi, Elena; Capalbo, Silvana Franca; Gobbi, Marco; Visani, Giuseppe; Salvucci, Marzia; Capodanno, Isabella; Tosi, Patrizia; Tiribelli, Mario; Scortechini, Anna Rita; Levato, Luciano; Maino, Elena; Binotto, Gianni; Gugliotta, Gabriele; Vignetti, Marco; Baccarani, Michele; Rosti, Gianantonio.

In: Cancer, Vol. 124, No. 10, 15.05.2018, p. 2228-2237.

Research output: Contribution to journalArticle

Efficace, F, Castagnetti, F, Martino, B, Breccia, M, D'Adda, M, Angelucci, E, Stagno, F, Cottone, F, Malato, A, Trabacchi, E, Capalbo, SF, Gobbi, M, Visani, G, Salvucci, M, Capodanno, I, Tosi, P, Tiribelli, M, Scortechini, AR, Levato, L, Maino, E, Binotto, G, Gugliotta, G, Vignetti, M, Baccarani, M & Rosti, G 2018, 'Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib', Cancer, vol. 124, no. 10, pp. 2228-2237. https://doi.org/10.1002/cncr.31323
Efficace, Fabio ; Castagnetti, Fausto ; Martino, Bruno ; Breccia, Massimo ; D'Adda, Mariella ; Angelucci, Emanuele ; Stagno, Fabio ; Cottone, Francesco ; Malato, Alessandra ; Trabacchi, Elena ; Capalbo, Silvana Franca ; Gobbi, Marco ; Visani, Giuseppe ; Salvucci, Marzia ; Capodanno, Isabella ; Tosi, Patrizia ; Tiribelli, Mario ; Scortechini, Anna Rita ; Levato, Luciano ; Maino, Elena ; Binotto, Gianni ; Gugliotta, Gabriele ; Vignetti, Marco ; Baccarani, Michele ; Rosti, Gianantonio. / Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib. In: Cancer. 2018 ; Vol. 124, No. 10. pp. 2228-2237.
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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. {\circledC} 2018 American Cancer Society.",
author = "Fabio Efficace and Fausto Castagnetti and Bruno Martino and Massimo Breccia and Mariella D'Adda and Emanuele Angelucci and Fabio Stagno and Francesco Cottone and Alessandra Malato and Elena Trabacchi and Capalbo, {Silvana Franca} and Marco Gobbi and Giuseppe Visani and Marzia Salvucci and Isabella Capodanno and Patrizia Tosi and Mario Tiribelli and Scortechini, {Anna Rita} and Luciano Levato and Elena Maino and Gianni Binotto and Gabriele Gugliotta and Marco Vignetti and Michele Baccarani and Gianantonio Rosti",
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TY - JOUR

T1 - Health-related quality of life in patients with chronic myeloid leukemia receiving first-line therapy with nilotinib

AU - Efficace, Fabio

AU - Castagnetti, Fausto

AU - Martino, Bruno

AU - Breccia, Massimo

AU - D'Adda, Mariella

AU - Angelucci, Emanuele

AU - Stagno, Fabio

AU - Cottone, Francesco

AU - Malato, Alessandra

AU - Trabacchi, Elena

AU - Capalbo, Silvana Franca

AU - Gobbi, Marco

AU - Visani, Giuseppe

AU - Salvucci, Marzia

AU - Capodanno, Isabella

AU - Tosi, Patrizia

AU - Tiribelli, Mario

AU - Scortechini, Anna Rita

AU - Levato, Luciano

AU - Maino, Elena

AU - Binotto, Gianni

AU - Gugliotta, Gabriele

AU - Vignetti, Marco

AU - Baccarani, Michele

AU - Rosti, Gianantonio

N1 - © 2018 American Cancer Society.

PY - 2018/5/15

Y1 - 2018/5/15

N2 - 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. © 2018 American Cancer Society.

AB - 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. © 2018 American Cancer Society.

U2 - 10.1002/cncr.31323

DO - 10.1002/cncr.31323

M3 - Article

C2 - 29499087

VL - 124

SP - 2228

EP - 2237

JO - Cancer

JF - Cancer

SN - 0008-543X

IS - 10

ER -