Health state utility and quality of life measures in patients with chronic myeloid leukemia in France.
Chronic myeloid leukemia
Health utility score
Quality of life
Tyrosine kinase inhibitor
Journal
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
ISSN: 1573-2649
Titre abrégé: Qual Life Res
Pays: Netherlands
ID NLM: 9210257
Informations de publication
Date de publication:
Jul 2021
Jul 2021
Historique:
accepted:
08
02
2021
pubmed:
3
3
2021
medline:
24
7
2021
entrez:
2
3
2021
Statut:
ppublish
Résumé
Tyrosine kinase inhibitors (TKIs) have dramatically improved the prognosis of chronic myeloid leukemia (CML). We aimed to assess health state utility and quality of life (QoL) in French patients with CML in real-life setting, to study the determinants of utility score and to compare health-related QoL values to general population norms. We conducted a cross-sectional study in 412 patients with CML. Data were collected by electronic survey. Three patient-reported outcomes questionnaires were used: EORTC QLQ-C30, EORTC QLQ-CML24 and EuroQol EQ-5D-3L. Health state utility values were computed using the French value set. We computed deviations from reference norms from the general population. We studied the determinants of health utility score using multiple regression models. The mean utility score (SD) was 0.72 (0.25) in the chronic phase and 0.84 (0.21) in treatment-free remission, with marked variations by gender. Patients with CML had a deviation from the reference norm of -0.15 on average (SD: 0.25). In terms of QoL, social functioning, role functioning and cognitive functioning were notably impacted with a mean difference of -16.0, -13.1 and -11.7 respectively. Fatigue, dyspnea and pain were the symptoms with the highest deviation from general population norms (mean difference of 20.6, 14.0 and 8.3 respectively). In the multiple regression analysis, fatigue was the most important independent predictor of the utility score. Although TKIs prevent the disease from progressing and even allow remission without treatment, QoL in patients with CML is notably altered. The utility scores deteriorate with CML symptoms.
Identifiants
pubmed: 33651280
doi: 10.1007/s11136-021-02794-5
pii: 10.1007/s11136-021-02794-5
doi:
Substances chimiques
4-amino-5-(4-methylphenyl)-7-(tert-butyl)pyrazolo(3,4-d)pyrimidine
0
Pyrazoles
0
Pyrimidines
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2021-2032Subventions
Organisme : Agence Nationale de la Recherche
ID : ANR-10-IBHU-0001
Références
Apperley, J. F. (2015). Chronic myeloid leukaemia. Lancet (London, England), 385(9976), 1447–1459. https://doi.org/10.1016/S0140-6736(13)62120-0 .
doi: 10.1016/S0140-6736(13)62120-0
Le Guyader-Peyrou, S., Belot, A., Maynadié, M., Binder-Foucard, F., Remontet, L., Troussard, X., et al. (2016). Cancer incidence in France over the 1980–2012 period: Hematological malignancies. Revue D’epidemiologie Et De Sante Publique, 64(2), 103–112. https://doi.org/10.1016/j.respe.2015.12.017 .
doi: 10.1016/j.respe.2015.12.017
pubmed: 26973179
Höglund, M., Sandin, F., & Simonsson, B. (2015). Epidemiology of chronic myeloid leukaemia: An update. Annals of Hematology, 94(Suppl 2), S241-247. https://doi.org/10.1007/s00277-015-2314-2 .
doi: 10.1007/s00277-015-2314-2
pubmed: 25814090
Foulon, S., Cony-Makhoul, P., Guerci-Bresler, A., Delord, M., Solary, E., Monnereau, A., et al. (2019). Using healthcare claims data to analyze the prevalence of BCR-ABL-positive chronic myeloid leukemia in France: A nationwide population-based study. Cancer Medicine, 8(6), 3296–3304. https://doi.org/10.1002/cam4.2200 .
doi: 10.1002/cam4.2200
pubmed: 31038849
pmcid: 6558491
Bower, H., Björkholm, M., Dickman, P. W., Höglund, M., Lambert, P. C., & Andersson, T.M.-L. (2016). Life expectancy of patients with chronic myeloid leukemia approaches the life expectancy of the general population. Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology, 34(24), 2851–2857. https://doi.org/10.1200/JCO.2015.66.2866 .
doi: 10.1200/JCO.2015.66.2866
Mahon, F.-X. (2017). Treatment-free remission in CML: Who, how, and why? Hematology American Society of Hematology. Education Program, 2017(1), 102–109. https://doi.org/10.1182/asheducation-2017.1.102 .
doi: 10.1182/asheducation-2017.1.102
pubmed: 29222243
pmcid: 6142562
Rea, D., Ame, S., Berger, M., Cayuela, J.-M., Charbonnier, A., Coiteux, V., et al. (2018). Discontinuation of tyrosine kinase inhibitors in chronic myeloid leukemia: Recommendations for clinical practice from the French Chronic Myeloid Leukemia Study Group. Cancer, 124(14), 2956–2963. https://doi.org/10.1002/cncr.31411 .
doi: 10.1002/cncr.31411
pubmed: 29723417
Efficace, F., & Cannella, L. (2016). The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors. Hematology American Society of Hematology Education Program, 2016(1), 170–179. https://doi.org/10.1182/asheducation-2016.1.170 .
doi: 10.1182/asheducation-2016.1.170
pubmed: 27913477
pmcid: 6142524
Hahn, E. A., Glendenning, G. A., Sorensen, M. V., Hudgens, S. A., Druker, B. J., Guilhot, F., et al. (2003). Quality of life in patients with newly diagnosed chronic phase chronic myeloid leukemia on imatinib versus interferon alfa plus low-dose cytarabine: Results from the IRIS Study. Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology, 21(11), 2138–2146. https://doi.org/10.1200/JCO.2003.12.154 .
doi: 10.1200/JCO.2003.12.154
Trask, P. C., Cella, D., Besson, N., Kelly, V., Masszi, T., & Kim, D.-W. (2012). Health-related quality of life of bosutinib (SKI-606) in imatinib-resistant or imatinib-intolerant chronic phase chronic myeloid leukemia. Leukemia Research, 36(4), 438–442. https://doi.org/10.1016/j.leukres.2011.10.011 .
doi: 10.1016/j.leukres.2011.10.011
pubmed: 22036634
Whiteley, J., Reisman, A., Shapiro, M., Cortes, J., & Cella, D. (2016). Health-related quality of life during bosutinib (SKI-606) therapy in patients with advanced chronic myeloid leukemia after imatinib failure. Current Medical Research and Opinion, 32(8), 1325–1334. https://doi.org/10.1185/03007995.2016.1174108 .
doi: 10.1185/03007995.2016.1174108
pubmed: 27045164
Park, J. S., Lee, S.-E., Jeong, S. H., Jang, E.-J., Choi, M.-Y., Kim, H.-J., et al. (2016). Change of health-related profiles after Imatinib cessation in chronic phase chronic myeloid leukemia patients. Leukemia & Lymphoma, 57(2), 341–347. https://doi.org/10.3109/10428194.2015.1049166 .
doi: 10.3109/10428194.2015.1049166
Efficace, F., Castagnetti, F., Martino, B., Breccia, M., D’Adda, M., Angelucci, E., et al. (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 .
doi: 10.1002/cncr.31323
pubmed: 29499087
Efficace, F., Stagno, F., Iurlo, A., Breccia, M., Cottone, F., Bonifacio, M., et al. (2020). Health-related quality of life of newly diagnosed chronic myeloid leukemia patients treated with first-line dasatinib versus imatinib therapy. Leukemia, 34(2), 488–498. https://doi.org/10.1038/s41375-019-0563-0 .
doi: 10.1038/s41375-019-0563-0
pubmed: 31477798
Efficace, F., Baccarani, M., Breccia, M., Alimena, G., Rosti, G., Cottone, F., et al. (2011). Health-related quality of life in chronic myeloid leukemia patients receiving long-term therapy with imatinib compared with the general population. Blood, 118(17), 4554–4560. https://doi.org/10.1182/blood-2011-04-347575 .
doi: 10.1182/blood-2011-04-347575
pubmed: 21750313
Phillips, K. M., Pinilla-Ibarz, J., Sotomayor, E., Lee, M. R., Jim, H. S. L., Small, B. J., et al. (2013). Quality of life outcomes in patients with chronic myeloid leukemia treated with tyrosine kinase inhibitors: A controlled comparison. Supportive Care in Cancer: Official Journal of the Multinational Association of Supportive Care in Cancer, 21(4), 1097–1103. https://doi.org/10.1007/s00520-012-1630-5 .
doi: 10.1007/s00520-012-1630-5
Reed, S. D., Anstrom, K. J., Ludmer, J. A., Glendenning, G. A., & Schulman, K. A. (2004). Cost-effectiveness of imatinib versus interferon-alpha plus low-dose cytarabine for patients with newly diagnosed chronic-phase chronic myeloid leukemia. Cancer, 101(11), 2574–2583. https://doi.org/10.1002/cncr.20694 .
doi: 10.1002/cncr.20694
pubmed: 15493042
Labeit, A. M., Copland, M., Cork, L. M., Hedgley, C., Foroni, L., Osborne, W. L., et al. (2015). Assessment of quality of life in the NCRI spirit 2 study comparing imatinib with dasatinib in patients with newly-diagnosed chronic phase chronic myeloid leukaemia. Blood, 126(23), 4024.
doi: 10.1182/blood.V126.23.4024.4024
Szabo, S. M., Levy, A. R., Davis, C., Holyoake, T. L., & Cortes, J. (2010). A multinational study of health state preference values associated with chronic myelogenous leukemia. Value in Health: The Journal of the International Society for Pharmacoeconomics and Outcomes Research, 13(1), 103–111. https://doi.org/10.1111/j.1524-4733.2009.00573.x .
doi: 10.1111/j.1524-4733.2009.00573.x
Guest, J. F., Naik, N., Sladkevicius, E., Coombs, J., & Gray, E. J. (2012). Utility values for chronic myelogenous leukemia chronic phase health states from the general public in the United Kingdom. Leukemia & Lymphoma, 53(5), 928–933. https://doi.org/10.3109/10428194.2011.631155 .
doi: 10.3109/10428194.2011.631155
Guest, J. F., Gray, E. J., Szczudlo, T., & Magestro, M. (2014). Utility values for specific chronic myeloid leukemia chronic phase health states from the general public in the United Kingdom. Leukemia & Lymphoma, 55(8), 1870–1875. https://doi.org/10.3109/10428194.2013.854886 .
doi: 10.3109/10428194.2013.854886
EuroQol Group. (1990). EuroQol–a new facility for the measurement of health-related quality of life. Health Policy (Amsterdam, Netherlands), 16(3), 199–208.
doi: 10.1016/0168-8510(90)90421-9
Aaronson, N. K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N. J., et al. (1993). The European organization for research and treatment of cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute, 85(5), 365–376.
doi: 10.1093/jnci/85.5.365
Efficace, F., Baccarani, M., Breccia, M., Saussele, S., Abel, G., Caocci, G., et al. (2014). International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: The EORTC QLQ-CML24. Quality of Life Research: An International Journal of Quality of Life Aspects of Treatment, Care and Rehabilitation, 23(3), 825–836. https://doi.org/10.1007/s11136-013-0523-5 .
doi: 10.1007/s11136-013-0523-5
Chevalier, J., & de Pouvourville, G. (2013). Valuing EQ-5D using time trade-off in France. The European Journal of Health Economics: HEPAC: Health Economics in Prevention and Care, 14(1), 57–66. https://doi.org/10.1007/s10198-011-0351-x .
doi: 10.1007/s10198-011-0351-x
pubmed: 21935715
Efficace, F., Iurlo, A., Patriarca, A., Stagno, F., Bee, P.-C., Ector, G., et al. (2020). Validation and reference values of the EORTC QLQ-CML24 questionnaire to assess health-related quality of life in patients with chronic myeloid leukemia. Leukemia & Lymphoma. https://doi.org/10.1080/10428194.2020.1838509 .
doi: 10.1080/10428194.2020.1838509
Szende, A., Janssen, B., & Cabases, J. (2014). Self-reported population health: An international perspective based on EQ-5D. Netherlands: Springer.
doi: 10.1007/978-94-007-7596-1
Liegl, G., Petersen, M. A., Groenvold, M., Aaronson, N. K., Costantini, A., Fayers, P. M., et al. (2019). Establishing the European Norm for the health-related quality of life domains of the computer-adaptive test EORTC CAT Core. European Journal of Cancer (Oxford, England: 1990), 107, 133–141. https://doi.org/10.1016/j.ejca.2018.11.023 .
doi: 10.1016/j.ejca.2018.11.023
Cocks, K., King, M. T., Velikova, G., Martyn St-James, M., Fayers, P. M., & Brown, J. M. (2011). Evidence-based guidelines for determination of sample size and interpretation of the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30. Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology, 29(1), 89–96. https://doi.org/10.1200/JCO.2010.28.0107 .
doi: 10.1200/JCO.2010.28.0107
Coretti, S., Ruggeri, M., & McNamee, P. (2014). The minimum clinically important difference for EQ-5D index: A critical review. Expert Review of Pharmacoeconomics & Outcomes Research, 14(2), 221–233. https://doi.org/10.1586/14737167.2014.894462 .
doi: 10.1586/14737167.2014.894462
Pickard, A. S., Neary, M. P., & Cella, D. (2007). Estimation of minimally important differences in EQ-5D utility and VAS scores in cancer. Health and Quality of Life Outcomes, 5, 70. https://doi.org/10.1186/1477-7525-5-70 .
doi: 10.1186/1477-7525-5-70
pubmed: 18154669
pmcid: 2248572
Kvam, A. K., Fayers, P. M., & Wisloff, F. (2011). Responsiveness and minimal important score differences in quality-of-life questionnaires: A comparison of the EORTC QLQ-C30 cancer-specific questionnaire to the generic utility questionnaires EQ-5D and 15D in patients with multiple myeloma. European Journal of Haematology, 87(4), 330–337. https://doi.org/10.1111/j.1600-0609.2011.01665.x .
doi: 10.1111/j.1600-0609.2011.01665.x
pubmed: 21668504
Kulpeng, W., Sompitak, S., Jootar, S., Chansung, K., & Teerawattananon, Y. (2014). Cost-utility analysis of dasatinib and nilotinib in patients with chronic myeloid leukemia refractory to first-line treatment with imatinib in Thailand. Clinical Therapeutics, 36(4), 534–543. https://doi.org/10.1016/j.clinthera.2014.02.008 .
doi: 10.1016/j.clinthera.2014.02.008
pubmed: 24635968
Arnold, D., Girling, A., Stevens, A., & Lilford, R. (2009). Comparison of direct and indirect methods of estimating health state utilities for resource allocation: Review and empirical analysis. BMJ (Clinical Research Esd.), 339, b2688. https://doi.org/10.1136/bmj.b2688 .
doi: 10.1136/bmj.b2688
Devlin, N. J., & Brooks, R. (2017). EQ-5D and the EuroQol Group: Past, Present and Future. Applied Health Economics and Health Policy, 15(2), 127–137. https://doi.org/10.1007/s40258-017-0310-5 .
doi: 10.1007/s40258-017-0310-5
pubmed: 28194657
pmcid: 5343080
Zulbaran-Rojas, A., Lin, H.-K., Shi, Q., Williams, L. A., George, B., Garcia-Manero, G., et al. (2018). A prospective analysis of symptom burden for patients with chronic myeloid leukemia in chronic phase treated with frontline second- and third-generation tyrosine kinase inhibitors. Cancer Medicine, 7(11), 5457–5469. https://doi.org/10.1002/cam4.1808 .
doi: 10.1002/cam4.1808
pubmed: 30318751
pmcid: 6246941