Health state utility estimation of Mycobacterium avium complex pulmonary disease using a time trade-off approach

References

• Diel R, Lipman M, Hoefsloot W. High mortality in patients with Mycobacterium avium complex lung disease: a systematic review. BMC Infect Dis. 2018;18(1):206.
   PubMed Web of Science ®Google Scholar
• Furuuchi K, Morimoto K, Yoshiyama T, et al. Interrelational changes in the epidemiology and clinical features of nontuberculous mycobacterial pulmonary disease and tuberculosis in a referral hospital in Japan. Respir Med. 2019;152:74–80.
   PubMed Web of Science ®Google Scholar
• Izumi K, Morimoto K, Hasegawa N, et al. Epidemiology of adults and children treated for nontuberculous mycobacterial pulmonary disease in Japan. Ann Am Thorac Soc. 2018;16:341–347.
   Web of Science ®Google Scholar
• Marras TK, Mendelson D, Marchand-Austin A, et al. Pulmonary nontuberculous mycobacterial disease, Ontario, Canada, 1998–2010. Emerg Infect Dis. 2013;19:1889–1891.
   PubMed Web of Science ®Google Scholar
• Namkoong H, Kurashima A, Morimoto K, et al. Epidemiology of pulmonary nontuberculous mycobacterial disease, Japan. Emerg Infect Dis. 2016;22(6):1116–1117.
   PubMed Web of Science ®Google Scholar
• Morimoto K, Iwai K, Uchimura K, et al. A steady increase in nontuberculous mycobacteriosis mortality and estimated prevalence in Japan. Ann Am Thorac Soc. 2014;11(1):1–8.
   PubMedGoogle Scholar
• Ito Y, Hirai T, Fujita K, et al. Increasing patients with pulmonary Mycobacterium avium complex disease and associated underlying diseases in Japan. J Infect Chemother. 2015;21(5):352–356.
   PubMed Web of Science ®Google Scholar
• Morimoto K, Hasegawa N, Izumi K, et al. A laboratory-based analysis of nontuberculous mycobacterial lung disease in Japan from 2012 to 2013. Ann Am Thorac Soc. 2017;14(1):49–56.
   PubMed Web of Science ®Google Scholar
• Daley CL, Iaccarino JM, Lange C, et al. Treatment of nontuberculous mycobacterial pulmonary disease: an official ATS/ERS/ESCMID/IDSA clinical practice guideline. Eur Respir J. 2020;56:2000535.
   PubMed Web of Science ®Google Scholar
• Maekawa K, Ito Y, Oga T, et al. High-resolution computed tomography and health-related quality of life in Mycobacterium avium complex disease. Int J Tuberc Lung Dis. 2013;17(6):829–835.
   PubMed Web of Science ®Google Scholar
• Diel R, Nienhaus A, Ringshausen FC, et al. Microbiologic outcome of interventions against mycobacterium avium complex pulmonary disease: a systematic review. Chest. 2018;153(4):888–921.
   PubMed Web of Science ®Google Scholar
• Lee BY, Kim S, Hong Y, et al. Risk factors for recurrence after successful treatment of Mycobacterium avium complex lung disease. Antimicrob Agents Chemother. 2015;59(6):2972–2977.
   PubMed Web of Science ®Google Scholar
• van Ingen J, Obradovic M, Hassan M, et al. Nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex – disease burden, unmet needs, and advances in treatment developments. Expert Rev Respir Med. 2021;15(11):1387–1401.
   PubMed Web of Science ®Google Scholar
• Aliberti S, Sotgiu G, Castellotti P, et al. Real-life evaluation of clinical outcomes in patients undergoing treatment for non-tuberculous mycobacteria lung disease: a ten-year cohort study. Respir Med. 2020;164:105899.
   PubMed Web of Science ®Google Scholar
• Boyle DP, Zembower TR, Qi C. Relapse versus reinfection of Mycobacterium avium complex pulmonary disease. Patient characteristics and macrolide susceptibility. Ann Am Thorac Soc. 2016;13(11):1956–1961.
   PubMed Web of Science ®Google Scholar
• Kumar K, Daley CL, Griffith DE, et al. Management of Mycobacterium avium complex and Mycobacterium abscessus pulmonary disease: therapeutic advances and emerging treatments. Eur Respir Rev. 2022;31:210212.
   PubMed Web of Science ®Google Scholar
• Mehta M, Marras TK. Impaired health-related quality of life in pulmonary nontuberculous mycobacterial disease. Respir Med. 2011;105:1718–1725.
   PubMed Web of Science ®Google Scholar
• van Ingen J, Wagner D, Gallagher J, et al. Poor adherence to management guidelines in nontuberculous mycobacterial pulmonary diseases. Eur Respir J. 2017;49:1601855.
   PubMed Web of Science ®Google Scholar
• Griffith DE, Eagle G, Thomson R, CONVERT Study Group, et al. Amikacin liposome inhalation suspension for treatment-refractory lung disease caused by Mycobacterium avium complex (CONVERT). a prospective, open-label, randomized study. Am J Respir Crit Care Med. 2018;198(12):1559–1569.
   PubMed Web of Science ®Google Scholar
• Insmed Incorporated. ARIKAYCE® (amikacin liposome inhalation suspension) for oral inhalation use [prescribing information]. Bridgewater (NJ): Insmed Incorporated; 2018. [cited 2022 Oct 26]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/207356s000lbl.pdf
   Google Scholar
• Winthrop KL, Flume PA, Thomson R, et al. Amikacin liposome inhalation suspension for Mycobacterium avium complex lung disease: a 12-month open-label extension clinical trial. Ann Am Thorac Soc. 2021;18(7):1147–1157.
   PubMed Web of Science ®Google Scholar
• Cho S, Kim H, Kim SH, et al. Utility estimation of hypothetical chronic obstructive pulmonary disease health states by the general population and health professionals. Health Qual Life Outcomes. 2015;13:34.
   PubMed Web of Science ®Google Scholar
• Rowen D, Mulhern B, Banerjee S, et al. Comparison of general population, patient, and carer utility values for dementia health states. Med Decis Making. 2015;35(1):68–80.
   PubMed Web of Science ®Google Scholar
• Shah A, Ng X, Shah R, et al. Psychometric validation of the EQ-5D-3L in patients with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC). Patient Relat Outcome Meas. 2021;12:45–54.
   PubMed Web of Science ®Google Scholar
• Center for Outcomes Research and Economic Evaluation for Health National Institutes of Public Health C2H. Guideline for preparing cost-effectiveness evaluations to the central social insurance medical council. Tokyo, Japan: National Institute of Public Health (C2H); 2022. Available from: https://c2h.niph.go.jp/tools/guideline/guideline_en.pdf
   Google Scholar
• Matza LS, Stewart KD, Lloyd AJ, et al. Vignette-based utilities: usefulness, limitations, and methodological recommendations. Value Health. 2021;24(6):812–821.
   PubMed Web of Science ®Google Scholar
• Hall F, de Freitas HM, Kerr C, et al. Estimating utilities/disutilities for high-risk metastatic hormone-sensitive prostate cancer (mHSPC) and treatment-related adverse events. Qual Life Res. 2019;28(5):1191–1199.
   PubMed Web of Science ®Google Scholar
• Lloyd A, Piglowska N, Ciulla T, et al. Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy. Br J Ophthalmol. 2019;103(11):1610–1614.
   PubMed Web of Science ®Google Scholar
• Matza LS, Deger KA, Vo P, et al. Health state utilities associated with attributes of migraine preventive treatments based on patient and general population preferences. Qual Life Res. 2019;28(9):2359–2372.
   PubMed Web of Science ®Google Scholar
• Insmed Incorporated. CONVERT trial: data on file. Bridgewater (NJ): Insmed Incorporated; 2021.
   Google Scholar
• Leitman A. NTM lung disease: patient experiences and preferences. FDA public workshop: development of antibacterial drugs for treatment of nontuberculous mycobacterial disease. Silver Spring (MD): U. S. Food and Drug; 2019. [cited 2022 Sept 1]. Available from: https://www.fda.gov/media/124058/download
   Google Scholar
• Pravosud V, Mannino DM, Prieto D, et al. Symptom burden and medication use among patients with nontuberculous mycobacterial lung disease. Chronic Obstr Pulm Dis. 2021;8(2):243–254.
   PubMed Web of Science ®Google Scholar
• Devlin NJ, Tsuchiya A, Buckingham K, et al. A uniform time trade off method for states better and worse than dead: feasibility study of the ‘lead time’ approach. Health Econ. 2011;20:348–361.
   PubMed Web of Science ®Google Scholar
• Jones PW, Quirk FH, Baveystock CM, et al. A self-complete measure of health status for chronic airflow limitation. The St. George’s Respiratory Questionnaire. Am Rev Respir Dis. 1992;145:1321–1327.
   PubMed Web of Science ®Google Scholar
• Saint George’s University of London. St. George’s Respiratory Questionnaire. 2022 [cited 2022 Dec 11]. Available from: https://www.sgul.ac.uk/research/research-operations/research-administration/st-georges-respiratory-questionnaire/sgrq
   Google Scholar
• Ogawa T, Asakura T, Suzuki S, et al. Longitudinal validity and prognostic significance of the St George’s Respiratory Questionnaire in Mycobacterium avium complex pulmonary disease. Respir Med. 2021;185:106515.
   PubMed Web of Science ®Google Scholar
• Hajiro T, Nishimura K, Tsukino M, et al. Comparison of discriminative properties among disease-specific questionnaires for measuring health-related quality of life in patients with chronic obstructive pulmonary disease. Am J Respir Crit Care Med. 1998;157:785–790.
   PubMed Web of Science ®Google Scholar
• Mukuria C, Rowen D, Brazier JE, et al. Deriving a preference-based measure for myelofibrosis from the EORTC QLQ-C30 and the MF-SAF. Value Health. 2015;18:846–855.
   PubMed Web of Science ®Google Scholar
• Mulhern B, Rowen D, Jacoby A, et al. The development of a QALY measure for epilepsy: NEWQOL-6D. Epilepsy Behav. 2012;24:36–43.
   PubMed Web of Science ®Google Scholar
• Rowen D, Mulhern B, Banerjee S, et al. Estimating preference-based single index measures for dementia using DEMQOL and DEMQOL-Proxy. Value Health. 2012;15:346–356.
   PubMed Web of Science ®Google Scholar
• Ministry of Education Culture, Sports, Science and Technology, Ministry of Health, Labour and Welfare, and Ministry of Economy, Trade and Industry. Ethical guidelines for medical and biological research involving human subjects (in Japanese). Tokyo: Ministry of Education, Culture, Sports, Science and Technology, Ministry of Health, Labour and Welfare; 2022. [cited 2022 Jan 4]. Available from: https://www.mhlw.go.jp/content/000757566.pdf
   Google Scholar
• Japan Statistics Bureau of the Ministry of Internal Affairs and Communications. Table 2–1. Population by male and female, age/education, employment status/jobs (15 years old and over) – Japan. e-Stat Japan in statistics. Tokyo: National Statistics Center; 2017. [cited 2022 Sept 28]. Available from: https://www.e-stat.go.jp/stat-search/files?page=1&layout=datalist&toukei=00200532&tstat=000001107875&cycle=0&tclass1=000001107876&tclass2=000001107877&tclass3val=0
   Google Scholar
• Asakura T, Funatsu Y, Ishii M, et al. Health-related quality of life is inversely correlated with C-reactive protein and age in Mycobacterium avium complex lung disease: a cross-sectional analysis of 235 patients. Respir Res. 2015;16:145.
   PubMed Web of Science ®Google Scholar
• Kawahara K, Tabusadani M, Yamane K, et al. Health-related quality of life associates with clinical parameters in patients with NTM pulmonary disease. Int J Tuberc Lung Dis. 2021;25:299–304.
   PubMed Web of Science ®Google Scholar
• Yagi K, Asakura T, Namkoong H, et al. Association between six-minute walk test parameters and the health-related quality of life in patients with pulmonary Mycobacterium avium complex disease. BMC Pulm Med. 2018;18:114.
   PubMed Web of Science ®Google Scholar
• Fukushima K, Kitada S, Komukai S, et al. First line treatment selection modifies disease course and long-term clinical outcomes in Mycobacterium avium complex pulmonary disease. Sci Rep. 2021;11:1178.
   PubMed Web of Science ®Google Scholar
• Hong JY, Lee SA, Kim SY, et al. Factors associated with quality of life measured by EQ-5D in patients with nontuberculous mycobacterial pulmonary disease. Qual Life Res. 2014;23:2735–2741.
   PubMed Web of Science ®Google Scholar
• Efthymiadou O, Mossman J, Kanavos P. Health related quality of life aspects not captured by EQ-5D-5L: results from an international survey of patients. Health Policy. 2019;123(2):159–165.
   PubMed Web of Science ®Google Scholar
• Marras TK, Mirsaeidi M, Vinnard C, et al. Guidelines-based treatment associated with improved economic outcomes in nontuberculous mycobacterial lung disease. J Med Econ. 2019;22:1126–1133.
   PubMed Web of Science ®Google Scholar
• Ringshausen FC, Wagner D, de Roux A, et al. Prevalence of nontuberculous mycobacterial pulmonary disease, Germany, 2009–2014. Emerg Infect Dis. 2016;22:1102–1105.
   PubMed Web of Science ®Google Scholar
• Mahlich J, Dilokthornsakul P, Sruamsiri R, et al. Cultural beliefs, utility values, and health technology assessment. Cost Eff Resour Alloc. 2018;16:19.
   PubMed Web of Science ®Google Scholar
• Purba FD, Hunfeld JAM, Iskandarsyah A, et al. Employing quality control and feedback to the EQ-5D-5L valuation protocol to improve the quality of data collection. Qual Life Res. 2017;26:1197–1208.
   PubMed Web of Science ®Google Scholar
• Ramos-Goñi JM, Oppe M, Slaap B, et al. Quality control process for EQ-5D-5L valuation studies. Value Health. 2017;20:466–473.
   PubMed Web of Science ®Google Scholar
• Insmed I. CONVERT trial: data on file. Bridgewater (NJ): Insmed Incorporated; 2021.
   Google Scholar
• Japan MoHLaW. [Comprehensive Survey of Living Conditions 2019] (Japanese). Tokyo: Director General for Statistics and Information Policy; 2020 [cited 26 Oct 2022]. Available from: https://www.mhlw.go.jp/toukei/saikin/hw/k-tyosa/k-tyosa19/index.html
   Google Scholar
• Ministry oIAaC. Final report of the 2015 population and households of Japan. Tokyo: Director General Statistics Bureau; 2018 [cited 26 Oct 2022]. Available from: https://www.stat.go.jp/english/data/kokusei/2015/final_en/final_en.html#Summary
   Google Scholar
• Ministry oIAaC. Annual report on the labour force survey 2017. Tokyo; 2017 [cited 26 Oct 2022]. Available from: https://www.stat.go.jp/english/data/roudou/report/2017/index.html
   Google Scholar
• Organisation fEC-oaDO. OECD. OECD Reviews of public health: Japan: a healthier tomorrow. Paris: OECD Publishing; 2019 [cited 26 Oct 2022]. Available from: https://doi.org/10.1787/9789264311602-en
   Google Scholar