Health state utilities associated with treatment options for acute myeloid leukemia (AML)

References

• Faderl S, Kantarjian HM. Chapter 59. Clinical manifestations and treatment of acute myeloid leukemia. In: R. Hoffman, editors. Hematology - basic principles and practice. Philadelphia (PA): Elsevier; 2018. p. 2333.
   Google Scholar
• Kantarjian H. Acute myeloid leukemia—major progress over four decades and glimpses into the future. Am J Hematol. 2016;91:131–145.
   PubMed Web of Science ®Google Scholar
• Korol EE, Wang S, Johnston K, et al. Health-related quality of life of patients with acute myeloid leukemia: a systematic literature review. Oncol Ther. 2017;5:1–16.
   PubMed Web of Science ®Google Scholar
• National Cancer Institute and SEER Program. SEER Cancer Stat Facts: Acute Myeloid Leukemia (AML). 2017 [cited 2017 Dec 21]; Available from: http://seer.cancer.gov/statfacts/html/amyl.html.
   Google Scholar
• De Kouchkovsky I, Abdul-Hay M. Acute myeloid leukemia: a comprehensive review and 2016 update. Blood Cancer J. 2016;6:e441.
   PubMed Web of Science ®Google Scholar
• Murphy T, Yee KWL. Cytarabine and daunorubicin for the treatment of acute myeloid leukemia. Expert Opin Pharmacother. 2017;18:1765–1780.
   PubMed Web of Science ®Google Scholar
• Huang LW, Olin RL. Emerging therapeutic modalities for acute myeloid leukemia (AML) in older adults. J Geriatr Oncol. 2017;8:417–420.
   PubMed Web of Science ®Google Scholar
• Perl AE. The role of targeted therapy in the management of patients with AML. Hematology Am Soc Hematol Educ Program. 2017;2017:54–65.
   PubMedGoogle Scholar
• Sallman DA, Lancet JE. What are the most promising new agents in acute myeloid leukemia? Curr Opin Hematol. 2017;24:99–107.
   PubMed Web of Science ®Google Scholar
• Stein EM, Tallman MS. Emerging therapeutic drugs for AML. Blood. 2016;127:71–78.
   PubMed Web of Science ®Google Scholar
• Stone RM. Which new agents will be incorporated into frontline therapy in acute myeloid leukemia? Best Pract Res Clin Haematol. 2017;30:312–316.
   PubMed Web of Science ®Google Scholar
• Wei AH, Tiong IS. Midostaurin, enasidenib, CPX-351, gemtuzumab ozogamicin, and venetoclax bring new hope to AML. Blood. 2017;130:2469–2474.
   PubMed Web of Science ®Google Scholar
• Brunetti C, Anelli L, Zagaria A, et al. CPX-351 in acute myeloid leukemia: can a new formulation maximize the efficacy of old compounds? Expert Rev Hematol. 2017;10:853–862.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Goldberg SL, Feldman EJ, et al. Phase II, multicenter, randomized trial of CPX-351 (cytarabine:daunorubicin) liposome injection versus intensive salvage therapy in adults with first relapse AML. Cancer. 2015;121:234–242.
   PubMed Web of Science ®Google Scholar
• Jazz Pharmaceuticals. Highlights of Prescribing Information: VYXEOS™ (daunorubicin and cytarabine) liposome for injection, for intravenous use. Initial US Approval. Palo Alto (CA): Jazz Pharmaceuticals; 2017. p. 22.
   Google Scholar
• Lancet JE, Cyr P, Sacks N, et al. CPX-351 enables administration of consolidation treatment in the outpatient setting and increases the time spent out of the hospital after completion of AML treatment compared with 7 + 3. Blood. 2015;126:4507.
   Web of Science ®Google Scholar
• Lancet JE, Uy GL, Cortes JE, et al. CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia. JCO. 2018;36:2684–2692.
   PubMed Web of Science ®Google Scholar
• Cressman S, Karsan A, Hogge DE, et al. Economic impact of genomic diagnostics for intermediate-risk acute myeloid leukaemia. Br J Haematol. 2016;174:526–535.
   PubMed Web of Science ®Google Scholar
• Levy AR, Zou D, Risebrough N, et al. Cost-effectiveness in Canada of azacitidine for the treatment of higher-risk myelodysplastic syndromes. Curr Oncol. 2014;21:e29–e40.
   PubMed Web of Science ®Google Scholar
• National Institute for Health and Care Excellence (NICE). Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. Technology appraisal guidance [TA399]. London (UK): NICE; 2016.
   Google Scholar
• Brazier J, Ratcliffe J, Salomon JA, et al. Measuring and valuing health benefits for economic evaluation. 2nd ed. Oxford (UK): Oxford University Press; 2017.
   Google Scholar
• Kurosawa S, Yamaguchi T, Miyawaki S, et al. A Markov decision analysis of allogeneic hematopoietic cell transplantation versus chemotherapy in patients with acute myeloid leukemia in first remission. Blood. 2011;117:2113–2120.
   PubMed Web of Science ®Google Scholar
• Sung L, Buckstein R, Doyle JJ, et al. Treatment options for patients with acute myeloid leukemia with a matched sibling donor: a decision analysis. Cancer. 2003;97:592–600.
   PubMed Web of Science ®Google Scholar
• Kurosawa S, Yamaguchi T, Mori T, et al. Patient-reported quality of life after allogeneic hematopoietic cell transplantation or chemotherapy for acute leukemia. Bone Marrow Transplant. 2015;50:1241–1249.
   PubMedGoogle Scholar
• Leunis A, Redekop WK, Uyl-de Groot CA, et al. Impaired health-related quality of life in acute myeloid leukemia survivors: a single-center study. Eur J Haematol. 2014;93:198–206.
   PubMed Web of Science ®Google Scholar
• Slovacek L, Slovackova B, Jebavy L, et al. Psychosocial, health and demographic characteristics of quality of life among patients with acute myeloid leukemia and malignant lymphoma who underwent autologous hematopoietic stem cell transplantation. Sao Paulo Med J. 2007;125:359–361.
   PubMed Web of Science ®Google Scholar
• Cressman S, Savoie ML, Couban S, et al. Health utility during the first two years of treatment for acute myeloid leukemia and myelodysplastic syndrome. 58th American Society of Hematology Annual Meeting and Exposition. December 3–6, 2016.
   Google Scholar
• National Institute for Health and Care Excellence (NICE). Azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia. NICE technology appraisal guidance 218. London (UK): NICE; 2011. p. 30.
   Google Scholar
• National Institute for Health and Care Excellence (NICE). Lenalidomide for treating myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality. NICE technology appraisal guidance 322. London (UK): NICE; 2014. p. 54.
   Google Scholar
• Uyl-de Groot CA, Lowenberg B, Vellenga E, et al. Health utility during the first two years of treatment for acute myeloid leukemia and myelodysplastic syndrome. Br J Haematol. 1998;100:629–636.
   PubMed Web of Science ®Google Scholar
• Castejon N, Cappelleri JC, Cuervo J, et al. Estimating health state utilities for patients with acute myeloid leukemia (PSY84). International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting; May 20-24, 2017; Boston, MA.
   Google Scholar
• Hensen M, Joshi N, Xu W, et al. Assessment of utility values for treatment-related health states of acute myeloid leukemia in the United Kingdom (PCN157). International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting; May 20-24, 2017. Boston, MA.
   Google Scholar
• National Institute for Health and Care Excellence (NICE). Process and methods guides: Guide to the methods of technology appraisal 2013 (PMG9). London (UK): NICE; 2013. p. 93.
   Google Scholar
• Brennan VK, Dixon S. Incorporating process utility into quality adjusted life years: a systematic review of empirical studies. Pharmacoeconomics. 2013;31:677–691.
   PubMed Web of Science ®Google Scholar
• Kuppermann M, Shiboski S, Feeny D, et al. Can preference scores for discrete states be used to derive preference scores for an entire path of events? An application to prenatal diagnosis. Med Decis Making. 1997;17:42–55.
   PubMed Web of Science ®Google Scholar
• MacKeigan LD, O’Brien BJ, Oh PI. Holistic versus composite preferences for lifetime treatment sequences for type 2 diabetes. Med Decis Making. 1999;19:113–121.
   PubMed Web of Science ®Google Scholar
• Matza LS, Stewart KD, Gandra SR, et al. Acute and chronic impact of cardiovascular events on health state utilities. BMC Health Serv Res. 2015b;15:173.
   PubMed Web of Science ®Google Scholar
• Cancer Research UK. Acute myeloid leukaemia (AML): Symptoms. 2016 [cited 2017 Dec 20]. Available from: http://about-cancer.cancerresearchuk.org/about-cancer/acute-myeloid-leukaemia-aml/symptoms.
   Google Scholar
• Redaelli A, Stephens JM, Brandt S, et al. Short- and long-term effects of acute myeloid leukemia on patient health-related quality of life. Cancer Treat Rev. 2004;30:103–117.
   PubMed Web of Science ®Google Scholar
• Leukemia & Lymphoma Society (LLS). AML: Signs and Symptoms. 2016 [cited 2017 Dec 20]. Available from: https://www.lls.org/leukemia/acute-myeloid-leukemia/signs-and-symptoms.
   Google Scholar
• Tidy C. and ThePatient.info. Acute Myeloid Leukaemia. 2016 [cited 2017 Dec 20]. Available from: https://patient.info/doctor/acute-myeloid-leukaemia-pro.]
   Google Scholar
• Al-Ali HK, Jaekel N, Niederwieser D. The role of hypomethylating agents in the treatment of elderly patients with AML. J Geriatr Oncol. 2014;5:89–105.
   PubMed Web of Science ®Google Scholar
• CancerMPact and Kantar Health, Treatment Architecture: United States - Acute Myeloid Leukemia. December 2015, v1.2.
   Google Scholar
• Dohner H, Weisdorf DJ, Bloomfield CD. Acute myeloid leukemia. N Engl J Med. 2015;373:1136–1152.
   PubMed Web of Science ®Google Scholar
• Fey MF, Buske C, ESMO Guidelines Working Group. Acute myeloblastic leukaemias in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2013;24:vi138–vi143.
   PubMed Web of Science ®Google Scholar
• Kolitz JE, Strickland SA, Cortes JE, et al. Efficacy by consolidation administration site: subgroup analysis of a phase III study of CPX-351 versus 7 + 3 in older adults with newly diagnosed, high-risk acute myeloid leukemia (AML). JCO. 2017;35:7036.
   Web of Science ®Google Scholar
• London Cancer Alliance and South East London Cancer Network. HiDAC (High Dose Cytarabine) for AML. 2012 [cited 2017 Dec 20]. Available from: http://www.londoncanceralliance.nhs.uk/media/36431/AML_HiDAC__protocol_v1.0.pdf.
   Google Scholar
• National Comprehensive Cancer Network (NCCN), NCCN Clinical Practice Guidelines in Oncology - Acute Myeloid Leukemia. 2017. p. MS-77.
   Google Scholar
• Cancer Research UK. Coping with acute myeloid leukaemia (AML). 2016 [cited 2017 Dec 20]. Available from: http://about-cancer.cancerresearchuk.org/about-cancer/acute-myeloid-leukaemia-aml/living-with/coping-acute-myeloid-leukaemia.
   Google Scholar
• Ritchie EK, Roboz GJ. Levels of care: defining best supportive care in elderly patients with acute myeloid leukemia. Curr Hematol Malig Rep. 2010;5:95–100.
   PubMed Web of Science ®Google Scholar
• Leukemia & Lymphoma Society (LLS). Allogeneic Stem Cell Transplantation. 2016 [cited 2017 Dec 20]. Available from: https://www.lls.org/treatment/types-of-treatment/stem-cell-transplantation/allogeneic-stem-cell-transplantation.
   Google Scholar
• Jazz Pharmaceuticals. Vyxeos CPX-351 301 trial. Data on file VYX-2017-019.
   Google Scholar
• Canadian Agency for Drugs and Technologies in Health (CADTH). Guidelines for the economic evaluation of health technologies: Canada. Ottawa (CAN): CADTH; 2006.
   Google Scholar
• Attema AE, Brouwer WB. On the (not so) constant proportional trade-off in TTO. Qual Life Res. 2010;19:489–497.
   PubMed Web of Science ®Google Scholar
• Meyers J, Yu Y, Kaye JA, et al. Medicare fee-for-service enrollees with primary acute myeloid leukemia: an analysis of treatment patterns, survival, and healthcare resource utilization and costs. Appl Health Econ Health Policy. 2013;11:275–286.
   PubMedGoogle Scholar
• Matza LS, Sapra SJ, Dillon JF, et al. Health state utilities associated with attributes of treatments for hepatitis C. Eur J Health Econ. 2015;16:1005–1018.
   PubMed Web of Science ®Google Scholar
• Rowen D, Brazier J. Health utility measurement. In: Glied S, Smith P, editors. The Oxford Handbook of Health Economics. New York (NY): Oxford University Press; 2011. p. 788–813.
   Google Scholar
• Dolan P. Modeling valuations for EuroQol health states. Med Care. 1997;35:1095–1108.
   PubMed Web of Science ®Google Scholar
• Dolan P, Gudex C, Kind P, et al. The time trade-off method: results from a general population study. Health Econ. 1996;5:141–154.
   PubMed Web of Science ®Google Scholar
• Arnesen T, Trommald M. Are QALYs based on time trade-off comparable?—A systematic review of TTO methodologies. Health Econ. 2005;14:39–53.
   PubMed Web of Science ®Google Scholar
• Matza LS, Boye KS, Feeny DH, et al. The time horizon matters: results of an exploratory study varying the timeframe in time trade-off and standard gamble utility elicitation. Eur J Health Econ. 2016;17:979–990.
   PubMed Web of Science ®Google Scholar
• Scalone L, Stalmeier PFM, Milani S, et al. Values for health states with different life durations. Eur J Health Econ. 2015;16:917–925.
   PubMed Web of Science ®Google Scholar
• Clinical Trials.Gov and Jazz Pharmaceuticals. Phase III study of CPX-351 versus 7 + 3 in patients 60–75 years old with untreated high risk (secondary) acute myeloid leukemia (301). ClinicalTrials.gov Identifier: NCT01696084. [Last update February 27, 2018]; Available from: https://clinicaltrials.gov/ct2/show/results/NCT01696084?term=NCT01696084&rank=1&view=results [Last accessed January 10, 2019].
   Google Scholar