Cost-Effectiveness Analysis of Gemtuzumab Ozogamicin for First-Line Treatment of Patients with Cd-33 Positive Acute Myeloid Leukaemia in Spain

References

• Tallman MS, Wang ES, Altman JK, et al. Acute myeloid leukemia, version 3.2019, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2019;17(6):721–749. doi:10.6004/jnccn.2019.0028
   PubMed Web of Science ®Google Scholar
• Visser O, Trama A, Maynadié M, et al. Incidence, survival and prevalence of myeloid malignancies in Europe. Eur J Cancer. 2012;48(17):3257–3266. doi:10.1016/j.ejca.2012.05.024
   PubMed Web of Science ®Google Scholar
• Deschler B, Lübbert M. Acute myeloid leukemia: epidemiology and etiology. Cancer. 2006;107(9):2099–2107. doi:10.1002/cncr.22233
   PubMed Web of Science ®Google Scholar
• De Kouchkovsky I, Abdul-Hay M. Acute myeloid leukemia: a comprehensive review and 2016 update. Blood Cancer J. 2016;6(7):e441. doi:10.1038/bcj.2016.50
   PubMed Web of Science ®Google Scholar
• National Cancer Institute. Cancer stat facts: acute myeloid leukemia (AML); 2020. Available from: https://seer.cancer.gov/statfacts/html/amyl.html. Accessed April 12, 2021.
   Google Scholar
• Horvath Walsh LE, Rider A, Piercy J, et al. Real-world impact of physician and patient discordance on health-related quality of life in US patients with acute myeloid leukemia. Oncol Ther. 2019;7(1):67–81. doi:10.1007/s40487-019-0094-x
   PubMed Web of Science ®Google Scholar
• Pandya BJ, Chen CC, Medeiros BC, et al. Economic and clinical burden of acute myeloid leukemia episodes of care in the United States: a retrospective analysis of a commercial payer database. J Manag Care Spec Pharm. 2020;26(7):849–859. doi:10.18553/jmcp.2020.19220
   PubMed Web of Science ®Google Scholar
• Ferrara F, Schiffer CA. Acute myeloid leukaemia in adults. Lancet. 2013;381(9865):484–495. doi:10.1016/S0140-6736(12)61727-9
   PubMed Web of Science ®Google Scholar
• Loh KP, Pardee TS, Klepin HD. Acute myeloid leukemia in older adults. Geriatric Oncology. 2020:501–520.
   Google Scholar
• Döhner H, Estey EH, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424–447.
   PubMed Web of Science ®Google Scholar
• Fournier E, Duployez N, Ducourneau B, et al. Mutational profile and benefit of gemtuzumab ozogamicin in acute myeloid leukemia. Blood. 2020;135(8):542–546. doi:10.1182/blood.2019003471
   PubMed Web of Science ®Google Scholar
• Milligan DW, Grimwade D, Cullis JO, et al. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450–474. doi:10.1111/j.1365-2141.2006.06314.x
   PubMed Web of Science ®Google Scholar
• Talati C, Sweet K. Recently approved therapies in acute myeloid leukemia: a complex treatment landscape. Leuk Res. 2018;73:58–66. doi:10.1016/j.leukres.2018.09.001
   PubMed Web of Science ®Google Scholar
• Dombret H, Gardin C. An update of current treatments for adult acute myeloid leukemia. Blood. 2016;127(1):53–61. doi:10.1182/blood-2015-08-604520
   PubMed Web of Science ®Google Scholar
• Thol F, Schlenk RF. Gemtuzumab ozogamicin in acute myeloid leukemia revisited. Expert Opin Biol Ther. 2014;14(8):1185–1195. doi:10.1517/14712598.2014.922534
   PubMed Web of Science ®Google Scholar
• Castaigne S, Pautas C, Terré C, et al. Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): a randomised, open-label, phase 3 study. Lancet. 2012;379(9825):1508–1516. doi:10.1016/S0140-6736(12)60485-1
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Mylotarg 5 mg powder for solution for infusion summary of product characteristics. London: European Medicines Agency; 2020. Available from: https://www.ema.europa.eu/en/documents/product-information/mylotarg-epar-product-information_es.pdf. Accessed April 12, 2021.
   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(7):2113–2120. doi:10.1182/blood-2010-05-285502
   PubMed Web of Science ®Google Scholar
• Leunis A, Redekop WK, van Montfort KA, et al. The development and validation of a decision-analytic model representing the full disease course of acute myeloid leukemia. Pharmacoeconomics. 2013;31(7):605–621. doi:10.1007/s40273-013-0058-1
   Web of Science ®Google Scholar
• Wang HI, Aas E, Howell D, et al. Long-term medical costs and life expectancy of acute myeloid leukemia: a probabilistic decision model. Value Health. 2014;17(2):205–214. doi:10.1016/j.jval.2013.12.007
   PubMed Web of Science ®Google Scholar
• López Bastida J, Oliva J, Antoñanzas F, et al. Spanish recommendations on economic evaluation of health technologies. Eur J Health Econ. 2010;11:513–520. doi:10.1007/s10198-010-0244-4
   Web of Science ®Google Scholar
• Lambert J, Pautas C, Terré C, et al. Gemtuzumab ozogamicin for de novo acute myeloid leukemia: final efficacy and safety updates from the open-label, phase III ALFA-0701 trial. Haematologica. 2019;104(1):113–119. doi:10.3324/haematol.2018.188888
   PubMed Web of Science ®Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics forFludarabina 50 mg polvo para solución inyectable y para perfusión EFG [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/60616/60616_ft.pdf. Accesssed April 12, 2021
   Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics forCitarabina Pfizer 500 mg polvo y disolvente para solución inyectable y para perfusión [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/49154/49154_ft.pdf. Accessed April 12, 2021.
   Google Scholar
• European Medicines Agency. Summary of product characteristics for Filgrastim HEXAL 30 MU/0,5 mL solución inyectable o para perfusión en jeringa precargada. Londres: Agencia Europea de Medicamentos; 2020. Available from: https://www.ema.europa.eu/en/documents/product-information/filgrastim-hexal-epar-product-information_es.pdf. Accessed April 12, 2021.
   Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics forIdarubicina Sandoz 1 mg/mL concentrado para solución para perfusión [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/75242/FichaTecnica_75242.html.pdf. Accessed April 12, 2021.
   Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics for Hydrea 500 mg cápsulas duras [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/61633/FichaTecnica_61633.html.pdf. Accessed April 12, 2021.
   Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics forVidaza 25 mg/mL polvo para suspensión inyectable [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/08488001/FT_08488001.pdf. Accessed April 12, 2021.
   Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics forDacogen 50 mg polvo para concentrado para solución para perfusión [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/112792001/FT_112792001.pdf. Accessed April 12, 2021.
   Google Scholar
• National Institute of Statistics (Instituto Nacional de Estadística; INE). 2018 mortality tables of Spanish population. National results. INEbase. Madrid: Instituto Nacional de Estadística. Available from: http://www.ine.es. Accessed April 12, 2021.
   Google Scholar
• National Institute for Health and Care Excellence (NICE). Technology appraisal No. 399. Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. Company evidence submission. Data on file (provided by Pfizer). November 25, 2015.
   Google Scholar
• McKenzie L, van der Pol M. Mapping the EORTC QLQ C-30 onto the EQ-5D instrument: the potential to estimate QALYs without generic preference data. Value Health. 2009;12(1):167–171. doi:10.1111/j.1524-4733.2008.00405.x
   PubMed Web of Science ®Google Scholar
• Kurosawa S, Yamaguchi H, Yamaguchi T, et al. Decision analysis of postremission therapy in cytogenetically intermediate-risk acute myeloid leukemia: the impact of FLT3 internal tandem duplication, nucleophosmin, and CCAAT/enhancer binding protein alpha. Biol Blood Marrow Transplant. 2016;22(6):1125–1132. doi:10.1016/j.bbmt.2016.03.015
   Web of Science ®Google Scholar
• Ara R, Brazier JE. Populating an economic model with health state utility values: moving toward better practice. Value Health. 2010;13(5):509–518. doi:10.1111/j.1524-4733.2010.00700.x
   PubMed Web of Science ®Google Scholar
• Scottish Medicines Consortium. Defibrotide, 80mg/mL, concentrate for solution for infusion (Defitelio®). SMC No. (967/14). May 9, 2014. Available from: https://www.scottishmedicines.org.uk/media/1543/defibrotide__defitelio__final_may_2014_for_website.pdf. Accessed April 16, 2021
   Google Scholar
• Kantarjian HM, DeAngelo DJ, Stelljes M, et al. Inotuzumab ozogamicin versus standard of care in relapsed or refractory acute lymphoblastic leukemia: final report and long-term survival follow-up from the randomized, phase 3 INO-VATE study. Cancer. 2019;125(14):2474–2487. doi:10.1002/cncr.32116
   PubMed Web of Science ®Google Scholar
• Consejo General de Colegios Oficiales de Farmacéuticos. Base de datos del Conocimiento Sanitario - Bot Plus 2.0 [Health Knowledge database – bot Plus 2.0] [Internet]. Madrid: Consejo General de Colegios Oficiales de Farmacéuticos; 2020. Available from: https://botplusweb.portalfarma.com/. Accessed April 12, 2021.
   Google Scholar
• Ministry of Health, Social Services and Equality. Informative list of medicines affected by the deductions established in Royal Decree Law 8/2010 of 20 May, which adopts extraordinary measures to reduce the public deficit. [Internet]. Madrid: ; 2010. Available from: http://www.msssi.gob.es/profesionales/farmacia/pdf/DeduccionesJulio2019.pdf.
   Google Scholar
• Oblikue Consulting. Base de datos de costes sanitarios eSalud [eSalud health cost database] [Internet]. Barcelona: Oblikue Consulting; 2020. Available from: http://www.oblikue.com/bddcostes/. Accessed April 12, 2021.
   Google Scholar
• National Institute of Statistics (Instituto Nacional de Estadísitica; INE). Available from: http://www.ine.es. Accessed April 12, 2021.
   Google Scholar
• Ortega Eslava A, Marín Gil R, Fraga Fuentes MD, et al. Guidelines on economic evaluation and budgetary impact in drug assessment reports. Practical guide associated with the MADRE v 4.0 programme. Madrid: SEFH (ed.); 2016. Available from: http://gruposdetrabajo.sefh.es/genesis/index.php?option=com_content&view=article&id=11&Itemid=13. Accessed April 12, 2021.
   Google Scholar
• Vallejo-Torres L, García-Lorenzo B, Rivero-Arias O, Pinto-Prades JL. The societal monetary value of a QALY associated with EQ-5D-3L health gains. Eur J Health Econ. 2020;21(3):363–379. doi:10.1007/s10198-019-01140-4
   PubMed Web of Science ®Google Scholar
• Spanish Agency of Medicine and Sanitary Products. Summary of product characteristics for Mylotarg 5 mg polvo para concentrado para solución para perfusión [Internet]. Madrid: Spanish Agency of Medicine and Sanitary Products.; 2020. Available from: https://cima.aemps.es/cima/pdfs/es/ft/1181277001/FT_1181277001.pdf. Accessed April 12, 2021.
   Google Scholar
• Larson RA, Sievers EL, Stadtmauer EA, et al. Final report of the efficacy and safety of gemtuzumab ozogamicin (Mylotarg) in patients with CD33-positive acute myeloid leukemia in first recurrence. Cancer. 2005;104(7):1442–1452. doi:10.1002/cncr.21326
   PubMed Web of Science ®Google Scholar
• Petersdorf SH, Kopecky KJ, Slovak M, et al. A phase 3 study of gemtuzumab ozogamicin during induction and postconsolidation therapy in younger patients with acute myeloid leukemia. Blood. 2013;121(24):4854–4860. doi:10.1182/blood-2013-01-466706
   PubMed Web of Science ®Google Scholar
• Pautas C, Raffoux E, Lambert J, et al. Outcomes following hematopoietic stem cell transplantation in patients treated with standard chemotherapy with or without gemtuzumab ozogamicin for acute myeloid leukemia. Bone Marrow Transplant. 2021. doi:10.1038/s41409-020-01207-4
   Web of Science ®Google Scholar
• Hills RK, Castaigne S, Appelbaum FR, et al. Addition of gemtuzumab ozogamicin to induction chemotherapy in adult patients with acute myeloid leukaemia: a meta-analysis of individual patient data from randomised controlled trials. Lancet Oncol. 2014;15(9):986–996. doi:10.1016/S1470-2045(14)70281-5
   PubMed Web of Science ®Google Scholar
• Mokgokong R, Mamolo C, Cappelleri JC, et al. Cost-effectiveness of gemtuzumab ozogamicin in combination with standard of care chemotherapy (daunorubicin and cytarabine) for first-line treatment of acute myeloid leukaemia. Presented at the 59th Annual Scientific Meeting of the British Society for Haematology (BSH); April 1–3, 2019; Glasgow, Scotland.
   Google Scholar
• Gong Q, Zhou L, Xu S, et al. High doses of daunorubicin during induction therapy of newly diagnosed acute myeloid leukemia: a systematic review and meta-analysis of prospective clinical trials. PLoS One. 2015;10(5):e0125612. doi:10.1371/journal.pone.0125612
   PubMed Web of Science ®Google Scholar
• Li X, Xu S, Tan Y, Chen J. The effects of idarubicin versus other anthracyclines for induction therapy of patients with newly diagnosed leukaemia. Cochrane Database Syst Rev. 2015;6:CD010432.
   PubMed Web of Science ®Google Scholar