References
- World Health Organization. Haematopoietic stem cell transplantation HSCtx. 2020. Available from: https://www.who.int/transplantation/hsctx/en/.
- Cheuk DK. Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: prophylaxis and treatment controversies. World J Transplant. 2012;2(2):27–34.
- Carreras E. Veno-occlusive disease of the liver after hemopoietic cell transplantation. Eur J Haematol. 2000;64(5):281–291.
- Svahn BM, Remberger M, Alvin O, et al. Increased costs after allogeneic haematopoietic SCT are associated with major complications and re-transplantation. Bone Marrow Transplant. 2012;47(5):706–715.
- Carreras E. Early Complications after HSCT. In: The EBMT handbook. 6th ed. European School of Haematology. Cham (Switzerland): Springer Nature Switzerland AG; 2012. p. 177–195.
- Coppell JA, Richardson PG, Soiffer R, et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010;16(2):157–168.
- NHS England Clinical Commissioning. Clinical commissioning policy: use of defibrotide in severe veno-occlusive disease following stem cell transplant; 2015. Available from: https://www.england.nhs.uk/wp-content/uploads/2020/11/Defibrotide-in-severe-veno-occlusive-disease-following-stem-cell-transplant.pdf
- Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation. Bone Marrow Transplant. 2018;53(2):138–145.
- Carreras E, Diaz-Ricart M, et al. Early complications of endothelial origin. In: Carreras E, Dufour C, Mohty M, editors. The EBMT handbook: hematopoietic stem cell transplantation and cellular therapies. Cham (Switzerland): Springer Nature Switzerland AG; 2019. p. 315–322.
- Dalle JH, Giralt SA. Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: risk factors and stratification, prophylaxis, and treatment. Biol Blood Marrow Transplant. 2016;22(3):400–409.
- Wallhult E, Kenyon M, Liptrott S, et al. Management of veno-occlusive disease: the multidisciplinary approach to care. Eur J Haematol. 2017;98(4):322–329.
- Carreras E. [Prevention and treatment of hepatic veno-occlusive disease]. Gastroenterol Hepatol. 2011;34(9):635–640.
- Defitelio. Defitelio. Summary of product characteristics. 2019 [cited 2019 Apr 2]. Available from: https://pp.jazzpharma.com/pi/defitelio.gb.SPC.pdf
- Richardson PG, Riches ML, Kernan NA, et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016;127(13):1656–1665.
- Richardson PG, Soiffer RJ, Antin JH, et al. Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial. Biol Blood Marrow Transplant. 2010;16(7):1005–1017.
- Kernan NA, Grupp S, Smith AR, et al. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018;181(6):816–827.
- Corbacioglu S, Carreras E, Mohty M, et al. Defibrotide for the treatment of hepatic veno-occlusive disease: final results from the international compassionate-use program. Biol Blood Marrow Transplant. 2016;22(10):1874–1882.
- European Medicines Agency. Defitelio EPAR public assessment report; 2013. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/defitelio
- FDA. FDA approves first treatment for rare disease in patients who receive stem cell transplant from blood or bone marrow; 2016 [cited 2020 May 6]. Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-rare-disease-patients-who-receive-stem-cell-transplant-blood-or-%E2%80%A61/3.
- Belsey J, Kemadjou EN, Isaila M, et al. Defibrotide cost-effectiveness in Canada for the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) with multi-organ dysfunction (MOD) after HSCT. Biol Blood Marrow Transplant. 2019;25:S100–S289.
- Lopez Bastida J, Oliva J, Antonanzas F, et al. [A proposed guideline for economic evaluation of health technologies]. Gac Sanit. 2010;24(2):154–170.
- Puig-Junoy J, Oliva Moreno J, Trapero Bertran M, et al. Guia y recomendaciones para la realizacion y presentacion de evaluaciones economicas y analisis de impacto presupuestario de medicamentos en el ambito del Catsalut; 2014. Available from: https://catsalut.gencat.cat/web/.content/minisite/catsalut/proveidors_professionals/medicaments_farmacia/farmaeconomica/caeip/gaeip_publica_castellano_octubre2014_catsalut.pdf
- Remberger M, Ackefors M, Berglund S, et al. Improved survival after allogeneic hematopoietic stem cell transplantation in recent years. A single-center study. Biol Blood Marrow Transplant. 2011;17(11):1688–1697.
- Nivison-Smith I, Simpson JM, Dodds AJ, et al. Relative survival of long-term hematopoietic cell transplant recipients approaches general population rates. Biol Blood Marrow Transplant. 2009;15(10):1323–1330.
- Ministerio de Sanidad, Consumo y Bienestar Social. Portal Estadístico del SNS – Registro de Altas de los Hospitales Generales del Sistema Nacional de Salud. CMBD. Norma Estatal [2020 Nov 2]. Available from: https://www.mscbs.gob.es/estadEstudios/estadisticas/cmbd.htm.
- Aberg F, Maklin S, Rasanen P, et al. Cost of a quality-adjusted life year in liver transplantation: the influence of the indication and the model for end-stage liver disease score. Liver Transpl. 2011;17(11):1333–1343.
- Szende A, Janssen B, Cabases J, editors. Self-reported population health: an international perspective based on EQ-5D. 2014. Available from: https://www.springer.com/gp/book/9789400775954
- Goldman JM, Majhail NS, Klein JP, et al. Relapse and late mortality in 5-year survivors of myeloablative allogeneic hematopoietic cell transplantation for chronic myeloid leukemia in first chronic phase. J Clin Oncol. 2010;28(11):1888–1895.
- Briggs AH. Handling uncertainty in cost-effectiveness models. Pharmacoeconomics. 2000;17(5):479–500.
- Sacristan JA, Oliva J, Del Llano J, et al. Que es una tecnologia sanitaria eficiente en Espana. Gac Sanit. 2002;16(4):334–343.
- Vallejo-Torres L, Garcia-Lorenzo B, Serrano-Aguilar P. Estimating a cost-effectiveness threshold for the Spanish NHS. Health Econ. 2018;27(4):746–761.
- Sacristan JA, Rovira J, Ortun V, et al. Utilization of economic assessments of health interventions. Med Clin (Barc). 2004;122(20):789–795.
- Perales MA, Bonafede M, Cai Q, et al. Real-world economic burden associated with transplantation-related complications. Biol Blood Marrow Transplant. 2017;23(10):1788–1794.
- Cao Z, Villa KF, Lipkin CB, et al. Burden of illness associated with sinusoidal obstruction syndrome/veno-occlusive disease in patients with hematopoietic stem cell transplantation. J Med Econ. 2017;20(8):871–883.
- Lucas-Alcahuz B, Calpe-Armero P, Ferriols-Lisart R, et al. Cost-effectiveness analysis of treatment with defibrotide for patients with severe sinusoidal obstruction syndrome. Eur J Clin Pharm Atención Farm. 2019;21(1):6–12.
- Veenstra DL, Guzauskas GF, Villa KF, et al. The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant. J Med Econ. 2017;20(5):453–463.
- Richardson P, Aggarwal S, Topaloglu O, et al. Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS). Bone Marrow Transplant. 2019;54(12):1951–1962.