Carcedo Rodriguez, DavidArtola Urain, TeresaChinea Rodriguez, AnabelleGarcía Torres, EstefaníaGonzález Vicent, MartaGutiérrez García, GonzaloRegueiro García, AlexandraCalvo Hidalgo, MarcosVillacampa, Alba2023-02-092023-02-092021http://hdl.handle.net/10668/17586This study evaluated cost-effectiveness of defibrotide vs best supportive care (BSC) for the treatment of hepatic veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) with multiorgan dysfunction (MOD) post-hematopoietic cell transplantation (HCT) in Spain. A two-phase Markov model, comprising a 1-year acute phase with daily cycles and a lifetime long-term phase with annual cycles, was adapted to the Spanish setting. The model included a cohort of patients with severe VOD/SOS (defined as VOD/SOS with MOD) post-HCT. For the acute phase, efficacy and VOD/SOS-related length of stay were obtained from a phase 3 defibrotide study (NCT00358501). VOD/SOS-related hospital stays were 7.5 and 23.2 days in defibrotide-treated and BSC patients, respectively. Defibrotide-treated patients spent 30% of their stay in the intensive care unit vs 60% in BSC patients. Assumptions for the long-term phase and utility values were obtained from the literature. Costs were from the Spanish Health System perspective (€2019). Defibrotide cost was based on 25 mg/kg/day over 17.5 days, using local expert opinion. Life-years (LYs), quality-adjusted LYs (QALYs), and costs were estimated over a lifetime horizon, applying a 3% discount rate for costs and outcomes. Sensitivity analyses assessed the robustness of the results. Defibrotide produced an additional 1.214 QALYs and 1.348 LYs vs BSC, with a total cost of €33,708 more than BSC alone. However, defibrotide resulted in savings up to €16,644/patient for cost of hospital stay. Difference between costs and effective measures led to ratios of €27,757/QALY and €25,007/LY gained. Additional hospital stays had the greatest influence on base-case results. Probabilistic analysis confirmed the robustness of the deterministic results. Limitations include use of historical controls and assumptions extrapolated from the literature. This cost-effectiveness model, adapted to the Spanish setting, showed that defibrotide is a cost-effective alternative to BSC alone in patients with severe VOD/SOS post-HCT.enAttribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/DefibrotideE20I00Spaincost-effectivenessmultiorgan dysfunctionsinusoidal obstruction syndromeveno-occlusive diseaseCost-Benefit AnalysisFibrinolytic AgentsHematopoietic Stem Cell TransplantationHepatic Veno-Occlusive DiseaseHumansPolydeoxyribonucleotidesSpainresearch article33858278open access10.1080/13696998.2021.19167491941-837Xhttps://www.tandfonline.com/doi/pdf/10.1080/13696998.2021.1916749?needAccess=true