Cost-effectiveness analysis of defibrotide in the treatment of patients with severe veno-occlusive disease/sinusoidal obstructive syndrome with multiorgan dysfunction following hematopoietic cell transplantation in Spain

Carcedo, David; Urain, Teresa Artola; Rodríguez, A. Chinea; Torres, E; Vicent, Marta González; Gutiérrez-García, Gonzalo; García, Alexandra Regueiro; Hidalgo, M; Villacampa, Alba

doi:10.1080/13696998.2021.1916749

Cited by 5 publications

(3 citation statements)

References 31 publications

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“…Additionally, multiple studies have highlighted the cost-effectiveness of defibrotide in treating VOD/SOS compared with the best supportive care. These studies indicate that defibrotide not only serves as a cost-effective alternative but also contributes a substantial increase in quality-adjusted life years 13–15…”

Section: Introductionmentioning

confidence: 96%

Hepatic veno-occlusive disease complicated with extreme hyperammonaemia (920 µmol/L) treated with defibrotide, lactulose, rifampin and haemodialysis

Kabat,

Gill,

Kim

et al. 2024

BMJ Case Rep

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Hepatic veno-occlusive disease (VOD)/sinusoidal obstructive syndrome (SOS) is a severe complication that can occur following haematopoietic stem cell transplant (HSCT) with high-intensity conditioning chemotherapy regimens. Severe VOD/SOS, often characterised by multiorgan failure, is associated with a high mortality rate. This case report details the complex clinical course of a male patient in his mid-20s, recently diagnosed with B cell acute lymphoblastic leukaemia, who underwent allogeneic HSCT. Based on the 2023 European Society for Blood and Marrow Transplantation (EBMT) criteria, the patient developed very severe VOD/SOS, prompting immediate treatment with defibrotide. Unexpectedly, he developed profound hyperammonaemia exceeding 900 µmol/L, leading to encephalopathy and cerebral oedema. Despite aggressive interventions including defibrotide, lactulose, rifampin and haemodialysis, the patient passed away due to cerebral oedema and pulseless electrical activity arrest. We theorise the hyperammonaemia is disproportionate to his hepatic dysfunction and is possibly secondary to an acquired defect of the urea synthesis consistent with idiopathic hyperammonaemia, a rare complication in patients receiving intense conditioning chemotherapy.

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Section: Introductionmentioning

confidence: 96%

Hepatic veno-occlusive disease complicated with extreme hyperammonaemia (920 µmol/L) treated with defibrotide, lactulose, rifampin and haemodialysis

Kabat,

Gill,

Kim

et al. 2024

BMJ Case Rep

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“…The high costs of DF are controversial [20]. A recent study evaluated the cost-effectiveness of DF compared to best supportive care (BSC) for the treatment of SOS with MOF after HSCT [21]. They found a lower SOS-related hospitalization duration (7.5 vs. 23.2 days) and proportional less stay in intensive care unit (30% vs 60%) in DF treated patients compared to BSC patients, which implies a better cost effectiveness in patients receiving DF.…”

Section: Introductionmentioning

confidence: 99%

“…They found a lower SOS-related hospitalization duration (7.5 vs. 23.2 days) and proportional less stay in intensive care unit (30% vs 60%) in DF treated patients compared to BSC patients, which implies a better cost effectiveness in patients receiving DF. The analysis based on data of the study of Richardson et al [12], which compared 102 patients treated with DF (between 2006 and 2008) with a historical control group (n = 32, treated between 1995 and 2007) and the results were adapted to the Spanish health system (2019) [21]. In an editorial about this study, Gratwohl criticizes that the study is based on data which was partly collected over 20 years ago and adapted to today's health system.…”

Section: Introductionmentioning

confidence: 99%

Low Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease in adults undergoing allogenic stem cell transplantation with prophylactic ursodiol and low-dose heparin

Stutz

Halter

Heim

et al. 2022

Bone Marrow Transplant

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Hepatic sinusoidal obstruction syndrome (SOS)/veno-occlusive disease (VOD) is a complication after allogenic hematopoietic stem-cell transplantation (allo-HSCT) with high mortality. The purpose of this study was to assess the incidence and outcome of SOS in patients after allo-HSCT with the impact of ursodeoxycholic acid (UDCA) and low-dose heparin as SOS prophylaxis. Out of 1016 patients, 23 developed SOS, with a cumulative incidence of 2.3% (95% CI 1.3–3.3) 6 months after HSCT. Approximately one quarter of these patients (26.1%) had late-onset SOS. A high proportion were very severe SOS cases (74%), and 83% of the patients were treated with defibrotide (DF). In multivariate analysis, advanced disease (p = 0.003), previous HSCT (p = 0.025) and graft versus host disease (GvHD) prophylaxis by post-transplant cyclophosphamide (PTCy) (p = 0.055) were associated with the development of SOS. The 1-year overall survival (OS) was significantly lower in the SOS group compared to patients without SOS (13% versus 70%, p = 0.0001). In conclusion, we found a low incidence of SOS in patients receiving low-dose heparin and UDCA prophylactically, but among SOS patients, a high mortality. Low-dose heparin and UDCA might be a prophylactic approach for SOS.

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Cost-effectiveness of defibrotide for treatment of severe veno-occlusive disease: it is time for evidence based economic evaluations

Gratwohl

2021

Journal of Medical Economics

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Rodriguez et al present in this issue a cost-effectiveness analysis of Defibrotide (Defitelio R ) for the treatment of severe veno-occlusive disease in the context of hematopoietic stem cell transplantation (HSCT) [1]. According to their analysis, Defibrotide might produce 1.214 QALYs compared to best supportive care, with a total cost of €33,708. They conclude that Defibrotide offers a cost-effective therapy, with ratios of €27,757/QALY and €25,007/LY gained [1]. Their complex analysis follows the rules and roles of economic evaluations for novel drugs, and the authors admit some limitations. Still, they suggest that Defibrotide is cost effective within a

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Cost-effectiveness analysis of defibrotide in the treatment of patients with severe veno-occlusive disease/sinusoidal obstructive syndrome with multiorgan dysfunction following hematopoietic cell transplantation in Spain

Abstract: View related articles View Crossmark dataCost-effectiveness analysis of defibrotide in the treatment of patients with severe veno-occlusive disease/sinusoidal obstructive syndrome with multiorgan dysfunction following hematopoietic cell transplantation in Spain

Cited by 5 publications

References 31 publications

Hepatic veno-occlusive disease complicated with extreme hyperammonaemia (920 µmol/L) treated with defibrotide, lactulose, rifampin and haemodialysis

Hepatic veno-occlusive disease complicated with extreme hyperammonaemia (920 µmol/L) treated with defibrotide, lactulose, rifampin and haemodialysis

Low Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease in adults undergoing allogenic stem cell transplantation with prophylactic ursodiol and low-dose heparin

Cost-effectiveness of defibrotide for treatment of severe veno-occlusive disease: it is time for evidence based economic evaluations

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