Cargando…

Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

The objective was to undertake an early cost-effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA) without FVIII-antibodies. We also aimed to i...

Descripción completa

Detalles Bibliográficos
Autores principales:	ten Ham, Renske M.T., Walker, Simon M., Soares, Marta O., Frederix, Geert W.J., Leebeek, Frank W.G., Fischer, Kathelijn, Coppens, Michiel, Palmer, Stephen J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8820916/ https://www.ncbi.nlm.nih.gov/pubmed/35141470 http://dx.doi.org/10.1097/HS9.0000000000000679

Ejemplares similares

Toward Transplantation of Liver Organoids: From Biology and Ethics to Cost-effective Therapy
por: ten Dam, Marjolein J.M., et al.
Publicado: (2023)

Treatment of acquired hemophilia A, a balancing act: results from a 27‐year Dutch cohort study
por: Schep, Sarah J., et al.
Publicado: (2020)

Hepatitis C virus in hemophilia: Health‐related quality of life after successful treatment in the sixth Hemophilia in the Netherlands study
por: Isfordink, Cas J., et al.
Publicado: (2021)

Correction Notice: Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
Publicado: (2022)

Joint status of patients with nonsevere hemophilia A
por: Zwagemaker, Anne‐Fleur, et al.
Publicado: (2022)

In patients with hemophilia, a decreased thrombin generation profile is associated with a severe bleeding phenotype
por: Verhagen, Marieke J.A., et al.
Publicado: (2023)

Socioeconomic participation of persons with hemophilia: Results from the sixth hemophilia in the Netherlands study
por: van Balen, Erna C., et al.
Publicado: (2022)

The bleeding phenotype in people with nonsevere hemophilia
por: Kloosterman, Fabienne R., et al.
Publicado: (2022)

New Developments in Diagnosis and Management of Acquired Hemophilia and Acquired von Willebrand Syndrome
por: Leebeek, Frank W. G.
Publicado: (2021)

Role of Regulatory Cells in Immune Tolerance Induction in Hemophilia A
por: Schep, Sarah J., et al.
Publicado: (2021)

Generic PROMIS item banks in adults with hemophilia for patient‐reported outcome assessment: Feasibility, measurement properties, and relevance
por: Kuijlaars, Isolde A. R., et al.
Publicado: (2021)

Validation of PROMIS Profile‐29 in adults with hemophilia in the Netherlands
por: van Balen, Erna C., et al.
Publicado: (2021)

Similar sports participation as the general population in Dutch persons with haemophilia; results from a nationwide study
por: Versloot, Olav, et al.
Publicado: (2021)

Desmopressin for bleeding in non‐severe hemophilia A: Suboptimal use in a real‐world setting
por: Zwagemaker, Anne‐Fleur, et al.
Publicado: (2022)

Health and treatment outcomes of patients with hemophilia in the Netherlands, 1972–2019
por: Hassan, Shermarke, et al.
Publicado: (2021)

Clinical outcomes in hemophilia: Towards development of a core set of standardized outcome measures for research
por: Dover, Saunya, et al.
Publicado: (2020)

The experiences and attitudes of hemophilia carriers around pregnancy: A qualitative systematic review
por: Punt, Marieke C., et al.
Publicado: (2020)

Population Pharmacokinetics of Clotting Factor Concentrates and Desmopressin in Hemophilia
por: Preijers, Tim, et al.
Publicado: (2020)

Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B
por: Schmidt, Manfred, et al.
Publicado: (2023)

Association between Sports Participation, Factor VIII Levels and Bleeding in Hemophilia A
por: Bukkems, Laura H., et al.
Publicado: (2022)

Design of a Prospective Study on Pharmacokinetic-Guided Dosing of Prophylactic Factor Replacement in Hemophilia A and B (OPTI-CLOT TARGET Study)
por: Goedhart, Tine M.H.J., et al.
Publicado: (2022)

Antithrombotic Treatment in Patients With Hemophilia: an EHA-ISTH-EAHAD-ESO Clinical Practice Guidance
por: Schutgens, Roger E.G., et al.
Publicado: (2023)

Mortality, life expectancy, and causes of death of persons with hemophilia in the Netherlands 2001–2018
por: Hassan, Shermarke, et al.
Publicado: (2020)

The Pharmacoeconomic Benefits of Pemetrexed Dose Individualization in Patients With Lung Cancer
por: de Rouw, Nikki, et al.
Publicado: (2022)

In silico evaluation of limited sampling strategies for individualized dosing of extended half-life factor IX concentrates in hemophilia B patients
por: Preijers, T., et al.
Publicado: (2021)

Reduced cardiovascular morbidity in patients with hemophilia: results of a 5-year multinational prospective study
por: Van Der Valk, Paul, et al.
Publicado: (2022)

Predicting Individual Changes in Terminal Half-Life After Switching to Extended Half-Life Concentrates in Patients With Severe Hemophilia
por: Versloot, Olav, et al.
Publicado: (2022)

Check Your Checklist: The Danger of Over- and Underestimating the Quality of Economic Evaluations
por: Frederix, Geert W. J.
Publicado: (2019)

Determinants of successful immune tolerance induction in hemophilia A: systematic review and meta-analysis
por: Oomen, Ilja, et al.
Publicado: (2022)

Treatment‐related risk factors for inhibitor development in non‐severe hemophilia A after 50 cumulative exposure days: A case‐control study
por: Abdi, Amal, et al.
Publicado: (2021)

Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia
por: Thorat, Teja, et al.
Publicado: (2018)

Use of Social Media in the Assessment of Relative Effectiveness: Explorative Review With Examples From Oncology
por: Kalf, Rachel RJ, et al.
Publicado: (2018)

Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks
por: Teela, Lorynn, et al.
Publicado: (2023)

The factor VIII treatment history of non‐severe hemophilia A
por: Abdi, Amal, et al.
Publicado: (2020)

Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe
por: ten Ham, Renske M.T., et al.
Publicado: (2018)

Hemophilia B in a female with intellectual disability caused by a deletion of Xq26.3q28 encompassing the F9
por: Stoof, Sara C. M., et al.
Publicado: (2018)

Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set
por: van Balen, Erna C., et al.
Publicado: (2021)

Novel F8 and F9 gene variants from the PedNet hemophilia registry classified according to ACMG/AMP guidelines
por: Andersson, Nadine G., et al.
Publicado: (2020)

Desmopressin in nonsevere hemophilia A: patient perspectives on use and efficacy
por: Romano, Lorenzo G.R., et al.
Publicado: (2023)

Inhibitor incidence in an unselected cohort of previously untreated patients with severe hemophilia B: a PedNet study
por: Male, Christoph, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_gdlik8nc022anjbc3i29qc1jnk