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Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA.
Appl Health Econ Health Policy
December 2024
Department of Pharmacy Systems, Outcomes, and Policy, Retzky College of Pharmacy, University of Illinois Chicago, Chicago, IL, USA.
Article Synopsis
- - The study evaluates the cost-effectiveness of a new gene therapy, Etranacogene dezaparvovec (EDZ), for severe hemophilia B, comparing it to the traditional treatment, factor IX (FIX) prophylaxis over a lifetime perspective in the U.S.
- - Results show that despite EDZ costing $3.5 million, it offers lifetime savings of $11 million and slightly improves quality of life by 0.64 QALYs compared to FIX, which has very high annual costs.
- - The cost-effectiveness of EDZ is influenced significantly by capping annual cost offsets, emphasizing how these limits can help balance price and value for healthcare systems.
Interventional Oncology Meets Immuno-oncology: Combination Therapies for Hepatocellular Carcinoma.
Radiology
November 2024
From the Departments of Radiology (R.B., J.C.) and Digestive Diseases (Hepatology) (J.C.), Yale University School of Medicine, New Haven, Conn; Department of Radiology, Feinberg School of Medicine, Northwestern University, Chicago, Ill (R.S.); Department of Medical Oncology, Geffen School of Medicine, University of California Los Angeles, Los Angeles, Calif (R.F.); Center for Cancer Research, National Institutes of Health, Bethesda, Md (T.F.G.); Department of Radiology, Hadassah Hebrew University Medical Center, Hebrew University, Jerusalem, Israel (S.N.G.); and Department of Biomedical Engineering, Yale School of Engineering and Applied Sciences, 789 Howard Ave, Clinic Bldg 363H, New Haven, CT 06520 (J.C.).
The management of hepatocellular carcinoma (HCC) is undergoing transformational changes due to the emergence of various novel immunotherapies and their combination with image-guided locoregional therapies. In this setting, immunotherapy is expected to become one of the standards of care in both neoadjuvant and adjuvant settings across all disease stages of HCC. Currently, more than 50 ongoing prospective clinical trials are investigating various end points for the combination of immunotherapy with both percutaneous and catheter-directed therapies.
View Article and Find Full Text PDFCommentary on Novitas LCD.
Bladder Cancer
December 2023
Department of Urology, UT Health San Antonio, San Antonio, TX, USA.
Complement Activation Is Associated With Disease Severity in Multiple Sclerosis.
Neurol Neuroimmunol Neuroinflamm
March 2024
From the Department of Neurology (J.O., A.M.M., A.O., S. Meier, E.W., T.D., M.D.S., M.L., B.F.-B., C. Granziera, L.K., D.L., J.K.); Multiple Sclerosis Centre and Research Center for Clinical Neuroimmunology and Neuroscience (RC2NB) (J.O., S.A.S., A.M.M., A.O., S. Meier, E.W., T.D., P.B., M.D.S., M.L., B.F.-B., C. Granziera, L.K., D.L., J.K.), Departments of Biomedicine and Clinical Research, University Hospital and University of Basel, Switzerland; Department of Neurology with Institute of Translational Neurology (K.S., H.W., J.D.L.), University Hospital 4 Münster, Germany; Clinical Trial Unit (S.A.S., P.B.), Department of Clinical Research, University Hospital Basel, University of Basel, Basel, Switzerland; Octavebio Bioscience (F.Q.), Menlo Park, CA; Division of Medical Immunology (I.H.), Laboratory Medicine, University Hospital Basel, Switzerland; Medica Laboratory (A.R.), Zürich; Department of Neurology (L.A.), Cantonal Hospital, Aarau; Department of Neurology (S. Mueller), Cantonal Hospital St. Gallen; Department of Neurology (A.S.), Inselspital, Bern University Hospital and University of Bern; Department of Clinical Neurosciences (P.H.L., C.B.), Division of Neurology; Diagnostic Department (P.H.L.), Division of Laboratory Medicine; Department of Pathology and Immunology (P.H.L.), Faculty of Medicine, University of Geneva; Division of Neurology (C.P., R.A.D.P.), Department of Clinical Neurosciences, Lausanne University Hospital (CHUV) and University of Lausanne; Neurocentre of Southern Switzerland (C. Gobbi), Multiple Sclerosis Centre, Ospedale Civico; Faculty of Biomedical Sciences (C. Gobbi), Università della Svizzera Italiana (USI), Lugano, Switzerland; Translational Imaging in Neurology (ThINk) Basel (C. Granziera), Department of Biomedical Engineering, Faculty of Medicine, University Hospital Basel and University of Basel; and Division of Internal Medicine (M.T.), University Hospital Basel and Clinical Immunology, Department of Biomedicine, University of Basel, Basel, Switzerland.
Article Synopsis
- The study investigates the role of complement components (CCs) and activation products (CAPs) in multiple sclerosis (MS), particularly focusing on how their levels are affected by the presence of intrathecal IgM synthesis, which is linked to higher disease severity.
- By analyzing samples from 112 clinically isolated syndrome (CIS) patients and 127 MS patients, it was found that specific complement levels in the cerebrospinal fluid (CSF) were significantly higher in those with MS compared to control groups.
- Key findings indicate that increased levels of complement components like C3a and C4a in the CSF correlate with worse disability and disease progression in MS patients, emphasizing the relationship between complement activation and neurodegeneration in
Effect of Incomplete Cryoablation and Matrix Metalloproteinase Inhibition on Intratumoral CD8 T-Cell Infiltration in Murine Hepatocellular Carcinoma.
Radiology
February 2024
From the Department of Radiology and Biomedical Imaging (A.S., J.G.S., D.N., J.T., S.K.M., D.C., J.D., D.C.M., J.C.), Section of Digestive Diseases, Department of Internal Medicine (S.J.R., J.C.), Section of Medical Oncology, Department of Medicine (D.C.M.), and Section of Surgical Oncology, Department of Surgery (D.C.M.), Yale University School of Medicine, 300 Cedar St, The Anlyan Center, N312A, New Haven, CT 06520; Department of Diagnostic and Interventional Radiology and Neuroradiology, University Hospital Essen, Essen, Germany (A.S., A.W.); Department of Radiology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität, Berlin, Germany (A.B., V.K.); Department of Biomedical Engineering, School of Engineering & Applied Science, Yale University, New Haven, Conn (D.C., J.D., J.C.); and Department of Diagnostic and Interventional Radiology and Neuroradiology, Asklepios Clinic Altona, Hamburg, Germany (A.W.).
Background Image-guided tumor ablation is the first-line therapy for early-stage hepatocellular carcinoma (HCC), with ongoing investigations into its combination with immunotherapies. Matrix metalloproteinase (MMP) inhibition demonstrates immunomodulatory potential and reduces HCC tumor growth when combined with ablative treatment. Purpose To evaluate the effect of incomplete cryoablation with or without MMP inhibition on the local immune response in residual tumors in a murine HCC model.
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