PHARMAC funds longer-acting treatments for haemophilia

Source: PharmacoEconomics and Outcomes News - Category: Drugs & Pharmacology Source Type: news

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Condition:   Hemophilia A Intervention:   Biological: valoctocogene roxaparvovec Sponsor:   BioMarin Pharmaceutical Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
The development of an immune response against therapeutic factor VIII is the major complication in hemophilia A patients. Oligomannose carbohydrates at N239 and/or N2118 on factor VIII allow its binding to the macrophage mannose receptor expressed on human dendritic cells, thereby leading to factor VIII endocytosis and presentation to CD4+ T lymphocytes. Here, we investigated whether altering the interaction of factor VIII with mannose-sensitive receptors on antigen-presenting cells may be a strategy to reduce factor VIII immunogenicity. Gene transfer experiments in factor VIII-deficient mice indicated that N239Q and/or N2...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Conclusions: Low self-esteem in hemophilia patients indicates the importance of lifelong psychosocial support. Patients with pathologic HJHS are at risk of low-esteem. Using OSIQ with HJHS during follow-up of hemophilia patients may be useful for management.
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Online Articles: Original Articles Source Type: research
Replacement therapy with coagulation factor VIII (FVIII) represents the current clinical treatment for patients affected by hemophilia A (HA). This treatment while effective is, however, hampered by the formation of antibodies which inhibit the activity of infused FVIII in up to 30% of treated patients. Immune tolerance induction (ITI) protocols, which envisage frequent infusions of high doses of FVIII to confront this side effect, dramatically increase the already high costs associated to a patient's therapy and are not always effective in all treated patients. Therefore, there are clear unmet needs that must be addressed...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
CONCLUSION: This large, real-world data set indicates that rFIXFc activity in plasma samples can be accurately measured with the majority of routine OSC and CS assay methods. Given the variation in FIX assay procedures between sites, it is important that individual laboratories qualify their in-house methods for monitoring of rFIXFc activity. PMID: 32202380 [PubMed - as supplied by publisher]
Source: International Journal of Laboratory Hematology - Category: Hematology Authors: Tags: Int J Lab Hematol Source Type: research
Authors: Lillicrap D, Fijnvandraat K, Young G, Mancuso ME Abstract Introduction: Novel non-replacement therapies (e.g. emicizumab) have improved the management of patients with hemophilia A with and without inhibitors while introducing new challenges and increasing the complexity of clinical decision-making.Areas covered: Use of emicizumab can substantially delay initial exposure to FVIII thereby altering the natural history of inhibitor development, but it remains unclear whether later exposure to FVIII might modify the incidence of inhibitor development. Moreover, decisions regarding initiation of immune toleranc...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Conclusions: Though few patients have become positive for HBsAg and HIV Ab, HCV is still the major virus of concern for hemophiliacs who have undergone orthopedic procedures. Hepatitis B vaccination should be given to the high-risk population including hemophilia patients as soon as possible. PMID: 32190141 [PubMed]
Source: Archives of Medical Science - Category: General Medicine Tags: Arch Med Sci Source Type: research
Abstract Von Willebrand factor (VWF) and coagulation factor VIII (FVIII) circulate as a complex in plasma and have a major role in the hemostatic system. VWF has a dual role in hemostasis. It promotes platelet adhesion by anchoring the platelets to the subendothelial matrix of damaged vessels and it protects FVIII from proteolytic degradation. Moreover, VWF is an acute phase protein that has multiple roles in vascular inflammation and is massively secreted from Weibel-Palade bodies upon endothelial cell activation. Activated FVIII on the other hand, together with coagulation factor IX forms the tenase complex, an ...
Source: Sub-Cellular Biochemistry - Category: Biochemistry Authors: Tags: Subcell Biochem Source Type: research
CONCLUSIONS: after 12 weeks of observation, both approaches significantly reduced bleeds per animal and increased the proportion of bleed-free animals compared to controls (43% vs. 0%, respectively [AAV]; 75% vs. 8%, respectively [injection]). Both approaches resulted in an anti-FVIII inhibitory response in 20-37% of treated animals, similar to HA patients. Inhibitory antibodies were refractory to clinical improvement (reduction of bleeds) only in the AAV-based prophylaxis. An integrated model-based analysis of data on FVIII exposure and bleeding events was performed. This predicted the bleeding risk at any given circulati...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
"Whatever Works": Innovations in the Treatment of Hemophilia in the United States 1783-1950. Can Bull Med Hist. 2020 Mar 20;:e386092019 Authors: Rushton AR Abstract Treatment of the bleeding disorder hemophilia in the nineteenth century was empirical, based on clinical experience. Medications, transfusions of human or animal blood, and injections of blood sera were utilized in an attempt to halt life-threatening hemorrhages. After 1900, the application of clinical laboratory science facilitated the utilization of anti-coagulated blood and donor blood compatibility tests for safer emergency t...
Source: Medical History - Category: History of Medicine Authors: Tags: Can Bull Med Hist Source Type: research
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