Economic impact model of delayed inhibitor development in patients with hemophilia A receiving emicizumab for the prevention of bleeding events.
Conclusions: This study suggests that prophylaxis with emicizumab results in cost savings compared to FVIII prophylaxis in HA. PMID: 31530050 [PubMed - as supplied by publisher]
ConclusionsHemophilia A with high-titer inhibitors is a disease that generates significant pressure on the Colombian health system, mainly linked to the cost of factors and bypassing agents.
We present a case of a 68-year-old Hispanic female with a history of factor VII deficiency who presented with shortness of breath, chest pain, and palpitations and was found to have pulmonary embolism. Our patient did not have any of the above-mentioned thrombotic risk factors. Our case and review of the literature show that factor VII deficiency does not provide protection against thrombosis. PMID: 31590173 [PubMed - as supplied by publisher]
An 85-year-old woman was admitted for examination of anemia and black stools. A blood test revealed a reduction in her hemoglobin level from 12.7 to 6.6 g/dL (7.9×103 to 4.1×103 mol/L) over a month, suggesting upper gastrointestinal bleeding. Esophagogastroduodenoscopy revealed a large esophageal polyp near the esophagogastric junction (Figure 1). The polyp that was fragile was accidentally resected and removed endoscopically. Histopathological examination of the resected specimen revealed a hematoma containing numerous Enterococcus faecalis bacterial masses without epithelial tissue (Figure 2).
CONCLUSIONS: These results show that low-normal FV levels can enhance thrombin generation in hemophilia A. Further explorations with the mathematical model suggest a potential mechanism: lowering FV reduces competition between FV and FVIII for FXa on activated platelet surfaces (APS), which enhances FVIII activation and rescues thrombin generation in FVIII-deficient blood. PMID: 31562694 [PubMed - as supplied by publisher]
CONCLUSION: This population PK model derived from real world data adequately describes FVIII levels following perioperative administration of the FVIII/VWF plasma-derived concentrate (Haemate® P/Humate P®) and will help to facilitate future dosing in VWD patients. PMID: 31557387 [PubMed - as supplied by publisher]
CONCLUSIONS: Our results provide HLAcII peptidomic level explanations for several important clinical observations/issues including the differential immunogenicity of distinct FVIIIs and the role of HLAcII genetics in inhibitor development. PMID: 31556206 [PubMed - as supplied by publisher]
ConclusionsThese findings suggest that SHED-HLC-based micro-hepatic tissues might be a promising source for treating pediatric refractory diseases, including chronic liver fibrosis and hemophilia A.
CONCLUSIONS: Increasing the affinity of rFVIIa to activated platelets resulted in approximately 50-fold increased potency both in vitro and in the mouse model. The correlation of in vivo with in vitro data using maximally activated platelets supports that these assay conditions are relevant when evaluating platelet-targeted haemostatic concepts. PMID: 31549480 [PubMed - as supplied by publisher]
AbstractEmicizumab (Hemlibra®), a recombinant, humanized, bispecific monoclonal antibody, restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab is approved in several countries, including in the USA and Japan, for the routine prophylaxis of bleeding episodes in patients with haemophilia A with or without FVIII inhibitors. It is also approved in the EU for the routine prophylaxis of bleeding episodes in patients with haemophilia A with inhibitors or severe haemophilia A without inhibitors. In phas...
CONCLUSION: As hemophilia is an orphan disease, patients are mainly treated in specialized centers. For patients who live far from these centers or have limited access to a training there for other reasons, the physical medicine consultation hour and the implementation of online exercise instructions offer individually adapted exercise information for a regular home-based training to benefit from increased physical fitness and joint stability. PMID: 31535221 [PubMed - as supplied by publisher]