Roche presents a broad range of data for Hemlibra demonstrating continued benefits for people with haemophilia A at the ISTH 2019 Congress
Roche today announced new data for Hemlibra ® (emicizumab) across multiple pivotal studies in people with haemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on 6-10 July in Melbourne, Australia.
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...
CONCLUSIONS: Our findings suggest that a careful comprehensive geriatric assessment of acquired TTP patients is necessary. It is important to look for other autoimmune diseases and such age-related comorbidities as osteoporosis, arterial hypertension, ischemic heart disease and cerebrovascular disease. PMID: 32201091 [PubMed - as supplied by publisher]
CONCLUSION: This extension study demonstrated the long-term safety and efficacy of weekly rIX-FP in pediatric patients. Additionally, it showed that adequate bleed protection can be achieved with 10- or 14-day rIX-FP regimens in selected pediatric patients while maintaining safety. PMID: 32185782 [PubMed - as supplied by publisher]
This study investigated the myocardial functions, blood pressure changes, arterial stiffness, and risk factors associated with cardiovascular diseases in children with hemophilia.
BAY 81-8973 (Kovaltry ®), a recombinant factor VIII (rFVIII) product, was efficacious and well tolerated in paediatric previously treated patients (PTPs) with severe haemophilia A for ≥50 exposure days (EDs) in the LEOPOLD Kids study. Because long-term prophylaxis (≥100 EDs) can provide substantial patient benefits, FVIII products should demonstrate long-term safety and efficacy.
CONCLUSIONS: CM in vitro is procoagulant and prothrombotic. CM in vivo can augment myocardial damage and can be prohemostatic in the presence of bleeding. CM's procoagulant and antifibrinolytic activities likely involve, at least in part, its ability to bind factor Xa and enhance thrombin generation. Future work is needed to clarify CM's pathophysiology and its mechanistic influences on hemostasis or thrombosis. PMID: 32102568 [PubMed - as supplied by publisher]
CONCLUSIONS: rIX-FP extended interval prophylaxis provides dosing flexibility and, in selected patients, a 21-day regimen may provide an alternative option to minimize treatment burden and individualize treatment. PMID: 32078256 [PubMed - as supplied by publisher]
CONCLUSIONS: Herein, we demonstrate the aberrant impact of a FVIII synonymous mutation on its transcription, activity and pathological outcomes. Our data underline the importance of increasing the knowledge regarding the functional consequences of F8 mutations and their link to inhibitor development and an effective replacement therapy. PMID: 32078252 [PubMed - as supplied by publisher]
CONCLUSION: We demonstrated that the poly(T)-track in F8 intron 13 is a deletion hotspot. We recommend that deletions in this region should be specifically investigated in all genetically unresolved mild HA patients. PMID: 32073743 [PubMed - as supplied by publisher]
CONCLUSIONS: We conclude that in non-severe HA ABO and VWF levels do not substantially influence the variability of FVIII levels and age has only minor effects on it, which is important information for the diagnostic procedures. PMID: 32073230 [PubMed - as supplied by publisher]