European Commission approves Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors

Roche today announced that the European Commission has approved Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A (congenital factor VIII deficiency, FVIII
Source: Roche Media News - Category: Pharmaceuticals Source Type: news

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Acquired factor V deficiency (AFVD) is a rare autoimmune bleeding disorder. Unlike acquired hemophilia, bypass therapies with recombinant activated factor VII and activated prothrombin complex concentrates are ineffective for severe bleeding due to AFVD. Although several treatment strategies have been attempted, a standard of care for severe hemorrhage induced by AFVD is lacking. Herein, we report a case of AFVD with severe bleeding that responded to plasma exchange (PE) combined with immunosuppression. We also reviewed previously reported AFVD cases with severe hemorrhage and suggest that PE may be an effective initial tr...
Source: Acta Haematologica - Category: Hematology Source Type: research
Hemophilia A (HA) is a congenital bleeding disorder resulting from absence or deficiency of clotting factor VIII (FVIII). The symptoms consist of more or less severe hemorrhages as a result of trauma, surgical procedures, or spontaneous bleedings. These most likely occur into the joints (hemarthrosis) leading over the long term to progressive and irreversible hemophilic arthropathy, but bleeding may also occur into muscles, soft tissues and other sites (e.g. central nervous system) with potentially life-threatening consequences and, not least, drastic impairment in the individual's quality of life (QoL) [1].
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
Purpose of review Nonsevere hemophilia A (NSHA) patients have received relatively little clinical and research attention as compared with their severe counterparts. There is increasing recognition that despite their milder bleeding phenotype, the management of NSHA can be a challenge, with most management decisions largely inferred from severe hemophilia A data. This review focuses on some of the more recent developments in the field of NSHA. Recent findings Epidemiologic studies suggest that NSHA remain under-recognized and under-diagnosed globally. As the NSHA population ages, they are susceptible to age-related com...
Source: Current Opinion in Hematology - Category: Hematology Tags: HEMOSTASIS AND THROMBOSIS: Edited by Paul F. Bray Source Type: research
CONCLUSIONS: We were unable to identify any relevant trials on the efficacy and safety of rituximab for treating inhibitors in people with hemophilia. The research evidence available is from case reports and case series. Randomized controlled trials are needed to evaluate the efficacy and safety of rituximab for this condition. However, prior to the publication of any possible future randomized controlled trials, meta-analysis of case reports and case series may provide some evidence. PMID: 32761818 [PubMed - in process]
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
The deficiency or abnormal activity of von Willebrand factor, a multi-adhesive protein which binds platelets to exposed subendothelium and carries factor VIII in circulation, is responsible for von Willebrand disease, the most frequent inherited bleeding disorder. Clinical symptoms are characterized by mucous membrane and soft tissue bleeding, bleeding after surgery and rarely joint and gastrointestinal bleeding. Intriguingly, also factor VIII, the protein deficient in hemophilia A, may be variably reduced because VWF stabilizes it into circulation.
Source: Thrombosis Research - Category: Hematology Authors: Tags: Review Article Source Type: research
Abstract Hemophilia A (HA) and B (HB) are X-linked bleeding disorders caused by mutations in the F8 or F9 gene that result in the absence, or reduced activity, of the corresponding clotting factor. The severity of bleeding and related complications is proportional to the amount of residual circulating functional factor. The development of a safe and effective hemophilia treatment lasted several decades and has been mainly based on clotting factor replacement. Advances in the engineering and manufacturing of clotting concentrates have led to the widespread availability of extended half-life products that reduced th...
Source: Hamostaseologie - Category: Hematology Authors: Tags: Hamostaseologie Source Type: research
(Wake Forest Baptist Medical Center) WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
ier C Abstract Congenital haemophilia A (HA) is caused by deficiency of coagulation factor VIII (FVIII) activity, leading to spontaneous or traumatic bleeding events. While FVIII replacement therapy can treat and prevent bleeds, approximately 30% of patients with severe HA develop inhibitor antibodies that render FVIII replacement therapy ineffective. The bypassing agents (BPAs), activated prothrombin complex concentrate (aPCC) and recombinant activated FVII, first approved in 1977 and 1996, respectively, act to generate thrombin independent of pathways that involve factors IX and VIII. Both may be used in patient...
Source: Hamostaseologie - Category: Hematology Authors: Tags: Hamostaseologie Source Type: research
Abstract Hemophilia A, characterized by absent or ineffective coagulation factor VIII (FVIII), is a serious bleeding disorder that entails severe and potentially life-threatening bleeding events. Current standard therapy still involves replacement of FVIII, but is often complicated by the occurrence of neutralizing alloantibodies (inhibitors). Management of patients with inhibitors is challenging and necessitates immune tolerance induction for inhibitor eradication and the use of bypassing agents (activated prothrombin complex concentrates or recombinant activated factor VII), which are expensive and not always ef...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: Thromb Haemost Source Type: research
DISCUSSION: During the six-month clinical study period, the use of Kedrion FIX resulted in a safe and effective pd-FIX concentrate with excellent PK characteristics. PMID: 32712218 [PubMed - as supplied by publisher]
Source: European Journal of Pharmaceutical Sciences - Category: Drugs & Pharmacology Authors: Tags: Eur J Pharm Sci Source Type: research
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