The therapeutic approach to genetic bleeding disorders has had a big and rapid evolution since the second half of the XX century thanks to the production of the first plasma-derived concentrate of coagulation factor. However, the lack of viral inactivation procedures of the concentrates, and the lack of knowledge about the possibility of transmission of viral agents using the same, has caused in the population of hemophilic patients epidemic of HIV and HCV-relate infection . From 1985 onwards, the concentrated plasma derivatives are subjected to viral inactivation techniques, also combined with increasingly effective technology. In addition, the accurate viral donor screening tests become widely available using led to the disappearance of new infections by the most feared pathogens. Moreover, the possibility of transmitting agents such as parvovirus B19, also low pathogenic, or prions, associated with Creutzfeldt-Jakob disease, are reasons to maintain high surveillance of plasma-derived products. There could be also the possibility that micro-organisms responsible for outbreaks of viral haemorrhagic fevers in Africa, or West Nile virus can be transmit through blood. Since 1990, the concentrates obtained in recombinant cell culture thanks to the techniques of recombinant DNA and subjected to multiple purification steps and in the treatment of viral inactivation have conferred the advantage of an almost absolute security of not transmitting viral infections.
7 September 2021
DAMOCTOCOG ALFA PEGOL: A REVIEW IN HAEMOPHILIA A
Damoctocog alfa pegol (Jivi®) is approved in the USA, EU, Japan and Canada for the treatment and prophylaxis of previously treated patients aged ≥ 12 years with haemophilia A. Formulated with a 60 kDa polyethylene glycol (PEG) moiety, damoctocog alfa pegol is an intravenously (IV) administered recombinant factor VIII (rFVIII) product with a longer terminal half-life than non-PEGylated FVIII […]
7 September 2021
Health-related quality-of-life and treatment satisfaction of individuals sati with hemophilia A treated with turoctocog alfa pegol (n8-gP): a new recombinant extended half-life FVIII
Prophylactic treatment regimens lead to improvements in health-related quality- of-life (HRQoL) among individuals with hemophilia. Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction (TS). To investigate the HRQoL and TS of patients with […]
15 March 2021
Target joint resolution in patients with haemophilia A receiving long‐term prophylaxis with BAY 94‐9027
BAY 94‐9027 (damoctocog alfa pegol; Jivi®; Bayer AG, Germany) is an extended half‐life B‐domain–deleted, site‐specifically PEGylated recombinant FVIII. Due to its prolonged half‐life, BAY 94‐9027 has the potential to maintain FVIII at a higher haemostatic level for longer periods versus standard‐acting agents.This longer half‐life also allows less frequent dosing, which in turn could help to […]
18 January 2021
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A
Factor VIII replacement products have improved the care of patients with hemophilia A, but the short half-life of these products affects the patients’ quality of life. The half-life of recombinant factor VIII ranges from 15 to 19 hours because of the von Willebrand factor chaperone effect. BIVV001 (rFVIIIFc-VWF-XTEN) is a novel fusion protein designed to […]
25 November 2020
Target joint resolution in patients with haemophilia A receiving long‐term prophylaxis with BAY 94‐9027
BAY 94‐9027 (damoctocog alfa pegol; Jivi®; Bayer AG, Germany) is an extended half‐life B‐domain–deleted, site‐specifically PEGylated recombinant FVIII. Due to its prolonged half‐life, BAY 94‐9027 has the potential to maintain FVIII at a higher haemostatic level for longer periods versus standard‐acting agents.This longer half‐life also allows less frequent dosing, which in turn could help to […]
27 January 2020
DAMOCTOCOG ALFA PEGOL: A REVIEW IN HAEMOPHILIA A
Damoctocog alfa pegol (Jivi®) is approved in the USA, EU, Japan and Canada for the treatment and prophylaxis of previously treated patients aged ≥ 12 years with haemophilia A. Formulated with a 60 kDa polyethylene glycol (PEG) moiety, damoctocog alfa pegol is an intravenously (IV) administered recombinant factor VIII (rFVIII) product with a longer terminal half-life than non-PEGylated FVIII […]
24 January 2020
Health-related quality-of-life and treatment satisfaction of individuals sati with hemophilia A treated with turoctocog alfa pegol (n8-gP): a new recombinant extended half-life FVIII
Prophylactic treatment regimens lead to improvements in health-related quality- of-life (HRQoL) among individuals with hemophilia. Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction (TS). To investigate the HRQoL and TS of patients with […]
21 January 2020
Status of Recombinant Factor VIII Concentrate Treatment for Hemophilia a in Italy: Characteristics and Clinical Benefits
The current interest in recombinant factor VIII (rFVIII) products stems from the fact that they offer a technological solution to prolonging the half-life of and reducing the risk of formation of alloantibodies (inhibitors) against FVIII in treated patients with hemophilia A (HA). The Italian health care system has authorized the use of a wide range […]
19 January 2020
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
Adeno-associated virus (AAV)-mediated gene therapy is a promising treatment option for patients with hemophilia A. Up-to-date 3 years follow-up on the efficacy and safety of a single administration of AAV5-hFVIII-SQ are available. Fifteen subjects, older than 18 years, affected by severe hemophilia A (factor VIII level, ≤1 IU per deciliter) were treated with a single […]
17 January 2020
Laboratory measurement of factor replacement therapies in the treatment of congenital haemophilia: A United Kingdom Haemophilia Centre Doctors’ Organisation guideline
Discrepancies among several assays results can be observed within the laboratory monitoring of FVIII and FIX replacement therapy, this is especially true for the extended half‐life products. This guideline has evaluated available published data and advices on the assays of choice for laboratory measurement of replacement therapy in Haemophilia A and B without inhibitors. Each […]
15 January 2020
A Molecular Revolution in the Treatment of Hemophilia
A recently published review has focused on the evolution of treatment landscape of hemophilia A and B, two monogenetic bleeding disorders (coagulation factor VIII and IX deficiency) treated with systemic protein replacement therapy up to the last ten years. Now, several drugs, ranging from antibody to gene to RNA therapy, are transforming this treatment. Replacement […]
16 October 2019
Comorbidities in hemophilia not related to hemophilia and therapeutic prescription concerns
The longest life expectancy makes hemophiliacs susceptible to diseases not related to haemophilia and belonging to the general population as cerebro-cardiovascular pathologies and malignacies. Predictable is also that the younger patients, children and adolescents, suffer from disorders caused by well-being: obesity, metabolic syndrome, abnormal glucose tolerance. In severe elderly hemophiliacs chronic arthropathy is the most […]
2 November 2018
Prophylaxis with emicizumab in hemophiliacs A without inhibitor
Emicizumab represents a therapeutic innovation not only for haemophiliacs A with inhibitor but also for those without inhibitor. This is what the recently published Haven 3 study has shown. Thephase 3 study was performed on 152 enrolled patients aged ≥ 12 years and randomly assigned witha 2: 2: 1 ratio in 4 groups in order […]
20 December 2017
A promising approach for treatment of severe hemophilia A patients with inhibitor.
Current standard therapy for severe hemophilia A is regular infusions of either plasma-derived or recombinant factor VIII (FVIII) for prophylaxis and for treating bleeding episodes when they occur (“on demand” therapy). Nowadays the most important complication of replacement therapy is that a significant number of patients develop neutralizing alloantibodies (inhibitors) against exogenous FVIII. Inhibitors are […]
9 November 2017
Brief report on gene therapy for severe hemophilia
Patients affected by severe hemophilia A and B must be treated with plasmaderived or recombinant factor replacement with significant improvement in morbidity and mortality, as well as in their quality of life. Nowadays prophylaxis represents the golden standard for the management of hemophilia, using fixed doses of factor VIII/factor IX (FVIII/FIX) concentrates at fixed timing […]
10 October 2017
Recent innovations in replacement and no-replacement therapy of hemophilia
The most important and prevalent inherited bleeding disorders are hemophilia A and B, rare recessive X-linked diseases (1/5.000 and 1/30.000 male respectively). The severity of disesase is based on the levels of factor VIII (FVIII) and factor IX (FIX) into the bloodstream: <1% (severe type), 1 to 5% (moderate type), >5 to <40% (mild type). […]
9 March 2017
A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects
ACE910 is a recombinant humanized bispecific antibody that binds to activated factor IX and factor X and mimics the cofactor function of factor VIII (FVIII). This first-in-human study examined the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ACE910 in healthy male adults. A total of 40 Japanese and 24 white subjects were randomized to […]