How we choose factor VIII to treat hemophilia.

@article{Mannucci2012HowWC,
  title={How we choose factor VIII to treat hemophilia.},
  author={Pier Mannuccio Mannucci and Maria Elisa Elisa Mancuso and Elena Santagostino},
  journal={Blood},
  year={2012},
  volume={119 18},
  pages={
          4108-14
        }
}
In high-income countries, the large availability of coagulation factors for replacement therapy of patients with hemophilia A has raised the life expectancy of these lifelong bleeders to that of males from the general population. The practicing clinician is offered a multitude of choices among several commercial brands of factor VIII extracted from human plasma or engineered from mammalian cell cultures by means of recombinant DNA technology. This article has the goal to offer our opinions on… 

Tables from this paper

Recommendations for factor VIII product source to treat patients with haemophilia A.
TLDR
The striking current novelty is that the results of SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers), a study with a randomised design, have transformed the aforementioned perception into evidence, and that this new information should be now conveyed without ambiguity to patients during the decision-making process.
The increased demand for plasma-derived factor VIII in Italy.
TLDR
A significant increase in the demand for plasma-derived FVIII was recorded between 2011 and 2014 in Italy, one of the highest in Europe even though with a large variability across the Italian regions.
Emerging drugs for the treatment of hemophilia A and B
TLDR
The emerging drugs for hemophilia treatment seem to be very promising and the extended half-life of FVIII/IX concentrates will improve the adherence of patients to therapy.
Tailoring hemostatic therapies to lower inhibitor development in previously untreated patients with severe hemophilia A
TLDR
Interest is currently directed toward the potential for lesser immunogenicity of novel hemostatic agents designed to decrease the dosing frequency or avoid/delay the need of FVIII replacement therapy.
Factor VIII products in haemophilia A: one size fits all?
TLDR
The development of alloantibodies (inhibitors) that inactivate the coagulant activity of factor VIII (FVIII) is the major problem for the management of persons with severe haemophilia A and it is theoretically possible to decrease the inhibitor cumulative incidence by acting upon modifiable risk factors.
The inhibitors – a challenge for the management of patients with hereditary haemophilia A
  • R. Mihăilă
  • Medicine, Biology
    Romanian journal of internal medicine = Revue roumaine de medecine interne
  • 2018
TLDR
Plasma-derived factor VIII is less immunogenic, but not entirely safe from the point of view of the possibility of transmitting biological agents, and plasma-derived or recombinant factor VIII products must co-exist on the market for the benefit of haemophilic patients.
The history of hemophilia.
TLDR
The discovery of "antihemophilic globulin" in the middle of the 20th century paved the way to the production of cryoprecipitate and then of FVIII and FIX concentrates, which revolutionized the treatment of hemophilia through the widespread adoption of home treatment and prophylaxis regimens, which dramatically improved the quality of life and life expectancy of persons with hemophilias during the past decade.
2017 Clinical trials update: Innovations in hemophilia therapy
TLDR
The nonfactor replacement strategies provide a promising treatment option for patients with inhibitors, presently the greatest unmet medical need in hemophilia.
The current and future role of plasma-derived clotting factor concentrate in the treatment of haemophilia A.
  • B. Kevane, N. O'Connell
  • Biology, Medicine
    Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis
  • 2018
Evolution of the European guidelines for the clinical development of factor VIII products: little progress towards improved patient management
  • P. Mannucci
  • Medicine
    Haemophilia : the official journal of the World Federation of Hemophilia
  • 2013
TLDR
This article reviews the evolution of the European regulations on new FVIII products, lists a number of regulatory requirements whose scientific and/or clinical rationale is perhaps questionable and recommends keeping such requirements in reasonable limits of feasibility, without jeopardizing current high standards of efficacy and safety.
...
1
2
3
4
5
...

References

SHOWING 1-10 OF 75 REFERENCES
Plasma-derived versus recombinant factor VIII concentrates for the treatment of haemophilia A: plasma-derived is better.
  • P. Mannucci
  • Medicine, Biology
    Blood transfusion = Trasfusione del sangue
  • 2010
TLDR
The most challenging complication of current therapy has become the development of inhibitory alloantibodies, which renders replacement therapies ineffective, limit the access of patients to a safe and effective standard of care and predispose them to an unacceptably high risk of morbidity and mortality.
Human recombinant DNA-derived antihemophilic factor (factor VIII) in the treatment of hemophilia A. recombinant Factor VIII Study Group.
TLDR
Recombinant factor VIII has biologic activity comparable to that of plasma factor VIII and is safe and efficacious for the treatment of hemophilia A.
Role of von Willebrand factor in immune tolerance induction
TLDR
Current data support use of FVIII concentrates containing VWF in immune tolerance induction; other variables may also contribute to the relative success of this treatment.
Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates.
TLDR
Despite a lower recovery compared with pdFIX, rFIX controlled hemorrhage in a wide variety of settings and may provide a safety advantage in terms of risk from blood-borne pathogens.
Back to the future: a recent history of haemophilia treatment
  • P. Mannucci
  • Biology, Medicine
    Haemophilia : the official journal of the World Federation of Hemophilia
  • 2008
TLDR
It is likely that further improvements in replacement therapy will occur in the near future, through the availability of new‐therapeutic tools such as factors VIII and IX with longer half‐lives, more potent bypassing agents and factors extracted from the milk of transgenic animals.
Immune tolerance therapy utilizing factor VIII/von Willebrand factor concentrate in haemophilia A patients with high titre factor VIII inhibitors
TLDR
This retrospective study describes 25 patients who were considered poor prognosis because of clinical and laboratory characteristics, which made ITI less likely to be successful or because of a poor response to initial ITI with a monoclonal/recombinant FV III concentrate, who were treated with FVIII/VWF concentrate as part of their ITI.
Hemophilia treatment in developing countries: products and protocols.
TLDR
There is wide variability not only in the types of products used but also in the doses administered for control or treatment of bleeding in different countries, which shows that the combined experience in the developing world in providing hemophilia services should be used to define standards of care that are practical and to set achievable goals.
French previously untreated patients with severe hemophilia A after exposure to recombinant factor VIII : incidence of inhibitor and evaluation of immune tolerance.
TLDR
Immune tolerance was difficult to achieve in French previously untreated patients with severe hemophilia A despite a follow-up period of 16 to 30 months: immune tolerance was complete in only one out of the 3 patients undergoing low dose ITR and in one of the 5 patients with high dose I TR.
Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly purified plasma-derived concentrate.
TLDR
Patients with severe hemophilia A received only one brand of highly purified factor VIII concentrate prepared by conventional chromatography with a solvent-detergent step for viral inactivation showed a low incidence of inhibitor compared with previous studies using high purity plasma-derived or recombinant products.
...
1
2
3
4
5
...