Literature DB >> 22411872

How we choose factor VIII to treat hemophilia.

Pier Mannuccio Mannucci1, Maria Elisa Mancuso, Elena Santagostino.   

Abstract

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 how to choose among the different products, that we consider interchangeable relevant to their clinical efficacy in the control of bleeding and safety from pathogen transmission. Hence, the main determinants of our choices are price and the risk of occurrence of factor VIII inhibitory alloantibodies. With this as background, we present the rationale underlying the choices for different categories of patients with severe hemophilia A: previously untreated patients, multiply treated patients, and patients undergoing immune tolerance induction with large doses of factor VIII to eradicate inhibitors. Mention is also made to the possible strategies that should be implemented to make available coagulation factors for replacement therapy in developing countries.

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Year:  2012        PMID: 22411872     DOI: 10.1182/blood-2012-01-394411

Source DB:  PubMed          Journal:  Blood        ISSN: 0006-4971            Impact factor:   22.113


  26 in total

Review 1.  Present and future challenges in the treatment of haemophilia: the patient's perspective.

Authors:  Romano Arcieri; Angelo C Molinari; Stefania Farace; Giuseppe Mazza; Alberto Garnero; Gabriele Calizzani; Paola Giordano; Emily Oliovecchio; Lorenzo Mantovani; Lamberto Manzoli; Paul Giangrande
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

Review 2.  Present and future challanges in the treatment of haemophilia: a clinician's perspective.

Authors:  Pier Mannuccio Mannucci; Massimo Franchini
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

Review 3.  The demand for factor VIII and for factor IX and the toll fractionation product surplus management.

Authors:  Gabriele Calizzani; Samantha Profili; Fabio Candura; Monica Lanzoni; Stefania Vaglio; Livia Cannata; Giancarlo M Liumbruno; Massimo Franchini; Pier Mannuccio Mannucci; Giuliano Grazzini
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

Review 4.  Plasma-derived medicinal products self-sufficiency from national plasma: to what extent?

Authors:  Vincenzo De Angelis; Antonio Breda
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

5.  Pathogen safety of long-term treatments for bleeding disorders: still relevant to current practice.

Authors:  Giovanni Di Minno; Mariana Canaro; James W Ironside; David Navarro; Carlo Federico Perno; Andreas Tiede; Lutz Gürtler
Journal:  Haematologica       Date:  2013-10       Impact factor: 9.941

6.  High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis.

Authors:  Andrea Messori; Flora Peyvandi; Sabrina Trippoli; Roberta Palla; Frits R Rosendaal; Pier Mannuccio Mannucci
Journal:  Blood Transfus       Date:  2017-05-15       Impact factor: 3.443

7.  Recommendations for factor VIII product source to treat patients with haemophilia A.

Authors:  Romano Arcieri; Gabriele Calizzani; Fabio Candura; Pier Mannuccio Mannucci
Journal:  Blood Transfus       Date:  2017-03-02       Impact factor: 3.443

8.  The increased demand for plasma-derived factor VIII in Italy.

Authors:  Romano Arcieri; Gabriele Calizzani; Fabio Candura; Pier Mannuccio Mannucci
Journal:  Blood Transfus       Date:  2016-07-28       Impact factor: 3.443

9.  Factor VIII inhibitors: von Willebrand factor makes a difference in vitro and in vivo.

Authors:  Q Shi; E L Kuether; J A Schroeder; C L Perry; S A Fahs; J Cox Gill; R R Montgomery
Journal:  J Thromb Haemost       Date:  2012-11       Impact factor: 5.824

10.  Factor VIII inhibitors in hemophilia A: rationale and latest evidence.

Authors:  Char Witmer; Guy Young
Journal:  Ther Adv Hematol       Date:  2013-02
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