octanate (R): over 20 years of clinical experience in overcoming challenges in haemophilia A treatment

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F20%3A10410727" target="_blank" >RIV/00216208:11130/20:10410727 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00064203:_____/20:10410727

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=oK3HZJ5Rv4" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=oK3HZJ5Rv4</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1177/2040620720914692" target="_blank" >10.1177/2040620720914692</a>

Jazyk výsledku

angličtina

Název v původním jazyce

octanate (R): over 20 years of clinical experience in overcoming challenges in haemophilia A treatment

Popis výsledku v původním jazyce

Treatment of haemophilia A with FVIII replacement has evolved over the past decades to adapt to the needs of patients. octanate (R), a plasma-derived, double virus-inactivated, von Willebrand factor (VWF)-containing FVIII concentrate, has been used in clinics worldwide for over 20 years. First licensed in 1998 in Germany, octanate (R) is approved in over 80 countries for the prevention and treatment of bleeding and for surgical prophylaxis in patients with haemophilia A, and in over 40 countries for immune tolerance induction (ITI). The manufacturing process for octanate (R) was developed to ensure high viral safety and effectively eliminates both enveloped and nonenveloped viruses. Over the past 20 years, the excellent safety and efficacy of octanate (R) have been demonstrated in pivotal clinical trials in adult and paediatric previously treated patients (PTPs) for on-demand treatment, prophylaxis and as surgical cover. Importantly, octanate (R) has displayed low immunogenicity in previously untreated patients (PUPs), with only 9.8% of PUPs developing FVIII inhibitors. octanate (R) has also shown to be highly effective in inhibitor elimination when used as ITI therapy. In a population of patients with high risk of ITI failure, success was achieved in 79.2% of patients (70.8% complete success), even when using exceptionally stringent success criteria. No relapses were observed. Here we present an overview of the clinical data with octanate (R) that support its use in a range of patient populations and clinical indications.

Název v anglickém jazyce

octanate (R): over 20 years of clinical experience in overcoming challenges in haemophilia A treatment

Popis výsledku anglicky

Treatment of haemophilia A with FVIII replacement has evolved over the past decades to adapt to the needs of patients. octanate (R), a plasma-derived, double virus-inactivated, von Willebrand factor (VWF)-containing FVIII concentrate, has been used in clinics worldwide for over 20 years. First licensed in 1998 in Germany, octanate (R) is approved in over 80 countries for the prevention and treatment of bleeding and for surgical prophylaxis in patients with haemophilia A, and in over 40 countries for immune tolerance induction (ITI). The manufacturing process for octanate (R) was developed to ensure high viral safety and effectively eliminates both enveloped and nonenveloped viruses. Over the past 20 years, the excellent safety and efficacy of octanate (R) have been demonstrated in pivotal clinical trials in adult and paediatric previously treated patients (PTPs) for on-demand treatment, prophylaxis and as surgical cover. Importantly, octanate (R) has displayed low immunogenicity in previously untreated patients (PUPs), with only 9.8% of PUPs developing FVIII inhibitors. octanate (R) has also shown to be highly effective in inhibitor elimination when used as ITI therapy. In a population of patients with high risk of ITI failure, success was achieved in 79.2% of patients (70.8% complete success), even when using exceptionally stringent success criteria. No relapses were observed. Here we present an overview of the clinical data with octanate (R) that support its use in a range of patient populations and clinical indications.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

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OECD FORD obor

30205 - Hematology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2020

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Therapeutic Advances in Hematology

ISSN

2040-6207

e-ISSN

—

Svazek periodika

11

Číslo periodika v rámci svazku

April

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

12

Strana od-do

2040620720914692

Kód UT WoS článku

000529418600001

EID výsledku v databázi Scopus

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