Low incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A treated with octanate((R)): Final report from a prospective study

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F18%3A10375312" target="_blank" >RIV/00216208:11130/18:10375312 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00064203:_____/18:10375312

Výsledek na webu

<a href="https://doi.org/10.1111/hae.13385" target="_blank" >https://doi.org/10.1111/hae.13385</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1111/hae.13385" target="_blank" >10.1111/hae.13385</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Low incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A treated with octanate((R)): Final report from a prospective study

Popis výsledku v původním jazyce

IntroductionOctanate((R)) is a human, plasma-derived, von Willebrand factor-stabilized coagulation factor VIII (FVIII) concentrate with demonstrated haemostatic efficacy in previously treated patients with haemophilia A. AimThis prospective, open-label study aimed to assess the immunogenicity of octanate((R)) in previously untreated patients (PUPs). MethodsThe study monitored development of FVIII inhibitors in 51 PUPs. Tolerability, viral safety, FVIII recovery and efficacy of octanate((R)) for the prevention and treatment of bleeds and in surgical procedures were also assessed. ResultsFive (9.8%) of the 51 patients developed inhibitors during the study, 4 of which (7.8%) were high titre. Three inhibitor cases (5.9%) were considered clinically relevant; 2 were transient inhibitors that disappeared during regular octanate((R)) treatment without a change in dose or treatment frequency. Amongst 45 patients with FVIII:C &lt;1% at baseline and who received 20 exposure days (EDs) or had &lt;20 EDs but developed an inhibitor, inhibitor incidence was 11.1% (6.7% clinically relevant). All clinically relevant inhibitors developed within 20EDs of on-demand treatment. No inhibitors developed in PUPs receiving prophylaxis. All patients who developed inhibitors had either intron 22 inversions or large deletions. Irrespective of the reason for administration, haemostatic efficacy was rated as excellent in 99.6% of all infusions (4700 of 4717 infusions), and no complications were reported in 23 surgical procedures. Mean incremental in vivo recovery was 2.0%/IU/kg (0.7) and 1.9%/IU/kg (+/- 0.5) for the first and second assessments, respectively. Tolerability was rated very good in 99.9% of infusions. ConclusionIn PUPs with severe haemophilia A, octanate((R)) demonstrated haemostatic efficacy with a low rate of inhibitor development.

Název v anglickém jazyce

Low incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A treated with octanate((R)): Final report from a prospective study

Popis výsledku anglicky

IntroductionOctanate((R)) is a human, plasma-derived, von Willebrand factor-stabilized coagulation factor VIII (FVIII) concentrate with demonstrated haemostatic efficacy in previously treated patients with haemophilia A. AimThis prospective, open-label study aimed to assess the immunogenicity of octanate((R)) in previously untreated patients (PUPs). MethodsThe study monitored development of FVIII inhibitors in 51 PUPs. Tolerability, viral safety, FVIII recovery and efficacy of octanate((R)) for the prevention and treatment of bleeds and in surgical procedures were also assessed. ResultsFive (9.8%) of the 51 patients developed inhibitors during the study, 4 of which (7.8%) were high titre. Three inhibitor cases (5.9%) were considered clinically relevant; 2 were transient inhibitors that disappeared during regular octanate((R)) treatment without a change in dose or treatment frequency. Amongst 45 patients with FVIII:C &lt;1% at baseline and who received 20 exposure days (EDs) or had &lt;20 EDs but developed an inhibitor, inhibitor incidence was 11.1% (6.7% clinically relevant). All clinically relevant inhibitors developed within 20EDs of on-demand treatment. No inhibitors developed in PUPs receiving prophylaxis. All patients who developed inhibitors had either intron 22 inversions or large deletions. Irrespective of the reason for administration, haemostatic efficacy was rated as excellent in 99.6% of all infusions (4700 of 4717 infusions), and no complications were reported in 23 surgical procedures. Mean incremental in vivo recovery was 2.0%/IU/kg (0.7) and 1.9%/IU/kg (+/- 0.5) for the first and second assessments, respectively. Tolerability was rated very good in 99.9% of infusions. ConclusionIn PUPs with severe haemophilia A, octanate((R)) demonstrated haemostatic efficacy with a low rate of inhibitor development.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30204 - Oncology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2018

Kód důvěrnosti údajů

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

Název periodika

Haemophilia

ISSN

1351-8216

e-ISSN

—

Svazek periodika

24

Číslo periodika v rámci svazku

2

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

8

Strana od-do

221-228

Kód UT WoS článku

000428795000020

EID výsledku v databázi Scopus

2-s2.0-85044579919