Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria

Abstract To prevent bleeding in severe haemophilia A [SHA, defined as factor VIII (FVIII) activity < 1%] regular prophylactic FVIII replacement therapy is required, and the benefits of factor products with extended half-life (EHL) over traditional standard half-life (SHL) are still being debated....

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Autores principales:	Cihan Ay, Clemens Feistritzer, Joachim Rettl, Gerhard Schuster, Anna Vavrovsky, Leonard Perschy, Ingrid Pabinger
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/8e84b37a487c40b5a14d05ed968045e9
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spelling	oai:doaj.org-article:8e84b37a487c40b5a14d05ed968045e92021-12-02T18:02:49ZBleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria10.1038/s41598-021-92245-52045-2322https://doaj.org/article/8e84b37a487c40b5a14d05ed968045e92021-06-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-92245-5https://doaj.org/toc/2045-2322Abstract To prevent bleeding in severe haemophilia A [SHA, defined as factor VIII (FVIII) activity < 1%] regular prophylactic FVIII replacement therapy is required, and the benefits of factor products with extended half-life (EHL) over traditional standard half-life (SHL) are still being debated. We performed a multi-centre, retrospective cohort study of persons with SHA in Austria aiming to compare clinical outcomes and factor utilization in patients with SHA, who switched from prophylaxis with SHL to an EHL. Data were collected from haemophilia-specific patient diaries and medical records. Twenty male persons with SHA (median age: 32.5 years) were included. The most common reason for switching to the EHL was a high bleeding rate with SHL. Switch to rFVIII-Fc resulted in a significantly decreased annualized bleeding rate (ABR; median difference (IQR): − 0.3 (− 4.5–0); Wilcoxon signed-rank test for matched pairs: Z = − 2.7, p = 0.008) and number of prophylactic infusions per week (− 0.75 (− 1.0–0.0); Z = − 2.7, p = 0.007). Factor utilization was comparable to prior prophylaxis with SHL (0.0 (− 15.8–24.8) IU/kg/week; Z = − 0.4, p = 0.691). In summary, switch to EHL (rFVIII-Fc) was associated with an improved clinical outcome, reflected by ABR reduction, and less frequent infusions, without significantly higher factor usage.Cihan AyClemens FeistritzerJoachim RettlGerhard SchusterAnna VavrovskyLeonard PerschyIngrid PabingerNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-8 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Cihan Ay Clemens Feistritzer Joachim Rettl Gerhard Schuster Anna Vavrovsky Leonard Perschy Ingrid Pabinger Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
description	Abstract To prevent bleeding in severe haemophilia A [SHA, defined as factor VIII (FVIII) activity < 1%] regular prophylactic FVIII replacement therapy is required, and the benefits of factor products with extended half-life (EHL) over traditional standard half-life (SHL) are still being debated. We performed a multi-centre, retrospective cohort study of persons with SHA in Austria aiming to compare clinical outcomes and factor utilization in patients with SHA, who switched from prophylaxis with SHL to an EHL. Data were collected from haemophilia-specific patient diaries and medical records. Twenty male persons with SHA (median age: 32.5 years) were included. The most common reason for switching to the EHL was a high bleeding rate with SHL. Switch to rFVIII-Fc resulted in a significantly decreased annualized bleeding rate (ABR; median difference (IQR): − 0.3 (− 4.5–0); Wilcoxon signed-rank test for matched pairs: Z = − 2.7, p = 0.008) and number of prophylactic infusions per week (− 0.75 (− 1.0–0.0); Z = − 2.7, p = 0.007). Factor utilization was comparable to prior prophylaxis with SHL (0.0 (− 15.8–24.8) IU/kg/week; Z = − 0.4, p = 0.691). In summary, switch to EHL (rFVIII-Fc) was associated with an improved clinical outcome, reflected by ABR reduction, and less frequent infusions, without significantly higher factor usage.
format	article
author	Cihan Ay Clemens Feistritzer Joachim Rettl Gerhard Schuster Anna Vavrovsky Leonard Perschy Ingrid Pabinger
author_facet	Cihan Ay Clemens Feistritzer Joachim Rettl Gerhard Schuster Anna Vavrovsky Leonard Perschy Ingrid Pabinger
author_sort	Cihan Ay
title	Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
title_short	Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
title_full	Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
title_fullStr	Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
title_full_unstemmed	Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria
title_sort	bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia a in austria
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/8e84b37a487c40b5a14d05ed968045e9
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Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria

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