Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life

Abstract Introduction In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient‐reported outcomes (PROs) and health‐related quality of life (HRQoL) are important to evaluate with new treatments. Aims To asses...

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Autores principales:	Mairead O’Donovan, Eimear Quinn, Kate Johnston, Evelyn Singleton, Julie Benson, Brian O'Mahony, Declan Noone, Cleona Duggan, Ruth Gilmore, Kevin Ryan, James S. O'Donnell, Niamh M. O’Connell
Formato:	article
Lenguaje:	EN
Publicado:	Wiley 2021
Materias:	extended half‐life concentrate hemophilia B health‐related quality of life pain patient‐reported outcomes Diseases of the blood and blood-forming organs RC633-647.5
Acceso en línea:	https://doaj.org/article/cce7db211eaf4a6f804488289b87edbe
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spelling	oai:doaj.org-article:cce7db211eaf4a6f804488289b87edbe2021-11-29T09:35:28ZRecombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life2475-037910.1002/rth2.12602https://doaj.org/article/cce7db211eaf4a6f804488289b87edbe2021-10-01T00:00:00Zhttps://doi.org/10.1002/rth2.12602https://doaj.org/toc/2475-0379Abstract Introduction In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient‐reported outcomes (PROs) and health‐related quality of life (HRQoL) are important to evaluate with new treatments. Aims To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis. Methods Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following two years of rFIXFc prophylaxis. The PROBE questionnaire has four domains: demographics, general health, haemophilia‐specific, and European Quality of Life 5‐Dimensions (EQ‐5D‐5L) questionnaire. Results Twenty‐three participants completed the questionnaire at both time points. The number of activities where chronic pain occurred and interfered with the activity was reduced by 25% and 33%, respectively (P < .001), following two years of rFIXFc prophylaxis. There was a 9% decrease in chronic pain during the second year of rFIXFc prophylaxis compared to baseline, but the rate remained high, at 74%. A 25% reduction in the number of affected activities of daily living (ADLs) was reported following 2 years of rFIXFc prophylaxis (P = .007). The most common health problems were arthritis, hypertension, anxiety/depression, and gingivitis. The median EQ‐5D‐5L score was similar following two years of rFIXFc prophylaxis, 0.76 (range, −0.01 to 0.95), compared to 0.77 (range, 0.36‐1) at baseline. Conclusion This study of real‐world patient experience using PROs demonstrates a reduction in chronic pain and improvement in ADLs in participants after switching to rFIXFc prophylaxis. It provides important insights into patient‐identified health care needs and living with severe hemophilia B.Mairead O’DonovanEimear QuinnKate JohnstonEvelyn SingletonJulie BensonBrian O'MahonyDeclan NooneCleona DugganRuth GilmoreKevin RyanJames S. O'DonnellNiamh M. O’ConnellWileyarticleextended half‐life concentratehemophilia Bhealth‐related quality of lifepainpatient‐reported outcomesDiseases of the blood and blood-forming organsRC633-647.5ENResearch and Practice in Thrombosis and Haemostasis, Vol 5, Iss 7, Pp n/a-n/a (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	extended half‐life concentrate hemophilia B health‐related quality of life pain patient‐reported outcomes Diseases of the blood and blood-forming organs RC633-647.5
spellingShingle	extended half‐life concentrate hemophilia B health‐related quality of life pain patient‐reported outcomes Diseases of the blood and blood-forming organs RC633-647.5 Mairead O’Donovan Eimear Quinn Kate Johnston Evelyn Singleton Julie Benson Brian O'Mahony Declan Noone Cleona Duggan Ruth Gilmore Kevin Ryan James S. O'Donnell Niamh M. O’Connell Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
description	Abstract Introduction In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient‐reported outcomes (PROs) and health‐related quality of life (HRQoL) are important to evaluate with new treatments. Aims To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis. Methods Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following two years of rFIXFc prophylaxis. The PROBE questionnaire has four domains: demographics, general health, haemophilia‐specific, and European Quality of Life 5‐Dimensions (EQ‐5D‐5L) questionnaire. Results Twenty‐three participants completed the questionnaire at both time points. The number of activities where chronic pain occurred and interfered with the activity was reduced by 25% and 33%, respectively (P < .001), following two years of rFIXFc prophylaxis. There was a 9% decrease in chronic pain during the second year of rFIXFc prophylaxis compared to baseline, but the rate remained high, at 74%. A 25% reduction in the number of affected activities of daily living (ADLs) was reported following 2 years of rFIXFc prophylaxis (P = .007). The most common health problems were arthritis, hypertension, anxiety/depression, and gingivitis. The median EQ‐5D‐5L score was similar following two years of rFIXFc prophylaxis, 0.76 (range, −0.01 to 0.95), compared to 0.77 (range, 0.36‐1) at baseline. Conclusion This study of real‐world patient experience using PROs demonstrates a reduction in chronic pain and improvement in ADLs in participants after switching to rFIXFc prophylaxis. It provides important insights into patient‐identified health care needs and living with severe hemophilia B.
format	article
author	Mairead O’Donovan Eimear Quinn Kate Johnston Evelyn Singleton Julie Benson Brian O'Mahony Declan Noone Cleona Duggan Ruth Gilmore Kevin Ryan James S. O'Donnell Niamh M. O’Connell
author_facet	Mairead O’Donovan Eimear Quinn Kate Johnston Evelyn Singleton Julie Benson Brian O'Mahony Declan Noone Cleona Duggan Ruth Gilmore Kevin Ryan James S. O'Donnell Niamh M. O’Connell
author_sort	Mairead O’Donovan
title	Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
title_short	Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
title_full	Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
title_fullStr	Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
title_full_unstemmed	Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life
title_sort	recombinant factor ix‐fc fusion protein in severe hemophilia b: patient‐reported outcomes and health‐related quality of life
publisher	Wiley
publishDate	2021
url	https://doaj.org/article/cce7db211eaf4a6f804488289b87edbe
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Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life

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