Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity …

VR Byams, JR Baker, C Bailey, NT Connell… - Expert review of …, 2023 - Taylor & Francis
ABSTRACT Background The National Hemophilia Foundation (NHF) conducted extensive
all-stakeholder inherited bleeding disorder (BD) community consultations to inform a …

[HTML][HTML] Hemophilia trials in the twenty‐first century: defining patient important outcomes

BA Konkle, M Skinner, A Iorio - Research and Practice in Thrombosis and …, 2019 - Elsevier
Abstract Treatment for hemophilia has advanced dramatically over the past 5 decades.
Success of prophylactic therapy in preventing bleeding and decreasing associated …

[HTML][HTML] Patient‐relevant health outcomes for hemophilia care: development of an international standard outcomes set

EC van Balen, B O'Mahony, MH Cnossen… - Research and practice …, 2021 - Elsevier
Background Patient‐relevant health outcomes for persons with hemophilia should be
identified and prioritized to optimize and individualize care for persons with hemophilia …

Outcome measures in hemophilia: current and future perspectives

S Benemei, L Boni, G Castaman - Expert Review of Hematology, 2024 - Taylor & Francis
Introduction Hemophilia can detrimentally affect patients' quality of life and likelihood of
survival. In the evolving landscape of therapies, the therapeutic gain of each treatment must …

Non‐severe haemophilia: is it benign?–insights from the PROBE study

C Chai‐Adisaksopha, D Noone, R Curtis, N Frick… - …, 2021 - Wiley Online Library
Introduction There are limited data on the impact of haemophilia on health status and health‐
related quality of life (HRQL) in people with non‐severe (mild and moderate) haemophilia …

Pain and functional disability amongst adults with moderate and severe haemophilia from the Irish personalised approach to the treatment of haemophilia (iPATH) …

M Kennedy, B O'Mahony, S Roche… - European Journal of …, 2022 - Wiley Online Library
Objectives To establish the prevalence of pain and functional disability in Irish adults with
moderate and severe haemophilia, and to examine demographic and lifestyle influences …

[HTML][HTML] User-centered development and testing of the online patient-reported outcomes, burdens, and experiences (PROBE) survey and the myPROBE app and …

F Germini, VB Debono, D Page, V Zuk… - JMIR Human …, 2022 - humanfactors.jmir.org
Background: The Patient-Reported Outcomes, Burdens, and Experiences (PROBE)
questionnaire is a tool for assessing the quality of life and disease burden in people living …

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

M O'Donovan, E Quinn, K Johnston, E Singleton… - Research and Practice …, 2021 - Elsevier
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 …

Health utilities in adults with hemophilia A: A retrospective cohort study

S Hirniak, AN Edginton, A Iorio, MW Alsabbagh… - …, 2024 - Wiley Online Library
Introduction Haemophilia A negatively affects a patient's quality of life. There is a limited
amount of health utility data (a measure of health‐related quality of life) available for patients …

Canadian clinical experience on switching from standard half‐life recombinant factor VIII (rFVIII), octocog alfa, to extended half‐life rFVIII, damoctocog alfa pegol, in …

D Matino, F Germini, AKC Chan, K Decker… - …, 2024 - Wiley Online Library
Introduction Damoctocog alfa pegol (BAY 94–9027, Jivi®) is an extended half‐life
recombinant factor (F) VIII replacement, indicated for the treatment of haemophilia A in …