Open AccessThe impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
Author(s) -
Sun Haowei Linda,
Yang Ming,
Poon ManChiu,
Lee Adrienne,
Robinson K. Sue,
Sholzberg Michelle,
Wu John,
Iorio Alfonso,
Blanchette Victor,
Carcao Manuel,
Klaassen Robert J.,
Jackson Shan
Publication year - 2021
Publication title -
research and practice in thrombosis and haemostasis
Language(s) - English
Resource type - Journals
ISSN - 2475-0379
DOI - 10.1002/rth2.12601
Subject(s) - quality of life (healthcare) , psychology , mood , haemophilia , eq 5d , mental health , medicine , physical therapy , family medicine , clinical psychology , psychiatry , health related quality of life , pediatrics , psychotherapist , disease
Background Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) were the only available extended half‐life (EHL) products in Canada during 2016 to 2018. Objectives To evaluate if patient‐reported outcome measures (PROMs) improved in Canadian persons with hemophilia who switched from standard half‐life (SHL) to EHL products (rFVIIIFc/rFIXFc). Patients/Methods This prospective cohort study enrolled persons with moderate or severe hemophilia aged ≥6 years who switched to rFVIIIFc/rFIXFc (2016‐2018) and those who remained on SHL. Health‐related quality of life (HRQoL) was assessed using the Haemophilia‐specific Quality of Life (Haem‐A‐QoL) and 36‐item Short‐Form Survey (SF‐36) at baseline, 3‐months, 12 months, and 24 months. Other PROMs included the Work Productivity and Impairment Questionnaire, chronic pain scale, partner/parent ratings of mood, International Physical Activity Questionnaire, and Treatment Satisfaction Questionnaire for Medication. We identified meaningful changes using minimally important difference for SF‐36 and responder definition for Haem‐A‐QoL. Results We enrolled 25 switchers (16 rFVIIIFc, 9 rFIXFc) and 33 nonswitchers. Those switched to rFVIIIFc/rFIXFc had improved overall HRQoL, and improved subscale physical activity, mental health, and social functioning at 3 months. The rFIXFc switchers had improved chronic pain and ability to engage in normal activities while the rFVIIIFc switchers had improved treatment satisfaction. There was no change in work impairment after the switch. Observed improvement disappeared by 24 months in most domains. Conclusion Switching from SHL to rFVIIIFc/rFIXFc resulted in short‐term meaningful improvement in overall HRQoL and other PROMs in a small proportion. Longitudinal changes on PROMs are affected by ceiling effects and response shift, warranting further studies in instrument optimization in the era of EHL and nonfactor products.