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The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

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‐...

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Autores principales: Sun, Haowei (Linda), Yang, Ming, Poon, Man‐Chiu, Lee, Adrienne, Robinson, K. Sue, Sholzberg, Michelle, Wu, John, Iorio, Alfonso, Blanchette, Victor, Carcao, Manuel, Klaassen, Robert J., Jackson, Shannon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8505988/
https://www.ncbi.nlm.nih.gov/pubmed/34667922
http://dx.doi.org/10.1002/rth2.12601
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author Sun, Haowei (Linda)
Yang, Ming
Poon, Man‐Chiu
Lee, Adrienne
Robinson, K. Sue
Sholzberg, Michelle
Wu, John
Iorio, Alfonso
Blanchette, Victor
Carcao, Manuel
Klaassen, Robert J.
Jackson, Shannon
author_facet Sun, Haowei (Linda)
Yang, Ming
Poon, Man‐Chiu
Lee, Adrienne
Robinson, K. Sue
Sholzberg, Michelle
Wu, John
Iorio, Alfonso
Blanchette, Victor
Carcao, Manuel
Klaassen, Robert J.
Jackson, Shannon
author_sort Sun, Haowei (Linda)
collection PubMed
description 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.
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spelling pubmed-85059882021-10-18 The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B Sun, Haowei (Linda) Yang, Ming Poon, Man‐Chiu Lee, Adrienne Robinson, K. Sue Sholzberg, Michelle Wu, John Iorio, Alfonso Blanchette, Victor Carcao, Manuel Klaassen, Robert J. Jackson, Shannon Res Pract Thromb Haemost Original Articles 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. John Wiley and Sons Inc. 2021-10-11 /pmc/articles/PMC8505988/ /pubmed/34667922 http://dx.doi.org/10.1002/rth2.12601 Text en © 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH). https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Articles
Sun, Haowei (Linda)
Yang, Ming
Poon, Man‐Chiu
Lee, Adrienne
Robinson, K. Sue
Sholzberg, Michelle
Wu, John
Iorio, Alfonso
Blanchette, Victor
Carcao, Manuel
Klaassen, Robert J.
Jackson, Shannon
The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title_full The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title_fullStr The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title_full_unstemmed The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title_short The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B
title_sort impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia a and hemophilia b
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8505988/
https://www.ncbi.nlm.nih.gov/pubmed/34667922
http://dx.doi.org/10.1002/rth2.12601
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