Résumé
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.
| Langue d'origine | English |
|---|---|
| Numéro d'article | e12601 |
| Journal | Research and Practice in Thrombosis and Haemostasis |
| Volume | 5 |
| Numéro de publication | 7 |
| DOI | |
| Statut de publication | Published - oct. 2021 |
Note bibliographique
Funding Information:This study is funded by an investigator‐initiated grant from Bioverativ.
Funding Information:
HS attended advisory boards for Bayer, Novo Nordisk, Octapharma, Pfizer, Sanofi, and Shire/Takeda; and received research support from Octapharma. MY has no conflicts of interest to declare. M‐CP has received grant funding from Bayer and CSL Behring; has been an ad hoc speaker for Bayer, Novo Nordisk, and Pfizer; and attended advisory board meetings of Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Pfizer, Roche, and Takeda. AL has received research grants from Bayer and Biovertiv/Sanofi; was a speaker/participant in advisory boards for Bayer, Novo Nordisk, Pfizer, and Shire/Takeda; was an ad hoc speaker for Bayer, Novo Nordisk, and Pfizer; attended advisory board meetings of Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Pfizer, Roche, and Takeda; and received grant funding from Bayer and CSL Behring. KSR has received research funding from Roche; and was a speaker/participant in advisory boards for Celgene and Roche. MS has received consultancy/advisory board fees from Octapharma, NovoNordisk, Bayer, and Takeda. JW has received research funding from Bayer and honoraria from Bayer, Bioverativ, CSL Behring Novo Nordisk, Octapharma, Pfizer, and Shire. AI’s institution has received project‐based funding via research or service agreements with Bayer, CSL, Grifols, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sobi, and Takeda. VB reports that he is Chair of the International Prophylaxis Study Group, a cooperative study group that is funded by education grants from Bayer Healthcare, Bioverativ/Sanofi, Novo Nordisk, Pfizer, Shire/Takeda, and Spark Therapeutics to the Hospital for Sick Children Foundation. He has received fees for participation in Advisory Boards/Education events supported by Amgen, Bayer, Novo. Nordisk, Pfizer, Roche and Shire/Takeda and for participation in Data Safety Monitoring Boards for Octapharma and Shire/Takeda. He has received investigator‐initiated, industry‐supported research grants from Novo Nordisk, Bioverativ/Sanofi and Shire/Takeda. In addition, he has a patent on the CHO‐KLAT with royalties paid to the Hospital for Sick Children, Laurentian University, University of Manitoba, and Dr Victoria Price. MC has received research support from Bayer, Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Octapharma, Pfizer, and Shire/Takeda; and honoraria for speaking/participating in advisory boards from Bayer, Biotest, Bioverativ/Sanofi, CSL Behring, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, and Shire/Takeda. RJK has received speaker and/or consultant fees from Agios Pharmaceuticals Inc., Amgen, Hoffmann La Roche LTD, Shire Pharma Canada ULC, Novo Nordisk Canada Inc, Octapharma AG, Takeda, and Sanofi‐Genzyme. SJ has received research grants from Sanofi, Canadian Hemophilia Society; and honoraria from Pfizer/BMS, Takeda, Octapharma, Bayer, Roche; and consulting fees from Hemalytic.
Publisher Copyright:
© 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).
ASJC Scopus Subject Areas
- Hematology
PubMed: MeSH publication types
- Journal Article
ODD de l’ONU
Cette action contribue à la réalisation des objectifs de développement durable suivants
