Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes

Haowei Sun; Ming Yang; Man Chiu Poon; Adrienne Lee; K. Sue Robinson; Michelle Sholzberg; John Wu; Alfonso Iorio; Victor Blanchette; Manuel Carcao; Robert J. Klaassen; Shannon Jackson

doi:10.1111/hae.14369

Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes

Haowei Sun, Ming Yang, Man Chiu Poon, Adrienne Lee, K. Sue Robinson, Michelle Sholzberg, John Wu, Alfonso Iorio, Victor Blanchette, Manuel Carcao, Robert J. Klaassen, Shannon Jackson

Medicine

Résultat de recherche: Article › examen par les pairs

12 Citations (Scopus)

Résumé

Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018. Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc. Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018. Factor utilization and annualized bleeding rates (ABR) were collected at baseline, 1-year and 2-years. Due to low prospective enrolment (n = 25 switchers), prospective and retrospective data were pooled. Results: 125 switchers (93 rFVIIIFc, 32 rFIXFc) and 33 non-switchers were included. The median age was 17 (rFVIIIFc) and 38 years (rFIXFc). Prior to switch, over 80% were on prophylaxis. There was a statistically significant reduction in the prescribed weekly prophylactic dose after the switch to rFVIIIFc/rFIXFc for all age groups, with a corresponding reduction (15-16%) in actual annualized FIX utilization in switchers (combined adults and children) to rFIXFc, and a smaller non-significant reduction in actual annualized FVIIII utilization (7%) in children who switched to rFVIIIFc. A significant reduction in the median ABR was only observed in children who switched to rFVIIIFc, but not in adults who switched to rFVIIIFc or rFIXFc. Conclusion: Switching from SHL to EHL products led to a small reduction in factor utilization, while preserving a low ABR in children and adults with haemophilia. Further patient-reported outcomes data will further elucidate the role of EHL in the haemophilia landscape.

Langue d'origine	English
Pages (de-à)	751-759
Nombre de pages	9
Journal	Haemophilia
Volume	27
Numéro de publication	5
DOI	https://doi.org/10.1111/hae.14369
Statut de publication	Published - sept. 2021

Note bibliographique

Funding Information:
Haowei Sun attended advisory boards for Bayer, Novo Nordisk, Octapharma, Pfizer, Sanofi, Shire/Takeda; and research support from Octapharma. Ming Yang has no conflicts of interest to declare. Man‐Chiu Poon has received grant funding from Bayer and CSL Behring; ad hoc speaker for Bayer, Novo Nordisk, and Pfizer; attended advisory board meetings of Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Pfizer, Roche, and Takeda. Adrienne Lee has received research grants from Bayer and Biovertiv/Sanofi; and was a speaker/participant in advisory boards for Bayer, Novo Nordisk, Pfizer, and Shire/Takeda; 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. Katherine Sue Robinson has received research funding from Roche; was a speaker/ participant in advisory boards for Celgene and Roche. Michelle Sholzberg has received consultancy/advisory board fees from Octapharma, NovoNordisk, Bayer and Takeda. John Wu has received research funding from Bayer; and honoraria from Bayer, Bioverativ, CSL Behring Novo Nordisk, Octapharma, Pfizer and Shire. Alfonso Iorio'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. Victor Blanchette reports that he is Chair of the International Prophylaxis Study Group (IPSG), a co‐operative 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 (“SickKids”) 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 (DSMB) for Octapharma and Shire/Takeda. He has received Investigator initiated, Industry supported research grants from Novo Nordisk, Bioverativ/Sanofi and Shire/Takeda. In addition, Dr. Blanchette has a patent on the CHO‐KLAT Health‐Related Quality of Life Tool with royalties paid to the Hospital for Sick Children, Laurentian University, University of Manitoba, and Dr. Victoria Price. Manuel Carcao 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. Robert J. Klaassen 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. Shannon Jackson has received research grants from Sanofi, Canadian Haemophilia Society; and honoraria from Pfizer/BMS, Takeda, Octapharma, Bayer, Roche; and consulting fees from Haemolytic. This study is funded by an investigator‐initiated grant from Bioverativ.

Publisher Copyright:
© 2021 John Wiley & Sons Ltd.

ASJC Scopus Subject Areas

Hematology
Genetics(clinical)

PubMed: MeSH publication types

Journal Article
Observational Study

Accès au document

10.1111/hae.14369

Autres fichiers et liens

Citer

Sun, H., Yang, M., Poon, M. C., Lee, A., Robinson, K. S., Sholzberg, M., Wu, J., Iorio, A., Blanchette, V., Carcao, M., Klaassen, R. J., & Jackson, S. (2021). Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes. Haemophilia, 27(5), 751-759. https://doi.org/10.1111/hae.14369

Sun, H, Yang, M, Poon, MC, Lee, A, Robinson, KS, Sholzberg, M, Wu, J, Iorio, A, Blanchette, V, Carcao, M, Klaassen, RJ & Jackson, S 2021, 'Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes', Haemophilia, vol. 27, n° 5, pp. 751-759. https://doi.org/10.1111/hae.14369

@article{56565ae884d64b95b0564da8bef7fced,

title = "Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes",

abstract = "Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018. Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc. Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018. Factor utilization and annualized bleeding rates (ABR) were collected at baseline, 1-year and 2-years. Due to low prospective enrolment (n = 25 switchers), prospective and retrospective data were pooled. Results: 125 switchers (93 rFVIIIFc, 32 rFIXFc) and 33 non-switchers were included. The median age was 17 (rFVIIIFc) and 38 years (rFIXFc). Prior to switch, over 80% were on prophylaxis. There was a statistically significant reduction in the prescribed weekly prophylactic dose after the switch to rFVIIIFc/rFIXFc for all age groups, with a corresponding reduction (15-16%) in actual annualized FIX utilization in switchers (combined adults and children) to rFIXFc, and a smaller non-significant reduction in actual annualized FVIIII utilization (7%) in children who switched to rFVIIIFc. A significant reduction in the median ABR was only observed in children who switched to rFVIIIFc, but not in adults who switched to rFVIIIFc or rFIXFc. Conclusion: Switching from SHL to EHL products led to a small reduction in factor utilization, while preserving a low ABR in children and adults with haemophilia. Further patient-reported outcomes data will further elucidate the role of EHL in the haemophilia landscape.",

author = "Haowei Sun and Ming Yang and Poon, {Man Chiu} and Adrienne Lee and Robinson, {K. Sue} and Michelle Sholzberg and John Wu and Alfonso Iorio and Victor Blanchette and Manuel Carcao and Klaassen, {Robert J.} and Shannon Jackson",

note = "Funding Information: Haowei Sun attended advisory boards for Bayer, Novo Nordisk, Octapharma, Pfizer, Sanofi, Shire/Takeda; and research support from Octapharma. Ming Yang has no conflicts of interest to declare. Man‐Chiu Poon has received grant funding from Bayer and CSL Behring; ad hoc speaker for Bayer, Novo Nordisk, and Pfizer; attended advisory board meetings of Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Pfizer, Roche, and Takeda. Adrienne Lee has received research grants from Bayer and Biovertiv/Sanofi; and was a speaker/participant in advisory boards for Bayer, Novo Nordisk, Pfizer, and Shire/Takeda; 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. Katherine Sue Robinson has received research funding from Roche; was a speaker/ participant in advisory boards for Celgene and Roche. Michelle Sholzberg has received consultancy/advisory board fees from Octapharma, NovoNordisk, Bayer and Takeda. John Wu has received research funding from Bayer; and honoraria from Bayer, Bioverativ, CSL Behring Novo Nordisk, Octapharma, Pfizer and Shire. Alfonso Iorio'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. Victor Blanchette reports that he is Chair of the International Prophylaxis Study Group (IPSG), a co‐operative 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 (“SickKids”) 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 (DSMB) for Octapharma and Shire/Takeda. He has received Investigator initiated, Industry supported research grants from Novo Nordisk, Bioverativ/Sanofi and Shire/Takeda. In addition, Dr. Blanchette has a patent on the CHO‐KLAT Health‐Related Quality of Life Tool with royalties paid to the Hospital for Sick Children, Laurentian University, University of Manitoba, and Dr. Victoria Price. Manuel Carcao 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. Robert J. Klaassen 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. Shannon Jackson has received research grants from Sanofi, Canadian Haemophilia Society; and honoraria from Pfizer/BMS, Takeda, Octapharma, Bayer, Roche; and consulting fees from Haemolytic. This study is funded by an investigator‐initiated grant from Bioverativ. Publisher Copyright: {\textcopyright} 2021 John Wiley & Sons Ltd.",

year = "2021",

month = sep,

doi = "10.1111/hae.14369",

language = "English",

volume = "27",

pages = "751--759",

journal = "Haemophilia",

issn = "1351-8216",

publisher = "Wiley-Blackwell",

number = "5",

}

TY - JOUR

T1 - Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates

T2 - A Canadian observational study of real-world outcomes

AU - Sun, Haowei

AU - Yang, Ming

AU - Poon, Man Chiu

AU - Lee, Adrienne

AU - Robinson, K. Sue

AU - Sholzberg, Michelle

AU - Wu, John

AU - Iorio, Alfonso

AU - Blanchette, Victor

AU - Carcao, Manuel

AU - Klaassen, Robert J.

AU - Jackson, Shannon

N1 - Funding Information: Haowei Sun attended advisory boards for Bayer, Novo Nordisk, Octapharma, Pfizer, Sanofi, Shire/Takeda; and research support from Octapharma. Ming Yang has no conflicts of interest to declare. Man‐Chiu Poon has received grant funding from Bayer and CSL Behring; ad hoc speaker for Bayer, Novo Nordisk, and Pfizer; attended advisory board meetings of Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Pfizer, Roche, and Takeda. Adrienne Lee has received research grants from Bayer and Biovertiv/Sanofi; and was a speaker/participant in advisory boards for Bayer, Novo Nordisk, Pfizer, and Shire/Takeda; 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. Katherine Sue Robinson has received research funding from Roche; was a speaker/ participant in advisory boards for Celgene and Roche. Michelle Sholzberg has received consultancy/advisory board fees from Octapharma, NovoNordisk, Bayer and Takeda. John Wu has received research funding from Bayer; and honoraria from Bayer, Bioverativ, CSL Behring Novo Nordisk, Octapharma, Pfizer and Shire. Alfonso Iorio'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. Victor Blanchette reports that he is Chair of the International Prophylaxis Study Group (IPSG), a co‐operative 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 (“SickKids”) 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 (DSMB) for Octapharma and Shire/Takeda. He has received Investigator initiated, Industry supported research grants from Novo Nordisk, Bioverativ/Sanofi and Shire/Takeda. In addition, Dr. Blanchette has a patent on the CHO‐KLAT Health‐Related Quality of Life Tool with royalties paid to the Hospital for Sick Children, Laurentian University, University of Manitoba, and Dr. Victoria Price. Manuel Carcao 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. Robert J. Klaassen 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. Shannon Jackson has received research grants from Sanofi, Canadian Haemophilia Society; and honoraria from Pfizer/BMS, Takeda, Octapharma, Bayer, Roche; and consulting fees from Haemolytic. This study is funded by an investigator‐initiated grant from Bioverativ. Publisher Copyright: © 2021 John Wiley & Sons Ltd.

PY - 2021/9

Y1 - 2021/9

N2 - Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018. Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc. Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018. Factor utilization and annualized bleeding rates (ABR) were collected at baseline, 1-year and 2-years. Due to low prospective enrolment (n = 25 switchers), prospective and retrospective data were pooled. Results: 125 switchers (93 rFVIIIFc, 32 rFIXFc) and 33 non-switchers were included. The median age was 17 (rFVIIIFc) and 38 years (rFIXFc). Prior to switch, over 80% were on prophylaxis. There was a statistically significant reduction in the prescribed weekly prophylactic dose after the switch to rFVIIIFc/rFIXFc for all age groups, with a corresponding reduction (15-16%) in actual annualized FIX utilization in switchers (combined adults and children) to rFIXFc, and a smaller non-significant reduction in actual annualized FVIIII utilization (7%) in children who switched to rFVIIIFc. A significant reduction in the median ABR was only observed in children who switched to rFVIIIFc, but not in adults who switched to rFVIIIFc or rFIXFc. Conclusion: Switching from SHL to EHL products led to a small reduction in factor utilization, while preserving a low ABR in children and adults with haemophilia. Further patient-reported outcomes data will further elucidate the role of EHL in the haemophilia landscape.

AB - Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018. Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc. Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018. Factor utilization and annualized bleeding rates (ABR) were collected at baseline, 1-year and 2-years. Due to low prospective enrolment (n = 25 switchers), prospective and retrospective data were pooled. Results: 125 switchers (93 rFVIIIFc, 32 rFIXFc) and 33 non-switchers were included. The median age was 17 (rFVIIIFc) and 38 years (rFIXFc). Prior to switch, over 80% were on prophylaxis. There was a statistically significant reduction in the prescribed weekly prophylactic dose after the switch to rFVIIIFc/rFIXFc for all age groups, with a corresponding reduction (15-16%) in actual annualized FIX utilization in switchers (combined adults and children) to rFIXFc, and a smaller non-significant reduction in actual annualized FVIIII utilization (7%) in children who switched to rFVIIIFc. A significant reduction in the median ABR was only observed in children who switched to rFVIIIFc, but not in adults who switched to rFVIIIFc or rFIXFc. Conclusion: Switching from SHL to EHL products led to a small reduction in factor utilization, while preserving a low ABR in children and adults with haemophilia. Further patient-reported outcomes data will further elucidate the role of EHL in the haemophilia landscape.

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Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes

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PubMed: MeSH publication types

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