Measuring the impact of changing from standard half-life (SHL) to extended half-life (EHL) FVIII prophylaxis on health-related quality of life (HRQoL) in boys with moderate/severe haemophilia A: Lessons learned with the CHO-KLAT tool

Manuel Carcao, Laura Zunino, Nancy L. Young, Saunya Dover, Vanessa Bouskill, Pamela Hilliard, Victoria E. Price, Victor S. Blanchette

Résultat de recherche: Articleexamen par les pairs

15 Citations (Scopus)

Résumé

Introduction: In many countries, there is a shift from standard half-life (SHL) to extended half-life (EHL) clotting factor concentrates (CFCs). Aim: To describe the experience of switching from SHL to an EHL FVIII CFC and the impact of this on frequency of infusions, factor consumption, bleeding rates and HRQoL using the Canadian Hemophilia Kids’ Life Assessment Tool (CHO-KLAT). Methods: A retrospective chart review was conducted at a single haemophilia treatment centre in 2018 that included boys (ages: 4-18 years) with moderate/severe haemophilia A, without inhibitors, who switched from a SHL to an EHL FVIII CFC in the previous 2 years and for whom HRQoL data were available. Results: The study cohort comprised 38 boys [mean (SD) age: 11.0 (3.4) years] with moderate (n = 5)/severe (n = 33) haemophilia A. The switch was associated with a 33% reduction in the number of weekly infusions from a median of 3.5 to 2.3 (P <.0001) and a 17% reduction in median FVIII consumption from 103 IU/kg/wk to 85.5 IU/kg/wk (P =.004). There was no significant change in annualized joint bleed rates or in CHO-KLAT scores. Conclusions: Despite documenting several benefits of switching to EHL FVIII (less infusions, lower factor consumption with no increase in bleeding), our study did not demonstrate any improvement in HRQoL. We conclude that either the current CHO-KLAT tool is not optimized to measure burden of treatment administration in boys with low bleed rates switching from SHL to EHL FVIII CFCs or that a reduction of 1.2 infusions/week does not result in a meaningful change in HRQoL.

Langue d'origineEnglish
Pages (de-à)73-78
Nombre de pages6
JournalHaemophilia
Volume26
Numéro de publication1
DOI
Statut de publicationPublished - janv. 1 2020

Note bibliographique

Funding Information:
M. Carcao: Reports having received research support from Bayer, Bioverativ/Sanofi, CSL Behring, Novo Nordisk, Octapharma, Pfizer and Shire. He has also received honoraria for speaking/participating in advisory boards from Bayer, Bioverativ/Sanofi, Biotest, CSL Behring, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche and Shire. L. Zunino reports no conflicts. N. Young reports no conflicts. S. Dover reports no conflicts. V. Bouskill reports no conflicts. P. Hilliard: Reports acting as a paid consultant for Alnylam and Pfizer. V. Price reports no conflicts. VS Blanchette: Reports receiving speakers fees/honoraria for participation in Educational events/Advisory Boards from Bayer Health Care, Bioverativ/Sonofi, Novo Nordisk, Pfizer, Roche, Shire and Spark Therapeutics. Member of Data Safety Monitoring Boards for Octapharma and Shire. Research Grants from Bayer Health Care, Bioverativ and Shire. Chairman of the International Prophylaxis Study Group (IPSG), a non‐for‐profit collaborative study group supported by grants from Bayer Health Care, Bioverativ, Novo Nordisk, Pfizer, Shire and Spark Therapeutics to the Hospital for Sick Children Foundation, Toronto, Canada.

Publisher Copyright:
© 2019 John Wiley & Sons Ltd

ASJC Scopus Subject Areas

  • Hematology
  • Genetics(clinical)

PubMed: MeSH publication types

  • Journal Article

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