Patterns of joint damage in severe haemophilia A treated with prophylaxis

Rachel Goren, Eleanor Pullenayegum, Victor S. Blanchette, Saunya Dover, Manuel Carcao, Sara J. Israels, Anthony Chan, Georges E. Rivard, MacGregor Steele, Stéphanie Cloutier, Robert J. Klaassen, Roona Sinha, Victoria E. Price, Nicole Laferriere, Elizabeth Paradis, John K. Wu, Brian M. Feldman

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Resumen

Objective: The primary objective of this study was to assess whether there are different patterns (classes) of joint health in young boys with severe haemophilia A (SHA) prescribed primary tailored prophylaxis. We also assessed whether age at first index joint bleed, blood group, FVIII gene abnormality variant, factor VIII trough level, first-year bleeding rate and adherence to the prescribed prophylaxis regimen significantly predicted joint damage trajectory, and thus class membership. Methods: Using data collected prospectively as part of the Canadian Hemophilia Primary Prophylaxis Study (CHPS), we implemented a latent class growth mixture model technique to determine how many joint damage classes existed within the cohort. We used a multinomial logistic regression to predict the odds of class membership based on the above predictors. We fitted a survival model to assess whether there were differences in the rate of dose escalation across the groups. Results: We identified three distinct classes of trajectory: persistently low, moderately increasing and rapidly increasing joint scores. By multinomial regression, we found that only age at first index joint bleed predicted rapidly increasing joint scores. The rapidly increasing joint score class group moved through dose escalation significantly faster than the other two groups. Conclusions: Using tailored prophylaxis, boys with SHA follow one of three joint health trajectories. By using knowledge of disease trajectories, clinicians may be able to adjust treatment according to a subject's predicted long-term joint health and institute cost-effective programmes of prophylaxis targeted at the individual subject level.

Idioma originalEnglish
Páginas (desde-hasta)666-673
Número de páginas8
PublicaciónHaemophilia
Volumen27
N.º4
DOI
EstadoPublished - jul. 2021
Publicado de forma externa

Nota bibliográfica

Funding Information:
The Canadian Hemophilia Primary Prophylaxis Study was initially funded by grants from the Medical Research Council of Canada/Pharmaceutical Manufacturers Association of Canada Partnership Fund and the Bayer/Canadian Blood Services/Hema‐Quebec Partnership Fund. Subsequent renewals were funded by Bayer Canada Inc. Dr. Blanchette reports having received fees for participation in Advisory Boards for Amgen, Bayer, Novo Nordisk, Pfizer, Roche and Shire and Data Monitoring Boards for Octapharma and Takeda He holds grants from Sanofi and Takeda and is the non‐remunerated Chair of the International Prophylaxis Study Group, outside the submitted work. Dr. Carcao reports having received research support from Bayer, Bioverativ/Sonafi, CSL Behring, Novo Nordisk, Octapharma, Pfizer and Shire/Takeda. He has also received honoraria for speaking/participating in advisory boards from Bayer, Bioverativ/Sonofi, Biotest, CSL Behring, Grifols, LFB, Novo Nordisk, Octapharma, Pfizer, Roche and Shire/Takeda. Dr. Klaassen reports personal fees from Agios Pharmaceuticals Inc, Amgen Inc, Hoffman‐LaRoche LTD, Shire Pharma Canada ULC, Novo Nordisk Canada Inc, Octapharma AG, Takeda, and Sanofi outside the submitted work. Dr. Feldman holds a patent (2009) for the Hemophilia Joint Health Score (HJHS) 2.1 with royalties paid to The Hospital for Sick Children, Centre Hospitalier Universitaire Sainte Justine, the Regents of the University of Colorado, Karolinska Hospital and the University Medical Center Utrecht, 2009. The HJHS is available under licence by The Hospital for Sick Children. ©

Funding Information:
This study was a sub-study of the Canadian Hemophilia Primary Prophylaxis Study, a cooperative initiative undertaken by the Association of Hemophilia Clinic Directors of Canada (AHCDC).

Publisher Copyright:
© 2021 John Wiley & Sons Ltd

ASJC Scopus Subject Areas

  • Hematology
  • Genetics(clinical)

PubMed: MeSH publication types

  • Journal Article

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