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Patterns of joint damage in severe haemophilia A treated with prophylaxis
Author(s) -
Goren Rachel,
Pullenayegum Eleanor,
Blanchette Victor S.,
Dover Saunya,
Carcao Manuel,
Israels Sara J.,
Chan Anthony,
Rivard Georges E.,
Steele MacGregor,
Cloutier Stéphanie,
Klaassen Robert J.,
Sinha Roona,
Price Victoria E.,
Laferriere Nicole,
Paradis Elizabeth,
Wu John K.,
Feldman Brian M.
Publication year - 2021
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.14345
Subject(s) - medicine , multinomial logistic regression , haemophilia , logistic regression , bleed , cohort , odds ratio , regimen , odds , demography , physical therapy , pediatrics , surgery , statistics , mathematics , sociology
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.