Persons with severe hemophilia A exhibit significant variations in clinical phenotype; approximately 10% are milder bleeders. This study explored factors that might contribute to this inter-patient heterogeneity. Using a multi-center prospective study following boys with severe hemophilia A on a tailored primary prophylaxis regimen, age at first joint bleed, body mass index, protocol adherence and family activity level were modeled on the age at escalation from once-weekly to twice-weekly and alternate day infusions. Escalation, based on bleeding frequency, served as a surrogate marker of bleeding severity. We also assessed the roles of clinical and hemostatic parameters in predicting age at first joint bleed using a cross-sectional study. Results suggest that a delayed first joint bleed indicates later escalation (milder bleeding pattern) and that a longer lag time is associated with an earlier onset of joint bleeding. Larger studies are needed to definitively identify predictors of clinical heterogeneity in these patients.
| Identifer | oai:union.ndltd.org:TORONTO/oai:tspace.library.utoronto.ca:1807/18325 |
| Date | 20 January 2010 |
| Creators | Hang, Marissa Xi |
| Contributors | Feldman, Brian M. |
| Source Sets | University of Toronto |
| Language | en_ca |
| Detected Language | English |
| Type | Thesis |
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