Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

• Published in: Research and practice in thrombosis and haemostasis 2020-02, Vol.4 (2), p.318-325
• Main Authors: Dover, Saunya, Blanchette, Victor S., Wrathall, Darius, Pullenayegum, Eleanor, Kazandjian, Daniel, Song, Byron, Hawes, Sue Ann, Cloutier, Stéphanie, Rivard, Geroges E., Klaassen, Robert J., Paradis, Elizabeth, Laferriere, Nicole, Stain, Ann Marie, Chan, Anthony K., Israels, Sara J., Sinha, Roona, Steele, MacGregor, Wu, John K.M., Feldman, Brian M.
• Format: Article
• Language: English
• Subjects: bleeding; factor VIII; Hemophilia; hemophilia A; Literature reviews; Original; Original : Hemostasis; Patient compliance; Pediatrics; prophylaxis; Sensitivity analysis; treatment adherence and compliance
• ISSN: 2475-0379; 2475-0379
• DOI: 10.1002/rth2.12301

Standard of care for persons with severe hemophilia A includes regular replacement of factor VIII (FVIII). Prophylaxis regimens using standard half‐life (SHL) FVIII concentrates, while effective, are costly and require frequent intravenous infusions. This study evaluated the adherence of 56 boys with severe hemophilia A to tailored, frequency‐escalated prophylaxis with an SHL recombinant FVIII concentrate. We reviewed the factor infusion and bleeding logs of study subjects. Adherence to the prescribed regimen was calculated on a weekly basis, and bleeding rates were determined from self/proxy‐reported bleeding logs. The primary outcome was adherence to the prescribed prophylaxis regimen. The median (range of values [ROV]) weekly adherence to prophylaxis was 85.7% (37.4%‐99.8%). The median (ROV) adherent weeks on steps 1 (weekly), 2 (twice weekly), and 3 (alternate‐day) were 92.9% (50%‐100%), 80.3 (32%‐96%), and 72.6% (14%‐98%); relative to step 1, subjects were less likely to be adherent on steps 2 and 3 (P