HHE Haematology supplement 2018 - Page 16

residual or subclinical haemarthroses, and hence at risk of subsequent arthropathy, due to insufficient trough FVIII levels, and those who request it after having learned about the benefits of these new products. A monocentre case study in France showed that, out of 48 patients who switched to rFVIIIFc, 32 changed their dosing schedules, reducing the injection frequency or introducing prophylaxis. Of those 48 patients, 40 received long-term prophylaxis, without any obvious preference for a particular treatment pre-switch, and 8 were treated on demand. After switching to rFVFc, prophylaxis was continued or initiated in 47 patients, with a good/excellent qualitative assessment of efficacy in 44 out of 45 patients evaluated. As of now, FVIII trough levels (1–4%) were shown to be consistent with the clinical outcomes, and no inhibitor development was observed. Although larger and longer surveys need to be conducted to determine QoL and health economic outcomes, rFVIIIFc may optimise outcomes in some patients by increasing the level of protection in patients at risk of residual bleedings, and by making prophylaxis acceptable and feasible among patients reluctant to changes in their treatment plans. Reconsider treatment goals in haemophilia B: Clinical experience with rFIXFc One of the major achievements of prophylaxis regimens for haemophilia patients is sustained protection from bleeds; however, among the treatment goals reported by patients to be important to them is decreased injection frequency to reduce the treatment burden and improve QoL. In addition, having weekly injections allows patients and/or their carers to remember to take their medication, which contributes to improved adherence. However, these patient-related benefits are related to clinical outcomes. The anticipated desired outcomes with rFIXFc are to achieve and maintain low ABRs, as well as attain adequate FIX levels (individualised for the patient’s bleeding phenotype, joint status, and activity levels), prevent bleeding when undergoing surgical or invasive medical procedures, and enhance patient experience. 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