Changes in health‐related quality of life with treatment of longer‐acting clotting factors: results in the A‐LONG and B‐LONG clinical studies. (7th July 2016) - Wyrwich, K. W.
Abstract : Introduction: In haemophilia, prophylactic infusion of replacement factor can result in improvements in health‐related quality of life (HRQoL) when compared with episodic treatment. The Haemophilia‐specific Quality of Life (Haem‐A‐QoL) questionnaire assessed HRQoL in adults with severe haemophilia A or B who received prophylactic or episodic treatment with recombinant factor VIII or IX Fc fusion protein (rFVIIIFc or rFIXFc) in the A‐LONG or B‐LONG clinical studies. Aims: Understand changes in HRQoL during the A‐LONG and B‐LONG trials. Methods: Group‐level and individual‐level changes over time for the Haem‐A‐QoL key domains of 'Physical Health' and 'Sports & Leisure, ' and 'Total Score' were evaluated in adults through baseline and 6‐month HRQoL assessments. Previously determined responder definitions (RDs) were used for evaluating meaningful subject‐level HRQoL improvements. Results: The analysis included 67 A‐LONG and 51 B‐LONG subjects who completed the Haem‐A‐QoL (baseline and 6 months). While HRQoL improvements were observed among all treatment groups, greater improvements in HRQoL were observed among subjects who received episodic treatment pre‐study (and prophylaxis on‐study) compared to those who received hyphenate prophylaxis. Applying the RDs for interpreting 6‐month changes, 47.4%/33.3% ('Physical Health'), 35.9%/50.0% ('Sports & Leisure') and 23.9%/33.3% ('Total Score') of A‐LONG subjects who received individualized or weekly prophylaxis were classified as HRQoL responders. In B‐LONG, 69.2%/57.9% ('Physical Health'), 44.4%/56.7% ('Sports & Leisure') and 41.7%/44.1% ('Total Score') of subjects who received individualized or weekly prophylaxis were classified as HRQoL responders. Conclusion: Changes in Haem‐A‐QoL key domains and 'Total Score' suggest that prophylaxis with long‐acting rFVIIIFc or rFIXFc resulted in meaningful HRQoL improvements.