Premium
Changes in health‐related quality of life with treatment of longer‐acting clotting factors: results in the A‐ LONG and B‐ LONG clinical studies
Author(s) -
Wyrwich K. W.,
Krishnan S.,
Auguste P.,
Poon J.L.,
Maltzahn R.,
Yu R.,
Pierce G. F.,
Mei B.,
Mahlangu J.,
Mackensen S.
Publication year - 2016
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.12987
Subject(s) - medicine , haemophilia , quality of life (healthcare) , clotting factor , clinical trial , haemophilia a , haemophilia b , health related quality of life , pediatrics , physical therapy , disease , nursing
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 ( rFVIIIF c or rFIXF c) in the A‐ LONG or B‐ LONG clinical studies. Aims Understand changes in HRQ oL 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 HRQ oL assessments. Previously determined responder definitions ( RD s) were used for evaluating meaningful subject‐level HRQ oL improvements. Results The analysis included 67 A‐ LONG and 51 B‐ LONG subjects who completed the Haem‐A‐QoL (baseline and 6 months). While HRQ oL improvements were observed among all treatment groups, greater improvements in HRQ oL were observed among subjects who received episodic treatment pre‐study (and prophylaxis on‐study) compared to those who received hyphenate prophylaxis. Applying the RD s 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 HRQ oL 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 HRQ oL responders. Conclusion Changes in Haem‐A‐QoL key domains and ‘Total Score’ suggest that prophylaxis with long‐acting rFVIIIF c or rFIXF c resulted in meaningful HRQ oL improvements.