Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies
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Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies. / Wyrwich, K W; Krishnan, S; Auguste, P; Poon, J-L; von Maltzahn, R; Yu, R; Pierce, G F; Mei, B; Mahlangu, J; von Mackensen, S.
In: HAEMOPHILIA, Vol. 22, No. 6, 11.2016, p. 866-872.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies
AU - Wyrwich, K W
AU - Krishnan, S
AU - Auguste, P
AU - Poon, J-L
AU - von Maltzahn, R
AU - Yu, R
AU - Pierce, G F
AU - Mei, B
AU - Mahlangu, J
AU - von Mackensen, S
N1 - © 2016 John Wiley & Sons Ltd.
PY - 2016/11
Y1 - 2016/11
N2 - 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.
AB - 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.
KW - Journal Article
U2 - 10.1111/hae.12987
DO - 10.1111/hae.12987
M3 - SCORING: Journal article
C2 - 27385432
VL - 22
SP - 866
EP - 872
JO - HAEMOPHILIA
JF - HAEMOPHILIA
SN - 1351-8216
IS - 6
ER -
