CHANGES IN JOINT HEALTH WERE EVALUATED¹

Study Design¹

• This post hoc study assessed change in joint health among 47 adult and adolescent males (≥12 years of age) with severe hemophilia A who were treated with ELOCTATE prophylaxis
• Subjects completed the A-LONG pivotal trial, enrolled in the ASPIRE extension study (data cutoff of December 8, 2014), and had available mHJHS data
• Forty-seven subjects had mHJHS data at all 4 time points (A-LONG baseline, ASPIRE baseline, Year 1, and Year 2)
• Joint health assessments included the change in mHJHS from A-LONG baseline to follow-up visits and comparisons of individual mHJHS domains. In addition, factors such as target joints, weight-bearing and non–weight-bearing joints, and prestudy dosing regimen were assessed for impact on mHJHS. Negative values indicate improvement

Study Limitations¹

• Data are reported from a post hoc analysis that was not powered to show statistical significance. There was a small sample size, no control group, and potential for interobserver variability
• The mHJHS is not a validated tool
• Further studies are needed to confirm these findings as well as the effect of improvements in joint health on overall quality of life

^a Instability was scored the same as in the old version of the HJHS: 0=none and 1=significant pathologic joint laxity.^1
b Global gait was scored as follows: 0=no difficulty with walking or climbing up/down stairs; 1=no difficulty with walking, but difficulty with stairs; 2=difficulty with walking and with stairs.^1
c Joint scoring was performed separately for 6 joints (left and right ankle, left and right elbow, left and right knee) with a score range of 0 to 19 plus the gait score (0–2). Score of 0=normal; score of 116=most severe disease.¹

HJHS=Hemophilia Joint Health Score; mHJHS=modified Hemophilia Joint Health Score.