ADVATE prophylaxis: post hoc analysis of joint bleeding episodes. (21st February 2014)

• Record Type: Journal Article
• Authors: Valentino, L. A.; Reyes, C. M.; Ewenstein, B.; Guzmán‐Becerra, N.; Wong, W. Y.; Schoenig‐Diesing, C.; Romanov, V.
• Is Part Of: Haemophilia. Volume 20:Number 5(2014:Sep.)
• Page Start: 630
• Page End: 638
• Publication Date: 2014-02-21
• Subjects: Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.12382
• Languages: English
• ISSNs: 1351-8216

Abstract

<abstract abstract-type="main" id="hae12382-abs-0001"> <title>Summary</title> <sec id="hae12382-sec-0001" sec-type="section"> <title>Introduction</title> <p>The ADVATE (rAHF‐PFM) Prophylaxis Study compared the efficacy of (i) standard factor (F) VIIII prophylaxis (SP) (20–40 IU kg<sup>−1</sup> every other day) vs. pharmacokinetic‐tailored prophylaxis (PKP) (20–80 IU kg<sup>−1</sup> every third day) and (ii) both prophylactic regimens with on‐demand therapy (OD) in 66 previously on‐demand‐treated patients (median age: 26 years; range: 7–59) with FVIII ≤2% and ≥8 joint haemorrhages in the year before enrolment.</p> </sec> <sec id="hae12382-sec-0002" sec-type="section"> <title>Aim</title> <p>The aim of this study was to evaluate joint bleeding episodes during the on‐demand and prophylactic study periods.</p> </sec> <sec id="hae12382-sec-0003" sec-type="section"> <title>Methods</title> <p>A <italic>post hoc</italic> analysis of joint bleeding episodes in the per protocol analysis set (<italic>n</italic> = 53) was conducted.</p> </sec> <sec id="hae12382-sec-0004" sec-type="section"> <title>Results</title> <p>The annualized joint bleeding rate (AJBR) was significantly lower for subjects treated with 12 months of SP (<italic>n</italic> = 30) or PKP (<italic>n</italic> = 23) as compared with 6 months of OD (<italic>n</italic> = 53): 55 {median AJBR 0.48 [interquartile range (IQR) 1.96]}, 72 [1.00 (4.07)] and 1164 [38.65 (24.81)] respectively (<italic>P</italic> < 0.0001). Median AJBR was comparable during both prophylaxis arms (0.5 and 1.0 respectively). In contrast, median AJBR during on‐demand therapy was 38.7 (<italic>P</italic> < 0.0001).</p> </sec> <sec id="hae12382-sec-0005" sec-type="section"> <title>Conclusion</title> <p>Both SP and PKP significantly increased the median number of days between joint bleeding episodes compared with OD: 268.9, 182.9 and 7.4 respectively (<italic>P</italic> < 0.0001). Study subjects experienced these joint‐protective benefits of prophylaxis regardless of the number of baseline target joints.</p> </sec> </abstract>