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Kraemmer, D; Königsbrügge, O; Moik, F; Wildner, B; Ay, C; Pabinger, I.
Pharmacokinetic-guided versus standard prophylaxis in hemophilia: a systematic review and meta-analysis.
J Thromb Haemost. 2023; 21(12): 3432-3449. Doi: 10.1016/j.jtha.2023.08.031
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Co-authors Med Uni Graz: Moik Florian
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Abstract:: BACKGROUND: With population pharmacokinetic (PK) modeling more readily available and PK-guided prophylaxis endorsed by current hemophilia guidelines, we conducted a systematic review to summarize current evidence in the literature. OBJECTIVES: To assess the efficacy of PK-guided compared with non-PK-guided prophylaxis. METHODS: We did not restrict inclusion to specific study design labels and included all studies consisting of at least one distinct cohort arm receiving PK-guided prophylaxis. We searched the following databases from inception to date of search: MEDLINE, Embase, CENTRAL, ClinicalTrials.gov, and the EU Clinical Trial Register. Following title, abstract, and full-text screening conducted independently by 2 review authors, we summarized studies qualitatively and synthesized included randomized clinical trials (RCTs) quantitatively by fitting random-effects models. RESULTS: Search of databases on February 3, 2023, yielded 25 studies fitting our inclusion criteria. Of those, only 2 RCTs and 17 nonrandomized studies included a standard prophylaxis comparator group. Furthermore, risk of bias in the latter was substantial, primarily due to before-after study designs and retrospective comparator groups. Thus, nonrandomized studies were only presented qualitatively. A random-effects meta-analysis of the 2 identified RCT remained inconclusive with regards to bleeding outcomes (ratio of means, 1.15; 95% CI, 0.85-1.56) and factor consumption (ratio of means, 0.82; 95% CI, 0.58-1.18). CONCLUSION: Evidence in the literature suggesting a clinical benefit of PK-guided over standard fixed-dose prophylaxis was weak and mainly found in nonrandomized studies limited by lack of concurrent controls, heterogeneity in outcome reporting, small sample sizes, and high risk of bias.
Find related publications in this database (Keywords): factor IX; factor VIII; hemophilia A; hemophilia B; hemorrhage; systematic review