Comparison of Drugs Used for Prophylaxis in Hemophilia a or B with Inhibitors: A Systematic Review and Frequentist Network Meta-Analysis of Randomized Clinical Trials
Abstract
Background:
Frequent infusions with hemostatic agents in hemophilia patients can lead to the formation of anti-drug antibodies that can bind with clotting factors and inhibit their function. Inhibitor formation leads to worsening of hemophilia symptoms and is difficult to treat with clotting factor concentrates and is categorized as hemophilia with inhibitors. This network meta-analysis (NMA) aims to assess the efficacy of different prophylactic drugs for hemophilia A and B with inhibitors and provide a comparison among these drugs.
Methods:
Cochrane and PRISMA guidelines were followed to conduct this NMA. A comprehensive literature search was performed in PubMed and Embase with keywords for “hemophilia” AND “drug therapy” from the inception of data till April 2024. We screened a total of 1052 articles and 6 randomized clinical trials (RCTs, N=457) were included in this NMA.The estimation of parameters across different models was accomplished using the graph theoretical approach, which entails constructing a Moore-Penrose pseudoinverse matrix with the Netmeta package in R (version 4.3.1). NMA was registered on PROSPERO CRD42024532136.
Results:
In 6 RCTs (N=457), 38 patients were treated with fitusiran prophylaxis, 114 patients with concizumab prophylaxis, 147 patients with emicizumab prophylaxis, 17 patients with FEIBA NF prophylaxis, 26 patients with AICC prophylaxis, and 115 patients were only treated on demand without prophylaxis. Patients' ages ranged from 7 to 75 years.…
How strong is the evidence?
Evidence Hub reads this as a meta-analysis, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 457 participants or records.
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