ArticleAdvances in therapy2025
Comparative Effectiveness of Valoctocogene Roxaparvovec and Efanesoctocog Alfa in the Treatment of Severe Hemophilia A: A Matching-Adjusted Indirect Comparison of Bleeding Frequency.
Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
2 citing papers in PubMed.
- Roctavian withdrawal: exploring the gap between innovation and healthcare system readiness in hemophilia A gene therapy.Research and practice in thrombosis and haemostasis · 2026Review
- The Molecular Pathology of Non-Malignant Haematological Disease.British journal of biomedical science · 2026Review
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHemophilia A, an X-linked recessive bleeding disorder, is characterized by reduced factor VIII (FVIII) activity. Hemophilia A can significantly impact a person's quality of life because of the risk of spontaneous bleeding. Treatment for hemophilia A aims to prevent bleeding from occurring. The innovation of gene therapies for use in hemophilia has the potential to replace prophylaxis treatment by enabling a single treatment infusion that sustains endogenous FVIII production for years. This analysis assessed the comparative effectiveness of valoctocogene roxaparvovec, a gene therapy, versus prophylactic FVIII replacement therapy with efanesoctocog alfa, given the current lack of comparative evidence between these treatments.
methodsComparison between valoctocogene roxaparvovec and efanesoctocog alfa was conducted using matching-adjusted indirect comparison (MAIC). Patient-level data from the phase III GENEr8-1 trial of valoctocogene roxaparvovec were reweighted to align with baseline characteristics of aggregate-level data from XTEND-1, a phase III trial of efanesoctocog alfa. Matching variables included proportion of patients with zero treated bleeds and mean annualized bleed rate (ABR) for treated bleeds prior to initiating therapy. Following reweighting, the proportion of patients who had experienced zero bleeds after 52 weeks was compared, along with mean ABR-each population was assessed for treated bleeds, treated joint bleeds, and treated spontaneous bleeds.
resultsFollowing MAIC weighting, patients treated with valoctocogene roxaparvovec had higher odds of experiencing zero treated bleeds (OR 2.68, 95% CI 1.18-6.14) and zero treated joint bleeds (OR 2.75, 95% CI 1.09-6.86) compared with efanesoctocog alfa. Odds of zero treated spontaneous bleeds were also higher for valoctocogene roxaparvovec, but not statistically significant. Mean ABRs for treated bleeds, treated joint bleeds, and treated spontaneous bleeds were similar between groups, with no statistically significant differences.
conclusionThis MAIC suggests that valoctocogene roxaparvovec provides a greater likelihood of patients experiencing zero treated bleeds compared with efanesoctocog alfa during the first year following treatment initiation.
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