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July 1, 2026Blood

One-stage Assay Factor VIII Activity Reflects AAV-Derived Factor VIII-Enhanced Thrombin Activation and Predicts Phenotype

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Authors

ASAnna R. SternbergCWConnor T. WatsonRDRJ Davidson

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Overview

Randomized trial examines AAV-derived factor VIII activity in hemophilia A, suggesting improved outcome predictions.

Key Points

  • This research aims to determine how one-stage assay FVIII activity reflects AAV-derived FVIII function and its predictive value for hemophilia A phenotype.
  • Compared AAV-derived FVIII-SQ activation and function in gene therapy-treated mice and SPK-8011 trial participants.
  • Analyzed assay discrepancies using one-stage assay (OSA) and chromogenic substrate assay (CSA) across both species.
  • Performed negative binomial regression analysis of 23 SPK-8011 participants over 99 cumulative patient-years.
  • AAV-FVIII-SQ showed enhanced thrombin-mediated activation detectable by OSA, but not by CSA.
  • OSA activity correlated better with hemostatic function compared to CSA, indicating its predictive value for annualized bleeding rate.
  • Findings suggest OSA FVIII activity is more reflective of AAV-FVIII-SQ function within the intrinsic tenase complex.

Cite This Study

Sternberg et al. (2026) studied this question.

synapsesocial.com/papers/6a44af635cd2549c8bc445a3https://doi.org/10.1182/blood.2025032890
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Default one-stage assay for hospitalized hemophilia A patients: A cautionary tale2025
  2. 2445 Hemophilia A: when the bleeding patient looks worse than lab testing would indicate2025
  3. 3Variability in combinations of <scp>APTT</scp> reagent and substrate plasma for a one‐stage clotting assay to measure factor <scp>VIII</scp> products2024 · 1 citations
  4. 4Overcoming Hemophilia A Gene Therapy Limitations with an Enhanced Function Factor VIII Variant2024 · 1 citations
  5. 5High agreement between one-stage clot-based and chromogenic factor VIII activity assays.2025