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Efficacy of rFIXFc versus N9-GP Prophylaxis in Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PARADIGM 2 Trials.

Maria Elisa MancusoDaniel ErikssonAletta FalkZalmai HakimiPiotr WojciechowskiMarlena WdowiakRobert Klamroth
Published in: Journal of blood medicine (2023)
The study did not reveal any significant differences in the efficacy of rFIXFc and N9-GP prophylaxis. Given differences in trough levels (rFIXFc dosing was targeted to achieve a trough 1-3 IU/dL above baseline versus a reported estimated N9-GP mean trough of 27.3 IU/dL), interpreting plasma FIX levels as potential surrogate efficacy markers requires consideration of compound-specific pharmacokinetic profiles.
Keyphrases
  • dna methylation
  • risk assessment