What question did this study set out to answer?

To evaluate the safety, pharmacodynamics, and efficacy of monthly fitusiran in individuals with moderate or severe hemophilia A or B with inhibitors.

February 17, 2021Journal of Thrombosis and HaemostasisOpen Access

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

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Authors

JPJohn PasiQueen Mary University of London TLToshko LissitchkovNational Specialized Hospital for Active Treatment of Hematologic Diseases VMVasily MamonovNational Medical Research Center for Hematology

Discussion

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Implication

Open-label phase 1 trial reveals reduced bleeding and lowered antithrombin in hemophilia A or B with inhibitors, suggesting improved hemostatic balance.

Key Points

To evaluate the safety, pharmacodynamics, and efficacy of monthly fitusiran in individuals with moderate or severe hemophilia A or B with inhibitors.
Open-label phase 1, part D trial enrolled 17 males with moderate to severe hemophilia A or B who possessed inhibitors.
Participants received three once-monthly subcutaneous doses of fitusiran at either 50 mg (n = 6) or 80 mg (n = 11) with follow-up for up to 112 days.
At nadir, mean (SEM) antithrombin activity decreased from baseline by 82.0% (2.2) in the 50 mg group and 87.4% (0.7) in the 80 mg group, which increased thrombin generation.
No thrombotic events occurred, and the most common adverse event was injection site erythema (n = 8); 11 of 17 participants (64.7%) had zero bleeding episodes over an observation period averaging 69.4 (SD 16.3) days.
Mean (SEM) Haemophilia Quality of Life Questionnaire for Adults scores improved from baseline, changing by -9.2 (2.9) overall and -12.3 (3.9) in the physical health domain.

Cite This Study

Pasi et al. (2021) studied this question.

synapsesocial.com/papers/6a94838b18fa85d8c46e5afa https://doi.org/10.1111/jth.15270

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Implication

Open-label phase 1 trial reveals reduced bleeding and lowered antithrombin in hemophilia A or B with inhibitors, suggesting improved hemostatic balance.

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

Discussion

Member takes

Implication

Key Points

Cite This Study

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

Discussion

Member takes

Implication

Key Points

Cite This Study

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