Reducing Treatment Burden in Hemophilia: Guy Young, MD


The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed improving outcomes in hemophilia with fitusiran.

“We're looking for treatments that reduce the treatment burden. We can do that 1 of 2 ways. One is by the mode of administration... Subcutaneous is a lot easier than IV...The other treatment burden issue is the frequency. Rather than having to have treatment1 to 2 to 3 times per week, we'd like to be able to administrate less often... Fitusiran is once per month in the ATLAS-INH study, and in the future we’ll explore other possible dosing regimens.”

Monthly prophylactic doses of fitusiran, an siRNA therapeutic targeting antithrombin, reduced bleeding in people with hemophilia A or B with or without inhibitors compared with those only given on-demand treatment.

These findings, from the phase 3 ATLAS-INH study (NCT03417102), were presented at the 63rd Annual American Society of Hematology (ASH) Meeting, December 11-14, 2021, by Guy Young, MD, director, Hemostasis and Thrombosis Program, Children’s Hospital of Los Angeles and professor of Pediatrics, Keck School of Medicine, University of Southern California.

Young and colleagues found that 65.8% of treated patients with both hemophilia A and B had treated bleeding events reduced to 0. In general, treated participants also had higher physical health domain health-related quality of life scores (both P <.0001).

GeneTherapyLive spoke with Young to learn more about unmet needs in people with hemophilia. He discussed treatment adherence issues with IV factor therapy and how less frequent fitusiran treatments could reduce patients’ treatment burden.

Young G, Srivastava A, Kavkli K, et al. Efficacy and safety of fitusiran prophylaxis, an siRNA therapeutic, in a multicenter phase 3 study (ATLAS-INH) in people with hemophilia A or B, with inhibitors (PwHI). Presented at: 63rd Annual ASH Meeting; December 11-14, 2021, Atlanta, GA. Abstract 4.
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