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The following is a summary of “Impact of Donidalorsen Taken Every 8 Weeks in Patients With Hereditary Angioedema: Two-Year Update,” published in the February 2024 issue of Journal of Allergy and Clinical Immunology by Manning et al.
Researchers conducted a retrospective study on donidalorsen Q8W in patients with Type 1/2 HAE, showing a 76% attack rate reduction and sustained efficacy with good tolerability over 2 years.
They used fixed dosing (Weeks 1–16, donidalorsen 80 mg Q4W) and flexible dosing (Weeks 17–105, 80 mg Q4W, 80 mg Q8W, or 100 mg Q4W).
The results showed 8 of 17 patients received donidalorsen Q8W; 5 remained attack-free, 3 returned to Q4W by Year 2 and 5 (63%) reported TEAEs; 1 (13%) had a drug-related TEAE (abdominal pain). Mean monthly attack rate was 0.04 (range 0.0–0.3; 95% CI −0.07 to 0.15; median 0.0) at Year 2 vs. 0.28 (range 0.0–1.7; 95% CI −0.23 to 0.78; median 0.0) at Year 1, showing 83% reduction from baseline (2.70). Mean prekallikrein change was −59.1 (Q8W) and −54.3 (Q4W). QoL scores improved to 27.4 in Year 2 and 25.0 in Year 1.
Investigators found donidalorsen Q8W was well-tolerated, showed durable efficacy in reducing HAE attacks, and had prekallikrein levels similar to Q4W dosing. They supported continued study of Q8W dosing.
Source: jacionline.org/article/S0091-6749(23)01522-1/fulltext
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