Photo Credit: SaevichMikalai

The following is a summary of “Patient-Reported Outcomes in the Phase III OASIS-HAE Study of Donidalorsen for Hereditary Angioedema,” published in the April 2025 issue of European Journal of Allergy and Clinical Immunology by Riedl et al. 

Hereditary angioedema (HAE) causes unpredictable swelling and severely affects quality of life (QoL). The OASIS-HAE study assessed donidalorsen for reducing attacks and improving disease control. 

Researchers conducted a retrospective study to assess donidalorsen’s impact on quality of life and patient-reported outcomes (PROs).  

They randomized patients with HAE to donidalorsen 80 mg or placebo every 4 or 8 weeks for 24 weeks in a double-blind, placebo-controlled study. PROs included Angioedema (AE)-QoL Questionnaire, angioedema control test (AECT), patient global impression of severity (PGIS), and work productivity and activity impairment questionnaire plus classroom impairment questions (WPAI+CIQ). 

The results showed 88% in the donidalorsen Q4W group vs. 45% in placebo had ≥6-point AE-QoL improvement. Least-squares mean (LSM) differences vs. placebo in AE-QoL domains were functioning Q4W −24.5, Q8W −16.1; fears/shame Q4W −23.9, Q8W −20.1; nutrition Q4W −15.7, Q8W −10.7. AECT scores improved: Q4W 6.0, Q8W 4.1. PGIS showed 82% (Q4W) vs. 44% (placebo) with decreased severity. Donidalorsen Q4W improved WPAI+CIQ presenteeism, work/school, and activity impairment. 

Investigators significantly improved QoL and other PROs with donidalorsen vs. placebo in patients with HAE. They observed consistent benefits across multiple PRO measures. 

Source: onlinelibrary.wiley.com/doi/full/10.1111/all.16563