Cinryze is Efficacious for Hereditary Angioedema (HAE) Attack Prevention In Pediatric Patients: Final Phase 3 Efficacy And Safety Results
Saturday, March 3, 2018
South Hall A2 (Convention Center)
Emel Aygören-Pürsün, MD, Daniel F. Soteres, MD FAAAAI, Sandra Nieto–Martinez, Jim Christensen, MD FAAAAI, Kraig W. Jacobson, MD FAAAAI, Dumitru Moldovan, MD, PhD, Arthur Van Leerberghe, Tom Tang, Peng Lu, MD, PhD, Inmaculada Martinez-Saguer, MD

C1 inhibitor (C1-INH; Shire, Lexington, MA, USA) is approved for the treatment and pre-procedural prevention of HAE attacks in patients aged ≥2 years (EU), and routine prevention of attacks in patients ≥6 years (EU) and ≥12 years (US). C1-INH’s safety and efficacy for attack prevention in children aged 6-11 years was investigated in a phase 3 study.


The multicenter single-blind study required children to have an average of ≥1.0 angioedema monthly attacks that were moderate, severe, or required acute treatment during the 12-week baseline observation period. In a crossover design, patients received 500U and 1000U C1-INH every 3-4 days for 12 weeks. The primary endpoint was the monthly-normalized number of attacks (NNA).


Of 12 enrolled patients (HAE type I), 7 (58.3%) were female with a median (range) age and BMI of 10.0 (7-11) years and 18.6 (13.1-28.2) kg/m2, respectively. NNA with 500U and 1000U differed significantly (mean [SD] within subject difference -0.4 [0.58]; P=0.035). The mean (SD) percent reduction in NNA compared to baseline (3.7 [3.2] attacks) was 71.1% (27.1%) and 84.5% (20.0%), respectively. Overall, 58.3% and 91.7% of patients achieved a ≥70% reduction from baseline with 500U and 1000U C1-INH, respectively. Cumulative attack severity, cumulative daily severity, and number of treatment-requiring acute attacks were reduced. There were no serious adverse events or discontinuations. Patient’s health status (EQ-5D Y) improved, particularly with 1000U C1-INH.


As observed in prior C1-INH studies, 500U and 1000U C1-INH were effective, safe, and well-tolerated for routine prevention of HAE attacks in children aged 6-11 years.