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Background: Patients with hereditary angioedema with C1 inhibitor deficiency or dysfunction have burdensome recurrent angioedema attacks. The safety, efficacy, and health‐related quality of life (HRQoL) outcomes of C1 inhibitor (C1‐INH) prophylaxis (intravenously administered) in patients aged 6‐11 years were investigated. Methods: Eligible patients were enrolled in a randomized, single‐blind, crossover, phase 3 trial. After a 12‐week baseline observation period (BOP), patients received 500 or 1000 U C1‐INH, twice weekly, for 12 weeks before crossing over to the alternate dose for 12 weeks. The primary efficacy end‐point was the monthly normalized number of angioedema attacks (NNA). HRQoL was assessed using the EuroQoL 5‐dimensional descriptive system youth version and visual analog scale (EQ‐VAS). Results: Twelve randomized patients had a median (range) age of 10.0 (7‐11) years. Mean (SD) percentage reduction in monthly NNA from BOP was 71.1% (27.1%) with 500 U and 84.5% (20.0%) with 1000 U C1‐INH. Mean (SD) within‐patient difference (−0.4 [0.58]) for monthly NNA with both doses was significant (P = 0.035 [90% CI, −0.706 to −0.102]). Cumulative attack severity, cumulative daily severity, and number of acute attacks treated were reduced. No serious adverse events or discontinuations occurred. Mean EQ‐VAS change from BOP to week 9 of treatment (500 U C1‐INH, 10.4; 1000 U C1‐INH, 21.6) was greater than the minimal important difference, indicating a meaningful HRQoL change. Conclusions: C1‐INH prophylaxis was effective, safe, and well tolerated in children aged 6‐11 years experiencing recurrent angioedema attacks. A post hoc analysis indicated a meaningful improvement in HRQoL with C1‐INH. Trial registration: ClinicalTrials.gov identifier NCT02052141. [ABSTRACT FROM AUTHOR]

Background Human plasma-derived nanofiltered C1 esterase inhibitor (C1 INH-nf) is used to treat acute angioedema attacks in patients with hereditary angioedema ( HAE), but data regarding use in children are sparse. Methods Patients 2 to <12 years of age, body weight ≥10 kg, with a diagnosis of HAE type I or II, were recruited for a multicenter open-label trial. Patients were recruited into 2 weight categories (10-25 kg, >25 kg). Each weight category included 2 dosing levels: C1 INH-nf (500 units [U], 1000 U) and C1 INH-nf (1000 U, 1500 U), respectively. Patients experiencing an angioedema attack were given a single intravenous dose. Primary efficacy end-point was the onset of unequivocal relief of the defining symptom within 4 h following initiation of C1 INH-nf treatment. Results Nine children were treated: 3 (10-25 kg) received 500 U; 3 (>25 kg) received 1000 U; and 3 (>25 kg) received 1500 U. The lower weight/higher dose category (10-25 kg, 1000 U) was not successfully enrolled. All patients completed the study. Most angioedema attacks (n = 5) were abdominal. All patients met the primary end-point; median time to unequivocal symptom relief was 0.5 (range: 0.25-2.5) h. Doses of C1 INH-nf ranged from 20.8 to 51.9 U/kg. Conclusions Treatment of a single angioedema attack with C1 INH-nf doses of 500 U (in patients 10-25 kg), 1000 U, and 1500 U (in patients >25 kg) were well tolerated. Doses of C1 INH-nf <1000 U may be appropriate in some pediatric patients. [ABSTRACT FROM AUTHOR]