C1 INHIBITOR ADMINISTRATION IN PEDIATRIC PATIENTS WITH HEREDITARY ANGIOEDEMA: PATIENT COMPLIANCE WITH INTRAVENOUS THERAPY

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Introduction Adherence in a pediatric population can be a barrier to therapy. Here we report compliance, safety, efficacy and QoL outcomes in patients aged 6-11years who received intravenous (IV) C1-INH for routine prevention of angioedema attacks. Methods In a single blind, crossover design, patients received IV 500U and 1000U of C1-INH every 3-4 days for 12 weeks. Endpoints assessed were the monthly normalized number of attacks (NNA), adverse event reporting, completion of the youth version of the EQ-5D-Y descriptive system/VAS questionnaire, and C1 INH treatment adherence. Results Of 12 enrolled patients, 7 (58.3%) were female with a median (range) age 10.0 (7-11) years. All 12 patients were administered ≥90% of all C1-INH doses. Ten (83.3%) patients were 100% compliant with receiving all doses (n=480 doses) across both treatment arms. Overall, 58.3% and 91.7% of patients achieved a ³70% reduction from baseline with 500U and 1000U C1-INH, respectively. No patients discontinued due to an AE and few injection site reactions were noted; one patient (8.3%) who received 1000U C1-INH reported 1 related TEAE of mild pruritus and 1 related TEAE of mild erythema. The mean (SD) change in the EQ-5D VAS score from baseline to week 9 of treatment with 500U and 1000U C1-INH was 10.4 (19.0) and 21.6 (13.4), respectively. Conclusions This study demonstrated that high levels of compliance with IV administration of C1-INH can be achieved in a pediatric population. IV administration of C1 INH is well tolerated, efficacious and indicative of improved QoL.