Recombinant human C1-esterase inhibitor for the prevention of acute hereditary angioedema attacks: a case report

Abstract

Background: Prophylaxis may be considered for frequent acute attacks of hereditary angioedema (HAE); moreover, prophylaxis efficacy may impact direct costs associated with medical utilization/medication.Objectives: To present data on the efficacy and cost savings of investigational prophylactic treatment with recombinant human C1-esterase inhibitor (rhC1-INH).Methods: Retrospective chart review of patient with HAE.Results: A white female aged 52 years who had experienced HAE attack symptoms since age 16 years was diagnosed at age 37 years with type 1 HAE. She received danazol 400 mg/d; attempts to lower her dose were unsuccessful. She continued to experience daily extremity swelling and had abdominal edema with intense pain severe enough to warrant hospitalization every 2–3 months. In 2012, she began prophylaxis with plasma-derived C1-INH (pdC1-INH) 1000 IU 3 times weekly (TIW) at a weekly cost of ∼$16,500. During a 12-month period (September 2013 to September 2014), authors documented 13 emergency room (ER) visits for acute HAE attacks, but other records suggest there were several additional ER visits. The pdC1-INH dose was increased to 1500 IU TIW in October 2014 at a weekly cost of ∼$24,829, and the number of ER visits declined (n = 9 during a 14-month period). In December 2015, patient began prophylaxis TIW with rhC1-INH 2100 IU for an annual cost savings of $400,660 versus pdC1-INH. From initiation of rhC1-INH to October 2017, patient required only 1 ER visit.Conclusions: rhC1-INH prophylaxis for HAE reduced occurrence of ER visits and provided potential cost benefits for both drug costs and medical utilization.