title: Safety and efficacy of physician-supervised self-managed C1 inhibitor replacement therapy.
abstract: BACKGROUND
  C1 inhibitor (C1INH) has recently been approved in the USA for the treatment of acute attacks in hereditary angioedema (HAE) patients. The literature suggests that treatment with C1INH is most effective when administered early in an attack. Home infusion of C1INH allows for the earliest possible intervention since patients can initiate therapy at the first sign of symptoms.
  
  
  METHODS
  We performed an observational, prospective study on 39 subjects with HAE utilizing two groups of patients: one receiving on-demand C1INH replacement therapy in a medical facility and the other self-managing on-demand C1INH replacement therapy in the home setting under the supervision of a treating physician. All subjects completed online questionnaires weekly for 8 weeks.
  
  
  RESULTS
  There were statistically significant decreases in attack duration (p < 0.0001), pain medication use (p < 0.0001) and graded attack severity (p < 0.005) in the subjects who received C1INH in the home setting versus the clinic-based group. Attack frequency was similar between the groups. The home group experienced more frequent injection-related side effects; however, the clinic group noted more severe adverse events from C1INH.
  
  
  CONCLUSIONS
  Physician-supervised self-managed C1INH replacement therapy is a safe and effective treatment for patients with HAE with potential benefits in diminishing attack duration and attack severity.
authors: University of California, San Diego, La Jolla, CA 92093-0732, USA.
authors: Louanne M
authors: LM
authors: Tourangeau
authors: Anthony J
authors: AJ
authors: Castaldo
authors: Donna K
authors: DK
authors: Davis
authors: James
authors: J
authors: Koziol
authors: Sandra C
authors: SC
authors: Christiansen
authors: Bruce L
authors: BL
authors: Zuraw
mesh_terms: D000328:Adult; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage* / Q000009:adverse effects; D018450:Disease Progression; D056947:Enzyme Replacement Therapy / Q000009:adverse effects*; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010817:Physician-Patient Relations; D011446:Prospective Studies; D012646:Self Administration / Q000009:adverse effects; D011795:Surveys and Questionnaires; D013997:Time Factors
chemical_list: D050718:Complement C1 Inhibitor Protein