549 Management of Twenty-Five Pediatric Patients with Hereditary Angioedema (Hae) Undergoing Home Treatment—A Clinical Surveillance Program

Abstract

BackgroundHereditary angioedema (HAE) is a rare disorder characterized by C1 esterase inhibitor (C1-INH) deficiency. Clinically, HAE is characterized by relapsing episodes of edema at various body sites followed by disease-free intervals of variable duration. Episodes of upper airway obstruction (usually laryngeal edema) are potentially life-threatening and many patients died by asphyxiation in the families. In literature Longhurst et al, Buygun et al and Levi et al showed improvement of quality of life in patients with hereditary angioedema due to home treatment with C1-inhibitor-concentrate.MethodsWe investigated in a cohort study the integration of a clinical surveillance program into home treatment of pediatric patients suffering from hereditary angioedema. Parameters investigated were overall coping of the pediatric patient with home treatment, documentation of efficacy/safety of C1-INH concentrate (Berinertâ P, CSL Behring, Marburg), regular control of laboratory parameters (C1-inhibitor (INH) activity, C1-INH antigen, C4, hepatitis A, -B, -C-, HIV-1/2-, and parvovirus B19 serology) and quality of life parameters (hospitalization, absence from school).ResultsTwenty-five pediatric HAE patients (6 male, 19 female) have so far been investigated. Twenty-one patients suffer from HAE type I, 4 patients from HAE type II. Median age is 13.7 years (range: 2.8–17.4 years), first diagnosis of HAE took place at the median age of 5 years (range: 0.1–15.9 years) and first manifestation of HAE at the median age of 3.9 years (range: 0.3–11.7 years). Plasma C4 complement was reduced in nearly all patients (median: 2.4 mg/dL; range: <1.4–10 mg/dL) except one patient. All patients coped well with home treatment, compliance was excellent, clinical findings during regular medical control remained in the normal range and all parameters confirmed an improved quality of life, e.g., patients had not been hospitalized nor had they been absent from school. There were no adverse drug reactions due to administration of C1-INH concentrate.ConclusionsHome treatment might be also a valuable option for pediatric HAE patients not affecting compliance negatively and providing a significant positive impact on health-related quality of life.