ABSTRACT
INTRODUCTION: Hereditary angioedema (HAE) with C1 inhibitor (C1-INH) deficiency is a rare autosomal dominant disease. Although the first symptoms can appear in childhood, the diagnosis's delay has a strong impact on the patient's quality of life. We analyzed clinical and laboratory characteristics and the drug therapy of pediatric patients with HAE in Brazil. METHODS: Medical records from 18 reference centers of HAE patients under 18 years of age were evaluated after confirmed diagnosis was performed by quantitative and/or functional C1-INH. RESULTS: A total of 95 participants (51 M:44 F; mean age: 7 years old) out of 17 centers were included; 15 asymptomatic cases were identified through family history and genetic screening. Angioedema attacks affected the extremities (73.5%), gastrointestinal tract (57%), face (50%), lips (42.5%), eyelids (23.7%), genitals (23.7%), upper airways (10%), and tongue (6.3%). Family history was present in 84% of patients, and the mean delay in the diagnosis was 3.9 years. Long-term prophylaxis (51/80) was performed with tranexamic acid (39/80) and androgens (13/80); and short-term prophylaxis (9/80) was performed with tranexamic acid (6/80) and danazol (3/80). On-demand therapy (35/80) was prescribed: icatibant in 7/35, fresh frozen plasma in 16/35, C1-INH plasma-derived in 11/35, and tranexamic acid in 12/35 patients. CONCLUSIONS: This is the first study on HAE pediatric patients in Latin America. Clinical manifestations were similar to adults. Drugs such as androgens and tranexamic acid were indicated off-label, probably due to restricted access to specific drugs. Educational programs should address pediatricians to reduce late diagnosis and tailored child therapy.
Subject(s)
Angioedemas, Hereditary/epidemiology , Adolescent , Anaphylaxis/etiology , Angioedemas, Hereditary/diagnosis , Angioedemas, Hereditary/therapy , Brazil/epidemiology , Child , Child, Preschool , Delayed Diagnosis , Disease Management , Female , Follow-Up Studies , Humans , Male , Public Health Surveillance , Quality of LifeABSTRACT
OBJECTIVE: This study analyzed the factors that predicted the hospitalization of children with asthma following standardized treatment in emergency care unit (ECU). METHODS: This retrospective study examined data collected from the clinical records of children, 14 years or younger, who were diagnosed with asthma (often with bronchopneumonia, pneumonia, or other illnesses) and treated at the ECU of Santo André from January 2005 to December 2009. The following data were analyzed: month and year of care, child's age and sex, period of observation, and need for hospitalization. A pediatrician confirmed the clinical diagnoses of all participants. The children were first given clinical treatments and were then admitted to ECU for follow-up assessment. RESULTS: The number of hospital admissions was analyzed, and correlations were found with regard to this variable and child age (χ(2) = 166.9; P = 0.00001), the presence of associated illnesses (χ(2) = 63.8; P < 0.00001), and the observation period length (χ(2) = 11.4; P = 0.009). The number of hospital admissions was not correlated with child sex (χ(2) = 0.013; P = 0.9) or time of year (χ(2) = 15.8; P = 0.1). The 3-day observation period was not significant (P = 0.4) with regard to the remainder of the variables in the multiple logistic regression analysis. CONCLUSIONS: Age, mainly children younger than 1 year, the presence of associated illnesses, and the observation period length predicted the hospitalization of children with asthma following treatment in ECU. Sex and seasonality did not affect the need for hospitalization.
