Clinical management of hereditary angio‐oedema in children
- 1 June 2002
- journal article
- review article
- Published by Wiley in Pediatric Allergy and Immunology
- Vol. 13 (3), 153-161
- https://doi.org/10.1034/j.1399-3038.2002.01014.x
Abstract
Hereditary angio‐oedema (HAE) results from the deficiency of C1‐esterase inhibitor (C1‐INH). The clinical picture of this autosomal dominant disorder is characterized by recurrent attacks of subcutaneous oedema and/or potentially life‐threatening swelling of the submucosa. This review discusses the authors' decade‐long experience obtained in the treatment and follow‐up of pediatric patients with HAE. Twenty‐six children with HAE were reviewed. Pedigree analysis was performed in all cases to identify afflicted relatives. C1‐INH concentrate was reserved for the emergency treatment of acute oedematous attacks, whereas tranexamic acid and danazol were administered for short‐ or long‐term prophylaxis. Follow‐up care included laboratory tests and abdominal ultrasound, which was repeated at regular intervals. Twenty‐one children had Type I HAE and five suffered from Type II HAE. Clinical manifestations of the disease first occured in children when 2.5–12 years of age. Oedema formation primarily afflicted subcutaneous tissues. Mechanical trauma was identified as a precipitating factor in 20 patients. Pedigree analysis revealed 24 patients with relatives who suffered from HAE. Long‐term prophylaxis with tranexamic acid or danazol was initiated in 11 patients; two children required short‐term prophylaxis. No drug‐related adverse effects were observed, except for one case of delayed menarche. Therapy improved serum complement parameters significantly and substantially reduced the frequency and severity of clinical episodes. Adequate prophylaxis and follow‐up care can spare pediatric patients from oedematous attacks caused by HAE. Undesirable adverse effects can be avoided and the patient's quality of life enhanced considerably by administering the lowest effective drug dose.Keywords
This publication has 25 references indexed in Scilit:
- Side effects of long-term prophylaxis with attenuated androgens in hereditary angioedema: Comparison of treated and untreated patients☆☆☆★Journal of Allergy and Clinical Immunology, 1997
- Hereditary Angioedema in ChildrenPediatric Asthma, Allergy & Immunology, 1996
- Reduction in transmission of hepatitis C after the introduction of a heat‐treatment step in the production of C1‐inhibitor concentrateTransfusion, 1995
- Hereditary and Acquired C1-Inhibitor DeficiencyMedicine, 1992
- Long-term prophylaxis with C1-inhibitor (C1 INH) concentrate in patients with recurrent angioedema caused by hereditary and acquired C1-inhibitor deficiencyJournal of Allergy and Clinical Immunology, 1989
- Complement in the Pathophysiology of Human DiseaseNew England Journal of Medicine, 1987
- Human C.hivin.1 inhibitor: primary structure, cDNA cloning, and chromosomal localizationBiochemistry, 1986
- Danazol and stanozolol in long-term prophylactic treatment of hereditary angioedemaJournal of Allergy and Clinical Immunology, 1980
- REPLACEMENT THERAPY IN HEREDITARY ANGIOEDEMA: SUCCESSFUL TREATMENT OF TWO PATIENTS WITH FRESH FROZEN PLASMAThe Lancet, 1969
- Hereditary Angioneurotic Edema: Two Genetic VariantsScience, 1965