title: [Long-term therapy of alpha 1-antitrypsin-deficiency-associated pulmonary emphysema with human alpha 1-antitrypsin].
abstract: alpha 1-antitrypsin (alpha 1-AT) deficiency is a genetic disorder characterized by low serum levels of alpha 1-AT and a high risk of pulmonary emphysema at a young age. The resulting surplus of proteases, mainly of neutrophil elastase, can be balanced by i.v. augmentation with alpha 1-AT. However, it is not clear if affected patients benefit from long-term augmentation therapy and no long-term safety data are available. We examined 443 patients with severe alpha 1-AT deficiency and pulmonary emphysema receiving weekly i.v. infusions of 60 mg/kg body weight alpha 1-AT in addition to their regular medication. The progression of the disease was assessed by repeated lung function measurements, particularly the decline in forced expiratory volume in 1 second (delta FEV1). 443 patients with alpha 1-AT deficiency tolerated augmentation therapy well with few adverse reactions. The delta FEV1 in 287 patients with available follow-up data was 57.1 +/- 31.1 ml per year. Stratified for baseline FEV1, the decline was 35.6 +/- 21.3 ml in the 108 patients with an initial FEV1 < 30% and 64.0 +/- 26.4 ml in the 164 with 30% < FEV1 < or = 65% of predicted normal (p = 0.0008). The remaining 15 patients had an initial FEV1 > 65%. Long-term treatment with i.v. alpha 1-antitrypsin in patients with severe alpha 1-Pi deficiency is feasible and safe. The decline in forced expiratory volume in one second is related to the initial forced expiratory volume in one second as in alpha 1-antitrypsin deficient patients not receiving augmentation therapy.
authors.affiliation: Ruhrlandklinik, Zentrum für Pneumologie und Thoraxchirurgie, Essen.
authors.forename: M
authors.initials: M
authors.lastname: Wencker
authors.forename: N
authors.initials: N
authors.lastname: Banik
authors.forename: R
authors.initials: R
authors.lastname: Buhl
authors.forename: R
authors.initials: R
authors.lastname: Seidel
authors.forename: N
authors.initials: N
authors.lastname: Konietzko
mesh_terms: D000328:Adult; D018450:Disease Progression; D005240:Feasibility Studies; D005260:Female; D005541:Forced Expiratory Volume / Q000187:drug effects; D006801:Humans; D007262:Infusions, Intravenous; D008134:Long-Term Care; D008297:Male; D008875:Middle Aged; D010641:Phenotype; D011656:Pulmonary Emphysema / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage*; D019896:alpha 1-Antitrypsin Deficiency / Q000235:genetics / Q000628:therapy*
chemical_list: D000515:alpha 1-Antitrypsin
vernacular_title: Langzeittherapie des alpha 1-Antitrypsin-mangelassoziierten Lungenemphysems mit humanem alpha 1-Antitrypsin.