[Evaluation after 2 years of substitutive treatment of PiZZ emphysema with alpha-1 antitrypsin. 9 cases].
Homozygous PiZZ individuals with a serum deficiency due to a defect in the secretion of the alpha 1-antitrypsin protein are at risk of developing severe panlobular emphysema. Tobacco smokers are particularly exposed to the disease which begins at an earlier age. Treatment by substitutive therapy with alpha 1-antitrypsin concentrates seems to be the only possibility. A two years' clinical trial was performed in 9 PiZZ patients, with more than 1,500 infusions being administered weekly. Serum AAT levels were used as guidelines to follow biochemical changes in the protease-antiprotease balance. From 0.16 g/l initially, the AAT level rose to 0.57 g/l after 7 months. No adverse reaction was observed during the trial; the concentrated protein was well accepted, ant the antielastase activity of the protein recovered after injection was equivalent to the activity injected. An attempt to administer the infusions monthly was stopped when we observed a dramatic decrease of the serum AAT level. Clinically, stabilization of the symptoms was noted. No degradation was observed in the patients who took part in the trial, even if no real improvement was detected.
Carles
P
P
Service de Médecine, Hôpital Purpan, Toulouse.
Constans
J
J
Pujazon
M C
MC
Arnaud
J
J
Lauque
D
D
Goudemand
M
M
D000328:Adult; D002985:Clinical Protocols; D005500:Follow-Up Studies; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D008875:Middle Aged; D010641:Phenotype; D011656:Pulmonary Emphysema / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D011669:Pulmonary Wedge Pressure; D012129:Respiratory Function Tests; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage / Q000032:analysis / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency
D000515:alpha 1-Antitrypsin
Bilan à deux ans du traitement substitutif de l'emphysème PiZZ par l'alpha 1-antitrypsine. Neuf cas.