[Evaluation of replacement therapy in emphysema caused by alpha 1-antitrypsin deficiency]. / Evaluación del tratamiento sustitutivo del enfisema por déficit de alfa-1-antitripsina.

Assessment of alpha 1-antitrypsin replacement therapy (AAT) for emphysema. Patient characteristics were analyzed along with the possible side effects of the treatment and its efficacy in maintaining appropriate AAT blood levels. Lung function changes were also studied. The treatment protocol began with 4 weekly intravenous doses of 60 mg/kg AAT (Prolastin) and continued with monthly doses of 240 mg/kg. AAT serum levels were measured before each dose. Every 6 months pulmonary function tests (spirometry, plethysmography and CO transfer) were performed. Thirteen patients (mean age 46 yr) have been studied since 1988. Mean initial FEV1 was 0.79 l. Over 250 doses have been infused with no significant side effects reported. AAT levels before treatment in 3 patients were lower than that considered protective (50 mg/dl). Function tests results indicated stabilization of spirometric values in most cases. Diagnosis of AAT deficiency is delayed considerably, meaning that significant functional deterioration takes place before replacement therapy begins. No side effects of treatment have been observed. Until an appropriate interval between doses has been established, each patient's AAT levels must be monitored.