However, whether IV augmentation therapy has clinical efficacy remains unproven by a randomized clinical trial. A total of 1,129 individuals were enrolled from 37 participating clinical centers. The results showed that patients receiving augmentation therapy experienced a lower mortality risk compared to those not receiving therapy. When IV augmentation therapy is considered, proper patient selection is essential. Table 2 presents criteria for initiating augmentation therapy in patients with ATT deficiency, and Table 3 lists management steps for such patients.
Advocates suggest that preventing any decline in lung function is important.
Skeptics cite the absence of evidence for efficacy when airflow obstruction is severe and suggest that augmentation therapy confers little benefit under these circumstances. Another controversy is whether to continue to administer augmentation therapy to patients who have had lung transplantation for end-stage emphysema due to ATT deficiency. No consensus exists on this issue, and most centers do not administer augmentation therapy after lung transplantation. In addition to IV augmentation therapy, optimal standard therapy for patients with airflow obstruction is crucial. This should include strict avoidance of cigarette smoking, maximal supportive therapy with bronchodilators, pulmonary rehabilitation, nutritional support, and oxygen when indicated.