| Augmentation therapy | | |
|
Intravenous augmentation therapy is recommended for:
individuals with FEV1 ≤65% pred;
in those with lung disease related to AATD and FEV1 >65% pred, there is need for a discussion with each individual regarding the potential benefits of reducing lung function decline with consideration of the cost of therapy and lack of evidence for such benefit;
individuals with necrotising panniculitis Intravenous augmentation therapy is not recommended for:
individuals with the MZ genotype of AATD;
individuals with lung disease due to AATD who continue to smoke;
individuals with AATD and emphysema or bronchiectasis who do not have airflow obstruction;
the treatment of liver disease due to AATD;
individuals who have undergone liver transplantation Additional recommendations regarding dosing of intravenous augmentation therapy:
weekly doses higher than the current US FDA-approved dose are not recommended;
monitoring of trough AAT blood levels to evaluate the adequacy of AAT augmentation dosing is not recommended
|
Several randomised clinical trials in severe AATD have shown intravenous augmentation therapy to reduce the progression of emphysema as assessed by CT densitometry There is no evidence to support efficacy of AAT augmentation therapy in PiSZ, PiMZ or current smokers of any protein phenotype Clinical trials have used fixed doses of AAT determined by body weight; whether individualising dosage based on trough levels for each patient has any benefit requires confirmation
|
| Lung volume reduction surgery and endoscopic bronchial valve placement | | |
| |
Surgical volume reduction and endoscopic bronchial valve placement may be considered in selected patients with AATD, but further studies are needed to confirm the role of such therapies The optimal results of these techniques are obtained when a careful appraisal of risks and benefits are performed by a multidisciplinary team experienced in lung volume reduction and AATD
|
| Lung transplantation | | |
| |
The survival benefit of lung transplant in AATD patients is not clear In general, patients with AATD have improved quality of life following lung transplantation Referral timing, rate of decline in lung function, health status and social support differ from patient to patient, and will have an influence on the evaluation for transplant The role of post-transplant augmentation therapy in particular needs to be explored
|