This protocol addresses patients with Alpha-1-antitrypsin deficiency (AATD) who have developed advanced liver disease as a direct consequence of their condition. The hepatic manifestation of AATD represents a distinct and serious complication requiring specific management considerations.
In patients with AATD who reach advanced stages of liver disease, approved pharmacological options for the hepatic disease are absent. The management approach for this sub-population is therefore sharply focused on a single definitive intervention, reserved for those meeting criteria for severe and terminal disease.
The protocol for this scenario centres on an organ-based surgical intervention as the primary management option for qualifying patients with advanced disease. The full eligibility criteria, workup requirements, and decision algorithm are contained in the complete protocol.