Single Center Experience with Amyloid Patients in the Current Era of Disease Specific Medications

Both amyloid light chain (AL) amyloidosis and transthyretin-related (TTR) amyloid have been viewed with reluctance to proceed with heart transplant. AL amyloid in particular has been a contraindation for heart transplant as the underlying disease process progresses. In the current era of proteasome inhibitors, AL amyloid patients may now be adequately treated and undergo heart transplant with subsequent bone marrow transplant. In our program, we have treated 7 AL amyloid patients who developed end-stage heart disease. These 7 patients subsequently received a heart transplant and 3 received a bone-marrow transplant 1-year later. All AL amyloid patients were treated with bortezomib. We have also transplanted 12 TTR patients and now present their outcomes. Between 2006 and 2014, we assessed 665 heart transplant patients and found 7 patients who had AL amyloid, 12 with TTR amyloid, and compared these to 15 restrictive non-amyloid patients. The following outcomes were assessed: 1-year actuarial survival, freedom from left ventricular dysfunction, freedom from non-fatal major adverse cardiac events (NF-MACE: myocardial infarction, new congestive heart failure, percutaneous coronary intervention, implantable cardioverter defibrillator/pacemaker insertion, stroke), and freedom from any treated rejection, both cellular and antibody mediated. Among the AL amyloid, TTR, and restrictive non-amyloid patients, there was no significant difference between the 3 groups in 1 year survival, freedom from left ventricular dysfunction, NF-MACE, and treated rejections, both cellular and antibody mediated (see table). No amyloid was observed in the endomyocardial biopsies after heart transplant. AL and TTR amyloid have acceptable outcomes after heart transplant. An approach using bortezomib in AL amyloid patients allows them to suppress amyloid production while subsequently waiting to undergo bone marrow transplant 1-year later.