Cardiac Transplantation for Amyloid Heart Disease: The United Kingdom Experience Simon W. Dubrey, FRCP, a Margaret M. Burke, FRCPath, b Philip N. Hawkins, FRCP, c and Nicholas R. Banner, FRCP a,b Background: Heart transplantation (TX) for cardiac amyloidosis is uncommon because of concern about progression of amyloid in other organs and the possibility of amyloid deposition in the donor heart. Methods: Records of all 24 patients with amyloid heart disease who have undergone TX in the United Kingdom were examined. Seventeen patients had AL amyloidosis (AL) and 7 had non-AL forms of amyloidosis (non-AL). Results: Survival of the 10 patients with AL who underwent TX but had no additional chemotherapy was 50%, 50%, and 20% at 1, 2, and 5 years, respectively; amyloid recurred in the grafts of these patients after a median of 11 months, and extra-cardiac amyloid deposition contributed to mortality in 70% of these patients. Survival of 7 patients with AL who also had chemotherapy was 71%, 71%, and 36% respectively and 2 patients remain alive. Survival of the 7 patients with non-AL was 86%, 86%, and 64% at 1, 2, and 5 years, respectively; 5 patients remain alive. One patient from this group had recurrence of amyloid in the graft at 60 months. Five-year survival for all 24 amyloid patients was 38%, compared to patients undergoing TX in the UK for other indications (n = 4,058) for whom it was 67% (p = 0.013). Conclusion: Regardless of the use of adjunctive chemotherapy, the 5-year survival after TX for cardiac AL amyloidosis was less than that after TX for other indications, and progression of the systemic disease contributed substantially to the increased mortality. In contrast, the 5-year survival after TX for non-AL amyloid, combined as necessary with liver or kidney TX, was similar to that after TX in general. J Heart Lung Transplant 2004;23:1142-53. Amyloidosis is the generic term for a group of disorders characterized by extracellular deposition of protein in a characteristic fibrilar form. In systemic AL amyloidosis, where most cases are associated with a plasma cell dyscrasia, 1,2 some patients benefit from chemotherapy directed towards the underlying monoclonal gammopa- thy. This approach is rarely effective in patients with severe cardiac involvement whose median survival is about 6 months. 3,4 Hereditary systemic amyloidosis is caused by deposition of amyloid fibrils derived from a range of genetically variant proteins. 5,6 The first report of successful heart transplantation for cardiac amyloidosis appeared in 1988. 7 Later publica- tions revealed that patients had received heart trans- plants for end-stage cardiac amyloid as early as 1984. 8 Aside from individual case reports, only two series have described the experience at individual transplant cen- ters. 9,10 More than 10 years have elapsed since the only multi-center survey reported the longer term outcome following heart transplantation. 11 Among centers per- forming heart transplantation, its application in AL amyloidosis remains contentious. 12 We report here the complete United Kingdom (UK) experience of heart transplantation for amyloid heart disease from its inception in 1984. PATIENTS AND METHODS All patients undergoing heart transplantation for amy- loidosis in the UK between 1982 and 2002 were identified from their explant pathology report by inter- rogating the pathology databases of all UK transplant centers. Their clinical and pathology records were reviewed with the agreement of the participating pa- thologists and clinicians. Documentation was cross checked with the National Transplant Database (UK Transplant) and with information held at the National Amyloidosis Center. Patients with AL amyloid were considered separately from those with non-AL types. From the a The Imperial College School of Medicine, National Heart and Lung Institute, London, the b Transplant Unit and Department of Pathology, Royal Brompton and Harefield Hospitals NHS Trust, Mid- dlesex, and c Department of Medicine, The National Amyloidosis Centre, The Royal Free Hospital, London, United Kingdom. Submitted June 5, 2003; accepted August 22, 2003. Reprint requests: NR Banner, FRCP, Harefield Hospital, Royal Brompton and Harefield NHS Trust, Harefield, Middlesex UB9 6JH, United Kingdom. E-mail: n.banner@rbh.nthames.nhs.uk Copyright © 2004 by the International Society for Heart and Lung Transplantation. 1053-2498/04/$–see front matter. doi:10.1016/ j.healun.2003.08.027 1142 CLINICAL HEART TRANSPLANTATION