Organ Transplantation in Hereditary Apolipoprotein AI Amyloidosis

Abstract

Patients with hereditary apolipoprotein AI (apoAI) amyloidosis often have extensive visceral amyloid deposits, and many develop end-stage renal failure as young adults. Solid organ transplantation to replace failing organ function in systemic amyloidosis is controversial due to the multisystem and progressive nature of the disease and the risk of recurrence of amyloid in the graft. We report the outcome of solid organ transplantation, including dual transplants in 4 cases, among 10 patients with apoAI amyloidosis who were followed for a median (range) of 16 (4-28) and 9 (0.2-27) years from diagnosis of amyloidosis and transplantation, respectively. Eight of 10 patients were alive, seven with a functioning graft at censor. Two patients died, one of disseminated cytomegalovirus infection 2 months after renal transplantation and the other of multisystem failure following severe trauma more than 13 years after renal transplantation. The renal transplant of one patient failed due to recurrence of amyloid after 25 years. Amyloid disease progression was very slow and the natural history of the condition was favorably altered in both cases in which the liver was transplanted. Failing organs in hereditary apoAI amyloidosis should be replaced since graft survival is excellent and confers substantial survival benefit.

Diagnosis, Pathogenesis, Treatment, and Prognosis of Hereditary Fibrinogen Aα-Chain Amyloidosis

“…The natural history of the renal decline in AFib was relatively slow compared with that in untreated systemic AL amyloidosis in which median time from diagnosis to ESRD is 7.5 to 14 mo, 23 but was substantially faster than in hereditary apoAI amyloidosis in which it is typically approximately 8 yr. 24 Despite the absence of therapy to diminish production of the amyloidogenic fibrinogen variant in most patients reported here, median patient survival from clinical presentation was more than 15 yr, contrasting markedly that of less than 2 yr and approximately 5 yr among untreated 25 and treated 26,27 patients with systemic AL amyloidosis respectively. Estimated median survival from commencement of dialysis with censoring at transplantation was 9.3 yr, substantially longer than the approximately 5-yr median survival among all nondiabetic U.K. patients aged 55 to 64 yr who commenced dialysis between 1997 and 2001 (Figure 5B).…”

Recurrence of Secondary Glomerular Disease after Renal Transplantation

“…However, in view of the increased risk of postoperative complications, a preoperative cardiovascular evaluation is mandatory even in asymptomatic patients. Specific treatment should be considered for particular forms of amyloidosis, that is, chemotherapy and autologous stem cell transplantation followed by kidney transplantation in progressive primary AL amyloidosis (55), a dual liver and kidney transplantation in hereditary amyloidosis and multivisceral involvement (42,43,56), a dual heart and kidney transplantation in severe and irreversible cardiac and renal involvement (57). Patients with familial Mediterranean fever should be treated regularly with colchicine (58,59).…”

Hereditary lysozyme amyloidosis – phenotypic heterogeneity and the role of solid organ transplantation

“…Interestingly, liver function tests were barely deranged and remained relatively stable during follow-up, which contrasts AL amyloidosis, in which patients with similar SAP scintigraphic appearances have markedly deranged liver biochemistry and may develop hepatic decompensation [19]. Both this and the finding that median time from presentation with renal dysfunction to ESRD in this cohort was 11.0 years indicate that the natural history of ALys is markedly slower than AL and other types of hereditary amyloidosis [15,20]. The mechanism by which amyloid accumulation in native organs appears to abate in ALys once patients have accumulated a large total body amyloid burden remains unclear.…”