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Blood, 1 February 2006, Vol. 107, No. 3, pp. 1227-1229.
Prepublished online as a Blood First Edition Paper on October 6, 2005; DOI 10.1182/blood-2005-08-3253.


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TRANSPLANTATION
Brief report

Sequential heart and autologous stem cell transplantation for systemic AL amyloidosis

Julian D. Gillmore, Hugh J. Goodman, Helen J. Lachmann, Mark Offer, Ashutosh D. Wechalekar, Jayshree Joshi, Mark B. Pepys, and Philip N. Hawkins

From the National Amyloidosis Centre, Centre for Amyloidosis and Acute Phase Proteins, Department of Medicine (Hampstead Campus), Royal Free and University College Medical School, London, United Kingdom.

Extensive cardiac amyloid deposition in systemic AL amyloidosis is associated with a grave prognosis. Heart transplantation is rarely performed because of the systemic and progressive nature of the disease. Patients with severe cardiac amyloid infiltration are ineligible for the preferred treatment of melphalan chemotherapy with stem cell transplantation (SCT) rescue because of the high risk for treatment-related mortality. Heart transplantation followed by SCT was performed in 5 patients with AL amyloidosis and predominant cardiomyopathy. Patients were followed up for a median of 95 months (range, 37-118 months) from diagnosis. At censor, 3 of 5 patients were well without evidence of intracardiac or extracardiac amyloid accumulation, and median overall survival by Kaplan-Meier estimate was not reached. Two patients died of progressive amyloidosis 33 and 90 months after heart transplantation after relapse of their underlying plasma cell dyscrasia. Heart transplantation followed by SCT is feasible in selected patients with cardiac AL amyloidosis and may confer substantial survival benefit.


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