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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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Submitted August 11, 2005
Accepted September 22, 2005
Sequential cardiac 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
National Amyloidosis Centre, Centre for Amyloidosis & Acute Phase Proteins, Department of Medicine (Hampstead Campus), Royal Free & University College Medical School, London, United Kingdom
* Corresponding author; email: j.gillmore{at}medsch.ucl.ac.uk.
Extensive cardiac amyloid deposition in systemic AL amyloidosis is associated with a grave prognosis. Cardiac transplantation is rarely performed due to the systemic and progressive nature of the disease. Patients with severe cardiac amyloid are ineligible for the preferred treatment of melphalan chemotherapy with stem cell rescue (SCT) due to the high risk of treatment-related mortality. Cardiac transplantation followed by SCT was performed in 5 patients with AL amyloidosis and predominant cardiomyopathy. Patients were followed for a median (range) of 95 (37-118) months from diagnosis. At censor, three of 5 patients were well without evidence of intra or extra-cardiac amyloid accumulation, and median overall survival by Kaplan-Meier estimate was not reached. Two patients died of progressive amyloid 33 and 90 months after cardiac transplantation following relapse of their underlying plasma cell dyscrasia. Cardiac transplantation followed by SCT is feasible in selected patients with cardiac AL amyloidosis and may confer substantial survival benefit.
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