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Sequential heart and autologous stem cell transplantation for systemic AL amyloidosis
Julian D Gillmore1, Hugh J Goodman, Helen J Lachmann
1National Amyloidosis Centre, Department of Medicine, Royal Free and University College Medical School, London, United Kingdom. j.gillmore@medsch.ucl.ac.uk
Insights
Heart transplantation followed by stem cell transplantation (SCT) is a feasible treatment for selected systemic AL amyloidosis patients with cardiac involvement. This approach may offer significant survival benefits, with some patients remaining well long-term.
Area of Science:
- Cardiology
- Hematology
- Transplantation Medicine
Background:
- Systemic AL amyloidosis with extensive cardiac amyloid deposition carries a poor prognosis.
- Heart transplantation is seldom performed due to disease progression.
- Severe cardiac amyloidosis often precludes melphalan chemotherapy with stem cell transplantation (SCT) rescue due to high mortality risks.
Purpose of the Study:
- To evaluate the feasibility and survival benefit of heart transplantation followed by SCT in patients with AL amyloidosis and predominant cardiomyopathy.
Main Methods:
- Five patients with AL amyloidosis and significant cardiac involvement underwent heart transplantation followed by SCT.
- Patients were monitored for long-term outcomes, including survival and evidence of amyloid recurrence.
Main Results:
- Three out of five patients were alive and well at follow-up (median 95 months), with no signs of amyloid accumulation.
- Two patients succumbed to progressive amyloidosis months after transplantation, linked to plasma cell dyscrasia relapse.
- Median overall survival was not reached, suggesting potential long-term benefit.
Conclusions:
- Heart transplantation followed by SCT is a viable option for carefully selected cardiac AL amyloidosis patients.
- This combined strategy may lead to substantial improvements in overall survival for this challenging patient group.
Abstract:
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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