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Published on: January 21, 2018
High-dose therapy with auto-SCT is feasible in high-risk cardiac amyloidosis
P Kongtim1, M H Qazilbash1, J J Shah2
1Department of Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Insights
High-dose therapy and auto-SCT (HDT-ASCT) is a viable treatment for light-chain amyloidosis with cardiac involvement. This approach demonstrated manageable treatment-related mortality and improved long-term survival outcomes.
Area of Science:
- Hematology
- Cardiology
- Oncology
Background:
- Cardiac involvement in light-chain amyloidosis (AL) significantly worsens prognosis.
- It is associated with increased treatment-related mortality (TRM) and morbidity during high-dose therapy and auto-SCT (HDT-ASCT).
Purpose of the Study:
- To evaluate the outcomes of patients with cardiac amyloidosis undergoing HDT-ASCT.
- To identify factors influencing survival in this high-risk patient group.
Main Methods:
- Retrospective analysis of 30 patients with cardiac amyloidosis who underwent HDT-ASCT.
- Melphalan-based conditioning regimens (140-200 mg/m²) were utilized.
- Median follow-up was 35 months.
Main Results:
- One-year TRM was 10%.
- Three-year overall survival (OS) and event-free survival (EFS) were 83% and 56.8%, respectively.
- Cumulative incidence of relapse at 3 years was 38.5%.
Conclusions:
- HDT-ASCT is well-tolerated in patients with high-risk cardiac amyloidosis.
- This therapy can lead to improved overall survival outcomes.
- Age >60, lack of novel induction therapy, and BM plasmacytosis >10% were negative survival factors.
Abstract:
Cardiac involvement in light-chain amyloidosis (AL) predicts poor prognosis and is associated with higher TRM and morbidity during high-dose therapy and auto-SCT (HDT-ASCT). We studied the outcomes of 30 patients with cardiac amyloidosis undergoing HDT-ASCT at our center between January 1998 and March 2012. The median age of the patients was 53 years (range, 36-74) with a median follow-up of 35 months (range, 0.4-97 months). Twenty-seven patients (90%) had more than one organ involved besides the heart with 37% with cardiac stage ⩾3. Melphalan-based conditioning regimen (140-200 mg/m(2)) was used for HDT-ASCT. One-year TRM is 10%. Three-year OS and EFS from HDT-ASCT was 83% and 56.8%, respectively. Cumulative incidence of relapse at 3 years was 38.5%. Negative factors affecting survival included age >60 years, lack of novel induction therapy and BM plasmacytosis >10%. We conclude that HDT-ASCT is well tolerated in patients with high-risk cardiac amyloidosis and can lead to improved overall outcomes.
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