Abstract |
In Ig light chain ( AL) amyloidosis, cardiac involvement is associated with worse prognosis and increased treatment-related complications. In this retrospective cohort study, we assessed survival, hematologic and cardiac responses to high-dose melphalan and auto-SCT (HDM/SCT) in patients with AL amyloidosis and cardiac involvement, stratified by cardiac biomarkers brain natriuretic peptide and Troponin I, analogous to the Mayo cardiac staging. Forty-seven patients underwent HDM/SCT based upon functional measures; six patients had modified cardiac stage I disease, seventeen had modified cardiac stage II disease and twenty-four had modified cardiac stage III disease. Treatment-related mortality was 4% for all patients and 8% for patients with stage III disease. Three-year survival was 88% and EFS was 47%; these did not differ by stage. By intention-to-treat analysis, 27% of patients achieved a hematologic complete response and 32% a very good partial response, of whom 70 and 45%, respectively, have not required additional therapy at 36 months. Cardiac response was achieved in 53% of patients. We conclude that with appropriate patient selection and a risk-adapted treatment approach, HDM/SCT is safe and effective in patients with AL amyloidosis and cardiac involvement.
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Authors | S Girnius, D C Seldin, H K Meier-Ewert, J M Sloan, K Quillen, F L Ruberg, J L Berk, G Doros, V Sanchorawala |
Journal | Bone marrow transplantation
(Bone Marrow Transplant)
Vol. 49
Issue 3
Pg. 434-9
(Mar 2014)
ISSN: 1476-5365 [Electronic] England |
PMID | 24317129
(Publication Type: Clinical Trial, Journal Article)
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Chemical References |
- Biomarkers
- Troponin I
- Natriuretic Peptide, Brain
- Melphalan
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Topics |
- Aged
- Amyloidosis
(complications, physiopathology, therapy)
- Biomarkers
(metabolism)
- Female
- Follow-Up Studies
- Heart Diseases
(complications, therapy)
- Hematopoietic Stem Cells
(cytology)
- Humans
- Immunoglobulin Light-chain Amyloidosis
- Kaplan-Meier Estimate
- Male
- Melphalan
(administration & dosage, therapeutic use)
- Middle Aged
- Natriuretic Peptide, Brain
(metabolism)
- Prognosis
- Proportional Hazards Models
- Retrospective Studies
- Stem Cell Transplantation
- Time Factors
- Treatment Outcome
- Troponin I
(metabolism)
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