Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement.

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.
AuthorsS Girnius, D C Seldin, H K Meier-Ewert, J M Sloan, K Quillen, F L Ruberg, J L Berk, G Doros, V Sanchorawala
JournalBone marrow transplantation (Bone Marrow Transplant) Vol. 49 Issue 3 Pg. 434-9 (Mar 2014) ISSN: 1476-5365 [Electronic] England
PMID24317129 (Publication Type: Clinical Trial, Journal Article)
Chemical References
  • Biomarkers
  • Troponin I
  • Natriuretic Peptide, Brain
  • Melphalan
  • Aged
  • Amyloidosis (complications, physiopathology, therapy)
  • Biomarkers (metabolism)
  • Female
  • Follow-Up Studies
  • Heart Diseases (complications, therapy)
  • Hematopoietic Stem Cells (cytology)
  • Humans
  • 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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