HOMEPRODUCTSSERVICESCOMPANYCONTACTFAQResearchDictionaryPharmaMobileSign Up FREE or Login

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

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.
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
Topics
  • 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)

Join CureHunter, for free Research Interface BASIC access!

Take advantage of free CureHunter research engine access to explore the best drug and treatment options for any disease. Find out why thousands of doctors, pharma researchers and patient activists around the world use CureHunter every day.
Realize the full power of the drug-disease research network!


Choose Username:
Email:
Password:
Verify Password: