title: 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.
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: S
authors: S
authors: Girnius
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: D C
authors: DC
authors: Seldin
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Cardiovascular Medicine, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: H K
authors: HK
authors: Meier-Ewert
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: J M
authors: JM
authors: Sloan
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA [3] Department of Pathology and Laboratory Medicine, Boston Medical Center, Boston, MA, USA.
authors: K
authors: K
authors: Quillen
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Cardiovascular Medicine, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: F L
authors: FL
authors: Ruberg
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Pulmonary, Critical Care, and Allergy, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: J L
authors: JL
authors: Berk
authors: Department of Biostatistics, Boston University School of Public Health, Boston, MA, USA.
authors: G
authors: G
authors: Doros
authors: 1] Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA [2] Section of Hematology-Oncology, Department of Medicine, Boston Medical Center, Boston, MA, USA.
authors: V
authors: V
authors: Sanchorawala
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000150:complications / Q000503:physiopathology* / Q000628:therapy*; D015415:Biomarkers / Q000378:metabolism; D005260:Female; D005500:Follow-Up Studies; D006331:Heart Diseases / Q000150:complications / Q000628:therapy*; D006412:Hematopoietic Stem Cells / Q000166:cytology; D006801:Humans; D000075363:Immunoglobulin Light-chain Amyloidosis; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000627:therapeutic use; D008875:Middle Aged; D020097:Natriuretic Peptide, Brain / Q000378:metabolism; D011379:Prognosis; D016016:Proportional Hazards Models; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D013997:Time Factors; D016896:Treatment Outcome; D019210:Troponin I / Q000378:metabolism
chemical_list: D015415:Biomarkers; D019210:Troponin I; D020097:Natriuretic Peptide, Brain; D008558:Melphalan