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

Bone Marrow Transplantation volume 49, pages 434–439 (2014)Cite this article

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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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Acknowledgements

We gratefully acknowledge our colleagues in the Clinical Trials Office, specifically Anthony Shelton, RN, Dina Brauneis, NP, and Kathleen T Finn, NP, and the staff of the Solomont Center for Cancer and Blood Disorders at Boston Medical Center who assisted with the multi-disciplinary evaluation and treatment of the patients.

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Authors and Affiliations

  1. Department of Medicine, Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA

    S Girnius, D C Seldin, H K Meier-Ewert, J M Sloan, K Quillen, F L Ruberg, J L Berk & V Sanchorawala

  2. Department of Medicine, Section of Hematology-Oncology, Boston Medical Center, Boston, MA, USA

    S Girnius, D C Seldin, J M Sloan, K Quillen & V Sanchorawala

  3. Department of Medicine, Section of Cardiovascular Medicine, Boston Medical Center, Boston, MA, USA

    H K Meier-Ewert & F L Ruberg

  4. Department of Pathology and Laboratory Medicine, Boston Medical Center, Boston, MA, USA

    K Quillen

  5. Department of Medicine, Section of Pulmonary, Critical Care, and Allergy, Boston Medical Center, Boston, MA, USA

    J L Berk

  6. Department of Biostatistics, Boston University School of Public Health, Boston, MA, USA

    G Doros

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  1. S Girnius
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  9. V Sanchorawala
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Correspondence to S Girnius.

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Girnius, S., Seldin, D., Meier-Ewert, H. et al. Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement. Bone Marrow Transplant 49, 434–439 (2014). https://doi.org/10.1038/bmt.2013.192

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