Low-dose continuous oral melphalan for the treatment of primary systemic (AL) amyloidosis

Authors

  • Vaishali Sanchorawala,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • Daniel G. Wright,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • David C. Seldin,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • Rodney H. Falk,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • John L. Berk,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • Laura M. Dember,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • Kathleen T. Finn,

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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  • Martha Skinner

    1. Department of Medicine of Boston Medical Center and the Amyloid Treatment and Research Program, Boston University School of Medicine, Boston, MA, USA
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Vaishali Sanchorawala, MD, Boston Medical Center, Section of Hematology/Oncology, 88 East Newton Street, Boston, MA 02118, USA. E-mail: Vaishali.Sanchorawala@bmc.org

Abstract

Summary.  Median survival of patients with AL amyloidosis with clinically significant cardiac involvement is 5 months when treated with cyclic melphalan and prednisone. We investigated a regimen of continuous oral melphalan as a single agent for patients with cardiac amyloidosis who were unable to tolerate prednisone or more aggressive chemotherapy. Thirty patients with amyloid cardiomyopathy were treated with continuous oral melphalan. Seven of 13 patients, evaluable after 3–4 months of treatment, achieved a partial haematological response and three achieved a complete haematological response; six patients have survived for > 1 year. This regimen appeared to be effective in inducing haematological responses in patients who received total doses of melphalan > 300 mg.

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