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An overview of the use of high-dose melphalan with autologous stem cell transplantation for the treatment of AL amyloidosis

Bone Marrow Transplantation volume 28, pages 637–642 (2001)Cite this article

Abstract

Primary or AL amyloidosis results from a plasma cell dyscrasia in which fibrillar light chain protein deposition leads to organ failure and death. Standard treatment for AL amyloidosis has been oral melphalan and prednisone. However, this form of treatment modifies the natural history of this lethal disease only marginally, extending median survival from 13 months following diagnosis to 17 months. At Boston University Medical Center, we have developed treatment protocols using high-dose intravenous melphalan with autologous peripheral blood stem cell transplantation (HDM/SCT) to treat AL amyloidosis, and we have treated over 200 patients with HDM/SCT during the past six years. This extensive experience has shown that patients with AL amyloidosis, despite multisystem involvement and compromised organ function can tolerate this aggressive form of treatment. Furthermore, HDM/SCT results in durable hematologic responses in a substantial proportion of patients, and such responses are associated with clinical improvement, decreased amyloid-related organ dysfunction, and prolonged survival. However, toxicity from treatment is high (overall peri-transplant mortality, 14%), particularly for those patients with clinically significant cardiac involvement. For this reason, we believe a multidisciplinary management approach is essential when using HDM/SCT for treatment of AL amyloidosis. Based on our experience, we believe that HDM/SCT is the treatment of choice for patients with AL amyloidosis who have a good performance status and limited cardiac involvement at the time of diagnosis. HDM/SCT offers the best chance for hematologic remission, prolongation of survival, and reversal of amyloid-related disease. At the same time, we believe that HDM/SCT should continue to be examined in the context of clinical trials, directed at developing approaches to broaden the applicability of this therapy by minimizing toxicity and to increase the likelihood of complete hematologic responses. Bone Marrow Transplantation (2001) 28, 637–642.

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Acknowledgements

We gratefully acknowledge the numerous current and former colleagues in the Stem Cell Transplant Program, Amyloid Research and Treatment Program, Clinical Trials Office, and Clinical Laboratories at Boston Medical Center who have assisted with the multidisciplinary evaluation and treatment of patients with AL amyloidosis. This work was supported in part by grants from the Food and Drug Administration FD-R-001346 and from the Gerry Foundation. Dr Seldin is a scholar of the Leukemia and Lymphoma Society of America.

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Authors and Affiliations

  1. Stem Cell Transplant Program of the Section of Hematology and Oncology, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    V Sanchorawala, DG Wright, DC Seldin, K Finn & K Quillen

  2. Amyloid Research and Treatment Program, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    V Sanchorawala, DG Wright, DC Seldin, LM Dember, K Finn, RH Falk, J Berk, K Quillen & M Skinner

  3. Amyloid Research and Treatment Program, Section of Renal Medicine, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    LM Dember

  4. Amyloid Research and Treatment Program, Section of Cardiology, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    RH Falk

  5. Amyloid Research and Treatment Program, Section of Pulmonology, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    J Berk

  6. Section of Rheumatology in the Department of Medicine, Amyloid Research and Treatment Program, Boston University School of Medicine, Boston University Medical Center, Boston, MA, USA

    M Skinner

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  1. V Sanchorawala
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Sanchorawala, V., Wright, D., Seldin, D. et al. An overview of the use of high-dose melphalan with autologous stem cell transplantation for the treatment of AL amyloidosis. Bone Marrow Transplant 28, 637–642 (2001). https://doi.org/10.1038/sj.bmt.1703200

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