Modified high-dose melphalan and autologous stem cell transplantation for immunoglobulin light chain amyloidosis Journal Article


Authors: Nguyen, V. P.; Landau, H.; Quillen, K.; Brauneis, D.; Shelton, A. C.; Mendelson, L.; Rahman, H.; Sloan, J. M.; Sarosiek, S.; Sanchorawala, V.
Article Title: Modified high-dose melphalan and autologous stem cell transplantation for immunoglobulin light chain amyloidosis
Abstract: High-dose melphalan and autologous stem cell transplantation (HDM/SCT) have been used in patients with immunoglobulin light chain (AL) amyloidosis for over 2 decades now with durable responses, prolonged survival, and decreasing treatment-related mortality. Historically, patients with poorer baseline functional status, advanced age, renal compromise, and cardiac involvement have been treated with a risk-adapted modified conditioning dose of melphalan (mHDM) of 100 to 140 mg/m2 before SCT. In part because of these baseline characteristics, patients receiving mHDM/SCT have had poorer outcomes compared with patients receiving full-dose melphalan at 200 mg/m2. With the advent of novel therapeutic agents such as proteasome inhibitors, immunomodulatory agents, and monoclonal antibodies for the treatment of AL amyloidosis, it is imperative to understand the long-term effects of mHDM/SCT. Here we report the long-term outcomes of 334 patients with AL amyloidosis treated with mHDM/SCT. Median overall survival was 6.1 years and median event-free survival 4.3 years, with median overall survival reaching 13.4 years for patients who had achieved a hematologic complete response (CR). Overall hematologic response rate was 69%, and treatment-related mortality was 3% after 2010. Thus, mHDM/SCT leads to prolonged survival and favorable outcomes, especially if a hematologic CR is achieved. © 2018 The American Society for Blood and Marrow Transplantation
Keywords: overall survival; stem cell transplantation; hematologic response; al amyloidosis; melphalan dose
Journal Title: Biology of Blood and Marrow Transplantation
Volume: 24
Issue: 9
ISSN: 1083-8791
Publisher: Elsevier Inc.  
Date Published: 2018-09-01
Start Page: 1823
End Page: 1827
Language: English
DOI: 10.1016/j.bbmt.2018.06.018
PROVIDER: scopus
PUBMED: 29933072
PMCID: PMC7553205
DOI/URL:
Notes: Article -- Export Date: 1 November 2018 -- Source: Scopus
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MSK Authors
  1. Heather Jolie Landau
    363 Landau
  2. Hafsa Rahman
    3 Rahman