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High-dose melphalan and stem cell transplantation in AL amyloidosis with elevated cardiac biomarkers

Bone Marrow Transplantation volume 53, pages 1593–1595 (2018)Cite this article

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Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease caused by misfolded light chains. These misfolded light chains form soluble toxic aggregates that deposit as amyloid fibrils in tissues and organs, leading to significant and permanent organ dysfunction. Although nearly any organ or combination of organs can be affected by AL amyloidosis, ~ 70% of individuals diagnosed with AL amyloidosis experience cardiac involvement [1]. Cardiac involvement is a major determinant of survival in patients with AL amyloidosis. Untreated patients with cardiac amyloidosis have historically had a median survival of 6–12 months [2], though recent advances in treatments directed toward the plasma cell dyscrasia have led to tremendous improvements in overall survival (OS) [3]. The development of novel therapeutic options for patients with cardiac amyloidosis has highlighted the need for accurate prognostication to help with risk stratification.

High-dose melphalan and stem cell transplantation (HDM/SCT) can induce hematologic responses and improve survival in carefully and highly selected patients with AL amyloidosis. However, patients with advanced cardiac involvement are at a higher risk of morbidity and mortality with this aggressive treatment and therefore may not be candidates for HDM/SCT [4].

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Authors and Affiliations

  1. Section of Hematology and Oncology, Stem Cell Transplant Program, Boston Medical Center, Boston, USA

    Paul S. White, Dina Brauneis, J. Mark Sloan, Karen Quillen, Shayna Sarosiek & Vaishali Sanchorawala

  2. Amyloidosis Center, Boston University School of Medicine, Boston, MA, USA

    Pooja Phull, J. Mark Sloan, Shayna Sarosiek & Vaishali Sanchorawala

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  1. Paul S. White
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Correspondence to Vaishali Sanchorawala.

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White, P.S., Phull, P., Brauneis, D. et al. High-dose melphalan and stem cell transplantation in AL amyloidosis with elevated cardiac biomarkers. Bone Marrow Transplant 53, 1593–1595 (2018). https://doi.org/10.1038/s41409-018-0242-1

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