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Stem cell transplantation

Organ responses after highdose melphalan and stemcell transplantation in AL amyloidosis

Leukemia volume 35, pages 916–919 (2021)Cite this article

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AL amyloidosis is the most common systemic amyloidoses. In this disorder, clonal plasma cells or B cells produce monoclonal immunoglobulin free light chains that misfold, aggregate, and accumulate in tissues leading to organ failure [1]. The degree of organ dysfunction, especially in regards to cardiac involvement, remains a crucial determinant of overall survival (OS). Treatment of AL amyloidosis aims to eradicate the clonal cells to prevent production of the amyloidogenic light chains thereby allowing improvement in organ function [2]. Although several therapeutic options exist, highdose melphalan and autologous stemcell transplantation (HDM/SCT) is associated with durable hematologic responses and prolonged OS for highly selected and fit patients [3,4,5,6]. Organ responses following HDM/SCT occur more frequently in those achieving hematologic responses. Previous studies from our center show that organ responses were achieved by 78% of the patients who achieved a hematologic complete response (CR) following HDM/SCT compared with 52% of patients who did not achieve a CR [7,8,9,10,11]. However, hematologic and organ response criteria have been modified and updated since these initial reports [12,13,14,15].

Hence, we sought to evaluate patients with AL amyloidosis treated with upfront HDM/SCT to determine the impact of hematologic response at 6 months on organ responses using the contemporary validated criteria.

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Fig. 1: Impact of organ and hematologic response on overall survival after highdose melphalan and stemcell transplantation in patients with AL amyloidosis.
Fig. 2: Organ response according to hematologic response at 6 months in patients with AL amyloidosis treated with HDM/SCT as frontline therapy.

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Acknowledgements

The authors acknowledge their colleagues in the Amyloidosis Center, Cancer Clinical Trials Office, and Center for Cancer and Blood Disorders at Boston Medical Center, who assisted with the multidisciplinary evaluation and treatment of the patients with AL amyloidosis.

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Authors and Affiliations

  1. Amyloidosis Center, Boston University School of Medicine, Boston, MA, 02118, USA

    Raphael Szalat, Shayna Sarosiek, Andrea Havasi, Dina Brauneis, J. Mark Sloan & Vaishali Sanchorawala

  2. Stem Cell Transplantation Program in the Section of Hematology and Oncology, Boston, MA, 02118, USA

    Raphael Szalat, Shayna Sarosiek, Dina Brauneis, J. Mark Sloan & Vaishali Sanchorawala

  3. Section of Renal Medicine, Boston Medical Center, Boston, MA, 02118, USA

    Andrea Havasi

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  1. Raphael Szalat
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Contributions

RS and VS designed research, performed research, analyzed data, and wrote the paper. RS performed statistical analysis. SS performed research, analyzed data, and critically reviewed the paper. JMS and DB critically reviewed the paper.

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Correspondence to Vaishali Sanchorawala.

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Szalat, R., Sarosiek, S., Havasi, A. et al. Organ responses after highdose melphalan and stemcell transplantation in AL amyloidosis. Leukemia 35, 916–919 (2021). https://doi.org/10.1038/s41375-020-1006-7

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