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Long-term outcome of high-dose melphalan and autologous stem cell transplantation for AL amyloidosis

Bone Marrow Transplantation volume 37, pages 937–943 (2006)Cite this article

Abstract

Light chain (AL) amyloidosis is the result of a clonal plasma cell expansion, in which amyloidogenic monoclonal light chains deposit in various tissues resulting in organ dysfunction and organ failure. The median survival of patients with AL amyloidosis without therapy is 10–14 months. Several phase II studies report haematological and clinical remission in up to 50% of patients after high-dose melphalan and autologous stem cell transplantation. We analysed retrospectively the long-term outcome of 19 patients treated in this way between August/1996 and December/2001. We observed a relatively high treatment-related mortality of 26%, but 12 patients (63%) were high-risk candidates. Eight patients (42%) surviving longer than 100 days achieved haematological remission and long-term survival, whereas 6 (32%) obtained no clear benefit from high-dose therapy. However, 62% of patients survived beyond 2 years and the median survival from transplant was 48 months (range 0–104 months).

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Acknowledgements

We especially thank our colleagues from the Department of Cardiology and the Department of Nephrology at the Hammersmith Hospital for excellent interdisciplinary co-operation.

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Authors and Affiliations

  1. Department of Haematology, Hammersmith Hospital NHS Trust, Imperial College School of Medicine, London, UK

    J B Perz, A Rahemtulla, C Giles, R M Szydlo, J Davis & J F Apperley

  2. National Amyloidosis Centre, Royal Free University College Medical School, London, UK

    D Gopaul, J Gillmore & P N Hawkins

  3. National Hospital for Neurology and Neurosurgery, University College, Queen Square, London, UK

    C J Mathias

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  1. J B Perz
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  2. A Rahemtulla
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  7. J Gillmore
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  9. P N Hawkins
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  10. J F Apperley
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Correspondence to J B Perz.

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Perz, J., Rahemtulla, A., Giles, C. et al. Long-term outcome of high-dose melphalan and autologous stem cell transplantation for AL amyloidosis. Bone Marrow Transplant 37, 937–943 (2006). https://doi.org/10.1038/sj.bmt.1705354

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