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Early intensification and autologous stem cell transplantation in patients with systemic AL amyloidosis: a single-centre experience

Frossard, Valérie ; Ketterer, Nicolas ; Rosselet, Anne ; Meier, Pascal ; Cairoli, Anne ; Duchosal, Michel ; Kovacsovics, Tibor

In: Annals of Hematology, 2009, vol. 88, no. 7, p. 681-685

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    Titre
    • Early intensification and autologous stem cell transplantation in patients with systemic AL amyloidosis: a single-centre experience
    Auteur
    • Frossard, Valérie. Department of Haematology, University Hospital CHUV, Lausanne, Switzerland
    • Ketterer, Nicolas. Department of Oncology, University Hospital CHUV, Lausanne, Switzerland
    • Rosselet, Anne. Department of Haematology, University Hospital CHUV, Lausanne, Switzerland
    • Meier, Pascal. Department of Nephrology, University Hospital CHUV, Lausanne, Switzerland
    • Cairoli, Anne. Department of Haematology, University Hospital CHUV, Lausanne, Switzerland
    • Duchosal, Michel. Department of Haematology, University Hospital CHUV, Lausanne, Switzerland
    • Kovacsovics, Tibor. Department of Haematology, University Hospital CHUV, Lausanne, Switzerland
    Type de document
    • Postprint
    Identifiant OAI-PMH
    • oai:doc.rero.ch:315341
    Summary
    Primary systemic amyloidosis (AL amyloidosis) continues to have a very poor prognosis. Most therapeutic strategies remain unsatisfactory. Conventional chemotherapy is known to offer at best only moderate efficacy. Several studies have yielded higher complete response rates after high-dose chemotherapy and autologous stem cell transplantation (ASCT) in addition to improving outcomes in a subgroup of patients. However, the superiority of an intensive approach in AL amyloidosis has not been confirmed in a randomised trial. The precise role of ASCT remains unclear. We report our experience in 16 patients diagnosed with AL amyloidosis and treated in a multidisciplinary approach with high-dose melphalan and ASCT. Median age was 59 (39-71)years. The kidneys were predominantly affected in 75% of cases; two or more organs were affected in 38%. Median time from diagnosis to transplantation was 2 (1-4)months. Three patients (19%) developed acute renal failure and required transient dialysis. Transplant-related mortality was 6% after 100days. Haematological complete response (CR) was obtained in nine (56%) and organ response in six (38%) patients. Nine out of 12 patients (75%) with kidney involvement exhibited a sustained clinical benefit at 12months. Half of all the patients (n = 8) were alive after a median follow-up of 33months, including two in continuous CR. This suggests that high-dose chemotherapy and ASCT are still valid treatment options in AL amyloidosis and that a significant number of patients with renal involvement might benefit from this approach