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

Ann Hematol. 2009 Jul;88(7):681-5. doi: 10.1007/s00277-008-0652-z. Epub 2008 Dec 6.

Abstract

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 100 days. 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 12 months. Half of all the patients (n = 8) were alive after a median follow-up of 33 months, 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.

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Aged
  • Amyloidosis / complications
  • Amyloidosis / mortality
  • Amyloidosis / therapy*
  • Female
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Humans
  • Kidney Diseases / therapy
  • Male
  • Melphalan / administration & dosage
  • Middle Aged
  • Myeloablative Agonists / administration & dosage
  • Remission Induction
  • Survival Rate
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Myeloablative Agonists
  • Melphalan