title: Early intensification and autologous stem cell transplantation in patients with systemic AL amyloidosis: a single-centre experience.
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.
authors.affiliation: Department of Haematology, University Hospital CHUV, Lausanne, Switzerland.
authors.forename: Valérie
authors.initials: V
authors.lastname: Frossard
authors.forename: Nicolas
authors.initials: N
authors.lastname: Ketterer
authors.forename: Anne
authors.initials: A
authors.lastname: Rosselet
authors.forename: Pascal
authors.initials: P
authors.lastname: Meier
authors.forename: Anne
authors.initials: A
authors.lastname: Cairoli
authors.forename: Michel A
authors.initials: MA
authors.lastname: Duchosal
authors.forename: Tibor
authors.initials: T
authors.lastname: Kovacsovics
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000150:complications / Q000401:mortality / Q000628:therapy*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006801:Humans; D007674:Kidney Diseases / Q000628:therapy; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage; D012074:Remission Induction; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D019653:Myeloablative Agonists; D008558:Melphalan