title: Allogeneic and syngeneic hematopoietic cell transplantation in patients with amyloid light-chain amyloidosis: a report from the European Group for Blood and Marrow Transplantation.
abstract: Using the European Group for Blood and Marrow Transplantation (EBMT) registry, we retrospectively studied 19 patients with AL (amyloid light-chain) amyloidosis who underwent allogeneic (allo; n = 15) or syngeneic (syn; n = 4) hematopoietic stem cell transplantation (SCT) between 1991 and 2003. For allo-SCT, full-intensity conditioning was used in 7 patients and reduced-intensity conditioning (RIC) in 8 patients. Engraftment was durable in 12 of those 15 patients. The median follow-up time is 19 months. Kaplan-Meier probabilities of overall and progression-free survival were 60% and 53% at 1 year, respectively. Overall, 40% of patients died of transplant-related mortality (TRM). Best hematologic response after SCT was complete remission (CR) and partial remission (PR) in 8 and 2 patients, respectively, leading to an organ response in 8 of these patients. Seven of the 10 patients in remission are long-term survivors. In 5 of 7 evaluable patients in CR, chronic graft-versus-host disease (GvHD) was observed, indicating the contribution of immune effects to disease control. The main clinical problem was cardiac failure in patients with poor performance status due to amyloidosis or in combination with severe infections. These data suggest that allo-SCT might be a promising and potentially curative treatment modality for selected patients with AL amyloidosis.
authors.affiliation: University of Heidelberg, Dept of Hematology/Oncology and Rheumatology, Im Neuenheimer Feld 410, 69120 Heidelberg, Germany. stefan_schoenland@med.uni-heidelberg.de
authors.forename: Stefan O
authors.initials: SO
authors.lastname: Schönland
authors.forename: Henk
authors.initials: H
authors.lastname: Lokhorst
authors.forename: Agnes
authors.initials: A
authors.lastname: Buzyn
authors.forename: Veronique
authors.initials: V
authors.lastname: Leblond
authors.forename: Ute
authors.initials: U
authors.lastname: Hegenbart
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Bandini
authors.forename: Andrew
authors.initials: A
authors.lastname: Campbell
authors.forename: Enric
authors.initials: E
authors.lastname: Carreras
authors.forename: Augustin
authors.initials: A
authors.lastname: Ferrant
authors.forename: Leanthe
authors.initials: L
authors.lastname: Grommisch
authors.forename: Peter
authors.initials: P
authors.lastname: Jacobs
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
authors.forename: Nigel
authors.initials: N
authors.lastname: Russell
authors.forename: Pierre
authors.initials: P
authors.lastname: Zachee
authors.forename: Hartmut
authors.initials: H
authors.lastname: Goldschmidt
authors.forename: Simona
authors.initials: S
authors.lastname: Iacobelli
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Gösta
authors.initials: G
authors.lastname: Gahrton
mesh_terms: D000328:Adult; D000686:Amyloidosis / Q000150:complications / Q000628:therapy*; D005334:Fever / Q000209:etiology; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006331:Heart Diseases / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006801:Humans; D008875:Middle Aged; D012074:Remission Induction; D012189:Retrospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D014185:Transplantation, Isogeneic