title: Induction bortezomib in Al amyloidosis followed by high dose melphalan and autologous stem cell transplantation: a single institution retrospective study.
abstract: BACKGROUND
  High-dose melphalan (HDM) followed by autologous stem cell transplant (ASCT) for light chain amyloidosis (AL) was performed in 31 patients at Oregon Health and Science University between 2005 and 2012. Fifteen patients had cardiac involvement.
  
  
  METHODS
  Patients received melphalan 200 mg/m(2) or dose-adjusted HDM (100-140 mg/m(2)) depending on high risk features. Thirteen patients proceeded directly to ASCT after diagnosis, 12 patients received a bortezomib-containing regimen, and 6 received a variety of other induction regimens.
  
  
  RESULTS
  The day 100 treatment-related mortality was 9.6%. Overall hematologic (ORR) and organ response rates (OR) in the whole cohort after ASCT were 77% and 58%. ORR and OR in the bortezomib pretreated group were 92% and 75% vs. 69% and 54% in the group that received no pretreatment. The median time to maximum hematologic response after ASCT was reduced in the group that received bortezomib induction (3 vs. 14 months). Overall cardiac response rate was 60%; 100% in patients pretreated with bortezomib and 43% in those without induction treatment. With a median follow-up of 2.9 years, the 3-year progression-free and overall survival rates in the entire cohort were 66% and 73% and in those with cardiac involvement, 73% and 80%.
  
  
  CONCLUSIONS
  We observed that bortezomib-based induction is well tolerated in patients with and without cardiac involvement and suggest that this approach be studied in prospective multi-institutional trials.
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR. Electronic address: scottem@ohsu.edu.
authors.forename: Emma C
authors.initials: EC
authors.lastname: Scott
authors.affiliation: Oregon Health and Science University Knight Cardiovascular Institute, Portland, OR.
authors.forename: Stephen B
authors.initials: SB
authors.lastname: Heitner
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: William
authors.initials: W
authors.lastname: Dibb
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Gabrielle
authors.initials: G
authors.lastname: Meyers
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Stephen D
authors.initials: SD
authors.lastname: Smith
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Farnoush
authors.initials: F
authors.lastname: Abar
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Tibor
authors.initials: T
authors.lastname: Kovacsovics
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Galit
authors.initials: G
authors.lastname: Perez-Avraham
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Alex
authors.initials: A
authors.lastname: Stentz
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Rachel
authors.initials: R
authors.lastname: Frires
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: James
authors.initials: J
authors.lastname: Dibb
authors.affiliation: Center for Hematologic Malignancies, Oregon Health and Science University Knight Cancer Institute, Portland, OR.
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
mesh_terms: D000328:Adult; D000368:Aged; D058227:Amyloidogenic Proteins / Q000032:analysis; D000686:Amyloidosis / Q000150:complications / Q000188:drug therapy* / Q000628:therapy; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D001897:Boronic Acids / Q000008:administration & dosage / Q000627:therapeutic use*; D000069286:Bortezomib; D009202:Cardiomyopathies / Q000209:etiology; D003520:Cyclophosphamide / Q000008:administration & dosage; D003907:Dexamethasone / Q000008:administration & dosage; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000032:analysis; D053208:Kaplan-Meier Estimate; D007674:Kidney Diseases / Q000209:etiology; D000077269:Lenalidomide; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000627:therapeutic use*; D061988:Proteasome Inhibitors / Q000008:administration & dosage / Q000627:therapeutic use*; D011719:Pyrazines / Q000008:administration & dosage / Q000627:therapeutic use*; D012074:Remission Induction; D012189:Retrospective Studies; D013792:Thalidomide / Q000008:administration & dosage / Q000031:analogs & derivatives; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D058227:Amyloidogenic Proteins; D001897:Boronic Acids; D007147:Immunoglobulin Light Chains; D019653:Myeloablative Agonists; D061988:Proteasome Inhibitors; D011719:Pyrazines; D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; D003520:Cyclophosphamide; D000077269:Lenalidomide; D008558:Melphalan
keywords: Amyloid; Bortezomib induction; Stem cell transplantation