title: Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death.
abstract: In light of major advances in immunoglobulin light chain (AL) amyloidosis, we evaluated the trends in presentation, management, and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000 to 2014. As compared with the 2 intervals 2000-2004 and 2005-2009, patients diagnosed in 2010-2014 were less likely to have >2 involved organs. Utilization of autologous stem cell transplant (ASCT) was similar across all periods, about one-third of patients, but there was an increase in the use of pre-ASCT bortezomib induction and of unattenuated melphalan conditioning in 2010-2014 compared with earlier periods. Non-ASCT first-line regimen changed with 65% of patients in 2010-2014 received bortezomib-based therapy, 79% of patients in 2005-2009 received melphalan-dexamethasone, and 64% of patients in 2000-2004 received melphalan-prednisone. The rate of better than very good partial response (VGPR) was higher in more recent periods (66% vs 58% vs 51%;P= .001), a change largely driven by improved VGPR rates in the non-ASCT population. Overall survival (OS) has improved, with inflection points for improvement differing for the ASCT and non-ASCT groups. In the ASCT population, the greatest gains were after 2010 (4-year OS, 91% compared with 73% and 65%). In the non-ASCT group, greatest gains were after 2005 (4-year OS, 38%, 32%, and 16%). Fewer patients died within 6 months of diagnosis in the 2 later periods (24% vs 25% vs 37%;P< .001). Overall, outcomes among patients with AL amyloidosis have improved with earlier diagnosis, higher rates of VGPR, lower early mortality, and improved OS.
authors.affiliation: Division of Hematology.
authors.forename: Eli
authors.identifier: 0000-0003-2210-2174
authors.initials: E
authors.lastname: Muchtar
authors.affiliation: Division of Hematology.
authors.forename: Morie A
authors.identifier: 0000-0002-3853-5196
authors.initials: MA
authors.lastname: Gertz
authors.affiliation: Division of Hematology.
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.affiliation: Division of Hematology.
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.affiliation: Division of Hematology.
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.affiliation: Division of Hematology.
authors.forename: Francis K
authors.initials: FK
authors.lastname: Buadi
authors.affiliation: Department of Cardiovascular Diseases, and.
authors.forename: Martha
authors.initials: M
authors.lastname: Grogan
authors.affiliation: Division of Hematology.
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.affiliation: Division of Hematology.
authors.forename: Prashant
authors.initials: P
authors.lastname: Kapoor
authors.affiliation: Division of Hematology.|Division of Nephrology and Hypertension, Mayo Clinic, Rochester, MN.
authors.forename: Nelson
authors.initials: N
authors.lastname: Leung
authors.affiliation: Division of Hematology.
authors.forename: Amie
authors.initials: A
authors.lastname: Fonder
authors.affiliation: Division of Hematology.
authors.forename: Miriam
authors.initials: M
authors.lastname: Hobbs
authors.affiliation: Division of Hematology.
authors.forename: Yi Lisa
authors.initials: YL
authors.lastname: Hwa
authors.affiliation: Division of Hematology.
authors.forename: Wilson
authors.initials: W
authors.lastname: Gonsalves
authors.affiliation: Division of Hematology.
authors.forename: Rahma
authors.initials: R
authors.lastname: Warsame
authors.affiliation: Division of Hematology.
authors.forename: Taxiarchis V
authors.initials: TV
authors.lastname: Kourelis
authors.affiliation: Division of Hematology.
authors.forename: Stephen
authors.initials: S
authors.lastname: Russell
authors.affiliation: Division of Hematology.
authors.forename: John A
authors.initials: JA
authors.lastname: Lust
authors.affiliation: Division of Hematology.
authors.forename: Yi
authors.initials: Y
authors.lastname: Lin
authors.affiliation: Division of Hematology.
authors.forename: Ronald S
authors.initials: RS
authors.lastname: Go
authors.affiliation: Division of Hematology.
authors.forename: Steven
authors.initials: S
authors.lastname: Zeldenrust
authors.affiliation: Division of Hematology.
authors.forename: Robert A
authors.initials: RA
authors.lastname: Kyle
authors.affiliation: Division of Hematology.
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.affiliation: Division of Hematology.
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000401:mortality / Q000628:therapy; D064592:Autografts; D000069286:Bortezomib / Q000008:administration & dosage*; D003907:Dexamethasone / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains; D060828:Induction Chemotherapy / Q000379:methods; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D015996:Survival Rate; D019172:Transplantation Conditioning
chemical_list: D007147:Immunoglobulin Light Chains; D000069286:Bortezomib; D003907:Dexamethasone; D008558:Melphalan