title: Stem cell transplantation compared with melphalan plus dexamethasone in the treatment of immunoglobulin light-chain amyloidosis.
abstract: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival.
  
  
  
  Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis.
  
  
  
  Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P < .01).
  
  
  
  Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone. Cancer 2016;122:2197-205. © 2016 American Cancer Society.
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Francis K
authors.initials: FK
authors.lastname: Buadi
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.|Division of Nephrology and Hypertension, Mayo Clinic, Rochester, Minnesota.
authors.forename: Nelson
authors.initials: N
authors.lastname: Leung
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: John
authors.initials: J
authors.lastname: Lust
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.|Department of Molecular Medicine, Mayo Clinic, Rochester, Minnesota.
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Russell
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, Minnesota.
authors.forename: Vera J
authors.initials: VJ
authors.lastname: Suman
authors.affiliation: Division of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, Minnesota.
authors.forename: Jennifer G
authors.initials: JG
authors.lastname: Le-Rademacher
authors.affiliation: Division of Hematology, Mayo Clinic, Rochester, Minnesota.
authors.forename: William J
authors.initials: WJ
authors.lastname: Hogan
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000175:diagnosis / Q000378:metabolism* / Q000401:mortality / Q000628:therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001706:Biopsy; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage; D005260:Female; D019650:Hematopoietic Stem Cell Mobilization; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D012720:Severity of Illness Index; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains; D003907:Dexamethasone; D008558:Melphalan
keywords: dexamethasone; immunoglobulin light chains; light-chain amyloidosis; melphalan; stem cell transplantation