title: Induction therapy with bortezomib and dexamethasone followed by autologous stem cell transplantation versus autologous stem cell transplantation alone in the treatment of renal AL amyloidosis: a randomized controlled trial.
abstract: BACKGROUND
  Although the use of bortezomib alone and in combination with steroids has shown efficacy in AL amyloidosis, its role in combination with high-dose melphalan and autologous stem cell transplantation (HDM/SCT) is unknown. In this study, we evaluated bortezomib in combination with dexamethasone (BD) for induction chemotherapy prior to HDM/SCT.
  
  
  METHODS
  This was a single-center, prospective, randomized controlled trial comparing induction therapy consisting of two BD cycles followed by HDM/SCT (BD + HDM/SCT) with HDM/SCT alone in the treatment of patients with newly diagnosed AL amyloidosis. The hematological and organ responses of the patients were assessed every three months post HDM/SCT.
  
  
  RESULTS
  Fifty-six patients newly diagnosed with renal (100%), cardiac (57.1%), liver (7.1%), or nervous system (8.9%) AL amyloidosis were enrolled in this study; 28 patients were assigned to each arm. Two patients died within 100 days of HDM/SCT (3.6% treatment-related mortality). The overall hematologic response rates in the BD + HDM/SCT arm and HDM/SCT arm at three, six and twelve months were 78.5% versus 50%, 82.1% versus 53.5% and 85.7% versus 53.5%, respectively. In the BD + HDM/SCT arm, 15 (53.5%) patients achieved a hematologic response after BD and before HDM/SCT. An intention-to-treat analysis revealed a higher rate of complete remission in the BD + HDM/SCT arm at both 12 and 24 months (67.9% and 70%, respectively) than with the HDM/SCT-only therapy (35.7% and 35%, respectively, P = 0.03). After a median follow-up of 28 months, the survival rates at 24 months post-treatment start were 95.0% in the BD + HDM/SCT group and 69.4% in the HDM/SCT alone group (P = 0.03).
  
  
  CONCLUSIONS
  Our preliminary data suggest that the outcome of treating AL amyloidosis with BD induction and HDM/SCT was superior to the outcome of the HDM/SCT treatment alone.
  
  
  BACKGROUND
  This trial has been registered at clinicaltrials.gov with the number NCT01998503.
authors.forename: Xianghua
authors.initials: X
authors.lastname: Huang
authors.forename: Qingwen
authors.initials: Q
authors.lastname: Wang
authors.forename: Wencui
authors.initials: W
authors.lastname: Chen
authors.forename: Caihong
authors.initials: C
authors.lastname: Zeng
authors.forename: Zhaohong
authors.initials: Z
authors.lastname: Chen
authors.forename: Dehua
authors.initials: D
authors.lastname: Gong
authors.forename: Haitao
authors.initials: H
authors.lastname: Zhang
authors.affiliation: Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing 210002, China. liuzhihong@nju.edu.cn.
authors.forename: Zhihong
authors.initials: Z
authors.lastname: Liu
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D001897:Boronic Acids / Q000008:administration & dosage*; D000069286:Bortezomib; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage*; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D007674:Kidney Diseases / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011719:Pyrazines / Q000008:administration & dosage*; D015996:Survival Rate / Q000639:trends; D014182:Transplantation, Autologous
chemical_list: D001897:Boronic Acids; D011719:Pyrazines; D000069286:Bortezomib; D003907:Dexamethasone