title: Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial.
abstract: The depth of hematologic response has been shown to correlate with survival and organ responses for patients with light chain (AL) amyloidosis. We conducted a prospective trial of 2 cycles of induction with bortezomib and dexamethasone on a twice a week schedule followed by conditioning with bortezomib and high-dose melphalan (HDM) and autologous stem cell transplantation (SCT). The objectives were hematologic responses, tolerability, and survival. Thirty-five patients were enrolled from 2010 to 2013. Of these, 30 proceeded with SCT, whereas 5 did not because of clinical deterioration during induction (n = 3) or complications after stem cell collection (n = 2). Two patients developed features of an autologous graft-versus-host disease-like syndrome post-SCT, which responded to steroids; no other unusual complications were seen. Treatment-related mortality occurred in 8.5% (3/35). Hematologic responses were achieved by 100% of the 27 assessable patients (63% complete response, 37% very good partial response [VGPR]) who completed the planned treatment. By intention-to-treat, hematologic responses occurred in 77% of patients (49% complete response, 29% VGPR). With a median follow-up of 36 months, the median overall survival and progression-free survival were not reached. In conclusion, incorporating bortezomib into induction and conditioning yielded a high rate of hematologic responses after HDM/SCT in patients with AL amyloidosis.
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts. Electronic address: vaishali.sanchorawala@bmc.org.
authors: Vaishali
authors: V
authors: Sanchorawala
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: Dina
authors: D
authors: Brauneis
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: Anthony C
authors: AC
authors: Shelton
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: Stephen
authors: S
authors: Lo
authors: Department of Biostatistics, Boston University School of Public Health, Boston, Massachusetts.
authors: Fangui
authors: F
authors: Sun
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: J Mark
authors: JM
authors: Sloan
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: Karen
authors: K
authors: Quillen
authors: Amyloidosis Center, Boston University School of Medicine and Boston Medical Center, Boston, Massachusetts.
authors: David C
authors: DC
authors: Seldin
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy*; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D000069286:Bortezomib / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D000075363:Immunoglobulin Light-chain Amyloidosis; D060828:Induction Chemotherapy / Q000379:methods*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D011446:Prospective Studies; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods*
chemical_list: D000970:Antineoplastic Agents; D000069286:Bortezomib; D008558:Melphalan
keywords: AL amyloidosis; Bortezomib; Induction; Melphalan; Stem cell transplantation