title: Combined use of bortezomib, cyclophosphamide, and dexamethasone induces favorable hematological and organ responses in Japanese patients with amyloid light-chain amyloidosis: a single-institution retrospective study.
abstract: Amyloid light-chain amyloidosis (ALA) is a rare disease with poor prognosis and is often associated with monoclonal gammopathy of undetermined significance, multiple myeloma, or Waldenström macroglobulinemia. Only high-dose melphalan with auto-peripheral blood stem cell transplantation (PBSCT) has shown high long-term hematological response rates, but combinations with novel agents, including bortezomib or lenalidomide, have recently shown high hematological response rates for AL amyloidosis patients. In the present study, we treated eight Japanese patients with AL amyloidosis using bortezomib, cyclophosphamide, and dexamethasone (CyBorD). Overall response rate was 100 %; four patients (50 %) had complete remissions (CR), two (25 %) had very good partial responses, and two (25 %) had partial responses. Five of six patients (83 %) had organ responses in the heart and/or kidney. A relapsed patient repeatedly achieved CR with the CyBorD treatment. One patient died of sudden cardiac arrest a month after normalization of his serum free light chain level, which may be attributable to his spending the previous 6 months undergoing PBSCT collection and high-dose melphalan with auto-PBSCT. Altogether, the CyBorD regimen achieved high levels of hematological responses relatively quickly (within 2-3 months). The CyBorD regimen, rather than high-dose melphalan treatment, could serve as a first-line therapy for Japanese patients with ALA.
authors.affiliation: Department of Hematology, Rheumatology & Infectious Diseases, Kumamoto University of Medicine, 1-1-1 Honjo, Kumamoto, 860-8556, Japan.
authors.forename: Yoshitaka
authors.initials: Y
authors.lastname: Kikukawa
authors.forename: Hiromichi
authors.initials: H
authors.lastname: Yuki
authors.forename: Sinya
authors.initials: S
authors.lastname: Hirata
authors.forename: Kazuhiko
authors.initials: K
authors.lastname: Ide
authors.forename: Hirotomo
authors.initials: H
authors.lastname: Nakata
authors.forename: Toshikazu
authors.initials: T
authors.lastname: Miyakawa
authors.forename: Naofumi
authors.initials: N
authors.lastname: Matsuno
authors.forename: Kisato
authors.initials: K
authors.lastname: Nosaka
authors.forename: Yuji
authors.initials: Y
authors.lastname: Yonemura
authors.forename: Tatsuya
authors.initials: T
authors.lastname: Kawaguchi
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Hata
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Mitsuya
authors.forename: Yutaka
authors.initials: Y
authors.lastname: Okuno
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism / Q000401:mortality / Q000628:therapy; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001897:Boronic Acids / Q000008:administration & dosage; D000069286:Bortezomib; D003520:Cyclophosphamide / Q000008:administration & dosage; D003907:Dexamethasone / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains; D000075363:Immunoglobulin Light-chain Amyloidosis; D007564:Japan; D008297:Male; D008875:Middle Aged; D008998:Monoclonal Gammopathy of Undetermined Significance / Q000188:drug therapy; D009101:Multiple Myeloma / Q000188:drug therapy; D011719:Pyrazines / Q000008:administration & dosage; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D001897:Boronic Acids; D007147:Immunoglobulin Light Chains; D011719:Pyrazines; D000069286:Bortezomib; D003907:Dexamethasone; D003520:Cyclophosphamide