title: Reduced intensity allogeneic stem cell transplantation for systemic primary amyloidosis refractory to high-dose melphalan.
abstract: Complete elimination of the plasma cell dyscrasia is a rational therapeutic goal, as intercepting supply of precursor protein is a necessary condition for a major regression of amyloid deposits. High-dose melphalan with autologous stem cell transplantation has shown the ability to induce complete hematological response (HR) along with recovery of organ dysfunction. However, the rate of HR with this treatment rarely exceeds 40%. We describe here the first known case of successful reduced intensity allogeneic stem cell transplantation (RIST) for a patient with primary amyloidosis complicated with nephrotic syndrome but without cardiac disease, who had obtained only partial HR by high-dose melphalan with autologous stem cell transplantation. RIST may be feasible and be capable of achieving complete HR along with recovery from nephrotic syndrome with acceptable toxicity.
authors.affiliation: First Department of Internal Medicine, Faculty of Medical Sciences, University of Fukui, Matsuoka, Japan. ykawai@fmsrsa.fukui-med.ac.jp
authors.forename: Yasukazu
authors.initials: Y
authors.lastname: Kawai
authors.forename: Keiichi
authors.initials: K
authors.lastname: Kinoshita
authors.forename: Hajime
authors.initials: H
authors.lastname: Arai
authors.forename: Atsushi
authors.initials: A
authors.lastname: Kuwata
authors.forename: Yoshitomo
authors.initials: Y
authors.lastname: Fukuoka
authors.forename: Masanobu
authors.initials: M
authors.lastname: Yamaoka
authors.forename: Shin
authors.initials: S
authors.lastname: Imamura
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Tsutani
authors.forename: Takanori
authors.initials: T
authors.lastname: Ueda
mesh_terms: D000686:Amyloidosis / Q000150:complications / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008558:Melphalan / Q000627:therapeutic use*; D008875:Middle Aged; D009404:Nephrotic Syndrome / Q000209:etiology / Q000628:therapy; D010265:Paraproteinemias / Q000150:complications / Q000628:therapy*; D011507:Proteinuria / Q000209:etiology / Q000628:therapy; D012074:Remission Induction / Q000379:methods; D016879:Salvage Therapy / Q000379:methods; D014184:Transplantation, Homologous; D017211:Treatment Failure
chemical_list: D008558:Melphalan