title: Autologous stem cell transplantation for AL amyloidosis: adjustment of melphalan dose by factors including BNP.
abstract: High-dose melphalan with autologous stem cell transplantation (HDM/ASCT) is a promising treatment option for eligible patients with systemic immunoglobulin light chain (AL) amyloidosis. We present the results of ASCT following risk-adapted melphalan conditioning on the basis of criteria proposed by our group, including B-type natriuretic peptide (BNP). Ten patients with primary systemic AL amyloidosis treated at our institute were evaluated. A full dose of melphalan (200 mg/m(2)) was administered to patients who met all the following: performance status, 0 or 1; number of organs involved, 2 or less; serum creatinine, 1.5 mg/dL or less; EF 50 % or more and BNP 200 pg/mL or less; otherwise 140 mg/m(2). The hematologic complete response was achieved in four and organ response was seen in two patients. The median event-free survival (EFS) of all patients was 21.5 months, and median overall survival (OS) was 47.0 months. EFS and OS were significantly longer for patients who received 200 mg/m(2) of melphalan than for those who received lower dose (EFS: not reached vs. 13.9 months, P = 0.0217; OS: not reached vs. 13.8 months, P = 0.0186). No treatment-related mortality within 100 days from ASCT was observed. Evaluation of cardiac diastolic function may contribute to safer HDM/ASCT and improve outcome of AL amyloidosis.
authors.affiliation: Department of Gastroenterology, Rheumatology, and Clinical Immunology, Sapporo Medical University School of Medicine, South 1, West 16, Chuou-ku, Sapporo, Hokkaido, Japan, haya@sapmed.ac.jp.
authors.forename: Toshiaki
authors.initials: T
authors.lastname: Hayashi
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Ikeda
authors.forename: Tetsuyuki
authors.initials: T
authors.lastname: Igarashi
authors.forename: Yumiko
authors.initials: Y
authors.lastname: Maruyama
authors.forename: Yuka
authors.initials: Y
authors.lastname: Aoki
authors.forename: Masanori
authors.initials: M
authors.lastname: Nojima
authors.forename: Tadao
authors.initials: T
authors.lastname: Ishida
authors.forename: Yasuhisa
authors.initials: Y
authors.lastname: Shinomura
mesh_terms: D000328:Adult; D000686:Amyloidosis / Q000175:diagnosis / Q000401:mortality / Q000503:physiopathology / Q000628:therapy*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007147:Immunoglobulin Light Chains; D000075363:Immunoglobulin Light-chain Amyloidosis; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use; D008875:Middle Aged; D020097:Natriuretic Peptide, Brain; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains; D020097:Natriuretic Peptide, Brain; D008558:Melphalan