title: High-dose melphalan and autologous stem cell transplantation for systemic light-chain amyloidosis: a single institution retrospective analysis of 40 cases.
abstract: We report our retrospective analysis of 40 patients who received high-dose melphalan and autologous stem cell transplantation for systemic immunoglobulin light-chain (AL) amyloidosis. Between 2006 and 2013, 40 patients with AL amyloidosis were transplanted at our medical center. Their median age was 54 years (range 32-70 years): 18 were male. The dominant organs involved were the heart in 13 patients, and kidney in 22: and other organs were involved in five. The median melphalan dose administered was 129 (range 50-200) mg/m(2), and the median infused CD34(+) cells was 2.69 (range 1.17-11.26) × 10(6)/kg. Of the 40 patients, 30 are alive after a median follow-up of 42 (range 12-94) months, and the 4-year estimated overall survival rate was 74% (95% CI 56-86%). Four patients died ≤ 100 days post-ASCT (heart failure in three patients, bacteremia in one). The 4-year estimated survival of the patients with cardiac involvement was 54%, significantly lower than that of the other patients (91%). Hematological and organ responses were 52 and 50%, respectively. Careful patient selection and experienced management are important, especially for patients with cardiac involvement. It is also important to develop additional treatment for patients who do not achieve a hematological and/or organ response.
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan. tsukada_nobuhiro@med.jrc.or.jp.
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Tsukada
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Masahiro
authors.initials: M
authors.lastname: Ikeda
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Sumito
authors.initials: S
authors.lastname: Shingaki
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Kanji
authors.initials: K
authors.lastname: Miyazaki
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Sohsuke
authors.initials: S
authors.lastname: Meshitsuka
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Yumiko
authors.initials: Y
authors.lastname: Yoshiki
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Yu
authors.initials: Y
authors.lastname: Abe
authors.affiliation: Department of Hematology, Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-ku, Tokyo, 150-8935, Japan.
authors.forename: Kenshi
authors.initials: K
authors.lastname: Suzuki
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000401:mortality / Q000628:therapy*; D009202:Cardiomyopathies / Q000401:mortality / Q000628:therapy*; D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007147:Immunoglobulin Light Chains; D007674:Kidney Diseases / Q000401:mortality / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains; D008558:Melphalan
keywords: AL amyloidosis; ASCT; BNP; Melphalan