title: Oral cyclic melphalan and dexamethasone for patients with AL amyloidosis.
abstract: OBJECTIVE
  Aggressive treatment of amyloid light chain (AL) amyloidosis with high-dose intravenous melphalan followed by autologous stem cell transplantation (HDM/SCT) is effective in inducing hematologic remission and clinical improvement. However, only selected patients with AL amyloidosis are eligible for HDM/SCT because of amyloid-associated organ dysfunction.
  
  
  METHODS
  We report on 70 patients with AL amyloidosis treated with oral cyclic melphalan and dexamethasone.
  
  
  RESULTS
  Of 48 evaluable patients who survived and returned for follow-up assessment, 6 patients (13%) achieved a complete hematologic response and 12 patients (25%) a partial hematologic response. Responses were non-inferior for patients receiving weekly "low-dose" dexamethasone compared with those receiving 4 day pulses. Median survival for the 70 patients has not yet been reached with a median follow-up of 17 months. Nineteen patients (27%) received additional treatment leading to improvement in survival.
  
  
  CONCLUSIONS
  Melphalan/dexamethasone can lead to hematologic responses and improvement in survival, particularly for those who can receive additional treatment for AL amyloidosis.
authors.affiliation: Section of Hematology-Oncology, Department of Medicine, Boston University School of Medicine, 820 Harrison Avenue, Boston, MA 02118, USA. vaishali.sanchorawala@bmc.org
authors.forename: Vaishali
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: David C
authors.initials: DC
authors.lastname: Seldin
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.forename: J Mark
authors.initials: JM
authors.lastname: Sloan
authors.forename: Gheorghe
authors.initials: G
authors.lastname: Doros
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000682:Amyloid / Q000378:metabolism; D000686:Amyloidosis / Q000188:drug therapy* / Q000378:metabolism; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage / Q000627:therapeutic use; D003907:Dexamethasone / Q000008:administration & dosage / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage / Q000627:therapeutic use; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000627:therapeutic use*; D008875:Middle Aged; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D000682:Amyloid; D018906:Antineoplastic Agents, Alkylating; D005938:Glucocorticoids; C035222:amyloid protein AL; D003907:Dexamethasone; D008558:Melphalan