title: Predictors of survival in patients with systemic light-chain amyloidosis and cardiac involvement initially ineligible for stem cell transplantation and treated with oral melphalan and dexamethasone.
abstract: The treatment of systemic light-chain (AL) amyloidosis with symptomatic cardiac involvement at diagnosis remains a challenge. We report the results of 40 consecutive newly diagnosed AL cardiac patients who were not candidates for stem cell transplant and therefore received monthly oral melphalan and dexamethasone. Median survival was 10.5 months and baseline predictors of survival included gender, troponin I and interventricular septal thickness. The most significant predictor of survival was response to therapy. The haematological response rate was 58% (23/40) with 13% (5/40) complete responses; most responses were noted in <3 cycles. Achievement of a rapid response to therapy extends survival.
authors.affiliation: Department of Medicine, Weill-Cornell School of Medicine, New York Hospital, NY, USA.
authors.forename: D
authors.initials: D
authors.lastname: Lebovic
authors.forename: J
authors.initials: J
authors.lastname: Hoffman
authors.forename: B M
authors.initials: BM
authors.lastname: Levine
authors.forename: H
authors.initials: H
authors.lastname: Hassoun
authors.forename: H
authors.initials: H
authors.lastname: Landau
authors.forename: Y
authors.initials: Y
authors.lastname: Goldsmith
authors.forename: M S
authors.initials: MS
authors.lastname: Maurer
authors.forename: R M
authors.initials: RM
authors.lastname: Steingart
authors.forename: A D
authors.initials: AD
authors.lastname: Cohen
authors.forename: R L
authors.initials: RL
authors.lastname: Comenzo
mesh_terms: D000284:Administration, Oral; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy*; D009202:Cardiomyopathies / Q000188:drug therapy*; D000075202:Contraindications; D003907:Dexamethasone / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000032:analysis*; D008297:Male; D008558:Melphalan / Q000627:therapeutic use*; D008875:Middle Aged; D011379:Prognosis; D012737:Sex Factors; D033581:Stem Cell Transplantation; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D007147:Immunoglobulin Light Chains; D003907:Dexamethasone; D008558:Melphalan