BACKGROUND: High-dose melphalan and autologous stem cell transplantation (HDM) is an effective treatment for systemic amyloid light chain (AL) amyloidosis but the eligibility criteria exclude many patients with this disorder. The aim of this study was to determine appropriate treatment strategies for systemic AL amyloidosis according to each patient's clinical condition in Japan. METHODS: Historical cohort study. Fifty-three patients with systemic AL amyloidosis (those with malignancies were excluded) were treated in our hospital with HDM (15 patients), melphalan + prednisolone (MP) (17 patients), vincristine + adriamycin + dexamethasone (VAD) (11 patients), or supportive treatment (no chemotherapy, 10 patients). We compared the survival rates among these treatment groups. RESULTS: Mean survival was significantly longer in the HDM group than in the other three groups (P < 0.01, log-rank test). This trend remained the same when patients were divided into those with and without cardiac amyloid involvement. Furthermore, in patients with heart involvement, survival in the VAD therapy group was significantly inferior to that in the MP therapy group (P < 0.01 by log-rank test). Significant factors related to the survival rate included the presence or absence of heart involvement and treatment modality. CONCLUSIONS: HDM should be considered the treatment of choice in eligible patients with systemic AL amyloidosis even in the presence of cardiac amyloidosis. If HDM is not eligible, indications for VAD therapy should be carefully evaluated in patients with cardiac amyloidosis.
BACKGROUND: High-dose melphalan and autologous stem cell transplantation (HDM) is an effective treatment for systemic amyloid light chain (AL) amyloidosis but the eligibility criteria exclude many patients with this disorder. The aim of this study was to determine appropriate treatment strategies for systemic AL amyloidosis according to each patient's clinical condition in Japan. METHODS: Historical cohort study. Fifty-three patients with systemic AL amyloidosis (those with malignancies were excluded) were treated in our hospital with HDM (15 patients), melphalan + prednisolone (MP) (17 patients), vincristine + adriamycin + dexamethasone (VAD) (11 patients), or supportive treatment (no chemotherapy, 10 patients). We compared the survival rates among these treatment groups. RESULTS: Mean survival was significantly longer in the HDM group than in the other three groups (P < 0.01, log-rank test). This trend remained the same when patients were divided into those with and without cardiac amyloid involvement. Furthermore, in patients with heart involvement, survival in the VAD therapy group was significantly inferior to that in the MP therapy group (P < 0.01 by log-rank test). Significant factors related to the survival rate included the presence or absence of heart involvement and treatment modality. CONCLUSIONS: HDM should be considered the treatment of choice in eligible patients with systemic AL amyloidosis even in the presence of cardiac amyloidosis. If HDM is not eligible, indications for VAD therapy should be carefully evaluated in patients with cardiac amyloidosis.
Authors: Angela Dispenzieri; Robert A Kyle; Martha Q Lacy; Terry M Therneau; Dirk R Larson; Matthew F Plevak; S Vincent Rajkumar; Rafael Fonseca; Philip R Greipp; Thomas E Witzig; John A Lust; Steven R Zeldenrust; Denise S Snow; Susan R Hayman; Mark R Litzow; Dennis A Gastineau; Ayalew Tefferi; David J Inwards; Ivana N Micallef; Stephen M Ansell; Luis F Porrata; Michelle A Elliott; Morie A Gertz Journal: Blood Date: 2004-01-22 Impact factor: 22.113
Authors: Martha Skinner; Vaishali Sanchorawala; David C Seldin; Laura M Dember; Rodney H Falk; John L Berk; Jennifer J Anderson; Carl O'Hara; Kathleen T Finn; Caryn A Libbey; Janice Wiesman; Karen Quillen; Niall Swan; Daniel G Wright Journal: Ann Intern Med Date: 2004-01-20 Impact factor: 25.391
Authors: Arnaud Jaccard; Philippe Moreau; Veronique Leblond; Xavier Leleu; Lotfi Benboubker; Olivier Hermine; Christian Recher; Bouchra Asli; Bruno Lioure; Bruno Royer; Fabrice Jardin; Frank Bridoux; Bernard Grosbois; Jérome Jaubert; Jean-Charles Piette; Pierre Ronco; Fabrice Quet; Michel Cogne; Jean-Paul Fermand Journal: N Engl J Med Date: 2007-09-13 Impact factor: 91.245