BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS: Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasone patient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P < .01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone. Cancer 2016;122:2197-205.
BACKGROUND: Autologous stem cell transplantation (SCT) is a common management strategy for select patients with immunoglobulin light-chain amyloidosis, but no trials have documented improved overall survival. METHODS: Eighty-nine patients with biopsy-proven immunoglobulin light-chain amyloidosis were allowed to select treatment with melphalan plus dexamethasone (n = 34) or SCT (n = 55); all patients were transplant eligible. Treatment preference resulted in imbalanced study arms. Patients who selected SCT were younger, more frequently had an Eastern Cooperative Oncology Group performance status score less than 2, had lower-stage amyloidosis, and had a lower incidence of cardiac amyloidosis. RESULTS:Patients receiving melphalan plus dexamethasone had a 3-year progression-free survival rate of 29.1% and an overall survival rate of 58.8%. Patients undergoing SCT had a 3-year progression-free survival rate of 51.7% and an overall survival rate of 83.6%. An attempt to match patients between the 2 arms in terms of risk produced 24 matched triplet sets (2 SCT patients for each melphalan-dexamethasonepatient); there was no difference in hematologic response, but there was better survival after autologous SCT. A propensity score-matched analysis of the cohorts (melphalan plus dexamethasone vs SCT) showed an overall mortality hazard ratio of 2.56 (P < .01). CONCLUSIONS: Although the study had limitations, similar hematologic responses and improved survival were observed after SCT versus melphalan plus dexamethasone. Cancer 2016;122:2197-205.
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: Vaishali Sanchorawala; Fangui Sun; Karen Quillen; J Mark Sloan; John L Berk; David C Seldin Journal: Blood Date: 2015-10-06 Impact factor: 22.113
Authors: Ashutosh D Wechalekar; Stefan O Schonland; Efstathios Kastritis; Julian D Gillmore; Meletios A Dimopoulos; Thirusha Lane; Andrea Foli; Darren Foard; Paolo Milani; Lisa Rannigan; Ute Hegenbart; Philip N Hawkins; Giampaolo Merlini; Giovanni Palladini Journal: Blood Date: 2013-03-11 Impact factor: 22.113
Authors: Christopher P Venner; Julian D Gillmore; Sajitha Sachchithanantham; Shameem Mahmood; Thirusha Lane; Darren Foard; Murielle Roussel; Lisa Rannigan; Simon Dj Gibbs; Jennifer H Pinney; Carol J Whelan; Helen J Lachmann; Philip N Hawkins; Ashutosh D Wechalekar Journal: Haematologica Date: 2014-09-05 Impact factor: 9.941
Authors: G Palladini; P Milani; A Foli; M Vidus Rosin; M Basset; F Lavatelli; M Nuvolone; L Obici; S Perlini; G Merlini Journal: Leukemia Date: 2014-07-25 Impact factor: 11.528
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
Authors: Cihan Heybeli; Andrew Bentall; Jiqiu Wen; Mariam Priya Alexander; Francis K Buadi; Fernando G Cosio; Patrick G Dean; Angela Dispenzieri; David Dingli; Mireille El Ters; Morie A Gertz; Amer Hatem; Prashant Kapoor; Hasan Khamash; Taxiarchis Kourelis; Shaji Kumar; Elizabeth C Lorenz; Martin Mai; Eli Muchtar; David L Murray; Mikel Prieto; Carrie A Schinstock; Mark D Stegall; Rahma Warsame; Nelson Leung Journal: Kidney Int Date: 2020-07-23 Impact factor: 10.612