Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: a matched case–control study on 174 patients

• Published in: Leukemia Vol. 28; no. 12; pp. 2311 - 2316
• Main Authors: Palladini, G, Milani, P, Foli, A, Vidus Rosin, M, Basset, M, Lavatelli, F, Nuvolone, M, Obici, L, Perlini, S, Merlini, G
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.12.2014; Nature Publishing Group
• Subjects: 631/67/1059/99; 692/699/1541; 692/699/67/1990/804; Aged; Alkylation; Amyloidosis; Amyloidosis - diagnosis; Amyloidosis - drug therapy; Amyloidosis - metabolism; Amyloidosis - mortality; Antineoplastic Combined Chemotherapy Protocols - adverse effects; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Boronic Acids - administration & dosage; Bortezomib; Cancer; Cancer Research; Case-Control Studies; Chemotherapy; Clinical trials; Cloning; Congestive heart failure; Critical Care Medicine; Dexamethasone; Dexamethasone - administration & dosage; Dosage; Drug dosages; Drug therapy; Hematology; Humans; Immunoglobulin Light Chains - metabolism; Intensive; Internal Medicine; Leukemia; Medical prognosis; Medicine; Medicine & Public Health; Melphalan; Melphalan - administration & dosage; Methods; Middle Aged; Multiple myeloma; Oncology; original-article; Patient outcomes; Patients; Peptides; Pyrazines - administration & dosage; Renal function; Response rates; Stem cell transplantation; Stem cells; Steroids; Survival; Toxicity; Transplantation; Transplants & implants; Treatment Outcome; Italy
• ISSN: 0887-6924; 1476-5551
• DOI: 10.1038/leu.2014.227

Oral melphalan and dexamethasone (MDex) is a standard treatment for patients with AL amyloidosis who are not eligible for stem cell transplantation at many referral centers. However, following encouraging reports on the activity of bortezomib combined with alkylators and dexamethasone, these combinations are being moved to frontline therapy. We compared the outcome of 87 patients treated with bortezomib plus MDex (BMDex) with that of 87 controls treated with MDex. Patients and controls were matched for age, cardiac and renal function and free light chain burden. A higher rate of complete responses was observed with BMDex (42 vs 19%), but this did not result in a survival improvement in the overall population. However, a significant survival advantage for BMDex was observed in patients without severe (New York Heart Association class III or IV) heart failure and with N-terminal pro-natriuretic peptide type-B <8500 ng/l. Patients treated with full-dose dexamethasone had similar response rates and survival whether they received bortezomib or not. Intermediate-risk patients who are not fit enough to receive high-dose dexamethasone are likely to take the greatest advantage from the addition of bortezomib to MDex.