title: Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: a matched case-control study on 174 patients.
abstract: 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.
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: G
authors: G
authors: Palladini
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy [3] Department of Internal Medicine, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy.
authors: P
authors: P
authors: Milani
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: A
authors: A
authors: Foli
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: M
authors: M
authors: Vidus Rosin
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: M
authors: M
authors: Basset
authors: Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy.
authors: F
authors: F
authors: Lavatelli
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: M
authors: M
authors: Nuvolone
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: L
authors: L
authors: Obici
authors: Department of Internal Medicine, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy.
authors: S
authors: S
authors: Perlini
authors: 1] Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo, Pavia, Italy [2] Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors: G
authors: G
authors: Merlini
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism / Q000401:mortality; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001897:Boronic Acids / Q000008:administration & dosage; D000069286:Bortezomib; D016022:Case-Control Studies; D003907:Dexamethasone / Q000008:administration & dosage; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D011719:Pyrazines / Q000008:administration & dosage; D016896:Treatment Outcome
chemical_list: D001897:Boronic Acids; D007147:Immunoglobulin Light Chains; D011719:Pyrazines; D000069286:Bortezomib; D003907:Dexamethasone; D008558:Melphalan