Bortezomib, melphalan, and dexamethasone for light-chain amyloidosis
Efstathios Kastritis
(1)
,
Xavier Leleu
(2)
,
Bertrand Arnulf
(3)
,
Elena Zamagni
(4)
,
Maria Teresa Cibeira
,
Fiona Kwok
(5)
,
Peter Mollee
(6)
,
Roman Hajek
(7)
,
Philippe Moreau
(8)
,
Arnaud Jaccard
(9)
,
Stefan O. Schonland
(10)
,
Robin Filshie
(11)
,
Emmanuelle Nicolas-Virelizier
(12)
,
Bradley Augustson
,
Maria-Victoria Mateos
(13)
,
Ashutosh Wechalekar
,
Eric Hachulla
(14)
,
Paolo Milani
,
Meletios A. Dimopoulos
(15)
,
Jean-Paul Fermand
(16)
,
Andrea Foli
,
Maria Gavriatopoulou
(15)
,
Catherine Klersy
,
Antonio Palumbo
(17)
,
Pieter Sonneveld
(18)
,
Hans Erik Johnsen
(19)
,
Giampaolo Merlini
(20)
,
Giovanni Palladini
(20)
1
NKUA -
National and Kapodistrian University of Athens
2 Hôpital Claude Huriez [Lille]
3 AP-HP - Hopital Saint-Louis [AP-HP]
4 UNIBO - Alma Mater Studiorum Università di Bologna = University of Bologna
5 Westmead Hospital [Sydney]
6 Princess Alexandra Hospital, Brisbane
7 Ostravská univerzita / University of Ostrava
8 Hôtel-Dieu de Nantes
9 CHU Limoges
10 University of Heidelberg, Medical Faculty
11 St. Vincent's Hospital, Sydney
12 Centre Léon Bérard [Lyon]
13 Universidad de Salamanca
14 INFINITE - Institute for Translational Research in Inflammation - U 1286
15 University of Athens Medical School [Athens]
16 Hôpital Saint-Louis
17 UNITO - Università degli studi di Torino = University of Turin
18 Erasmus MC - Erasmus University Medical Center [Rotterdam]
19 AAU - Aalborg University [Denmark]
20 UNIPV - Università degli Studi di Pavia = University of Pavia
2 Hôpital Claude Huriez [Lille]
3 AP-HP - Hopital Saint-Louis [AP-HP]
4 UNIBO - Alma Mater Studiorum Università di Bologna = University of Bologna
5 Westmead Hospital [Sydney]
6 Princess Alexandra Hospital, Brisbane
7 Ostravská univerzita / University of Ostrava
8 Hôtel-Dieu de Nantes
9 CHU Limoges
10 University of Heidelberg, Medical Faculty
11 St. Vincent's Hospital, Sydney
12 Centre Léon Bérard [Lyon]
13 Universidad de Salamanca
14 INFINITE - Institute for Translational Research in Inflammation - U 1286
15 University of Athens Medical School [Athens]
16 Hôpital Saint-Louis
17 UNITO - Università degli studi di Torino = University of Turin
18 Erasmus MC - Erasmus University Medical Center [Rotterdam]
19 AAU - Aalborg University [Denmark]
20 UNIPV - Università degli Studi di Pavia = University of Pavia
Maria Teresa Cibeira
- Fonction : Auteur
Bradley Augustson
- Fonction : Auteur
Ashutosh Wechalekar
- Fonction : Auteur
Eric Hachulla
- Fonction : Auteur
- PersonId : 1366882
- ORCID : 0000-0001-7432-847X
- IdRef : 031788866
Paolo Milani
- Fonction : Auteur
Andrea Foli
- Fonction : Auteur
Catherine Klersy
- Fonction : Auteur
Résumé
Oral melphalan and dexamethasone (MDex) were considered a standard of care in light-chain (AL) amyloidosis. In the past decade, bortezomib has been increasingly used in combination with alkylating agents and dexamethasone. We prospectively compared the efficacy and safety of MDex and MDex with the addition of bortezomib (BMDex).
This was a phase III, multicenter, randomized, open-label trial. Patients were stratified according to cardiac stage. Patients with advanced cardiac stage (stage IIIb) amyloidosis were not eligible. The primary end point was hematologic response rate at 3 months. This trial is registered with ClinicalTrials.gov identifier NCT01277016.
A total of 109 patients, 53 in the BMDex and 56 in the MDex group, received ≥ 1 dose of therapy (from January 2011 to February 2016). Hematologic response rate at 3 months was higher in the BMDex arm (79% vPv
BMDex improved hematologic response rate and overall survival. To our knowledge, this is the first time a controlled study has demonstrated a survival advantage in AL amyloidosis. BMDex should be considered a new standard of care for AL amyloidosis.