Scientific publications

A phase II trial of lenalidomide, dexamethasone and cyclophosphamide for newly diagnosed patients with systemic immunoglobulin light chain amyloidosis

May 14, 2015 | Magazine: British Journal of Haematology

Cibeira MT(1), Oriol A(2), Lahuerta JJ(3), Mateos MV(4), de la Rubia J(5), Hernández MT(6), Granell M(7), Fernández de Larrea C(1), San Miguel JF(8), Bladé J(1); PETHEMA cooperative study group.

(1) Amyloidosis and Myeloma Unit, Hospital Clínic, IDIBAPS, University of Barcelona, Barcelona, Spain.
(2) ICO-Hospital Universitari Germans Trias i Pujol, Barcelona, Spain.
(3) Hospital Doce de Octubre, Madrid, Spain.
(4) Hospital Clínico/Centro de Investigacion del Cáncer, Salamanca, Spain.
(5) Hospital La Fe, Valencia, Spain.
(6) Hospital Universitario de Canarias, Santa Cruz de Tenerife, Spain.
(7) Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
(8) Clínica Universidad de Navarra, CIMA, Pamplona, Spain.


Abstract

Immunomodulatory drugs have been shown to be of benefit in relapsed/refractory immunoglobulin light-chain (AL) amyloidosis. We designed a prospective, multicentre phase II trial of lenalidomide, dexamethasone and cyclophosphamide for newly diagnosed patients with AL amyloidosis not eligible for autologous stem-cell transplantation.

Twenty-eight patients were included in the study. Cardiac involvement was present in 23 patients; 14 of them had cardiac stage III. The overall haematological response rate was 46%, including complete and very good partial responses in 25% and 18% of patients respectively. Haematological response was mainly associated with absence of cardiac stage III and lower tumour burden. Organ response was observed in 46% of patients. After a median follow-up of 24 months, median progression-free and overall survival have not been reached, both being significantly longer in responders (P < 0·001 and P = 0·001 respectively). Seventeen patients have discontinued treatment, mostly due to amyloid-related death, disease progression or lack of response. Only 14% of the patients discontinued treatment due to therapy-related adverse events.

Our results support the efficacy of this regimen, with high quality responses and prolonged survival, as well as its tolerability, in patients with AL amyloidosis not eligible for stem cell transplant and without advanced cardiac involvement (clinicaltrials.gov identifier: NCT01194791).

CITATION  Br J Haematol. 2015 May 14. doi: 10.1111/bjh.13500.

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