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A phase II trial of lenalidomide, dexamethasone and cyclophosphamide for newly diagnosed patients with systemic immunoglobulin light chain amyloidosis
Author(s) -
Cibeira Maria T.,
Oriol Albert,
Lahuerta Juan J.,
Mateos MariaVictoria,
Rubia Javier,
Hernández Miguel T.,
Granell Miquel,
Fernández de Larrea Carlos,
San Miguel Jesús F.,
Bladé Joan
Publication year - 2015
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13500
Subject(s) - medicine , al amyloidosis , lenalidomide , tolerability , cyclophosphamide , amyloidosis , primary systemic amyloidosis , transplantation , gastroenterology , regimen , autologous stem cell transplantation , dexamethasone , surgery , adverse effect , oncology , chemotherapy , immunology , systemic disease , immunopathology , immunoglobulin light chain , antibody
Summary 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).