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Low‐dose continuous oral melphalan for the treatment of primary systemic (AL) amyloidosis
Author(s) -
Sanchorawala Vaishali,
Wright Daniel G.,
Seldin David C.,
Falk Rodney H.,
Berk John L.,
Dember Laura M.,
Finn Kathleen T.,
Skinner Martha
Publication year - 2002
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 - 10.1046/j.1365-2141.2002.03541.x
Subject(s) - melphalan , medicine , prednisone , al amyloidosis , regimen , cardiac amyloidosis , amyloidosis , chemotherapy , surgery , primary systemic amyloidosis , gastroenterology , systemic disease , immunopathology , immunology , antibody , immunoglobulin light chain
Summary.  Median survival of patients with AL amyloidosis with clinically significant cardiac involvement is 5 months when treated with cyclic melphalan and prednisone. We investigated a regimen of continuous oral melphalan as a single agent for patients with cardiac amyloidosis who were unable to tolerate prednisone or more aggressive chemotherapy. Thirty patients with amyloid cardiomyopathy were treated with continuous oral melphalan. Seven of 13 patients, evaluable after 3–4 months of treatment, achieved a partial haematological response and three achieved a complete haematological response; six patients have survived for > 1 year. This regimen appeared to be effective in inducing haematological responses in patients who received total doses of melphalan > 300 mg.

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