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Blood, 1 December 2004, Vol. 104, No. 12, pp. 3520-3526.
Prepublished online as a Blood First Edition Paper on August 12, 2004; DOI 10.1182/blood-2004-05-1924.
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Submitted May 21, 2004
Accepted July 20, 2004
Clinical efficacy of high dose dexamethasone with maintenance dexamethasone/alpha interferon in patients with primary systemic amyloidosis: results of United States intergroup trial southwest oncology group (SWOG) S9628
Madfhav V Dhodapkar*, Mohamad A Hussein, Erik Rasmussen, Alan Solomon, Richard A Larson, John J Crowley, and Bart Barlogie
Laboratory of Tumor Immunology and Immunotherapy, Rockefeller University and Hematology Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA
Cleveland Clinic Foundation, Cleveland, OH, USA
Southwest Oncology Group Statistical Center, Seattle, WA, USA
University of Tennessee Cancer Institute, Knoxville, TN, USA
University of Chicago and Cancer and Leukemia Group B, Chicago, IL, USA
University of Arkansas for Medical Sciences, Little Rock, AR, USA
* Corresponding author; email: dhodapm{at}mail.rockefeller.edu.
Current therapy of primary systemic (AL) amyloidosis with oral melphalan and prednisone remains unsatisfactory, with a median survival of only 13 months. Between 1996 and 2003, 93 patients with biopsy proven AL amyloidosis were enrolled in a prospective US national cooperative group trial. Treatment schema consisted of induction therapy with pulse dexamethasone (DEX), followed by maintenance therapy with DEX and alpha interferon. Hematologic complete remissions were observed in 24% and improvement in AL related organ dysfunction occurred in 45% of patients evaluable for response. Median survival of the entire cohort is 31 months, with an estimated 2 year overall (OS) and event free survival (EFS) of 60% and 52% respectively. Presence of congestive heart failure and increased level of serum beta 2 microglobulin (> 3.5 mg/L) were dominant predictors of adverse outcome. Estimated 2 year OS in transplant eligible patients with this approach was 78%. These data demonstrate for the first time in the context of a US multicenter prospective clinical trial, that front line therapy with a DEX based regimen in AL amyloidosis can lead to durable reversal of AL related organ dysfunction, and prolonged survival.

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