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Blood, 15 August 2008, Vol. 112, No. 4, pp. 1035-1038.
Prepublished online as a Blood First Edition Paper on May 23, 2008; DOI 10.1182/blood-2008-02-140954.


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Submitted February 22, 2008
Accepted April 1, 2008

First thalidomide clinical trial in multiple myeloma: a decade later

Frits van Rhee, John D. Shaughnessy, Jr., Elias Anaissie, David Siegel, Antje Hoering, Jerome Zeldis, Bonnie Jenkins, Seema Singhal, Jayesh Mehta, John Crowley, Sundar Jagannath, and Bart Barlogie*

Myeloma Institute for Research and Therapy, University of Arkansas for Medical Sciences, Little Rock
Hackensack University, Hackensack, NJ
Cancer Research and Biostatistics, CRAB, Seattle
Celgene Corporation, Celgene Corporation, Cambridge
Northwestern University, Chicago, IL
St. Vincent's Hospital, New York, NY

* Corresponding author; email: barlogiebart{at}uams.edu.

The clinical outcomes of 169 patients, enrolled in the first clinical trial of thalidomide for advanced or refractory myeloma, are updated. Seventeen patients remain alive and 10 event-free, with a median follow-up of 9.2 years. According to multivariate analysis of pre-treatment variables, cytogenetic abnormalities (CA), present in 47% of patients within 3 months of enrollment, and lambda light chain isotype both affected overall survival and event-free survival adversely. Forty percent of the 58 patients lacking these 2 unfavorable features, one-half of whom had no disease recurrence, survived at least 6 years, in contrast to fewer than 5% among those with 1 or 2 risk features (P<0.0001). Patients who had received cumulative thalidomide doses in excess of 42g in the first 3 months enjoyed superior overall and event-free survival. The poor outcome associated with lambda-type myeloma may relate to its over-representation in molecularly defined high-risk disease gleaned from studies in newly diagnosed myeloma.


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