Originally published as JCO Early Release 10.1200/JCO.2007.14.1853 on March 24 2008

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Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Thalidomide Plus Dexamethasone Compared With Dexamethasone As Initial Therapy for Newly Diagnosed Multiple Myeloma

S. Vincent Rajkumar, Laura Rosiñol, Mohamad Hussein, John Catalano, Wieslaw Jedrzejczak, Lela Lucy, Marta Olesnyckyj, Zhinuan Yu, Robert Knight, Jerome B. Zeldis, Joan Bladé

From the Mayo Clinic, Rochester, MN; Cleveland Clinic, Cleveland, OH; Frankston Hospital, Frankston, Australia; Medical Academy of Warsaw, Warsaw, Poland; Pharmion Corp Boulder, CO; Celgene Corporation, Summit, NJ; and Hospital Clinic, IDIBAPS, Barcelona, Spain

Corresponding author: S. Vincent Rajkumar, MD, Division of Hematology, Mayo Clinic, 200 First St SW, Rochester, MN 55905; e-mail: rajkumar.vincent{at}mayo.edu

Purpose: The long-term impact of thalidomide plus dexamethasone (thal/dex) as primary therapy for newly diagnosed multiple myeloma (MM) is unknown. The goal of this study was to compare thalidomide plus dexamethasone versus placebo plus dexamethasone (placebo/dex)as primary therapy for newly diagnosed MM.

Patients and Methods: In this double-blind, placebo-controlled trial, patients with untreated symptomatic MM were randomized to thal/dex (arm A) or to placebo plus dexamethasone (dex) (arm B). Patients in arm A received oral thalidomide 50 mg daily, escalated to 100 mg on day 15, and to 200 mg from day 1 of cycle 2 (28-day cycles). Oral dex 40 mg was administered on days 1 through 4, 9 through 12, and 17 through 20 during cycles 1 through 4 and on days 1 through 4 only from cycle 5 onwards. Patients in arm B received placebo and dex, administered as in arm A. The primary end point of the study was time to progression. This study is registered at http://ClinicalTrials.gov (NCT00057564).

Results: A total of 470 patients were enrolled (235 randomly assigned to thal/dex and 235 to placebo/dex). The overall response rate was significantly higher with thal/dex compared with placebo/dex (63% v 46%), P < .001. Time to progression (TTP) was significantly longer with thal/dex compared with placebo/dex (median, 22.6 v 6.5 months, P < .001). Grade 4 adverse events were more frequent with thal/dex than with placebo/dex (30.3% v 22.8%).

Conclusion: Thal/dex results in significantly higher response rates and significantly prolongs TTP compared with dexamethasone alone in patients with newly diagnosed MM.

published online ahead of print at www.jco.org on March 24, 2008.

Supported by Celgene Corporation, Summit, NJ; Pharmion Ltd, Windsor, United Kingdom; Public Health Service Grants No. CA93842, CA 107476, CA 62242, and CA 100080 (S.V.R.) from the National Cancer Institute, National Institutes of Health, and the Department of Health and Human Services; and Spanish Grant No. RD 06/0020/ (L.R., J.B.).

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.