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Treatment of Waldenstrom Macroglobulinemia with 2-Chlorodeoxyadenosine

Meletios A. Dimopoulos, MD; Hagop Kantarjian, MD; Elihu Estey, MD; Susan O'Brien, MD; Kay Delasalle, BS; Michael J. Keating, MBBS; Emil J. Freireich, MD; and Raymond Alexanian, MD
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From the University of Texas M. D. Anderson Cancer Center, Houston, Texas. Requests for Reprints: Meletios A. Dimopoulos, MD, University of Texas M. D. Anderson Cancer Center, Box 1, 1515 Holcombe Boulevard, Houston, TX 77030. Acknowledgments: The authors thank Dean Anthony and Rose Guevara for preparing and editing this manuscript. Grant Support: In part by the Hompe Myeloma Research Fund.

Copyright ©2004 by the American College of Physicians

Ann Intern Med. 1993;118(3):195-198. doi:10.7326/0003-4819-118-3-199302010-00007
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Objective: To evaluate the activity of 2-chlorodeoxyadenosine (2CdA) in the treatment of patients with Waldenstrom macroglobulinemia.

Design: Uncontrolled phase II trial.

Setting: Tertiary, referral cancer center.

Patients: Twenty-nine consecutive, symptomatic patients with Waldenstrom macroglobulinemia, of whom 9 were previously untreated.

Intervention: 2-Chlorodeoxyadenosine was administered as a continuous intravenous infusion at a dose of 0.1 mg/kg body weight per day for 7 days. Only two courses of 2CdA were given and responding patients were then followed without further treatment.

Measurements and Main Results: A total of 17 (59%) patients responded, including all of those who were newly diagnosed and 40% of those who had failed previous therapies. Treatment was well tolerated except for one death in a patient who had presented with severe pancytopenia. With a median follow-up of 7 months, only one responding patient has relapsed.

Conclusion: 2-Chlorodeoxyadenosine is a nucleoside analog that was effective in most patients with Waldenstrom macroglobulinemia and was associated with little toxicity.


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Figure 1.
Immunoglobulin M synthesis with 2-chlorodeoxyadenosine treatment in the nine previously untreated patients.
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Figure 2.
Marrow lymphocytes and hemoglobin levels in patients before and after treatment with 2-chlorodeoxyadenosine.Left.closed circlesopen circlesRight.closed circlesopen circles

Changes in marrow lymphocytes with 2-chlorodeoxyadenosine (2CdA) in responsive patients ( ) or unresponsive ( ) patients. Changes in hemoglobin level with 2CdA in responsive patients ( ) or unresponsive ( ) patients.

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