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Sydney 2005:
Novel Insights into the Biology & Therapy of Waldenstrom's Macuglobulinemia
By Steve Treon
04.12.05
Dr. Treon presented an update from the Third International Workshop on WM (Paris, France). Dr. Treon shared the early reports of a strong link to familial WM. In a study of 257 patients, 1 out of 5 patients have a first degree relative with WM or a B-cell disorder. Interestingly, patients with familial link also present symptoms at an earlier age, almost a decade before other patients with WM. Dr. Treon’s studies revealed significant abnormalities in 4 genes that are expressed in WM: BLIMP1, BCL6, PAX5, and XBP1. Additionally, WM patients tend to have excess bone marrow mast cells (BMMC), which express CD40L, a potent ligand that induces B-cell growth. With this information, several studies are in place to target BMMC or block CD40L. Treatment options include the use of monoclonal antibody, rituximab, with chemotherapy, and in combination with novel agents such as thalidomide and CC-5013 (Revlimid). Data from these studies are to be presented at the 2005 American Society of Hematology meeting. Additional studies for the relapsed/refractory patient involve the use of alemtuzumab or bortezomib. An interesting and more recent study is underway investigating the use of sildenafil (Viagra) in patients with WM.


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