Thalidomide: new indication. A last resort in myeloma.

(1) There is no reference treatment for patients with multiple myeloma who have relapsed, or not responded to first-line treatment. (2) Thalidomide has been granted temporary authorization in France for patients with multiple myeloma who have no other therapeutic option. (3) A non comparative trial of thalidomide therapy in 169 patients showed an increase in the survival time of a few months with an oral dose of 200 mg to 800 mg/day. In other trials the main endpoint was a fall in the circulating level of monoclonal immunoglobulin. (4) Three non comparative trials of the thalidomide + dexamethasone combination failed to show a survival advantage relative to thalidomide alone. There is no evidence that adding one or several cytotoxic agents to this combination improves outcomes, but assessment is limited to three small non comparative trials. (5) Thalidomide has many adverse effects, including neuropathies (often irreversible) and venous thromboembolism. It is highly teratogenic, and its use by men and women of childbearing potential is subject to strict conditions. (6) In practice, thalidomide monotherapy is justified for patients with multiple myeloma who have no other therapeutic option. All patients should be given comprehensive information on the risk-benefit ratio of this drug.