Background and objectives: Thalidomide is currently used as a very promising drug in patients with recurrent multiple myeloma or those refractory to chemotherapy. Literature data show prolonged survival in patients with advanced multiple myeloma treated with thalidomide but the optimal time and dose of thalidomide treatment remain to be established.
Design and methods: We have treated 53 refractory or relapsed myeloma patients with thalidomide (Grunenthal, Aachen). The patients received thalidomide orally as monotherapy at a starting dose of 200 mg daily, with a dose increase of 100 mg every week to a maximum well-tolerated dose of 400 mg. All the patients qualified for the therapy underwent clinical and laboratory assessments every 4 weeks. Laboratory tests included complete blood count, electrophoresis, immunoglobulin level, lactate dehydrogenase (LDH), C-reactive protein, b2 microglobulin concentration, liver and renal function tests and there was also a monthly neurological examination. Bone marrow aspiration was performed every 3 months during the 12-month treatment.
Results: Among 53 evaluable patients, a clinical response was observed in 27 (51%): there was a major response in 7 patients, a partial response in 12 and a minor response in 8.
Interpretation and conclusions: In responding patients the earliest response was observed after 4 weeks of treatment and the latest after 12 weeks of treatment. Our results, obtained during a long observation period, show that thalidomide is an effective drug, with an acceptable degree of toxicity, in patients with refractory multiple myeloma.