The effect of thalidomide on corticosteroid-dependent pulmonary sarcoidosis

To determine the benefit and corticosteroid-sparing potential of thalidomide in corticosteroid-dependent pulmonary sarcoidosis.

The study was a prospective open-label pilot study. Ten subjects were enrolled who had pulmonary sarcoidosis, required corticosteroid therapy for at least six months, and experienced a flare of pulmonary sarcoidosis within the previous 2 years when corticosteroids had been reduced or discontinued. Thalidomide was given at a dose of 200 mg/day for 24 weeks. Dose reductions were allowed in 50 mg/day increments for side effects. After 12 weeks, the baseline corticosteroid dose was halved for the remaining 12 weeks of the study.

There were no clinically or statistically significant changes in spirometry (FVC% predicted: week 0: 72 +/- 4, week 12: 75 +/- 5, week 24: 73 +/- 4; p = NS), quality of life (as measured by the Short Form-36), or dyspnea (as measured by the Transitional Dyspnea Index) between weeks 0, 12, and 24. Three (30%) subjects demonstrated clinical evidence of a corticosteroid-sparing effect from thalidomide. Three (30%) subjects had to withdraw from the study; two at week 4 because of an acute pulmonary flare of sarcoidosis, and one because of possible side effects. Although nine of 10 (90%) of subjects required a reduction of the thalidomide dose because of side effects, no adverse effects were severe. The most common final dose of thalidomide was 100 mg/day.

Thalidomide does not significantly improve pulmonary function or quality of life in patients with corticosteroid-dependent sarcoidosis. Few patients can tolerate doses of greater than 100 mg/day. Thalidomide may have been corticosteroid-sparing in a subgroup of these patients.