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


METHODS
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.


RESULTS
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.


CONCLUSIONS
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.
Judson
Marc A
MA
Division of Pulmonary and Critical Care Medicine, Medical University of South Carolina, Charleston, South Carolina 29425, USA.
Silvestri
Joanne
J
Hartung
Cindy
C
Byars
Teresa
T
Cox
Christopher E
CE
D000305:Adrenal Cortex Hormones / Q000008:administration & dosage / Q000627:therapeutic use*; D001741:Black or African American / Q000706:statistics & numerical data; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D008875:Middle Aged; D010865:Pilot Projects; D011241:Prednisone / Q000627:therapeutic use; D011446:Prospective Studies; D011788:Quality of Life; D017565:Sarcoidosis, Pulmonary / Q000188:drug therapy*; D011795:Surveys and Questionnaires; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D013997:Time Factors; D016896:Treatment Outcome; D044465:White People / Q000706:statistics & numerical data
D000305:Adrenal Cortex Hormones; D013792:Thalidomide; D011241:Prednisone; D006918:Hydroxyurea; D008727:Methotrexate