Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal disorder of unknown cause
with no effective treatment. Cough affects up to 80% of patients with IPF, is frequently
disabling, and lacks effective therapy.
To determine the efficacy of thalidomide in suppressing cough in patients with IPF.
24-week, double-blind, 2-treatment, 2-period crossover trial. (ClinicalTrials.gov
registration number: NCT00600028)
1 university center.
98 participants were screened, 24 were randomly assigned, 23 received treatment (78.3%
men; mean age, 67.6 years; mean FVC, 70.4% predicted), and 20 completed both treatment
periods.
The primary end point was cough-specific quality of life measured by the Cough Quality
of Life Questionnaire (CQLQ). Secondary end points were visual analogue scale of cough
and the St. George's Respiratory Questionnaire (SGRQ). For all measures, lower scores
equaled improved cough or respiratory quality of life.
CQLQ scores significantly improved with thalidomide (mean difference vs. placebo,
-11.4 [95% CI, -15.7 to -7.0]; P < 0.001). Thalidomide also significantly improved
scores on the visual analogue scale of cough (mean difference vs. placebo, -31.2 [CI,
-45.2 to -17.2]; P < 0.001). In participants receiving thalidomide, scores from the
total SGRQ, SGRQ symptom domain, and SGRQ impact domain improved compared with those
of participants receiving placebo. Adverse events were reported in 74% of patients
receiving thalidomide and 22% receiving placebo; constipation, dizziness, and malaise
were more frequent with thalidomide.
This was a single-center study of short duration and small sample size focused on
symptom-specific quality of life.
Thalidomide improved cough and respiratory quality of life in patients with IPF. A
larger trial is warranted to assess these promising results.
Celgene Corporation.