Background: 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
Objective: To determine the efficacy of thalidomide in
suppressing cough in patients with IPF.
Design: 24-week, double-blind, 2-treatment, 2-period
crossover trial. (ClinicalTrials.gov registration number: NCT00600028)
Setting: 1 university center.
Participants: 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.
Measurements: 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.
Results: CQLQ scores significantly improved with
thalidomide (mean difference vs. placebo, −11.4 [95% CI, −15.7 to
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
Limitation: This was a single-center study of short duration
and small sample size focused on symptom-specific quality of life.
Conclusion: Thalidomide improved cough and respiratory quality
of life in patients with IPF. A larger trial is warranted to assess these
Primary Funding Source: Celgene Corporation.