Maureen R. Horton, MD; Victoria Santopietro; Leena Mathew, BS; Karen M. Horton, MD; Albert J. Polito, MD; Mark C. Liu, MD; Sonye K. Danoff, MD; Noah Lechtzin, MD
Financial Support: Celgene Corporation.
Potential Conflicts of Interest: Disclosures can be
Reproducible Research Statement:Study protocol: Available from Dr. M.R. Horton
Statistical code and data set: Available from Dr.
Requests for Single Reprints: Maureen R. Horton, MD,
1830 East Monument Street, 5th Floor, Baltimore, MD 21205; e-mail,
Current Author Addresses: Drs. M.R. Horton, Danoff,
and Lechtzin: 1830 East Monument Street, 5th Floor, Baltimore, MD 21205.
Ms. Santopietro, Ms. Mathew, and Dr. Liu: 5501
Hopkins Bayview Circle, Asthma and Allergy Center, Baltimore, MD 21224.
Dr. K.M. Horton: JHOC 3253, 601 North Caroline
Street, Baltimore, MD 21287.
Dr. Polito: 301 St. Paul Place, McAuley Tower
Building, 4th Floor, Baltimore, MD 21202.
Author Contributions: Conception and design: M.R.
Horton, A.J. Polito, N. Lechtzin.
Analysis and interpretation of the data: M.R.
Horton, K.M. Horton, M.C. Liu, S.K. Danoff, N. Lechtzin.
Drafting of the article: M.R. Horton, S.K. Danoff,
Critical revision of the article for important
intellectual content: M.R. Horton, K.M. Horton, M.C. Liu, N. Lechtzin.
Final approval of the article: M.R. Horton, V.
Santopietro, K.M. Horton, M.C. Liu, S.K. Danoff, N. Lechtzin.
Provision of study materials or patients: L. Mathew,
A.J. Polito, M.C. Liu, S.K. Danoff, N. Lechtzin.
Statistical expertise: N. Lechtzin.
Obtaining of funding: M.R. Horton, L. Mathew.
Administrative, technical, or logistic support: L.
Mathew, A.J. Polito, M.C. Liu, N. Lechtzin.
Collection and assembly of data: M.R. Horton, V.
Santopietro, K.M. Horton.
Horton MR, Santopietro V, Mathew L, Horton KM, Polito AJ, Liu MC, et al. Thalidomide for the Treatment of Cough in Idiopathic
Pulmonary Fibrosis: A Randomized Trial. Ann Intern Med. 2012;157:398-406. doi: 10.7326/0003-4819-157-6-201209180-00003
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Published: Ann Intern Med. 2012;157(6):398-406.
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
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
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
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
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Pulmonary/Critical Care, Interstitial Lung Disease.
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