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Thalidomide Treatment for Cough in Patients With Idiopathic Pulmonary Fibrosis FREE

[+] Article and Author Information

The full report is titled “Thalidomide for the Treatment of Cough in Idiopathic Pulmonary Fibrosis. A Randomized Trial.” It is in the 18 September 2012 issue of Annals of Internal Medicine (volume 157, pages 398-406). The authors are M.R. Horton, V. Santopietro, L. Mathew, K.M. Horton, A.J. Polito, M.C. Liu, S.K. Danoff, and N. Lechtzin.


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Ann Intern Med. 2012;157(6):I-38. doi:10.7326/0003-4819-157-6-201209180-00001
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What is the problem and what is known about it so far?

Idiopathic pulmonary fibrosis (IPF) is a chronic lung disease that causes progressive shortness of breath and is frequently fatal. Patients with IPF also often have a debilitating cough for which there are no established effective treatments. Although thalidomide causes harm to developing fetuses when used to treat nausea in pregnant women, the drug's effects on the immune system suggested that it might be useful to treat IPF. In a previous inconclusive study of thalidomide's effect on IPF itself, some patients reported an improvement in their cough.

Why did the researchers do this particular study?

To determine whether thalidomide can improve the symptoms of cough in patients with IPF.

Who was studied?

24 patients with IPF treated at a single specialty clinic or who responded to an Internet announcement.

How was the study done?

Patients received thalidomide and placebo pills, each for 12 weeks. The order in which they received the treatments was randomly assigned. Patients rated the effect of cough on their quality of life, as well as the severity of their cough, using standardized questionnaires. Changes in the questionnaire scores after 12 weeks of thalidomide and placebo treatment were compared. Neither the patients nor the treating physicians were told which regimen was being administered. Patients received medication to prevent constipation, a known adverse effect of thalidomide.

What did the researchers find?

Quality of life related to cough improved more after 12 weeks of thalidomide treatment than after placebo. Cough severity also reduced more after thalidomide treatment. Patients had adverse effects more often during thalidomide treatment than with placebo, the most common being constipation in approximately one third of patients.

What were the limitations of the study?

The study was small and was conducted at only 1 highly specialized treatment center, so the applicability of the results to other patients and settings is unknown. The study was also of short duration.

What are the implications of the study?

Thalidomide may be a useful treatment for troubling cough in patients with IPF. Larger, more definitive studies are required to more thoroughly assess its effect and safety for such use.

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Author reply: To PMID 22986377. Ann Intern Med 2013;158(6):498-9.

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