In the first clinical trial to demonstrate an effective treatment for constant, disabling cough among people with idiopathic pulmonary fibrosis (IPF), researchers at the Johns Hopkins University School of Medicine found that taking thalidomide significantly reduced the cough and improved quality of life. Results of their study are published in the Annals of Internal Medicine on Sept. 18, 2012, in an article titled "Thalidomide for the Treatment of Cough in Idiopathic Pulmonary Fibrosis."
IPF is a progressive, fatal disorder that causes the lungs to become stiff and scarred, preventing oxygen from leaving the lungs to go to the rest of the body. The cause is unknown. Up to 80 percent of people with IPF have a dry, nagging cough, for which no effective treatment is available.
Thalidomide is a potent anti-inflammatory drug that was used to treat morning sickness and aid sleep in the 1950s. It was taken off the market in 1961 after it was shown to cause severe birth defects when women took the drug during pregnancy. Today, thalidomide is prescribed with strict controls to treat several diseases, including multiple myeloma and kidney cancer. It had not been studied for people with lung disease before.
"We performed a randomized, double-blind, placebo-controlled trial of thalidomide in patients with IPF to determine its effectiveness in suppressing cough," says lead author Maureen R. Horton, M.D., a pulmonary disease specialist and associate professor of medicine and environmental health sciences at the Johns Hopkins University School of Medicine.
"We found that low-dose thalidomide significantly reduced the cough and also improved the patients' quality of life, as demonstrated on established questionnaires known as the Cough Quality of Life Questionnaire and the St. George's Respiratory Questionnaire," says Horton.
For the study, patients either took low-dose thalidomide pills or a placebo for three months. Then there was a two-week "wash out" period in which the patients took nothing, followed by another three months when those who had taken the thalidomide went on the placebo and those who had been given the placebo started taking the thalidomide. Neither the patients nor the researchers knew which group the individuals were in.
Twenty patients completed both arms of the study - 15 men and five women. All were over age 50 and the mean age was 67.
Horton says the patients often noticed the difference within two weeks of taking the thalidomide. When they stopped the drug, their cough came back. "At the end of the study, all of the participants said they wanted to continue taking the medicine because their cough had improved."
On average, the patients reported that the frequency of their coughing decreased about 63 percent while they were taking thalidomide, and their respiratory-specific quality of life, such as the ability to do daily activities, improved about 20 percent. They also reported that the aspects of their life impacted by their cough also improved while they were on the drug.