Researchers at the Johns Hopkins University School of Medicine have found that thalidomide is an effective treatment for the cough associated with idiopathic pulmonary fibrosis (IPF). Up to 80% of people with IPF have a dry, nagging cough, for which no effective treatment is available.
The results of the study appear in the Annals of Internal Medicine.
Thalidomide is a 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, MD, 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 3 months. Then there was a 2-week "wash out" period in which the patients took nothing, followed by another 3 months when those who had taken the thalidomide went on the placebo and those who had been given the placebo started taking 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 patients often noticed the difference within 2 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% while they were taking thalidomide, and their respiratory-specific quality of life, such as the ability to do daily activities, improved about 20%. They also reported that the aspects of their life impacted by their cough also improved while they were on the drug.
"The constant cough caused by the disease can affect the quality of life in many ways," says Horton. "Some no longer go to church or to social gatherings because people think they are infectious. Other patients may have more pronounced urinary incontinence due to the cough, for example, so it has wide-ranging effects."
Side effects, such as constipation, dizziness and malaise were reported by 74% of the participants while they were taking thalidomide, and by 22% of those who were on a placebo.
Horton says the idea of testing thalidomide for cough among IPF patients came about because the drug is known to have a powerful effect on decreasing inflammation. Horton and her colleagues conducted a previous smaller study to see if thalidomide would help treat the IPF disease itself, and while the results of that research were not conclusive, they noticed that the patients had significant cough relief.
Source: Johns Hopkins Medicine