Long Discredited, Thalidomide Now Found to Suppress Cough in Patients With IPF
Baltimore—Thalidomide, used as an antinausea medication for pregnant women beginning in the mid-50s, was taken off the market in 1961 in a firestorm of controversy after more than 10,000 cases of related birth defects were reported in more than 46 nations.
It was discredited for decades and only began to be used with strict controls late in the 20th century for diseases such as multiple myeloma and kidney cancer. Now, the potent anti-inflammatory drug has shown promise in helping patients with a constant, disabling cough caused by idiopathic pulmonary fibrosis (IPF).
The study from the Johns Hopkins University School of Medicine was published recently in the Annals of Internal Medicine.
"We performed a randomized, double-blind, placebo-controlled trial of thalidomide in patients with IPF to determine its effectiveness in suppressing cough,"
said 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.
Up to 80% of patients with IPF, a progressive, fatal disorder that causes the lungs to become stiff and scarred, had a heretofore untreatable dry, nagging cough.
"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," Horton noted.
Patients participating in the double-blind study either took low-dose thalidomide pills or a placebo for 3 months. After a 2-week "wash out" period in which the patients took nothing, those who had taken the thalidomide went on the placebo and those who had been given the placebo started taking thalidomide for another three months.
The study was completed by 15 men and five women with a mean age of 67.
"At the end of the study, all of the participants said they wanted to continue taking the medicine because their cough had improved,” said Horton, who recounted that patients often noticed a difference after only 2 weeks on thalidomide.
On average, patient reports indicated a 63% decrease in coughing frequency when thalidomide was used and a 20% improvement in respiratory-specific quality of life.
Side effects, including 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, according to the report.
About 80,000 people in the U.S. have IPF, and the only cure is a lung transplant.