"The drug that has been the leading approach for treatment had never been rigorously tested in clinical research studies," said Mark Espeland, Ph.D., of Wake Forest, a study author. "Now that we know it isn't effective, research can focus on other possible treatments." Researchers at 10 clinical sites tested low-dose methotrexate, a drug that is used to treat lupus and other disorders, for managing hearing loss in autoimmune inner ear disease (AIED), a progressive disease caused by antibodies or immune cells damaging the inner ear. The hearing loss occurs over a few months and is often accompanied by ringing, hissing or roaring sounds.
AIED is rare and accounts for less than 1 percent of all cases of hearing impairment or dizziness. The condition is typically treated with high doses of steroids, such as prednisone, for a month. In patients who regain some hearing after steriods, methotrexate has been prescribed over the long term with the goal of preserving hearing.
"The question we wanted to answer was whether methotrexate can effectively be substituted for long-term steroid use, which isn't feasible because of potential side effects," said Espeland, a professor of biostatistics. "We found that methotrexate is no better than placebo at maintaining the restored hearing."
Espeland said it was natural to for physicians to turn to methotrexate as a possible treatment. The drug is safely and effectively used to treat rheumatoid arthritis and lupus, other forms of autoimmune disease.
The three-year study included 67 participants with rapidly progressing hearing loss in both ears. After taking steroids for a month, they were randomly assigned to take either methotrexate or an inactive pill made from sugar or starch, known as a placebo.
Espeland said the study is important because it could prevent people with AIED from getting a treatment that doesn't work. It also alerts researchers to focus attention on potential treatments for the disease.
"The results of this study clearly underscore the need for more effective and less toxic therapy for AIED, and that randomized, controlled trials are necessary to establish benefit," wrote the researchers in JAMA.
Wake Forest was the national coordinating center for the study, which was led by Dr. Jeffrey P. Harris, M.D., Ph.D., at the University of California in San Diego.
The study was a joint project of the National Institute of Deafness and Communication Disorders and the American Academy of Otolaryngology.
Media Contacts: Karen Richardson (krchrdsn@wfubmc.edu), Shannon Koontz (shkoontz@wfubmc.edu), Mark Wright (mwright@wfubmc.edu) at 336-716-4587.
Journal
JAMA