Oral fingolimod is no better than placebo for treating chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), according to results from a multinational randomized controlled trial (RCT).1 “There is no evidence from randomised controlled trials that any immunomodulatory treatments other than corticosteroids, intravenous or subcutaneous immunoglobulin, and plasma exchange are beneficial in CIDP,” wrote first author Richard Hughes, MD, of National Hospital for Neurology and Neurosurgery, London, UK, and colleagues.
CIDP refers to a group of rare neurological diseases that affect the peripheral nervous system. Although the cause has yet to be definitively established, CIDP is thought to result from an autoimmune process that causes the myelin sheath around nerves to degenerate, causing weakness, numbness and tingling of the extremities. In severe cases, CIDP can interfere with mobility.
Recommended first-line treatments for CIDP are generally effective and include corticosteroids or intravenous immunoglobulin (IVIg), and plasma exchange if these fail. However, not all patients respond adequately, and side effects as well as cost can limit their use. Other immunosuppressive and immunomodulatory agents are sometimes used in CIDP, but no formal evidence supports their efficacy.
Oral fingolimod is approved for treating relapsing-remitting multiple sclerosis, another inflammatory demyelinating disorder of the nervous system. Studies in rats have suggested that the drug may be a candidate for treating CIDP.
To test oral fingolimod in humans, researchers conducted a double-blind, randomized placebo-controlled trial in 48 neurology centers in Australia, Canada, Israel, Japan, the USA and nine countries in Europe between January 2013 and March 2016. Researchers randomized 106 participants who were already being treating with corticosteroids or IVIg to once daily fingolimod (0.5 mg, n=54) or placebo (n=52). Participants either discontinued IVIg treatment one day before receiving the study drug or underwent an eight-week steroid taper after randomization.
The primary endpoint was time to first worsening, defined as ≥ 1 point increase on the standardized Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale score.
1. Hughes R, Dalakas MC2, Merkies I, et al. Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial. Lancet Neurol. 2018;17:689-698.