This transcript has been edited for clarity.
Dear colleagues, I'm Christoph Diener from the University of Duisburg-Essen in Germany. Today I'd like to discuss six neurology studies that were published recently.
Edaravone for Amyotrophic Lateral Sclerosis (ALS)
The first study deals with a substance called edaravone, which was previously investigated in Japan in placebo-controlled trials. In a subgroup of patients with ALS, there seems to be a benefit of this substance. Edaravone was approved in the United States and Japan, but not by the European Medicines Agency.
What do we know about edaravone in everyday clinical practice? A group in Germany sought to answer that by performing a real-world study with 116 patients treated with edaravone and another 116 patients who received standard of care. Both groups were propensity score-matched and received treatment for 14 months.
Unfortunately, edaravone had no impact on the progression of the disease, mortality, and time to ventilation. This means, most probably, that edaravone is not effective in patients with ALS or in the subgroups of patients who seemed to benefit in the study in Japan.
Results of the ATRIL Trial
The next study deals with the treatment of spinocerebellar ataxia type 2. Investigators in France behind the ATRIL Trial randomized 45 patients with this condition to receive either riluzole, which is approved for ALS, or placebo.
Unfortunately, there was no benefit of riluzole for the primary and secondary endpoints, which means we still have no effective medical therapy for this condition.
Two Studies on Insomnia
The standard sleeping medications are either benzodiazepines or substances that work via the gamma-aminobutyric acid system. Now, however, we have a totally new generation of sleeping medications for the treatment of insomnia, the so-called orexin receptor antagonists. Orexins are produced in the hypothalamus and they play a very important role in the control of sleep.
The results of two randomized placebo-controlled studies with daridorexant, a novel orexin receptor antagonist, were recently published in Lancet Neurology. Both studies had more than 900 patients each and tested doses of daridorexant against placebo for a duration of 3 months.
They observed that the 25- and 50-mg doses were clearly superior to placebo for the induction of sleep and the quality of sleep, and there was no hangover for the next day. This seems to be the most important benefit of this new class of substances: They improve sleep without causing sleepiness or impacting awareness during the next day.
The US Food and Drug Administration (FDA) approved daridorexant at 25 mg and 50 mg for the treatment of adult patients with insomnia in early January.
An Effective Option for Idiopathic Hypersomnia
The next study deals with patients who have the opposite problem, idiopathic hypersomnia. These patients have sleeping disorders but also increased sleepiness during the day.
In this study, published in Lancet Neurology, investigators compared low-sodium oxybate with placebo in 154 adult patients with idiopathic hypersomnia. They observed a significant benefit of oxybate in the Epworth Sleepiness Scale, and the drug was relatively well tolerated. The fact that the drug has a low sodium content is also important for preventing hypotension.
Multiple Sclerosis (MS), Pregnancy, and Immunomodulatory Treatment
The next analysis comes from a German registry real-world study of 255 women with MS who became pregnant, which was published in JAMA Network Open. These women terminated their therapy with natalizumab either before pregnancy or during pregnancy.
Unfortunately, investigators reported that the women experienced a higher rate of MS exacerbations. About 10% had an increase in permanent disability after pregnancy. A possible consequence of these results would be that this treatment should be continued during pregnancy or should be changed to another immunomodulatory treatment prior to pregnancy.
Aspirin for COVID-19
The last study I wanted to talk about, which was published in The Lancet, deals with the treatment of COVID-19. We all know that COVID-19 leads to an increased risk for thromboembolic events, both in the venous system and in the arterial system.
RECOVERY trial investigators looked at whether adding aspirin has a benefit. They studied approximately 14,900 patients, half of whom received aspirin and the other half who did not. Aspirin had no impact on mortality and length of stay in the hospital. There were fewer thrombotic events on aspirin but a slightly increased risk for bleeding.
Dear colleagues, I hope you enjoyed hearing about these six interesting studies from January and February 2022, which might have an impact on how you treat your patients.
I am Christoph Diener from the Faculty of Medicine at the University of Duisburg-Essen. Thank you very much for listening and watching.
Medscape Neurology © 2022
Cite this: Neurology Journal Highlights: 6 New Studies - Medscape - Apr 18, 2022.