ACTH Superior to Treat Infantile Spasms, SESI-HRMS Serves as Viable OSA Screening Tool, Myeloid Microvesicles Diagnostic Value in MS

This week Neurology News Network covered a comparison study that looked at treatments for infantile spasms, correlations between biomarkers and obstructive sleep apnea severity, and the use of CSF myeloid microvesicles in predicting neuroinflammation and microglial/macrophage activity in vivo.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Findings from a recent study by the National Infantile Spasms Consortium suggest that adrenocorticotropic hormone (ACTH) and oral steroids are superior methods for treating infantile spasms when compared with nonstandard therapy. The prospective, multicenter, observational cohort study included 423 children with infantile spasm that began at 2 to 24 months. Using chart review, investigators found freedom from treatment failure rates were 46% (88 of 190) for ACTH, 44% (42 of 95) for oral steroids, 37% (32 of 87) for vigabatrin, and 8% (4/51) for nonstandard therapy. Investigators accounted for estimated superiority of standard therapies, and higher E-values (9.2 and 9) suggested findings would not be contradicted with unmeasured confounding. Vigabatrin, a GABA transaminase inhibitor, was estimated to be more effective than nonstandard therapies, but less effective than ACTH and oral steroids. Despite being underpowered for statistical significance, it was found to work for children with tuberous sclerosis complex (TSC), with freedom from treatment failure rates for those with TSC highest at 62% (13 of 21) when compared with other treatments at 29%.

Findings from a recent observational study confirmed a significant difference between patients with obstructive sleep apnea (OSA) and those without, as well as a correlation between biomarker levels and OSA severity, indicating that secondary electrospray ionization high-resolution mass spectrometry (SESI-HRMS) may be a viable screening method. A panel of 33 metabolites/breath biomarkers were validated in the observational study, confirming previous findings of the OSA-specific metabolic breath pattern in a pilot study.Future validation studies are necessitated investigate the value of SESI-HRMS in coordination with OSA screening questionnaires, and additionally, studies should include larger cohorts. Lead author Nora Nowak and colleagues wrote, “To the best of our knowledge, this is the first report of a validation of breath biomarkers for OSA. Previous studies using e-noses, offline gas chromatography couple to mass spectrometry, or enzyme immunoassays to analyze exhaled breath condensate have achieved promising results regarding the distinction between OSA patients and controls without OSA from exhaled breath. However, sample sizes in all these studies were limited and none of the results has been validated in an independent cohort of patients.”

Recently published findings suggest that CSF myeloid microvesicles (MVs) have diagnostic and prognostic value in predicting neuroinflammation and microglial/macrophage activity in vivo. The results also support possible use in clinical practice to compliment MRI and clinical evaluation at the moment of multiple sclerosis (MS) diagnosis. Investigators evaluated CSF myeloid MVs in 601 individuals with a diagnosis of neuroinflammatory, neurodegenerative, or no neurological disease at discharge or follow-up. Myeloid MVs were measured with flow cytometry as isolectin B4-positive events in fresh CSF. A post-hoc analysis showed statistically significant differences in median MV concentration between the neuroinflammatory group and the neurodegenerative/dementia group, and between the neuroinflammatory group and the control group. Notably, no differences were found between the neurodegenerative/dementia group and the CG.

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