The following is a summary of “Micro-RNA Signature in CSF Before and After Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis,” published in the December 2024 issue of Neurology by Pavlovic et al.
MicroRNAs (miRNAs) regulate gene expression and are dysregulated in people with multiple sclerosis (PwMS). Autologous hematopoietic stem cell transplantation (aHSCT) is a treatment option for PwMS.
Researchers conducted a prospective study to assess whether aHSCT affects microRNA expression in cerebrospinal fluid (CSF) in PwMS.
They analyzed 87 miRNAs in CSF samples from a discovery cohort (baseline: 4 pwMS & 4 HCs) using quantitative reverse transcription PCR. The top 22 miRNAs that discriminated between pwMS and HCs were then validated in 187 CSF samples (50 pwMS, 32 HCs). After excluding samples with quality issues, 133 samples (29 HCs, 33 pwMS at baseline, 30 pwMS at 1 year, 26 pwMS at 2 years, and 15 pwMS at 3-5 years) were analyzed.
The results showed 12 miRNAs were dysregulated in pwMS compared to HCs (q < 0.05), 4 miRNAs (miR-16-5p, miR-21-5p, miR-150-5p, and miR-146a-5p) were identified with strong correlations (r > 0.60, P<0.001) to clinical parameters. Additionally, 8 of the 12 miRNAs were differentially expressed in pwMS with gadolinium-enhancing lesions at baseline, and 4 were associated with prior disease-modifying treatment class (P<0.05). These 4 miRNAs decreased after aHSCT and remained low throughout the follow-up period (P<0.05).
They concluded that a 4-miRNA signature was associated with higher disease activity and may normalize after aHSCT in PwMS.
Source: neurology.org/doi/10.1212/NXI.0000000000200345
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