Influence of Efgartigimod on Curative Efficacy and Inflammation Factors of Patients with Long Segmental Myelitis

Summary

Efgartigimod has demonstrated significant efficacy and safety in patients with long-segment myelitis. Efgartigimod reduced serum levels of IL-6, IL-41, and IL-10 in LETM patients. The levels of these cytokines correlated with EDSS scores, suggesting their potential utility as biomarkers for predicting disease severity and treatment response in long-segment myelitis.

Abstract

The paper aims to evaluate the efficacy and impact of efgartigimod on clinical outcomes and inflammatory markers in patients with antibody-negative long-segment myelitis (LETM).This retrospective study enrolled 47 patients diagnosed with antibody-negative LETM, including 36 in the control group who received methylprednisolone alone and 11 in the observation group who received a combination of efgartigimod and methylprednisolone. Comparisons of the expanded disability status scale (EDSS), clinical frailty scale (CFS), and Health Survey Short Form-36 (SF-36) scores between the two groups were conducted. Concentrations of interleukin-6 (IL-6), IL-10, and IL-41 were detected using enzyme-linked immunosorbent assay (ELISA), and their correlations with EDSS scores were also evaluated. The potential predictors of therapeutic efficacy were identified using univariate and multivariate logistic regression analyses.Efgartigimod significantly reduced EDSS and CFS scores after 1 month of treatment, with the observation group demonstrating greater improvements than the control group. Efgartigimod improved the quality of life as assessed by the SF-36, with significant increases in physical functioning (PF) and vitality (VT) scores in the observation group (p < 0.05). Efgartigimod decreased serum cytokine (IL-41, IL-10, and IL-6) levels and EDSS scores at the time of treatment (p < 0.05). Serum cytokine levels were positively correlated with EDSS scores. The group (treatment strategy) and length of the involved segment were predictive factors of drug efficacy.Efgartigimod demonstrated significant efficacy and safety for the treatment of LETM. Efgartigimod lowered serum concentrations of IL-6, IL-41, and IL-10 in LETM patients. The levels of IL-6, IL-41, and IL-10 correlated with EDSS scores, indicating their potential as predictive biomarkers for disease severity and treatment efficacy in LETM.

Introduction

Long-segmental myelitis (LETM) is a rare but severe central nervous system disorder, typically defined by the involvement of three or more contiguous spinal cord segments, leading to motor, sensory, and autonomic dysfunction¹. Clinically, LETM is frequently associated with neuromyelitis optica spectrum disorder (NMOSD), which is primarily mediated by aquaporin-4 (AQP4) antibodies². However, a subset of LETM patients lacks detectable AQP4, myelin oligodendrocyte glycoprotein (MOG), or glial fibrillary acidic protein (GFAP) antibodies in serum, a condition termed antibody-negative LETM³. Intravenous....

Protocol

The research protocol was approved by the Ethics Committee of The Second Affiliated Hospital, Zhejiang University School of Medicine (No. 2025-0581). All experiments and procedures were performed according to the Declaration of Helsinki (as revised in 2013). This retrospective study included 47 patients hospitalized for LETM at the Second Affiliated Hospital, Zhejiang University School of Medicine. This study has obtained informed consent from all participants. This retrospective cohort study included all eligible patients. A post hoc power analysis was conducted to assess the adequacy of the sample size. The results showed that with the current sample size, the obser....

Results

A total of 47 patients were enrolled in this study across two cohorts. As shown in Table 1, there were no significant differences between the two groups in terms of age (p=0.414), sex (p=0.880), disease duration (p=0.144), and length of the involved spinal segments (p=0.945).

We evaluated the clinical efficacy of the two groups using the EDSS and CFS scores (Table 2). There were no significant differences in EDSS and CFS scores between the two groups before tr.......

Discussion

LETM frequently manifests as paralysis, limb weakness, urinary dysfunction, and other neurological symptoms²³. Methylprednisolone is the main clinical drug for the treatment of LETM and has a long history^24,25. However, there are some patients who do not respond²⁶. Efgartigimod is a reliable drug for the treatment of myasthenia gravis by reducing pathogenic IgG^27,.......

Materials

List of materials used in this article

Name	Company	Catalog Number	Comments
efgartigimod	Vetter Pharma-Fertigung GmbH & Co. KG, Germany	SJ20230008
ElaBoX Human Interleukin-10 Detection Kit	Solarbio, Beijing, China	SEKH-0018
ElaBoX Human Interleukin-6 Detection Kit	Solarbio, Beijing, China	SEKH-0014
GraphPad Prism 8	Graphpad, San Diego, CA, USA
Human IL-4 ELISA Kit	Thermo Fisher Scientific, Waltham, MA, USA	BMS225-2
IBM Corporation's SPSS 26.0	SPSS, Chicago, Illinois, USA
methylprednisolone	PFIZER SA,Bruxelles,Belgium	H20130302
Multiskan FC Enzyme-Linked Immunosorbent Assay (ELISA) Reader	Thermo Fisher Scientific, Waltham, MA, USA	1410101