Abstract Details

Title Tocilizumab for Refractory Neurosarcoidosis

Topic Autoimmune Neurology

Presentation(s) S30 - Autoimmune Neurology: Clinical CNS Autoimmune and Inflammatory Disorders (5:06 PM-5:18 PM)

Poster/Presentation
Number 009

Objective NA

Background

Neurosarcoidosis (NS) is a rare but important manifestation of sarcoidosis, characterized by granulomatous inflammation. Patients may present with diverse symptoms and risk permanent disability if inadequately treated. Corticosteroids are the mainstay of therapy, often followed by steroid-sparing agents and/or tumor necrosis factor (TNF) inhibitors. However, treatment failure or the development of neutralizing antibodies can limit their efficacy. Tocilizumab, an interleukin-6 receptor inhibitor, is a potential therapeutic alternative because of the role of IL-6 in granuloma formation.

Design/Methods

We retrospectively reviewed patients with NS evaluated at the Johns Hopkins Neurosarcoidosis Clinic and treated with tocilizumab between 2020–2025. Endpoints included: (1) proportion with clinical improvement or stabilization at six months, (2) MRI response (reduction in enhancement or lesion burden), (3) prednisone tapering or discontinuation, and (4) safety outcomes.

Results

Nine patients were treated with tocilizumab for a median of 18 months. Median age was 58 years; seven were female. All had systemic sarcoidosis and probable NS. Phenotypes included small fiber neuropathy (SFN) only (n=3), cranial neuropathy (CN) + SFN (n=4), CN + leptomeningeal disease (n=1), and CN + myelitis (n=1). All (9/9) had failed ≥3 prior immunosuppressants and 8/9 had received TNF inhibitors, with five developing anti-drug antibodies. Tocilizumab was given subcutaneously; 8/9 received concomitant immunosuppression. Prednisone tapering was achieved in 6/9 (three to ≤5 mg, three with prednisone discontinuation). Clinical improvement was observed in 7/9 patients, with MRI improvement noted in the two patients with CNS involvement. Overall, 7 out of 9 improved, and 2 out of 9 worsened clinically but not radiologically, necessitating discontinuation. No serious infections, or other major adverse events were observed.

Conclusions Tocilizumab appears to be a safe and generally effective option for refractory neurosarcoidosis. It facilitates corticosteroid tapering and achieves clinical and imaging improvement in most patients. Prospective studies are needed to clarify its role in treatment algorithms.

Authors/Disclosures
Munther Queisi, MD PRESENTER	Dr. Queisi has nothing to disclose.
Maria I. Bonilla	Miss Bonilla has nothing to disclose.
Clare M. Lambert, MD	Dr. Lambert has nothing to disclose.
David Acero-Garces, MD	Dr. Acero-Garces has nothing to disclose.
Carlos A. Pardo-Villamizar, MD (Johns Hopkins U, Med Dept of Neurology)	The institution of Dr. Pardo-Villamizar has received research support from National Institutes of Health. The institution of Dr. Pardo-Villamizar has received research support from Bart McLean Fund for Neuroimmunology Research .
Barney J. Stern, MD, FAAN (Johns Hopkins Outpatient Center)	Dr. Stern has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for Foundation for Sarcoidosis Research. The institution of Dr. Stern has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. The institution of Dr. Stern has received research support from NIH/NINDS. The institution of Dr. Stern has received research support from NIH/NINDS. The institution of Dr. Stern has received research support from NIH/NINDS. The institution of Dr. Stern has received research support from NIH/NINDS. Dr. Stern has received publishing royalties from a publication relating to health care.

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