Abstract:Neuromyelitis optica spectrum disorder (NMOSD) is a rare neurological inflammatory disorder, characterized by recurrent myelitis and optic neuritis, which can lead to severe paralysis and blindness.It is reported that more than 75% of NMOSD patients are seropositive for aquaporin-4 immunoglobulin G (AQP4-IgG) [1].
“…Other experimental therapy approaches, such as intravenous immunoglobulins (IVIG), early anti-CD20 therapy, and early anti-complement therapy, have been reported in single case series to possibly lead to favorable outcomes in acute attacks [ 65 , 127 , 132 , 147 ]. Future therapies may also include antibodies against the neonatal Fc receptor, which are currently being studied in an open-label trial [ 236 , ClinicalTrials.gov]. Fig.…”
Section: Treatment Of Nmosd: General Aspects and Outcome Measuresmentioning
This manuscript presents practical recommendations for managing acute attacks and implementing preventive immunotherapies for neuromyelitis optica spectrum disorders (NMOSD), a rare autoimmune disease that causes severe inflammation in the central nervous system (CNS), primarily affecting the optic nerves, spinal cord, and brainstem. The pillars of NMOSD therapy are attack treatment and attack prevention to minimize the accrual of neurological disability. Aquaporin-4 immunoglobulin G antibodies (AQP4-IgG) are a diagnostic marker of the disease and play a significant role in its pathogenicity. Recent advances in understanding NMOSD have led to the development of new therapies and the completion of randomized controlled trials. Four preventive immunotherapies have now been approved for AQP4-IgG-positive NMOSD in many regions of the world: eculizumab, ravulizumab - most recently-, inebilizumab, and satralizumab. These new drugs may potentially substitute rituximab and classical immunosuppressive therapies, which were as yet the mainstay of treatment for both, AQP4-IgG-positive and -negative NMOSD. Here, the Neuromyelitis Optica Study Group (NEMOS) provides an overview of the current state of knowledge on NMOSD treatments and offers statements and practical recommendations on the therapy management and use of all available immunotherapies for this disease. Unmet needs and AQP4-IgG-negative NMOSD are also discussed. The recommendations were developed using a Delphi-based consensus method among the core author group and at expert discussions at NEMOS meetings.
“…Other experimental therapy approaches, such as intravenous immunoglobulins (IVIG), early anti-CD20 therapy, and early anti-complement therapy, have been reported in single case series to possibly lead to favorable outcomes in acute attacks [ 65 , 127 , 132 , 147 ]. Future therapies may also include antibodies against the neonatal Fc receptor, which are currently being studied in an open-label trial [ 236 , ClinicalTrials.gov]. Fig.…”
Section: Treatment Of Nmosd: General Aspects and Outcome Measuresmentioning
This manuscript presents practical recommendations for managing acute attacks and implementing preventive immunotherapies for neuromyelitis optica spectrum disorders (NMOSD), a rare autoimmune disease that causes severe inflammation in the central nervous system (CNS), primarily affecting the optic nerves, spinal cord, and brainstem. The pillars of NMOSD therapy are attack treatment and attack prevention to minimize the accrual of neurological disability. Aquaporin-4 immunoglobulin G antibodies (AQP4-IgG) are a diagnostic marker of the disease and play a significant role in its pathogenicity. Recent advances in understanding NMOSD have led to the development of new therapies and the completion of randomized controlled trials. Four preventive immunotherapies have now been approved for AQP4-IgG-positive NMOSD in many regions of the world: eculizumab, ravulizumab - most recently-, inebilizumab, and satralizumab. These new drugs may potentially substitute rituximab and classical immunosuppressive therapies, which were as yet the mainstay of treatment for both, AQP4-IgG-positive and -negative NMOSD. Here, the Neuromyelitis Optica Study Group (NEMOS) provides an overview of the current state of knowledge on NMOSD treatments and offers statements and practical recommendations on the therapy management and use of all available immunotherapies for this disease. Unmet needs and AQP4-IgG-negative NMOSD are also discussed. The recommendations were developed using a Delphi-based consensus method among the core author group and at expert discussions at NEMOS meetings.
“…The patient experienced no adverse events during or subsequent to treatment. Earlier studies have demonstrated that the addition of the FcRn-monoclonal antibody, Batoclimab, to IVMP therapy enhances the EDSS scores in patients with acute NMOSD within 3–6 months [ 15 ]. Thus, FcRn antagonists are probably beneficial not only during the acute phase of NMOSD but may also contribute to sustained neurological function improvement.…”
“…The drug is undergoing phase II trials for Myasthenia Gravis and currently utilised in Neuromyelitis Optica. In TED it may decrease circulating TSHR and IGF-1 antibodies [ 146 , 147 ].…”
Section: Targeting Components Of the Inflammatory Cascadementioning
Aims
This review aims to provide an overview of the current understanding of TED and its pathophysiology. To describe the evidence base for current consensus treatment recommendations and newer biological therapies available as well as to present future therapeutic research.
Methods
We reviewed and assessed the peer-reviewed literature placing particular emphasis on recent studies evaluating the pathophysiology of TED, landmark trials forming the basis of current management and recent clinical trials informing future therapeutics. Searched were made in MEDLINE Ovid, Embase Ovid, US National Institutes of Health Ongoing Trials Register and EU Clinical Trials Register. Keywords included: “Thyroid Eye Disease”, “Graves Orbitopathy”, “Thyroid Orbitopathy” and “Graves’ Ophthalmopathy”.
Results and conclusions
The pathophysiology of TED involves a complex array of cellular and humoral based autoimmune dysfunction. Previous therapies have been broad-based acting as a blunt instrument on this mechanism with varying efficacy but often accompanied with a significant side effect profile. The recent development of targeted therapy, spearheaded by Teprotumumab has led to an array of treatments focusing on specific components of the molecular pathway optimising their impact whilst possibly minimising their side effect profile. Future challenges involve identifying the most effective target for each patient rather than any single agent being a panacea. Long-term safety profiles will require clarification as unintended immunological consequence downstream may become manifest as seen in other diseases. Finally, future novel therapeutics will entail significant expenditure and may lead to a divergence of available treatment modalities between healthcare systems due to funding disparities.
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