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Archives: Research Summaries

Quantifying the relationship between disability progression and quality of life in patients treated for NMOSD: Insights from the SAkura studies

Quantifying the relationship between disability progression and quality of life in patients treated for NMOSD: Insights from the SAkura studies

Journal: Multiple Sclerosis and Related Disorders; October 14, 2021

Author(s): Michael Levy, Alan R. Haycox, Ursula Becker, Cristina Costantino, Elisabetta Damonte, Gaelle Klingelschmitt, H. Christian von Büdingen, Gene Wallenstein, D. Di Maio, Lech Szczechowsk

How disability affects quality of life in patients treated for NMOSD

This study used data from published clinical trials for the approved therapy satralizumab to assess how disability affects quality of life in patients with NMOSD. NMOSD considerably affects patients’ quality of life because of pain, impaired physical and cognitive function, deficits in sensation and/or motor function, loss of vision, sexual/bowel/bladder dysfunction, and mental health challenges, all of which can impact their ability to work or perform day-to-day activities. In this study, disability and quality of life scores were correlated for NMOSD patients in the SAkuraStar and SAkuraSky studies, which evaluated the effects of satralizumab administered alone or along with immunosuppressants. The study showed that with every additional disability accrual, the patients’ quality of life scores reduced.

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Advances in the long-term treatment of neuromyelitis optica spectrum disorder

Advances in the long-term treatment of neuromyelitis optica spectrum disorder

Journal: Journal of Nervous System Disease; February 1, 2024

Author(s): Monique Anderson, Michael Levy

Long-term treatment approaches for NMOSD

Neuromyelitis optica spectrum disorder (NMOSD) is a rare inflammatory disorder of the central nervous system where myelin, the protective sheath surrounding neurons, gets eroded. occurs in approximately 1-5 individuals per 100,000 globally. It can affect the optic nerve, spinal cord, and brainstem. If left untreated, NMOSD attacks can lead to irreversible damage; therefore, treatment approaches focus on preventing repeat attacks. A better understanding of the underlying causes of NMOSD over the last 10 years has led to new FDA-approved treatments. This review discusses available approved and off-label long-term treatments used for NMOSD, treatments in the pipeline for adult and pediatric NMOSD patients, and considerations in pregnant patients and in the context of COVID-19. Patients would find this review helpful when discussing treatment options with their neurologist.

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Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial (S5.002)

Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial (S5.002)

Journal: Neurology; April 25, 2023

Author(s): Sean Pittock, Michael Barnett, Jeffrey Bennett, Achim Berthele, Jérôme de Sèze, Michael Levy, Ichiro Nakashima, Celia Oreja Guevara, Jacqueline Palace, Friedemann Paul, Carlo Pozzilli, Kerstin Allen, Yasmin Mashhoon, Marcus Yountz, and Ho Jin Kim

CHAMPION-NMOSD clinical trial: efficacy and safety of ravulizumab in NMOSD patients testing positive for AQP-4

Ravulizumab is a therapy for NMOSD that has recently (in 2024) been approved in the US, following the clinical trial called CHAMPION-NMOSD.  Ravulizumab is similar to eculizumab in its mechanism of action. This clinical trial study evaluated the safety and efficacy of ravulizumab in adult patients who tested positive for aquaporin-4 antibodies (AQP4+). In the trial, patients receiving ravulizumab showed a reduced risk of relapse and better disability scores than those receiving the placebo. While there were side effects, the overall safety of ravulizumab was similar to its documented safety profile in other conditions where it has been approved. Patients were followed up for  over 1.5 years on average, and the safety and efficacy results remained the same throughout this period.

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Ravulizumab in Aquaporin-4–Positive Neuromyelitis Optica Spectrum Disorder

Ravulizumab in Aquaporin-4–Positive Neuromyelitis Optica Spectrum Disorder

Journal: Annals of Neurology; March 3, 2023

Author(s): Sean J. Pittock, Michael Barnett, Jeffrey L. Bennett, Achim Berthele, Jérôme de Sèze, Michael Levy, Ichiro Nakashima, Celia Oreja-Guevara, Jacqueline Palace, Friedemann Paul, Carlo Pozzilli, Marcus Yountz, Kerstin Allen, Yasmin Mashhoon, Ho Jin Kim

Ravulizumab in NMOSD patients testing positive for AQP4

Ravulizumab is a therapy for NMOSD that has recently (in 2024) been approved in the US, following the clinical trial called CHAMPION-NMOSD.  This paper, which reviews data from the clinical trial, shows the safety and efficacy of ravulizumab in adult NMOSD patients who tested positive for aquaporin-4 antibodies (AQP4+). Ravulizumab was found to significantly reduce the risk of relapse in patients with AQP4+ NMOSD, with similar levels of safety as eculizumab for NMOSD and as ravulizumab in all other conditions where it has been approved.

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Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease: Current Insights into the Disease Pathophysiology, Diagnosis and Management

Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease: Current Insights into the Disease Pathophysiology, Diagnosis and Management

Journal: International Journal of Molecular Sciences; December 24, 2020

Author(s): Wojciech Ambrosius, Sławomir Michalak, Wojciech Kozubski, and Alicja Kalinowska

Overview of MOGAD: Disease behavior, diagnosis, and management

This review presents the current known information regarding MOGAD. is often misdiagnosed as MS or NMOSD but is considered a distinct condition. The most common initial symptom of MOGAD seems to be optic neuritis in adults and inflammation and demyelination of the brain and spinal cord in children. In adults, the disease tends to have relapses, which gradually increase disability. In children, demyelination is usually a one-time incident. Acute treatment directed at the immune system seems to be effective in preventing relapses and limiting disability. The detection of MOG antibodies with a highly specific and sensitive diagnostic test helps in differentiating MOGAD from MS and NMOSD. While there are no clinical trials and approved treatments for MOGAD, high-dose steroids and plasma exchange seem to be helpful for the treatment of acute attacks, whereas immunosuppressive therapies, such as steroids, oral immunosuppressants, and rituximab seem to be effective as maintenance treatment.

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MOG Antibodies Restricted to CSF in Children With Inflammatory CNS Disorders

MOG Antibodies Restricted to CSF in Children With Inflammatory CNS Disorders

Journal: Neurology; March 6, 2024

Author(s): Gemma Olivé-Cirera, Arlette L. Bruijstens, Elianet G. Fonseca, Li-Wen Chen, Eva Caballero, Eugenia Martinez-Hernandez, Mar Guasp, Maria Sepúlveda, Laura Naranjo, Raquel Ruiz-García, Yolanda Blanco, Albert Saiz, Josep O. Dalmau, and Thaís Armangue for the Spanish Pediatric MOGAD Study Group

What does detection of MOG antibodies only in cerebrospinal fluid mean in children with inflammatory disorders of the central nervous system?

In children with inflammatory disorders of the central nervous system (CNS), MOG antibodies may be detected only in the cerebrospinal fluid (CSF), only in the blood serum, or in both CSF and blood serum. In this study, serum and CSF were tested for MOG antibodies in groups of children with various types of neurological disorders— (a) demyelinating syndromes, including acute disseminated encephalomyelitis (ADEM); (b) non-ADEM encephalitis; and (c) noninflammatory neurologic disorders. The results showed that MOG antibodies may be detected in children with CNS inflammatory disorders. MOG antibody detection only in the CSF and not in the serum is more likely to be indicative of multiple sclerosis.

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A Comparative Review of Typical and Atypical Optic Neuritis: Advancements in Treatments, Diagnostics, and Prognosis

A Comparative Review of Typical and Atypical Optic Neuritis: Advancements in Treatments, Diagnostics, and Prognosis

Journal: Cureus; March 13, 2024

Author(s): Noah J. Spillers, Patrick M. Luther, Norris C. Talbot, Evan J. Kidder, Connor A. Doyle, Salim C. Lutfallah, Alyssa G. Derouen, Sridhar Tirumala, Shahab Ahmadzadeh, Sahar Shekoohi, Alan D. Kaye, Giustino Varrassi

Optic neuritis — different forms, treatments, diagnosis, and prognosis

Optic neuritis can lead to partial or total permanent vision loss if left untreated. Accurate diagnosis and prompt treatment are essential to prevent long-term eye damage. Optic neuritis is a common symptom of MS, NMOSD, or MOGAD. This review helps physicians evaluate optic neuritis on the basis of MRI and visual tests as a to differentiate between MS, NMOSD, and MOGAD.

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Why autoimmune disease is more common in women: X chromosome holds clues

Why autoimmune disease is more common in women: X chromosome holds clues

Journal: Nature; February 1, 2024

Author(s): Elie Dolgin

Why are women more likely than men to have autoimmune diseases?

Autoimmune diseases are up to 4 times more common in women than men, but there has been no evidence-based explanation for this until now, although several theories exist. New research on mice shows that this increased female association may be linked to a molecule called Xist found on X chromosomes. This molecule generates specific proteins when two X chromosomes are present, as in women, and some of these proteins seem to be linked to autoimmune diseases. When the Xist molecule was engineered into the DNA of male mice, these mice began developing signs of autoimmune disease almost as severe as in women. If these findings translate well to human studies, it could lead to new and better ways to diagnose and treat autoimmune diseases.

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Impact of neuromyelitis optica spectrum disorder on employment and income in the United States

Impact of neuromyelitis optica spectrum disorder on employment and income in the United States

Journal: Annals of Clinical and Translational Neurology; February 20, 2024

Author(s): Isabella Gomez Hjerthen, Cristina Trápaga Hacker, William Meador, Ahmed Z. Obeidat, Lucas Horta, Farrah J. Mateen

Impact of NMOSD on employment and income in the US

In this study, working-age NMOSD patients participated in a survey designed to understand factors like loss of jobs, income, and work hours associated with this disease in the US.

Of 127 participants (mostly female and Caucasian; average diagnosis age 38.7 years, average disease duration 6.4 years), among whom 94% were under immunotherapy, 56% lost a job due to NMOSD. While 80% were employed before diagnosis, only 68% were after diagnosis. 36% of participants said they no longer worked outside the home, and 68% of those who were employed before diagnosis had to reduce their work hours by about 18 hours per month after. The growth in average annual income was lower for patients than the estimated average in the US. 60% of participants had a regular unpaid caregiver; 34% of caregivers changed their work hours or job to help manage NMOSD.

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Worldwide Incidence and Prevalence of Neuromyelitis Optica: A Systematic Review

Worldwide Incidence and Prevalence of Neuromyelitis Optica: A Systematic Review

Journal: Neurology; December 11, 2020

Author(s): Viktoria Papp, Melinda Magyari, Orhan Aktas, Thomas Berger, Simon A Broadley, Philippe Cabre, Anu Jacob, Jun-Ichi Kira, Maria Isabel Leite, Romain Marignier, Katsuichi Miyamoto, Jacqueline Palace, Albert Saiz, Maria Sepulveda, Olafur Sveinsson, Zsolt Illes

How common is NMOSD around the world?

This study reviewed 33 published papers on NMOSD epidemiology (how a disease occurs in different groups and why) to determine the worldwide prevalence (number of people with a disease at a time), incidence (frequency of occurrence), and basic demographic characteristics of NMOSD and provide a critical overview of the published studies. The results showed that NMOSD is a rare disease worldwide. The incidence and prevalence were highest in the Afro-Caribbean region and the lowest in Australia and New Zealand; and highest in African ethnicity, lowest in White ethnicity. NMOSD occurred most commonly in Middle-aged adults and more in females than males.

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