Revised Recommendations Published to Improve Diagnosis and Treatment in NMOSD


Investigators identified practical guidelines for NMOSD management, emphasizing patient communication and targeted therapies, while providing recommendations for personalized approaches.

Achim Berthele, MD, associate professor, department of neurology, University Hospital rechts der Isar, Technical University of Munich

Achim Berthele, MD

The Neuromyelitis Optica Study Group (NEMOS) recently published practical recommendations for managing acute attacks and implementing preventive immunotherapies for patients with neuromyelitis optica spectrum disorders (NMOSD), a rare autoimmune disease.1 The review suggests that clinicians worldwide should communicate to patients and their caregivers about how to access available therapies as well as share their knowledge of the latest developments.

In the review, the group provided 3 core areas for clinical focus, including treating attacks, long-term therapy, and vaccinations. In the long-term therapy section of suggestions, authors centered on topics such as principal recommendations, off-label therapies, initiation, and selection criteria for aquaporin-4 immunoglobulin G antibodies (AQP4-IgG)-positive NMOSD, double-negative NMOSD, switching drugs, duration, as well as family planning and pregnancy.

Top Clinical Takeaways

  • Initiating attack therapy early in NMOSD is crucial, and a personalized approach to long-term immunotherapy, including monoclonal antibodies, is recommended for effective management.
  • The importance of equal and affordable access to NMOSD therapies and knowledge for patients and caregivers worldwide is emphasized in the revised manuscript.
  • Unmet needs in NMOSD therapy, including understanding the long-term disease course, optimal immunotherapy durations, and biomarkers for attack risk, underscore the importance of ongoing research.

“Recent insights into the pathogenesis of NMOSD have led to the development of novel targeted and highly effective therapies for patients with AQP4-IgG-positive NMOSD. These therapies provide a more personalized approach to treatment, considering factors, such as disease activity, age, comorbidities, family planning, side effects, route of administration, patient choice, availability, and costs,” senior author Achim Berthele, MD, associate professor, department of neurology, University Hospital rechts der Isar, Technical University of Munich, and colleagues wrote.1 “They also allow for switching between therapies in case of side effects or insufficient treatment response. However, long-term experience and therapy sequences, as well as general risk management for potential lifelong therapies, are still being developed. To gather these ‘real-world’ data, registries and platform trials should be established, and a standardized approach to data collection should be adopted.”

READ MORE: Vitamin D Deficiency Observed in Neuromyelitis Optica Spectrum Disorder

In the review, NEMOS conducted an overview of the current state of knowledge on treatments in NMOSD and provided statements as well as practical recommendations on the therapy management and use of all available immunotherapies for the disease. In addition, authors discussed unmet needs among patients with the disease and the pathogenesis of AQP4-IgG-negative NMOSD. The recommendations were developed by using a Delphi-based consensus method among the core author group and at expert discussions at NEMOS meetings.2

In the section on treating attacks, authors suggested initiating treatment as early as possible and consideration of an oral taper of glucocorticoids for up to 3 and 6 months to prevent subsequent attacks. Investigators suggested that long-term immunotherapy should be offered to patients with AQP4-IgG-positive NMOSD already after the first attack. Those who are stable on off-label therapies have no significant adverse effects; researchers noted that there is no need to be switched to different treatments. Additionally, long-term immunotherapy in AQP4-IgG-positive NMOSD should be initiated with a monoclonal antibody such as eculizumab (Soliris; Alexion)/ravulizumab (Ultomiris; AstraZeneca), inebilizumab (Uplizna; Horizon Therapeutics), rituximab, or satralizumab (Enspryng; Genentech), if available and accessible.

All told, the review concluded that long-term immunotherapy in double-negative NMOSD should be initiated after a second attack or after a severe first attack. If the treatment fails with classical immunosuppressive therapies, patients should switch to a monoclonal antibody, but if that fails, they should switch to a different one. Immunotherapy should be continued in stable patients with AQP4-IgG-positive NMOSD and requires closely monitored if treatment is temporarily or permanently discontinued because of adverse effects or patient decision.

Authors noted that patients of reproductive age with AQP4-IgG-positive NMOSD must be counseled early on regarding family planning options and the risks and benefits of both pregnancy and immunotherapies during pregnancy. In patients with active NMOSD, therapy initiation must not be postponed because of incomplete vaccination status and vaccinations should be updated according to national recommendations as soon as possible.

“Unmet needs in NMOSD therapy include understanding the long-term disease course, determining optimal immunotherapy durations, developing strategies for treatment cessation and de-escalation, and searching for biomarkers indicating attack risk,” Berthele et al noted.1 “Double-negative NMOSD, which affects a minority of patients, should also be addressed in international and collaborative research. Finally, NMOSD is a global disease, and patients and caregivers worldwide should have equal and affordable access to available therapies and therapeutic knowledge.”

1. Kümpfel T, Giglhuber K, Aktas O, et al. Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) - revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management. J Neurol. 2024;271(1):141-176. doi:10.1007/s00415-023-11910-z
2. Niederberger M, Spranger J. Delphi Technique in Health Sciences: A Map. Front Public Health. 2020;8:457. Published 2020 Sep 22. doi:10.3389/fpubh.2020.00457
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