Neuromielitis optica
Zapobieganie i profilaktyka

Neuromielitis optica (NMO) to autoimmunologiczna choroba demielinizacyjna ośrodkowego układu nerwowego, charakteryzująca się preferencyjnym zajęciem nerwów wzrokowych i rdzenia kręgowego. Ryzyko nawrotów przekracza 90%, co wymaga długoterminowej immunoterapii, zwłaszcza u pacjentów z obecnością przeciwciał AQP4-IgG, u których leczenie zapobiegawcze powinno być wdrożone już po pierwszym ataku. FDA zatwierdziła od 2019 roku cztery leki: ekulizumab (zmniejsza ryzyko nawrotu o 94%), rawulizumab (98,6%), inebilizumab (73%) oraz satralizumab (55%). Wcześniej stosowano azatioprynę (2,5 mg/kg/dzień), rytuksymab (redukcja nawrotów o 88%), mykofenolan mofetylu (87,4%) oraz metotreksat i mitoksantron, których stosowanie ogranicza się ze względu na toksyczność i brak danych. Terapia powinna trwać minimum 2-3 lata bez nawrotów, a decyzja o jej zakończeniu wymaga indywidualnej oceny ryzyka nawrotów i toksyczności leków.

Profilaktyka i zapobieganie nawrotom w neuromielitis optica

Neuromielitis optica (NMO), znana również jako choroba Devica, jest autoimmunologiczną chorobą demielinizacyjną ośrodkowego układu nerwowego, która preferencyjnie zajmuje nerwy wzrokowe i rdzeń kręgowy. Większość pacjentów z NMO doświadcza nawracających rzutów, które mogą prowadzić do poważnego i trwałego upośledzenia funkcji neurologicznych. Z tego powodu zapobieganie nawrotom jest kluczowym elementem terapii tej choroby.12

Znaczenie profilaktyki w NMO

W NMO prawdopodobieństwo nawrotu aktywności choroby wynosi ponad 90%, co podkreśla konieczność stosowania długoterminowego leczenia immunosupresyjnego. Wczesna identyfikacja i rozpoczęcie leczenia są niezwykle istotne w zarządzaniu chorobą. Zapobieganie nawrotom jest priorytetem terapeutycznym, ponieważ każdy atak może pozostawić pacjenta trwale niepełnosprawnym i pogorszyć jakość życia.123

U pacjentów z przeciwciałami przeciwko akwaporynie-4 (AQP4-IgG) zaleca się rozpoczęcie długoterminowej immunoterapii już po pierwszym ataku. Takich pacjentów uznaje się za zagrożonych nawrotem bezterminowo i należy rozważyć leczenie zapobiegawcze, nawet u osób z długotrwałą remisją kliniczną.12

Leki stosowane w profilaktyce NMO

Leczenie zapobiegawcze w NMO koncentruje się na szeregu leków immunosupresyjnych. Chociaż żaden z nich nie został zwalidowany w rygorystycznym randomizowanym badaniu, liczne badania retrospektywne i kilka ostatnich badań prospektywnych dostarczyły dowodów na skuteczność kilku leków w zapobieganiu zaostrzeniom NMO.12

Zatwierdzone leki immunosupresyjne

Od 2019 roku FDA zatwierdziła trzy leki do leczenia pacjentów z NMO z dodatnim wynikiem przeciwciał anty-AQP4:12

  • Ekulizumab (Soliris) – przeciwciało monoklonalne, które hamuje układ dopełniacza. Badania wykazały, że zmniejsza ryzyko nawrotu o około 94%.12
  • Inebilizumab (Uplizna) – zmniejsza ryzyko nawrotu o około 73% w porównaniu z osobami z NMOSD nieprzyjmującymi terapii.12
  • Satralizumab (Enspryng) – zmniejsza ryzyko nawrotu o około 55% w porównaniu z osobami z NMOSD nieprzyjmującymi terapii.12
  • Rawulizumab (Ultomiris) – najnowszy zatwierdzony lek, który zmniejsza ryzyko nawrotu o około 98,6%.12

Leki stosowane poza wskazaniami rejestracyjnymi

Przed zatwierdzeniem wyżej wymienionych leków, najczęściej stosowanymi preparatami w zapobieganiu nawrotom NMO były:12

  • Azatiopryna (Imuran, Azasan) – inhibitor syntezy DNA, jest przekształcany w analog purynowy zakłócający syntezę puryn (adeniny i guaniny). Hamuje proliferację komórek, szczególnie limfocytów. Typowa dawka to 2,5 mg/kg/dzień.12
  • Rytuksymab (Rituxan) – chimeryczne przeciwciało monoklonalne przeciwko białku CD20, które znajduje się głównie na powierzchni limfocytów B. Zmniejsza częstość nawrotów o 88%, przy czym 2/3 pacjentów osiąga całkowitą remisję podczas 10-letniego okresu badania.12
  • Mykofenolan mofetylu (CellCept) – prolek aktywnego metabolitu kwasu mykofenolowego, hamuje proliferację limfocytów poprzez inhibicję biosyntezy nukleotydów guanozynowych. Zmniejsza częstość nawrotów o 87,4% z 36% współczynnikiem niepowodzenia.12
  • Metotreksat (Trexall, Xatmep) – hamuje enzymy zależne od folianów i zakłóca syntezę puryn i tymidylanu.12
  • Mitoksantron – ze względu na potencjalną toksyczność, jego stosowanie powinno być ograniczone do przypadków opornych na inne leczenie.12
  • Prednizonkortykoidy w małych dawkach stosowane są częściej w innych częściach świata.12

Oprócz wyżej wymienionych leków, w leczeniu zapobiegawczym NMO stosowane są również:1

Zalecenia dotyczące terapii pierwszego rzutu

Zgodnie z najnowszymi zaleceniami, długoterminową immunoterapię u pacjentów z NMO z dodatnim wynikiem na obecność przeciwciał AQP4-IgG należy rozpocząć od jednego z przeciwciał monoklonalnych: ekulizumabu/rawulizumabu, inebilizumabu, rytuksymabu lub satralizumabu, jeśli są dostępne.1

Ze względu na większą dostępność danych klinicznych, azatiopryna, mykofenolan mofetylu i rytuksymab są najczęściej zalecanymi terapiami pierwszego rzutu w profilaktyce NMO. Z uwagi na potencjalną toksyczność mitoksantronu i ograniczone dane kliniczne dotyczące metotreksatu i cyklosporyny, lekarze powinni rozważyć ograniczenie ich stosowania do przypadków opornych na standardowe leczenie.12

Czas trwania leczenia zapobiegawczego

Nie ma konsensusu co do czasu trwania terapii zapobiegawczej w NMO, ale ogólnie za minimum uważa się 2-3 lata okresu bez nawrotów, biorąc pod uwagę ryzyko długoterminowej toksyczności tych leków. Decyzja o zaprzestaniu leczenia musi być oparta na starannej rozmowie między pacjentem a lekarzem prowadzącym, uwzględniającej historię nawrotów (częstość, nasilenie, powrót do zdrowia), toksyczność leczenia (faktyczną lub potencjalną), czas trwania leczenia i inne czynniki motywujące (np. chęć zajścia w ciążę).12

Szczepienia a profilaktyka NMO

Odpowiednie szczepienia są bezpieczne i skuteczne u większości pacjentów i mogą zapobiegać infekcjom oraz zmniejszać ryzyko nawrotów. Praktyki mające na celu zminimalizowanie ryzyka nawrotu związanego ze szczepionką obejmują krótkotrwałe, niskodiaskowe leczenie doustnymi steroidami na kilka dni przed szczepieniem oraz doustną dawkę leku przeciwhistaminowego w dniu szczepienia.1

U pacjentów z aktywną chorobą NMOSD, rozpoczęcie terapii nie powinno być odkładane z powodu niekompletnego statusu szczepień. Szczepienia powinny być aktualizowane zgodnie z krajowymi zaleceniami i standardami tak szybko, jak to możliwe.1

Profilaktyka NMO u kobiet w ciąży

Immunosupresja indukowana ciążą może wpływać na choroby autoimmunologiczne, takie jak NMO. Badania wykazują, że około 20-47% kobiet rozwija pierwsze objawy NMO podczas ciąży lub w ciągu roku po porodzie.1

Długoterminowa immunoterapia nie powinna być przerywana ani odkładana ze względu na chęć zajścia w ciążę. Przeciwciała monoklonalne (ekulizumab/rawulizumab, rytuksymab, tocilizumab) lub azatiopryna powinny być kontynuowane w trakcie ciąży.1

Przed zajściem w ciążę należy przerwać stosowanie:12

  • Mykofenolanu mofetylu (MMF) – 6 tygodni przed poczęciem
  • Metotreksatu (MTX) – 3 miesiące przed poczęciem
  • Cyklofosfamidu – 3 miesiące przed poczęciem

Ze względu na zwiększone ryzyko nawrotu po porodzie, szybkie wprowadzenie terapii profilaktycznej jest uzasadnione. Najbezpieczniejszymi opcjami leczenia podczas ciąży wydają się być azatiopryna i rytuksymab.12

Identyfikacja czynników wyzwalających nawroty

Identyfikacja potencjalnych czynników wyzwalających nawroty NMO jest obszarem trwających badań. Zrozumienie tych czynników, które mogą obejmować infekcje i stres, może pomóc pacjentom podjąć proaktywne kroki w celu zmniejszenia ryzyka nawrotu.1

Dobra higiena i mycie rąk są ważne, jeśli pacjent przyjmuje leki immunosupresyjne, podobnie jak posiadanie dobrego urologa w przypadku ryzyka infekcji dróg moczowych. Przewlekła immunosupresja wymaga regularnych badań skóry u dermatologa, ponieważ układ odpornościowy jest naszą najlepszą obroną przed rozwijającymi się komórkami nowotworowymi, a każde z tych leczenia może zakłócać jego normalne funkcjonowanie.1

Podsumowanie zaleceń profilaktycznych

NMO to przewlekła choroba, która wymaga długoterminowego zarządzania. Pacjenci potrzebują ścisłego monitorowania przez personel medyczny w celu dostosowania planów leczenia i zminimalizowania aktywności choroby oraz progresji niepełnosprawności.1

Chociaż NMO nie można wyleczyć, wczesna interwencja i leczenie mogą zatrzymać postęp choroby, zapobiec niepełnosprawności i nawrotom. Celem jest utrzymanie lub poprawa jakości życia pacjenta.1

Ważne jest, aby podkreślić, że niektóre leki immunomodulujące stosowane w leczeniu stwardnienia rozsianego (MS), takie jak interferon-β, fingolimod, natalizumab i alemtuzumab, nasilają progresję choroby NMO i nie powinny być stosowane w jej leczeniu.1

Kolejne rozdziały

Zapraszamy do dalszego czytania naszego leksykonu.

Wybierz kolejny rozdział z menu poniżej, aby otworzyć nową podstronę kompedium wiedzy i uzyskać szczegółowe informację o leku, substancji lub chorobie.

  1. 12.04.2026
  2. www.leksykon.com.pl

Materiały źródłowe

  • #1 Treatment of Neuromyelitis Optica: Review and Recommendations
    https://pmc.ncbi.nlm.nih.gov/articles/PMC3926208/
    Neuromyelitis optica (NMO) is an autoimmune demyelinating disease preferentially targeting the optic nerves and spinal cord. Preventive therapy in NMO has focused on a range of immunosuppressive medications, none of which have been validated in a rigorous randomized trial. However, multiple retrospective and a few recent prospective studies have provided evidence for the use of six medications for the prevention of NMO exacerbations: azathioprine, rituximab, mycophenolate mofetil, prednisone, methotrexate and mitoxantrone. […] We recommend preventive therapy with one of the six immunosuppressive regimens that have multiple studies reporting efficacy in NMO – azathioprine, rituximab, mycophenolate mofetil, methotrexate, prednisone and mitoxantrone. […] The duration of preventive treatment in NMO that is needed has not been adequately studied.
  • #1 NMOSD | Neuromyelitis Optica Spectrum Disorder | Prognosis & Management | SRNA
    https://wearesrna.org/living-with-myelitis/disease-information/neuromyelitis-optica-spectrum-disorder/prognosis-management/
    In NMOSD, the likelihood of recurrence of disease activity is greater than 90%. […] Therefore, it is generally thought that ongoing treatment with medications that suppress the immune system and prevent relapses is necessary. […] Good hygiene and hand washing are important if on immunosuppressants, as is having a good urologist if at risk for UTIs. […] Chronic immunosuppression requires regular skin exams with a dermatologist since our immune system is our best defense against cancer cells developing, and any of these treatments can interfere with its normal functioning. […] Mycophenolate mofetil and azathioprine are both twice daily pills which broadly suppress the immune system. […] Eculizumab reduced the risk of relapse by roughly 94%. […] Ravulizumab reduced the risk of relapse by roughly 98.6%.
  • #1
    https://link.springer.com/article/10.1007/s00415-023-11910-z
    Before 2019, recommendations on the long-term treatment of NMOSD, including ours, were mainly based on retrospective studies, case series, open-label trials, and expert opinions without any approved immunotherapies. […] Four therapies, eculizumab, inebilizumab, and satralizumab and most recently ravulizumab have been approved for use in AQP4-IgG-positive NMOSD since 2019. […] Long-term immunotherapy must be offered to patients with AQP4-IgG-positive NMOSD already after the first attack. […] Eculizumab/ravulizumab, inebilizumab, rituximab, satralizumab and tocilizumab are highly effective therapies for AQP4-IgG-positive NMOSD. […] Long-term immunotherapy in AQP4-IgG-positive NMOSD should be initiated with one of the monoclonal antibodies eculizumab/ravulizumab, inebilizumab, rituximab, or satralizumab, whenever those are available and accessible.
  • #1 Neuromyelitis optica spectrum disorder – Wikipedia
    https://en.wikipedia.org/wiki/Neuromyelitis_optica_spectrum_disorder
    Prophylactic treatment, to prevent relapses of NMO, is generally employed; but the exact duration of such treatment is debatable. […] FDA-approved pharmaceuticals against AQP4-IgG-positive NMOSD, shown to be effective in phase III clinical trials, first became available in 2019. […] These new drugs’ effectiveness against AQP4-IgG-negative NMOSD is unknown. […] Many treatments are used despite the lack of phase III clinical trials testing their efficacy. […] It is important to note that certain immunosuppressants used to treat MS such as interferon-, fingolimod, natalizumab, and alemtuzumab worsen NMO disease progression and should not be used to treat NMO.
  • #1 NMOSD | Neuromyelitis Optica Spectrum Disorder | Prognosis & Management | SRNA
    https://wearesrna.org/living-with-myelitis/disease-information/neuromyelitis-optica-spectrum-disorder/prognosis-management/
    Inebilizumab reduced the risk of relapse by roughly 73% compared to individuals with NMOSD not on therapy. […] Satralizumab reduced the risk of relapse by roughly 55% compared to individuals with NMOSD not on therapy. […] Azathioprine is contraindicated in pregnancy, so pregnancy planning is very important. […] Mycophenolate is also contraindicated in pregnancy, so, again, planning is imperative. […] Rituximab is safer in pregnancy than the other two previously described, […] Planned pregnancy is still recommended. […] Low-dose prednisone is used as well, more often in other parts of the world.
  • #1 Neuromyelitis optica spectrum disorders: from pathophysiology to therapeutic strategies | Journal of Neuroinflammation | Full Text
    https://jneuroinflammation.biomedcentral.com/articles/10.1186/s12974-021-02249-1
    Currently, long-term NMOSD relapse prevention includes off-label use of immunosuppressants, particularly rituximab. […] Long-term relapse prevention includes treatments based on data from retrospective observations, and prospective observational studies without control groups. The most commonly used treatments include azathioprine (AZA), mycophenolate (MMF), and rituximab. […] To minimize permanent neurologic disability, long-term relapse prevention treatment is recommended for all patients who are diagnosed with NMOSD. […] Off label use of some older immunosuppressive agents such as AZA, MMF, and rituximab have shown reductions in ARR, with disability stabilization in retrospective, prospective, and meta-analysis studies. […] Eculizumab was the first monoclonal approved by the Food and Drug Administration to prevent NMOSD relapses in AQP4-ab-positive patients in June 2019, followed by Inebilizumab (June 2020) and satralizumab (August 2020). […] Long-term impact of recently developed drugs remains to be established.
  • #1 SciELO Brazil – Treatment of neuromyelitis optica: an evidence based review Treatment of neuromyelitis optica: an evidence based review
    https://www.scielo.br/j/anp/a/rdSpmcLbFzPkCPK4cMfmZsw/
    Therefore, it is crucial that treatment is started as early as possible to avoid new relapses and further disability. […] The Guidelines on the use of therapeutic apheresis in clinical practice from the American Society for Apheresis published in 2010 considered that PE for NMO is accepted as second line therapy, as it may be helpful in recovery from acute attacks, although it does not prevent further relapses. […] Azathioprine is a DNA synthesis inhibitor, as it is converted to a purine analogue, with interference in the purines synthesis (adenine and guanine). It inhibits the proliferation of cells, especially lymphocytes. […] Rituximab is a chimeric monoclonal antibody against the protein CD20, which is primarily found on the surface of B cells and it has the property to eliminate B cells selectively. […] The use of corticosteroids and other immunosuppressant drugs for NMO treatment has been based on case reports or small series of cases rather than in double-blinded randomized studies.
  • #1 Treatment of neuromyelitis optica: state-of-the-art and emerging therapies
    https://pmc.ncbi.nlm.nih.gov/articles/PMC4229040/
    Due to the high morbidity associated with NMO exacerbations, immunosuppressive therapy is typically instituted after the first attack. […] Current preventative therapies are used to deplete immune cell populations, diminish circulating AQP4-IgG, or interfere with immune cell proliferation or activation. […] In addition to their use in the treatment of acute exacerbations, oral corticosteroids and plasma exchange can be used on a chronic basis for prophylactic therapy. […] Regular plasma exchange treatment in association with additional immunosuppression has also shown benefit in reducing relapse activity. […] Azathioprine is converted to nucleotide antimetabolites that interfere with lymphocyte proliferation. […] Mycophenolate mofetil, a prodrug of the active metabolite mycophenolic acid, suppresses lymphocyte proliferation by inhibiting guanosine nucleotide biosynthesis.
  • #1 Treatment of neuromyelitis optica: state-of-the-art and emerging therapies
    https://pmc.ncbi.nlm.nih.gov/articles/PMC4229040/
    Methotrexate inhibits folate-dependent enzymes and interferes with purine and thymidylate synthesis. […] Combination therapy with cytotoxic, immunomodulatory, and B-cell-depleting therapies is a fundamental approach to the treatment of many autoimmune disorders such as rheumatoid arthritis. […] Although no prospective clinical trials have been conducted in NMO, retrospective and prospective case series have been used to develop a framework to guide treatment decisions. […] Owing to the more-extensive availability of clinical data, azathioprine, mycophenolate mofetil and rituximab tend to be the most-recommended first-line therapies for NMO prophylaxis. […] Due to the potential toxicity of mitoxantrone and the limited clinical data on methotrexate and cyclosporine, physicians should consider restricting their use to refractory cases. […] Given the increased relapse rate following delivery, rapid introduction of prophylactic therapy could be warranted; however, the benefits of these therapies should be balanced against the benefits of breastfeeding.
  • #1 Neuromyelitis optica – Diagnosis and treatment – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/neuromyelitis-optica/diagnosis-treatment/drc-20375655
    Neuromyelitis optica can’t be cured. But treatment can sometimes lead to a long-term period with no symptoms, known as remission. NMO treatment involves therapies to reverse recent symptoms and prevent future attacks. […] Your healthcare professional might recommend that you take a lower dose of corticosteroids over time to prevent future NMO attacks and relapses. […] Monoclonal antibodies have been shown in clinical trials to be effective in reducing the risk of NMO relapses. These medicines include eculizumab (Soliris), satralizumab (Enspryng), inebilizumab (Uplizna), ravulizumab (Ultomiris) and rituximab (Rituxan). Many of these have been approved by the U.S. Food and Drug Administration (FDA) for prevention of relapses in adults. […] Your healthcare professional also might recommend taking a medicine that suppresses the immune system. This might include azathioprine (Imuran, Azasan), mycophenolate (Cellcept, Myhibbin), methotrexate (Trexall, Xatmep, others), cyclophosphamide or tocilizumab (Actemra). […] Intravenous immunoglobulins, also known as antibodies, may decrease the relapse rate of NMO.
  • #1
    https://journals.lww.com/annalsofian/fulltext/2015/18001/therapy_of_nmo_spectrum_disorders.5.aspx
    Patients who have the risk of relapse should receive long-term immunosuppression following treatment of an acute attack. […] Immunumodulators used as disease-modifying agent in MS have not been found to be effective in NMO. […] The duration of preventive treatment in NMO has not been adequately studied. […] While deciding to stop treatment, the clinician must discuss the matter with the patient taking into account the duration of treatment, history of relapse (frequency, severity, and recovery), treatment toxicity (actual or potential), and other factors (e. g., a woman’s desire to become pregnant). […] Generally oral steroid is continued after the treatment of an acute attack, then its dose is slowly reduced once the steroid-sparing immunosuppressive therapy becomes effective, thereby reducing the risks of long-term side effects of steroid.
  • #1 Neuromyelitis Optica Spectrum Disorder and COVID-19 FAQsmail2
    https://guthyjacksonfoundation.org/nmosd-covid-19-faqs/
    Appropriate vaccination is safe and effective in most patients and can prevent infection and reduce risks of relapse. Practices to minimize any remote risk of vaccine-related relapse are available and involve a short, low-dose oral steroid taper a few days before vaccination and an oral anti-histamine dose on the day of vaccination. Please consult your healthcare team to develop an immunization plan that is right for you. […] The best way to lower risks of secondary infection is to prevent primary infection due to the SARS-CoV-2 virus or other preventable infections through appropriate vaccination and exposure control practices.
  • #1
    https://link.springer.com/article/10.1007/s00415-023-11910-z
    Long-term immunotherapy should not be discontinued or postponed for the desire to become pregnant. Monoclonal antibodies (eculizumab/ravulizumab, rituximab, tocilizumab) or azathioprine should be continued during pregnancy. […] In patients with active NMOSD, therapy initiation must not be postponed due to incomplete vaccination status. Vaccinations should be updated according to national recommendations and standards as soon as possible.
  • #1 Neuromyelitis Optica – EyeWiki
    https://eyewiki.org/Neuromyelitis_Optica
    Pregnancy-induced immunosuppression can influence autoimmune conditions like NMO. Studies show around 20%47% of women develop their first symptoms of NMO during pregnancy or within a year postpartum. […] Prophylactic management: Continue immunosuppression to reduce the risk of postpartum relapse. Azathioprine (2.5mg/kg/day) and rituximab are safe treatment options. Discontinue the following before conception: Mycophenolate mofetil (MMF- 6 weeks prior), methotrexate (MTX), and cyclophosphamide (3 months prior). […] To conclude, special consideration is needed for teratogenic effects and the stage of pregnancy, while treating a pregnant patient with NMO.
  • #1
    https://journals.lww.com/annalsofian/fulltext/2015/18001/therapy_of_nmo_spectrum_disorders.5.aspx
    The NMO exacerbation does not increases in pregnancy, but increases in the postpartum period and in the year following childbirth. […] Owing to increased relapse rate following delivery, rapid introduction of prophylactic therapy is warranted. […] Rituximab is also preferred for treatment failure after initial attempts with agents such as mycophenolate mofetil, azathioprine, and methotrexate.
  • #1 Is there a Cure for Neuromyelitis Optica?
    https://www.dragarwal.com/blog/eye-wellness/is-there-a-cure-for-neuromyelitis-optica/
    Identifying potential triggers for NMO relapses is an ongoing area of research. Understanding these triggers, which can include infections and stress, may help patients take proactive steps to reduce their risk of relapse. […] NMO is often considered a lifelong condition, and long-term management is essential. Patients need close monitoring by healthcare providers to adjust treatment plans as needed and to minimise disease activity and disability progression.
  • #1 Neuromyelitis Optica (NMO) Treatment | Rush
    https://www.rush.edu/services/neuromyelitis-optica-nmo-services
    Treatment is most effective when it begins as early as possible. Early intervention can slow or stop progression of the disease and prevent disabilities. […] Their long-term goal is to prevent NMO attacks or relapses and give you the best quality of life possible. […] NMO is not a curable condition. But early intervention and treatment can stop progression of the disease, prevent disability and prevent relapses. The goal is to maintain or improve your quality of life.
  • #2
    https://journals.lww.com/annalsofian/fulltext/2015/18001/therapy_of_nmo_spectrum_disorders.5.aspx
    Neuromyelitis optica (NMO) is an autoimmune demyelinating condition of the central nervous system often associated with aquaporin-4 (AQP4) autoantibodies manifesting as severe optic neuritis and long segment myelitis with tendency to relapse. […] For relapse prevention, immunosuppressive agents that have been found to be effective are azathioprine, rituximab, mycophenolate mofetil, methotrexate, and mitoxantrone; although none of which have been validated in randomized, controlled trial. […] There is no consensus about the duration of preventive therapy, but generally 2-3 years of relapse-free period is considered the minimum, taking into account the risks of long-term toxicity of these agents. […] All these suggest that the most effective therapeutic option in NMO is immunosuppressive rather than immunomodulatory drugs and prevention of relapse is a therapeutic priority.
  • #2 Neuromyelitis Optica – EyeWiki
    https://eyewiki.org/Neuromyelitis_Optica
    Initial treatment for patients with suspected NMO or patients with acute attacks is intravenous glucocorticoids. Prevention of recurrent attacks is treated with long-term immunosuppression. AQP4-IgG seropositive patients are assumed to be at risk for relapse indefinitely and preventive treatment should be considered, even in patients with prolonged clinical remission. […] Guidelines for NMO management have been difficult to establish, since most studies involve a small number of patients. Treatment for acute episodes consists mainly of steroids (methylprednisolone 500-1000mg daily for 5-10 days) followed by plasmapheresis or intravenous immunoglobulin. Eculizumab and inebilizumab are humanized antibodies that have been studied in randomized controlled trials in patients with NMOSD and have shown efficacy in long term treatment. Other immunotherapies have also been used for long term management of NMOSD such as azathioprine, mycophenolate mofetil, rituximab, methotrexate, mitoxantrone, tocilizumab, and oral glucocorticoids.
  • #2 Treatment of neuromyelitis optica: state-of-the-art and emerging therapies
    https://pmc.ncbi.nlm.nih.gov/articles/PMC4229040/
    Methotrexate inhibits folate-dependent enzymes and interferes with purine and thymidylate synthesis. […] Combination therapy with cytotoxic, immunomodulatory, and B-cell-depleting therapies is a fundamental approach to the treatment of many autoimmune disorders such as rheumatoid arthritis. […] Although no prospective clinical trials have been conducted in NMO, retrospective and prospective case series have been used to develop a framework to guide treatment decisions. […] Owing to the more-extensive availability of clinical data, azathioprine, mycophenolate mofetil and rituximab tend to be the most-recommended first-line therapies for NMO prophylaxis. […] Due to the potential toxicity of mitoxantrone and the limited clinical data on methotrexate and cyclosporine, physicians should consider restricting their use to refractory cases. […] Given the increased relapse rate following delivery, rapid introduction of prophylactic therapy could be warranted; however, the benefits of these therapies should be balanced against the benefits of breastfeeding.
  • #2 What Is the Life Expectancy of Someone With NMO? NMO vs. MS
    https://www.medicinenet.com/what_is_the_life_expectancy_of_someone_with_nmo/article.htm
    More than 90% of NMO patients have recurring attacks which are generally severe, so prevention of relapse is an integral part of NMO treatment. Preventive medications are primarily immunosuppressants that lower immune activity in NMO patients. […] In 2019 and 2020, the FDA approved three medications to treat anti-APQ4 positive NMO. These medications are lab-engineered human antibodies that specifically target the anti-APQ4 antibodies in NMO, which include: Eculizumab (Soliris), Inebilizumab (Uplizna), Satralizumab-mwge (Enspryng). […] Immunosuppressants used off-label to treat NMO include: Azathioprine (Imuran, Azasan), Mycophenolate mofetil (CellCept), Rituximab (Rituxan).
  • #2 Neuromyelitis optica – Diagnosis and treatment – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/neuromyelitis-optica/diagnosis-treatment/drc-20375655
    Neuromyelitis optica can’t be cured. But treatment can sometimes lead to a long-term period with no symptoms, known as remission. NMO treatment involves therapies to reverse recent symptoms and prevent future attacks. […] Your healthcare professional might recommend that you take a lower dose of corticosteroids over time to prevent future NMO attacks and relapses. […] Monoclonal antibodies have been shown in clinical trials to be effective in reducing the risk of NMO relapses. These medicines include eculizumab (Soliris), satralizumab (Enspryng), inebilizumab (Uplizna), ravulizumab (Ultomiris) and rituximab (Rituxan). Many of these have been approved by the U.S. Food and Drug Administration (FDA) for prevention of relapses in adults. […] Your healthcare professional also might recommend taking a medicine that suppresses the immune system. This might include azathioprine (Imuran, Azasan), mycophenolate (Cellcept, Myhibbin), methotrexate (Trexall, Xatmep, others), cyclophosphamide or tocilizumab (Actemra). […] Intravenous immunoglobulins, also known as antibodies, may decrease the relapse rate of NMO.
  • #2
    https://link.springer.com/article/10.1007/s00415-023-11910-z
    Before 2019, recommendations on the long-term treatment of NMOSD, including ours, were mainly based on retrospective studies, case series, open-label trials, and expert opinions without any approved immunotherapies. […] Four therapies, eculizumab, inebilizumab, and satralizumab and most recently ravulizumab have been approved for use in AQP4-IgG-positive NMOSD since 2019. […] Long-term immunotherapy must be offered to patients with AQP4-IgG-positive NMOSD already after the first attack. […] Eculizumab/ravulizumab, inebilizumab, rituximab, satralizumab and tocilizumab are highly effective therapies for AQP4-IgG-positive NMOSD. […] Long-term immunotherapy in AQP4-IgG-positive NMOSD should be initiated with one of the monoclonal antibodies eculizumab/ravulizumab, inebilizumab, rituximab, or satralizumab, whenever those are available and accessible.
  • #2 Neuromyelitis Optica Spectrum Disorders
    https://practicalneurology.com/diseases-diagnoses/ms-immune-disorders/neuromyelitis-optica-spectrum-disorders/30129/
    We recommend prompt initiation of long-term immunosuppression once a diagnosis of NMOSD is made. No randomized clinical trial investigating preventative therapy in NMOSD has had final results published to date, therefore treatment is based on open-label prospective and retrospective studies. Given the available data, we prefer rituximab (RTX) for most patients with NMOSD. If there are contraindications to RTX, we typically use mycophenolate mofetil (MMF). […] Treatment with RTX decreased relapse rate by up to 88%, with 2 of 3 patients achieving complete remission during a 10-year study period, whereas MMF reduced relapse rates by 87.4% with a 36% failure rate and AZA reduced relapse rates by 72% with a 53% failure rate even when used concurrently with prednisone. […] Adequate RTX dose is important in order to maintain remission. Although the optimal dosing regimen has yet to be determined, we agree with dosing protocols tailored to an individual’s rate of immune reconstitution.
  • #2 Diagnosis and Treatment of Neuromyelitis Optica – touchNEUROLOGY
    https://touchneurology.com/multiple-sclerosis/journal-articles/diagnosis-and-treatment-of-neuromyelitis-optica/
    Neuromyelitis optica (NMO), an autoimmune inflammatory disease of the central nervous system (CNS), is characterized by severe attacks of optic neuritis and myelitis. […] For long-term relapse prevention, immunosuppressive drugs such as azathioprine, mycophenolate mofetil, rituximab, and mitoxantrone are recommended rather than the immunomodulatory agents used for MS. […] Following an attack of NMO, high-dose intravenous methylprednisolone should be started as soon as possible. Based on a randomized controlled trial that included patients with NMO and acute transverse myelitis, and other non-randomized experience, plasmapheresis is recommended to treat attacks that do not respond to intravenous steroids. […] For long-term relapse prevention, immunosuppressive drugs are recommended rather than the immunomodulatory agents used for MS patients. Azathioprine (2.5mg/kg/day) with oral corticosteroids (for example, prednisone beginning at 60mg/day or every other day for nine months, subsequently decreasing to the lowest possible maintenance dose or discontinuation) and mycophenolate mofetil (2g/day) with oral corticosteroids are recommended initial therapies.
  • #2 Diagnosis and Treatment of Neuromyelitis Optica – touchNEUROLOGY
    https://touchneurology.com/multiple-sclerosis/journal-articles/diagnosis-and-treatment-of-neuromyelitis-optica/
    For refractory cases, rituximab (1g intravenously twice separated by two weeks; repeated every six to nine months) and mitoxantrone (12mg/m2 every three months, with a maximum cumulative dose limited to 140mg/m2 primarily due to cardiotoxicity) have some evidence of efficacy in nonrandomized small series of NMO patients.
  • #2 Treatment of Neuromyelitis Optica: Review and Recommendations
    https://pmc.ncbi.nlm.nih.gov/articles/PMC3926208/
    For NMO or NMOSD patients with established relapsing disease, long term immunosuppression with the medications discussed below are recommended, and any decision to stop treatment must be based on a careful discussion between the patient and treating physician, taking into account past relapse history (frequency, severity, recovery), treatment toxicity (actual or potential), treatment duration, and external motivating factors (e.g., a woman’s desire to become pregnant). […] Based on relatively small retrospective and prospective case series, several treatments appear to be likely effective in preventing attacks and stabilizing disability in NMO patients. […] We recommend starting with one of four options for first-line monotherapy treatments for NMO: azathioprine, mycophenolate, rituximab, or prednisone, in doses and schedules according to Table 2.
  • #2 Treatment of Neuromyelitis Optica: Review and Recommendations
    https://pmc.ncbi.nlm.nih.gov/articles/PMC3926208/
    Neuromyelitis optica (NMO) is an autoimmune demyelinating disease preferentially targeting the optic nerves and spinal cord. Preventive therapy in NMO has focused on a range of immunosuppressive medications, none of which have been validated in a rigorous randomized trial. However, multiple retrospective and a few recent prospective studies have provided evidence for the use of six medications for the prevention of NMO exacerbations: azathioprine, rituximab, mycophenolate mofetil, prednisone, methotrexate and mitoxantrone. […] We recommend preventive therapy with one of the six immunosuppressive regimens that have multiple studies reporting efficacy in NMO – azathioprine, rituximab, mycophenolate mofetil, methotrexate, prednisone and mitoxantrone. […] The duration of preventive treatment in NMO that is needed has not been adequately studied.
  • #2 Neuromyelitis Optica Spectrum Disorders
    https://practicalneurology.com/diseases-diagnoses/ms-immune-disorders/neuromyelitis-optica-spectrum-disorders/30129/
    Because RTX can produce hypogammaglobulinemia which may be associated with significant infections, we monitor immunoglobulin levels prior to initiation and maintenance doses of RTX. […] For preventative therapy, methotrexate and cyclophosphamide are contraindicated during pregnancy, whereas AZA and RTX appear to have better safety profiles.
  • #2 Neuromyelitis Optica – EyeWiki
    https://eyewiki.org/Neuromyelitis_Optica
    Pregnancy-induced immunosuppression can influence autoimmune conditions like NMO. Studies show around 20%47% of women develop their first symptoms of NMO during pregnancy or within a year postpartum. […] Prophylactic management: Continue immunosuppression to reduce the risk of postpartum relapse. Azathioprine (2.5mg/kg/day) and rituximab are safe treatment options. Discontinue the following before conception: Mycophenolate mofetil (MMF- 6 weeks prior), methotrexate (MTX), and cyclophosphamide (3 months prior). […] To conclude, special consideration is needed for teratogenic effects and the stage of pregnancy, while treating a pregnant patient with NMO.
  • #3 Neuromyelitis optica spectrum disorders (NMOSD) – MS Australia
    https://www.msaustralia.org.au/nmosd-and-mogad/neuromyelitis-optica-spectrum-disorders/
    Early identification and treatment are very important in managing NMOSD. Treatment is usually broken into two different phases rescue therapy of the acute attack when it happens and relapse prevention, which is a more long-term approach. […] Relapse prevention usually involves a general immunosuppressive medication to prevent further attacks to the body and includes corticosteroids, azathioprine, mycophenolate, methotrexate, cyclophosphamide and rituximab. […] However, the approval of targeted treatments (similar to disease modifying treatments in MS) has significantly improved the management of NMOSD. On 1 April 2025, Ravulizumab (Ultomiris) was listed on the Pharmaceutical Benefits Scheme (PBS) for adults with NMOSD who are AQP4-antibody positive. This provides subsidised access to a highly effective treatment that can prevent relapses and reduce the severity of the disease. […] The inclusion of ravulizumab on the PBS represents a significant milestone for people living with NMOSD, ensuring broader access to a life-changing therapy that can help prevent relapses and improve quality of life.