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Neuromyelitis optica spectrum disorder (NMOSD): Treatment and prognosis

Neuromyelitis optica spectrum disorder (NMOSD): Treatment and prognosis
Literature review current through: Jan 2024.
This topic last updated: Jul 13, 2023.

INTRODUCTION — Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory disorder of the central nervous system characterized by severe, immune-mediated demyelination and axonal damage predominantly targeting optic nerves and the spinal cord.

The treatment and prognosis of NMOSD will be reviewed here. The epidemiology, pathogenesis, clinical manifestations, diagnosis of NMOSD are reviewed separately. (See "Neuromyelitis optica spectrum disorder (NMOSD): Clinical features and diagnosis".)

RATIONALE — The rationale for treatment of acute and recurrent attacks in NMOSD is based upon evidence that humoral autoimmunity plays a role in the pathogenesis of NMOSD and is driven by the high attack-related disability, poor prognosis, and overall high risk of mortality in untreated patients [1,2]. Thus, long-term immunotherapy is indicated for the prevention of attacks as soon as the diagnosis of NMOSD is made. (See 'Attack prevention' below.)

ACUTE ATTACKS

Definition — An attack of NMOSD is defined by objective neurologic worsening reflecting focal or multifocal central nervous system inflammation that develops acutely or subacutely in the absence of fever or infection, with a duration of at least 24 hours and occurring more than 30 days after the previous attack [3]. This definition is similar to the definition of a multiple sclerosis (MS) relapse.

Acute treatment — All patients with suspected NMOSD require treatment for acute attacks.

Glucocorticoid treatment — We suggest initial treatment with high-dose intravenous methylprednisolone (1 gram daily for three to five consecutive days), in agreement with expert panel recommendations and based upon studies of MS and idiopathic optic neuritis [1,4,5]. (See "Treatment of acute exacerbations of multiple sclerosis in adults", section on 'Initial therapy with glucocorticoids' and "Optic neuritis: Prognosis and treatment", section on 'Treatment'.)

Response rates – Observational evidence suggests that the response to initial treatment of an NMOSD attack is often suboptimal and that most patients will benefit from a second therapy. In a retrospective report of 871 attacks in 185 patients with NMOSD, the first treatment course (consisting of high-dose intravenous glucocorticoid in 84 percent of patients, plasma exchange in 8 percent, and other treatments in the remainder) was associated with complete remission in only 19 percent of patients and a partial remission or no remission in 65 and 16 percent, respectively [6]. Additional treatment courses, particularly a second treatment course, was associated with an increased remission rate. Plasma exchange was used more frequently in second (34 percent) and subsequent (50 percent or more) treatment courses.

Seronegative NMOSD – Seronegative NMOSD is managed in the same way as seropositive NMOSD.

Adjunctive plasma exchange — For patients with severe symptoms or vision loss that is poorly responsive to glucocorticoids, therapeutic plasma exchange is the suggested concomitant or rescue treatment [1,4,5]. Exchanges are carried out every other day up to a total of seven exchanges.

Limited retrospective and uncontrolled data suggest that patients with severe attacks of NMOSD do better if plasma exchange is started early as adjunctive therapy with glucocorticoids. One report of patients with severe attacks (defined by an Expanded Disability Status Scale (table 1) score ≥4 and/or visual acuity ≤20/200) found that initial treatment with intravenous glucocorticoids plus early therapeutic plasma exchange was associated with improved outcomes compared with delayed initiation of plasma exchange following glucocorticoid treatment [7].

Intravenous immune globulin has not been specifically evaluated for acute attacks of NMOSD and is rarely used in this setting.

ATTACK PREVENTION

Approach — Because the natural history of NMOSD is one of stepwise deterioration due recurrent attacks and accumulated disability, long-term immunotherapy is indicated for the prevention of attacks as soon as the diagnosis of NMOSD is made [1,5,8].

For patients with NMOSD who are seropositive for aquaporin-4 (AQP4)-immunoglobulin G (IgG) antibodies, we suggest treatment with eculizumab, inebilizumab, satralizumab, ravulizumab, or rituximab, rather than other immunosuppressive agents. However, the optimal drug regimen and treatment duration are yet to be determined.

Eculizumab, inebilizumab, and satralizumab are approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of AQP4-antibody positive NMOSD. In mid-2023, the EMA also approved ravulizumab for the treatment of adults with AQP4-seropositive NMOSD [9]. All these agents are very expensive; many clinicians use rituximab, tocilizumab, or other off-label immunotherapies for patients who meet NMOSD diagnostic criteria with or without AQP4-IgG seropositivity.

Immunotherapies

Eculizumab — Eculizumab is a humanized antibody that binds to the complement component C5 and inhibits the formation of C5b-induced membrane attack complex.

DoseEculizumab is administered intravenously at 900 mg weekly for the first four doses, followed by maintenance dosing of 1200 mg every two weeks beginning at week 5.

Adverse effectsEculizumab infusions are generally well tolerated. Commonly reported adverse events in clinical trials included headache, upper respiratory tract infections, back pain, and nausea. However, eculizumab treatment is associated with an increased risk of infection with Neisseria meningitidis; patients should be immunized with meningococcal vaccines and receive daily antimicrobial prophylaxis, as described separately.

In the United States, eculizumab is available only through a restricted program under a Risk Evaluation and Mitigation Strategy. (See "Treatment and prevention of meningococcal infection", section on 'Patients receiving C5 inhibitors'.)

Efficacy – High-quality data support the effectiveness of eculizumab. In the PREVENT randomized controlled trial of 143 patients with NMOSD who were seropositive for AQP4-IgG antibodies, treatment with intravenous eculizumab reduced the risk of relapse; the annualized relapse rates for the eculizumab and placebo groups were 0.02 and 0.35 (absolute risk reduction [ARR] 33 percent, rate ratio 0.04, 95% CI 0.01-0.15) [10]. Most patients in the trial were also taking concomitant immunotherapy (eg, azathioprine, glucocorticoids, mycophenolate mofetil) but not rituximab, which was excluded because its mechanism of action is incompatible with eculizumab. Data from an open-label extension phase of the trial suggested that eculizumab treatment was associated with a sustained reduction of the relapse rate [11].

Inebilizumab — Inebilizumab is a humanized monoclonal antibody that binds to the CD19 surface antigen of B cells, thereby depleting a wide range of lymphocytes derived from B cell lineage, including peripheral blood CD20 plasmablasts and plasma cells [12].

Dose and administrationInebilizumab is given by intravenous infusion as an initial dose of 300 mg, followed two weeks later by a second 300 mg dose [13]. Thereafter, inebilizumab is given as 300 mg infusion every six months, starting from the first infusion. Patients should be premedicated with a glucocorticoid (eg, methylprednisolone 80 to 125 mg), an antihistamine (eg, diphenhydramine 25 to 50 mg), and an antipyretic (eg, acetaminophen 500 to 650 mg) prior to every inebilizumab infusion. Patients should be monitored for infusion reactions during and for at least one hour after the end of the infusion. Administration of inebilizumab should be delayed for patients with active infection until the infection resolves. Patients should be screened for hepatitis B virus and tuberculosis before the first dose. Immunoglobulin levels should be monitored before treatment and at regular intervals during and after treatment. Flow cytometric analysis with an anti-CD20 antibody should be used to determine that circulating B cells continue to be depleted just before the next infusion.

Adverse reactions – The most common adverse reactions in the randomized trial were urinary tract infection, headache, arthralgia, nausea, and back pain [14].

Contraindications to inebilizumab are active hepatitis B infection, active or untreated latent tuberculosis, and previous life-threatening reaction to inebilizumab infusion [13]. Use during pregnancy is not advised because of potential harm to the fetus or newborn baby. Vaccination with live or live-attenuated vaccines is not recommended during or after treatment until B cell repletion.

EfficacyInebilizumab was evaluated in the N-MOmentum trial of 230 patients with NMOSD (with 92 percent seropositive for anti-AQP4 antibodies) who were randomly assigned in a 3:1 ratio to intravenous treatment with inebilizumab 300 mg (n = 175) or to placebo (n = 56) administered on days 1 and 15 [14]. The trial was stopped early after an interim analysis at 6.5 months determined efficacy as defined by fewer attacks among patients assigned to inebilizumab compared with those assigned to placebo (12 versus 39 percent, ARR 27 percent, hazard ratio [HR] 0.27, 95% CI 0.15-0.50). The rate of all adverse events and serious adverse events was similar between the inebilizumab and placebo groups. Limitations of this trial include the short duration of clinical follow-up and early stopping, which can lead to an overestimate of the treatment effect. Based upon these data, inebilizumab was approved by the FDA in June 2020 for the treatment of adults with NMOSD who are seropositive for anti-AQP4 antibodies [15]. However, further study is needed to establish the long-term efficacy and safety of inebilizumab relative to eculizumab, which has also shown efficacy in NMOSD.

Satralizumab — Satralizumab is a humanized monoclonal antibody that binds interleukin-6 (IL-6) receptors, thereby suppressing inflammation mediated by IL-6 signaling pathways.

DoseSatralizumab is given by subcutaneous injection with a loading dose of 120 mg at weeks 0, 2, and 4, followed by a maintenance dose of 120 mg every four weeks [16]. Screening for hepatitis B virus and tuberculosis are required before the first dose. Active infection should be excluded prior to every dose; treatment should be delayed for patients with active infection until resolved. Liver transaminase levels (ALT and AST) should be checked prior to first dose, and ALT, AST, and neutrophil counts should be monitored during treatment. Live or live-attenuated vaccines are not recommended during treatment.

Adverse effects – The most common adverse effects with satralizumab are nasopharyngitis, headache, upper respiratory tract infection, gastritis, rash, arthralgia, extremity pain, fatigue, and nausea. Satralizumab is contraindicated for patients with active hepatitis B infection, active or untreated latent tuberculosis, or known hypersensitivity to satralizumab.

Efficacy – Efficacy of satralizumab was demonstrated in two randomized controlled trials [17,18]. In the first trial, 83 patients with NMOSD were randomly assigned to subcutaneous injection with satralizumab 120 mg or placebo at weeks 0, 2, and 4, and every four weeks thereafter, in addition to their baseline immunosuppressant therapy [17]. At a median blinded treatment duration of 107 weeks, there were fewer relapses among patients assigned to satralizumab compared with those assigned to placebo (20 versus 43 percent, ARR 23 percent, HR 0.38, 95% CI 0.16-0.88). In the subgroup of 55 patients who were seropositive for anti-AQP4 antibodies, relapses were less frequent among patients assigned to satralizumab compared with placebo (11 versus 43 percent, ARR 32 percent, HR 0.21, 95% CI 0.06-0.75). In the subgroup of 28 patients who were seronegative for anti-AQP4 antibodies, the number of relapses was similar among patients assigned to satralizumab compared with placebo (36 versus 43 percent, ARR 7 percent, HR 0.66, 95% CI 0.2-2.24). For the entire cohort, the rate of all adverse events and serious adverse events was similar between the satralizumab and placebo groups.

The second trial of 95 patients with NMOSD was similar in design to the first, except that concomitant immunosuppressant therapy was not allowed [18]. Satralizumab monotherapy reduced the rate of relapse (30 percent, versus 50 percent for placebo, ARR 20 percent, HR 0.45, 95% CI 0.23-0.89) and was generally well tolerated.

Ravulizumab — Ravulizumab is a humanized antibody that binds to the same complement component C5 epitope as eculizumab, thereby inhibiting the formation of C5b-induced membrane attack complex. Ravulizumab has a longer half-life than eculizumab, which permits less frequent maintenance administration for ravulizumab (every eight weeks) compared with eculizumab (every two weeks) [19].

Dose – For patients with NMOSD and no prior treatment, ravulizumab is given intravenously with a weight-based loading dose of 2400 to 3600 mg on day one, followed by a weight-based maintenance dose of 3000 to 3600 mg once every eight weeks, starting two weeks after the loading dose [20].

Patients should be immunized with meningococcal vaccines at least two weeks prior to administering the first dose of ravulizumab.

Adverse effects – Meningococcal infections occurred in two patients (3 percent) treated with ravulizumab in the CHAMPION-NMOSD trial despite prior vaccination against Neisseria meningitidis; other common adverse events included COVID-19 in 24 percent and headache in 24 percent [20]. There were no deaths.

Efficacy – The open-label CHAMPION-NMOSD trial enrolled adult patients with AQP4-antibody positive NMOSD (n = 58) who were treated with ravulizumab [20]. A concurrent placebo control group was considered unethical given the availability of eculizumab. Therefore, a historical placebo control group (n = 47) from the eculizumab PREVENT trial was used as the comparator group. The median follow-up period for the ravulizumab and external control groups was 73.5 and 36.0 weeks, respectively. No patients in the ravulizumab group had an adjudicated on-trial relapse, compared with 20 patients in the historical placebo group (hazard ratio 0.014, 95% CI 0.000-0.103). Although cross-trial comparisons represent low-quality evidence and can be misleading, the risk reduction observed with ravulizumab in the CHAMPION-NMOSD trial was consistent with that observed with eculizumab in the PREVENT trial [20].

Rituximab — Rituximab is a chimeric monoclonal antibody that causes B cell depletion by binding to the CD20 antigen of B cell lymphocytes and to Fc receptors.

DoseRituximab is started with two 1 g infusions separated by a two-week interval, followed by a 1 g infusion every six months or earlier if CD19+ lymphocytes are >0.1 percent of total lymphocytes [21].

Adverse reactions – Potential adverse effects include infusion reactions, hypogammaglobulinemia, infection, reactivation of hepatitis B, and neutropenia.

Efficacy – In an open-label randomized trial of 86 patients who had NMOSD with or without AQP4 antibodies, the annualized relapse rate at 12 months was significantly reduced for patients assigned to rituximab compared with those assigned to azathioprine [22].

Evidence of efficacy is also supported by results from a small trial in Japan that randomly assigned 38 patients (36 women and 2 men) with AQP4 antibody-positive NMOSD in a 1:1 ratio to treatment with rituximab or placebo [23]. All the patients in the trial had relatively mild disease and were on concomitant oral glucocorticoids throughout the trial. Intravenous rituximab was loaded at 375 mg/m2 every week for four weeks, followed by a maintenance phase of rituximab 2000 mg (given as two 1000 mg doses separated by two weeks) at 24 and 48 weeks after randomization. At a median follow-up of 72 weeks, there were no relapses in patients assigned to rituximab and seven relapses in patients assigned to placebo (0 versus 37 percent, group difference, 36.8 percent, 95% CI 12.3-65.5 percent). One patient assigned to rituximab had an infusion reaction, but adverse events were otherwise similar in the two treatment groups. The small size of this trial prevents definitive conclusions, and additional research is needed to confirm safety and efficacy of rituximab for NMOSD.

One small observational study of patients with NMOSD who were seropositive for AQP4 or myelin oligodendrocyte glycoprotein antibodies found that rituximab treatment was associated with a lower risk of relapse compared with mycophenolate mofetil [24], and rituximab treatment was associated with a lower relapse rate compared with azathioprine in a small observational study of patients with NMOSD [25]. A systematic review of observational studies found that rituximab was associated with a reduced frequency of relapses and reduced neurologic disability in patients with NMOSD, but also noted concerns about the safety profile of rituximab [26].

Tocilizumab — Tocilizumab is a humanized IL-6 receptor antagonist.

Dose – The dose of tocilizumab is 8 mg/kg (maximum 800 mg) given intravenously every four weeks [27]. Interruption of dosing may be needed for laboratory abnormalities such as elevated liver enzymes, neutropenia, or thrombocytopenia.

Adverse effectsTocilizumab may cause dyslipidemia but is generally well tolerated. The most common adverse effects are upper respiratory infection, headache, hypertension, elevated alanine aminotransferase, and injection site reactions. Other adverse effects include neutropenia, thrombocytopenia, and serious infections, including mycobacterial and other opportunistic infections. Intestinal perforations have been reported, especially in older patients and in those with a history of diverticulitis, which is a contraindication to the use of tocilizumab.

Efficacy – In an open-label trial of 118 patients with NMOSD, intravenous tocilizumab (8 mg/kg every four weeks) was superior to azathioprine for reducing the relapse rate [27]. Tocilizumab treatment has also been associated with clinical stabilization or improvement in a small number of patients with refractory NMOSD who failed one or more of the "standard" immunosuppressive treatments (eg, glucocorticoids, mitoxantrone, rituximab, alemtuzumab) [28-32].

Other immunotherapies — The evidence of efficacy for systemic immunotherapy in preventing NMOSD attacks comes mainly from observational studies of agents including azathioprine [33,34], mycophenolate mofetil [35,36], methotrexate [37,38], mitoxantrone [39], and oral glucocorticoids [40]. Among these, the agents most often considered as effective treatments for NMOSD are azathioprine and mycophenolate mofetil [4,41,42].

One retrospective, nonrandomized study analyzed relapses among patients with NMOSD who were treated with azathioprine and concomitant prednisone (n = 32) for at least six months, or mycophenolate (n = 28) for at least six months, or rituximab (n = 30) for at least one month, and followed up after treatment for at least six months [43]. Treatment with these agents was associated with significant reductions of 72 to 88 percent in annualized relapse rates compared with baseline. As an example, the annualized relapse rate decreased from 2.26 before azathioprine treatment to 0.63 after treatment, a reduction of 72 percent. Treatment failure, defined as the development of any new central nervous system inflammatory event, varied from 33 to 53 percent. The retrospective design of these studies limits the strength of the findings.

In an observational study of five Chinese patients with highly active NMOSD that was poorly responsive to "standard" immunosuppressive agents, treatment with bortezomib (a proteosome inhibitor that depletes plasma cells) was associated with relapse-free status in four and clinical stabilization in all five [44].

Medications to avoid — Limited observational evidence suggests that treatment of NMOSD with interferon beta, natalizumab, or fingolimod is not effective and may be harmful [45-50]. There is no published evidence regarding the treatment of NMOSD with ocrelizumab.

Duration of therapy — Immunosuppression is usually continued for at least five years for patients who are seropositive for AQP4-IgG antibodies, including those presenting with a single attack, because they are at high risk for relapse or conversion to NMOSD [51]. However, many experts suggest that life-long therapy is appropriate, given the often-devastating nature of the disease.

PROGNOSIS

Natural history — The natural history of NMOSD is one of stepwise deterioration due to accumulating visual, motor, sensory, and bladder deficits from recurrent attacks. Most acute attacks or relapses worsen over days to a nadir and recover over several weeks to months with significant sequelae. There is no clear progressive phase, as occurs in multiple sclerosis. (See "Neuromyelitis optica spectrum disorder (NMOSD): Clinical features and diagnosis", section on 'Relapsing clinical course'.)

Although definitive proof is lacking, it is likely that treatment of acute attacks with glucocorticoids and/or plasma exchange and preventive treatment with immunotherapies has improved long-term outcomes of NMOSD [52].

Mortality — Mortality rates are high in NMOSD, most frequently secondary to neurogenic respiratory failure, which occurs with extension of cervical lesions into the brainstem or from primary brainstem lesions [1]. Cohort studies published from 2003 to 2009 of North American, Brazilian, and French West Indies populations reported mortality rates of 32 percent, 50 percent, and 25 percent, respectively, in NMOSD [53-55]. These studies may be biased towards more severe cases. Progress in the diagnosis and treatment of NMOSD is expected to decrease mortality rates.

Prognostic markers — Predictors of a worse prognosis include the number of relapses within the first two years, the severity of the first attack, older age at disease onset, and (perhaps) an association with other autoimmune disorders including autoantibody status [55-58]. These prognostic factors need to be confirmed in larger independent prospective studies.

The aquaporin-4 (AQP4) autoantibody (NMO-immunoglobulin G [IgG]) may be a marker for disease course and prognosis [59-61], though the available data are inconsistent [62]. In patients with recurrent optic neuritis, retrospective evidence suggests that AQP4-IgG seropositivity is associated with poor visual outcome and development of NMOSD [60]. A prospective study of 29 patients presenting with longitudinally extensive spinal cord lesions found 55 percent of the patients seropositive for AQP4-IgG relapsed within one year or converted to NMOSD, while none of the seronegative patients relapsed [61]. By contrast, a subsequent report noted that seronegative and seropositive NMOSD were similar in terms of relapse rate, severity, and long-term outcomes [62]. The discrepancy in these results may be due to differences in the determination of antibodies (cell-based versus enzyme-linked immunosorbent assays) and perhaps intermixing of patients with AQP4-seropositive NMOSD and patients with seronegative NMOSD who would now be identified as seropositive for myelin oligodendrocyte glycoprotein-IgG antibody, as well as with patients who have antibodies not yet identified.

NMOSD in pregnancy — There are only limited and retrospective data on the relationship of NMOSD and pregnancy. These suggest that NMOSD is associated with an increased risk of miscarriage [63] and that the annualized relapse rate of NMOSD is increased in the first three to six months of the postpartum period [64-67].

SOCIETY GUIDELINE LINKS — Links to society and government-sponsored guidelines from selected countries and regions around the world are provided separately. (See "Society guideline links: Multiple sclerosis and related disorders".)

SUMMARY AND RECOMMENDATIONS

Treatment of acute attacks

Glucocorticoid therapy – For patients with acute or recurrent attacks of NMOSD, we suggest initial treatment with high-dose intravenous methylprednisolone (1 gram daily for three to five consecutive days) (Grade 2C), with a low threshold for rapid initiation of plasma exchange. (See 'Glucocorticoid treatment' above.)

Adjunctive plasma exchange – For patients with severe symptoms or poorly responsive to glucocorticoids, we suggest treatment with plasma exchange (Grade 2C). Plasma exchange may be more effective if started early as adjunctive therapy with glucocorticoids. (See 'Adjunctive plasma exchange' above.)

Indication for preventive therapy – For patients with seropositive NMOSD, we recommend long-term immunotherapy (eg, eculizumab, inebilizumab, satralizumab, ravulizumab, or rituximab) (Grade 1B) to reduce the risk of relapse as soon as the diagnosis of NMOSD is made. For patients with seronegative NMOSD, we suggest long-term immunotherapy (eg, eculizumab, inebilizumab, satralizumab, or rituximab) (Grade 2C). (See 'Approach' above.)

Choice of agent – For patients with NMOSD who are seropositive for aquaporin-4 (AQP4)-immunoglobulin G (IgG) antibodies, we suggest treatment with eculizumab, inebilizumab, satralizumab, or ravulizumab, where available, rather than other immunosuppressive agents (Grade 2C). Several immunotherapies (eculizumab, inebilizumab, and satralizumab) are approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and ravulizumab is approved by the EMA. Many clinicians also use alternative immunotherapies (rituximab, tocilizumab) for patients who meet NMOSD diagnostic criteria with or without AQP4-IgG seropositivity. (See 'Attack prevention' above.)

Duration of preventive immunotherapy – Immunosuppression is usually continued for at least five years for patients who are seropositive for AQP4-IgG, including those presenting with a single attack, because they are at high risk for relapse. However, the optimal drug regimen and treatment duration for NMOSD, with or without AQP4 seropositivity, are yet to be determined, and many authorities suggest lifetime treatment. (See 'Attack prevention' above.)

Prognosis – The natural history of NMOSD is one of stepwise deterioration due to accumulating visual, motor, sensory, and bladder deficits from recurrent attacks. Long-term disability and mortality rates are high. Although unproven, it is likely that acute and preventive treatment with immunotherapies has improved the long-term outcomes of NMOSD. (See 'Prognosis' above.)

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Topic 134223 Version 5.0

References

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