Experts publish recommendations for modern NMOSD treatment

Updated guidelines offered after approval of 3 drugs to treat disease

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A person looks at two different treatment options.

An international team of experts has published evidence-based recommendations for how to best treat people with neuromyelitis optica spectrum disorder (NMOSD) who are positive for aquaporin-4 immunoglobulin G (AQP4-IgG), the most common type of disease-driving antibody.

The consensus statements were described in the study “International Delphi Consensus on the Management of AQP4-IgG+ NMOSD,” published in Neurology Neuroimmunology & Neuroinflammation.

NMOSD is an autoimmune disease driven by an abnormal immune response that causes damaging inflammation on healthy parts of the brain and spinal cord. In most patients, this autoimmune attack is driven by self-targeting antibodies against a protein called AQP4.

Until recently, there were no approved therapies to treat NMOSD, and the disease was generally managed with off-label therapies that reduce inflammation.

In 2019, Soliris (eculizumab) became the first therapy to be approved for AQP4-IgG-positive NMOSD in the U.S. Two other therapies, Uplizna (inebilizumab-cdon) and Enspryng (satralizumab-mwge), have since also been approved for the same indication.

Soliris is given via into-the-vein (intravenous) infusions every two weeks, Uplizna is administered by infusion twice yearly, and Enspryng is given through under-the-skin (subcutaneous) injections once a month.

These three medications differ in terms of their mechanism of action, though all of them broadly work to reduce inflammatory disease activity by suppressing the immune system.

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Panel of experts develop consensus statements to treat NMODS

Based on a review of the scientific literature and their own experience, an international panel of experts devised a set of consensus statements to help direct clinicians treating NMOSD in this new treatment landscape.

“The statements offer practical recommendations from experts related to the treatment of patients with AQP4-IgG–seropositive NMSOD,” they wrote.

The study was funded by Roche, which sells Enspryng through its subsidiary Chugai Pharmaceutical.

The panel agreed that all three approved therapies may be offered to adults who are newly diagnosed with NMOSD or who have had a disease relapse despite other treatments.

The decision on which specific therapy to offer depends on the expected efficacy and safety in each individual situation, as well as patient preferences about different administration routes and schedules.

The recommendations suggest patients experiencing a relapse while on treatment with one of these three therapies or an off-label treatment should be started on an approved therapy with a different mechanism of action.

The experts agreed that, when switching between the three approved NMOSD therapies, a new therapy can be started immediately after discontinuing an old one, after accounting for the time each therapy is active in the body.

Due to the risk of safety issues, the panel recommended each of the three approved therapies should be given alone, without any other NMOSD treatment. The experts also agreed that in some patients who are already on off-label immunosuppressive treatments, new approved therapies can be added before the older therapy is tapered off, though extra monitoring is recommended.

The group also recommended Enspryng should be the first therapy offered to adolescent patients, since this is the only therapy that has been tested in those as young as 12 in clinical trials.

The other two therapies may be given for adolescents who don’t respond to Enspryng, but clinical data for these therapies in such young patients are lacking, they noted.

Due to their immunosuppressive effects, all three approved therapies can increase the risk of infections. The panel agreed patients on any of these therapies should be regularly monitored for infections, and additional monitoring may be needed for adolescent and elderly patients or those who have other health conditions that increase infection risk.

Additional monitoring also may be recommended for pregnant patients; the experts noted that more research is needed to evaluate the safety of these therapies during pregnancy.

Staying up to date on vaccinations recommended

The panel also recommended patients should be up to date on all vaccinations before starting any of the approved NMOSD treatments. The experts noted, however, that there’s a need for clarification of official guidelines on infection-preventing measures in patients starting on these therapies, such as the optimal timing for certain vaccines and whether preventive antibiotic treatment should be given.

The experts agreed there is a need for standardized, validated measures that can be used to assess patient-reported quality of life in clinical trials, as well as more research into biological markers to measure NMOSD disease activity.

The panel also stressed the importance of collecting real-world and long-term data as the new therapies become more widely available, as well as further research to compare outcomes among the various treatments.

“More research is needed to improve individualized treatment strategy further,” the experts wrote.