We are in a new era of treating patients with NMOSD

Professor, NMOSD is a disease of the nervous system, somewhat resembling multiple sclerosis in its course and sometimes confused with it.

It's true. NMOSD (the acronym comes from the English name "neuromyelitis optica spectrum disorder," meaning a group of diseases with optic neuritis and spinal cord inflammation) is, like multiple sclerosis, an autoimmune disease of the nervous system, with a course of so-called "throws," or exacerbations, and periods of remission. However, the flares are usually more severe than in multiple sclerosis, and remission is not complete - meaning that after each flare, some symptoms remain, leading to increasing disability. The main culprit of symptoms in NMOSD is not demyelination, but inflammation leading to the death of cells in the nervous system, usually involving the optic nerves (unilaterally - or bilaterally) and the spinal cord - usually several of its segments. Characteristic symptoms of NMOSD resulting from spinal neuritis include paresis or paralysis of the lower extremities or both lower and upper extremities, increased muscle tone, sensory disturbances, severe pain in the extremities, paroxysmal painful muscle cramps and urinary and bowel disturbances. Typical symptoms resulting from optic neuritis, meanwhile, include eyeball pain and visual disturbances (ranging from visual fatigue and deterioration of visual acuity to total blindness). NMOSD can also involve a structure called the brainstem, the part of the nervous system between the medulla and the brain, and hence the symptoms of this disease can be quite non-obvious, such as persistent hiccups, nausea, vomiting, dizziness, facial pain, facial nerve palsy, hearing disorders, swallowing disorders or balance disorders.

NMOSD and MS differ not only in some of their symptoms and prognosis, but most importantly - in their mechanism of onset and response to treatment. Underlying NMOSD is a malfunction of the immune system, which produces antibodies against the body's own protein called aquaporin 4, located mainly in the optic nerves and spinal cord. As a result of the antibody attack on aquaporin 4, inflammation develops in these areas of nerve tissue and the cells that make up this tissue die. Since NMOSD is a disease with a dynamic, aggressive course, it is necessary to make a diagnosis as soon as possible, followed by the implementation of appropriate treatment as soon as possible.

How is NMOSD diagnosed?

To diagnose NMOSD, it is necessary to perform comprehensive diagnostic tests, usually carried out in a hospital setting: specialized blood tests (for the presence of antibodies to aquaporin 4), magnetic resonance imaging and cerebrospinal fluid examination. The results of these tests make it possible to distinguish between NMOSD and multiple sclerosis. This is extremely important, as some of the drugs used to treat multiple sclerosis may not only fail to help NMOSD, but may even worsen the course of the disease and the patients' condition.

As of November 2022, patients diagnosed with NMOSD can be treated in the B.138.FM drug program. What does this treatment consist of?

The treatment offered in the drug program is a chronic immunosuppressive treatment, called a disease-modifying treatment, which is designed to halt the progression of the disease and prevent further relapses. This is very important because, as I mentioned earlier, after each flare, some neurological deficit remains, leading to increasing disability. This immunotherapy therefore inhibits the development of disability. Patients included in the drug program receive therapy with an innovative drug - satralizumab. The drug belongs to a modern group of monoclonal antibodies, and works by inhibiting the inflammatory process and preventing neuronal death by affecting the cytokine system. This means that the drug strikes directly at the cause of the disease. Satralizumab is a drug administered subcutaneously, once every four weeks. It can be used as the only drug to modify the course of NMOSD or in combination treatment with other immunosuppressive therapy (such as oral corticosteroids, azathioprine or mycophenolate mofetil). The favorable safety profile and efficacy of satralizumab in a group of patients with NMOSD, with aquaporin 4 antibodies present, was confirmed by the results of the SAkuraSky and SAkuraSTAR clinical trials. Thanks to the provisions of the B.138.FM program, we have the opportunity in Poland to treat patients with seropositive NMOSD (i.e., with the presence of antibodies to aquaporin 4) in accordance with current medical knowledge and expert recommendations, including the recommendations of the expert panel of the Multiple Sclerosis and Neuroimmunology Section of the Polish Neurological Society in 2023 and the German group of scientists and clinicians who have been working on NMOSD (NEMOS) for years in 2024.

What are the eligibility criteria for chronic NMOSD treatment?

According to the provisions of the drug program B.138.FM, patients who meet all of the following criteria are eligible for satralizumab treatment: age over 12 years; diagnosis of NMOSD, based on current diagnostic criteria including confirmation of the presence of anti-equaporin 4 (anti-AQP4) antibodies in plasma; neurological status assessed using the EDSS scale of 0 to 6.5 points; no contraindications to the drug, as defined in the Summary of Product Characteristics; no previous treatment with another drug from the same group as satralizumab.

For patients who are of childbearing age, contraception is recommended. However, in pregnant patients, the inclusion of satralizumab treatment is at the discretion of the physician. Patients who have discontinued, due to pregnancy, satralizumab treatment under the drug program are automatically included in it, without the need for re-qualification, once the pregnancy has ended.

Eligibility of patients for treatment with satralizumab in the drug program is carried out by the Coordinating Team for the Treatment of Patients with NMOSD Spectrum, based on the application prepared by the neurologist caring for the patient in question and sent to the Team.

What is the effectiveness of satralizumab treatment?

The efficacy of satralizumab is supported by solid scientific evidence: the results of the SAkuraSky and SAkuraSTAR clinical trials, which showed a long-term and significant reduction in the frequency of episodes in patients with anti-AQP4 antibodies undergoing this therapy, as well as a favorable safety profile, and our observations from clinical practice. The Coordination Team has already qualified more than 130 patients nationwide for treatment in the drug program. We see that this is a drug that can prevent permanent disability, which is crucial in NMOSD. Previously used immunotherapy often failed to protect patients from episodes and patients were at risk of disability progression. It is important that in Poland there is an opportunity to chronically treat patients with NMOSD with such an effective and safe therapy as satralizumab.

Why is it so important that satralizumab treatment continues to be reimbursed for Polish patients?

The B.138.FM drug program is crucial for patients with NMOSD because it offers a treatment that goes beyond the previous therapeutic standard, effectively halting the progression of the disease. With the introduction of satralizumab, patients now have access to a treatment that directly hits the mechanism of the disease, resulting in effective prevention of further flares and limiting the accumulation of neurological deficits.

The drug program enables systematic chronic treatment of patients, which is extremely important because NMOSD is a disease with a very aggressive course. Incorporating therapy as soon as possible after diagnosis makes it possible to minimize the negative effects of flare-ups, and thus protect patients from increasing disability.

The continuation of this program is justified because there is currently no alternative with comparable efficacy and safety based on solid scientific evidence for patients with NMOSD.

Source: press mat.