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Challenging the Status Quo of Neuromyelitis Optica Spectrum Disorder Care

Twenty years ago, receiving a diagnosis of neuromyelitis optica spectrum disorder (NMOSD) or Devic’s disease was even rarer than it is today. This unpredictable autoimmune disease is still not well known and often misdiagnosed as multiple sclerosis (MS), because it also causes inflammation of the central nervous system, resulting in symptoms like pain, vision loss, paralysis and sensory loss. Individuals living with NMOSD may experience these symptoms as “attacks” or ”relapses,” which can cause permanent disability.[1]

When he first began his career, Dr. Robert Shin noticed that there was a lack of resources, education and treatment options for those living with NMOSD. However, in the last five years, treatment options have rapidly advanced with the FDA approval of medicines shown to reduce the risk of NMOSD attacks that can lead to permanent disability. That’s why Dr. Shin is challenging himself, his fellow health care professionals and NMOSD patients to learn about the approved treatments for NMOSD and determine which option may lead to the best patient outcomes.

To better understand the importance of early and active treatment of NMOSD, we interviewed Dr. Robert Shin, Professor of Neurology at MedStar Georgetown University Hospital.

Here’s what he had to say:

How has your experience diagnosing and treating NMOSD patients changed?

I remember a time when we debated whether NMOSD and MS were distinct disorders or if one was a variation of the other. Since then, a new diagnostic test has been developed that makes this distinction clearer. One of the main challenges with diagnosing NMOSD versus MS is that both conditions impact the spinal cord and brain. NMOSD can also present in a variety of ways, with those living with this condition showing different symptoms that may look unrelated, which is why it’s now called a spectrum disorder.

We were just diagnosing a small percentage of those living with NMOSD until a diagnostic test for NMOSD that measures the anti-aquaporin-4 (AQP4) antibody became available. Antibodies help your body identify foreign invaders, like bacteria and viruses. For individuals with autoimmune diseases like NMOSD, their antibodies may recognize some aspects of their bodies as “foreign” and attack themselves. In the case of NMOSD, the body is attacking specific areas of the optic nerve, brain stem and spinal cord. The AQP4 antibody is specific to NMOSD, with 75-80% of adults with NMOSD having cases that are AQP4 antibody positive.[2] People living with MS, however, are always negative for this blood test.

Now that we have this blood test available, we should be vigilant and I would argue we should test all patients who we suspect might have NMOSD.

Why is it important to identify NMOSD activity quickly?

While symptoms can be similar, unlike MS, NMOSD attacks can be severe and not often associated with full recovery. There is no overlap between MS and NMOSD therapies, so if we use certain MS medicines in treating those with NMOSD, they could potentially worsen the patient’s condition.

Now that we have treatments to help reduce the risk of attacks and possibly minimize the severity, getting the correct diagnosis early is critical to preventing irreversible damage. Time is of the essence.

I encourage patients to flag any new or worsening symptoms to their health care professionals so they can work together to determine if they have the right diagnosis and are being appropriately treated for their condition.

What has been your experience with FDA-approved treatments?

The idea of treating NMOSD with an FDA-approved medicine is very exciting because there’s data to validate the use of these therapies. When I first started learning about FDA-approved medicines, I found the UPLIZNA® (inebilizumab-cdon) trial compelling. UPLIZNA is a prescription medicine used to treat adults with NMOSD who are AQP4 antibody positive and works by depleting the B cells, which create the antibodies that can cause NMOSD.

Clinical trial results have shown the treatment is effective, as about 90% of patients were attack free after 28 weeks on UPLIZNA.[3] The study also showed that those taking UPLIZNA had side effects similar to placebo (meaning not on medicine) with the most common side effects being urinary tract infection and joint pain. These are not all the possible side effects of UPLIZNA. UPLIZNA is also the only approved standalone therapy for NMOSD with a convenient 90-minute, twice-yearly dosing after the two initial doses during the first four weeks of treatment, which means more treatment-free time for patients.

How have FDA-approved treatments changed the way you care for those with NMOSD?

The effect of the NMOSD-approved medicines is striking with sustained proven efficacy that makes me feel confident in these treatments. Before we used to say “NMOSD is devastating, attacks are severe and you don’t get a lot of recovery,” but if individuals receive accurate and timely diagnosis and get on these approved medicines, it’s much more manageable than it used to be. Those I treat with NMOSD are doing extremely well and if they do have relapses, I see relatively good recovery.

I feel we might need to rewrite the textbooks soon, as NMOSD might not be the completely disabling condition we previously thought. There’s a new sense of hope as long as we can recognize NMOSD early and get people on a highly effective therapy.

For those interested in learning more about the treatments for NMOSD, what resources are available?

For those living with or caring for someone with NMOSD, I recommend reviewing the resources from NMOSD advocacy groups like the Guthy-Jackson Charitable Foundation, The Siegel Rare Neuroimmune Association (SRNA) or The Sumaira Foundation for recent accurate information. There are also helpful resources specifically related to UPLIZNA like the NMOSD Nurse Advocates and PREVAIL virtual events for those who want to learn more about NMOSD and UPLIZNA.

You can also read personal stories, like Mary’s journey with NMOSD and UPLIZNA. Here’s a preview of what she had to say:

“Fast forward to today: I’m doing much better. Since starting the treatment, I’ve had no additional relapses. The symptoms that I do have, like a tightening pain in my abdomen, occasional trouble with balance, urinary and bowel issues as well as some numbness in my lower extremities, have not worsened since my first known attack. I have not experienced another attack since starting UPLIZNA.
Now, I get an infusion every six months, which fits in with my schedule well as someone with an active lifestyle. And because I get the dose twice a year, it’s easy to schedule and plan time with family.”

What advice do you have for those with NMOSD going into their next doctor’s appointment?

In general, it’s important to have a good relationship with your health care professional so that you feel comfortable communicating symptoms and health concerns. For those with NMOSD, it’s not always obvious that something might be going on (i.e., bladder or sensory symptoms) or that their symptoms may be worsening. These symptoms might not be immediately visible to your doctor so it’s important to let them know. I try to make my patients feel comfortable telling me all their symptoms.

It’s also important to be proactive with NMOSD treatment to help reduce the risk of an attack, so even if everything may appear to be fine and stable, I recommend patients have a conversation about the FDA-approved medicines.

Dr. Robert Shin participated in this interview in partnership with Horizon Therapeutics.


What is UPLIZNA?
UPLIZNA is a prescription medicine used to treat adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive. 

It is not known if UPLIZNA is safe or effective in children.

Who should not receive UPLIZNA?
You should not receive UPLIZNA if you have:
– had a life-threatening infusion reaction to UPLIZNA.
– an active hepatitis B virus infection.
– active or untreated inactive (latent) tuberculosis.

Before receiving UPLIZNA, tell your healthcare provider about all of your medical conditions, including if you:
– have or think you have an infection.– have ever taken, currently take, or plan to take medicines that affect your immune system, or other treatments for NMOSD. These medicines may increase your risk of getting an infection.– have or have ever had hepatitis B or are a carrier of the hepatitis B virus.– have or have ever had tuberculosis.– have had a recent vaccination or are scheduled to receive any vaccinations. You should receive any required vaccines at least 4 weeks before you start treatment with UPLIZNA.– are pregnant or plan to become pregnant. It is not known if UPLIZNA will harm your unborn baby. Females should use birth control (contraception) during treatment with UPLIZNA and for 6 months after your last infusion of UPLIZNA.– are breastfeeding or plan to breastfeed. It is not known if UPLIZNA passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you receive UPLIZNA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. 

What is the most important information I should know about UPLIZNA?
UPLIZNA may cause serious side effects, including: 
Infusion reactions. UPLIZNA can cause infusion reactions that can be serious or may cause you to be hospitalized. You will be monitored during your infusion and for at least 1 hour after each infusion of UPLIZNA for signs and symptoms of an infusion reaction. Tell your healthcare provider right away if you get any of these symptoms:

  • headache
  • sleepiness
  • fever
  • rash
  • nausea
  • shortness of breath
  • muscle aches 

If you develop an infusion reaction, your healthcare provider may need to stop or slow down the rate of your infusion and treat your symptoms.

Infections. Infections can happen during treatment with UPLIZNA. Tell your healthcare provider right away if you have an infection or get any of these symptoms:

  • painful and frequent urination 
  • nasal congestion, runny nose, sore throat, fever, chills, cough, body aches 
  • UPLIZNA taken before or after other medicines that weaken the immune system may increase your risk of getting infections. 
  • Hepatitis B virus (HBV) reactivation. Before starting treatment with UPLIZNA, your healthcare provider will do blood tests to check for hepatitis B viral infection. If you have ever had hepatitis B virus infection, the hepatitis B virus may become active again during or after treatment with UPLIZNA. Hepatitis B virus becoming active again (called reactivation) may cause serious liver problems, including liver failure or death. Your healthcare provider will monitor you if you are at risk for hepatitis B virus reactivation during treatment and after you stop receiving UPLIZNA.
  • Progressive Multifocal Leukoencephalopathy (PML). PML may happen with UPLIZNA. PML is a rare brain infection that leads to death or severe disability. Symptoms of PML may get worse over days to weeks. Call your healthcare provider right away if you get any of these symptoms:
      – weakness on one side of the body
      – changes in your vision
      – confusion
      – loss of coordination in your arms and legs
      – changes in thinking or memory
      – changes in your personality
  • Tuberculosis (TB). TB is caused by an infection in the lungs. Before starting treatment with UPLIZNA, your healthcare provider will check to see if you are at risk for getting TB or have ever had TB.
  • Vaccinations. Certain vaccines, called “live” or “live attenuated” vaccines, are not recommended in people receiving UPLIZNA. Talk to your healthcare provider before receiving any vaccinations. If you have a baby and you were receiving UPLIZNA during pregnancy, it is important to tell your baby’s healthcare provider about your UPLIZNA use so they can decide when your baby should receive any vaccine.

See “What are the possible side effects of UPLIZNA?” for more information about side effects.
How will I receive UPLIZNA?

  • UPLIZNA is given through a needle placed in a vein (IV or intravenous infusion) in your arm.
  • Before treatment with UPLIZNA, your healthcare provider will give you a corticosteroid medicine, an antihistamine, and a fever prevention medicine to help infusion reactions become less frequent and less severe. See “What is the most important information I should know about UPLIZNA?”
  • Your first dose of UPLIZNA will be given as 2 separate infusions, 2 weeks apart. 
  • Your next doses of UPLIZNA will be given as one infusion every 6 months. 
  • Each infusion will last about 1 hour and 30 minutes. After each infusion, you will be monitored by a healthcare provider for at least 1 hour.

What are the possible side effects of UPLIZNA?
UPLIZNA may cause serious side effects, including: 

  • See “What is the most important information I should know about UPLIZNA?”
  • low blood cell counts. UPLIZNA may cause a decrease in some types of blood cells. Your healthcare provider will do blood tests to check your blood cell counts.

The most common side effects include urinary tract infection and joint pain.
These are not all the possible side effects of UPLIZNA. 
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

P-UPZ-US-00547 01/23 © 2023 Horizon Therapeutics plc

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