Rare Disease

What is NOMID and Its Treatment

  • March 5, 2023
  • 3 mins read

NOMID is the most severe form of Cryopyrin-Associated Periodic Syndromes (CAPS), which are a group of autoinflammatory disorders seen in only 1 out of 1,000,000 individuals. This genetic disorder is caused by mutations in the CIAS1/NLRP3 gene. The majority of individuals diagnosed with NOMID have no other affected members in the family.

The term NOMID stands for Neonatal-Onset Multisystem Inflammatory Disease, which is also known as chronic infantile neurologic cutaneous articular (CINCA) syndrome. NOMID is a rare & systemic disease that causes persistent inflammation throughout the body, at birth or shortly after. This Neonatal-Onset Multisystem Inflammatory Disease is an autoinflammatory disease. In such diseases (autoinflammatory), the innate immune system (the one we are born with) is not working appropriately and typically causes inflammation of the body, even in absence of any injury or infection

Symptoms of NOMID:

The disease is characterized by a rash, fever, joint symptoms, and central nervous system symptoms. Throughout life, infections, and mental-physical stress can temporarily escalate the symptoms named flares.

In some patients, the symptoms of NOMID can be responsible for causing damage that usually progresses over time.

Children affected by the disease may grow more slowly than normal children, and over time the steady inflammation can lead to physical or intellectual disabilities and organ damage. The respective patients who are left untreated early in life, or whose symptoms are poorly controlled during treatment, are at risk for the most hazardous complications.

Diagnosis of NOMID:

Recognizing a rare disease, such as NOMID, is quite challenging. In most cases, diagnosis requires time and visits to multiple health specialists. There may be delays in diagnosing the disease because of the deficiency of recognition of its manifestations.

Neonatal-Onset Multisystem Inflammatory Disease is the most severe form of Cryopyrin-Associated Periodic Syndromes. Cryopyrin-Associated Periodic Syndromes can be recognized with blood tests for signs of inflammation and the existence of at least two symptoms. This rare disease is diagnosed based on the seriousness of these symptoms. A complete assessment of the signs and symptoms since birth is crucial so that Neonatal-Onset Multisystem Inflammatory Disease can be diagnosed and treated.

Treatment of NOMID:

Neonatal-Onset Multisystem Inflammatory Disease can be treated with KINERET (anakinra 100 mg/ 0.67 mL). The good news is that once the disease is recognized, a promising treatment is available. Kineret is the first ever and only medicine that has been approved by the FDA to treat Neonatal-Onset Multisystem Inflammatory Disease (NOMID).

The medicine Kineret inhibits interleukin-1-the underlying cause of inflammation in the disease. It blocks IL-1 protein. In those with NOMID, IL-1 forwards a signal that causes inflammation throughout the body. In clinical studies, Kineret 100 mg/0.67mL improved or stabilized the NOMID symptoms are rash, fever, joint pain, vomiting, headache, vision loss, hearing loss, and brain inflammation.

In studies, those taking treatment with Kineret reported a significant enhancement in signs-symptoms in the initial 90 days. The time duration for enhancement typically varies from patient to patient.

Dosage and Administration:

A health specialist will tell you how often and how much (each time) your child should take KINERET 100 mg. For children with Neonatal-Onset Multisystem Inflammatory Disease (NOMID), once-daily injections are typically recommended, but sometimes twice-daily administrations are considered. The half-life of Kineret is 4-6 hours, which means that the quantity of Kineret injection in your child’s body reduces by half every 4-6 hours. Taking the medicine as prescribed will help make sure that there is always sufficient medicine in your child’s body.