Neonatal-Onset Multisystem Inflammatory Disease (NOMID) is a rare autoinflammatory disorder that typically begins in infancy. It is characterized by recurrent episodes of inflammation affecting multiple parts of the body, including the joints, skin, and nervous system.
NOMID stands out as the most severe variant of Cryopyrin-Associated Periodic Syndromes (CAPS), a cluster of autoinflammatory conditions that affect just 1 in every 1,000,000 individuals. Interestingly, the majority of those diagnosed with NOMID have no relatives experiencing similar symptoms.
What Causes NOMID?
NOMID is caused by mutations in the NLRP3 gene, which leads to the overactivation of the innate immune system and excessive production of interleukin-1 (IL-1), a pro-inflammatory cytokine. This dysregulation of the immune system results in the recurrent inflammation seen in NOMID.
Symptoms of NOMID:
Symptoms of NOMID can vary in severity but often include:
- fever
- persistent rash
- joint swelling and pain
- headaches
- hearing loss
- vision problems
- neurological signs and symptoms such as seizures or developmental delays
First and Only FDA-approved Treatment for NOMID:
Kineret (anakinra) is the first and only FDA-approved therapeutic drug approved for the treatment of children and adults with neonatal-onset multisystem inflammatory disease (NOMID)—a form of cryopyrin-associated periodic syndromes (CAPS).
This medicinal product is supplied as 100 mg/0.67 mL solution for subcutaneous injection It is designed to work by blocking the action of interleukin-1, thereby reducing inflammation and alleviating symptoms. Kineret is typically administered as a daily subcutaneous (SC) injection.
The most common side effects in the first 6 months of the Kineret in patients with NOMID are injection site skin reactions, vomiting, fever, colds, joint pain, and headache.
Effectiveness of Kineret:
Studies have demonstrated that treatment with Kineret 100 mg/0.67 mL can lead to significant improvement in symptoms and quality of life (QoL) for individuals with NOMID. It helps reduce inflammation, alleviate pain, and prevent long-term complications associated with the disease.
Managing NOMID:
In addition to therapeutic drugs, managing NOMID often involves a multidisciplinary approach, including persistent monitoring by healthcare professionals (HCPs), physical therapy, and certain supportive care to address specific symptoms and complications.
Conclusion:
NOMID is a rare but severe autoinflammatory disorder that requires prompt diagnosis and appropriate treatment. Kineret has emerged as a valuable therapeutic option, providing relief from symptoms and improving outcomes for those living with NOMID. With the presence of Kineret, there is hope for better management and improved quality of life (QoL) for those affected by this condition.
Reference:
https://www.kineretrx.com/nomid
