NOMID Case Study

Patient insight: Quinn

Treated with KINERET since 2009.

Young girl smiling with her parents in the background

Patient age at first visit: 6 months

Patient age at diagnosis and initiation of KINERET treatment: 12 months

Relevant history/physical exam

  • Persistent urticaria-like rash
  • Recurrent high fevers
  • Failure to thrive (5th percentile for weight)
  • Mild facial dysmorphia (frontal bossing, saddle nose)
  • Mild developmental motor delays

Evaluation

Relevant lab tests during evaluation period

CRP: Ranged from 27 to 305 mg/L (normal <5 mg/L)

WBC: Ranged from 27.7 to 44.4 x 109/L (normal range 4-11 x 109/L)

Genetic testing: Negative for mutations in NLRP3/CIAS1 gene

Treating physician’s approach to evaluating Quinn:

1. Rule out infection and malignancy

  • Referrals to infectious disease specialists and oncologists
  • Extensive testing included lumbar punctures, colonoscopy, and bone marrow and skin biopsies

2. Consider autoinflammatory symptoms

  • Based on Quinn’s age and the severity of her symptoms, NOMID was a strong possibility

Diagnosis

Treating physician suspected NOMID and decided to try KINERET

  • Quinn had many of the symptoms associated with NOMID
    • Other common symptoms (eg, hearing loss and conjunctivitis) were not present
  • Quinn tested negative for NLRP3/CIAS1 mutation (mutation is absent in ~40% of cases)
  • 2016 clinical diagnostic criteria for CAPS do not “mandate evidence of a disease-causing NLRP3 mutation”

Results with KINERET treatment

Quinn’s response to KINERET confirmed treating physician’s suspected diagnosis of NOMID

  • CRP levels dropped from 300 mg/L to 3 mg/L overnight
    • Rash and irritability/pain also improved

Quinn has been taking KINERET since 2009

  • KINERET keeps Quinn’s NOMID symptoms under control
  • Infections and stress still cause periodic flares
    • Headache, fever, and fatigue are the primary symptoms
    • Quinn periodically takes acetaminophen during flares
  • Quinn is an active child who continues to meet age-appropriate developmental and growth milestones
    • Quinn started to self-inject at 8 years old

This case represents one patient's experience as interviewed in 2017. Individual outcomes may vary and are dependent on a patient's clinical history and profile, as well as their treating physician's discretion.

See the data
age NOTES
  • Timeline marker showing the start of birth
    • Born prematurely (34 weeks gestation)
    • Rash
    • Fever
    • Continued symptoms and
      failure to thrive
    • Consultations with multiple
      specialists
  • Timeline marker marking the 6 months of birth
    • Initial visit with treating physician
    • Treating physician evaluation
    • Included referrals to
      more specialists
  • Timeline marker marking the 12 months of birth
    • Hospitalized for aseptic meningitis
    • NOMID diagnosed
    • KINERET treatment began
  • Timeline marker marking the 8 years of age
    • Started self-injecting KINERET
  • Timeline marker marking the 11 years of age
    • Continues KINERET treatment
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Important Safety Information
INDICATION

KINERET® (anakinra) is an interleukin-1 receptor antagonist indicated for:

Rheumatoid Arthritis (RA). Reduction in signs and symptoms and slowing the progression of structural damage in moderately to severely active rheumatoid arthritis, in patients 18 years of age or older who have failed 1 or more disease-modifying antirheumatic drugs (DMARDs)

Cryopyrin-Associated Periodic Syndromes (CAPS). Treatment of Neonatal-Onset Multisystem Inflammatory Disease (NOMID)

Deficiency of Interleukin-1 Receptor Antagonist (DIRA). Treatment of Deficiency of Interleukin-1 Receptor Antagonist (DIRA)

CONTRAINDICATION

KINERET is contraindicated in patients with known hypersensitivity to E. coli–derived proteins, KINERET, or to any components of the product

IMPORTANT SAFETY INFORMATION

Serious Infections. In RA, discontinue use if serious infection develops. In KINERET-treated NOMID or DIRA patients, the risk of a disease flare when discontinuing KINERET treatment should be weighed against the potential risk of continued treatment. Do not initiate KINERET in patients with active infections

Use in combination with Tumor Necrosis Factor (TNF)-blocking agents is not recommended

Hypersensitivity reactions, including anaphylactic reactions and angioedema, and serious cutaneous reactions including drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. Patients with DIRA may have an increased risk of allergic reactions, particularly in the first several weeks after starting KINERET treatment

Immunosuppression. The impact of treatment with KINERET on active and/or chronic infections and the development of malignancies is not known

Immunizations. Live vaccines should not be given concurrently with KINERET

Neutrophil counts should be assessed prior to initiating KINERET treatment, and while receiving KINERET, monthly for 3 months, and thereafter quarterly for a period up to 1 year

Serious Adverse Reactions

RA: The most serious adverse reactions were: Serious Infections and Neutropenia, particularly when used in combination with TNF blocking agents.

NOMID and DIRA: The most serious adverse events were infections.

Most Common Adverse Reactions

RA: The most common adverse reactions (incidence ≥ 5%) are injection site reaction, worsening of rheumatoid arthritis, upper respiratory tract infection, headache, nausea, diarrhea, sinusitis, arthralgia, flu-like symptoms, and abdominal pain

NOMID: The most common AEs during the first 6 months of treatment (incidence > 10%) are injection site reaction, headache, vomiting, arthralgia, pyrexia, and nasopharyngitis

DIRA: The most common AEs are upper respiratory tract infections, rash, pyrexia, influenza-like illness, and gastroenteritis

Post-marketing Experience

Hepato-biliary disorders (elevations of transaminases; non-infectious hepatitis) and thrombocytopenia, including severe thrombocytopenia and DRESS have been identified during postapproval use of KINERET. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.

These are not all the possible risks associated with KINERET. Please see Full Prescribing Information for KINERET at https://www.KineretRxHCP.com/home.php
To report suspected adverse reactions, contact Sobi North America at 1-866-773-5274 or FDA at 1-800-FDA-1088
Click here for full Prescribing Information for KINERET.
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