The challenges of NOMID

NOMID is recognized as the most severe form of CAPS, a group of autoinflammatory disorders1

  • NOMID, which begins in infancy, is a lifelong and severely debilitating disease1,2
  • NOMID is very rare3
    • Estimated prevalence is less than 1 case per 1,000,000 people3
    • In the European Union, only 100 cases have been reported3
    • NOMID is associated with a number of clinical symptoms and markers1,2
    • It often presents in the first weeks of life as an urticarial-like skin rash and can progress to affect multiple organ systems, including skin, bones, and the central nervous system (CNS)2
    • Specific CNS manifestations include chronic aseptic meningitis, loss of vision, mental retardation, seizures, and hearing loss2
    • Additionally, elevated levels of SAA and CRP and elevated ESR may be present1,2
    • A 20% mortality rate before adulthood has been reported2

Early identification and treatment are important to avoid long-term complications, yet diagnosing NOMID is difficult4

  • Because NOMID is so rare, not many physicians have experience with NOMID to recognize the early signs3
  • The early signs can be so broad and nondescript that they are often attributed to more common diseases or causes, such as an infection5
  • Diagnosis is often made through a time-consuming process of eliminating other possible causes

IL-1β has been identified as playing an important role in the systemic inflammation and manifestations of NOMID and CAPS6

  • Spontaneous mutations in the CIAS1/NLRP3 gene have been identified in a majority of patients with CAPS, such as NOMID6
    • This mutation leads to an overabundance of the body's production of IL-1β, which leads to the manifestations of NOMID6

CAPS=cryopyrin-associated periodic syndrome; SAA=serum amyloid A; CRP=C-reactive protein; ESR=erythrocyte sedimentation rate.1

References

  1. Sibley CH, Plass N, Snow J, et al. Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra. Arthritis Rheum. 2012:66(7);2375-2386.
  2. Goldbach-Mansky R, Dailey NJ, Canna SW, et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1β inhibition. N Engl J Med. 2006;355:581-592.
  3. Orphanet Report Series – Prevalence of rare diseases: Bibliographic data. June 2013; Number 2.
  4. Hashkes PJ, Toker O. Autoinflammatory syndromes. Pediatr Clin N Am. 2012:59;447-470.
  5. Krause K, Grattan CE, Bindslev-Jensen C, et al. How not to miss autoinflammatory diseases masquerading as urticaria. Allergy. 2012:67;1465-1474.
  6. Kineret [Prescribing Information]. Stockholm, Sweden: Biovitrum AB; 2013.

See Quinn’s story, a rare case study in NOMID.

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Kineret improved both disease symptoms and serum markers of inflammation of NOMID in clinical trials1,2

Overall DSSS and each of its key symptoms improved over 60 months1

Diary Symptom Scores, key symptoms: 0=no symptoms, 1=mild, 2=moderate, 3=more severe, 4=severe.2

  • The figure shows mean values for observed cases at all given time points. ITT observed cases (n=29)
    • Improvements occurred in all individual disease symptoms comprising the DSSS1,2
    • Results for this endpoint were consistent across all subgroups including age, gender, presence of CIAS1 mutation, and disease phenotype1

Kineret significantly reduced key biomarkers of inflammation over 60 months2

  • Estimated changes from baseline in SAA, hsCRP, and ESR* levels from baseline were statistically significant at all time points through 60 months (P<0.0001)2

NOMID patients who were withdrawn from Kineret therapy responded again when treatment resumed1

  • For the 11 patients who went through a withdrawal phase, disease symptoms and key biomarkers of inflammation worsened after withdrawal and promptly responded to reinstitution of Kineret therapy1

Kineret provided additional clinical benefits for patients1

  • Treatment with Kineret appeared to be associated with improvement of, or stability in, assessments of other NOMID disease manifestations, up to 60 months
    • CNS
    • Audiogram
    • Visual acuity

*SAA=serum amyloid A; hsCRP=high-sensitivity C-reactive protein; ESR=erythrocyte sedimentation rate.

References

  1. Kineret [Prescribing Information]. Stockholm, Sweden: Biovitrum AB; 2013.
  2. Data on file. Stockholm, Sweden: Biovitrum AB; 2012.

IL-1—A mediator of inflammation

Overproduction of IL-1 has been implicated in autoinflammatory and autoimmune diseases such as NOMID and RA

Learn more >

Kineret has been evaluated in a prospective, long-term, open-label, uncontrolled study of 43 NOMID patients1

  • Kineret is the only FDA-approved medication for NOMID indicated for pediatric patients and adult patients1
  • 12+ years of clinical experience2 in adults with RA
  • Short half-life (4-6 hours) allowing for a short washout period if therapy needs to be interrupted for various reasons1

Adverse events (AEs) seen in NOMID clinical trials

  • There were 24 serious adverse events reported in 14 of the 43 treated patients, the most common type being infections1

References

  1. Kineret [Prescribing Information]. Stockholm, Sweden: Biovitrum AB; 2013.
  2. Data on file. Stockholm, Sweden: Biovitrum AB; 2012.
  3. Goldbach-Mansky R, Dailey N, Canna S, et al. Neonatal-onset multisystem inflammatory disease responsive to interleukin-1β inhibition. N Engl J Med. 2006;355(6):581-592.

IL-1—A mediator of inflammation

Overproduction of IL-1 has been implicated in autoinflammatory and autoimmune diseases such as NOMID and RA

Learn more >

Kineret safety profile in NOMID clinical trials

  • Most adverse events (AEs), including infections, occurred during the first 6 months of treatment. The incidence of AEs did not increase over time, and no new types of AEs emerged1
  • There were no permanent discontinuations of Kineret due to AEs1
  • There were no temporary or permanent discontinuations due to injection site reactions (ISRs). The majority of ISRs were mild (76%) or moderate (24%)1

Reference

  1. Kineret [Prescribing Information]. Stockholm, Sweden: Biovitrum AB; 2013.

IL-1—A mediator of inflammation

Overproduction of IL-1 has been implicated in autoinflammatory and autoimmune diseases such as NOMID and RA

Learn more >

Once-daily, subcutaneous dosing in NOMID*

  • The recommended starting dose is 1-2 mg/kg1
  • The dose can be individually adjusted to a maximum of 8 mg/kg daily to control active inflammation1
  • Adjust doses in 0.5- to 1.0-mg/kg increments1
  • Approved for all ages in NOMID (pediatric and adult)1
  • Physicians should consider administration of the prescribed Kineret® dose every other day for NOMID patients who have severe renal insufficiency or end-stage renal disease (defined as creatinine clearance <30 mL/min, as estimated from serum creatinine levels)1
  • Patients or caregivers should not be allowed to administer Kineret® until the patient or caregiver has demonstrated a thorough understanding of procedures and an ability to inject the product correctly1
  • Dosing should be adjusted using the graduated syringe for doses under 100 mg/day1

Calculating the daily dose of Kineret for your NOMID patient is easy1

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*Once-daily administration is generally recommended, but the dose may be split into twice-daily administrations1.

Reference

  1. Kineret [Prescribing Information]. Stockholm, Sweden: Biovitrum AB; 2013.

IL-1—A mediator of inflammation

Overproduction of IL-1 has been implicated in autoinflammatory and autoimmune diseases such as NOMID and RA

Learn more >

INDICATIONS

Kineret® is an interleukin-1 receptor antagonist indicated for:

Rheumatoid Arthritis (RA)

Cryopyrin-Associated Periodic Syndromes (CAPS)

IMPORTANT SAFETY INFORMATION