Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes

Cailin Sibley, Nikki Plass, Joseph Snow, Edythe A. Wiggs, Carmen C. Brewer, Kelly A. King, Christopher Zalewski, H. Jeffrey Kim, Rachel Bishop, Suvimol Hill, Scott M. Paul, Patrick Kicker, Zachary Phillips, Joseph G. Dolan, Brigitte Widemann, Nalini Jayaprakash, Frank Pucino, Deborah L. Stone, Dawn Chapelle, Christopher SnyderJohn A. Butman, Robert Wesley, Raphaela Goldbach-Mansky

Research output: Contribution to journalArticle

111 Citations (Scopus)

Abstract

Objective. Blocking interleukin-1 with anakinra in patients with the autoinflammatory syndrome neonatal-onset multisystem inflammatory disease (NOMID) reduces systemic and organ-specific inflammation. However, the impact of long-term treatment has not been established. This study was undertaken to evaluate the long-term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID. Methods. We conducted a cohort study of 26 NOMID patients ages 0.80-42.17 years who were followed up at the NIH and treated with anakinra 1-5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C-reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated. Results. Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P <0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P <0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverseevents other than viral infections were rare, and all patients continued to receive the medication. Conclusion. These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.

Original languageEnglish (US)
Pages (from-to)2375-2386
Number of pages12
JournalArthritis and Rheumatism
Volume64
Issue number7
DOIs
StatePublished - Jul 2012
Externally publishedYes

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Cryopyrin-Associated Periodic Syndromes
Interleukin 1 Receptor Antagonist Protein
Cohort Studies
Central Nervous System
Inflammation
Hearing
Parents
Cochlea
Virus Diseases
Patient Safety
Optic Nerve
Visual Fields
Interleukin-1
Leukocyte Count
Hearing Loss
C-Reactive Protein
Visual Acuity
Ear
Cerebrospinal Fluid
Albumins

ASJC Scopus subject areas

  • Immunology
  • Immunology and Allergy
  • Rheumatology
  • Pharmacology (medical)

Cite this

Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra : A cohort study to determine three- and five-year outcomes. / Sibley, Cailin; Plass, Nikki; Snow, Joseph; Wiggs, Edythe A.; Brewer, Carmen C.; King, Kelly A.; Zalewski, Christopher; Kim, H. Jeffrey; Bishop, Rachel; Hill, Suvimol; Paul, Scott M.; Kicker, Patrick; Phillips, Zachary; Dolan, Joseph G.; Widemann, Brigitte; Jayaprakash, Nalini; Pucino, Frank; Stone, Deborah L.; Chapelle, Dawn; Snyder, Christopher; Butman, John A.; Wesley, Robert; Goldbach-Mansky, Raphaela.

In: Arthritis and Rheumatism, Vol. 64, No. 7, 07.2012, p. 2375-2386.

Research output: Contribution to journalArticle

Sibley, C, Plass, N, Snow, J, Wiggs, EA, Brewer, CC, King, KA, Zalewski, C, Kim, HJ, Bishop, R, Hill, S, Paul, SM, Kicker, P, Phillips, Z, Dolan, JG, Widemann, B, Jayaprakash, N, Pucino, F, Stone, DL, Chapelle, D, Snyder, C, Butman, JA, Wesley, R & Goldbach-Mansky, R 2012, 'Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes', Arthritis and Rheumatism, vol. 64, no. 7, pp. 2375-2386. https://doi.org/10.1002/art.34409
Sibley, Cailin ; Plass, Nikki ; Snow, Joseph ; Wiggs, Edythe A. ; Brewer, Carmen C. ; King, Kelly A. ; Zalewski, Christopher ; Kim, H. Jeffrey ; Bishop, Rachel ; Hill, Suvimol ; Paul, Scott M. ; Kicker, Patrick ; Phillips, Zachary ; Dolan, Joseph G. ; Widemann, Brigitte ; Jayaprakash, Nalini ; Pucino, Frank ; Stone, Deborah L. ; Chapelle, Dawn ; Snyder, Christopher ; Butman, John A. ; Wesley, Robert ; Goldbach-Mansky, Raphaela. / Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra : A cohort study to determine three- and five-year outcomes. In: Arthritis and Rheumatism. 2012 ; Vol. 64, No. 7. pp. 2375-2386.
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abstract = "Objective. Blocking interleukin-1 with anakinra in patients with the autoinflammatory syndrome neonatal-onset multisystem inflammatory disease (NOMID) reduces systemic and organ-specific inflammation. However, the impact of long-term treatment has not been established. This study was undertaken to evaluate the long-term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID. Methods. We conducted a cohort study of 26 NOMID patients ages 0.80-42.17 years who were followed up at the NIH and treated with anakinra 1-5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C-reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated. Results. Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P <0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P <0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverseevents other than viral infections were rare, and all patients continued to receive the medication. Conclusion. These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.",
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T1 - Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra

T2 - A cohort study to determine three- and five-year outcomes

AU - Sibley, Cailin

AU - Plass, Nikki

AU - Snow, Joseph

AU - Wiggs, Edythe A.

AU - Brewer, Carmen C.

AU - King, Kelly A.

AU - Zalewski, Christopher

AU - Kim, H. Jeffrey

AU - Bishop, Rachel

AU - Hill, Suvimol

AU - Paul, Scott M.

AU - Kicker, Patrick

AU - Phillips, Zachary

AU - Dolan, Joseph G.

AU - Widemann, Brigitte

AU - Jayaprakash, Nalini

AU - Pucino, Frank

AU - Stone, Deborah L.

AU - Chapelle, Dawn

AU - Snyder, Christopher

AU - Butman, John A.

AU - Wesley, Robert

AU - Goldbach-Mansky, Raphaela

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N2 - Objective. Blocking interleukin-1 with anakinra in patients with the autoinflammatory syndrome neonatal-onset multisystem inflammatory disease (NOMID) reduces systemic and organ-specific inflammation. However, the impact of long-term treatment has not been established. This study was undertaken to evaluate the long-term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID. Methods. We conducted a cohort study of 26 NOMID patients ages 0.80-42.17 years who were followed up at the NIH and treated with anakinra 1-5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C-reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated. Results. Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P <0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P <0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverseevents other than viral infections were rare, and all patients continued to receive the medication. Conclusion. These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.

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