TY - JOUR
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 H.
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
PY - 2012/7
Y1 - 2012/7
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.
AB - 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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U2 - 10.1002/art.34409
DO - 10.1002/art.34409
M3 - Article
C2 - 22294344
AN - SCOPUS:84863228835
SN - 0004-3591
VL - 64
SP - 2375
EP - 2386
JO - Arthritis and rheumatism
JF - Arthritis and rheumatism
IS - 7
ER -