Sustained response and prevention of damage progression in patients with neonatal‐onset multisystem inflammatory disease treated with anakinra: a cohort study to …

CH Sibley, N Plass, J Snow, EA Wiggs… - Arthritis & …, 2012 - Wiley Online Library
CH Sibley, N Plass, J Snow, EA Wiggs, CC Brewer, KA King, C Zalewski, HJ Kim, R Bishop…
Arthritis & Rheumatism, 2012Wiley Online Library
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 …
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. Adverse events 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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