Neonatal treatment of CINCA syndrome

Pediatr Rheumatol Online J. 2014 Dec 15:12:52. doi: 10.1186/1546-0096-12-52. eCollection 2014.

Abstract

Chronic Infantile Neurological Cutaneous Articular (CINCA) syndrome, also called Neonatal Onset Multisystem Inflammatory Disease (NOMID) is a chronic disease with early onset affecting mainly the central nervous system, bones and joints and may lead to permanent damage. We report two preterm infants with severe CINCA syndrome treated by anti-interleukin-1 in the neonatal period, although, so far, no experience with this treatment in infants younger than three months of age has been reported. A review of the literature was performed with focus on treatment and neonatal features of CINCA syndrome.

Case report: Two cases suspected to have CINCA syndrome were put on treatment with anakinra in the early neonatal period due to severe clinical presentation. We observed a rapid and persistent decline of clinical signs and systemic inflammation and good drug tolerance. Diagnosis was confirmed in both cases by mutations in the NLRP3/CIAS1-gene coding for cryopyrin. As particular neonatal clinical signs polyhydramnios and endocardial overgrowth are to be mentioned.

Conclusion: We strongly suggest that specific treatment targeting interleukin-1 activity should be started early. Being well tolerated, it can be introduced already in neonates presenting clinical signs of severe CINCA syndrome in order to rapidly control inflammation and to prevent life-long disability.

Keywords: Anakinra; CINCA; IL-1 receptor; IL-1β; NOMID; Neonatal treatment.

Publication types

  • Case Reports

MeSH terms

  • Antirheumatic Agents / therapeutic use*
  • Carrier Proteins / genetics
  • Cryopyrin-Associated Periodic Syndromes / diagnosis
  • Cryopyrin-Associated Periodic Syndromes / drug therapy*
  • Cryopyrin-Associated Periodic Syndromes / genetics
  • Female
  • Humans
  • Infant, Newborn
  • Infant, Premature
  • Interleukin 1 Receptor Antagonist Protein / therapeutic use*
  • Interleukin-1 / antagonists & inhibitors*
  • Male
  • Mutation / genetics
  • NLR Family, Pyrin Domain-Containing 3 Protein
  • Time Factors
  • Treatment Outcome

Substances

  • Antirheumatic Agents
  • Carrier Proteins
  • Interleukin 1 Receptor Antagonist Protein
  • Interleukin-1
  • NLR Family, Pyrin Domain-Containing 3 Protein
  • NLRP3 protein, human