Abstract
ObjectiveAnakinra has been increasingly used in off-label indications as well as dosing and mode of administration in a variety of inflammatory conditions. We aimed to review our clinical practice and compare treatment outcomes with published data.MethodsClinical data from electronic records were retrospectively reviewed for patients treated with anakinra over the past 6 years for autoinflammatory diseases (AID).ResultsFrom 47 eligible patients (27 female patients), 32 were children. Macrophage activation syndrome (MAS) was the indication for anakinra therapy in 42.6% of patients. Systemic juvenile idiopathic arthritis (SJIA) was the most common underlying diagnosis (19/47) followed by the spectrum of AID. Off-label use was noted in 38.3% patients. Recommended dose was exceeded in 21 children (mean induction dose 5.1, highest dose 29.4 mg/kg/day) and two adults; five patients were treated intravenously. The mean treatment duration for SJIA was 1.4 years, that for AID was 2.2 years, and that for patients with higher anakinra dose was 9.7 (19.3) months. The mean follow-up duration was 2.7 (1.7) years. Treatment was effective in the majority of SJIA and cryopyrinopathy patients as well as those with MAS. Anakinra was well-tolerated without any major adverse effects even in patients with long-term administration of higher than recommended doses including two infants treated with a dose of over 20 mg/kg/day.ConclusionOur results support early use of anakinra in the individually tailored dosing. In patients with hyperinflammation, anakinra may be lifesaving and may even allow for corticosteroid avoidance. Further studies are needed in order to set up generally accepted response parameters and define condition-specific optimal dosing regimen.
Highlights
Anakinra, a biosynthetic analog of interleukin-1 (IL-1) receptor antagonist, increases its natural downregulating action of blocking the two isoforms of IL-1 from binding to their receptor
Anakinra has been successfully used in hyperinflammatory conditions associated with cytokine storm including macrophage-activation syndrome (MAS), severe COVID-19, as well as paediatric inflammatory multisystem syndrome temporarily associated with SARS-CoV-2 infection (PIMS-TS) [14–23]
MAS in systemic juvenile idiopathic arthritis (SJIA) accounted for 15/20 MAS patients; in three cases, MAS complicated Syndrome of Undifferentiated Recurrent Fevers (SURF) and two patients had other conditions—one polyarticular JIA and one NLR Family CARD Domain Containing 4-associated AID (NLRC4-AID) (Table 1)
Summary
A biosynthetic analog of interleukin-1 (IL-1) receptor antagonist, increases its natural downregulating action of blocking the two isoforms of IL-1 from binding to their receptor. Resulting reduction of proinflammatory signaling has major therapeutic implications in a wide variety of diseases where dysregulation of innate immune mechanisms leads to IL-1 overproduction [1–3]. Autoimmunity, the two mechanisms may co-exist, creating a pathophysiological continuum [2]. Mainly periodic fever syndromes and systemic juvenile idiopathic arthritis (SJIA)/adult-onset Still’s disease (AOSD) may benefit from IL1 blockade [4–6]. European Medicines Agency (EMA)-approved indications of anakinra currently include apart from rheumatoid arthritis only cryopyrin-associated periodic syndromes (cryopyrinopathy, CAPS), familial Mediterranean fever (FMF), and SJIA/AOSD [7], its off-label use in other conditions has been demonstrated [8–13]. Anakinra has been successfully used in hyperinflammatory conditions associated with cytokine storm including macrophage-activation syndrome (MAS), severe COVID-19, as well as paediatric inflammatory multisystem syndrome temporarily associated with SARS-CoV-2 infection (PIMS-TS) [14–23]
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