Table 3

Safety findings reported in observational studies of CAPS, HIDS/MKD and TRAPS

Study, year, country (pub type)Study designPopulationTreatment(s)NSafety findings
†Guerrero et al 2017,52 SpainRetrospective (Tertiary care hospital)CAPS (MWS)Canakinumab6Canakinumab was well-tolerated; injection-site reactions (1 patient).
Kuemmerle-Deschner et al 2016,40 GermanyProspective (Hospital)CAPS (FCAS, MWS, CINCA/NOMID)Canakinumab68AEs, 73 no. (infections and infestations, 45 no.; general and administrative site conditions, 16 no.); SAEs (2 no. (one infection and another musculoskeletal event))
Anton et al 2015,34 SpainRetrospective (Cohort)CAPS (FCAS, MWS)Anakinra5Injection-site reactions (60%); no infections.
Canakinumab8No injection-site reactions; upper RTI (1 patient); acute appendicitis (1 patient).
Kuemmerle-Deschner et al 2013,59 GermanyProspective (Cohort)CAPS (MWS)Anakinra12Injection-site reaction (42%) (mild); upper RTI (33%); weight gain (≥5 kg). No SAEs.
Canakinumab14No injection-site reactions; upper RTI (29%); transient headache (14%). SAE, 1 patient (vertigo requiring hospitalisation)
Eroglu et al 2016,36 TurkeyProspective (Clinics)CAPSAnti-IL-1 (Anakinra, Canakinumab)14Macrophage activation syndrome (2 patients). No SAE requiring hospitalisation.
Mehar et al 2016,43 AustraliaRetrospective (Physician survey)CAPS (FCAS, MWS, CINCA/NOMID)Anakinra13Injection-site pain (54%).
Russo et al 2014,65 EnglandProspective (Clinics)CAPS (MWS, CINCA/NOMID)Canakinumab10Canakinumab was well-tolerated. No injection-site reactions. No serious infections.
Chang et al 2014,50 U.S.Retrospective (Patient survey)CAPS (FCAS, CINCA/NOMID)Anakinra9Anakinra was well-tolerated.
†Navarrete et al 2014,61 SpainRetrospective (Clinics)CAPS (MWS)Canakinumab10No AE by any patient.
Kuemmerle-Deschner et al 2011,58 GermanyProspective (OL)CAPS (MWS)Anakinra12Anakinra was well-tolerated. No SAEs.
Neven et al 2010,62 EuropeRetrospective (Clinics)CAPS (CINCA/NOMID)Anakinra10Mild injection-site reactions.
†Hoffman et al 2016,37 InternationalProspective (Registry)CAPS
(FCAS, MWS, CINCA/NOMID)
Canakinumab288IR/100 pyrs for overall AEs was 100.0 (FCAS, 78.1; MWS, 113.4; NOMID, 119.0). Most common AEs: infections and infestations (IR/100 pyrs, 39.1). SAEs, 86 patients (IR/100 pyrs, 16.3) (infections (IR/100 pyrs, 5.0)). Discontinuations, 22 pts (8%) (AEs, 5; poor efficacy and patient choice, 10; other reasons, 7). One death (due to metastatic rectal adenocarcinoma).
Rossi-Semerano et al 2015,86 FranceRetrospective (Physician survey)HIDSAnakinra10SAE, 1 patient (severe bronchitis)
Galeotti et al 2012,78 FranceRetrospective (Physician survey)HIDSCanakinumab6Injection-site reactions (1 patient), recurrent pharyngitis (1 patient) and transient hepatitis (1 patient). Overall, well-tolerated.
Anakinra9Injection-site reactions (4 patients), shivers and hypothermia (1 patient) and bacterial pneumonia (1 patient). Overall, well tolerated.
Bodar et al 2011,70 The NetherlandsProspective (Clinics)HIDSAnakinra11Injection-site reactions (2 patients), mild upper RTI (2 patients).
†Cakan et al 2017,72 TurkeyRetrospective (Clinics)CAPS (FCAS, CINCA/NOMID)Canakinumab3No AEs observed under canakinumab treatment.
HIDSCanakinumab2
†Salugina et al 2017,87 RussiaRetrospective (Clinics)CAPSTocilizumab2Satisfactory tolerability of all treatments observed in all patients.
Canakinumab9
HIDSTocilizumab1
TRAPSTocilizumab2
Canakinumab4
†Salugina et al 2016,88 RussiaProspective (Clinics)CAPSCanakinumab8Canakinumab was satisfactorily tolerated. No SAEs reported.
TRAPS3
Cantarini et al 20106,4 ItalyRetrospective (Case-series)TRAPSEtanercept7Injections-site reactions (2 patients). No SAEs observed.
Gattorno et al 2008,79 ItalyProspectiveTRAPSAnakinra5Injections-site reactions (all 5 patients). No SAEs observed.
Drewe et al 2003,77 U.K.Prospective (Case-series)TRAPSEtanercept7Injection-site reactions (1 patient), upper RTI (1 patient). No SAEs or hospital admissions reported.
  • †Studies published as conference abstracts only.

  • AE, adverse event; CAPS, cryopyrin-associated periodic syndromes; CINCA, chronic infantile neurological cutaneous and articular syndrome; FCAS, familial cold autoinflammatory syndrome; HIDS, hyperimmunoglobulin D syndrome; MKD, mevalonate kinase deficiency; MWS, Muckle–Wells syndrome; NOMID, neonatal-onset multisystem inflammatory disease; RTI, respiratory tract infection; SAE, serious AE; TRAPS, TNF receptor-associated periodic syndrome; UTI, urinary tract infection.

  • N is number of patients who received a particular treatment.