Decreased quality of life and societal impact of cryopyrin-associated periodic syndrome treated with canakinumab: a questionnaire based cohort study

Orphanet J Rare Dis. 2018 Apr 20;13(1):59. doi: 10.1186/s13023-018-0799-1.

Abstract

Background: Cryopyrin-associated periodic syndrome (CAPS) is a rare disease. Knowledge on the quality of life (QoL) and the disease's societal impact is limited. Canakinumab is used in increasing frequency for the treatment of CAPS.

Methods: Observational study in Dutch CAPS patients. Patients completed questionnaires regarding treatment with canakinumab at baseline and retrospectively. Quality of life was assessed using the EQ-5D-5L in adults and CHQ-PF50 in children. Impact on work and school was assessed. Caregivers' quality of life was assessed using the CarerQol.

Results: Mean quality of life scores during treatment with canakinumab were 0.769 (EQ-5D-5L), 51.1 (CHQ-P) and 57-1 (CHQ-M). Most patients experienced problems on the pain/discomfort dimension. Higher disease activity and the presence of complications negatively influenced QoL. Half of the patients with a paid job reported absenteeism from work due to CAPS, for an average of 8.7 days in a 4-week period. All schoolgoing patients (N = 5) reported absence from school due to CAPS, for an average of 2.9 days. Caregivers reported gaining a lot fulfillment from providing care for their family members.

Conclusion: QoL during treatment is lower than in the general Dutch population. CAPS leads to productivity loss and absenteeism from school, and impacts the quality of life in informal caregivers.

Keywords: Anakinra; Canakinumab; Chronic infantile neurologic cutaneous and arthritis syndrome; Cryopyrin-associated periodic syndrome; Muckle Wells syndrome; Neonatal onset multisystem inflammatory disease; Quality of life; Societal impact.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized
  • Child
  • Child, Preschool
  • Cohort Studies
  • Cryopyrin-Associated Periodic Syndromes / drug therapy*
  • Cryopyrin-Associated Periodic Syndromes / physiopathology*
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Interleukin 1 Receptor Antagonist Protein / therapeutic use
  • Male
  • Middle Aged
  • Quality of Life
  • Retrospective Studies
  • Surveys and Questionnaires
  • Young Adult

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Interleukin 1 Receptor Antagonist Protein
  • canakinumab