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

作者：Mulders Manders Catharina M^*; Kanters Tim A; van Daele Paul L A; Hoppenreijs Esther; Legger G Elizabeth; van Laar Jan A M; Simon Anna; Hakkaart van Roijen Leona

来源：Orphanet Journal of Rare Diseases, 2018, 13(1): 59.

DOI：10.1186/s13023-018-0799-1

• Cryopyrin-associated periodic syndrome
• Muckle Wells syndrome
• Neonatal onset multisystem inflammatorydisease
• Chronic infantile neurologic cutaneous and arthritis syndrome
• Canakinumab
• Anakinra
• Quality of life
• Societal impact

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.

• 出版日期2018-4-20
• 单位河北医科大学