TY - JOUR
T1 - Decreased quality of life and societal impact of cryopyrin-associated periodic syndrome treated with canakinumab
T2 - A questionnaire based cohort study
AU - Mulders-Manders, Catharina M.
AU - Kanters, Tim A.
AU - van Daele, Paul L. A.
AU - Hoppenreijs, Esther
AU - Legger, G. Elizabeth
AU - van Laar, Jan A. M.
AU - Simon, Anna
AU - Hakkaart-van Roijen, Leona
PY - 2018/4/20
Y1 - 2018/4/20
N2 - 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.
AB - 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.
KW - Cryopyrin-associated periodic syndrome
KW - Muckle Wells syndrome
KW - Neonatal onset multisystem inflammatorydisease
KW - Chronic infantile neurologic cutaneous and arthritis syndrome
KW - Canakinumab
KW - Anakinra
KW - Quality of life
KW - Societal impact
KW - INSTRUMENT
KW - FRENCH
U2 - 10.1186/s13023-018-0799-1
DO - 10.1186/s13023-018-0799-1
M3 - Article
SN - 1750-1172
VL - 13
JO - Orphanet journal of rare diseases
JF - Orphanet journal of rare diseases
M1 - 59
ER -