Dmitrii Peshko1, Ekaterina Kulbachinskaya2, Ilya Korsunskiy3, Elena Kondrikova1, Federica Pulvirenti4, Isabella Quinti4, Oleg Blyuss5, Audrey Dunn Galvin6, Daniel Munblit7. 1. Department of Paediatrics, Sechenov University, Moscow, Russia. 2. Department of Paediatrics, Sechenov University, Moscow, Russia; The Research and Clinical Institute for Pediatrics named after Academician Yuri Veltischev of the Pirogov Russian National Research Medical University, Moscow, Russia. 3. Department of Paediatrics, Sechenov University, Moscow, Russia; Moscow City Paediatric Hospital #9 named after Speransky, Moscow, Russia. 4. Department of Molecular Medicine, Sapienza University of Rome, Rome, Italy. 5. Wolfson Institute of Preventive Medicine, Queen Mary University of London, London, United Kingdom; Department of Applied Mathematics, Lobachevsky State University of Nizhny Novgorod, Nizhny Novgorod, Russia. 6. School of Applied Psychology, University College Cork, Cork, Ireland. 7. Department of Paediatrics, Sechenov University, Moscow, Russia; Department of Paediatrics, Imperial College London, London, United Kingdom; The In-VIVO Global Network, an Affiliate of the World Universities Network (WUN), New York, NY; Healthcare Department of Moscow, Moscow Research and Clinical Center for Neuropsychiatry, Moscow, Russia. Electronic address: daniel.munblit08@imperial.ac.uk.
Abstract
BACKGROUND: Primary immunodeficiency diseases (PIDs) are a group of diseases that have been found to have an adverse impact on quality of life and health-related quality of life (HRQOL). OBJECTIVE: To systematically assess available evidence on the HRQOL of patients with PID. METHODS: We performed a literature search of all studies reporting HRQOL assessments in patients with PID published in English from inception to April 11, 2017, using MEDLINE and EMBASE. RESULTS: Of 1699 articles, 37 met the inclusion criteria. HRQOL was assessed by using various generic instruments. Child Health Questionnaire - Parent Form 50 and short-form 36 were the most frequently used (for children and adults, respectively). No PID-specific HRQOL instruments were used for children. HRQOL is significantly lower in adults with PID (mean score difference, -24.46; 95% CI, -34.57 to -14.34) and children (-10.06; 95% CI, -12.95 to -7.17) compared with the reference population and lower than in patients with other chronic conditions. There is a general agreement between child- and parent-reported data, although parents report child school-related HRQOL as more impaired than do children (6.19; 95% CI, 0.38 to 11.99). Most studies were of low to moderate quality and had methodological limitations. CONCLUSIONS: Available evidence suggests that patients with PID have a lower HRQOL than do healthy individuals and patients with other chronic conditions, including diabetes mellitus and juvenile idiopathic arthritis. No disease-specific instruments are available for children, and few options are available for adults. This finding highlights the need for developing PID-specific instruments that would allow for a more sensitive evaluation of PID impact on patient health and psychological well-being, school/work, and social activities.
BACKGROUND:Primary immunodeficiency diseases (PIDs) are a group of diseases that have been found to have an adverse impact on quality of life and health-related quality of life (HRQOL). OBJECTIVE: To systematically assess available evidence on the HRQOL of patients with PID. METHODS: We performed a literature search of all studies reporting HRQOL assessments in patients with PID published in English from inception to April 11, 2017, using MEDLINE and EMBASE. RESULTS: Of 1699 articles, 37 met the inclusion criteria. HRQOL was assessed by using various generic instruments. Child Health Questionnaire - Parent Form 50 and short-form 36 were the most frequently used (for children and adults, respectively). No PID-specific HRQOL instruments were used for children. HRQOL is significantly lower in adults with PID (mean score difference, -24.46; 95% CI, -34.57 to -14.34) and children (-10.06; 95% CI, -12.95 to -7.17) compared with the reference population and lower than in patients with other chronic conditions. There is a general agreement between child- and parent-reported data, although parents report child school-related HRQOL as more impaired than do children (6.19; 95% CI, 0.38 to 11.99). Most studies were of low to moderate quality and had methodological limitations. CONCLUSIONS: Available evidence suggests that patients with PID have a lower HRQOL than do healthy individuals and patients with other chronic conditions, including diabetes mellitus and juvenile idiopathic arthritis. No disease-specific instruments are available for children, and few options are available for adults. This finding highlights the need for developing PID-specific instruments that would allow for a more sensitive evaluation of PID impact on patient health and psychological well-being, school/work, and social activities.
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