Measuring parent proxy-reported quality of life of 11 rare diseases in children in Zhejiang, China

Abstract Background: It has become increasingly important to measure the health-related quality of life (HRQoL) of rare diseases in children and adolescents in recent decades. Much attention has been paid to investigate the HROoL of a specific rare disease by self-report in previous studies. This study aimed to evaluate and compare the HROoL of 11 rare diseases in Chinese children by parent proxy-report, to explore the factors associated with HROoL of patients, and to understand the problems of most concern. Methods: A total of 651 children aged from 2 to 18 were enrolled from the Children’s Hospital Affiliated Zhejiang University in 2018. Their parents completed the parent proxy-report version of the Pediatric Quality of Life InventoryTM 4.0 (PedsQLTM 4.0). Independent samples t-test, one-way ANOVA, or Kruskal-Wallis H test was used to compare HROoL scores between groups. Multilevel linear regression models with random intercept were applied to analyze the relationship between socioeconomic variables and both the total score and subdomain scores.Results: The total PedsQL scores of Patent ductus arteriosus(PDA), Infantile agranulocytosis, Autoimmune thrombocytopenia(ITP), Polysyndactyly, Hirschsprung disease, Cleft lip and palate, Tetralogy of fallot, Myasthenia gravis, Guillain-barre syndrome, Glycogen storage disease(GSD), and Langerhans cell histiocytosis children were 79.65±5.46, 95.88±3.48, 71.39±3.27, 91.77±6.35, 76.18±6.92, 96.33±4.22, 77.85±8.90, 95.99±3.31, 85.77±4.56, 82.97±4.13 and 77.6±5.15, respectively. Age was significantly associated with physical functioning, school functioning, and psychosocial health scores. The household registration place was significantly related to the total score. The most urgent desire of patients was to reduce the overall medical costs.Conclusions: This study showed that patients with Patent ductus arteriosus(PDA) had the lowest physical functioning score, while patients with Autoimmune thrombocytopenia(ITP) scored the lowest in the emotional functioning, social functioning, school functioning, psychosocial health, and total scores. Incentive policies should be further adopted to improve orphan drug availability and reduce the economic burden of rare diseases.

Download Full-text

Measuring parent proxy-reported quality of life of 11 rare diseases in children in Zhejiang, China

10.21203/rs.3.rs-18385/v2 ◽

2020 ◽

Author(s):

Qisheng Gao ◽

Shanshan Wang ◽

Jianping Ren ◽

Xin Wen

Keyword(s):

Quality Of Life ◽

Patent Ductus Arteriosus ◽

Rare Diseases ◽

Ductus Arteriosus ◽

Psychosocial Health ◽

School Functioning ◽

Autoimmune Thrombocytopenia ◽

Parent Proxy ◽

Patent Ductus

Abstract Background: It becomes increasingly important to measure the health-related quality of life (HRQoL) of rare diseases in children and adolescents in recent decades. Much attention has been paid to investigate the HROoL of one specific rare disease by self-report in previous studies. This study aimed to evaluate and compare the HROoL of 11 rare diseases in Chinese children by parent proxy-report, to explore the factors associated with HROoL of patients and to know the problems of most concern.Methods: A total of 651 children aged from 2 to 18 were enrolled from the Children’s Hospital Affiliated Zhejiang University in 2018. Their parents completed the parent proxy-reports version of the Pediatric Quality of Life InventoryTM 4.0 (PedsQLTM). Independent samples t-test, one-way ANOVA, or Kruskal-Wallis H test was used to compare HROoL scores between groups. Multilevel linear regression models with random intercept were applied to analyze the relationship between socioeconomic variables and both the total score and sub-domain scores.Results: The total PedsQL scores of Patent ductus arteriosus, Infantile agranulocytosis, Autoimmune thrombocytopenia, Polysyndactyly, Hirschsprung disease, Cleft lip and palate, Tetralogy of fallot, Myasthenia gravis, Guillain-barre syndrome, Glycogen storage disease, and Langerhans cell histiocytosis children were 79.65±5.46, 95.88±3.48, 71.39±3.27, 91.77±6.35, 76.18±6.92, 96.33±4.22, 77.85±8.90, 95.99±3.31, 85.77±4.56, 82.97±4.13 and 77.6±5.15, respectively. Age was significantly associated with physical functioning, school functioning, and psychosocial health score. Gender and The household registration place was significantly associated with the overall score. The most urgent desire of patients was reducing the overall medical costs.Conclusions: These data show that Patent ductus arteriosus scores lowest in physical functioning, Autoimmune thrombocytopenia (ITP) ranks the lowest in the emotional functioning score, social functioning score, school functioning score, psychosocial health score, and total score. Incentive policies should be further taken to improve orphan drug availability and reduce the financial burden of rare diseases.

Download Full-text

Measuring parent proxy-reported quality of life of 11 rare diseases in children in Zhejiang, China

Health and Quality of Life Outcomes ◽

10.1186/s12955-020-01572-0 ◽

2020 ◽

Vol 18 (1) ◽

Author(s):

Qisheng Gao ◽

Shanshan Wang ◽

Jianping Ren ◽

Xin Wen

Keyword(s):

Quality Of Life ◽

Rare Diseases ◽

Physical Functioning ◽

Psychosocial Health ◽

Self Report ◽

School Functioning ◽

Proxy Report ◽

Parent Proxy Report ◽

Parent Proxy

Abstract Background It has become increasingly important to measure the health-related quality of life (HRQoL) of rare diseases in children and adolescents in recent decades. Much attention has been paid to investigate the HROoL of a specific rare disease by self-report in previous studies. This study aimed to evaluate and compare the HROoL of 11 rare diseases in Chinese children by parent proxy-report, to explore the factors associated with HROoL of patients, and to understand the problems of most concern. Methods A total of 651 children aged from 2 to 18 were enrolled from the Children’s Hospital Affiliated Zhejiang University in 2018. Their parents completed the parent proxy-report version of the Pediatric Quality of Life Inventory™ 4.0 (PedsQL™ 4.0). Independent samples t-test, one-way ANOVA, or Kruskal–Wallis H test was used to compare HROoL scores between groups. Multilevel linear regression models with random intercept were applied to analyze the relationship between socioeconomic variables and both the total score and subdomain scores. Results The total PedsQL scores of Patent ductus arteriosus (PDA), Infantile agranulocytosis, Autoimmune thrombocytopenia (ITP), Polysyndactyly, Hirschsprung disease, Cleft lip and palate, Tetralogy of fallot, Myasthenia gravis, Guillain–barre syndrome, Glycogen storage disease, and Langerhans cell histiocytosis children were 79.65 ± 5.46, 95.88 ± 3.48, 71.39 ± 3.27, 91.77 ± 6.35, 76.18 ± 6.92, 96.33 ± 4.22, 77.85 ± 8.90, 95.99 ± 3.31, 85.77 ± 4.56, 82.97 ± 4.13 and 77.6 ± 5.15, respectively. Age was significantly associated with physical functioning, school functioning, and psychosocial health scores. The household registration place was significantly related to the total score. The most urgent desire of patients was to reduce the overall medical costs. Conclusions This study showed that patients with PDA had the lowest physical functioning score, while patients with ITP scored the lowest in the emotional functioning, social functioning, school functioning, psychosocial health, and total scores. Incentive policies should be further adopted to improve orphan drug availability and reduce the economic burden of rare diseases.

Download Full-text

Measuring parent proxy-reported quality of life of 11 rare diseases in children in Zhejiang, China

10.21203/rs.3.rs-18385/v1 ◽

2020 ◽

Author(s):

Qisheng Gao ◽

Shanshan Wang ◽

Jianping Ren ◽

Xin Wen

Keyword(s):

Quality Of Life ◽

Patent Ductus Arteriosus ◽

Rare Diseases ◽

Ductus Arteriosus ◽

Psychosocial Health ◽

School Functioning ◽

Autoimmune Thrombocytopenia ◽

Parent Proxy ◽

Patent Ductus

Abstract Background It is increasingly important to measure the health-related quality of life (HRQoL) of rare disease in children and adolescents in recent decades. Much attention has been paid to investigate the HROoL of one specific rare disease by self-report in previous studies. This study aimed to evaluate and compare the HROoL of 11 rare diseases in Chinese children by parent proxy-report, to explore the factors associated with HROoL of patients and to know the problems of most concern. Methods A total of 651 children aged from 2 to 18 were enrolled from the Children’s Hospital Affiliated Zhejiang University in 2018. Their parents completed the parent proxy-reports version of the Pediatric Quality of Life Inventory TM 4.0 (PedsQL TM ). Independent-samples t -test, one-way ANOVA or Kruskal-Wallis H test was used to compare HROoL scores between groups. Multilevel linear regression models with random intercepts was applied to analyze the relationship between socioeconomic variables and both the total score and sub-domain scores. Results The total PedsQL scores of Patent ductus arteriosus, Infantile agranulocytosis, Autoimmune thrombocytopenia, Polysyndactyly, Hirschsprung disease, Cleft lip and palate, Tetralogy of fallot, Myasthenia gravis, Guillain-barre syndrome, Glycogen storage disease and Langerhans cell histiocytosis children were 79.65±5.46, 95.88±3.48, 71.39±3.27, 91.77±6.35, 76.18±6.92, 96.33±4.22, 77.85±8.90, 95.99±3.31, 85.77±4.56, 82.97±4.13 and 77.6±5.15, respectively. Age was significantly associated with physical functioning, school functioning and psychosocial health score. Gender and household registration place were significantly associated with overall score. The most urgent desire of patients was reducing the overall medical costs. Conclusions These data show that Patent ductus arteriosus scores lowest in physical functioning, Autoimmune thrombocytopenia (ITP) ranks the lowest in emotional functioning score, social functioning score, school functioning score, psychosocial health score and total score. Incentive policies should be further adopted to improve orphan drug availability and reduce the financial burden of rare diseases.

Download Full-text

Use of the Pediatric Quality of Life Inventory to Assess the Health-Related Quality of Life in Children with Recurrent Respiratory Papillomatosis

Annals of Otology Rhinology & Laryngology ◽

10.1177/000348940511400701 ◽

2005 ◽

Vol 114 (7) ◽

pp. 499-503 ◽

Cited By ~ 31

Author(s):

Jonathan P. Lindman ◽

Linda S. Lewis ◽

Neil Accortt ◽

Brian J. Wiatrak

Keyword(s):

Quality Of Life ◽

Psychosocial Health ◽

School Functioning ◽

Healthy Children ◽

Quality Of Life Inventory ◽

Parent Proxy Report ◽

Related Quality ◽

Health Related ◽

Parent Proxy

Objectives: The objective of this study was to use the Pediatric Quality of Life Inventory (PedsQL), a 23-question modular instrument designed to measure the health-related quality of life (HRQOL) in children and adolescents, to compare the HRQOL in children with recurrent respiratory papillomatosis (RRP) with the HRQOLs reported for healthy children and children with other chronic medical conditions. Methods: The PedsQL version 4.0 Generic Core Scales consist of 23 questions in 4 subscales (Physical, Emotional, Social, and School Functioning) for parent-proxy reporting on the HRQOL in children ages 2 to 4 years, parent reporting for children 5 to 18 years, and child self-reporting for ages 5 to 7 years (age-adjusted questions and rating scales) and 8 to 18 years. The questionnaires were administered in person or by telephone to 22 children with RRP and (or, for children 2 to 4 years of age) one parent recruited from a tertiary pediatric otolaryngology practice. The results were compared with validated norms for healthy children and scores for children whose parents reported a chronic medical condition. Results: Compared with healthy controls, the children 5 to 18 years of age with RRP self-reported a significantly (p < .05) worse HRQOL as measured by the PedsQL Total Score, Psychosocial Health score (a combination of Emotional, Social, and School Functioning Scores), and Social Functioning and School Functioning scores. Self-reported scores for the children 5 to 18 years of age with RRP compared with children with other chronic illnesses followed the same trend, and the difference approached statistical significance (p = .05) for the School Functioning Subscale scores. The parent-proxy report (for children 2 to 18 years of age) scores for children with RRP were significantly lower (p < .0001) on every scale and in total compared with scores for healthy children. Compared with scores of children with other chronic medical conditions, the parent-proxy report scores were significantly lower for psychosocial health (p = .005) and school functioning (p<.0001). Conclusions: Children with RRP report a lower quality of life than do those who are healthy, and a quality of life similar to that of those who have other chronic medical problems. The parent reports also reflect a lower quality of life for children affected by this disease, as compared with healthy children. The PedsQL demonstrated a statistically significant low HRQOL in children with RRP; however, the clinical implications of this finding appear trivial. A distinct, disease-specific survey for RRP, if developed, would have enhanced responsiveness and sensitivity to change (due to the natural course of the disease and/or surgical treatments).

Download Full-text

Quality of life in Hungarian patients with cystic fibrosis

Orvosi Hetilap ◽

10.1556/oh.2013.29612 ◽

2013 ◽

Vol 154 (20) ◽

pp. 784-791 ◽

Cited By ~ 2

Author(s):

Réka Bodnár ◽

Klára Holics ◽

Rita Ujhelyi ◽

László Kádár ◽

Lajos Kovács ◽

...

Keyword(s):

Quality Of Life ◽

Cystic Fibrosis ◽

Self Report ◽

Clinical Severity ◽

Proxy Report ◽

Parent Proxy Report ◽

Multisystemic Disease ◽

Cystic Fibrosis Questionnaire ◽

Parent Proxy

Introduction: Cystic fibrosis is a progressive multisystemic disease which affects the quality of life of patients. Aim: The aim of the study was to evaluate quality of life in Hungarian patients with cystic fibrosis. Methods: Validated Hungarian translation of The Cystic Fibrosis Questionnaire – Revised was used to measure quality of life. Clinical severity was determined on the basis of Shwachman–Kulczycki score. Lung function was measured using spirometry. Results: 59 patients were included from five centres in Hungary. The relationships between 8–13 year-old children self-report and parent proxy report was 0.77 (p<0.001) in physical functioning, 0.07 (p<0.001) in emotional functioning, 0.51 (p<0.001) in eating, 0.21 (p<0.001) in treatment burden, 0.54 (p<0.001) in body image, 0.49 (p<0.001) in respiratory symptoms and 0.40 (p<0.001) in digestive symptoms domains. Conclusions: In contrast to physical domains weak correlations were observed between answers obtained from children and their parents in psychosocial domains. The perception of both patients and their parents should be assessed when measuring quality of life in paediatric patients with cystic fibrosis. Orv. Hetil., 2013, 154, 784–791.

Download Full-text

Agreement between child self-report and parent proxy-report to evaluate Quality of Life in Children with Cancer

Psycho-Oncology ◽

10.1002/pon.828 ◽

2005 ◽

Vol 14 (2) ◽

pp. 125-134 ◽

Cited By ~ 124

Author(s):

Pi-Chen Chang ◽

Chao-Hsing Yeh

Keyword(s):

Quality Of Life ◽

Self Report ◽

Children With Cancer ◽

Proxy Report ◽

Parent Proxy Report ◽

Parent Proxy

Download Full-text

Quality of life in young patients after cone reconstruction for Ebstein anomaly

Cardiology in the Young ◽

10.1017/s1047951119000726 ◽

2019 ◽

Vol 29 (06) ◽

pp. 756-760 ◽

Cited By ~ 1

Author(s):

Kristen L. Sessions ◽

Charlotte Van Dorn ◽

Joseph A. Dearani ◽

Simrit Warring ◽

Kaitlin Leopold ◽

...

Keyword(s):

Quality Of Life ◽

Young Patients ◽

Chronic Health Conditions ◽

Health Conditions ◽

Full Time ◽

Ebstein Anomaly ◽

Proxy Report ◽

Parent Proxy Report ◽

Parent Proxy

AbstractObjective:To evaluate the health status and quality of life of young patients who had cone reconstruction for Ebstein anomaly.Methods:We reviewed all patients who had cone reconstruction from 2007 to 2016 at our institution. Prospective surveys were mailed to all eligible patients. Quality of life was assessed using the PedsQL 4.0 Generic Core Scales, including four domains: physical, emotional, social, and school functioning.Results:Of 116 eligible patients, 72 (62%) responded. About 96% reported their health as excellent or good, and 52% were symptom-free. Only 37% of patients were taking any medications, the most common of which was aspirin (30%). Only 19% had been hospitalised for cardiac reasons following cone reconstruction. The average self-reported quality of life was 85.3/100, whereas the average parent proxy-reported quality of life was 81.8/100. There was no difference by self or parent proxy-report in quality of life between cone reconstruction patients and healthy children; however, quality of life was significantly better compared with children with other chronic health conditions. By self-report and parent proxy-report, 15.1 and 16.7% of patients were deemed “at risk” for reduced quality of life, respectively. Socially, 63/64 (98%) patients over 5 years old were either full-time students or working full-time.Conclusion:Children with Ebstein anomaly following cone reconstruction have excellent quality of life comparable with healthy peers and significantly better than other children with chronic health conditions. Families of children with Ebstein anomaly can expect excellent quality of life, long-term health status, and social functioning following cone reconstruction.

Download Full-text