Quality of Life of Developmentally Normal Children With Epilepsy and Their Siblings.

BACKGROUND: Quality of life (QOL) is a fundamental and multidimensional concept that should be considered with health problems, specifically chronic diseases, such as epilepsy. There have been limited studies on how pediatric epilepsy impacts the QOL of siblings of affected individuals. Hence, we studied the impact of epilepsy on the QOL of affected children and their siblings. OBJECTIVE: This study aimed to assess the QOL of developmentally normal children with epilepsy and their siblings and the association of QOL scores with the clinicodemographic profile. METHODS: This study was conducted at the University College of Medical Sciences and Guru Tegh Bahadur Hospital, New Delhi, India, a tertiary care hospital. The QOL of children (4-12 years) with epilepsy was assessed using epilepsy-specific questionnaires, i.e., Quality of Life in Childhood Epilepsy Questionnaire-55 (QOLCE-55), which covers the cognitive, emotional, social, and physical domains, and Pediatric Quality of Life Epilepsy Module (Peds QL EM), which covers the impact, cognitive, sleep, executive, and mood/ behavior domains. QOL in siblings was assessed using the Peds QL Inventory, which covers the following domains: physical, emotional, social, and school. The principal investigator administered these questionnaires to parents in Hindi/ English. Scoring was done as per standard instructions of the questionnaire. Clinical and demographic data were recorded in a pro forma. RESULT: The median QOLCE-55 score was 81.12, with a range of 74.65-86.34, and the median Peds QL EM score was 89.31, with a range of 75.58-94.48. Overall, Cronbach's alpha of QOLCE-55 and Peds QL EM was >0.8. Breakthrough seizures (≥10) affected the overall QOL (p=0.001) and all domains of QOLCE-55 (except emotional function (p=0.44)) and Peds QL EM (except sleep/fatigue domain (p=0.59)). Age, sex, parental education, socioeconomic status, and type of epilepsy did not affect the overall QOL (p>0.05). The QOL of siblings was not affected as per the Peds QL Inventory score (median score 100) and self-made questionnaire. CONCLUSION: Our results suggest that the QOL of children with epilepsy was compromised, whereas the QOL of their siblings was not affected.

Mapping the Peds QLTM 4.0 onto CHU-9D: a cross-sectional study in functional dyspepsia population from China.

Objective: The study aims to develop a mapping algorithm from the Pediatric Quality of Life Inventory™ 4. 0 (Peds QL 4.0) onto Child Health Utility 9D (CHU-9D) based on the cross-sectional data of functional dyspepsia (FD) children and adolescents in China. Methods: A sample of 2,152 patients with FD completed both the CHU-9D and Peds QL 4.0 instruments. A total of six regression models were used to develop the mapping algorithm, including ordinary least squares regression (OLS), the generalized linear regression model (GLM), MM-estimator model (MM), Tobit regression (Tobit) and Beta regression (Beta) for direct mapping, and multinomial logistic regression (MLOGIT) for response mapping. Peds QL 4.0 total score, Peds QL 4.0 dimension scores, Peds QL 4.0 item scores, gender, and age were used as independent variables according to the Spearman correlation coefficient. The ranking of indicators, including the mean absolute error (MAE), root mean squared error (RMSE), adjusted R2, and consistent correlation coefficient (CCC), was used to assess the predictive ability of the models. Results: The Tobit model with selected Peds QL 4.0 item scores, gender and age as the independent variable predicted the most accurate. The best-performing models for other possible combinations of variables were also shown. Conclusion: The mapping algorithm helps to transform Peds QL 4.0 data into health utility value. It is valuable for conducting health technology evaluations within clinical studies that have only collected Peds QL 4.0 data.

Assessing quality of life in pediatric fibrous dysplasia and McCune Albright syndrome: PEDS-QL and HADS data from the Fibrous Dysplasia Foundation Patient Registry.

PURPOSE: The International FD/MAS Consortium recently encouraged using the Pediatric Quality of Life Inventory (PEDS-QL) and the Hospital Anxiety and Depression scales (HADS) in clinical care. This study examines scores on these measures among pediatric fibrous dysplasia and McCune Albright (FD/MAS) patients to initiate consideration of their use in clinical treatment. METHODS: This is a retrospective analysis of pediatric data from 39 minors, ages 2-17, entered in the Fibrous Dysplasia Foundation Patient Registry from July 2016 to December 2018. Sample means and score distributions are compared to general population and chronic disease benchmarks. Associations with medical and demographic variables are also explored. RESULTS: Mean PEDS-QL scores for children 2-7 were inconclusive in determining at risk status for impaired quality of life (QOL). Individual score distributions suggested up to half experienced extensive physical or social impairment. Means and individual score distributions for the physical and psychosocial components of the PEDS-QL for children 8-17 suggested many were at risk of impaired QOL. Over half of 13-17 year-olds met the clinical benchmark for anxiety. Older males scored better than females on the PEDS-QL and HADS. Pain frequency was associated with physical function for older children. CONCLUSIONS: Older children with FD/MAS may be more compromised in terms of psychosocial QOL than previously reported. Clinicians should be attentive to the influence of gender on QOL in older children. Online patient registries associated with rare diseases have the potential to serve as efficient and cost-effective mechanisms to jumpstart examination of new measures in consideration for clinical use.

What is the future of patient-reported outcomes in sickle-cell disease?

INTRODUCTION: Sickle cell disease (SCD) is a complex, chronic disease caused by abnormal polymerization of hemoglobin, which leads to severe pain episodes, fatigue, and end-organ damage. Patient reported outcomes (PROs) have emerged as a critical tool for measuring SCD disease severity and response to treatment. AREAS COVERED: Authors review the key issues involved when deciding to use a PRO in a clinical trial. We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries.

Faktor-faktor yang Mempengaruhi Psikososial Pesakit Kanakkanak Talasemia

@#Introduction Thalassemia disease is a serious health problem as it gives psychosocial burden not only to the patients but also to the patients’ ecosystem. Among the difficulties faced by patients are psychosocial problems, especially in the process patients’ growth and development. Therefore, this study examines what factors can influenced the psychosocial of Thalassemic patients.. Methods A cross-sectional study was carried out among 161 of Thalasemia Major children at Women and Children Hospital, Likas and Keningau District Hospital, Sabah from February to April 2013. The validated PedsQL 4.0 Generic Core Scale and Social Support questionnaire has been used. Results The results showed that the overall psychosocial score was 68.50 (SP 15.18) with the lowest average school psychosocial score of 58.38 (SP 17.82). Only gender factor showed a significant relationship with psychosocial score of emotional function. There is no significant relationship noted between other sociodemographic factors with overall psychosocial scores or with the psychosocial scores of each function. In relation to subscale social support factors, there was a significant relationship between the psychosocial scores of each function and overall with social support factors of parents, close friends, classmates and teachers. The results of linear regression multivariate analysis suggest that social support factors from classmates is a significant factor in the psychosocial impact of Thalassemia Major pediatric patients. Conclusions In conclusion, social support factors primarily from peers are very important in influencing the psychosocial of Thalassemia Major children in overcoming their life's functionality like other normal children

Health related quality of life and family impact of type 1 diabetes among adolescents in Saudi Arabia.

AIM: To measure the health related quality of life (HRQoL) among Saudi Arabian adolescents with type 1 diabetes mellitus (T1DM) and the impact the disease has on the family. METHODOLOGY: A cross sectional study was conducted involving 315 adolescent patients (12-18 years) and their caregivers. Adolescent HRQoL was assessed by adolescents and their parents completing the Peds QL™ Diabetes Module 3.0. Family impact was assessed by the parent completing the Peds QL™ Family Impact module (FIM). RESULTS: Adolescents reported a cumulative mean HRQoL score of 64.8, while parents reported significantly lower scores of 60.3 (p=0.003). The lowest scores reported by both adolescents and parents were for "Worry". Female gender and late adolescent age were predictors of lower HRQoL for adolescents with T1DM. The FIM showed low scores for "Emotional functioning" (59.8) and high scores for "Family relationships" (80.9). CONCLUSION: These findings emphasize the importance of an interdisciplinary, biopsychosocial and family centered care approach to adolescents with a chronic disease. Future work could assess the effectiveness of direct care involvement of adolescent and mental health experts in improving the HRQoL for this population.

Osteogénesis imperfecta: estudio de la calidad de vida en los niños / Osteogenesis imperfecta: quality of life in children

La calidad de vida relacionada con la salud (CVRS) en los niños con osteogénesis imperfecta (OI) ha sido poco comunicada. Se la evaluó con el cuestionario Peds QL versión 4.0, en 65 familias y 42 niños, con una media de edad de 7,76 años (35 con OI de tipo I y 30 con OI de tipo III-IV). Hubo diferencias signifcativas en el dominio físico según los niños y los padres, entre los niños con OI de tipo I y aquellos con OI de tipo III-IV. En el dominio social solo los padres consideraron menor CVRS en las formas III-IV. En el análisis multivariado de las formas graves, para los padres la mejor CVRS se asoció con menor défcit de estatura (coef. β = 3,8; p= 0,039), menor número de fracturas (coef. β= 0,69; p= 0,003) y mayor dosis de pamidronato (coef. β= 1,44; p= 0,037). Para los niños, la CVRS fue mejor cuando hubo adherencia al tratamiento (coef. β= 19,41; p= 0,03).

The impact produced by osteogenesis imperfecta in childrens' quality of life almost has not been reported; 65 children, 38 males, were evaluated according to the questionnaire PedsQL 4.0 Argentinean Spanish version. Median age was 7.76 years; 35 children with OI type I were compared with 30 type III-IV (according to Sillence Classifcation) fnding signifcant difference in the physical domain in both visions, children and parents, and only in parents' vision in the social area. Multivariate analysis showed an association between better PedQL scores and treatment compliance (coef. β= 19.41 p= 0.03) in children's vision. In parental report on the other hand, the association was found with greater pamidronate doses (coef. β 1.44 p=0.037), lower height compromise (coef. β= 3.8; p= 0.039) and less number of fractures (coef. β= 0.69; p= 0.003).

Osteogénesis imperfecta: estudio de la calidad de vida en los niños / Osteogenesis imperfecta: quality of life in children

La calidad de vida relacionada con la salud (CVRS) en los niños con osteogénesis imperfecta (OI) ha sido poco comunicada. Se la evaluó con el cuestionario Peds QL versión 4.0, en 65 familias y 42 niños, con una media de edad de 7,76 años (35 con OI de tipo I y 30 con OI de tipo III-IV). Hubo diferencias signifcativas en el dominio físico según los niños y los padres, entre los niños con OI de tipo I y aquellos con OI de tipo III-IV. En el dominio social solo los padres consideraron menor CVRS en las formas III-IV. En el análisis multivariado de las formas graves, para los padres la mejor CVRS se asoció con menor défcit de estatura (coef. β = 3,8; p= 0,039), menor número de fracturas (coef. β= 0,69; p= 0,003) y mayor dosis de pamidronato (coef. β= 1,44; p= 0,037). Para los niños, la CVRS fue mejor cuando hubo adherencia al tratamiento (coef. β= 19,41; p= 0,03).(AU)

The impact produced by osteogenesis imperfecta in childrens quality of life almost has not been reported; 65 children, 38 males, were evaluated according to the questionnaire PedsQL 4.0 Argentinean Spanish version. Median age was 7.76 years; 35 children with OI type I were compared with 30 type III-IV (according to Sillence Classifcation) fnding signifcant difference in the physical domain in both visions, children and parents, and only in parents vision in the social area. Multivariate analysis showed an association between better PedQL scores and treatment compliance (coef. β= 19.41 p= 0.03) in childrens vision. In parental report on the other hand, the association was found with greater pamidronate doses (coef. β 1.44 p=0.037), lower height compromise (coef. β= 3.8; p= 0.039) and less number of fractures (coef. β= 0.69; p= 0.003).(AU)