Health-Related Quality of Life Assessments by Children and Adolescents with Sickle Cell Disease and Their Parents in Portugal.

Clara Abadesso,Maria Céu Machado,G Allen Finley,Susana Pacheco

doi:10.3390/children9020283

Clara Abadesso, Maria Céu Machado + Show 2 more

Open Access

https://doi.org/10.3390/children9020283

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Abstract

Health-Related Quality of Life (HRQL) can be used to measure the impact of Sickle Cell Disease (SCD) on the child and their family and is generally reduced. No research has yet measured HRQL in Portuguese pediatric SCD patients. Objectives: (1) Describe and compare HRQL of children with SCD reported by them and their parents; (2) Compare with a pediatric population with no SCD; (3) Find predictive factors of HRQL in SCD children. Methods: Descriptive, case-control study that included sixty-eight children and adolescents with SCD (aged 3 to 18 years) and their parents. Control group—children with no SCD, matched by age, gender and ethnic background. HRQL was assessed using the multidimensional self-report PedsQL® 4.0 Generic Scales. Summary scores for overall HRQL and subscale scores for physical, emotional, social and school functioning were compared within groups (children-parents) and with the control group. Clinical and socio-demographic variables were analyzed to find predictive factors of HRQL in pediatric SCD patients. Results: Children with SCD and their parents had significantly lower overall and all subdomains of HRQL, compared with the control group. Children with SCD also rated lower when compared with their parents (only significant for social functioning), with low to moderate correlations. Children and parent reports declined with increasing age. Higher pain frequency was associated with worse total and psychosocial domains of HRQL. The number of hospitalizations was a predictor of worse school score, and female gender was a predictor of worse emotional score. Conclusions: SCD significantly affects children’s HRQL. Parents can provide a good proxy report, although both evaluations are beneficial. Disease status, like number of hospitalizations and frequency of pain, influences HRQL. Interventions in SCD should consider improvements in HRQL as an important outcome.

Highlights

Sickle Cell Disease (SCD) is one of the most common genetic disorders in African descent population and is characterized by chronic hemolytic anemia, recurrent painful acute episodes and multiple complications
The number of hospitalizations was a predictor of worse school score, and female gender was a predictor of worse emotional score
For children above 5 years a total of 57 parent–child pairs were studied; two reports were only answered by the child a total of 57 parent–child pairs were studied; two reports were only answered by the child and one was onlyand answered by parents (Figure 1)

Summary

Introduction

Sickle Cell Disease (SCD) is one of the most common genetic disorders in African descent population and is characterized by chronic hemolytic anemia, recurrent painful acute episodes and multiple complications. It requires acute care visits, frequent hospitalizations and contributes to increased morbidity and early mortality [1]. The acute and chronic complications lead to predictable disease-related symptoms that significantly impact patient’s well-being [3]. The patient’s perspective on health and well-being has become an accepted method to determine the impact of the disease and its treatment [4], as a Patient-Reported Outcome (PRO), and it has been widely used in clinical practice and research [4,5]

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