Assessing Impact on Quality of Life and Fatigue in Children With Sickle Cell Disease: A Comparison of Child and Parent-Reported Outcomes
Sahar Mahate, Jessica Chicola, Eena Lin, Lindsey Nguyen, Trina Basu, Margarita Cubbler, Krystal Hunter, Christian Bruni, April Douglass-Bright, Rafat AhmedAbstract
Background
Sickle cell disease (SCD) is an autosomal recessive genetic disease characterized by the hemoglobin variant known as hemoglobin S (HbS), which causes red blood cells (RBCs) to assume a characteristic crescent or “sickle” shape. These sickle-shaped RBCs cause vascular obstructions, or vaso-occlusion in capillaries, which would manifest as intense pain. SCD is a lifelong and painful disease, yet there is insufficient literature regarding the impact of SCD on pediatric patients’ quality of life (QOL). A literature review identifies only 5 articles evaluating the experience of pediatric patients with SCD. Among the studies, greater age was a consistent predictor of worsening QOL, as well as female gender, increased social determinants of health, and socioemotional problems. However, none of these studies take a prospective approach to characterizing the QOL of pediatric patients with SCD; our study aims to fill that gap. The primary objective is to evaluate QOL and fatigue among pediatric patients with and without SCD. Secondary objectives include identifying factors that influence QOL and fatigue in these populations and assessing congruency of QOL and fatigue scores among patients and parents.
Methods
QOL and fatigue are evaluated using the PedsQL Sickle Cell Module and the PedsQL Multidimensional Fatigue Scale, including child self-report and parent proxy-report forms, which were reverse-scored and transformed to a 0-100 scale. Patients, aged 5 – 18 years, and their parents completed surveys recruited from the Pediatric Hematology/Oncology and General Pediatric clinics at Cooper University Hospital from Oct – Dec 2025.
Results
A total of 91 participants were included in the analysis, including 43 children with SCD and 48 controls without chronic illness. Child participants range in age from 5 to 18 years, with a mean age of 10 years. Children with SCD demonstrated significantly lower quality of life (QOL) scores compared to controls (mean 57.90 ± 22.14 vs. 92.78 ± 9.70, p < 0.001). Fatigue scores were significantly higher among children with SCD (2.62 ± 1.21) compared to controls (1.56 ± 1.01, p < 0.001). There were no significant differences in QOL or fatigue scores across age groups (QOL: p = 0.579; fatigue: p = 0.190). Additionally, no significant differences were observed between child-reported and parent-reported QOL (75.65 ± 23.36 vs. 76.97 ± 25.22, p = 0.796) or fatigue scores (2.11 ± 1.29 vs. 2.00 ± 1.16, p = 0.675). Significant associations were identified between SCD status and demographic variables. Children with SCD were more likely to identify as Black (95.3%, p < 0.001). Ethnicity also differed significantly between groups, with patients who identified as Hispanic less likely to have a diagnosis of SCD (71.4% vs 28.6%, p < 0.001). Hospitalizations and emergency department visits were significantly higher among children with SCD (p = 0.012), with 30.4% of SCD patients reporting three visits compared to only 3.3% of controls reporting three hospitalizations.
Conclusions
Preliminary findings suggest that pediatric patients with SCD experience significantly lower quality of life and greater fatigue compared with healthy controls. Further analysis will include comparisons of QOL and fatigue scores between SCD and healthy children stratified by significant demographic variables. Understanding how demographics influence QOL and fatigue can also allow for improvement of management and resources in patients with SCD. Prioritizing fatigue management and improvement of QOL is central to improving long-term health outcomes for children living with SCD.