Quality of Life Worsens in Older Children with Sickle Cell Disease

Maite Houwing, MD

Young children with sickle cell disease generally report worse metrics for health-related quality of life (QoL) than those without the hematologic disease, according to new findings from an Amsterdam-based study.

New cross-sectional data analysis from the Erasmus University Medical Centers and Emma Children’s Hospital in The Netherlands suggest children may begin to experience QoL burdens due to sickle cell disease once they’ve reached age 5 and beyond. The investigators stressed the significance of timely sickle disease diagnosis in young children, as this may help to better prepare clinicians in addressing burdensome effects and symptoms of the disease in their youngest patients.

Led by Maite E. Houwing, MD, investigators sought to better interpret the health-related QoL of young children living with sickle cell disease in their country. There are approximately 1500 cases of sickle cell disease in The Netherlands—half of which are in children. Though clinical advances in European and North American countries have given children with sickle cell disease a significantly decreased risk of mortality, issues persist in fully treating the disease.

“With prolongation of survival, greater attention must be focused on outcomes beyond survival including health-related QoL to assess the multidimensional impact of sickle cell disease on patient's overall well-being,” they wrote.

Houwing and colleagues conducted a retrospective, cross-sectional analysis of QoL evaluations in children aged 0-7 years old with sickle cell disease being treated at either health center. Their assessment included the TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL) for infants aged 0-1 years old and the Pediatric Quality of Life Inventory (PedsQL) for children aged 2-7 years old.

Children eligible for assessment were treated with a caregiver at either center between April 2012 and October 2020. The analysis included 136 caregivers and 136 children. Over the observed time period, their data included 46 infants (0-1 year old), 19 toddlers (2-4), and 93 young children (5-7).

A total of 46 patients (33.8%) completed the TAPQOL and 112 (82.4%) completed the PedsQL; 22 (16.2%) completed both evaluations.

Investigators observed that infants and toddlers with sickle cell generally did not fare significantly worse than the general population in QoL scores; infants with sickle cell scored significantly lower on TAPQOL’s skin and positive mood measures, but no other differences were reported.

Young children with sickle cell disease, however, scored significantly lower than the general pediatric population on PedsQL total score (P <.001) and 4 of the evaluation’s 5 subscales:

• Psychosocial health (P <.001)
• Physical health (P <.001)
• Social functioning (P <.005)
• School functioning (P <.001)

The team additionally observed a correlation between higher age and lower health-related QoL scores among children with sickle cell disease. Previous trial findings, they noted, have supported that correlation up to age 18.

They hypothesized that lower health-related QoL scores in older children with sickle cell disease may be due to their “growing into deficit,” a clinical phenomenon generally described in pediatric chronic diseases.

“While children may function normally at a very young age, over time with increasing day-to-day demands, they may fail to acquire normal, age-appropriate developmental milestones,” they explained. “As a result, psychological problems may become more visible over time when higher cognitive functions, such as emotion regulation, are required.”

Though the study had apparent limitations in lack of causal relationship interpretation and selection bias due to the required Dutch- and/or English-speaking requirements, Houwing and colleagues concluded the findings lend to the need for prioritized pediatric sickle cell diagnosis, as well as future assessment of the specific burdens of the disease in children such as vaso-occlusive crises.

“The recognition of the specific areas that require appropriate interventions will help enable targeting of resources to improve quality of life for children with sickle cell disease,” they concluded.

The study, “Health-related quality of life in infants, toddlers and young children with sickle cell disease,” was published online in Pediatric Blood & Cancer.