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Prepubertal growth and skeletal maturation in children with sickle cell disease
Insights
Children with sickle cell (SS) disease show significant growth deficits by age 2, impacting height and weight. Hemoglobin SC disease does not affect growth in children.
Area of Science:
- Pediatrics
- Genetics
- Hematology
Background:
- Sickle cell disease (SCD) encompasses genetic hemoglobinopathies with varying clinical severity.
- Homozygous sickle cell (SS) disease and hemoglobin SC (SC) disease are common forms with distinct pathophysiologies.
- Understanding growth patterns in pediatric SCD is crucial for clinical management and predicting long-term outcomes.
Purpose of the Study:
- To longitudinally assess and compare physical growth (weight, height) and skeletal maturity in children with SS disease and SC disease against normal controls.
- To establish growth standards for pediatric SCD populations.
- To investigate the early impact of SCD genotypes on physical development.
Main Methods:
- Longitudinal study of 298 children with SS disease and 157 with SC disease (birth to 9 years).
- Comparison with 231 age- and sex-matched children with normal hemoglobin (AA) genotype.
- Radiologic assessment of bone age at the wrist at 5 and 8 years for skeletal maturity.
Main Results:
- Children with SC disease exhibited growth comparable to normal (AA) children.
- Children with SS disease showed statistically significant, progressive deficits in weight and height starting before 2 years of age.
- By 9 years, SS disease resulted in an average deficit of approximately 1 standard deviation below the normal mean for age. Skeletal maturity was significantly retarded in SS disease at 8 years, but not at 5 years.
Conclusions:
- SS disease has an early and significant negative impact on physical growth and skeletal development in children.
- SC disease appears to have a benign effect on prepubertal growth, supporting its milder clinical profile.
- These findings provide essential data for clinical expectations and understanding the etiology of growth impairment in SCD.
Abstract:
In a longitudinal study of 298 children with homozygous sickle cell (SS) disease and 157 children with hemoglobin SC disease, between birth and 9 years of age, observations of weight and height were made. These were compared with similar data derived from an age- and sex-matched group of 231 children with a normal hemoglobin (AA) genotype. Growth in children with SC disease was not significantly different from that in normal children, but children with SS disease had statistically significant, and progressive, deficits in both weight and height before 2 years of age. The average deficit approached 1 SD below the normal mean for age by 9 years. Observations of skeletal maturity, based on radiologic assessment of bone age at the wrist, were made on a proportion of these children at 5 and 8 years of age. Children with SS disease were significantly retarded at 8 years but not 5 years, which is consistent with increasing deficit in height. These observations confirm the early impact of SS disease on physical development and provide standards from which clinical expectations of growth may be derived. The relevance of these findings and their relationship to the characteristic delay in pubertal development is discussed together with a review of possible etiologic factors. The benign nature of SC disease is endorsed by the absence of an effect on growth in the prepubertal child.