Abstract
BackgroundIn young children, β-thalassemia major (β-TM) is associated with potentially severe clinical characteristics, including poor growth, feeding difficulties, hepatosplenomegaly, bone metabolic disorders, and skeletal abnormalities.MethodsIn this study, we reviewed the demographic and clinical characteristics (e.g., age, sex, duration of blood transfusion and chelating therapy, and vitamin supplementation) and serum biomarker levels (e.g., iron accumulation, bone metabolism, liver, kidney, and thyroid function markers) of 32 patients that received regular blood transfusion at a single center in southern China with the aim of stratifying the risk of severe complications such as osteopenia, endocrinopathies, and multi-organ failures.ResultsAlthough all patients exhibited moderately to strongly elevated serum ferritin levels, this biomarker was significantly higher in children older than ≥5 years, compared to younger children (*p < 0.05, 1512 ± 192.6 vs. 2337 ± 299.8 ng/ml, Mann-Whitney U test). Older children had a significantly lower 25-hydroxy vitamin D3 (25(OH)D3) level, compared to younger children (**p < 0.01, 34.25 ± 11.06 vs. 23.05 ± 9.95 ng/ml, Mann-Whitney U test). No age-related differences were observed in serum calcium, phosphorus, and PTH levels. Regarding liver function, the serum alanine aminotransferase (ALT) level was significantly increased in children older than ≥5 years, compared to younger children (*p < 0.05, 19.17 ± 2.44 vs. 43.45 ± 9.82I U/ml, Mann-Whitney U test). However, no age-related differences were observed in the serum levels of other liver or kidney and thyroid biomarkers.ConclusionsOur results suggest that in older children, hepatic iron overload may be associated with a low serum concentration of 25(OH)D3, an indicator of vitamin D deficiency and altered bone metabolism. Iron accumulation may also be associated with a higher concentration of ALT, a sensitive marker of liver malfunction. These findings may provide important clinical indications of the need for intervention to prevent severe complications in children with β thalassemia.
Highlights
In young children, β-thalassemia major (β-TM) is associated with potentially severe clinical characteristics, including poor growth, feeding difficulties, hepatosplenomegaly, bone metabolic disorders, and skeletal abnormalities
We retrospectively reviewed the data of 32 children who visited Shenzhen Children’s Hospital (SZCH) for regular blood transfusions between January and June 2018
We evaluated serum biomedical markers of liver function, bone metabolism, kidney function, and thyroid function and compared these values to the normal ranges according to the laboratory standards of the medical diagnostic lab at SZCH
Summary
Β-thalassemia major (β-TM) is associated with potentially severe clinical characteristics, including poor growth, feeding difficulties, hepatosplenomegaly, bone metabolic disorders, and skeletal abnormalities. Thalassemia is usually asymptomatic or associated with only mild anemia, patients with severe disease require lifelong blood transfusions for survival [1]. Patients with β-TM are homozygous or compound heterozygous carriers of beta0 or beta+ genes [2]. Most such patients develop symptoms of β-TM during early childhood, generally between the ages of 6 and 24 months. These clinical symptoms may include poor growth, feeding difficulties, hepatosplenomegaly, bone metabolic disorders, and skeletal abnormalities. Children with β-TM must receive regular blood transfusions to prevent severe complications and maintain normal physiological growth [2, 3]
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