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Published on: January 29, 2018
Normal lumbar bone mineral density in optimally treated children and young adolescents with beta-thalassaemia major
Athanasios Christoforidis1, Eirini Kazantzidou, Ioanna Tsatra
1Thalassaemia Unit, 1st Paediatric Department, Aristotle University of Thessaloniki, Greece. christoforidis@doctors.org.uk
Insights
Optimal treatment for beta-thalassaemia major prevents bone density loss in young patients. Regular monitoring is crucial for maintaining bone health into adulthood and improving quality of life.
Area of Science:
- Pediatrics
- Hematology
- Endocrinology
Background:
- Beta-thalassaemia major is linked to osteopenia/osteoporosis due to complex factors.
- Decreased bone mineral density (BMD) is frequently observed even in young patients.
- This study investigates BMD in children and young adults with beta-thalassaemia major receiving standard care.
Purpose of the Study:
- To assess bone mineral density (BMD) in children and young adults with beta-thalassaemia major.
- To correlate BMD with auxological, clinical, and laboratory parameters.
- To evaluate the effectiveness of regular transfusions and chelation therapy on bone health.
Main Methods:
- Studied 35 young patients (5-20 years) with beta-thalassaemia major.
- Assessed lumbar BMD using dual-energy X-ray absorptiometry (DXA).
- Calculated Z-scores against age- and sex-matched normal populations; excluded patients with endocrinopathy or hormonal therapy.
Main Results:
- All patients had normal BMD Z-scores (mean 0.42 for girls, -0.41 for boys).
- A statistically significant gender difference in Z-scores was observed (p=0.018).
- A decline in Z-scores with age indicated delayed bone mass acquisition in thalassaemic patients compared to controls.
Conclusions:
- Effective conventional treatment prevents osteopenia/osteoporosis in young beta-thalassaemia major patients.
- Close surveillance, screening, and early management of endocrine complications are vital.
- Ensuring normal bone health in adulthood improves the quality of life for individuals with beta-thalassaemia major.
Objective:
Osteopenia/osteoporosis of multi-factorial pathogenetic mechanism is reported to be a significant cause of morbidity in adult patients with beta-thalassaemia major. Even in young patients, decreased Bone Mineral Density (BMD) values are a consistent finding in the literature. This study was performed in order to assess BMD in children and young adults with beta-thalassaemia major, regularly transfused and sufficiently chelated, along with auxological, clinical and laboratory parameters.
Design:
Thirty-five young thalassaemic patients (19 F, 16 M, aged 5-20 yr) were studied. Lumbar BMD was assessed by dual X-ray absorptiometry (DXA) and Z-scores were calculated according to bone density values using age- and sex-matched normal population. None of the patients presented with clinical or laboratory signs of endocrinopathy and none was receiving hormonal replacement therapy.
Results:
All BMD Z-scores were within normal range, with a mean Z-score of 0.42 for girls and -0.41 for boys (statistically significant gender difference, p=0.018). When correlated with age, a decline in Z-scores was observed, indicating a delay in bone mass acquisition with advancing age in the thalassaemic group compared to controls.
Conclusions:
Optimal conventional treatment prevents the manifestation of osteopenia/osteoporosis during the first two decades of life in patients with beta-thalassaemia major. However, close surveillance with regular screening, preventive intervention and early management of possible endocrine complications are essential in order to secure normal bone health during adulthood and improve quality of life in the thalassaemic population.
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