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Impact of long-term iron chelation therapy on growth and endocrine functions in thalassaemia

Vincenzo De Sanctis1, Malgorzata Roos, Theo Gasser

  • 1Department of Reproduction and Growth, Paediatric and Adolescent Unit, S Anna Hospital, Ferrara, Italy. vdesanctis@libero.it

Journal of Pediatric Endocrinology & Metabolism : JPEM
|June 9, 2006
PubMed

Insights

Daily desferrioxamine (DFX) therapy in beta-thalassaemia major patients improved sexual maturation and endocrine issues. However, short stature remained a persistent complication despite advancements in treatment.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Hematology

Background:

  • Children with beta-thalassaemia major treated with modern therapies are now reaching adulthood.
  • Evaluating long-term complications of iron overload is crucial.
  • Daily subcutaneous desferrioxamine (DFX) therapy's impact on preventing these complications needs assessment.

Purpose of the Study:

  • To evaluate the effects of early-life desferrioxamine (DFX) chelation therapy on growth and endocrine complications in beta-thalassaemia major patients.
  • To assess the prevention of iron overload-related complications through DFX therapy.

Main Methods:

  • A multi-centre study involving 238 patients aged 2-17 years with beta-thalassaemia major.
  • Evaluation of height, weight, endocrine status, hematological variables, and DFX compliance.
  • Statistical analysis using the LMS method and Mann-Whitney test.

Main Results:

  • 12.4% of patients had growth hormone insufficiency, 2.1% primary hypothyroidism, and 1.7% hypoparathyroidism.
  • Delayed puberty affected 18.4% of boys and 17.7% of girls.
  • While initial height was normal, a progressive decline in growth was observed over years; DFX positively impacted sexual maturation and endocrine complications.

Conclusions:

  • Daily subcutaneous desferrioxamine (DFX) therapy shows a positive effect on sexual maturation and endocrine complications in beta-thalassaemia major.
  • Short stature remains a persistent challenge despite advances in DFX treatment.
  • Early initiation of chelation therapy is vital for managing beta-thalassaemia major complications.

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