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ASCAT 2024 | Addressing the growth and development challenges in thalassemia
Maddalena Casale, MD, University of Campania Studies “Luigi Vanvitelli,” Naples, Italy, discusses the growth and development challenges facing pediatric patients with thalassemia, particularly those who are transfusion-dependent and at higher risk for complications due to iron overload. Dr Casale highlights two key priorities: improving the prediction of endocrine complications beyond traditional iron overload markers and addressing the disparity in growth disorders between children with thalassemia in low- and high-resource countries. This interview took place at the 19th Annual Scientific Conference of the Academy for Sickle Cell and Thalassaemia (ASCAT 2024) in London, UK.
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Transcript
Thalassemia and especially transfusion-dependent patients have the higher risk of growth and development complications, basically due to transfusion and iron toxicity. But giant strides have been made since children with thalassemia developed severe growth and development disorders to now, when at least in high income countries most children with thalassemia have normal growth and development.
In my opinion, there are two main challenges to prioritize nowadays...
Thalassemia and especially transfusion-dependent patients have the higher risk of growth and development complications, basically due to transfusion and iron toxicity. But giant strides have been made since children with thalassemia developed severe growth and development disorders to now, when at least in high income countries most children with thalassemia have normal growth and development.
In my opinion, there are two main challenges to prioritize nowadays. First, we know that traditional iron overload parameters are correlated to endocrine complications, but they are not able to predict the onset of a new endocrine complication since even patients with optimal iron balance develop new endocrine complication or growth disorders. This is a great challenge for preventing these complications, which are the most common disorders in thalassemia nowadays.
And for the second challenge, I want to use the slogan of this ASCAT 2024, Achieving Equitable Progress. This is another priority considering the huge differences in the rates of growth and development disorder in children living in low-resource countries and those living in high-resource countries. We know this is a problem related to the different organizations of the health services in different countries. However, we must work to create the conditions so that every child can benefit from the basic level of assistance that reduces the onset of irreversible complications, such as short stature, puberty delay, hypogonadism and infertility. Simple and reliable oxylogical assessments should be provided to any child at any centre, referring to more intensive care those children with growth and development disorders.
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