Background: Relatively little is known about endocrine function, bone mineral health, and growth during oral iron chelation therapy (OIC) in β-thalassemia major patients (BMT) on treatment with deferasirox.
Aims of the study: To measure the final adult standing height (FA-Ht) and the frequency of endocrine complications in relation to their liver iron content (LIC) and insulin-like growth factor 1 (IGF-I) concentration. Patients were grouped into two groups according to their iron chelation therapy.
Patients and methods: The first group (Group A; 15 patients, 6 females and 9 males) received oral iron chelation therapy (OIC) with deferasirox for 6 years before puberty; the second group (Group B; 40 patients) attained the FA-Ht before the use of OIC (iron chelation therapy with deferoxamine (DFO) given subcutaneously, since the age of 2 years). In both groups LIC was measured using FerriScan R2-MRI method.
Results: Patients with BTM who received OIC for 6 years or more before their end of growth were significantly taller and had lower LIC assessed by FerriScan R2-MRI, and lower fasting glucose level (FBG) and liver enzymes (ALT and AST) concentration, and higher IGF-1 SDS versus those who did not receive OIC before attaining FA-Ht. The prevalence of endocrinopathies, including hypothyroidism, impaired fasting glucose and hypogonadism were significantly lower in Group A versus Group B. The IGF-1-SDS did not differ between the two groups. Neither ferritin level nor IGF-1 concentrations were correlated with the Ht-SDS.
|Group A: OIC||Group B: No OIC|
|Number of patients||15||40|
|HtSDS < −-2||6.6%||52.5%*|
|DM: Diabetes mellitus, IFG: Impaired fasting glucose, IGF-1: insulin growth factor-1; SDS: standard deviation score; (*P<0.05).|
Conclusions: The use of OIC years before the end of puberty was associated with a significantly lower prevalence of endocrinopathies, lower LIC and higher FA-Ht. Proper blood transfusion and early use of intensive oral chelation can improve the final height of patients with thalassemia major.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology