ESPE Abstracts (2018) 89 P-P2-286

ESPE2018 Poster Presentations Multisystem Endocrine Disorders P2 (17 abstracts)

Final Adult Height, Insulin-Like Growth Factor 1 (IGF-I) Concentration and Endocrine Complications in Adolescents and Young Adults with β-Thalassemia Major (BTM) Who Received Oral Iron Chelation (OIC) in Comparison with Those Who Did Not Use OIC

Ashraf Soliman a, , Mohamed Yassin a & Vincenzo De Sanctis c


aHamad Medical Center, Doha, Qatar; bUniversity of Alexandria, Alexandria, Egypt; cQuisisana Hospital, Ferrara, Italy


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, six females and nine 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.

Table 1 Growth and endocrine function in adults who received oral iron chelation (OIC) vs those who did not receive OIC before attaining final adult height.
Group A: OICGroup B: No OIC
Number of patients1540
DM6.6%2.5%
IFG6.6%17.5%*
Hypothyroid0.0%10.0%*
IGF-1 <−220.0%87.5%*
HtSDS < −26.6%52.5%*
Hypogonadism13.3%40.0%*
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.

Volume 89

57th Annual ESPE (ESPE 2018)

Athens, Greece
27 Sep 2018 - 29 Sep 2018

European Society for Paediatric Endocrinology 

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