Growth hormone – insulin-like growth factor-I axis and bone mineral density in adults with thalassemia major

"Growth hormone – insulin-like growth factor-I axis and bone mineral density in adults with thalassemia major" 

1 Department of Pediatrics, Hamad Medical Center, Doha, Qatar
2 Pediatric and Adolescent Outpatient Clinic, Quisisana Hospital, Ferrara, Italy
3 Department of Hematology, Hamad Medical Center, Doha, Qatar
4 Department of Clinical Chemistry, Hamad Medical Center, Doha, Qatar

è la prima pubblicazione internazionale del 2014 che vede protagonista Quisisana con un gruppo di Ricercatori del Qatar. Gli Autori si occupano da molti anni di complicanze endocrine nella talassemia. Sono partiti dai problemi della crescita, pubertà e successivamente  della attività riproduttiva. Oggi possono essere considerati all'avanguardia anche nel campo delle endocrinopatie dei soggetti giovani adulti con talassemia. La collaborazione internazionale di Quisisana continuerà anche per il 2014. 


Introduction: Bone disease and short stature are frequent clinical features of patients with beta-thalassaemia major. Dysfunction of the GH-IGF-1 axis has been described in many thalassemics children and adolescents with short stature and reduced growth velocity. Assessment of the GH-IGF-1 axis in short adults with TM after attainment of final height may be required to select those who are candidates for replacement therapy and to prevent the development of bone disease. The aim of our study was to investigate GH secretion in adult thalassemic patients in relation to their bone mineral density (BMD) and serum ferritin concentrations.

Materials and Methods: We performed clonidine stimulation test in 30 thalassemic patients (18 males, 12 females) with a mean age of 31.5± 7.2 years. The cut-off level for GH response was set at 7ug/l, according to the literature. Serum ferritin, IGF-I, liver enzymes, alkaline phosphatase (ALP) and type 1 Collagen Carboxy Telopeptide (CCT1) were also determined.

Results: We diagnosed GH deficiency (GHD) in 12 patients (40%) and IGF-I deficiency (IGF-I SDS).

Conclusions: We conclude that GH status should be tested in adult thalassemic patients especially those with short stature and/or decreased BMD. Clonidine test appears to be effective and safe in adults with TM. If the diagnosis of adult GHD is established, GH treatment may be considered for possible improvement of bone mineral density and heart function in patients with TM.

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