Background Cardiovascular Magnetic Resonance (CMR) by late gadolinium enhancement (LGE) allows to detect myocardial fibrosis. Myocardial fibrosis was shown to be a relative common finding in large cohort of Italian thalassemia major (TM) patients mainly related to HCV infection, but specific studies involving only pediatric patients are not available. Our aim was to investigate the prevalence and clinical-instrumental correlates of myocardial fibrosis in pediatric TM patients.
CONTEXT GnRH analogs (GnRHa) are considered the treatment of choice for central precocious puberty (CPP). During GnRHa administration, the suppression of the pituitary-gonadal axis results in decreased rates of linear growth and skeletal maturation and in improved adult height. However, in some patients, the growth deceleration is so marked that the expected improvement in predicted adult height is not achieved. OBJECTIVE The objective of this study was to assess whether the addition of oxandrolone (Ox) may affect the height outcome of patients with CPP and growth deceleration during GnRHa treatment. DESIGN This was an open-label, clinical study. SETTING The study was performed at a pediatric endocrinology referral clinic. PATIENTS Twenty patients with CPP and marked growth deceleration during GnRHa treatment were studied. INTERVENTIONS Treatment consisted of GnRHa (Leuprorelina, 3.75 mg im every 28 d) alone (10 patients) or in combination with Ox (0.06 mg/kg.d by mouth) (10 patients). MAIN OUTCOME MEASURE The main outcome measure was the patients' adult height. RESULTS The adult height of the patients treated with GnRHa plus Ox was significantly higher than pretreatment predicted adult height (162.6 +/- 2.3 vs. 154.8 +/- 1.7 cm, mean +/- sem; P < 0.05) and target height (162.6 +/- 2.3 vs. 158.0 +/- 1.9; P > 0.05). Patients treated with GnRHa alone reached an adult height similar to the pretreatment predicted adult height (151.9 +/- 1.2 vs. 155.4 +/- 2.1 cm) but significantly lower than target height (151.9 +/- 1.2 vs. 156.6 +/- 1.4 cm; P < 0.005). No side effects were recorded in either group of patients. CONCLUSIONS Combined GnRHa and Ox therapy is a viable treatment option for children with CPP and marked growth deceleration during treatment with GnRHa alone.