Changes with growth hormone treatment in growth hormone deficient children

A total of 54 previously untreated patients (15 girls, 39 boys) with poor growth due to idiopathic growth hormone deficiency (IGHD) were treated with human growth hormone (hGH), continuously up to 4 years. All of the patients had a peak hGH level which was below 10 ng/mL after at least two pharmacological tests and/or blunted physiologic hGH secretion, and their height was below ?2.5 s.d. for age and gender. After the 1st year of therapy, height velocity (HV) increased significantly when compared with baseline (from 3.18 ±0.76 cm/year to 9.17±1.03 cm/year; P <0.001), declined during the 2nd year and then remained significantly higher than pretreatment HV. When considering improvement in height expressed by height standard deviation score (SDS), during the therapy all of the patients showed a significant gain ± 1.72±1.09 (from ?4.11±0.61 to ?2.21±0.48). The height values were significantly higher than pretreatment, but remained below ?2 s.d. after 4 years of hGH therapy in our patients. Increased height velocity has been sustained, but height improvement after therapy was inversely correlated to height SDS for chronological age of patients at the start of therapy. In conclusion post-treatment height has been shown to be related to height deficit at the beginning of therapy. Therapy was well tolerated with no local or systemic adverse effects or acceleration of bone age.     

Association of empty sella and neuroendocrine disorders in childhood

Empty sella syndrome (ESS) is a multicausal entity. The incidence of primary empty sella syndrome (PESS) in children with neuroendocrine dysfunction is not known. In the pediatric age group, frequency seems to have been underestimated. A total of 117 cases of neuroendocrine disorders, including complete growth hormone deficiency, primary hypothyroidism with pituitary resistance to thyroid hormone, obesity, central precocious puberty, hypothalamic hypogonadism and central diabetes insipidus, have been studied with computed tomography and/or magnetic resonance imaging of sellar region for etiologic evaluation. Twenty-one patients were found to have PESS. We noted a high incidence of PESS in children with neuroendocrine dysfunction (17.9%). Children with neuroendocrine dysfunction should be investigated with respect to PESS, and children with PESS recognized coincidentally should be studied with the particular consideration of subclinical neuroendocrine dysfunction.     

Recombinant human growth hormone treatment in children with thalassemia major

BACKGROUND: To evaluate the growth hormone reserve and the growth hormone response to recombinant human growth hormone (GH) in prepubertal thalassemic children with growth retardation. METHODS: Twenty thalassemic patients with short stature and delayed bone age were studied. Patients were randomized into GH-treated (n = 10) and non-GH treated (control; n = 10) groups. The GH-treated group received recombinant human (rh)-GH (Genotropin) at the dose of 0.7 IU/kg per week for 12 months. RESULTS: There was a significant discordance between GH response to pharmacologic stimuli and physiological secretion of GH/GHRH testing. Following the administration of rhGH, growth velocity increased from 2.47 +/- 0.48 cm/year to 6.27 +/- 0.76 cm/year (P = 0.005), whereas there was not a similar change in the non-GH-treated group. The height velocities of the two groups during the 1 year follow-up period were significantly different (6.27 +/- 0.76 vs 3.99 +/- 0.34 cm/year; P = 0.025). There were significant differences between the height velocity improvements and height velocity standard deviation scores of the two groups as well. CONCLUSION: The present study has demonstrated that rhGH is a safe and efficacious mode of treatment in thalassemic children.     

When is open ureterolithotomy indicated for the treatment of ureteral stones?

AIM: Improvements in extracorporeal shock wave lithotripsy (ESWL) and ureteroscopy have almost eradicated the need for open surgery in ureteral stones. The aim of this study was to assess characteristics of patients who underwent open ureterolithotomy. METHODS: During a 5-year period, a total of 654 patients with ureteral stones were treated. Initial management consisted of ureteroscopy in 524 patients, ESWL in 62 patients and percutaneous nephrolithotomy (PCNL) in 12 patients. Open surgery was performed in 56 patients. Stone location, size and success rates were retrospectively analyzed. RESULTS: Ureteroscopy resulted in successful stone removal in 94%, 98% and 98.5% of proximal, mid and distal ureteral stones, respectively. A total of 14 patients with ureteroscopy failure were referred for open surgery. ESWL treatment resulted in success in 55 patients (88%), and those with ESWL failure were referred for either ureteroscopy (n = 3) or open surgery (n = 4). Open surgery was performed in a total of 56 patients, 38 of whom had been referred from other centers. Stone location was proximal ureter in 25 (44.6%) patients (stone size: 2-12 cm(2)), mid ureter in five (8.9%) patients (stone size: 2-6 cm(2)) and distal ureter in 26 (46.4%) patients (stone size: 4-9 cm(2)). A history of previous unsuccessful endourological procedure was observed in 33 (58%) of 56 patients. Children under age 16 (range 1-15 years) comprised 17.8% of patients undergoing open surgery. CONCLUSION: Open surgery, which is nowadays being replaced with laparoscopic techniques, is generally indicated for failed endourological procedures (58%), particularly in centers that do not have flexible ureteroscopy or laser lithotriptor, and in patients with larger stones (>3 cm). Children (17.8%) are also candidates for open surgery, if specifically designed endourological equipment is not available.     

Evaluation of the pancreas reserve in siblings of type I diabetic children

INTRODUCTION: The purpose of this study was to determine the pancreas reserve in siblings of diabetic patients by screening islet cell antibodies (ICA), insulin auto antibodies (IAA), reduced C-peptide levels, first-phase insulin release and the derangement of cellular immunity (reduction of natural killer cells, abnormality of the T cell subpopulations). METHODS AND RESULTS: Twelve siblings (aged 9.3 +/- 2.8 years) of diabetic children were evaluated and results were compared with the control group (12.1 +/- 3.5 years). For siblings of the diabetic children, fasting, post-prandial and glucagon response C-peptide mean values were 2.2 +/- 1.2, 7.2 +/- 7.1 and 5.3 +/- 3.6 ng/mL, respectively, while in the control group they were 1.5 +/- 0.8, 3.6 +/- 2.0 and 5.1 +/- 2.9 ng/mL, respectively. There were no differences between the two groups. In 33%, postprandial C-peptide, and in 11% of the siblings, glucagon response C-peptide values were exaggerated. In siblings the first phase insulin release (FPIR) during an intravenous glucose tolerance test was 128.5 +/- 96.6 (above the 50th percentile) and stimulated insulin release (SIR) was 103.8 +/- 92.5 (above 25th percentile). Sibling values were significantly lower than the control group (FPIR 152.4 +/- 42.5, P = 0.01; SIR 134.9 +/- 38.2, P = 0.01). Values for FPIR (in two children) and SIR (three cases) were below the 5th percentile. In one, FPIR and SIR levels were both below the 1st percentile. Islet cell antibodies and IAA were also present in this subject. Treatment with nicotinamide was started in the cases with FPIR and SIR below the 5th percentile. We did not observe overt diabetic symptoms during the follow-up period of more than 3 years. CONCLUSION: We recommend that borderline insulin secretion be tested annually in siblings who show insufficient FPIR.