Treatment of Growth Hormone Deficiency via Daily Intravascular Injections in a Child with Bleeding Disorder.

OBJECTIVES: The standard of treatment for pediatric growth hormone deficiency (GHD) is daily subcutaneous recombinant human growth hormone (rhGH) injections. The efficacy of rhGH treatment given as daily intravenous (IV) boluses is not known. Case Presentation. A female with protein C deficiency, a severe bleeding disorder characterized by thrombosis formation, was diagnosed with GHD at age four years. She has been receiving daily protein C infusion through a permanent port since the newborn period. GHD was treated with daily IV rhGH boluses given through the port following protein C infusion. She has reached a growth rate of 12 cm/year and had no side effects. Surprisingly, serum insulin-like growth factor-1 (IGF1) levels did not rise despite an excellent clinical response. CONCLUSIONS: IV administration may be an alternative route for GHD treatment in eligible patients with permanent vascular access. A rise in serum IGF1 levels may not be needed to achieve the growth-promoting effect of rhGH.

Risk factors for hospitalization in youth with type 1 diabetes: Development and validation of a multivariable prediction model.

OBJECTIVE: To develop a multivariable prediction model to identify patients with type 1 diabetes at increased risk of hospitalization for diabetic ketoacidosis or hyperglycemia with ketosis in the 12 months following assessment. METHODS: Retrospective review of clinical data from patients with type 1 diabetes less than 17 years old at a large academic children's hospital (5732 patient years, 652 admissions). Data from the previous 12 months were assessed on October 15, 2015, 2016, 2017, and 2018, and used to predict hospitalization in the following 12 months using generalized estimating equations. Variables that were significant predictors of hospitalization in univariate analyses were entered into a multivariable model. 2014 to 2016 data were used as a training dataset, and 2017 to 2019 data for validation. Discrimination of the model was assessed with receiver operator characteristic curves. RESULTS: Admission in the preceding year, hemoglobin (Hb)A1c, non-commercial insurance, female sex, and non-White race were all individual predictors of hospitalization, but age, duration of diabetes and number of office visits in the preceding year were not. In multivariable analysis with threshold P < .0033, admissions in the previous 12 months, HbA1c, and non-commercial insurance remained as significant predictors. The model identified a subset of ~8% of the patients with a collective 42% risk of hospitalization, thus increased 5-fold compared with the 8% risk of hospitalization in the remaining 93% of patients. Similar results were obtained with the validation dataset. CONCLUSION: Our multivariable prediction model identified patients at increased risk of admission in the 12 months following assessment.

Diazoxide for the Treatment of Hypoglycemia Resulting From Dumping Syndrome in a Child.

Dumping syndrome-associated hypoglycemia is caused by an exaggerated hyperinsulinemic response to glucose absorption in the small intestine. Diazoxide acts on the ATP-sensitive potassium channels and prevents insulin secretion and, thus, should be beneficial for the treatment of hypoglycemia secondary to dumping syndrome. We report on the efficacy of diazoxide in a pediatric patient with dumping syndrome. A 6-year-old girl born at 32 weeks' gestation age with resultant short gut syndrome and liver failure, who had undergone liver, small bowel, and pancreas transplantation at 1 year of age, developed late dumping-like symptoms with postprandial hypoglycemia, headaches, tremors, and irritability. She experienced relief of symptoms with oral intake. An oral glucose tolerance test showed a fasting and 2-hour blood glucose of 3.9 and 2.8 mmol/L, respectively. A gastric emptying study confirmed the diagnosis of dumping. A diet with 2 g of fiber and cornstarch and antimotility medications failed to improve the dumping symptoms. Diazoxide was started orally at a dose of 3 mg/kg/d and was increased to 5 mg/kg/d, divided every 8 hours, after 1 month, with improvement of postprandial blood glucose values (3.6 to 5.0 mmol/L). No hypertrichosis, fluid retention, respiratory concerns, or other side effects were noted. Several duodenal dilations were performed, with resultant improvement of gastric emptying. She was eventually weaned from diazoxide, and no further episodes of substantial hypoglycemia occurred. In conclusion, diazoxide was efficacious and safe for the treatment of hypoglycemia secondary to dumping syndrome in children. It could be of particular use as a bridging therapy for children awaiting more definitive surgical interventions.