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This extremely rare condition results in early-onset insulin-dependent diabetes mellitus (IDDM) and exocrine pancreatic insufficiency.

Congenital Pancreatic Hypoplasia.

Approximately 10 cases of pancreatic agenesis have been reported.

Autosomal recessive transmission. The genetic defect results from a mutation of the human insulin promoter factor-1 (IPF1) gene, which is located on 13q12.1.

The homeodomain protein IPF1 is critical for the development of the pancreas and is a key factor in the regulation of the insulin gene in the beta cells of the endocrine pancreas. Targeted disruption of the gene encoding IPF1 results in failure of the pancreas to develop (pancreatic agenesis). Intrauterine growth retardation appears to be related to the fact that insulin is a major intrauterine growth factor.

Exclude Mucoviscidosis, which is the most common cause of exocrine pancreatic insufficiency in childhood. The findings of intrauterine growth retardation (insulin is a known prenatal growth factor that does not cross the placenta in clinically significant amounts) and early-onset IDDM combined with clinical and biochemical evidence of pancreatic exocrine insufficiency may fit the diagnosis of congenital pancreatic aplasia/hypoplasia. In contrast to cases with absence of islets of Langerhans or complete pancreatic aplasia, serum levels of C peptide and glucagon may be measurable in partial aplasia. The diagnosis of pancreatic agenesis is difficult to establish in the newborn period. The size of the infant makes use of endoscopic retrograde cholangiopancreatography hazardous, and normal newborn comparisons are not well established for radiologic examinations. Clues pointing to the diagnosis include intrauterine growth retardation, failure to thrive, persistent hyperglycemia, polyuria, glycosuria, and steatorrhea.

Intrauterine growth retardation with early-onset IDDM is typical. Affected infants may develop profound polydipsia and polyuria because of increased osmotic load resulting in rapidly progressing dehydration. Hyperglycemia in newborns can be associated with an increased incidence of intraventricular hemorrhage. Overly aggressive insulin therapy can result in hypoglycemia with adverse neurologic sequelae. Ketoacidosis is a rare finding in these patients, and some researchers hypothesized this finding results from the lack of hyperglucagonemia. Despite appropriate insulin and exocrine pancreas hormone replacement therapy, some patients fail to gain weight. Serum insulin levels may be very low or undetectable. Limited joint mobility has been reported in some patients.

Admit the patient 24 to 48 hours preoperatively to optimize insulin therapy and try to keep the blood glucose levels slightly higher (i.e., 5-12 mmol/liter) than generally accepted for older children. These patients should be booked at the beginning of the operating list. Different treatment options are available for the management of IDDM. We recommend the treatment you commonly use and with which you are familiar. One regimen consists of omitting the morning dose of insulin, starting an intravenous glucose-insulin infusion (either separately or mixed), and adding potassium as required. Preoperative laboratory investigations should include a 24-hour serum glucose profile, glycosylated hemoglobin or fructosamine, fasting blood glucose level, complete blood count, and serum concentrations ...

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