Improved growth with growth hormone therapy in a child with glycogen storage disease Ib

In type Ib glycogen storage disease (GSD) growth is typically affected and short stature is a common feature. Reported use and effect of growth hormone (GH) therapy in children with GSD is limited. We report on the use of substitutive GH treatment in a poorly growing adolescent female with GSD type Ib. The patient's growth velocity increased from a baseline level of 2.5cm/y to an average growth velocity during GH therapy of 8.7 cm/y. During GH therapy this patient did not demonstrate metabolic decompensation but increased levels of cholesterol and triglycerides were seen (A‐1). Conclusion : Patients with GSD may experience an improvement in growth response with GH treatment. Prior to GH therapy, treatment of hyperlipidemia associated with GSD should allow the therapy to be safely tolerated.