Growth Hormone Therapy in Neonatal Patients With Methylmalonic Acidemia

BackgroundInformation regarding growth hormone (GH) therapy in neonatal patients with methylmalonic acidemia (MMA) is lacking. We present our experience with GH therapy in neonatal patients with MMA.MethodsFour neonatal patients with mut0 type MMA were identified through newborn screening for elevated propionylcarnitine (C3) levels. GH therapy (0.6 IU/kg/week, subcutaneously) was prescribed for patient 1 after 1 month of admission, and was prescribed for patients 2, 3 and 4 on the 1st day of admission. We evaluated weight, skin erosion, hospital stay, and serum levels of C3 after GH therapy.ResultsAll of the neonatal patients with MMA displayed obvious weight gain and distinct improvement in skin erosions after GH therapy. The duration of hospital stay for patients 2, 3 and 4 was reduced compared to that of patient 1. However, the metabolic effects of GH therapy on reducing serum levels of C3 seem to be indeterminate.ConclusionOur clinical findings suggest that GH therapy has potentially beneficial effects on neonatal patients with MMA.