Growth Hormone Therapy in Neonatal Patients With Methylmalonic Acidemia

Background: Information 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. Methods: Four neonatal patients with mut 0 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 1 st day of admission. We evaluated weight, skin erosion, hospital stay, and serum levels of C3 after GH therapy. Results: All 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. Conclusion: Our clinical findings suggest that GH therapy has potentially beneficial effects on neonatal patients with MMA. [J Chin Med Assoc 2009;72(9):462–467]