Growth hormone therapy in neonatal patients with methylmalonic acidemia.
BACKGROUND
Information regarding growth hormone (GH) therapy in neonatal patients with methylmalonic academia (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 1st 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.


CONCLUSIONS
Our clinical findings suggest that GH therapy has potentially beneficial effects on neonatal patients with MMA.
Kao
Chuan-Hong
CH
Department of Pediatrics, Far Eastern Memorial Hospital, Taipei, Taiwan, ROC.
Liu
Mei-Ying
MY
Liu
Tze-Tze
TT
Hsiao
Kwang-Jen
KJ
Cheng
Kang-Hsiang
KH
Huang
Cheng-Hung
CH
Lin
Hsiang-Yu
HY
Niu
Dau-Ming
DM
D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D008764:Methylmalonic Acid / Q000097:blood*
D019382:Human Growth Hormone; D008764:Methylmalonic Acid