European Journal of Pediatrics

, Volume 165, Issue 9, pp 618–624

Effects of arginine treatment on nutrition, growth and urea cycle function in seven Japanese boys with late-onset ornithine transcarbamylase deficiency

  • Hironori Nagasaka
  • Tohru Yorifuji
  • Kei Murayama
  • Mitsuru Kubota
  • Keiji Kurokawa
  • Tomoko Murakami
  • Masaki Kanazawa
  • Tomozumi Takatani
  • Atsushi Ogawa
  • Emi Ogawa
  • Shigenori Yamamoto
  • Masanori Adachi
  • Kunihiko Kobayashi
  • Masaki Takayanagi
Original Paper

DOI: 10.1007/s00431-006-0143-y

Cite this article as:
Nagasaka, H., Yorifuji, T., Murayama, K. et al. Eur J Pediatr (2006) 165: 618. doi:10.1007/s00431-006-0143-y

Abstract

Background

The aim of this study was to investigate the effects of arginine on nutrition, growth and urea cycle function in boys with late-onset ornithine transcarbamylase deficiency (OTCD). Seven Japanese boys with late-onset OTCD enrolled in this study resumed arginine treatment after the cessation of this therapy for a few years. Clinical presentations such as vomiting and unconsciousness, plasma amino acids and urinary orotate excretion were followed chronologically to evaluate urea cycle function and protein synthesis with and without this therapy. In addition to height and body weight, blood levels of proteins, lipids, growth hormone (GH), insulin-like growth factor-I (IGF-I) and IGF-binding protein -3 (IGFBP-3) were monitored.

Results

The frequency of hyperammonemic attacks and urinary orotate excretion decreased significantly following the resumption of arginine treatment. Despite showing no marked change in body weight, height increased gradually. Extremely low plasma arginine increased to normal levels, while plasma glutamine and alanine levels decreased considerably. Except for a slight increase in high-density lipoprotein cholesterol level, blood levels of markers for nutrition did not change. In contrast, low serum IGF-I and IGFBP-3 levels increased to age-matched control levels, and normal urinary GH secretion became greater than the level observed in the controls.

Conclusion

Arginine treatment is able to reduces attacks of hyperammonemia in boys with late-onset OTCD and to increase their growth.

Keywords

Arginine deficiencyGrowth hormoneNutrition and growthPlasma glutamine and alanineOrnithine transcarbamylase deficiencyOrotate

Abbreviations

FFA

Free fatty acids

GH

Growth hormone

IGF-I

Insulin-like growth factor-I

IGFBP-3

Insulin-like growth factor binding protein-3

OTCD

Ornithine transcarbamylase deficiency

TKB

Total ketone body

TP

Total protein

Copyright information

© Springer-Verlag 2006

Authors and Affiliations

  • Hironori Nagasaka
    • 1
  • Tohru Yorifuji
    • 2
  • Kei Murayama
    • 1
  • Mitsuru Kubota
    • 3
  • Keiji Kurokawa
    • 2
  • Tomoko Murakami
    • 1
  • Masaki Kanazawa
    • 4
  • Tomozumi Takatani
    • 1
  • Atsushi Ogawa
    • 4
  • Emi Ogawa
    • 4
  • Shigenori Yamamoto
    • 4
  • Masanori Adachi
    • 5
  • Kunihiko Kobayashi
    • 3
  • Masaki Takayanagi
    • 1
  1. 1.Division of MetabolismChiba Children’s HospitalMidori-ku, ChibaJapan
  2. 2.Department of PediatricsKyoto University Graduate School of MedicineKyoto-shi, KyotoJapan
  3. 3.Department of PediatricsHokkaido University Graduate School of Medicine Sapporo, HokkaidoJapan
  4. 4.Department of PediatricsChiba University Graduate School of MedicineMidori-ku, ChibaJapan
  5. 5.Division of Endocrinology and MetabolismKanagawa Children’s Medical CenterYokohamaJapan