Document Type

Article

Original Publication Date

1982

Journal/Book/Conference Title

The New England Journal of Medicine

Volume

306

Issue

23

First Page

1387

Last Page

1392

DOI of Original Publication

10.1056/NEJM198206103062303

Comments

Originally published at http://dx.doi.org/10.1056/NEJM198206103062303

Date of Submission

January 2015

Abstract

Abstract

Children with inborn errors of urea synthesis accumulate ammonium and other nitrogenous precursors of urea, leading to episodic coma and a high mortality rate. We used alternative pathways for the excretion of waste nitrogen as substitutes for the defective ureagenic pathways in 26 infants. These pathways involve synthesis and excretion of hippurate after sodium benzoate administration, and of citrulline and argininosuccinate after arginine supplementation.

The children were treated for seven to 62 months; 22 survived. The mean plasma level of ammonium (±S.E.) was 36±2 μmol per liter, and that of benzoate was 1.5±1.0 mg per deciliter. Alternative pathways accounted for between 28 and 59 per cent of the total "effective" excretion of waste nitrogen. Nineteen infants had normal height, weight, and head circumference, and 13 had normal intellectual development.

Activation of alternative pathways of waste nitrogen excretion can prolong survival and improve clinical outcome in children with inborn errors of urea synthesis. (N Engl J Med. 1982; 306:1387–92.)

Rights

From The New England Journal of Medicine, Batshaw, M.L, Brusilow, S., Waber, L., et al., Treatment of Inborn Errors of Urea Synthesis — Activation of Alternative Pathways of Waste Nitrogen Synthesis and Excretion, Vol. 306, Page 1387, Copyright © 1982 Massachusetts Medical Society. Reprinted with permission.

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