Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy

Margaret M. McGovern, Melissa P. Wasserstein, Alan Aron, Susan P. Perrine

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Objectives: To determine the bioehemieal and elinieal effeets of intravenous arginine butyrate in X-linked Adrenoleukodystrophy (X-ALD). Study design: Arginine butyrate was intravenously infused over a 4-month period in a patient with the rapid cerebral form of X-ALD. Very long chain fatty acids (VLCFA), complete blood counts, and serum chemistries were monitored, and serial MRI of the brain and clinical neurologic examinations were performed. Results: All blood chemical and hematologic values remained within the normal range for age throughout the therapy. After completion of the first day of infusion, the C 26:0 value fell from 1.01 μg/mL to 0.445 μg/mL, which is below the mean value for an X-ALD heterozygote. Throughout the remainder of the trial, all C26:0 levels fell below the mean -1 SD for X-ALD hemizygotes (mean, 1.18 μg/mL, 1 SD = 0.53), ranging from 0.321 to 0.565 μg/mL. Despite reduction of the plasma VLCFA, the patient continued to deteriorate neurologically. Conclusions: Intravenous arginine butyrate resulted in a rapid decrease in plasma VLCFA but no effect on the neurologic progression of the disease in this patient. Additional studies are needed to determine minimum effective dosage and interval, what proportion of patients respond, and whether the agent can prevent neurologic degeneration.

Original languageEnglish (US)
Pages (from-to)709-713
Number of pages5
JournalJournal of Pediatrics
Volume142
Issue number6
DOIs
StatePublished - Jun 1 2003
Externally publishedYes

Fingerprint

Adrenoleukodystrophy
Fatty Acids
Hemizygote
Blood Cell Count
Neurologic Examination
Heterozygote
Nervous System Diseases
Nervous System
Reference Values
arginine butyrate
Brain
Serum

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy. / McGovern, Margaret M.; Wasserstein, Melissa P.; Aron, Alan; Perrine, Susan P.

In: Journal of Pediatrics, Vol. 142, No. 6, 01.06.2003, p. 709-713.

Research output: Contribution to journalArticle

McGovern, Margaret M. ; Wasserstein, Melissa P. ; Aron, Alan ; Perrine, Susan P. / Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy. In: Journal of Pediatrics. 2003 ; Vol. 142, No. 6. pp. 709-713.
@article{d0d2b3e7514941bf808f2410ed101c1e,
title = "Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy",
abstract = "Objectives: To determine the bioehemieal and elinieal effeets of intravenous arginine butyrate in X-linked Adrenoleukodystrophy (X-ALD). Study design: Arginine butyrate was intravenously infused over a 4-month period in a patient with the rapid cerebral form of X-ALD. Very long chain fatty acids (VLCFA), complete blood counts, and serum chemistries were monitored, and serial MRI of the brain and clinical neurologic examinations were performed. Results: All blood chemical and hematologic values remained within the normal range for age throughout the therapy. After completion of the first day of infusion, the C 26:0 value fell from 1.01 μg/mL to 0.445 μg/mL, which is below the mean value for an X-ALD heterozygote. Throughout the remainder of the trial, all C26:0 levels fell below the mean -1 SD for X-ALD hemizygotes (mean, 1.18 μg/mL, 1 SD = 0.53), ranging from 0.321 to 0.565 μg/mL. Despite reduction of the plasma VLCFA, the patient continued to deteriorate neurologically. Conclusions: Intravenous arginine butyrate resulted in a rapid decrease in plasma VLCFA but no effect on the neurologic progression of the disease in this patient. Additional studies are needed to determine minimum effective dosage and interval, what proportion of patients respond, and whether the agent can prevent neurologic degeneration.",
author = "McGovern, {Margaret M.} and Wasserstein, {Melissa P.} and Alan Aron and Perrine, {Susan P.}",
year = "2003",
month = "6",
day = "1",
doi = "10.1067/mpd.2003.201",
language = "English (US)",
volume = "142",
pages = "709--713",
journal = "Journal of Pediatrics",
issn = "0022-3476",
publisher = "Mosby Inc.",
number = "6",

}

TY - JOUR

T1 - Biochemical effect of intravenous arginine butyrate in X-linked adrenoleukodystrophy

AU - McGovern, Margaret M.

AU - Wasserstein, Melissa P.

AU - Aron, Alan

AU - Perrine, Susan P.

PY - 2003/6/1

Y1 - 2003/6/1

N2 - Objectives: To determine the bioehemieal and elinieal effeets of intravenous arginine butyrate in X-linked Adrenoleukodystrophy (X-ALD). Study design: Arginine butyrate was intravenously infused over a 4-month period in a patient with the rapid cerebral form of X-ALD. Very long chain fatty acids (VLCFA), complete blood counts, and serum chemistries were monitored, and serial MRI of the brain and clinical neurologic examinations were performed. Results: All blood chemical and hematologic values remained within the normal range for age throughout the therapy. After completion of the first day of infusion, the C 26:0 value fell from 1.01 μg/mL to 0.445 μg/mL, which is below the mean value for an X-ALD heterozygote. Throughout the remainder of the trial, all C26:0 levels fell below the mean -1 SD for X-ALD hemizygotes (mean, 1.18 μg/mL, 1 SD = 0.53), ranging from 0.321 to 0.565 μg/mL. Despite reduction of the plasma VLCFA, the patient continued to deteriorate neurologically. Conclusions: Intravenous arginine butyrate resulted in a rapid decrease in plasma VLCFA but no effect on the neurologic progression of the disease in this patient. Additional studies are needed to determine minimum effective dosage and interval, what proportion of patients respond, and whether the agent can prevent neurologic degeneration.

AB - Objectives: To determine the bioehemieal and elinieal effeets of intravenous arginine butyrate in X-linked Adrenoleukodystrophy (X-ALD). Study design: Arginine butyrate was intravenously infused over a 4-month period in a patient with the rapid cerebral form of X-ALD. Very long chain fatty acids (VLCFA), complete blood counts, and serum chemistries were monitored, and serial MRI of the brain and clinical neurologic examinations were performed. Results: All blood chemical and hematologic values remained within the normal range for age throughout the therapy. After completion of the first day of infusion, the C 26:0 value fell from 1.01 μg/mL to 0.445 μg/mL, which is below the mean value for an X-ALD heterozygote. Throughout the remainder of the trial, all C26:0 levels fell below the mean -1 SD for X-ALD hemizygotes (mean, 1.18 μg/mL, 1 SD = 0.53), ranging from 0.321 to 0.565 μg/mL. Despite reduction of the plasma VLCFA, the patient continued to deteriorate neurologically. Conclusions: Intravenous arginine butyrate resulted in a rapid decrease in plasma VLCFA but no effect on the neurologic progression of the disease in this patient. Additional studies are needed to determine minimum effective dosage and interval, what proportion of patients respond, and whether the agent can prevent neurologic degeneration.

UR - http://www.scopus.com/inward/record.url?scp=0038809137&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0038809137&partnerID=8YFLogxK

U2 - 10.1067/mpd.2003.201

DO - 10.1067/mpd.2003.201

M3 - Article

C2 - 12838202

AN - SCOPUS:0038809137

VL - 142

SP - 709

EP - 713

JO - Journal of Pediatrics

JF - Journal of Pediatrics

SN - 0022-3476

IS - 6

ER -