Literature DB >> 29147990

Assessment of the Effect of Once Daily Nitisinone Therapy on 24-h Urinary Metadrenalines and 5-Hydroxyindole Acetic Acid Excretion in Patients with Alkaptonuria After 4 Weeks of Treatment.

A S Davison1,2, B Norman3, A M Milan4,3, A T Hughes4,3, M Khedr4,3, J Rovensky5, J A Gallagher3, L R Ranganath4,3.   

Abstract

BACKGROUND: One of the major metabolic consequences of using nitisinone to treat patients with alkaptonuria is that circulating tyrosine concentrations increase. As tyrosine is required for the biosynthesis of catecholamine neurotransmitters, it is possible that their metabolism is altered as a consequence. Herein we report the 24-h urinary excretion of normetadrenaline (NMA), metadrenaline (MA), 3-methoxytyramine (3-MT) (catecholamine metabolites) and 5-hydroxyindole acetic acid (5-HIAA, metabolite of serotonin) in a cohort of AKU patients before and after a 4-week treatment trial with nitisinone.
MATERIALS AND METHODS: 24 h urinary excretions of NMA, MA, 3-MT and 5-HIAA were determined by liquid chromatography tandem mass spectrometry. Interassay coefficient of variation was <10% for all analytes measured, at all concentrations tested.
RESULTS: Urine samples were assayed at baseline (pre-nitisinone, n = 36) and 4 weeks later; 7 received no nitisinone (4 male, mean age (±SD) 46.3 (16.4) years), and 29 received a daily dose of nitisinone [1 mg (n = 7, 6 male, mean age 45.9 (10.9) years), 2 mg (n = 8, 5 male, mean age 43.9 (13.7) years), 4 mg (n = 8, 5 male, mean age 47.3 (10.7) years) and 8 mg (n = 6, 4 male, mean age 53.8 (8.3) years)]. 3-MT concentrations increase significantly (p < 0.01, at all doses) following nitisinone therapy but not in a dose-dependent manner. NMA concentrations decreased (p < 0.05, at all doses) following nitisinone therapy at all doses. 5-HIAA concentrations decreased following nitisinone therapy and were significantly lower at a daily dose of 8 mg only (p < 0.05).
CONCLUSIONS: This study shows that catecholamine and serotonin metabolism is altered by treatment with nitisinone.

Entities:  

Keywords:  5-Hydroxyindole acetic acid; Alkaptonuria; Metadrenalines

Year:  2017        PMID: 29147990      PMCID: PMC6122050          DOI: 10.1007/8904_2017_72

Source DB:  PubMed          Journal:  JIMD Rep        ISSN: 2192-8304


  30 in total

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2.  Studies of Alcaptonuria: Inheritance of 47 Cases in Eight Highly Inter-related Dominican Kindreds.

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5.  An update on molecular genetics of Alkaptonuria (AKU).

Authors:  Andrea Zatkova
Journal:  J Inherit Metab Dis       Date:  2011-07-01       Impact factor: 4.982

6.  Disprocynium24, a novel inhibitor of the extraneuronal monoamine transporter, has potent effects on the inactivation of circulating noradrenaline and adrenaline in conscious rat.

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7.  Increase of CSF tyrosine and impaired serotonin turnover in tyrosinemia type I.

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Review 9.  Recent advances in management of alkaptonuria (invited review; best practice article).

Authors:  Lakshminarayan R Ranganath; Jonathan C Jarvis; James A Gallagher
Journal:  J Clin Pathol       Date:  2013-03-13       Impact factor: 3.411

10.  Natural history of alkaptonuria.

Authors:  Chanika Phornphutkul; Wendy J Introne; Monique B Perry; Isa Bernardini; Mark D Murphey; Diana L Fitzpatrick; Paul D Anderson; Marjan Huizing; Yair Anikster; Lynn H Gerber; William A Gahl
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1.  Impact of Nitisinone on the Cerebrospinal Fluid Metabolome of a Murine Model of Alkaptonuria.

Authors:  Andrew S Davison; Brendan P Norman; Hazel Sutherland; Anna M Milan; James A Gallagher; Jonathan C Jarvis; Lakshminarayan R Ranganath
Journal:  Metabolites       Date:  2022-05-25

2.  Metabolomic studies in the inborn error of metabolism alkaptonuria reveal new biotransformations in tyrosine metabolism.

Authors:  Brendan P Norman; Andrew S Davison; Juliette H Hughes; Hazel Sutherland; Peter Jm Wilson; Neil G Berry; Andrew T Hughes; Anna M Milan; Jonathan C Jarvis; Norman B Roberts; Lakshminarayan R Ranganath; George Bou-Gharios; James A Gallagher
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3.  Serum Amino Acid Profiling in Patients with Alkaptonuria Before and After Treatment with Nitisinone.

Authors:  A S Davison; B P Norman; E A Smith; J Devine; J Usher; A T Hughes; M Khedr; A M Milan; J A Gallagher; L R Ranganath
Journal:  JIMD Rep       Date:  2018-05-13

4.  Assessing the effect of nitisinone induced hypertyrosinaemia on monoamine neurotransmitters in brain tissue from a murine model of alkaptonuria using mass spectrometry imaging.

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5.  Evaluation of the serum metabolome of patients with alkaptonuria before and after two years of treatment with nitisinone using LC-QTOF-MS.

Authors:  Andrew S Davison; Brendan P Norman; Gordon A Ross; Andrew T Hughes; Milad Khedr; Anna M Milan; James A Gallagher; Lakshminarayan R Ranganath
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6.  Long-term low dose nitisinone therapy in adults with alkaptonuria shows no cognitive decline or increased severity of depression.

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7.  Dietary restriction of tyrosine and phenylalanine lowers tyrosinemia associated with nitisinone therapy of alkaptonuria.

Authors:  Juliette H Hughes; Peter J M Wilson; Hazel Sutherland; Shirley Judd; Andrew T Hughes; Anna M Milan; Jonathan C Jarvis; George Bou-Gharios; Lakshminarayan R Ranganath; James A Gallagher
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8.  Musculoskeletal manifestations in Alkaptonuria: A cross-sectional study.

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  8 in total

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