Evidence map›Paper›PMID 29427049›Full record

ArticleMetabolic brain disease2018

Favourable outcome in a child with symptomatic diagnosis of Glutaric aciduria type 1 despite vertical HIV infection and minor head trauma.

Angeline Thomas, Els F M Dobbels, Priscilla E Springer, Christelle Ackermann, Mark F Cotton, Barbara Laughton

Abstract readCase Reports
PubMed Publisher
In one paragraph

Article in Metabolic brain disease, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact, top 99% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed, 1 citations in OpenAlex.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 2 institutions in 1 country.

Angeline ThomasDevelopmental Paediatrics, Department of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa.
Els F M DobbelsFamily Clinical Research Unit, Department of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa.
Priscilla E SpringerDevelopmental Paediatrics, Department of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa.
Christelle AckermannDepartment of Radiology, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa.
Mark F CottonFamily Clinical Research Unit, Department of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa.
Barbara LaughtonFamily Clinical Research Unit, Department of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, Francie van Zijl Drive, Tygerberg, Cape Town, South Africa. BL2@sun.ac.za.
Tygerberg Hospital · ZAStellenbosch University · ZA

Funding

Longitudinal Neuroimaging and Cognitive Study of HIV-Infected ChildrenR01HD071664 · NICHD · MASSACHUSETTS GENERAL HOSPITAL · PI LAUGHTON, BARBARA, MEINTJES, ERNESTA MARIA · 2011 to 2015
$2.4M
Eunice Kennedy Shriver National Institute of Child Health and Human Development R01 HD071664NICHD NIH HHS R01 HD071664NIH HHS HHSN272200800014cNIH HHS U19 A153217
6 · The paper itself

Abstract

The first case of Glutaric aciduria Type 1(GA1) in an African child was reported in 2001. GA1 has a prevalence of 1:5000 in black South Africans. Although early diagnosis is essential for a favourable outcome, newborn screening is not routine in South Africa where an estimated 320,000 children have HIV infection. Neurodevelopmental delay and encephalopathy are complications of both HIV and GA1. In such a setting it is important to recognise that HIV and GA1 can occur simultaneously. We present an HIV-infected South African male child of Xhosa descent with macrocephaly who commenced combination antiretroviral therapy (ART) at 8 weeks of age in a clinical trial which included a neurodevelopmental sub-study. He developed short-lived focal seizures at 16 months after minor head trauma. Neurological examination was normal. Neuroimaging showed temporal lobe atrophy, subtle hyperintense signal change in the globus pallidus, and focal haemosiderosis in the right Sylvian fissure region. As findings were not in keeping with HIV encephalopathy, a urine metabolic screen was undertaken which suggested GA1. Genetic testing confirmed Arg293Trp mutation. He began L-carnitine and a low protein diet as a restricted diet was not practicable. At 21 months he developed pulmonary tuberculosis, requiring 6 months treatment. He did not develop any neurologic motor symptoms. Serial neurodevelopmental and neuropsychological test scores until 9 years were similar to healthy neighbourhood controls, except for mild language delay at 3½ years. Detection of GA1, probably facilitated through participation in a clinical trial, was pivotal for a favourable outcome. The concomitant use of ART and anti-tuberculous therapy in a child with GA1 appears safe.

Indexed as

Amino Acid Metabolism, Inborn ErrorsAtrophyBrainBrain DiseasesBrain Diseases, MetabolicCarnitineCraniocerebral TraumaGlutaryl-CoA DehydrogenaseHIV InfectionsHumansInfantMaleTreatment OutcomeCarnitineGlutaryl-CoA DehydrogenaseAfricaAntiretroviral therapyChildrenGlutaric aciduriaHIVMacrocephaly

Identifiers

PMID29427049
OpenAlexW2790296563

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.