Evidence map›Paper›PMID 42192320›Full record

ArticleBMC pediatrics2026

Neonatal HIV prophylaxis is associated with accelerated presentation and clinical progression of MPV17-related mitochondrial neurohepatopathy.

Penelope C Rose, Helena Rabie, Ronalda de Lacy, Gillian Riordan, George van der Watt, Magriet van Niekerk, Ronald van Toorn, Tshepang Mokoto, Surita Meldau

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
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Article in BMC pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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4 · The record

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5 · Who and what money

Authors and funding

9 authors.

Penelope C RoseDepartment of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, PO Box 241, Cape Town, 8000, South Africa. p.rose@rn.dk.
Helena RabieDepartment of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, PO Box 241, Cape Town, 8000, South Africa.
Ronalda de LacyPaediatric Neurology and Gastroenterology, Department of Paediatrics and Child Health, Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa.
Gillian RiordanPaediatric Neurology and Gastroenterology, Department of Paediatrics and Child Health, Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa.
George van der WattDivision of Chemical Pathology, Department of Pathology, University of Cape Town, Cape Town, South Africa.
Magriet van NiekerkDepartment of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, PO Box 241, Cape Town, 8000, South Africa.
Ronald van ToornDepartment of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, PO Box 241, Cape Town, 8000, South Africa.
Tshepang MokotoDepartment of Paediatrics and Child Health, Tygerberg Hospital and Stellenbosch University, PO Box 241, Cape Town, 8000, South Africa.
Surita MeldauDivision of Chemical Pathology, Department of Pathology, University of Cape Town, Cape Town, South Africa.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundMPV17-related mitochondrial DNA depletion syndrome is a rare, lethal, autosomal recessive primary mitochondrial disorder characterised by infantile onset liver disease and neurological features, including hypotonia, developmental delay, failure to thrive and neuropathy.

methodsThe aim of this study was to describe the presentation and clinical course of infants diagnosed with MPV17 neurohepatopathy, comparing those who were HIV-exposed on antiretroviral therapy (ART), including zidovudine and nevirapine to prevent perinatal HIV transmission, to infants who were not HIV exposed, using data from a multicentre MPV17 natural history study in South Africa.

resultsBetween 2013 and 2024, 25 infants were diagnosed with MPV17 neurohepatopathy, 8 (32%) of whom were HIV-exposed and received ART at birth (7 received zidovudine), none were HIV-infected. Median birth weight was lower at 2.45 kg (IQR 2.28-2.71) in infants who were HIV-exposed compared to 2.86 kg (IQR 2.54-3.13) in HIV-unexposed infants (p = 0.02). Symptom onset occurred much earlier at a median of 3 days of age (IQR 0-10 days) in HIV-exposed infants compared to 60 days (IQR 14-90) in HIV-unexposed (p = 0.006). Infants exposed to HIV were more likely to develop liver failure (p = 0.02).

conclusionsPerinatal therapy with the nucleoside reverse transcriptase inhibitor zidovudine, a known mitochondrial toxin, was associated with accelerated clinical presentation and clinical course of MPV17 neurohepatopathy. Our findings suggest that less toxic antiretroviral therapy should be considered for perinatal HIV prophylaxis in HIV-exposed infants, particularly in our setting where there is a high carrier frequency of a single pathogenic MPV17 variant.

Indexed as

Anti-HIV AgentsHIV InfectionsInfectious Disease Transmission, VerticalMembrane ProteinsMitochondrial DiseasesMitochondrial ProteinsDisease ProgressionFemaleHumansInfantInfant, NewbornMaleNevirapineSouth AfricaZidovudineAnti-HIV AgentsMembrane ProteinsMitochondrial ProteinsMPV17 protein, humanNevirapineZidovudineAntiretroviralHIVMitochondrial diseaseMPV17NeurohepatopathyToxicityZidovudine

Identifiers

PMID42192320
PMCPMC13560266

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