Evidence map›Paper›PMID 41815511›Full record

ArticleAnnals of the Child Neurology Society2025

Fetal and Perinatal Brain MRI Findings in Adaptor Protein Complex 4-Associated Hereditary Spastic Paraplegia.

Alexandra K Brooks, Vicente Quiroz, Luca Schierbaum, Julian E Alecu, Katerina Bernardi, Nicole Battaglia, Amy Tam, Joshua Rong, Gregor Kasprian, Edward Yang and 1 more

Registry-linked trialAbstract read
In one paragraph

Article in Annals of the Child Neurology Society, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04712812 (Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia), which is not on this 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
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.

NCT04712812 recruitingnot on this map

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

Typeobservational_patient_registrySponsorBoston Children's HospitalRan2020 to 2030Enrolled700ConditionsHereditary Spastic Paraplegia, SPG47, SPG50, SPG51
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

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

11 authors.

Alexandra K BrooksMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0002-5121-7686
Vicente QuirozMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Luca SchierbaumMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Julian E AlecuMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Katerina BernardiMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Nicole BattagliaMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Amy TamMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0003-2072-3436
Joshua RongMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Gregor KasprianDepartment of Biomedical Imaging and Image-Guided Therapy, Medical University of Vienna, Vienna, Austria.
Edward YangDivision of Neuroradiology, Department of Radiology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Darius Ebrahimi-FakhariMovement Disorders Program, Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0002-0026-4714

Funding

Development of a Translational Research Platform to Understand and treat Defective Protein Trafficking in Childhood-Onset Hereditary Spastic ParaplegiaK08NS123552 · NINDS · BOSTON CHILDREN'S HOSPITAL · PI EBRAHIMI-FAKHARI, DARIUS · 2021 to 2025
$1.1M
NINDS NIH HHS K08 NS123552
6 · The paper itself

Abstract

Purpose: Adaptor protein complex 4-associated hereditary spastic paraplegia (AP-4-HSP) is a rare childhood-onset neurogenetic disorder. With gene replacement therapies advancing, early-potentially prenatal-diagnosis holds significant clinical promise. We aimed to characterize fetal and perinatal brain MRI features of AP-4-HSP to assess whether early imaging can prompt timely diagnosis, counseling, and interventions. Methods: In this retrospective analysis, we reviewed prenatal imaging from 303 individuals with genetically confirmed AP-4-HSP enrolled in the Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (NCT04712812). Four patients (covering SPG47, SPG50, SPG52) with fetal, perinatal, or early postmortem imaging available were selected for detailed neuroradiologic evaluation. Systematic assessment documented several structural anomalies, correlated with genotype and clinical progression. Results: Fetal imaging between 22 and 38 weeks' gestation revealed ventriculomegaly, corpus callosum hypoplasia, reduced periventricular white matter, and hippocampal under-rotation across all subtypes. The SPG52 patient exhibited additional severe features, including gyral immaturity and pontine/vermis hypoplasia. Postnatal follow-up demonstrated progressive white matter volume reduction and delayed myelination. Conclusions: This study demonstrates that fetal and perinatal brain MRI can detect early, consistent neurodevelopmental abnormalities in AP-4-HSP, reinforcing its classification as both a neurodevelopmental and neurodegenerative disorder. Integration of prenatal neuroimaging with molecular diagnostics could enable earlier recognition, family counseling, and access to emerging gene therapies. These findings support the incorporation of fetal brain MRI into diagnostic protocols for suspected neurogenetic conditions.

Indexed as

adaptor protein complex 4gene therapyhereditary spastic paraplegianeurodevelopmentprenatal imagingthin corpus callosum

Identifiers

PMID41815511
PMCPMC12974989

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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Registered trials

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 Socratic graph.