Neurodevelopmental Disorder with Psychomotor Delay, Hearing Loss, and Spasticity Caused by Compound Heterozygous SPATA5L1 Variants—Expanding Phenotype

<b>Background:</b><i>SPATA5L1</i>-related neurodevelopmental disorder is a recently described condition characterized by psychomotor delay, sensorineural hearing loss, and variable motor dysfunction. Because only a few cases have been reported, the full phenotypic spectrum remains poorly defined. Expanding clinical characterization is crucial for improving early diagnosis and targeted management. <b>Case Presentation:</b> We report a 24-month-old female with compound heterozygous <i>SPATA5L1</i> variants c.1918C>T (p.Arg640Ter) and c.2066G>T (p.Gly689Val). She presented with global psychomotor delay, bilateral sensorineural hearing loss, strabismus, and craniofacial dysmorphism. Brain MRI showed cortical and white matter atrophy, delayed myelination, and a thin corpus callosum. Vojta neurodevelopmental assessment demonstrated an 11-month motor delay, abnormal responses in all seven Vojta postural reactions, and persistent primitive reflexes. Early EEG recordings were without significant changes, whereas abnormalities emerged later in the clinical course. Genetic testing confirmed the variants in trans. <b>Management and Outcomes:</b> Early rehabilitation including reflex locomotion therapy was initiated. The persistence of primitive reflexes, central hypotonia, and pathological postural reactions provided a coherent neuromotor profile and indicated a high vulnerability to atypical motor development, and do not rule out the possibility of later evolution toward a spastic-dystonic motor pattern. These findings, combined with neuroimaging abnormalities, refined the patient's neuromotor phenotype and guided individualized therapeutic planning. <b>Conclusions:</b> This case expands the clinical and neurodevelopmental spectrum associated with <i>SPATA5L1</i> variants and highlights the diagnostic value of integrating genomic sequencing with structured motor assessments. Early, multidimensional evaluation may improve recognition of rare neurodevelopmental disorders and support more precise prognostication and rehabilitation strategies.

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Journal
Journal of Clinical Medicine
Published
2025-11-28
DOI
https://doi.org/10.3390/jcm14238442
Primary Topic
Genomics and Rare Diseases
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article
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article

Neurodevelopmental Disorder with Psychomotor Delay, Hearing Loss, and Spasticity Caused by Compound Heterozygous SPATA5L1 Variants—Expanding Phenotype

Artur Polczyk, Mateusz Biela, Robert Śmigiel, Agnieszka Pawelak et al.
Journal of Clinical Medicine
Genomics and Rare Diseases
article

Neurodevelopmental Disorder with Psychomotor Delay, Hearing Loss, and Spasticity Caused by Compound Heterozygous SPATA5L1 Variants—Expanding Phenotype

Artur Polczyk, Mateusz Biela, Robert Śmigiel, Agnieszka Pawelak, Ewelina Wolańska, Agnieszka Zubkiewicz‐Kucharska, Anna Zimny
article en

Abstract

<b>Background:</b><i>SPATA5L1</i>-related neurodevelopmental disorder is a recently described condition characterized by psychomotor delay, sensorineural hearing loss, and variable motor dysfunction. Because only a few cases have been reported, the full phenotypic spectrum remains poorly defined. Expanding clinical characterization is crucial for improving early diagnosis and targeted management. <b>Case Presentation:</b> We report a 24-month-old female with compound heterozygous <i>SPATA5L1</i> variants c.1918C>T (p.Arg640Ter) and c.2066G>T (p.Gly689Val). She presented with global psychomotor delay, bilateral sensorineural hearing loss, strabismus, and craniofacial dysmorphism. Brain MRI showed cortical and white matter atrophy, delayed myelination, and a thin corpus callosum. Vojta neurodevelopmental assessment demonstrated an 11-month motor delay, abnormal responses in all seven Vojta postural reactions, and persistent primitive reflexes. Early EEG recordings were without significant changes, whereas abnormalities emerged later in the clinical course. Genetic testing confirmed the variants in trans. <b>Management and Outcomes:</b> Early rehabilitation including reflex locomotion therapy was initiated. The persistence of primitive reflexes, central hypotonia, and pathological postural reactions provided a coherent neuromotor profile and indicated a high vulnerability to atypical motor development, and do not rule out the possibility of later evolution toward a spastic-dystonic motor pattern. These findings, combined with neuroimaging abnormalities, refined the patient's neuromotor phenotype and guided individualized therapeutic planning. <b>Conclusions:</b> This case expands the clinical and neurodevelopmental spectrum associated with <i>SPATA5L1</i> variants and highlights the diagnostic value of integrating genomic sequencing with structured motor assessments. Early, multidimensional evaluation may improve recognition of rare neurodevelopmental disorders and support more precise prognostication and rehabilitation strategies.

Journal of Clinical MedicineVol. 14(23)
Wroclaw Medical University (PL), Wroclaw University of Health and Sport Sciences (PL), Academy of Physiotherapy in Wrocław (PL)
Openalex Percentile: Top 61%
Genomics and Rare Diseases
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