Case Report: SATB2-Associated Syndrome Overlapping With Clinical Mitochondrial Disease Presentation: Report of Two Cases

SATB2-associated syndrome (SAS) is an autosomal dominant neurogenetic multisystemic disorder. We describe two individuals with global developmental delay and hypotonia who underwent an extensive evaluation to rule out an underlying mitochondrial disorder before their eventual diagnosis of SAS. Although the strict application of the clinical mitochondrial disease score only led to the designation of “possible” mitochondrial disorder for these two individuals, other documented abnormalities included nonspecific neuroimaging findings on magnetic resonance imaging and magnetic resonance spectroscopy, decreased complex I activity on muscle biopsy for patient 2, and variation in the size and relative proportion of types of muscle fibers in the muscle biopsies that were aligned with mitochondrial diseases. SAS should be in the differential diagnoses of mitochondrial disorders, and broad-spectrum diagnostic tests such as exome sequencing need to be considered early in the evaluation process of undiagnosed neurodevelopmental disorders.

[1]  R. Frye Mitochondrial Dysfunction in Autism Spectrum Disorder: Unique Abnormalities and Targeted Treatments. , 2020, Seminars in pediatric neurology.

[2]  A. Vanderver,et al.  Epilepsy and Electroencephalographic Abnormalities in SATB2-Associated Syndrome. , 2020, Pediatric neurology.

[3]  V. Dubowitz,et al.  Histological and Histochemical Stains and Reactions , 2020, Muscle Biopsy.

[4]  A. Munnich,et al.  High predictive value of brain MRI imaging in primary mitochondrial respiratory chain deficiency , 2018, Journal of Medical Genetics.

[5]  R. Horvath,et al.  Revisiting mitochondrial diagnostic criteria in the new era of genomics , 2017, Genetics in Medicine.

[6]  B. Peterlin,et al.  Comprehensive use of extended exome analysis improves diagnostic yield in rare disease: a retrospective survey in 1,059 cases , 2017, Genetics in Medicine.

[7]  Joan,et al.  Prevalence and architecture of de novo mutations in developmental disorders , 2017, Nature.

[8]  W. Chung,et al.  Clinical application of whole-exome sequencing across clinical indications , 2015, Genetics in Medicine.

[9]  R. Frye,et al.  Primary Mitochondrial Disease and Secondary Mitochondrial Dysfunction: Importance of Distinction for Diagnosis and Treatment , 2016, Molecular Syndromology.

[10]  Marni J. Falk,et al.  Diagnosis and management of mitochondrial disease: a consensus statement from the Mitochondrial Medicine Society , 2014, Genetics in Medicine.

[11]  S. Rahman,et al.  Complex I deficiency: clinical features, biochemistry and molecular genetics , 2012, Journal of Medical Genetics.

[12]  Marni J. Falk,et al.  Neurodevelopmental Manifestations of Mitochondrial Disease , 2010, Journal of developmental and behavioral pediatrics : JDBP.

[13]  S. Packman,et al.  Relationship of primary mitochondrial respiratory chain dysfunction to fiber type abnormalities in skeletal muscle , 2005, Clinical genetics.

[14]  M. Zeviani,et al.  Clinical and molecular findings in children with complex I deficiency. , 2004, Biochimica et biophysica acta.

[15]  Giovanni Cioni,et al.  Proton MR spectroscopy of mitochondrial diseases: analysis of brain metabolic abnormalities and their possible diagnostic relevance. , 2003, AJNR. American journal of neuroradiology.

[16]  C. Sewry Immunocytochemical analysis of human muscular dystrophy , 2000, Microscopy research and technique.