Abstract
Schinzel-Giedion syndrome is a rare recognizable malformation syndrome defined by characteristic facial features, profound developmental delay, severe growth failure, and multiple congenital anomalies. The causative gene of Schinzel-Giedion syndrome, SETBP1, has been identified, but limited cases have been confirmed by molecular analysis. We present a 9-month-old girl affected by West syndrome with Schinzel-Giedion syndrome. Congenital severe hydronephrosis, typical facial features, and multiple anomalies suggested a clinical diagnosis of Schinzel-Giedion syndrome. Hypsarrhythmia occurred at 7 months of age and was temporarily controlled by adrenocorticotropic hormone (ACTH) therapy during 5 weeks. SETBP1 mutational analysis showed the presence of a recurrent mutation, p.Ile871Thr. The implications in management of Schinzel-Giedion syndrome are discussed.
Keywords
SET binding protein 1 (SETBP1)
adrenocorticotropic hormone (ACTH)
epilepsy
MeSH Terms
Abnormalities, Multiple/drug therapy,genetics,pathology
Adrenocorticotropic Hormone/therapeutic use
Brain/pathology,physiopathology
Carrier Proteins/genetics
Craniofacial Abnormalities/complications,drug therapy,genetics,pathology
DNA Mutational Analysis
Diagnosis, Differential
Electroencephalography
Female
Hand Deformities, Congenital/complications,drug therapy,genetics,pathology
Hormones/therapeutic use
Humans
Infant
Intellectual Disability/complications,drug therapy,genetics,pathology
Mutation
Nails, Malformed/complications,drug therapy,genetics,pathology
Nuclear Proteins/genetics
Spasms, Infantile/complications,drug therapy,genetics,pathology
Chemicals
Carrier Proteins
Hormones
Nuclear Proteins
SETBP1 protein, human
Adrenocorticotropic Hormone
Authors & Affiliations
6 authors, click to expand affiliations / ORCID
Miyake Fuyu
Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan.
Kuroda Yukiko
Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan.
Naruto Takuya
Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan.
Ohashi Ikuko
Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan.
Takano Kyoko
Division of Neurology, Kanagawa Children's Medical Center, Yokohama, Japan.
Kurosawa Kenji
Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan
[email protected].
Supplementary Concepts
Schinzel-Giedion syndrome (Disease)