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Glycosylphosphatidylinositol biosynthesis defect 17(GPIBD17)

MedGen UID:
1648437
Concept ID:
C4747891
Disease or Syndrome
Synonyms: GLYCOSYLPHOSPHATIDYLINOSITOL BIOSYNTHESIS DEFECT 17; GPIBD17
 
Gene (location): PIGH (14q24.1)
 
Monarch Initiative: MONDO:0060724
OMIM®: 618010

Definition

Glycosylphosphatidylinositol biosynthesis defect-17 (GPIBD17) is an autosomal recessive disorder characterized by variable neurologic deficits that become apparent in infancy or early childhood. Patients may present with early-onset febrile or afebrile seizures that tend to be mild or controllable. Other features may include learning disabilities, autism, behavioral abnormalities, hypotonia, and motor deficits. The phenotype is relatively mild compared to that of other GPIBDs (summary by Nguyen et al., 2018). For a discussion of genetic heterogeneity of GPI biosynthesis defects, see GPIBD1 (610293). [from OMIM]

Clinical features

From HPO
Clinodactyly of the 5th finger
MedGen UID:
340456
Concept ID:
C1850049
Congenital Abnormality
Clinodactyly refers to a bending or curvature of the fifth finger in the radial direction (i.e., towards the 4th finger).
Clinodactyly of the 5th toe
MedGen UID:
871256
Concept ID:
C4025741
Anatomical Abnormality
Bending or curvature of a fifth toe in the tibial direction (i.e., towards the big toe).
Growth delay
MedGen UID:
99124
Concept ID:
C0456070
Pathologic Function
A deficiency or slowing down of growth pre- and postnatally.
Abnormal ear morphology
MedGen UID:
1640667
Concept ID:
C4703491
Anatomical Abnormality
Any structural anomaly of the ear.
Aggressive behavior
MedGen UID:
1375
Concept ID:
C0001807
Individual Behavior
Behavior or an act aimed at harming a person, animal, or physical property (e.g., acts of physical violence; shouting, swearing, and using harsh language; slashing someone's tires).
Febrile seizure (within the age range of 3 months to 6 years)
MedGen UID:
3232
Concept ID:
C0009952
Disease or Syndrome
A febrile seizure is any type of seizure (most often a generalized tonic-clonic seizure) occurring with fever (at least 38 degrees Celsius) but in the absence of central nervous system infection, severe metabolic disturbance or other alternative precipitant in children between the ages of 3 months and 6 years.
Seizure
MedGen UID:
20693
Concept ID:
C0036572
Sign or Symptom
A seizure is an intermittent abnormality of nervous system physiology characterized by a transient occurrence of signs and/or symptoms due to abnormal excessive or synchronous neuronal activity in the brain.
Dysplastic corpus callosum
MedGen UID:
98128
Concept ID:
C0431369
Congenital Abnormality
Dysplasia and dysgenesis of the corpus callosum are nonspecific descriptions that imply defective development of the corpus callosum. The term dysplasia is applied when the morphology of the corpus callosum is altered as a congenital trait. For instance, the corpus callosum may be hump-shaped, kinked, or a striped corpus callosum that lacks an anatomically distinct genu and splenium.
Bilateral tonic-clonic seizure
MedGen UID:
141670
Concept ID:
C0494475
Sign or Symptom
A bilateral tonic-clonic seizure is a seizure defined by a tonic (bilateral increased tone, lasting seconds to minutes) and then a clonic (bilateral sustained rhythmic jerking) phase.
Global developmental delay
MedGen UID:
107838
Concept ID:
C0557874
Finding
A delay in the achievement of motor or mental milestones in the domains of development of a child, including motor skills, speech and language, cognitive skills, and social and emotional skills. This term should only be used to describe children younger than five years of age.
Poor speech
MedGen UID:
341172
Concept ID:
C1848207
Finding
Generalized myoclonic seizure
MedGen UID:
892704
Concept ID:
C4021759
Disease or Syndrome
A generalized myoclonic seizure is a type of generalized motor seizure characterized by bilateral, sudden, brief (<100 ms) involuntary single or multiple contraction of muscles or muscle groups of variable topography (axial, proximal limb, distal). Myoclonus is less regularly repetitive and less sustained than is clonus.
Overfriendliness
MedGen UID:
867995
Concept ID:
C4022386
Mental or Behavioral Dysfunction
A form of hypersociability that presents as mostly inappropriate friendliness towards others.
Specific learning disability
MedGen UID:
871302
Concept ID:
C4025790
Mental or Behavioral Dysfunction
Impairment of certain skills such as reading or writing, coordination, self-control, or attention that interfere with the ability to learn. The impairment is not related to a global deficiency of intelligence.
Generalized hypotonia
MedGen UID:
346841
Concept ID:
C1858120
Finding
Generalized muscular hypotonia (abnormally low muscle tone).
Microcephaly
MedGen UID:
1644158
Concept ID:
C4551563
Finding
Head circumference below 2 standard deviations below the mean for age and gender.
Osteomyelitis
MedGen UID:
10497
Concept ID:
C0029443
Disease or Syndrome
Osteomyelitis is an inflammatory process accompanied by bone destruction and caused by an infecting microorganism.
Chronic otitis media
MedGen UID:
75751
Concept ID:
C0271441
Disease or Syndrome
Chronic otitis media refers to fluid, swelling, or infection of the middle ear that does not heal and may cause permanent damage to the ear.
Hypertriglyceridemia
MedGen UID:
167238
Concept ID:
C0813230
Finding
An abnormal increase in the level of triglycerides in the blood.
Abnormality of alkaline phosphatase level
MedGen UID:
892397
Concept ID:
C4025328
Finding
An abnormality of alkaline phosphatase level.
High palate
MedGen UID:
66814
Concept ID:
C0240635
Congenital Abnormality
Height of the palate more than 2 SD above the mean (objective) or palatal height at the level of the first permanent molar more than twice the height of the teeth (subjective).
Dry skin
MedGen UID:
56250
Concept ID:
C0151908
Sign or Symptom
Skin characterized by the lack of natural or normal moisture.
Nevus spilus
MedGen UID:
87538
Concept ID:
C0346099
Neoplastic Process
A tan, regularly bordered patch with darker macules within the lesion.

Professional guidelines

PubMed

Zurutuza L, Muller F, Gibrat JF, Taillandier A, Simon-Bouy B, Serre JL, Mornet E
Hum Mol Genet 1999 Jun;8(6):1039-46. doi: 10.1093/hmg/8.6.1039. PMID: 10332035

Recent clinical studies

Etiology

Hosokawa K, Sugimori N, Katagiri T, Sasaki Y, Saito C, Seiki Y, Mochizuki K, Yamazaki H, Takami A, Nakao S
Eur J Haematol 2015 Sep;95(3):230-8. Epub 2015 Feb 4 doi: 10.1111/ejh.12484. PMID: 25404431

Diagnosis

Ng BG, Freeze HH, Himmelreich N, Blau N, Ferreira CR
Mol Genet Metab 2024 May;142(1):108476. Epub 2024 Apr 10 doi: 10.1016/j.ymgme.2024.108476. PMID: 38653092Free PMC Article
Stolarczyk M, Veit G, Schnúr A, Veltman M, Lukacs GL, Scholte BJ
Am J Physiol Lung Cell Mol Physiol 2018 Apr 1;314(4):L555-L568. Epub 2017 Dec 14 doi: 10.1152/ajplung.00458.2017. PMID: 29351448
Hosokawa K, Sugimori N, Katagiri T, Sasaki Y, Saito C, Seiki Y, Mochizuki K, Yamazaki H, Takami A, Nakao S
Eur J Haematol 2015 Sep;95(3):230-8. Epub 2015 Feb 4 doi: 10.1111/ejh.12484. PMID: 25404431
Tashima Y, Taguchi R, Murata C, Ashida H, Kinoshita T, Maeda Y
Mol Biol Cell 2006 Mar;17(3):1410-20. Epub 2006 Jan 11 doi: 10.1091/mbc.e05-11-1005. PMID: 16407401Free PMC Article
Kennard ML, Richardson DR, Gabathuler R, Ponka P, Jefferies WA
EMBO J 1995 Sep 1;14(17):4178-86. doi: 10.1002/j.1460-2075.1995.tb00091.x. PMID: 7556058Free PMC Article

Therapy

Hosokawa K, Sugimori N, Katagiri T, Sasaki Y, Saito C, Seiki Y, Mochizuki K, Yamazaki H, Takami A, Nakao S
Eur J Haematol 2015 Sep;95(3):230-8. Epub 2015 Feb 4 doi: 10.1111/ejh.12484. PMID: 25404431

Prognosis

Hosokawa K, Sugimori N, Katagiri T, Sasaki Y, Saito C, Seiki Y, Mochizuki K, Yamazaki H, Takami A, Nakao S
Eur J Haematol 2015 Sep;95(3):230-8. Epub 2015 Feb 4 doi: 10.1111/ejh.12484. PMID: 25404431
Anand M, Rush JS, Ray S, Doucey MA, Weik J, Ware FE, Hofsteenge J, Waechter CJ, Lehrman MA
Mol Biol Cell 2001 Feb;12(2):487-501. doi: 10.1091/mbc.12.2.487. PMID: 11179430Free PMC Article
Kawaguchi K, Wada H, Mori A, Takemoto Y, Kakishita E, Kanamaru A
Br J Haematol 1999 Apr;105(1):80-4. PMID: 10233366

Clinical prediction guides

Anand M, Rush JS, Ray S, Doucey MA, Weik J, Ware FE, Hofsteenge J, Waechter CJ, Lehrman MA
Mol Biol Cell 2001 Feb;12(2):487-501. doi: 10.1091/mbc.12.2.487. PMID: 11179430Free PMC Article
Zurutuza L, Muller F, Gibrat JF, Taillandier A, Simon-Bouy B, Serre JL, Mornet E
Hum Mol Genet 1999 Jun;8(6):1039-46. doi: 10.1093/hmg/8.6.1039. PMID: 10332035
Kawaguchi K, Wada H, Mori A, Takemoto Y, Kakishita E, Kanamaru A
Br J Haematol 1999 Apr;105(1):80-4. PMID: 10233366

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