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Liberfarb syndrome(LIBF)

MedGen UID:
1709796
Concept ID:
C5394404
Disease or Syndrome
Synonyms: LIBF; Short stature-skeletal dysplasia-retinal degeneration-intellectual disability-sensorineural hearing loss syndrome; SPONDYLOEPIMETAPHYSEAL DYSPLASIA, LIBERFARB TYPE
SNOMED CT: Short stature, skeletal dysplasia, retinal degeneration, intellectual disability, sensorineural hearing loss syndrome (1284851009); Liberfarb syndrome (1284851009)
 
Gene (location): PISD (22q12.2)
 
Monarch Initiative: MONDO:0030045
OMIM®: 618889
Orphanet: ORPHA589442

Definition

Liberfarb syndrome is a progressive disorder involving connective tissue, bone, retina, ear, and brain. Patients exhibit severe short stature and scoliosis with thoracic kyphosis and lumbar hyperlordosis. Severe joint laxity results in dislocations of elbows, hips, and knees. Eye findings are consistent with early-onset retinal degeneration, and there is moderate to severe early-onset hearing loss. Microcephaly is apparent by school age, and patients exhibit developmental delay and intellectual deficits (Peter et al., 2019). Clinical variability has been observed, with some patients presenting differences in the severity and location of skeletal dysplasia involvement as well as variation in other features of the syndrome (Girisha et al., 2019; Zhao et al., 2019). [from OMIM]

Clinical features

From HPO
Short stature
MedGen UID:
87607
Concept ID:
C0349588
Finding
A height below that which is expected according to age and gender norms. Although there is no universally accepted definition of short stature, many refer to "short stature" as height more than 2 standard deviations below the mean for age and gender (or below the 3rd percentile for age and gender dependent norms).
Sensorineural hearing loss disorder
MedGen UID:
9164
Concept ID:
C0018784
Disease or Syndrome
A type of hearing impairment in one or both ears related to an abnormal functionality of the cochlear nerve.
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.
Intellectual disability
MedGen UID:
811461
Concept ID:
C3714756
Mental or Behavioral Dysfunction
Intellectual disability, previously referred to as mental retardation, is characterized by subnormal intellectual functioning that occurs during the developmental period. It is defined by an IQ score below 70.
Scoliosis
MedGen UID:
11348
Concept ID:
C0036439
Disease or Syndrome
The presence of an abnormal lateral curvature of the spine.
Metaphyseal striations
MedGen UID:
376512
Concept ID:
C1849081
Finding
Longitudinal densities on radiographs located in a metaphysis (the narrow region of a long bone between the epiphysis and the diaphysis).
Delayed epiphyseal ossification
MedGen UID:
351324
Concept ID:
C1865200
Finding
Microcephaly
MedGen UID:
1644158
Concept ID:
C4551563
Finding
Head circumference below 2 standard deviations below the mean for age and gender.
Retinal degeneration
MedGen UID:
48432
Concept ID:
C0035304
Finding
A nonspecific term denoting degeneration of the retinal pigment epithelium and/or retinal photoreceptor cells.
Optic disc pallor
MedGen UID:
108218
Concept ID:
C0554970
Finding
A pale yellow discoloration of the optic disc (the area of the optic nerve head in the retina). The optic disc normally has a pinkish hue with a central yellowish depression.
Bone spicule pigmentation of the retina
MedGen UID:
323029
Concept ID:
C1836926
Finding
Pigment migration into the retina in a bone-spicule configuration (resembling the nucleated cells within the lacuna of bone).
Retinal pigment epithelial mottling
MedGen UID:
347513
Concept ID:
C1857644
Finding
Mottling (spots or blotches with different shades) of the retinal pigment epithelium, i.e., localized or generalized fundal pigment granularity associated with processes at the level of the retinal pigment epithelium.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVLiberfarb syndrome

Professional guidelines

PubMed

Rose PS, Levy HP, Liberfarb RM, Davis J, Szymko-Bennett Y, Rubin BI, Tsilou E, Griffith AJ, Francomano CA
Am J Med Genet A 2005 Oct 15;138A(3):199-207. doi: 10.1002/ajmg.a.30955. PMID: 16152640
Liberfarb RM, Levy HP, Rose PS, Wilkin DJ, Davis J, Balog JZ, Griffith AJ, Szymko-Bennett YM, Johnston JJ, Francomano CA, Tsilou E, Rubin BI
Genet Med 2003 Jan-Feb;5(1):21-7. doi: 10.1097/00125817-200301000-00004. PMID: 12544472

Recent clinical studies

Diagnosis

Pinto E, Pinto C, Ramos C, Alves JE
Pediatr Radiol 2023 Mar;53(3):561-563. Epub 2022 Sep 22 doi: 10.1007/s00247-022-05503-7. PMID: 36136119

Clinical prediction guides

Pinto E, Pinto C, Ramos C, Alves JE
Pediatr Radiol 2023 Mar;53(3):561-563. Epub 2022 Sep 22 doi: 10.1007/s00247-022-05503-7. PMID: 36136119
Peter VG, Quinodoz M, Pinto-Basto J, Sousa SB, Di Gioia SA, Soares G, Ferraz Leal G, Silva ED, Pescini Gobert R, Miyake N, Matsumoto N, Engle EC, Unger S, Shapiro F, Superti-Furga A, Rivolta C, Campos-Xavier B
Genet Med 2019 Dec;21(12):2734-2743. Epub 2019 Jul 2 doi: 10.1038/s41436-019-0595-x. PMID: 31263216Free PMC Article

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