MECKEL-GRUBER SYNDROME

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    MECKEL-GRUBER SYNDROME

     

    DEFINITION:

    An autosomal recessive disorder of unknown etiology characterized by an occipital encephalocele, cystic dysplastic kidneys, and polydactyly.

    EPIDEMIOLOGY:

    • incidence: over 200 cases reported
    • age of onset:
      • prenatally by ultrasound or at birth
    • risk factors:
      • familial - autosomal recessive
        • chrom.#: 17q21-q24
        • gene: ?
      • M = F
      • prevalent in Finland (1:9000 births), and among the Tatars and the Gujarati Indians

    PATHOGENESIS:

    1. Background

    • also called Meckel Syndrome, Gruber Syndrome, Dysencephalia Splanchnocytica
    • originally described by J.R. Meckel in 1822 and later by G.B. Gruber in 1934 who called the condition Dysencephalia Splanchnocytica

    2. Pathogenesis

    • unknown

    CLINICAL FEATURES:

    1. Central Nervous System Manifestations

    • occipital encephalocele
    • anencephaly, microcephaly, or hydrocephaly +/- Arnold-Chiari Malformation
    • cerebral and cerebellar hypoplasia
    • absent olfactory lobes and tract, corpus callosum, and septum pallucidum

    2. Facial Manifestations

    • broad, round face
    • low, sloping forehead
    • eyes:
      • hypo- or hypertelorism, upslanting palpebral fissures, microphthalmia, coloboma of the iris, cataracts
    • broad, flattened nose
    • broad cheeks
    • low set ears
    • hypoplastic or absent philtrum and/or nasal septum
    • mouth:
      • wide, full lips, cleft palate +/- cleft lip, natal teeth, buccal frenula, small lobulated or cleft tongue
    • micrognathia
    • short, webbed neck
    • Potter-like facies with severe oligohydramnios

    3. Renal Manifestations

    • bilateral cystic dysplasia with grossly enlarged kidneys
    • absent, hypoplastic, or duplicated ureters
    • absent or hypoplastic bladder
    • urethra absent or not connected to the bladder

    4. Limb Malformations

    • postaxial polydactyly usually bilateral involving both hands and feet
    • partial or total syndactyly of the fingers and toes
    • clinodactyly
    • ulnar deviation of the hands
    • clubfeet
    • short, bowed limbs

    5. Genital Manifestations

    • ambiguous genitalia
    • absent ovaries or testes (cryptorchidism)

    6. Cardiac Manifestations

    • septal defects (ASD, VSD)
    • Patent Ductus Arteriosus
    • Coarctation of the Aorta
    • Pulmonary Stenosis

    7. Gastrointestinal Manifestations

    1. Liver and Biliary System

    • hepatomegaly with microscopic cysts and fibrosis
    • absent gall bladder
    • dilated bile ducts

    2. Spleen

    • absent, accessory, or splenomegaly

    3. Adrenals

    • absent or hypoplastic

    4. Others

    • omphalocele
    • intestinal malrotation
    • imperforate anus

    8. Respiratory Manifestations

    • hypoplastic lungs

    INVESTIGATIONS:

    1. Prenatal Diagnosis

    • occipital encephalocele or cystic dysplastic kidneys detected by ultrasound as early as midtrimester
    • elevated maternal and amniotic fluid alpha fetoprotein (with encephalocele or anencephaly)

    MANAGEMENT:

    1. Supportive

    • most cases are stillborn or die within a few days to weeks of delivery
    • oldest known survivor died at 4 months of age
    • genetic counselling

    ADDITIONAL REFERENCES:

    1. Jones, K.L., Smith's Recognizable Patterns of Human Malformation (5th Edition). p. 184-185. (1997).
    2. Baraitser, M. and R.M. Winter. Color Atlas of Congenital Malformation Syndromes (1st Edition). p. 64. (1996).
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    Pediatric Database - MECKEL-GRUBER SYNDROME

    Pediatric Organization - Pedbase [at] Gmail.com