Multiplex Ligation-Dependent Probe Amplification (MLPA)
Beckwith-Weidemann syndrome (BWS) is a congenital overgrowth condition associated with neonatal hypoglycemia, macroglossia, macrosomia, hemihypertrophy and increased risk for embryonal tumors. Russell-Silver syndrome (RSS) is a congenital condition characterized by stunted grow, limb length asymmetry, and developmental delay. Testing can confirm a suspected clinical diagnosis of BWS or RSS.
Disease Overview
Incidence
BWS: ~1/10,000-13,700 newborns
RSS: ~1/100,000 newborns
Symptoms
BWS (Major Findings) | RSS |
---|---|
Macrosomia Visceromegaly Hemihyperplasia Embryonal tumors in childhood (eg, Wilms tumor, hepatoblastoma, neuroblastoma, rhabdomyosarcoma) Macroglossia Omphalocele Renal abnormalities Ear creases or pits |
Pre- and postnatal growth deficiency Proportionate short stature Limb length asymmetry Developmental delay and/or learning disabilities Triangular facies, broad forehead, narrow chin |
Genetics
Etiology
Causes of BWS
- 50% have loss of maternal methylation on chromosome 11p15 imprinting center (IC)2
- 20% have paternal uniparental disomy (UPD) for chromosome 11p15
- 5% have gain of methylation in maternal IC1
- Pathogenic sequence variants in CDKN1C
- 5-10% of nonfamilial cases
- ~40% of familial cases
- <1% cytogenetic abnormalities involving 11p15
Causes of RSS
- 35-50% have hypomethylation of paternal IC1
- 10% have maternal UPD of chromosome 7
- ~40% have an unknown genetic mechanism
Inheritance
- Sporadic in 85% of BWS cases and 60% of RSS cases
- Autosomal dominant in 15% of BWS cases due to parent-of-origin transmission
Penetrance
- Complete for RSS
- Incomplete for BWS due to methylation (eg, individuals with a paternally inherited CDKN1C pathogenic variant will not show features of BWS)
Test Interpretation
Clinical sensitivity/specificity: 75% for BWS; 35-50% for RSS
Analytical sensitivity/specificity: 99%
Results
Result | BWS | RSS |
---|---|---|
Positive |
IC2 hypomethylation AND normal IC1 methylation IC1 hypermethylation AND hypomethylation of IC2 IC1 hypermethylation AND normal methylation of IC2 |
IC1 hypomethylation |
Negative |
Normal methylation patterns:
|
Normal methylation patterns:
|
Limitations
Molecular mechanisms causing BWS or RSS that do not affecting methylation patterns are not assessed, including:
- Maternal UPD of chromosome 7
- Chromosomal translocations, inversions, deletions, or duplications
- Pathogenic CDKN1C sequence variants, deletions/duplications
- Diagnostic errors can occur due to rare sequence variations.
Confirm diagnosis of BWS or RSS in individuals with a suspected clinical diagnosis