Arthrogryposis Multiplex Congenita
Aetiology
- Non-progressive disorder
- Lack of anterior horn cells
- Myopathic, neuropathic, or mixed
Diagnosis
- Confirmed by:
- Enzyme tests
- Nerve conduction studies (NCS)
- Muscle biopsy at 3 months
Features
- Multiple joint contractures – joints lack skin creases
- Normal facies
- Normal intelligence
- No visceral involvement
Affected Body Areas
- Upper Limbs:
- Shoulder internal rotation and adduction
- Elbow extension contractures
- Management:
- Leave one arm extended
- Manipulation under anesthesia (MUA) & serial casting
- Release and supracondylar osteotomy
- One arm for feeding, other for self-care
- Hips:
- Teratologic dislocations
- Open reduction +/- femoral shortening or VDRO
- Knees:
- Early release of hamstrings
- Feet:
- Congenital vertical talus and rigid club foot
- Will not respond to casting
- Open release; if unsuccessful – talectomy
- Aim for a plantigrade, shoeable foot
- Spine:
- C-shaped large curve scoliosis
- May need fusion to pelvis if progressing
Distal Arthrogryposis Syndrome
- Autosomal dominant
- Similar features to Arthrogryposis Multiplex Congenita
- Hands and feet more affected than proximal joints
Larsen’s Syndrome
- Similar to Arthrogryposis Multiplex Congenita but contractures are milder
- Features:
- Multiple dislocated joints
- Cervical spine kyphosis:
- May progress and cause myelopathy
Multiple Pterygium Syndrome
- Joint contractures with webbing on the flexor aspect
- Surgical caution:
- Neurovascular bundle close during release of webs
Friedrich’s Ataxia
Characteristics
- Most common spinocerebellar disorder
- Autosomal recessive
- Lack of Frataxin protein
- Affects mitochondria
- Onset: 10–15 years
- Death: By 50 years (cardiomyopathy)
Clinical Features & Management
- Staggering, wide-based ataxic gait
- Increased polyphasic waveforms on NCS
- Cardiomyopathy
- Cavovarus/Equinovarus – stiff:
- Scoliosis:
- Needs fusion due to aggressive progression (not always down to pelvis)
Spinal Muscular Atrophy (SMA)
Characteristics
- Most common genetic disease causing pediatric death
- 1 in 10,000 live births
- Loss of anterior horn cells in the spinal cord
- Autosomal recessive
Classification
- SMA1:
- Onset <6 months
- Death by 2 years (respiratory failure)
- SMA2:
- Onset <24 months
- Life expectancy: 15–20 years
- SMA3:
- Onset >24 months
- Normal life expectancy
- Independently stands
Clinical Features & Management
- Weakness develops from proximal to distal
- Hip Dislocations:
- Usually asymptomatic; non-operative treatment
- Scoliosis:
- Severe, progressive
- Large curves causing respiratory compromise
- Aggressive treatment with long fusions
- May reduce walking capability
- Feet:
- Equinovarus common
- Aim for pain-free, plantigrade, shoeable foot
- Use tenotomy & osteotomy rather than transfer
Polio
- Viral infection causing loss of anterior horn cells in spine and brainstem
- Features:
- Often unilateral
- No sensory deficit
- Weakness and smaller limb typical
- No new cases since vaccination, but sequelae still treated
Hereditary Motor Sensory Neuropathies
Charcot-Marie-Tooth Disease (CMT)
Aetiology
- Autosomal dominant disorder causing predominantly distal motor and sensory deficits
- Family history often positive
Types
- HSMN 1 (CMT – Myelopathic/Hypertrophic):
- More common
- Onset: 10–20 years
- Demyelination of nerves
- Nerve conduction studies: Prolonged latencies, reduced velocities
- Absent reflexes
- HSMN 2 (CMT – Neuropathic):
- Less common
- Onset: 20–30 years
- Wallerian degeneration without demyelination
- EMGs: Near normal
- Present reflexes
- HSMN 3 (Dejerine-Sottas Disease):
- Autosomal recessive
- Onset: Infancy
- More severe manifestations:
- Foot drop
- Scoliosis
- Difficulty ambulating
Clinical Features of All Types
- Motor > sensory affliction
- Most affected muscles: Peronei and Tibialis Anterior
- Predominantly foot symptoms:
- Cavus, Cavovarus, hammer toes
- Management:
- Surgery aims to maintain flexibility with transfers/osteotomy
- Avoid fusion where possible – young patients
- Rare Features:
- Hip dislocation
- Scoliosis
- Intrinsic wasting of hands
Guillain-Barre Syndrome
- Idiopathic post-viral polyneuropathy
- Features:
- Symmetric ascending motor neuropathy
- Self-limiting
- Death by respiratory failure
Hemihypertrophy
- Unilateral limb hypertrophy
- Idiopathic cause
- Associated with:
- Neurofibromatosis Type 1 (NF1)
- Renal abnormalities (e.g., Wilm’s tumor)
Klippel-Trenaunay Syndrome
- Hemihypertrophy with associated hemangiomas and AV malformations
- Management:
- Amputation or embolization often necessary
Proteus Syndrome
- Overgrowth of hands and feet with spinal deformity
- Bizarre facial features
- Must differentiate from NF1 and McCune-Albright Syndrome
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