Tuesday 10 December 2013

Charcot-Marie-Tooth disease

MC inherited neurological disorder
1886 described together
Professor Jean Martin Charcot and his student Pierre Marie – Peroneal Muscular Atrophy
Howard Henry Tooth – Peroneal Progressive Muscular Atrophy. Attributed it correctly to neuropathy.
1912 Hoffman described peroneal muscular atrophy with thickened nerve; Hoffman’s disease.

Hereditary Sensory and Motor Neuropathy type I, II and III (Dejerine-Sotta’s disease)

Classification
Type 1
Demyelinating;
further subdivided A (MC, 70%), B & C.
Autosomal dominant
Type 2
Axonal degeneration;
Autosomal dominant or recessive
Type C
Intermediate between axonal degeneration and demyelinating
Autosomal dominant



Clinical Features

Incidence of all types of CMT varies from 1 in 5000 to 1 in 2500
Presents with
Progressive weakness and atrophy of distal muscles
Depressed DTRs
Slowed nerve conduction velocities
Family h/o similar disease
Varied age of onset
Most commonly onset at the 2nd decade of life
CMT Type 2 onset 3rd decade of life
Normal motor milestone
Incidence equal in both sexes, severity more in boys with CMTX

Physical signs

Diminished to absent DTRs
Ankle jerk lost before knee jerk
Sensory loss
2/3rd of the patients
More common in CMT 1 than CMT 2
Muscular weakness
Varied involvement of muscle
MC muscles involved are tibialis anterior and peroneus brevis. May involve all the muscles of the calf
Most severe form – generalized muscle weakness → inability to walk
Atrophy of calf muscles → stork leg appearance
Gait changes
Early stages – slight foot drop seen only in swing phase
Progressively develop complete plantigrade foot with hyperflexion of knee then hip and elevation of hemipelvis
Steppage gait
Foot deformity
Pes cavus, pes cavovarus or claw toe
Hands show atrophy of intrinsic muscles
Progressive scoliosis of spine seen in adolescent and teens
Sensorineural deafness maybe seen in CMTX.

Investigations

Blood tests – known mutations
EMG-NCV
Demyelinating: decrease in velocity with amplitude same as normal
Axonal degeneration: same velocity with decrease amplitude
EMG shows fibrillation due to denervation
Nerve Biopsy
Sural nerve is used
Demyelinating – Onion Bulb appearance
Type 2 – Axonal degeneration
Muscle biopsy – atrophic muscles
MRI Spine
Diffuse enlargement of cauda equina, nerve roots and ganglia
Sonography
Enlargement of median nerve
Medical management
No medical treatment to slow or stop the disease
71% of the patients present with severe neuropathic pain requiring medications

Orthopaedic manifestations

Foot
MC manifestation is pes cavovarus
Atrophy due to denervation of the intrinsic muscles → contractures → elevation of the medial longitudinal arch because of the contracture of plantar fascia
Plantar flexion of the first ray and forefoot equinus → varus of the hind foot
Claw toes – absent intrinsic muscles

Radiographs

Meary’s angle
Angle between the longitudinal axis of talus and the 1st metatarsal
Normal 0-5°
Coleman’s block test
Patient made to stand on a block of wood with the 1st metatarsal head falling off the block
Supple hind foot varus – corrects
Fixed varus – does not correct
Surgical management
Olney divided surgical management into two components
Deformity correction
Soft tissue release
Plantar fascia release
Capsulotomies
Bony fusions or osteotomies
1st metatarsal osteotomy
Calcaneal osteotomy
Triple arthrodesis
Rebalancing of muscle forces
Tendon transfers
Dorsiflexion weakness – EDL or posterior tibial transfer
Overpowering peroneus longus – transfer to peroneus brevis
Hindfoot equinus – tendoachilles lengthening
Claw toes
Paralyses and contracture of intrinsic muscles
Toe extensors recruited to dorsiflex ankle
Jones transfer of the long extensors to the metatarsal to help ankle dorsiflexion
Fusion of IP joint of great toe and PIP joint of the other toes
Hip
Dysplasia of hip
Subtle weakness of the proximal musculature → progressive dysplasia of hip
Asymptomatic till adolescent
Treatment similar to idiopathic adolescent dysplasia of hip

Spine
Scoliosis seen in 37%
Similar to idiopathic scoliosis with increased kyphosis
Highest risk in girls & CMT 1
May present with hyperkyphosis without scoliosis

Treatment

Orthosis
Posterior spinal fusion – failure of orthotic management and with progressive curves
Hand
Onset of hand symptoms occur in 1st to 3rd decade of life; 8 years after the appearance of lower limb symptoms
Present with intrinsic muscle weakness with clawing of ring and small digits occur.
Involvement of ulnar and median nerve innervated forearm and intrinsic muscles
Sparing of radial nerve innervated muscles
Functional problems:
Loss of opposition
Loss of side to side pinch
Clawing of fingers

Management

Electrodiagnostic studies done to determine the best muscle for tendon transfer
Transfers without pulley or if required with static pulley preferred to tendon or tendon loop pulley
Opponensplasty – extensor carpi ulnaris or extensor indicisproprius
Side to side pinch – extensor pollicis brevis, abductor pollicislongus or extensor indicis to 1st dorsal interosseous or adductor pollicis
Muscle transfer done in flexion of MCP joints to compensate extrinsic extensors
Delayed till age when patient can understand the limitations and aftercare
The disease and deformity can be progressive
Transfer requires protection from excessive abuse
Dejerine-Sottas Disease
(Hypertrophic Interstitial Neuritis)
Severe, infantile-onset demyelinating polyneuropathy
Related to same gene as CMT 1A
Enlarged peripheral nerves – proliferation of perineural and endoneural tissues
Classical onion bulb appearance
Presenting complaint
Disability of gait under 3 years of age
Floppy muscles, thickened nerves.
Severe cases present with respiratory failure at birth.
Similar findings as CMT with increased severity and earlier age of onset.
No treatment
Steroids can be given in severe cases.