What is the management of Charcot-Marie-Tooth (CMT) disease?

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Last updated: April 9, 2025View editorial policy

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From the Research

Management of Charcot-Marie-Tooth disease (CMT) primarily focuses on supportive care and symptom management as there is currently no cure for this hereditary peripheral neuropathy. Treatment begins with a multidisciplinary approach involving neurologists, physical therapists, occupational therapists, and orthopedic specialists.

Key Components of Management

  • Physical therapy is essential to maintain muscle strength, prevent contractures, and improve balance and coordination.
  • Ankle-foot orthoses (AFOs) or other orthotic devices are commonly prescribed to provide stability and prevent falls by compensating for foot drop and ankle weakness.
  • For pain management, medications such as gabapentin (starting at 300mg daily, gradually increasing to 300-600mg three times daily), pregabalin (75-150mg twice daily), or amitriptyline (10-25mg at bedtime) may be used to address neuropathic pain, as suggested by 1.

Rehabilitation and Orthotics

  • Rehabilitation plays a major role in treatment, with aerobic, stretching, and strengthening exercises being effective in improving strength and general fitness, as shown in 2.
  • Orthotic devices are the mainstay of maintaining mobility and ambulation and upper extremity function.

Genetic Counseling and Avoidance of Neurotoxic Medications

  • Genetic counseling should be offered to patients and families to understand inheritance patterns and risks for future generations.
  • Patients should avoid neurotoxic medications like vincristine, cisplatin, and high doses of vitamin A, which can worsen neuropathy, as noted in 3.

Individualized Approach

  • The management approach needs to be individualized based on the specific CMT subtype, disease progression, and the patient's unique symptoms, as CMT encompasses a heterogeneous group of disorders with varying clinical presentations and genetic causes, as discussed in 4 and 5.

Monitoring and Surgical Intervention

  • Regular monitoring for complications like progressive deformities (pes cavus, hammer toes) is important, with surgical intervention considered for severe cases that affect mobility or cause pain.
  • Weight management and maintaining overall health are also important as obesity can exacerbate mobility issues.

References

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This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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