Is there sufficient evidence for the use of anabolic steroids to treat Charcot-Marie-Tooth (CMT) disease?

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Last updated: March 24, 2025View editorial policy

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From the Guidelines

There is currently insufficient evidence to recommend anabolic steroids for the treatment of Charcot-Marie-Tooth disease (CMT). The most recent and highest quality study, published in 2021 in the journal Clinical Nutrition 1, states that there are insufficient consistent clinical data to recommend currently approved androgenic steroids to increase muscle mass, with a strength of recommendation of none and a level of evidence classified as low. This guideline, although focused on clinical nutrition in cancer, highlights the lack of evidence for using anabolic steroids to improve muscle mass in general, which can be applied to the context of CMT.

When considering the treatment of CMT, it is crucial to prioritize evidence-based approaches that focus on supportive care, physical therapy, orthotic devices, and pain management. CMT is a complex hereditary peripheral neuropathy that affects the nerves controlling muscles and transmitting sensory information, and its management should be tailored to the specific type of CMT and the patient's symptom profile.

Key considerations in the management of CMT include:

  • Supportive care to manage symptoms and prevent complications
  • Physical therapy to maintain muscle strength and function
  • Orthotic devices to support mobility and reduce the risk of falls
  • Pain management strategies to address neuropathic pain

The use of anabolic steroids, given their potential for significant side effects including liver damage, cardiovascular complications, hormonal imbalances, and psychological effects, as noted in various clinical contexts 1, is not justified without robust evidence demonstrating their efficacy in treating CMT. Therefore, patients with CMT should work closely with neurologists specializing in neuromuscular disorders to develop personalized, evidence-based treatment plans.

From the Research

Anabolic Steroids in Charcot-Marie-Tooth Disease Treatment

  • There is no mention of anabolic steroids as a treatment for Charcot-Marie-Tooth (CMT) disease in the provided studies 2, 3, 4, 5, 6.
  • The studies focus on rehabilitation approaches, such as exercise, physiotherapy, and orthotics, as well as potential therapeutic strategies like genetic therapy and molecule inhibitors 2, 3, 5, 6.
  • Massage therapy is also explored as a potential complementary treatment for reducing pain and improving range of motion in CMT patients 4.
  • The lack of evidence on anabolic steroids in CMT treatment suggests that other approaches are being prioritized in research and clinical practice 2, 3, 4, 5, 6.

Current Treatment Approaches

  • Rehabilitation and occupational therapy are currently the main management strategies for CMT patients 2, 3.
  • Exercise, including strength and endurance training, is shown to improve functionality and activities of daily living (ADLs) in CMT patients 5, 6.
  • Orthotics and other assistive devices are also used to maintain mobility and ambulation in CMT patients 3, 5.

Professional Medical Disclaimer

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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