What is the management and treatment for a patient with MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes) syndrome?

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Last updated: June 16, 2025View editorial policy

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From the Guidelines

The management of MELAS syndrome focuses on supportive care, symptom management, and preventing metabolic crises as there is no definitive cure. The treatment typically includes a combination of coenzyme Q10, L-arginine, and L-carnitine, which aim to improve mitochondrial function and energy production 1. These supplements are used in the following doses: coenzyme Q10 (100-300 mg daily), L-arginine (0.4-0.5 g/kg/day during acute stroke-like episodes and 150-300 mg/kg/day for maintenance), and L-carnitine (50-100 mg/kg/day). Additional medications may include riboflavin, creatine, and vitamin E, which are used to reduce oxidative stress and improve energy metabolism. During acute stroke-like episodes, intravenous L-arginine should be administered over 30-60 minutes 1. It is essential to avoid valproic acid, which can worsen mitochondrial dysfunction. Management also requires a multidisciplinary approach with neurologists, cardiologists, endocrinologists, and other specialists to address specific organ involvement. Regular monitoring of cardiac function, hearing, vision, and blood glucose is essential, and patients should maintain adequate hydration, avoid fasting, and follow a balanced diet potentially enriched with complex carbohydrates. Physical therapy helps manage myopathy symptoms, while seizures require appropriate anticonvulsant therapy. These interventions aim to reduce oxidative stress, improve energy metabolism, and enhance nitric oxide production to improve cerebral blood flow, thereby reducing the frequency and severity of stroke-like episodes and other MELAS manifestations. Some key points to consider in the management of MELAS syndrome include:

  • The use of L-arginine as a nitric oxide precursor to improve endothelial function 1
  • The importance of avoiding valproic acid due to its potential to worsen mitochondrial dysfunction
  • The need for regular monitoring of cardiac function, hearing, vision, and blood glucose
  • The role of physical therapy in managing myopathy symptoms
  • The use of appropriate anticonvulsant therapy to manage seizures.

From the Research

Management and Treatment of MELAS Syndrome

The management and treatment of MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes) syndrome is largely symptomatic and involves a multidisciplinary team approach 2.

  • Medications: The drugs most often used to treat MELAS syndrome include antioxidants and various vitamins aimed at minimizing the demands on the mitochondria and supporting and maximizing their function 3. Some of the most frequently prescribed agents include:
    • Coenzyme Q(10)
    • L-arginine
    • B vitamins
    • Levocarnitine
  • L-arginine Therapy: Unblinded studies have shown that L-arginine therapy improves stroke-like episode symptoms and decreases the frequency and severity of these episodes 2.
  • Citrulline and Arginine Supplementation: Supplementing the NO precursors arginine and citrulline restores NO production in MELAS syndrome, and citrulline supplementation has been shown to decrease plasma lactate levels 4.
  • Ketogenic Diet: A ketogenic diet should be initiated as a supportive measure for patients with MELAS syndrome 5.
  • Antiepileptic Drugs: Seizures prior to or during a stroke-like episode or paroxysmal EEG-activity during a stroke-like episode should be initially treated with antiepileptic drugs (AEDs) with low mitochondrion-toxicity 5.
  • Regional Anesthesia: Regional anesthesia, such as spinal anesthesia, may be a satisfactory choice for patients with MELAS syndrome undergoing surgery, but it should only be performed when neurological abnormalities of spinal cord or peripheral nerves are definitely ruled out 6.

Monitoring and Support

  • EEG Recordings: All patients with stroke-like episodes need to have an EEG recorded, irrespective of whether they have manifesting seizures or not 5.
  • Multidisciplinary Team: Management of MELAS syndrome should involve a multidisciplinary team approach to address the various clinical manifestations and multisystem effects of the disease 2.

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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