Drug Information

As an initiative of the International Mito Patients (IMP), a group of experts in mitochondrial disease carried out a study into the safety of drugs in mitochondrial patients. The study took place on an international level and resulted in the publication of a list of drugs to be used with caution in primary mitochondrial disease.

It is important to remember that side effects can occur with any medicine in any patient. This can affect anyone taking the medicine and may not be related to your mitochondrial disease. It is therefore essential that you take the advice of your doctor and read the information given with the medicine.

What is the list?

This is a list of medicines (drugs) that should be avoided or used with caution in people affected by a primary mitochondrial disease. Patients suspected of having a primary mitochondrial disease, but in whom the diagnosis has not yet been confirmed by doctors, may also consult this list. The list was compiled by a group of experts in mitochondrial disease (doctors, pharmacists and scientists), after careful consideration and consultation.

What is a primary mitochondrial disease?

This is a genetic disorder that affects the function of the mitochondria. Mitochondria are tiny power stations present inside our cells and are responsible for making the energy that powers everything that happens inside our bodies.

How is a primary mitochondrial disease diagnosed?

Primary mitochondrial disease is diagnosed by doctors after a series of tests that may include blood and urine tests, brain scan (MRI), muscle biopsy and, most importantly, genetic testing. It is not always possible to identify the genetic cause in every affected individual, and in these cases a clinical diagnosis of “probable mitochondrial disease” may be made.

How is a primary mitochondrial disease treated?

Currently, there are no cures for most mitochondrial diseases. This means that supportive treatments and medicines are extremely important. These include medicines for treating seizures (anticonvulsants), antibiotics for treating bacterial infections, medicine for managing pain, controlling fevers, treating diabetes and heart disease, and for administering general anaesthesia safely when an operation is needed.

Why did the list of drugs to be avoided need updating?

We saw that the existing list of medicines thought to be unsuitable (contraindicated) in patients with mitochondrial disease was very long and contained drugs that we felt could be useful in treating patients with mitochondrial disease. Therefore, we thought it was important that each medicine on the list was reviewed and updated with the latest clinical and scientific evidence.

What was the process by which the list was reviewed and updated?

Our group consisted of sixteen doctors, pharmacists and scientists and each was assigned a group of drugs to evaluate. These experts then spent 2 months researching the evidence for whether the medicine was harmful for mitochondrial disease patients for each of the drugs assigned to them. The group then met for a two day workshop to discuss the evidence for > 50 drugs and decide whether or not they could safely be used in mitochondrial disease. This process of evaluating evidence is known as a Delphi workshop, and is widely accepted as a valid scientific method.

What were the conclusions of the workshop?

After a thorough review of the evidence, we concluded that most drugs on the previous list could be used safely in people affected by primary mitochondrial disease. The drugs studied and considered safe to use are listed in Table 1. The drugs considered dangerous, or to be used with caution are listed in Table 2. The drug valproic acid (an antiepileptic drug also known as sodium valproate or Epilim) should not be given to patients with mutations in a gene called POLG and not used in any patient who could have a primary mitochondrial disease until POLG mutation is ruled out. Table 2 also lists drugs that need careful evaluation and situations in which doctors may want to change the way that these medicines are used or prescribed for patients affected by particular types of mitochondrial disease.

Why is this list not the same as other lists of drugs to be avoided in mitochondrial diseases? 

This is a NEW UPDATED VERSION to replace all previous lists of medicines to be avoided in primary mitochondrial disease, based on the latest scientific and clinical evidence.

What should I do with the list?

It is very important that you consult your doctor whenever you are unwell. You may, however, wish to share the list of medicines considered safe to use and the list with points of attention with your doctor(s) and discuss with them what this means for you and your particular type of mitochondrial disease.

For the full scientific publication, click here.