Ethical considerations in the management of children with mitochondrial disorders

The following information sheet has come directly from Great Ormond Street Hospital to accompany the questionnaire link at the bottom of the page:

Participation Information Sheet for completion of questionnaire: Treatments for mitochondrial disorders and the use of social media: a questionnaire for parents

We would like to invite you to take part in the above research study. Before you decide we would like you to understand why the research is being done and what it will involve. 


We want to explore this area in more detail and welcome your views. Mitochondrial disorders are rare conditions in children that have many different genetic causes. There are many challenges in treatment which often lead to questions about the best way forward with regard to management for a child with a mitochondrial condition. This may include making decisions about experimental or new treatments for which there is limited scientific evidence. Understandably, having a child with a rare condition for which treatment is currently limited may lead parents to look for information on the internet and social media.  

This study aims to understand the range of ethical issues which arise for parents and professionals in caring for children with mitochondrial conditions. The aim is to develop  an ethical framework to support professionals and parents in managing children with these conditions. This will be by inviting parents to complete a questionnaire. Understanding ethical challenges is an important part of the day to day  practice modern medicine.

What’s involved?

A questionnaire asking about your use of social media and innovative and experimental treatments will be available to parents (approximately 100) who are members of the UK mitochondrial support group, The Lily Foundation to complete. The Lily Foundation will send you the link to complete this questionnaire on SurveyMonkey.

If having read the information sheet you would like to take part in this research study, please complete the questionnaire. By completing the questionnaire fully, you will consenting to the data which is non identifiable to be analysed in the study. If you do not wish to take part please do not complete the questionnaire.          

What are the possible benefits of taking part? 

There are no direct benefits for taking part. The study information may help to inform management for a wider group of patients with mitochondrial disorders and help with future management of patients with these conditions.

What are the possible disadvantages and risks of taking part?

Some parents may find answering questions about their child’s medical condition upsetting. If you're affected in this way, please contact The Lily Foundation at [email protected] or telephone 0300 400 1234.

You have the right to withdraw from the study at any point whilst completing the questionnaire. Once completed, it will not be possible to withdraw as no personal details are included that would identify you.

Further supporting information

The study is being carried out with an expert in mitochondrial disorders and ethicists. The study is being carried out during a 3 month consultant sabbatical and will continue for a further 6 months to ensure all data is analysed. The information collected will be anonymous which means no personal data which could identify you are included. Anonomised data will be stored electronically on the Trust and Principal Investigator’s computer so it can be analysed.

The analysis of the data This will be by ‘qualitative’ analysis- looking at the points that parents make about different questions and whether these fall into common themes. Percentages will also be calculated for the replies on the questionnaires, which is ‘quantitative’ analysis. The same questionnaire will be completed by members of the Australian Mitochondrial Support Group, AMDF and the results of the questionnaire combined with those of the UK. Anonomised data will be analysed in collaboration with the Bioethics team at Melbourne Royal Children’s Hospital. Some of the analysis will be carried out with the Bioethics team at Melbourne Royal Children’s Hospital and some of this will be carried out at Great Ormond Street Hospital for Children NHS Foundation Trust.

Anonomised data will be stored for up to 15 years on the computer at Ormond Street Hospital for Children NHS Foundation Trust and may be used for future research. Our procedures for handling, processing, storage and destruction of data are compliant with the Data Protection Act 1998.

The overall results will be discussed at meetings for Bioethics or mitochondrial disorders and a paper written about the results with the aim of publishing this in a medical journal.

Dr Sarah Aylett is the consultant leading the research and has been funded by the GOSH Charity to develop skills in clinical ethics and to undertake this work.

Harm - In the event that something does go wrong you are harmed during the research and this is due to someone’s negligence then you may have grounds for a legal action for compensation – but you may have to pay your legal costs. The normal NHS complaints mechanisms will still be available to you.

Who has reviewed the study? - All research in the NHS is looked at by an independent group of people, called a Research Ethics Committee. This study has been reviewed and given a favourable ethical opinion for conduct in the NHS by Health Research Authority.

Contact for further information - If you would like any further information about this study you could contact:

Name: Dr Sarah Aylett

Designation: Consultant Paediatric Neurologist

Hospital/Department:  Neurosciences, Great Ormond Street Hospital for Children NHS Foundation Trust

Tel: +44 207 405 9200 ext 8356

Email: [email protected]

Thank you for taking the time to read this information sheet.

    Link to Questionnaire 

    The questionnaire will close on Monday 9th April 2018.