EXPLORE

• Research type

  Research Study

• Full title

  Exploring the EXperience of Patients and Caregivers Living with mitOchondRial Stroke-Like Episodes

• IRAS ID

  314820

• Contact name

  Yi Shiau Ng

• Contact email

  yi.ng@ncl.ac.uk

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  1 years, 0 months, 1 days

• Research summary

  Mitochondria are the ‘batteries’ of the cell that provide the energy we need. When they are faulty, it leads to mitochondrial disease.

  Mitochondrial disease has many causes and symptoms.

  Some people with mitochondrial disease experience Stroke Like Episodes (SLEs). They affect the brain causing symptoms like those seen in a stroke.

  SLEs are serious events. They involve seizures and cause permanent damage to the brain. Those affected have repeated episodes throughout their lives.

  SLEs cause problems with memory, understanding, and speech (we call these ‘cognitive’ processes). They also cause problems with movement and balance. As more SLEs happen, these problems get worse.

  We can treat some SLE symptoms but currently have no way to stop them happening or fix the damage they cause.

  It is important that SLEs are recognised as early as possible when they happen. This allows medication for the seizures to be given and can help reduce damage to the brain.

  Guidelines about recognising and treating SLEs are available for healthcare professionals. However, we do not currently have guidelines for people who experience SLEs and don’t know much about their, or their caregivers, experiences.

  This study is run by the Wellcome Centre for Mitochondrial Research, Newcastle University. It will take place at Newcastle University.

  This aim is to collect information about patient and caregiver experiences of SLEs. This will help us understand what improvements to current treatments are needed, and what patient/caregiver guidelines would be useful. It will also help us design future research studies.
  
  We will invite a small number of people (approx. 20 adults) affected by, or who care for someone with, SLEs, to attend a focus group and/or interview.

  Participants will be asked about their experiences of SLEs including treatments received and how/if they are able to recognise when SLEs are happening.
  Lay summary of study results: Why was the EXPLORE study needed?
  Stroke-like episodes (SLEs) are serious neurological emergencies experienced by a subgroup of patients living with mitochondrial disease, including those with MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes). These events can cause seizures, confusion, headaches, language and visual disturbances, often leading to cognitive decline and loss of independence. Although clinical guidelines exist to support medical management, there is currently no curative treatment.
  While early recognition and treatment are critical to improving outcomes, little is known about how patients and caregivers recognise symptoms, access urgent care, and experience recovery and rehabilitation. Understanding these lived experiences is essential to identify barriers to timely treatment, improve service provision, and inform the design of future research.

  Who designed, led and funded the trial?
  EXPLORE was designed and led by researchers and clinicians at Newcastle University (Translational and Clinical Research Institute) and The Newcastle upon Tyne Hospitals NHS Foundation Trust.
  The study was funded by Newcastle University Faculty of Medical Sciences (FMS) 2022 Tilly Hale Engagement Award.

  What did it investigate?
  This study explored how people with mitochondrial disease and their caregivers experience stroke-like episodes (SLEs).
  We looked at how well symptoms are recognised, how families seek urgent medical help, and what their experiences are during hospital admission. We also explored whether patients receive physical or cognitive rehabilitation afterwards, and how SLEs affect daily life and quality of life in the long term.

  Where and when did the trial take place?
  EXPLORE took place remotely via Microsoft Teams between October 2022 and June 2024. Focus groups and interviews were conducted in person or online, allowing participants from across the UK to take part.

  Who took part and what was involved?
  Participants included:
  - Adults with mitochondrial disease (including MELAS) who had experienced a stroke-like episode (SLE)
  - Caregivers or family members of individuals who had experienced an SLE
  Patients and caregivers took part in either an interview (1-1) or in a small focus group (2–3 participants per group). Both a patient and their caregiver could participate (in different groups. Participation involved discussing their experiences of recognising symptoms, accessing emergency care, hospital admission, rehabilitation, and the longer-term impact of SLEs on daily life.
  Each session provided a supportive environment for participants to share their experiences and perspectives.

  How many people took part?
  A total of 17 people took part in the EXPLORE study. Of these, 12 were caregivers and 5 were patients.
  What were the results?
  The findings showed several consistent themes across interviews and focus groups. Overall, participants described:
  - Difficulty recognising and diagnosing stroke-like episodes, with delays and limited awareness in emergency settings
  - Mixed hospital experiences, often feeling unheard or unsupported, unless specialist teams were involved
  - Limited rehabilitation and follow-up care after discharge.
  - Major long-term impacts on memory, speech, physical health, mental health, work, relationships, and finances.
  Stroke-like episodes were described as life-changing events with lasting impact on both patients and caregivers.

  Was the study useful?
  Yes. The study provided valuable insights into the real-world experiences of patients and caregivers living with stroke-like episodes. It highlighted challenges in recognising symptoms, accessing urgent care, and obtaining appropriate rehabilitation and long-term support.

  Importantly, the findings have already been used to develop a survey to measure caregiver experiences. The results will also help shape future research and improvements in clinical services.

  What happens next?
  It is important that research into patient and caregiver experience continues. The survey developed following EXPLORE will allow experiences to be measured more widely and systematically, helping to identify areas where care can be improved.

  Where can I find out more?
  For further information about the EXPLORE study, please contact the research team at: nuth.mitoresearch@nhs.net The full study protocol is available on a publicly accessible repository: https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fdoi.org%252F10.25405%252Fdata.ncl.21303930%2FNBTI%2FVezDAQ%2FAQ%2Fa5f7babf-c61c-4ed6-a76b-65f4b79f6e18%2F1%2FGKBmILmx_S&data=05%7C02%7Cleicestercentral.rec%40hra.nhs.uk%7Cd5599adef6f941524b0e08de7ea1bd51%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C639087430296712407%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=0eoWUaC2lDYll%2FyqcwR7hwd8APkZx0zZEmDfVrsoR1s%3D&reserved=0

• REC name

  East Midlands - Leicester Central Research Ethics Committee

• REC reference

  22/EM/0166

• Date of REC Opinion

  7 Oct 2022

• REC opinion

  Further Information Favourable Opinion