Assessing the Burden of Illness of Refractory Myasthenia Gravis_v0.1
Research type
Research Study
Full title
Assessing the Burden of Illness (BOI) of Refractory Myasthenia Gravis in England
IRAS ID
225861
Contact name
Sophie Graham
Contact email
Sponsor organisation
Alexion Pharmaceuticals
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
0 years, 6 months, 0 days
Research summary
What is the humanistic burden of illness in patients with Generalised Myasthenia Gravis in England?
Myasthenia Gravis (MG) is a rare disease that causes weakness of the muscles that control the limbs, face, mouth, throat and chest. The disease typically affects women in their forties and men in their sixties. Patients who are treated are able to lead a near normal quality of life and are able to carry out daily activities. However, 10-20% of patients do not respond to their treatment and therefore experience continuous muscle weakness, and have difficulty swallowing, chewing, breathing and walking. They experience symptoms such as eyelid droop, slurred speech, weak arms and legs, and shortness of breath. These patients are known to have ‘refractory’ myasthenia gravis. At present there are no available treatments for this group of patients and therefore in order to inform new treatments the quality of life of these patients needs to be assessed. Quality of life in patients with ‘refractory’ myasthenia gravis has never knowingly been previously assessed. This study therefore aims to compare the quality of life of patients that are ‘refractory’ with myasthenia patients that are not refractory by using a patient questionnaire. The questionnaires used to assess quality of life in these patients are the MG-quality of life and MG-activities of daily living which have been used and trialed before many times in patients with myasthenia gravis. The questionnaire will take around 15 minutes to complete and patients will be able to complete them in their home. They will also not be given any interventions and their normal treatment will not be affected. Patients will be asked to be on the study by their GP and can withdraw from the study at any time. Patient questionnaires will not be identifiable and all patient information will be kept confidential at all times.
REC name
East of Scotland Research Ethics Service REC 1
REC reference
17/ES/0137
Date of REC Opinion
27 Oct 2017
REC opinion
Further Information Favourable Opinion