COHERE is an abbreviation of Core Outcome Set for Head, nEck and REspiratory disease in Mucopolysaccharidosis Type II.
The study aims to standardise the way outcomes are reported in research examining head and neck and respiratory issues in individuals with Mucopolysaccharidosis Type II. Researchers aim to establish outcomes for studies in two patient: those aged up to 11 years old and those aged 12 to 25-years-old.
Study type: Electronic survey (in two parts, approximately 18 months apart)
Funder: Shire International GmbH, a Takeda company, under IIR-GBR-000133
Core Outcome Sets
A Core Outcome Set (COS) is an agreed minimum set of outcomes that should be measured and reported in all research in a specific condition. Use of a COS would not limit the outcomes that could be recorded in a study, it would simply require that the COS was used as a minimum set of outcomes.
The below video is a plain language summary produced by the COMET Initiative, which explains what outcomes are and the problems with using different outcomes in research.
The importance of Core Outcome Sets
Ensuring that different trials report their outcomes in the same way enables researchers, doctors and patients to compare the effects of different treatments with greater ease and accuracy.
We also know from initiatives to standardise the reporting of outcomes in other fields, that patients can have significantly different opinions as to which outcomes are important to them when compared to doctors. That is why in the COHERE study the same weight will be given to views of patients, parents, clinicians and scientists.
How do we aim to achieve our goal?
Researchers working on the COHERE study will develop Core Outcome Set for head, neck and respiratory disease in MPS II for ages up to 11-years-old and 12 to 25-years-old. Researchers will use an electronic survey to determine what are the most important outcome measures for head, neck and respiratory disease in MPS II. This survey will allow individuals (patients, parents, doctors, nurses and scientists) to rank outcomes according to their importance and will allow participants to reflect on the opinions of others, before reaching a final decision on what are the key outcomes that should be measured in future studies.
Who can take part?
- Indivduals with MPS II, aged 12-25 years old
- Parents/carers of individuals with MPS II
- Clinicians responsible for direct care and management of at least two individuals with MPS Type II in the last 12 months
- Scientists who have worked in the field of MPS II within the last five years.
How can I take part?
Please download one of the below participant information sheets which is best suited to you before deciding to take part. Clicking links will open Word documents.
- COHERE Participant Information Sheet for clinicians and scientists
- COHERE Participant Information Sheet for parents and guardians
- COHERE Patient Information Sheet for patients over 16 years old
- COHERE Patient Information Sheet for 12 to 16-years-olds
If you are happy to proceed, please continue to complete one or more of the below surveys which are best suited to you. Clicking links will open online surveys.
- COS for up to 11-year-olds – this survey is for parents, clinicians and scientists
- COS for 12 to 25 year olds – this survey is for patients [16-25 years old], parents, clinicians and scientists
- COS for 12 to 25-year-olds – this survey is for patients aged 12 to 15, who need their parent to register
More information about this study
Please get in touch via: COS4MPS2@mft.nhs.uk
This study is supported by:
More information about MPS
The mucopolysaccharidoses (MPS) are a family of inherited metabolic disorders, caused by specific lysosomal enzyme deficiencies, with resultant accumulation of partially degraded glycosaminoglycans (GAG) within tissues. The ubiquitous nature of GAG within the body means that a number of different organ systems can be affected. Eleven distinct types of MPS disorders have been classified, according to the particular enzyme that is deficient.