COHERE

• Research type

  Research Study

• Full title

  Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II

• IRAS ID

  261752

• Contact name

  Iain Alexander Bruce

• Contact email

  iain.bruce@manchester.ac.uk

• Sponsor organisation

  Manchester NHS Foundation Trust

• Duration of Study in the UK

  1 years, 0 months, 1 days

• Research summary

  The Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by specific enzyme deficiencies which result in accumulation of partially degraded glycosaminoglycans (GAGs) within various tissues. As GAGs are common in the body a number of different organ systems can be affected.
  Involvement of the upper and lower respiratory tract in MPS Type II results in significant airway compromise, with progressive airway obstruction being responsible for a significant proportion of the morbidity and mortality associated with this condition. Hearing loss is a universal finding in MPS, with a third of patients suffering with severe-profound hearing loss.
  There is an unmet need for strong clinical evidence to guide treatment of head, neck and respiratory disease in MPS disorders.A Core Outcome Set (COS) describes the minimum outcome data that should be measured in a clinical study for a particular condition. The lack of an agreed core outcome set for MPS II in general, and specifically head, neck and respiratory disease, makes comparison between studies difficult. In addition, purchasers and regulatory bodies are likely to prefer evidence from clinical studies that adhere to an agreed set of outcome measures. There is also a lack of information detailing patient and parent perspectives on the MPS disorders. The ideal COS for head, neck and respiratory disease associated with MPS II would combine both patient/parent and clinician opinion and could be used in the design of all subsequent clinical studies. Additionally, the tendency to only report those findings that researchers consider to be significant or positive can lead to outcome reporting bias (ORB). The development of a COS can help to negate the effect of ORB in future study designs and limit variability of chosen outcomes in studies investigating different interventions in the same organ system.
  

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  20/EE/0017

• Date of REC Opinion

  19 Mar 2020

• REC opinion

  Further Information Favourable Opinion