Currently, PCD management guidelines are mostly based on research conducted on other respiratory conditions (such as cystic fibrosis) alongside expert opinion. To improve understanding of the condition and improve the outcomes and the quality of life for people with PCD, it is of the utmost importance that we design and carry out research with PCD patients.
All centres are involved in research and will offer their patients the chance to take part in clinical trials. You may also receive information about research via PCD Support UK. These may be local, national, or international studies. This information aims to answer your questions about research and to help you decide whether you want to be involved.
PCD research aims to: Research undertaken at your local PCD centre hopes to not only improve your care but also improve the care throughout the country and further afield. Without research there would be no new medicines or tests, improved treatments, or better ways of treating people with PCD.
A European Respiratory Taskforce for PCD has recently created an evidence-based guideline for PCD diagnostics. This will improve the testing for PCD internationally; ensuring centres all around the world are using the best evidence to inform their decisions for diagnosing PCD. This may be further improved in the future as new techniques for diagnosis become available.
Improve our understanding of the causes and development PCD
Improve diagnostic techniques and reduce time to diagnosis/exclusion of PCD
Help prevent and reduce severity and frequency that PCD patients become ill with chest or ENT problems
Look for the best ways to treat infections and improve the quality of life for people living with PCD
Develop new treatments and medicines
What is research?
There are four main types of research you may be invited to participate in:
Most children in England have been invited to share their anonymised data on a local and/or international database. (PCD and the PCD Registry are two international databases that record the diagnostic history and follow up data for adults and children with PCD). As this information is collected, we will be able to learn more about PCD as well as learning about the relationship between the genetics and the characteristics of the condition.
This type of research is usually conducted through a series of interviews with patients and families to find out how their condition affects them individually so we can have a better idea of how to best care for PCD patients.
Cells are basic units that make up the human body to perform all the functions we need. For example, there are cells that line the airways of our lungs that produce mucus and other cells that grow cilia. Together they form the basis of mucociliary clearance to keep our lungs clean. Human cells and tissue are vital for medical research. They can be collected during many routine procedures, including:
blood tests
operations
biopsies (tissue samples taken for examination e.g. nasal brushings)
Researchers can use these cells and tissues to learn about how PCD starts and develops, and how new drugs and tests might work before clinical trials begin.
New treatments or medicines need to be tested to see if they are of benefit to patients. The treatments PCD patients undertake are known to be safe but rarely have their effectiveness tested with PCD patients. Many of the treatments have been tested on cystic fibrosis patients or patients with other respiratory conditions but have not been demonstrated to be effective in patients with PCD. An example of a clinical trial is the recent study looking at the use of the antibiotic Azithromycin in patients with PCD. This was part of the BESTCILIA programme.
Frequently asked questions about research
All research participation is voluntary and may be withdrawn at any time. Each study will have its own requirements. It is important you read the patient information sheet and ask any questions you may have before deciding whether you/your child wants to take part. You may wish to consider:
Number of visits
Treatment changes
Time it will take you
Tests you will be required to undertake
If you are happy to get involved in a research study, you will need to give informed consent to show that you understand what is being asked of you and to confirm you agree to take part. You should not give your consent if you are unclear about any aspect of the research. Not all research requires in-person participation. Some research studies such as the COVID-PCD study (see section “Advertisements for Research”) can be done remotely and do not require you to visit the hospital to participate.
If you take part in a research study, you may have more contact with your PCD team which may improve your understanding of your condition and its management. In many cases, the research may not help you directly, but it may provide vital information that will help people with PCD in the future. The aims of the study will be explained to you before you give your consent.
To eliminate any risks to patients, all research must be approved by a Research Ethics Committee – a panel of health and social care professionals and members of the public with knowledge of, and/or an interest in research. Each study will be different, and any known risks will be explained clearly to you.
Each study will have a patient information sheet with a named person for you to contact with any concerns. You can also contact your local Patient Advice and Liaison Service (PALS) with any concerns you may have.
The PCD National Service works together within the UK but also collaborates internationally as part of BEAT-PCD funded by the European Respiratory Society. A network coordinating research from basic science to clinical care for PCD.
The UK Cilia Network brings together research and clinicians working on structure and function of cilia.
You can also help PCD research by volunteering to be a patient or public representative. This is an important role that will help ensure research is patient-centred and that patients and the public are involved in the design of research studies. Representatives will be able to offer a unique perspective and have a personal understanding that doctors, nurses, and researchers may lack. If this is something you feel you may be able to help with, please complete the PCD Patient and Public Involvement Registration Form and email it to [email protected].
For more information about ear, nose and throat management please look at the website for ENT UK
Simon Whalley and Chris McManus’ study into ‘Living with PCD’ published in October 2006
The UK Pub Med Central website aims to make medical articles freely available to researchers. If you follow the link and enter “Primary Ciliary Dyskinesia” into the “search article” box it finds around 67 articles on the subject which can be viewed, downloaded etc.
