Patients and healthcare professionals identify research priorities for Alpha-1 antitrypsin deficiency

A summary of research published in ERJ Open Research

A number of key priorities have been identified by people with Alpha-1 antitrypsin deficiency and healthcare professionals, to help guide future research in the field.

Alpha-1 is a rare condition, and it is important for international research teams to work together to gather enough information from people living with the condition to help us understand more about it. This study aimed to guide this future research by looking at the key issues that matter to patients and healthcare professionals.

What did the study look at?

Two surveys were shared between July 2018 and December 2019 to collect thoughts on research priorities. 93 healthcare professionals and 438 patients completed the surveys, which asked about a range of topics including research priorities and barriers to treatments for Alpha-1. The surveys were put together by the European Alpha-1 Research Collaboration (EARCO).

What do the results show?

The main research and management priorities identified by patients and healthcare professionals included:

• understanding more about the natural history of the condition,
• improving information for physicians and general practitioners to help improve early recognition of the condition,
• access to specialised centres, and
• personalising treatment and having equal access to existing therapies. In countries where a specific treatment for Alpha-1, known as augmentation therapy, was not paid for by the state, access to this was listed as the most relevant area.

Why is this important?

Rare conditions, such as Alpha-1, face key hurdles when it comes to research. The low numbers of people with the condition make large studies difficult and there is limited funding for research into rare conditions. It is important that there is collaboration around the world to improve our knowledge and improve care for people living with the condition.

This study is an important step towards directing this research, by collecting together the areas that are considered most relevant by people living with the condition and by healthcare professionals working in the field.

Read the original research paper

Title: Research Priorities in alpha-1 antitrypsin deficiency: Results of a patients’ and healthcare providers’ international survey from the EARCO Clinical Research Collaboration

https://openres.ersjournals.com/content/erjor/early/2020/10/15/23120541.00523-2020.full.pdf