TABLE 4

Most important research areas as rated by patients and caregivers

Research areaTotalNon-reimbursed countriesReimbursed countries
Improving knowledge of AATD, in particular among general practitioners999999
Access to AATD specialised centres979996
Access to reliable, easy to understand information about living with AATD979996
Being able to recognise an exacerbation979897
Targeted screening programmes: COPD and asthma patients969499
Having an action plan for exacerbations and easy access to healthcare during episodes969995
A personalised integrated care plan including therapeutic physical activity959198
Education for physicians on diagnostic techniques, algorithm, interpretation of results959597
Regular communication between healthcare professional team and AATD patient949497
Having access to pulmonary rehabilitation and being taught the techniques and how to use the equipment at home939593
Diagnosis in liver disease patients (children and adults)939295
Educational programmes regarding regional/national resources to diagnose and refer AATD patients939695
Smoking cessation938896
Develop better ways of teaching people to use their medicines e.g. inhalers, oxygen929094
Diagnosis of non-respiratory diseases associated with AATD919095
Develop other aspects of integral care (e.g. physical activity, caregiver support, maintaining work or schooling, nutrition, psychological care, sex life, daily life)918594
Role of pulmonary rehabilitation918995
Availability of organ (lung and/or liver) donation909192
Role of nutrition909193
Use of vaccines to prevent exacerbations909390
Relationship between AATD and other diseases909489
More evidence on effectiveness of augmentation therapy909492
Different evolution of the disease among patients899191
Synthetic AAT production to cover the demand899188
Evidence on the effect of augmentation therapy on reducing exacerbations899290
Development of an international AATD registry899189
Alternatives to IV augmentation therapy (e.g. inhaled augmentation therapy)898994

Data are presented as the % of participants that considered each item to be important or very important. AATD: α1-antitrypsin deficiency; COPD: chronic obstructive pulmonary disease; AAT: α1-antitrypsin; IV: intravenous.