Descriptive Analysis of the Expert Opinion Regarding the Initiation of Augmentation Therapy in Alpha-1-Antitrypsin-Deficiency

Descriptive Analysis of the Expert Opinion Regarding the Initiation of Augmentation Therapy in Alpha-1-Antitrypsin-Deficiency - NA

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00013943

Enrollment

Registered

2018-02-05

Start date

2018-04-13

Completion date

Unknown

Last updated

2025-04-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

E88.0 J44.8

Interventions

Group 1: The study Population consists of 50 expertes for AATD (Alpha-1-antitrypsin deficiency) that will evaluate 500 virtual clinical cases (10 cases per expert) and make the decision to start augme

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) 5-year work experience in the field of AATD (2) 5 treated AATD-patients/year

Exclusion criteria

Exclusion criteria: none

Design outcomes

Primary

Measure	Time frame
Descriptive analysis of all those factors which would lead to an augmentation therapy. Due to the lack of a prespecified questionnaire, we created one. After a phone call, experts in the field of AATD will get the questionnaire and complete it once. No follow-up is planned. The Analyse factors include AAT serum level, genotype, FEV1, diffusion capacity, FEV1-decline, diffusion capacity decline, age, lung function decline, CT-scan, smoking history, symptoms and exacerbations.	—

Measure

Time frame

Descriptive analysis of all those factors which would lead to an augmentation therapy. Due to the lack of a prespecified questionnaire, we created one. After a phone call, experts in the field of AATD will get the questionnaire and complete it once. No follow-up is planned. The Analyse factors include AAT serum level, genotype, FEV1, diffusion capacity, FEV1-decline, diffusion capacity decline, age, lung function decline, CT-scan, smoking history, symptoms and exacerbations.

—

Secondary

Measure	Time frame
Individual factors of the experts (Age, gender, country of origin). These will be taken from the questionnaire.	—

Countries

Belgium, Croatia, Czechia, France, Germany, Ireland, Italy, Netherlands, Poland, Portugal, Romania, Spain, Switzerland, Turkey, United Kingdom

Contacts

Public ContactTimm Greulich

Philipps-Unversität Marburg FB Humanmedizin, Innere Medizin, Pneumologie

alpha1@med.uni-marburg.de+49 (0)6421-5866451

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026