Author
Prof. Dr. Dr. Robert Bals, Alpha1 Germany eV, as published in Alpha1 Journal 2/2022.
Improving quality of life is an important goal in the treatment of chronic diseases. Questionnaires are often used to assess this. This allows for the measurement of various aspects of quality of life.
Developing such questionnaires is a complex process to ensure that they accurately reflect quality of life and, for example, capture different manifestations of a disease. It is also important to determine whether a treatment leads to a change in quality of life. These questionnaires are called "patient-reported outcome measures" (PROMs), meaning treatment successes are reported by the patients themselves and not measured by a medical procedure. Such PROMs are often used in clinical trials for new medications.
Quality-of-life questionnaires have been developed for common diseases, such as COPD and liver diseases. These questionnaires have often been used in studies of alpha-1 antitrypsin deficiency (AATD), without a thorough investigation into their suitability for this condition. Developing quality-of-life questionnaires specifically for AATD would be an important goal to accurately measure quality of life in this condition. This would also facilitate the conduct of studies to develop new therapies.
We are currently starting a project to develop quality-of-life questionnaires (PROMs) for the AATM. These include questions about general quality of life as well as liver or lung disease.
Before practical implementation, we conducted a literature review of existing questionnaires to avoid duplication and to identify the current state of research. To the questions selected from this review, we added several questions that seemed important to the participants. The first phase of the study involves participants answering questions online. The study has been approved by the Ethics Committee of the Saarland Medical Association and complies with data protection regulations. In a second phase, we administered the questionnaires during outpatient visits to examine their practical application in everyday practice. We expect that by the end of the project, a validated AATM quality of life questionnaire will be available for widespread use.
Alpha1 Germany e. V., Saarland University, RWTH Aachen University, the University of Marburg, and the University Hospital of Regensburg are working closely together on this project. CSL Behring is providing financial support; no personal data will be shared with the company.
We invite those affected to participate in various ways.
We sent out invitation letters at the end of October. Many of you have already participated in this first part of the study and are therefore contributing to the success of the project. We would like to take this opportunity to thank you. For questions and further information, please contact the study center in Homburg (Prof. Robert Bals, Tel. +49 6841 16 15051, alpha1@uks.eu or Alpha1 Germany).
Caption for image: The aim of the project is to develop a questionnaire that measures the quality of life of individuals with alpha-1 antitrypsin deficiency. The institutions mentioned are working closely together on this project, and the data will be analyzed jointly.
Prof. Dr. Dr. Robert Bals
