Amyotrophic Lateral Sclerosis (ALS) is a rare disease characterized by a progressive and irreversible degeneration of central (central nervous system) and peripheral (bulb and spinal cord) motor neurons. From a pathophysiological point of view, the mechanisms explaining central and peripheral neurodegeneration in ALS are still not fully understood. Preventing this neuronal death is a key challenge to propose an effective treatment and thus slow down the progression of the disease. ALS can be characterized by different grading and classification systems used at the time of initial diagnosis and/or during follow-up, with some being predominantly used in clinical trials.
In this context, the objective of this project is to establish expert consensus to:
- Identify the specific issues in the ALS patient journey ;
- Define classification criteria, disease progression criteria and measurement tools used in Central Eastern European (CEE) daily practice ;
- Identify the current ALS management and treatment objectives in CEE ;
- Define the current medical needs for HCPs in ALS management.
To achieve this, you will access an online questionnaire comprising about thirty questions. The time required to complete it is a maximum of twenty minutes. This survey requires no preparatory work, so there is no need to search for precise answers to the questions in your patients' records or databases. We would like to know your overall practices rather than exhaustively patient by patient.
Data collection follows the Delphi principle1, based on your expertise in real-life ALS patient management. The Delphi methodology is iterative, meaning that if you agree to participate in phase 1, you will have to participate in a second round of questionnaire response, addressing specific points requiring clarification or comments from you, in May 2024. In this context, the aggregated responses from the first round will be provided to you.
There is no right or wrong answer; the only answer we seek is the one that most accurately reflects your real clinical practice and your reflection on the disease.
This survey requires no preparatory work, so there is no need to search for precise answers to the questions in your patients' records or databases. We would like to know your overall practices rather than exhaustively patient by patient.
All your responses will be strictly confidential, anonymous, and analyzed in aggregate only.
We appreciate your interest and participation in this project,
Dr Magdalena KUZMA-KOZAKIEWICZ, Poland
Prof. Eva VLČKOVÁ, Czech Rep.
Prof. Monika KOPRUŠAKOVA TURČANOVA, Slovakia
Dr Mirea HANČEVIĆ, Croatia
Prof. Blaž KORITNIK, Slovenia
Dr Judit MOLNAR, Hungary
1. Niederberger, M. & Spranger, J. Delphi Technique in Health Sciences: A Map. Front Public Health 8, 457 (2020).