David Liuzzo via Wikimedia Commons (CC 2.0)
OUR CURRENT PROJECT
DUE TO THE RARITY OF THE DISEASE, ONLY A MULTICENTER APPROACH IS FEASIBLE
10 different clinics participate in our project.
How should future therapies and clinical trials be designed?
Our current study comprehensively surveys MSA- and Parkinson patients on how clinical trials and disease-modifying therapies for these diseases should be designed. In our questionnaire we are asking which side effects are particularly severe and which would be bearable.
Moreover, we are assessing the willingness of patients to take therapy-associated risks. We are specifically investigating whether the willingness to take risks correlates with the disease burden (assessed using the health-associated quality of life). Furthermore, our goal is to find out more about the general attitude of patients towards clinical trials and possible barriers that prevents them from participating.
How do MSA and M. Parkinson patients dream?
We are investigating potentially burdensome, but so far not assessed, symptoms linked to the contents of the patients' dreams. For that purpose, we use a 'dream questionnaire'. The majority of MSA patients suffer from a REM-Sleep-Behaviour-Disorder. Moreover, 50% of all Parkinson patients suffer from this disorder.
The disorder is characterised by a loss of muscle tension during the REM-sleep. This leads to the patients living out their often vivid and sometimes even violent dreams. We hypothesise that these brutal and possibly disturbing dreams increase the disease burden of MSA and M. Parkinson patients.
Elicitation of clinical data of MSA and Parkinson patients.
For the analysis we need a comprehensive picture of the health state of our participants. We are collecting some data retrospectively from the patients' medical records. Among other things, we are checking for additional diseases, such as terminal cancer, which could potentially increase the suffering and the patients' willingness to take risks. Moreover, it is invaluable for us to have information about the cognitive state of our patients and to know in which stage of the disease they are.
Patients in a later stage of the disease, which may already be tied to a wheelchair, suffer more than patients which aren't physically impaired and are still able to take care of themselves. Moreover, we are collecting information about symptoms specific to MSA and Parkinson to improve the characterisation of the disease burden, such as incontinence, impotence, orthostatic hypotension, REM sleep behavior disorder, slowing movement (bradykinesia), tremor and stiffness (rigor).
WHAT DO WE WANT TO ACCOMPLISH WITH OUR RESEARCH?
YAMSA e. V. PRESENTED THEIR PROJECT AT THE ALPHA-SYNUCLEIN-CONFERENCE IN ATHENS 2017
OUR FUTURE PROJECTS
In accordance to our statues, we want to support and carry out additional multi-central MSA research projects - our current project sets the foundation and the starting point of this. With our current study we are already supporting the clincis at the participating hospitals with the creation of a list of patients
that show general interest in participating in future studies concerning MSA. This could lead to a possible follow-up project: A nationwide patient registry of German-speaking MSA patients.
We have started with the recruitment of MSA patients for our project in January 2019 in Marburg, Dresden and Beelitz. All other clinics will start by April 2019 as well. We are expecting first results in December 2019.
We presented part of our project at the "6. International MSA Congress" in New York City in March 2018:
The abstract presented there was published in Clinical Autonomic Research (2018) 28:142.
Tabea Barthel*, Alexander Maximilian Bernhardt*, Lioba Kahmann*, Emre Kocakavuk*, Eliana Nachman*, Marc Oeller*, Kevin Peikert*, Malte Roderigo*, Andreas Rossmann*, Jeremy Schmidt*, Isabel Weigelt*, Lea Olivia Wilhelm*, Armin Giese, Wolfgang Hermann Oertel (03/2018): Assessing the Willingness of Multiple System Atrophy Patients to Take Risks in Clinical Trials. 6th International MSA Congress, New York City, 1. – 2. März 2018.
* contributed equally