200 Chapter 6 can be gained into the aetiology of this exercise intolerance (ClinicalTrials.gov ID: NCT05413876). Among other parameters, maximum oxygen uptake (V’O2 max) is measured and compared to individuals of identical sex, age and weight from a reference database. However, the accuracy, reproducibility and relevance of an incremental CPX test is less than optimal, as it is not representative for exercise during daily activities, in which sudden bouts of maximal activity do not regularly occur. In the FEISTY, an intermittent CPX test is an extension of the ‘block workload test’, in which a step-change from rest to a relatively low constant workload is studied and provides a view in the dynamic properties of gas transport during exercise [48]. This study is an attempt to identify functional outcome parameters, related to the patient’s ability to participate in everyday life. These are needed to be improve in FD research, since current outcome parameters used to assess for example the effectiveness of ERT, such as cardiac mass, do not reflect the patients ability to participate in daily life. In the FEISTY, we will evaluate exercise capacity using a new CPX protocol and evaluate if the protocol can be used to evaluate existing ERT and future FD treatments, with a better relation to functional outcome than currently used parameters.
RkJQdWJsaXNoZXIy MTk4NDMw