Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study.

Meng YuanEleni-Rosalina AndrinopoulouMichelle E KruijshaarAglina LikaLaurike HarlaarAns T van der PloegDimitris RizopoulosNadine A M E van der Beek

Published in: Orphanet journal of rare diseases (2020)

Participation, daily-life activities, and the physical component of quality of life of adult Pompe patients are positively correlated to physical outcomes. This work serves as a first step towards assessing how changes over time in physical outcomes are related to changes in PROMs, and to define the minimal change in physical outcomes required to make an important difference for the patient.

Keyphrases

late onset
patient reported outcomes
physical activity
mental health
early onset
end stage renal disease
chronic kidney disease
ejection fraction
newly diagnosed
type diabetes
replacement therapy
young adults
prognostic factors
adipose tissue
risk factors
insulin resistance
metabolic syndrome
skeletal muscle