Cardiopulmonary exercise test to quantify enzyme replacement response in pediatric Pompe disease.

Ronen Bar-YosephHanna MandelGur MainzerMichal GurGalit TalGeorge ShalloufehYazeed Toukan

Published in: Pediatric pulmonology (2018)

CPET is safe for pediatric Pompe patients. ERT may benefit exercise capacity in patients with less advanced disease. Individualized assessment by CPET, 6MWT, and motor function may help ERT adjustment by providing precise quantification of the response to treatment. Additional studies are needed to clarify the benefit of this assessment protocol.

Keyphrases

late onset
replacement therapy
end stage renal disease
high intensity
ejection fraction
newly diagnosed
physical activity
chronic kidney disease
randomized controlled trial
peritoneal dialysis
prognostic factors
young adults