We would like to share the latest publication based on the data generated through the IPA/ Erasmus MC Pompe survey.
Together with the International Pompe Association (IPA), the Erasmus University Medical Center (Erasmus MC) based in Rotterdam, Netherlands, has systematically collected data on patients with Pompe disease since 2002. Earlier findings from the survey include the demonstration of a positive effect of ERT on survival. Using data from this survey, it was investigated whether ERT reduces the risk that a patient will need a wheelchair or respiratory support. This is the first study to provide evidence that ERT with alglucosidase alfa reduces the risk that adult patients with Pompe disease will become wheelchair dependent (patients who received ERT had a 64% smaller probability of becoming wheelchair dependent than an untreated patient). Nevertheless, a proportion of treated patients still become wheelchair dependent at some point in their life. Hence, while ERT shows positive clinical effects in adult patients with Pompe disease, it has been concluded that there is still room for improvement. With regard to the risk for starting respiratory support, no differences could be detected. The IPA survey is an open cohort into which patients are continually included. It has been shown to be a good reflection of the clinical spectrum of adult patients with Pompe disease.
Enzyme Replacement Therapy Reduces The Risk For Wheelchair Dependency In Adult Pompe Patients (PDF, 609 KB)
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