The International Pompe Association (IPA) encourages patients to participate in the IPA/Erasmus MC Pompe Survey. The survey is designed to better understand the progression of the disease and its effect on quality of life. Patient-reported data is gathered through paper and/or online questionnaires that have to be filled in by patients once a year.
The IPA/Erasmus MC Pompe Survey (“the Survey”) is a joint international study of the International Pompe Association (IPA), Erasmus MC in Rotterdam (NL) and Genzyme Corporation. It is the continuation of a study conducted since 2002 by the IPA and the Erasmus MC (“Pompe Questionnaire”).
The Survey was designed to gather data about Pompe disease directly from patients through questionnaires with the objective to better understand the progression of disease over time. Understanding the disease allows physicians to anticipate the future needs of their patients and provides a baseline against which investigational therapies can be tested. Another goal is to find out how patients themselves experience the long-term effects of the available treatments and the supporting measures, including enzyme replacement therapy with Myozyme®.
All individuals aged 16 and above, with a confirmed diagnosis of Pompe disease, may participate in the Survey. All patients who participated in the earlier version of the Survey will be asked to participate again, afterwards other patients will be invited to join.
You will be asked to fill out an extensive questionnaire regarding your medical history and current physical condition, as well as a number of standardized questionnaires about the effect of the disease on your activities and quality of life. You will be asked which language you prefer for filling out the questionnaire, and whether you prefer a paper version or an online version through a secured internet page (preferred) to fill out and return/submit the questionnaire. Currently the Survey is available in Dutch, German, English and French. In 2010 Spanish and Italian translations will become available, and a survey for children under 16 years of age will be developed.
As a participant in the Survey, you will be informed regularly of the results on a group level by the Erasmus MC and/or the IPA. This will be done in various ways, for instance through a newsletter, through the website of the International Pompe Association (www.worldpompe.org) and through presentations at patient meetings.
For questions about the Survey please contact a representative of your national patient organization or any other member of the IPA Board, or Dr. Deniz Güngör at Erasmus MC.
For more detailed information, please click here (PDF, 119 kB).