Pompe Registry

 

Expanding knowledge about Pompe disease

The Pompe Registry, sponsored by Sanofi Genzyme, is a global resource dedicated to improving understanding of the clinical variability, and variability in progression, of Pompe disease.
The Pompe Registry works with physicians to compile patient outcome data from routine clinical practice, to provide the medical community with resources to:

  • Encourage collaboration and shared expertise between colleagues
  • Support the clinical practice of evidence-based medicine
  • Develop individualized care plans based on benchmark comparisons of similar patients
  • Facilitate significant research publications

                                                                                       

 

Pompe Registry objectives

The Pompe Registry is an ongoing, observational database that tracks the natural history and outcomes of patients with Pompe disease. All Pompe disease patients are eligible for enrollment irrespective of their treatment status, and all physicians managing patients with Pompe disease are encouraged to participate in the Pompe Registry.

The Pompe Registry provides a repository that enables exchange of information and aggregate data, to facilitate clinical decision making and data reports, as well as serving as a vehicle for collaborative studies.

 

The primary objectives of the Pompe Registry are to:

  • Enhance understanding of the clinical variability and variability in the progression of Pompe disease; aid the identification of key manifestations of the disease and understand their natural history
  • Assist the Pompe disease medical community with development of recommendations for monitoring patients and reporting patient outcomes, to help optimize patient care
  • Characterize and describe the Pompe disease population as a whole
  • Evaluate the long-term effectiveness and safety of available treatment options and support measures, including enzyme replacement therapy (ERT).

Role of participating physicians

Participating physicians are requested to submit participating patients’ data on a regular basis. It is recommended that data be submitted to the Pompe Registry according to the Recommended Schedule of Assessments found in the Pompe Registry Protocol. Pompe Registry staff are available upon request to assist with any data collection questions.

Through the Pompe Registry, the Pompe community has access to important information on a disease that affects a small patient population. Management guidelines and publications from the Pompe Registry may thus contribute to earlier diagnosis and intervention.

 

Benefits of participation

The Pompe Registry has several informative features that offer unique benefits to participating physicians. Physicians' contributions of patient data to the Pompe Registry database benefit all other Pompe Registry participants, as data are pooled to study trends or address specific questions. Participating physicians are encouraged to submit requests for specific information from the database, to facilitate the management of Pompe patients.

The Registry allows physicians to:

  • Request individualized patient reports and informative clinical summaries to help monitor their patient’s disease status
  • Provide a repository for the exchange of aggregate clinical data among physicians to facilitate clinical decision-making
  • Access information on current treatment guidelines and practice patterns

 

Available reports

There are several Pompe Registry-specific reports available to participating physicians. These reports provide important clinical summaries of individual patients, as well as analytical summaries of the overall Registry population. Please visit www.pomperegistry.com to learn more.

Confidentiality

Confidentiality and patient privacy are of primary concern to the Pompe Registry. The Registry complies with applicable national privacy regulations and other state and local laws relating to medical information. All patient and physician information submitted to the Pompe Registry is maintained as confidential:

  • Patients are referenced by Registry ID number only
  • Patients must authorize release of their clinical data to the Registry
  • No site-to-site data comparisons are made

 

Useful links

 

Please find some useful websites for further expanding your knowledge on Pompe disease.

 

International Pompe Association
c/o VSN, Luitenant Generaal van Heutszlaan 6
3743 JN Baarn, The Netherlands
Phone +31(35)5480480
E-mail info@worldpompe.org info@worldpompe.org
www.worldpompe.org

 

More links:

The Pompe Center at the Erasmus Medical Center

National Institute of Neurological Disorders and Stroke

 

 

 

 

 

  GZEMEA.PD.14.11.0313f (1) – March 2019