Article
German nation-wide registry-based neurofibromatosis type 2 service (NF2-Netzwerk)
Etablierung eines nationalen Register-basierten Netzwerkes für Neurofibromatose Typ 2 (NF2-Netzwerk)
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Authors
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Anna Cecilia Lawson McLean
- Helios Klinikum Erfurt, Klinik für Neurochirurgie, Erfurt, Deutschland
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Said Farschtschi
- University Medical Center Hamburg-Eppendorf, Hamburg, Deutschland
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Isabel Gugel
- University Hospital Tübingen, Tübingen, Deutschland
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Martin Ulrich Schuhmann
- University Hospital Tübingen, Tübingen, Deutschland
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Steffen Rosahl
- Helios Klinikum Erfurt, Klinik für Neurochirurgie, Erfurt, Deutschland
| Published: | June 4, 2021 |
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Outline
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Objective: Neurofibromatosis type 2 (NF2) is a rare autosomal-dominant genetic disease with a prevalence of 1:60,000. Patients typically develop various tumours in the central and peripheral nervous systems and other disorders leading to a wide range of deficits. Due to the complexity of the disease, multidisciplinary teams in a specialised centers are required for optimal treatment of these patients. Despite the existence of such specialized centers, there is no systematic nationally coordinated approach to the treatment of NF2 in Germany.
Methods: Analogous to the UK national NF2 service delivery, we plan to implement a coordinated network of specialized NF2 centers [RS1] in Germany. These specialized centers will share an online-accessible registry that will capture prospective data from all patients treated at those centers and associated health care facilities. In addition to clinical data, patient reported outcome measures (PROMs), including quality of life and psychological questionnaires, will be collected digitally from patients via smart app. Data storage and handling is based strictly regulated according to current EU legislation and GCP standards.
Results: The aim of this approach is to improve the quality of patient care, resulting in higher and longer lasting quality of life as well as lower morbidity and mortality. Patient data will be accessible online for all participating medical professionals resulting in lower workload, a more complete medical profile of patients, timely notification for diagnostic follow-up, interventions and rehabilitation, and avoidance of unnecessary and duplicated investigations. Additionally, prospective data from a large multicenter cohort will be collected, which will improve the basis for high-quality research studies. The layout of the registry with the first 100 patients entered will be presented.
Conclusion: A nationally-coordinated approach of NF2 care in Germany is expected to improve the treatment and patient-based outcomes in this rare disease.
