The PROspective German NOn-CF BronchiectaSIS Patient Registry
Bronchiectasis is typically associated with cystic fibrosis (CF). However, it is a heterogeneous disease complex with a variety of possible non-CF causes. As a result, there are significant diagnostic and therapeutic challenges. The chronic and progressive course of the disease and its accompanying clinical symptoms impose an extraordinary burden on the patient. Everyday functionality and social participation become increasingly limited.
Bronchiectasis is characterised by persistent dilation of the bronchi and bronchioles. Its pathophysiology is not fully understood. A widely accepted model describes a “vicious cycle” of inflammation, airway damage, mucus retention, and increased bacterial colonisation of the bronchial mucosa. The dilated bronchi are likely the common endpoint of various heterogeneous bronchial diseases that predispose to chronic airway inflammation. The etiology of non-CF bronchiectasis ranges from structural and chronically obstructive damage to local and systemic infections and immunosuppression.
Unlike genetically determined CF bronchiectasis, whose causal genetic defect is known, non-CF bronchiectasis is not hereditary. Therefore, the treatment of bronchiectasis must be tailored to its etiology and specific characteristics. This aspect is a focus of the PROGNOSIS registry study.
The current data on non-CF bronchiectasis is limited. While the diagnosis has become more frequent over the last century, knowledge about the most commonly used therapies remains scarce, reflecting a significant lack of epidemiological, basic scientific, and clinical studies. This is evidenced by the fact that at the start of the study in 2015, no pharmacotherapy for this indication was approved. Prescriptions are required to be given “off-label”. Therefore, obtaining more information on the effectiveness and safety of potential therapeutic strategies is crucial for the development of effective treatment approaches and concepts.
With the PROGNOSIS registry study, a national, representative, prospective, observational, and longitudinal database is being established throughout Germany. Currently, more than 1,700 patients at over 25 study centers are enrolled in the non-CF bronchiectasis registry. PROGNOSIS collaborates closely with the European Bronchiectasis Registry EMBARC (The European Multicentre Bronchiectasis Audit and Research Collaboration), which is supported by the European Respiratory Society (ERS) as a Clinical Research Collaboration. The data from PROGNOSIS represent Germany within the European Bronchiectasis Registry EMBARC.
PROGNOSIS started recruiting its first patients in the third quarter of 2015.
The goals of PROGNOSIS include:
- Establishing and developing a nationwide, representative, prospective, observational (non-interventional), and longitudinal patient registry. Adult patients from 25-35 centers across sectors, ranging from pulmonology practices to university hospitals, are recruited into the registry.
- Addressing important epidemiological questions, such as the distribution of etiologies and capturing the current healthcare reality of non-CF bronchiectasis (including quality of life, health economics, and pharmacoepidemiology).
- Establishing a rational framework for the differential diagnosis and prognostic markers of non-CF bronchiectasis.
- Facilitating access to future clinical studies for participating centers.
- Developing a German-language guideline for the diagnosis and management of non-CF bronchiectasis in collaboration with the German Respiratory Society (DGP) e.V.
- Supporting the establishment of a translational research network on non-CF bronchiectasis in Germany and Europe.
- Tracking emerging healthcare services and healthcare costs.
Ongoing since 2015
Adult patients diagnosed with bronchiectasis without evidence of cystic fibrosis are to be included. They will be observed for a minimum period of three years. Biomaterials are currently being collected from 500 patients and stored in a biomaterial bank for further research purposes and made available to researchers.
25 German study centers
Multicenter prospective and longitudinal observational study in Germany
Sputum, serum, EDTA plasma, EDTA whole blood, urine
CAPNETZ Foundation, MHH, BREATH/DZL, EMBARC European Bronchiectasis Registry, Hannover Unified Biobank (HUB), 2mt Software GmbH
The establishment of the PROGNOSIS registry was made possible with the support of Bayer, Grifols, Novartis, Insmed, and Infectopharm. Currently, as part of a cooperative project between DZL and the German Center for Infection Research (DZIF), a biobank is being established to provide biomaterials for addressing the goals of the cooperative project and other scientific projects.
ROLE OF CAPNETZ
At Hannover Medical School (MHH), Non-CF bronchiectasis is of significant importance, and the establishment of the PROGNOSIS registry is primarily initiated and coordinated by medical staff from the MHH. Competent support is provided by the leading actors of the registry in the planning and execution of the registry study.
The DZL partner CAPNETZ Foundation supports the German Non-CF Bronchiectasis Registry PROGNOSIS in various aspects. This includes programming the study database for clinical data collection, submitting study documents to regulatory authorities, recruiting participating centers, data management, and collaboration with the involved study centers within the scientific network. Currently, utilizing the established biomaterial logistics of CAPNETZ Foundation, biomaterial from 500 study patients is collected during study visits to establish a central biomaterial bank.
In early 2023, PROGNOSIS became an associated partner of the German Center for Lung Research (DZL) within BREATH (Biomedical Research in Endstage and Obstructive Lung Disease Hannover), located at MHH. PROGNOSIS receives support in terms of marketing and public relations within this framework.
For further information, please visit the official website of the PROGNOSIS Bronchiectasis Registry: www.bronchiektasen-register.de