GSD/GLA registry Freiburg - Register for Gorham-Stout-Disease (GSD) and generalised lymphatic anomalies (GLA) -
Organizational Data
- DRKS-ID:
- DRKS00007182
- Recruitment Status:
- Recruiting complete, study complete
- Date of registration in DRKS:
- 2015-06-17
- Last update in DRKS:
- 2023-07-28
- Registration type:
- Retrospective
Acronym/abbreviation of the study
GSD/GLA-Registry
URL of the study
No Entry
Brief summary in lay language
The GLA is a very rare disease which is marked by an increase of lymphatic vessels. Besides, bones, internal organs, soft part fabrics and the skin can be concerned. This disease appears predominantly with children and young adults. Treatments can be carried out according to propagation of the lymphatic vessels by drugs and if necessary also by radiotherapy. At the moment there is still no academically secure knowledge about the origin of the diesease and the suitable risk factors which favour the origin of the GLA. The GSD is an extremely rare disease with which bones are dissolved and are replaced with lymphatic tissue. The messenger's material Interleukin-6 seems to be involved in these disease events decisively. Bone-diminishing cells and the appearance of distensions in the area of the blood vessels and lymphatic vessels seem to be significant with these illness events. At the moment there is still no generally approved therapy procedure. Beside surgical interventions, radiotherapy and chemotherapy as well as Bisphosphonate and interferon alpha 2 are applied. GSD as well as GLA are seldom appearing diseases with partly serious complications. The aim of the register is the systematic and central registration of the illness course of the affected patients. By the collection of this information and scientific evaluation we get more knowledge about these diseases. The strategies of treatment can be thereby improved in the longer term for the affected patients, according to the actual knowledge.
Brief summary in scientific language
The Gorham-Stout-Disease (GSD) and the generalised lymphatic anomalies (GLA) are rare diseases. Both appear predominantly with children and young adults and one can separate them hardly of each other. The GLA is marked by a vague increase of lymphatic vessels, among the rest, bones, internal organs, soft tissue and skin can be concerned. It also often comes to hemorrhages. With the GSD it comes above all for dissolving of osseous substance and the skeletal segments are replaced by lymphatic vessels. It also comes to the infestation of soft tissue, as well as internal organs like the spleen, the liver and the lung. Thus it can come to serious complications like osseous breaks or lung failure. Nevertheless, the illness course as well as the development of the diseases varies a lot individually. Different attempts of treatment with drugs, radio therapy and also surgical interventions are undertaken. There is not a standardised procedure of treatment, nevertheless, up to now. Also the causes of the diseases are still widely unknown. With the GSD the messenger's material Interleukin-6 seems to be involved in the illness events decisively. The aim of the register is the systematic registration of patients with GSD/GLA with the help of a registration curve. By the accumulated information and scientific evaluation it is possible to get to know the illnesses better and to optimise in the longer term the strategies of treatment according to the actual level of knowledge. Besides the care of the affected patients and the consultation of the treating doctors can be improved thus. It may also answer scientific questions, as for example the aetiology of the diseases.
Health condition or problem studied
- ICD10:
- M89.5 - Osteolysis
- ICD10:
- I89 - Other noninfective disorders of lymphatic vessels and lymph nodes
- ICD10:
- D18.1 - Lymphangioma, any site
- Free text:
- Gorham-Stout-Disease (GSD)
- Free text:
- Generalized Lymphatic Anomalies (GLA)
- Healthy volunteers:
- No
Interventions, Observational Groups
- Arm 1:
- The aim of the register is the systematic and central registration of the illness course of patients with Gorham-Stout-Disease and generalised lymphatic anomalies. By the collection of this information and the scientific evaluationwe win more knowledge about these diseases. The strategies of treatment can be thereby improved in the longer term for the affected patients, according to the actual knowledge.
Endpoints
- Primary outcome:
- The aim of the registry is the systematic and central registration of the illness course of the patients with Gorham-Stout-Disease (GSD) and generalised lymphatic anomalies (GLA) with the help of a registration curve.
- Secondary outcome:
- By the accumulated information and scientific evaluation it is possible to get to know the diseases better and to optimise in the longer term the strategies of treatment according to the actual level of knowledge. Besides the care of the affected patients and the consultation of the treating doctors can be improved thus. It may also answer scientific questions, as for example the aetiology of the diseases.
Study Design
- Purpose:
- Prognosis
- Retrospective/prospective:
- No Entry
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- No Entry
- Study type non-interventional:
- Epidemiological study
Recruitment
- Recruitment Status:
- Recruiting complete, study complete
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- Austria
- Germany
- Switzerland
- Number of study centers:
- Multicenter study
- Recruitment location(s):
-
- Medical center Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin Freiburg
Recruitment period and number of participants
- Planned study start date:
- No Entry
- Actual study start date:
- 2014-09-18
- Planned study completion date:
- No Entry
- Actual Study Completion Date:
- 2022-12-23
- Target Sample Size:
- 50
- Final Sample Size:
- 31
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- no minimum age
- Maximum Age:
- no maximum age
- Additional Inclusion Criteria:
- All patients with Gorham-Stout-Disease/the generalised lymphatic anomalies which have given their written consent are enclosed.
Exclusion Criteria
The patients with missing, or secluded consent are excluded to the participation in the register.
Addresses
Primary Sponsor
- Address:
- Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin, Klinik IV, Pädiatrische Hämatologie/OnkologieMathildenstr. 179106 FreiburgGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
- Investigator Sponsored/Initiated Trial (IST/IIT):
- Yes
Contact for Scientific Queries
- Address:
- Universitätsklinikum Freiburg, ZKJ, Pädiatrische Hämatologie/Onkologie,Prof. Dr. med Jochen RösslerMathildenstr. 179106 FreiburgGermany
- Telephone:
- 0761-270-43000
- Fax:
- 0761-270-46990
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Contact for Public Queries
- Address:
- Universitätsklinikum Freiburg, Zentrum für Kinder- und JugendmedizinDr. med. Friedrich KappMathildenstraße 179106 FreiburgGermany
- Telephone:
- +49 761 270 46211
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Principal Investigator
- Address:
- Universitätsklinikum Freiburg, ZKJ, Pädiatrische Hämatologie/Onkologie,Prof. Dr. med Jochen RösslerMathildenstr. 179106 FreiburgGermany
- Telephone:
- 0761-270-43000
- Fax:
- 0761-270-46990
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Sources of Monetary or Material Support
Institutional budget, no external funding (budget of sponsor/PI)
- Address:
- Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin, Klinik IV, Pädiatrische Hämatologie/OnkologieMathildenstr. 179106 FreiburgGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Ethics Committee
Address Ethics Committee
- Address:
- Ethik-Kommission der Albert-Ludwigs-Universität FreiburgEngelberger Str. 2179106 FreiburgGermany
- Telephone:
- +49-761-27072600
- Fax:
- +49-761-27072630
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2014-06-18
- Ethics committee number:
- 310/14
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2014-09-18
Further identification numbers
- Other primary registry ID:
- No Entry
- EudraCT Number:
- No Entry
IPD - Individual Participant Data
- Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
- No
- IPD Sharing Plan:
- only by publication
Study protocol and other study documents
- Study protocols:
- No Entry
- Study abstract:
- No Entry
- Other study documents:
- No Entry
- Background literature:
- No Entry
- Related DRKS studies:
- No Entry
Publication of study results
- Planned publication:
- No Entry
- Publikationen/Studienergebnisse:
- Andreoti TA, Berg S, Holm A, Angerer M, Oberlin M, Foeldi E, Baumgartner I, Niemeyer CM, Rössler J, Kapp FG. Complex Lymphatic Anomalies: Report on a Patient Registry Using the Latest Diagnostic Guidelines. Lymphat Res Biol. 2023 Jun;21(3):230-243. doi: 10.1089/lrb.2022.0041. Epub 2023 Jan 27. PMID: 36706428.
- Date of first publication of study results:
- 2023-01-27
- DRKS entry published for the first time with results:
- 2023-07-28
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- No Entry