FRESH-PEF73379-en
Primary Ciliary Dyskinesia: Identification of specific severity criteria and phenotype-genotype correlation study
RaDiCo-DCP
| Name | Country code |
|---|---|
| France | fr |
RaDiCo-DCP is a French national cohort dedicated to primary ciliary dyskinesia (PCD), a rare respiratory disease of autosomal recessive genetic origin. It results from structural or functional abnormalities of the cilia, tiny structures found in particular in the respiratory tract, which normally clear mucus. This dysfunction causes recurrent infections of the upper and lower airways from early childhood, which may progress to nasal polyposis, bronchiectasis and, in about one third of patients, respiratory failure. Half of cases are accompanied by a laterality defect of the organs (Kartagener syndrome), and infertility is more frequently observed, particularly in men. Around thirty genes have been identified to date, accounting for approximately half of cases. The severity of the disease varies greatly from person to person and its causes are still incompletely understood.
The main objective of this cohort study is to better understand the factors that influence the progression of PCD in order to identify, as early as possible, patients who are at risk of developing a more severe form of the disease. This knowledge will help improve medical follow-up and enable more personalized care. The study also aims to identify new genes involved in PCD, to better understand the links between genetic characteristics, cilia abnormalities, and disease manifestations, and to assess the impact of PCD on patients' quality of life, particularly with regard to ENT complications and fertility issues.
The cohort includes children and adults with confirmed PCD from several hospitals in France. Participants are monitored regularly as part of their routine care. Researchers collect medical data, test results, information on quality of life, and, when available, data from genetic analyses and biological collections. The data are pseudonymized and analyzed securely to ensure participants' confidentiality.
By compiling this information over several years, the RaDiCo-DCP cohort helps improve our understanding of this rare disease, optimize patient care, and promote the development of more personalized medicine.
['Clinical data','Paraclinical data (non-biological)','Paraclinical data (non-biological) : Imaging','Paraclinical data (non-biological) : Anthropometry','Paraclinical data (non-biological) : Function testing','Biological data','Environmental / exposure data','Genetic / genomic data','Participant-reported health data']
Individus
| Topic | Vocabulary |
|---|---|
| General medicine | health theme |
| Infectious disease medicine | health theme |
| Medical genetics | health theme |
| Otolaryngology | health theme |
| Paediatrics | health theme |
| Public, environmental and occupational health | health theme |
| Pulmonary medicine | health theme |
| Surgery | health theme |
| Primary ciliary dyskinesia | cim-11 |
| Place of Residence | other environmental determinant |
| Socio-demographic and economic determinants | health determinant |
| Socio-demographic and economic determinants: Age | health determinant |
| Environmental determinants | health determinant |
| Environmental determinants: Other | health determinant |
| Behavioral determinants | health determinant |
| Behavioral determinants: Addiction | health determinant |
| Biological determinants | health determinant |
| Biological determinants: Genetic predisposition | health determinant |
| Biological determinants: Sex | health determinant |
['Auvergne Rhône-Alpes','Grand Est','Hauts-de-France','Ile-de-France','Occitanie','Provence - Alpes - Côte d'Azur']
{
"level_sex_clusion_I": [
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D005260"
},
"value": "Female"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D008297"
},
"value": "Male"
}
],
"level_age_clusion_I": [
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D007231"
},
"value": "Infant, Newborn (birth to 28 days)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D007223"
},
"value": "Infant (28 days to 2 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D002675"
},
"value": "Child, Preschool (2 to 5 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D002648"
},
"value": "Child (6 to 12 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D000293"
},
"value": "Adolescent (13 to 18 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D055815"
},
"value": "Young Adult (19 to 24 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D000328"
},
"value": "Adult (25 to 44 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D008875"
},
"value": "Middle Aged (45 to 64 years)"
},
{
"concept": {
"vocab": "MeSH",
"vocabURI": "D000368"
},
"value": "Aged (65 to 79 years)"
}
],
"level_type_clusion_I": "Patients population",
"level_type_clusion_other": "",
"clusion_I": "All prevalent patients and incidents included in the RaDiCo-DCP cohort must: • Have a confirmed diagnosis of PCD based on at least one of the following diagnostic criteria: Kartagener syndrome (association of chronic sinusitis, bronchiectasis and situs inversus), and\/or evidence of specific abnormalities of the ciliary ultrastructure, and\/or identification of unambiguous mutations in a PCD gene. • Have at least one annual follow-up visit in accordance with standard practice. Patients who meet the following criteria will not be included: • Patient with an unconfirmed diagnosis of PCD; • Patient with a concomitant progressive pathology that may interfere with the evaluation of PCD-related manifestations.",
"clusion_E": "Patients meeting the following criteria will not be eligible for inclusion:\r\nPatient with an unconfirmed diagnosis of PCD.\r\nPatient with a concomitant progressive condition that may interfere with the assessment of PCD-related manifestations."
}
| Name |
|---|
| Bernard;MAITRE |
| Name | Role |
|---|---|
| INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) | sponsor |
| Name |
|---|
| AGENCE NATIONALE DE LA RECHERCHE (ANR) |
| MINISTERE DE L’ENSEIGNEMENT SUPERIEUR, DE LA RECHERCHE ET DE L’ESPACE |
| Name |
|---|
| Sonia;GUEGUEN |
| INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) |
| Filière Maladies Rares pulmonaires RespiFil. / European Reference Network ERN-LUNG |
| Agency name |
|---|
| CNIL |
| CESREES |
| Other |
['Through organizations (health services or institutions, schools, businesses, etc.)']
['{"concept":{"vocab":"CESSDA","vocabURI":"Other"},"value":"Other"}']
Access on specific project only
Observational Study
| Start | End |
|---|---|
| 2017-05-02 | 2027-12-31 |
Other
Annual for 5 years.
| Standard |
|---|
| ['HPO, ICD10, Snomed CT, Orpha Codes and ORDO, Drug dictionary (DCIs)'] |
['Data Management Plan and Data Validation Plan. Continuous data management (automatic control rules and query system)']
| Name | |
|---|---|
| Juliette;CARTON | juliette.carton@radico.fr |
Requests for access to RaDiCo-DCP data (aggregated or individual) will be considered by the Scientific Committee following the submission of a summary of a specific research project, as defined in the Charter of access to resources.
Prior to any publication, the PRS Lead, in consultation with the PRS Manager if applicable, shall determine—by mutual agreement with the relevant project committee and based on each individual’s respective intellectual contributions—the order of authorship and the number of authors from each entity participating in the project. Publications must include at least one author from the Promoter. Provided that this practice is accepted by the publishers of these scientific publications, RaDiCo will be listed as a co-author of the publication. In the event of refusal by the publisher, the members of the RaDiCo Consortium Agreement will provide the PRS Manager or the PRS Lead with the list of co-authors.
Before publication, articles must be submitted to the relevant Project committee for review. Users who wish to publish must take into account all comments made by the relevant committee, provided that such comments are well-founded and not arbitrary. The terms and conditions for publication and dissemination are specified in the PRS Agreement.
{"extLink":{"title":"COAR","uri":"http://purl.org/coar/access_right/c_16ec"},"value":"Restricted access"}
| Indicate if special permissions are required to access a resource |
|---|
| No |
| Name | |
|---|---|
| Juliette;CARTON | juliette.carton@radico.fr |
| Sonia;GUEGUEN | sonia.gueguen@radico.fr |
FRESH-PEF73379-en
| Name |
|---|
| Sonia GUEGUEN |