A Retrospective/Prospective, Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) With Clinical Significance.
Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
SUMMARY
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation. Different medical entities classified as RADs by ORPHA classification are most of them chronic life threating disorders with many unmet needs for their proper clinical management creating an impact on European health systems. RADs present diagnostic challenges and their appropriate management requires from specialised multidisciplinary teams in Centers of expertise. Although there are some examples of well-established national registries on RADs in EU, the lack of recommendations for Rare disease registries implementation and the lack of standards for interoperability has led to the fragmentation or unavailability of data on prevalence, survival, main clinical manifestations or treatments in most of the European countries.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 100
Healthy Volunteers: f
View:
• Patients must meet all of the following criteria to be included in the RADeep Registry
• Age from 0-100, both female and male
• Diagnosed as RADs (SCD, THAL, PKD, and other RADs THAL according to ORPHANET classification)
• Able and willing to provide written informed consent (patient or legal representative for minors)
Locations
Other Locations
Spain
Vall d'hebron Research Institute - Vall d'Hebron Research Institute - University Hospital Vall d'Hebrón (VHIR/HUVH)
RECRUITING
Barcelona
Contact Information
Primary
María del Mar Manú Pereira, PhD
mar.manu@vhir.org
0034934893000
Backup
Victoria Gutiérrez Valle, Msc
victoria.gutierrez@vhir.org
0034934893000
Time Frame
Start Date:2021-11-30
Estimated Completion Date:2036-11
Participants
Target number of participants:32564
Treatments
Sickle cell anaemia and other related sickle diseases
Patients with sickle cell disease and related diseases in current regular follow-ups in European-Union health centers
Thalassemia and related diseases
Patients with Thalassemia disease and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.
Pyruvate Kinase Deficiency and related diseases
Patients with Pyruvate Kinase Deficiency and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.
Red Blood Cell membrane disorders and related diseases
Patients with Reb Blood Cell membrane disorders and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.