Improving the health of children and adolescents with autoinflammatory diseases
Organizational Data
- DRKS-ID:
- DRKS00032447
- Recruitment Status:
- Recruiting planned
- Date of registration in DRKS:
- 2024-01-04
- Last update in DRKS:
- 2024-01-04
- Registration type:
- Prospective
Acronym/abbreviation of the study
Pro-AID
URL of the study
No Entry
Brief summary in lay language
Autoinflammatory diseases (AIDs) are rare chronic diseases in which recurrent or permanent inflammation occurs due to dysregulation of the innate immune system. Affected children and adolescents are at increased risk for comorbidities and socioeconomic burden. Early therapy with complete inflammation control can prevent organ damage, reduced quality of life and shortened life expectancy. Pro-AID investigates the current health care situation of children and adolescents with AID in Germany and the psychosocial burden of those affected and their families. Pro-AID is a prospective observational study that uses the structures of the national paediatric rheumatology database (NPRD). Over a total of 3 years, disease characteristics and disease activity as well as the therapy of children and adolescents with AID are recorded by physicians every six months within routine outpatient paediatric rheumatology appointments. Parents of affected children and adolescents as well as patients (12 years and older) report on their disease burden, family situation, psychosocial stress, and unmet needs as well as their satisfaction with the care they receive. The data are used to examine whether the patients are cared for according to the current recommendations of the Society for Paediatric Rheumatology and what the treatment results are. In addition, the subjective burden of disease (e.g. the quality of life of those affected, days absent from school), family burden and satisfaction with care are evaluated. Based on the information gained, measures for optimising the care of children and adolescents with AID will be developed with those affected and patient organisations. The knowledge gained shall be incorporated into future AID treatment recommendations.
Brief summary in scientific language
Autoinflammatory diseases (AID) are rare chronic diseases in which recurring or permanent inflammation occurs due to dysregulation of the innate immune system. Affected children and adolescents are at increased risk of comorbidities and socio-economic burden. Early effective treatment can prevent organ damage, reduced quality of life and shortened life expectancy. The Pro-AID project analyses the current health care situation of children and adolescents with AID and the psychosocial burden of affected patients and their families. Pro-AID is a prospective observational study incorporated in the national paediatric rheumatology database (NPRD). Over a total of three years, disease characteristics and activity as well as the treatment of children and adolescents with AID are recorded approximately every six months in routine care. Parents of affected children and adolescents as well as patients (aged 12 and over) report on their disease burden, family situation, psychosocial stress and unmet needs as well as their satisfaction with care. The primary study objective is to investigate whether patients are being treated in accordance with the current recommendations of the paediatric rheumatology society. The primary study outcome is disease activity. Secondary outcomes include the subjective burden of disease (e.g. quality of life of those affected, days missed from school), family burden and satisfaction with care. Based on the information obtained, measures to optimise the care of children and adolescents with AIDs are developed together with patient organisations. The knowledge gained will be incorporated into the further development of AID treatment recommendations.
Health condition or problem studied
- Free text:
- FMF (familial Mediterranean fever), NLRP3-AID (CAPS = Cryopyrin associated periodic syndromes), MKD (mevalonate kinase deficiency), TRAPS (TNF receptor associated periodic fever syndrome), PFAPA, DIRA (Deficiency of the IL-1 receptor antagonist), DITRA (Deficiency of the IL-36 receptor antagonist), NLRP12-AID (FCAS2 = Familial cold autoinflammatory syndrome 2), Aicardi-Goutières Syndrome (AGS), STING-Associated Vasculopathy with Onset in Infancy (SAVI), Chilblain Lupus, CANDLE (PRAAS) Syndrom, COPA Syndrome, Deficiency of Adenosine Deaminase 2 (DADA2), M. Behcet, unclassified and other AID
- ICD10:
- E85.0 - Non-neuropathic heredofamilial amyloidosis
- ICD10:
- M35.2 - Behçet disease
- Free text:
- ORPHA:210115 ORPHA:325004 ORPHA:342 ORPHA:32960 ORPHA:42642 ORPHA:247868 ORPHA:309025 ORPHA:51 ORPHA:404546 ORPHA:90280 ORPHA:117 ORPHA:425120 ORPHA:208650 ORPHA:444092
- Healthy volunteers:
- No
Interventions, Observational Groups
- Arm 1:
- Prospective observation of prevalent and incident cases with AID approximately every 6 months for a total of 24 months - Physician reports on diagnosis, age at onset of disease at baseline every 6 months on disease activity, therapy, clinical manifestations, concomitant diseases, selected laboratory parameters at the last visit, additionally on disease damage (using ADDI) - Patient/parent information on symptom onset, doctor contacts and time to diagnosis at baseline every 6 months on overall health, coping with illness, quality of life, family burden (FaBel), adherence to therapy, satisfaction with therapy
Endpoints
- Primary outcome:
- Disease activity (physician global assessment on a numerical rating scale of 0-10, NRS) at each visit
- Secondary outcome:
- Disease activity (patient global assessment on a numerical rating scale of 0-10) at each visit Damage (ADDI) at last visit Coping with disease, NRS, at each visit Quality of life (PedsQL 3.0 and 4.0, EQ-5D), at each visit Family burden (FaBel), at each visit Therapy adherence (JAMAR), at each visit Satisfaction with therapy (Likert scale), at each visit
Study Design
- Purpose:
- Health care system
- Retrospective/prospective:
- Prospective
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- Longitudinal study
- Study type non-interventional:
- Epidemiological study
Recruitment
- Recruitment Status:
- Recruiting planned
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- Austria
- Germany
- Number of study centers:
- Multicenter study
- Recruitment location(s):
-
- University medical center Kinderklinik der Medizinischen Hochschule Hannover Hannover
- University medical center Klinik für Kinder- und Jugendmedizin Erlangen
- Medical center Kinderklinik Amsterdamer Straße Köln
- Medical center Asklepios Kinderklinik Sankt Augustin Sankt Augustin
- Medical center St. Marien Krankenhaus Landshut
- Medical center Olgahospital Stuttgart
- Medical center St. Josef Stift Sendenhorst
- Medical center Klinikum Bremen-Mitte Bremen
- University medical center Klinik für Kinder- und Jugendmedizin Ulm
- University medical center Dr. von Hauner'sches Kinderspital München
- Medical center Kinderrheumaklinik Garmisch-Partenkirchen
- University medical center Charité Berlin
- University medical center Klinik für Kinder- und Jugendmedizin Tübingen
- University medical center Department für Kinder- und Jugendmedizin Innsbruck
Recruitment period and number of participants
- Planned study start date:
- 2024-01-15
- Actual study start date:
- No Entry
- Planned study completion date:
- 2028-12-31
- Actual Study Completion Date:
- No Entry
- Target Sample Size:
- 250
- Final Sample Size:
- No Entry
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- no minimum age
- Maximum Age:
- 18 Years
- Additional Inclusion Criteria:
- - autoinflammatory disease diagnosed by paediatric rheumatologist - written informed consent
Exclusion Criteria
- Lack of cognitive maturity to understand the requirements and content of the project - Diagnoses: systemic JIA, CRMO
Addresses
Primary Sponsor
- Address:
- Deutsches Rheuma-Foschungszentrum Berlin10117 BerlinGermany
- 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:
- Deutsches Rheuma-Forschungszentrum BerlinDr. med. Tatjana WelzelCharitéplatz 110117 BerlinGermany
- Telephone:
- +493028460632
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Contact for Public Queries
- Address:
- Deutsches Rheuma-Forschungszentrum BerlinDr. med. Tatjana WelzelCharitéplatz 110117 BerlinGermany
- Telephone:
- +493028460632
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Principal Investigator
- Address:
- Deutsches Rheuma-Forschungszentrum BerlinProf. Dr. med. Kirsten MindenCharitéplatz 110117 BerlinGermany
- Telephone:
- +493028460669
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://www.drfz.de
Sources of Monetary or Material Support
Private sponsorship (foundations, study societies, etc.)
- Address:
- Kindness for Kids - Stiftung für Kinder mit Seltenen Erkrankungen82319 StarnbergGermany
- 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 an der Medizinischen Fakultät der RWTH AachenWendlingweg 252074 AachenGermany
- Telephone:
- +49-241-8089963
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- http://www.medizin.rwth-aachen.de/cms/Medizin/Die-Fakultaet/Organisation/~coeq/Ethik-Kommission/
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2023-06-20
- Ethics committee number:
- EK 384/19
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2023-10-27
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:
- No Entry
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:
- No Entry
- Date of first publication of study results:
- No Entry
- DRKS entry published for the first time with results:
- No Entry
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- No Entry