More optimal prediction of SCD disease severity will lead to more precise management and treatment and development of novel therapeutic options.
ID
Bron
Verkorte titel
Aandoening
Sickle cell disease
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
1. To describe the natural course of disease e.g. symptoms and complications, growth, psychosocial and neurocognitive development, socioeconomic and demographic characteristics of children and adults with SCD;
2. To identify (molecular) genetic and epigenetic, biological, demographic and psychological and therapeutic determinants for morbidity, mortality and treatment outcome in children and
adults with SCD;
3. To investigate the long-term effects of current and future therapies on symptoms and complications, preservation of organ function, growth, psychosocial and neurocognitive development in children and adults with SCD;
4. To evaluate and improve aspects of health care organization and other (perceived) care aspects in children and adults with SCD by measurement of patient and treatment characteristics, patient-reported outcomes, patient reported experiences and to perform analyses of associations between these factors and health care outcome.
Achtergrond van het onderzoek
This long term retrospective and prospective observational cohort study in sickle cell disease
(SCD) patients in the Netherlands aims to determine the natural history of SCD and to
identify modifying factors which contribute to morbidity and mortality of the disease.
Doel van het onderzoek
More optimal prediction of SCD disease severity will lead to more precise management and treatment and development of novel therapeutic options.
Onderzoeksopzet
Data will be collected retrospectively and prospectively at participants' half yearly regular clinic visits as part of standard care.
Onderzoeksproduct en/of interventie
NA
Publiek
Wetenschappelijk
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
- A diagnosis of SCD of any genotype;
- Written informed consent by the patient or legal guardians, and pediatric consent when indicated.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
- Any medical or social reason, which obstructs or inhibits study participation according to treating physician;
- Patient or legal guardians unable or unwilling to give consent, or lack of pediatric consent when indicated.
Opzet
Deelname
Voornemen beschikbaar stellen Individuele Patiënten Data (IPD)
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL7873 |
| Ander register | METC Erasmus MC : MEC-2019-0436 |