No registrations found.
ID
Source
Brief title
Health condition
Sickle cell disease
Sponsors and support
Intervention
Outcome measures
Primary outcome
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.
Secondary outcome
NA
Background summary
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.
Study objective
More optimal prediction of SCD disease severity will lead to more precise management and treatment and development of novel therapeutic options.
Study design
Data will be collected retrospectively and prospectively at participants' half yearly regular clinic visits as part of standard care.
Intervention
NA
Inclusion criteria
- A diagnosis of SCD of any genotype;
- Written informed consent by the patient or legal guardians, and pediatric consent when indicated.
Exclusion criteria
- 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.
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL7873 |
| Other | METC Erasmus MC : MEC-2019-0436 |