Sickle cell disease
Conditions
Interventions
None listed
Sponsors
None listed
Eligibility
Inclusion criteria
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
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 outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. | — |
Contacts
Public ContactIneke van Vliet
Erasmus Medical Center
Outcome results
None listed