MedPath

Sickle Cell Outcome Research

Recruiting
Conditions
Sickle cell disease
Registration Number
NL-OMON29491
Lead Sponsor
A
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
Not specified
Target Recruitment
1000
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.

Study & Design

Study Type
Observational non invasive
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
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;<br>2. To identify (molecular) genetic and epigenetic, biological, demographic and psychological and therapeutic determinants for morbidity, mortality and treatment outcome in children and<br>adults with SCD;<br>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;<br>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 Measures
NameTimeMethod
© Copyright 2025. All Rights Reserved by MedPath