Sickle Cell Disease
Conditions
Keywords
sickle cell disease, iron over load
Brief summary
A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
Interventions
None listed
Sponsors
Novartis
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female patients with HbSS, HbS/beta-thalassemia and HbSC * Age \> 2 years old. * Written informed consent by the patient or legal guardians, and pediatric assent where indicated.
Exclusion criteria
* Patients with Sickle Cell trait (HbAS) are not eligible for the study * Patient or legal guardians unable or unwilling to give consent, or pediatric assent where indicated.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Document current treatment patterns, natural history and outcomes in patients with sickle cell disease | up to 5 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Data collection | up to 5 years | Collection of the following data:Current therapies used for the treatment of SCD Current transfusion practices, Difference in treatments between pediatric and adult patients, Use of chelation therapies, Frequency and types of crises including Frequency of hospitalizations, Incidence of end organ damage (caridac, renal, pulmonary,liver), Quality of life assessed by PedsQL TM Pediatric Quality of Life Inventory for patients 2- \<18 years old and SF-36® Health Survey for patients 18 years old and older |
| Measure Sickle cell crisis and hospitalizations | up to 5 years | To evaluate whether patients on regular transfusion protocol have fewer crisis and hospitalizations |
Countries
United States
Outcome results
None listed