The Dallas Hereditary Spherocytosis Cohort Study
The purpose of this study is to
- better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy
- evaluate and describe the health-related quality of life in children with HS.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||The Dallas Hereditary Spherocytosis Cohort Study|
- Health related quality of life [ Time Frame: Approximately at 5 years ] [ Designated as safety issue: No ]PedsQL measurement
- Primary indications for splenectomy [ Time Frame: Approximately at 5 years ] [ Designated as safety issue: No ]Primary indication for splenectomy determined at time of splenectomy, if performed.
- Complications of HS [ Time Frame: Approximately at 5 years and at 10 years ] [ Designated as safety issue: No ]
- Complications of splenectomy [ Time Frame: Approximately at 5 years ] [ Designated as safety issue: No ]
- Diagnosis of HS [ Time Frame: At enrollment ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples Without DNA
|Study Start Date:||May 2010|
|Study Completion Date:||May 2013|
|Primary Completion Date:||May 2013 (Final data collection date for primary outcome measure)|
Patients with a new or established diagnosis of HS seen at Children's Medical Center will be asked to enroll in the study. Previous and current medical records will be reviewed to systematically catalogue their history of HS, including diagnosis, complications, hospitalizations, medications and laboratory data. Health-related quality of life questionnaires will be given to the patients and their parents at enrollment and periodically during the follow-up. Those who agree will have up to three small samples of blood collected and frozen for future laboratory studies of complications associated with HS and/or splenectomy.
We anticipate enrolling approximately 200 children and young adults with HS in this study and following them until adulthood (age 18-21 years).