The Dallas Hereditary Spherocytosis Cohort Study

Terminated

• Conditions: Hereditary Spherocytosis

• Registration Number: NCT01141621
• Lead Sponsor: University of Texas Southwestern Medical Center

Brief Summary

The purpose of this study is to

1. better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy

2. evaluate and describe the health-related quality of life in children with HS.

Detailed Description

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).

Study Timeline

• Study Start: 2010-05
• Study First Submitted: 2010-06-04
• Study First Submitted QC: 2010-06-08
• Study First Posted: 2010-06-10
• Status Verified: 2013-05
• Primary Completion: 2013-05
• Study Completion: 2013-05
• Last Update Submitted: 2013-05-13
• Last Update Posted: 2013-05-14

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 55

Inclusion Criteria

• Diagnosis of HS with or without prior splenectomy
• Age 0 - 21 years
• Spanish-speaking subjects are eligible to participate

Exclusion Criteria

• Unable to provide contact information for follow-up

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Health related quality of life	Approximately at 5 years	PedsQL measurement

Secondary Outcome Measures

Name	Time	Method
Complications of HS	Approximately at 5 years and at 10 years
Diagnosis of HS	At enrollment
Primary indications for splenectomy	Approximately at 5 years	Primary indication for splenectomy determined at time of splenectomy, if performed.
Complications of splenectomy	Approximately at 5 years