The Dallas Hereditary Spherocytosis Cohort Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01141621
Recruitment Status : Terminated (Insufficient staff to continue pursuing project)
First Posted : June 10, 2010
Last Update Posted : May 14, 2013
Information provided by (Responsible Party):
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.

Condition or disease
Hereditary Spherocytosis

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 Type : Observational
Actual Enrollment : 55 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Dallas Hereditary Spherocytosis Cohort Study
Study Start Date : May 2010
Actual Primary Completion Date : May 2013
Actual Study Completion Date : May 2013

Primary Outcome Measures :
  1. Health related quality of life [ Time Frame: Approximately at 5 years ]
    PedsQL measurement

Secondary Outcome Measures :
  1. Primary indications for splenectomy [ Time Frame: Approximately at 5 years ]
    Primary indication for splenectomy determined at time of splenectomy, if performed.

  2. Complications of HS [ Time Frame: Approximately at 5 years and at 10 years ]
  3. Complications of splenectomy [ Time Frame: Approximately at 5 years ]
  4. Diagnosis of HS [ Time Frame: At enrollment ]

Biospecimen Retention:   Samples Without DNA

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Center for Cancer and Blood Disorders outpatient clinics

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

Responsible Party: University of Texas Southwestern Medical Center Identifier: NCT01141621     History of Changes
Other Study ID Numbers: IRB # 022010-024
First Posted: June 10, 2010    Key Record Dates
Last Update Posted: May 14, 2013
Last Verified: May 2013

Keywords provided by University of Texas Southwestern Medical Center:
congenital hemolytic anemia
quality of life

Additional relevant MeSH terms:
Spherocytosis, Hereditary
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn