Curative Versus Disease-Modifying Therapies in Children With Severe Sickle Cell Disease

NCT01369160 | Completed DMC

• 1 other identifier: 261-2005
• Study Type: observational
• Target: 33
• Locations: 1 country
• Sites: 2

Brief Summary

The research proposed is a pilot study of pediatric and adolescent/young adult patients who have received the curative intervention (MSD-SCT), disease-modifying interventions (HU or CT) or SCC (control), with respect to three clinically important outcomes: quality-of-life (QOL), neurocognitive function, and reproductive potential. Comparable cohorts will be identified for each of the groups, drawing from patients treated by the SCD program of Children's Healthcare of Atlanta (CHOA). QOL measures and neuropsychiatric testing and will be administered. Reproductive endocrine function markers (laboratory studies and pubertal staging), will be collected and analyzed. A tracking system of such patients will also be developed, gathering available retrospective data and setting up a mechanism for collection of new data.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (2)

• quality of life

  5 years after last patient enrolled

• neuropsychiatric testing

  1 year after last patient enrolled

Study Arms (4)

1

Chronic Transfusion

Behavioral: Quality of Life measures

2

hydroxyurea

Behavioral: Quality of Life measures

3

matched sibling donor stem cell transplantation (MSD-SCT)

Behavioral: Quality of Life measures

4

standard comprehensive care (SCC, control)

Behavioral: Quality of Life measures

Interventions

Quality of Life measures BEHAVIORAL

measuring QOL with different therapies

1; 2; 3; 4

Eligibility Criteria

• Age: 3 Years - 23 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Greater than or equal to 3 years of age
• Homozygous hemoglobin S (HbSS)
• Severe disease, defined as having one or more of the following:
• recurrent (2 or more episodes per year) acute chest syndrome (ACS),
• frequent (3 or more episodes per year) vaso-occlusive pain events, defined as episode lasting 4 hours and requiring hospitalization or outpatient treatment with parenteral narcotics
• Any combination of 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years.
• any stroke, defined as central nervous system (CNS) event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings
• At least one year has elapsed since start of therapy for severe disease (CT, HU, MSD-BMT or SCC).

You may not qualify if:

• Inadequate medical records to support eligibility criteria
• Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).

Sponsors & Collaborators

• Emory University: lead

Study Sites (2)

Children's Healthcare of Atlanta/Emory University

Atlanta, Georgia, 30322, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Ann Haight, MD

  Children's Healthcare of Atlanta

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: April 22, 2009
• First Posted: June 8, 2011
• Study Start: May 1, 2005
• Primary Completion: May 1, 2007
• Study Completion: March 1, 2014
• Last Updated: May 28, 2014

Record last verified: 2014-05

Locations

US (2)