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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ann E. Haight, Emory University
ClinicalTrials.gov Identifier:
NCT01369160
First received: April 22, 2009
Last updated: May 23, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention
Sickle Cell Disease
Behavioral: Quality of Life measures

Study Type: Observational
Official Title: Curative vs Disease-Modifying Therapies in Children With Severe Sickle Cell Disease: A Pilot, Cross-Sectional Study

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • quality of life [ Time Frame: 5 years after last patient enrolled ] [ Designated as safety issue: No ]
  • neuropsychiatric testing [ Time Frame: 1 year after last patient enrolled ] [ Designated as safety issue: No ]

Enrollment: 33
Study Start Date: May 2005
Study Completion Date: March 2014
Primary Completion Date: May 2007 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
1
Chronic Transfusion
Behavioral: Quality of Life measures
measuring QOL with different therapies
2
hydroxyurea
Behavioral: Quality of Life measures
measuring QOL with different therapies
3
matched sibling donor stem cell transplantation (MSD-SCT)
Behavioral: Quality of Life measures
measuring QOL with different therapies
4
standard comprehensive care (SCC, control)
Behavioral: Quality of Life measures
measuring QOL with different therapies

Detailed Description:

sickle cell disease (SCD), but a significant proportion experience clinically severe disease requiring more aggressive intervention. Widely applicable curative therapy with a favorable toxicity profile remains elusive for such patients.

Three distinct intervention strategies are currently available for children with severe sickle cell disease (SCD): oral hydroxyurea (HU), chronic blood transfusions (CT), and allogeneic hematopoietic stem cell transplantation (SCT) from an HLA-matched sibling donor (MSD). Each intervention has distinct advantages and disadvantages. Many patients do not receive specific intervention, and continue standard comprehensive care (SCC).

Though indications for these therapies overlap, to date there are no comparative outcomes data, leaving families and physicians without adequate information upon which to base therapeutic decisions. The gold standard for obtaining such data would be a randomized, prospective study comparing each intervention, though this may or may not be feasible to conduct. Before such a trial is considered, a large cross-sectional trial should be conducted to establish comparisons among the four therapeutic groups (HU, SCT, CT, SCC) with respect to the outcomes that clinicians and families deem most important.

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.

  Eligibility

Ages Eligible for Study:   3 Years to 23 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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).

Exclusion Criteria:

  • Inadequate medical records to support eligibility criteria
  • Patients less than 1 year from start of therapy (CT, HU, MSD-BMT or SCC).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01369160

Locations
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Investigators
Principal Investigator: Ann Haight, MD Children's Healthcare of Atlanta
  More Information

No publications provided

Responsible Party: Ann E. Haight, Assistant Professor, Emory University
ClinicalTrials.gov Identifier: NCT01369160     History of Changes
Other Study ID Numbers: 261-2005
Study First Received: April 22, 2009
Last Updated: May 23, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 21, 2014