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

May 23, 2014 updated by: Ann E. Haight, Emory University

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

33

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta/Emory University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 23 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
quality of life
Time Frame: 5 years after last patient enrolled
5 years after last patient enrolled
neuropsychiatric testing
Time Frame: 1 year after last patient enrolled
1 year after last patient enrolled

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Emory University

Investigators

  • Principal Investigator: Ann Haight, MD, Children's Healthcare of Atlanta

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2005

Primary Completion (Actual)

May 1, 2007

Study Completion (Actual)

March 1, 2014

Study Registration Dates

First Submitted

April 22, 2009

First Submitted That Met QC Criteria

June 7, 2011

First Posted (Estimate)

June 8, 2011

Study Record Updates

Last Update Posted (Estimate)

May 28, 2014

Last Update Submitted That Met QC Criteria

May 23, 2014

Last Verified

May 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 261-2005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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