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Cognitive, Academic and Psychosocial Functioning in Long-Term Survivors of Pediatric Stem Cell Transplantation

Completed
Conditions
Stem Cell Transplant During Childhood
Allogeneic Bone Marrow Transplant During Childhood
Interventions
Other: Questionnaire
Other: Neurocognitive Evaluation
Registration Number
NCT01701492
Lead Sponsor
St. Jude Children's Research Hospital
Brief Summary

This is an observational study to collect information by use of performance-based measures and survey questionnaires. It does not include interventions aimed at altering patient outcome.

Advances in pediatric hematopoietic stem cell transplantation (SCT) have resulted in improved survival and prompted increased attention to the potential adverse late effects of this procedure. Survivors of SCT are thought to be at risk for neurocognitive deficits as a result of their exposure to a number of potentially neurotoxic agents. Prior studies done by our group and others have demonstrated generally stable cognitive function in the first 5 years following transplant, with little evidence of significant declines. However, there has been almost no research to date on the status of very long-term (\> 5 years post-transplant) survivors. In this study, we will evaluate a large sample of long-term survivors of allogeneic SCT using measures of intelligence, academic achievement, and specific cognitive functions such as attention, working memory and processing speed. We will also obtain measures of behavioral functioning and quality of life. We will examine how this group of survivors are functioning relative to normative expectations, and in comparison to community controls without a history of serious illness, matched on age, gender, race/ethnicity, and socioeconomic status. We will also examine the relationship between cognitive function and psychosocial function and quality of life in this population.

Detailed Description

This study will involve a one-time assessment using both performance based measures as well as both self- and parent-report questionnaires. During their yearly visit to St. Jude, eligible patients who agree to participate will undergo a single neurocognitive evaluation. This evaluation will take place in the Psychology Clinic. The administration time for the assessment battery is estimated at 2.5-3.5 hours, with variability depending on the age of the patient and the speed of completion of the tasks. All measures selected for the study have well-established psychometric properties, including acceptable reliability and validity. All measures were standardized on large, representative samples of children and adults and have age-specific norms.

PRIMARY OBJECTIVE:

To examine and describe psychological outcomes in long-term (\> 5 years) survivors of allogeneic bone marrow or stem cell transplantation (SCT) in childhood. This will be conducted in a one-time cross-sectional assessment using both performance based measures and survey questionnaires. Comparisons will be made to established normative data on well validated standardized instruments and to control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status.

* To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement.

* To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions.

* To examine psychosocial function and quality of life among long-term SCT survivors using both self- and parent-report.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
133
Inclusion Criteria
  • Treated with allogeneic bone marrow or stem cell transplant at St. Jude Children's Research Hospital (SJCRH)
  • ≤ 21 years at time of transplant
  • > 5 years from date of last transplant
  • Currently at least 8 years of age
  • English as primary language
Exclusion Criteria
  • Transplanted for metabolic storage disorder, osteogenesis imperfecta, osteopetrosis, or dyskeratosis congenita.
  • History of central nervous system (CNS) injury/disease predating or unrelated to reason for SCT
  • Major sensory or motor impairment that would preclude valid cognitive assessment. However, we will not exclude participants with mild cognitive impairments or special educational needs.
  • Prior participation in BMTPE protocol at St. Jude Children's Research Hospital.

Control Group Inclusion Criteria

  • Age at least 8 years
  • No known history of serious illness
  • Demographic match to a St. Jude stem cell transplant patient

Control Group Exclusion Criteria

  • Major sensory or motor impairment that would preclude valid cognitive assessment.
  • History of specific CNS disease/injury (e.g., Down Syndrome, traumatic brain injury). However, we will not exclude participants with mild cognitive impairments or special educational needs.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Stem Cell Transplant SurvivorsNeurocognitive EvaluationAll participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation.
Normal control participantsQuestionnaireControl participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation.
Stem Cell Transplant SurvivorsQuestionnaireAll participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation.
Normal control participantsNeurocognitive EvaluationControl participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation.
Primary Outcome Measures
NameTimeMethod
Intelligence and academic achievement1 day

To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement when compared to established normative data on well validated standardized instruments as well as to control participants in the community.

Performance in specific neuropsychologic domains.1 day

To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions compared to established normative data on well validated standardized instruments as well as to control participants in the community.

Psychosocial function1 day

To examine psychosocial function among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.

Quality of life1 day

To examine quality of life among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

St. Jude Children's Research Hospital

🇺🇸

Memphis, Tennessee, United States

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