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

NCT01701492 | Completed

• 1 other identifier: XPD12-077 BMTPE3
• Study Type: observational
• Target: 133
• Locations: 1 country
• Sites: 1

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.

Conditions

Stem Cell Transplant During Childhood; Allogeneic Bone Marrow Transplant During Childhood

Keywords

pediatric; hematopoietic stem cell transplantation; allogeneic bone marrow transplantation

Outcome Measures

Primary Outcomes (4)

• Intelligence and academic achievement

  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.

  1 day

• Performance in specific neuropsychologic domains.

  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.

  1 day

• Psychosocial function

  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.

  1 day

• Quality of life

  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.

  1 day

Study Arms (2)

Stem Cell Transplant Survivors

All participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation.

Other: Questionnaire; Other: Neurocognitive Evaluation

Normal control participants

Control 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.

Other: Questionnaire; Other: Neurocognitive Evaluation

Interventions

Questionnaire OTHER

A one-time assessment using both performance-based measures as well as both self- and parent-report questionnaires.

Normal control participants; Stem Cell Transplant Survivors

Neurocognitive Evaluation OTHER

A one-time neurocognitive evaluation conducted in the Psychology Clinic

Normal control participants; Stem Cell Transplant Survivors

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Survivors of SCT who are \> 5 years since transplant and are returning for a transplant clinic outpatient follow-up visit will be recruited to participate on study. We will recruit patients beginning at age 8 years, with no upper age limit, although we anticipate that most patients will be between 8 and 25 years at the time of assessment. For patients in the age range 8-21 we will obtain data by both self- and parent-report. However, if patients age 18-21 present to clinic without a parent, they will still be eligible for study, and for these patients, as well as those ≤ 21 years, we will obtain performance based and self-report measures only.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• St. Baldrick's Foundation: collaborator

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Interventions

Surveys and Questionnaires

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Sean Phipps, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2012
• First Posted: October 5, 2012
• Study Start: October 19, 2012
• Primary Completion: January 23, 2018
• Study Completion: January 23, 2018
• Last Updated: May 18, 2018

Record last verified: 2018-05

Locations

US (1)