Identifying Predictors of Poor Health-Related Quality-of-Life Among Pediatric Hematopoietic Stem Cell Donors

To compare donors to their non-donor counterparts and healthy controls as well as to generate trajectory classes based on longitudinal patterns of donor HRQoL and identify predictors of poor donor HRQoL.

Study Overview

• Status: Active, not recruiting
• Conditions: Quality of Life

Detailed Description

There is limited data to show the effects that donation has on pediatric donors to their siblings. There is widespread agreement that it is critical to investigate the medical and psychosocial aspects of sibling pediatric HSC donation for multiple reasons including (a) the vulnerability of the pediatric population undergoing donation, (b) increasing use of pediatric HSC donation as a therapeutic option, (c) evidence that ~20% of pediatric HSC donors experience clinically important HRQoL deficits, and (d) the impact that improved understanding of factors predicting poor HRQoL will have on our ability to develop guidelines and/or interventions for assisting at-risk donors/families. The proposed study will help to compare donors to their non-donor counterparts and healthy controls as well as to generate trajectory classes based on longitudinal patterns of donor HRQoL and identify predictors of poor donor HRQoL.

• Study Type: Observational
• Enrollment (Actual): 754

Contacts and Locations

• Study Contact: Name: Brandan Butler, B.A.; Phone Number: 7634063280; Email: bbutler@nmdp.org
• Study Contact Backup: Name: Lisa Erickson; Phone Number: 76340644710; Email: lericks2@nmdp.org

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • California
    • Duarte, California, United States, 91010
      • City of Hope
    • Loma Linda, California, United States, 92354
      • Loma Linda University
    • Los Angeles, California, United States, 90027
      • Children's Hospital of Los Angeles
    • San Diego, California, United States, 92123
      • Rady Children's Hospital San Diego
    • San Francisco, California, United States, 94143
      • University of California San Francisco Medical Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Yale New Haven Hospital
  • District of Columbia
    • Washington, District of Columbia, United States, 20910
      • Children's National Medical Center
  • Florida
    • Miami, Florida, United States, 33155
      • Niklaus Children's Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville Hospital - James Brown Cancer Center
  • Louisiana
    • New Orleans, Louisiana, United States, 70118
      • Children's Hospital of New Orleans/LSUHSC
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • NIH/NCI
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Spectrum Health
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • The Children's Mercy Hospitals and Clinics
    • Saint Louis, Missouri, United States, 63110
      • St. Louis Children's Hospital
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan-Kettering Cancer Center
    • New York, New York, United States, 10595
      • Westchester Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina Hospitals
    • Durham, North Carolina, United States, 27705
      • Duke University Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Vanderbilt University Medical Center
  • Texas
    • Dallas, Texas, United States, 75235
      • Children's Health (formerly Children's Medical Center Dallas)
    • San Antonio, Texas, United States, 78229
      • Methodist Healthcare System of San Antonio/Texas Transplant Institute
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah Blood and Marrow Transplant Program - Pediatrics
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Children's Hospital of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Pediatric donors, their recipients and caregivers, non-donor siblings from the donor-recipient families, and non-donor siblings of patients receiving unrelated transplants.

Description

Inclusion Criteria:

• Participants must fall into one of the following categories:

  • Donor between the age of 5 and 17 who is donating to a sibling
  • Parent/caregiver of study participating donor
  • Recipient sibling aged 5 to 17 of study participating donor
  • Any of the donor's non-donor/non-recipient siblings between 5 and 17
  • Any child between 5 and 17 with a brother or sister (also between 5 and 17) receiving a transplant from an unrelated source

• Be willing and able to provide signed informed consent:

  • Adults must give consent for their children's and, if applicable, their own participation
  • Assent will be obtained in accordance with guidelines at the participant's transplant institution
• Be willing and able to respond to psychological assessment questions
• Must be the donor's first donation
• Recipient must consent to the CIBMTR research database

Exclusion Criteria:

• For families with pediatric donors aged 5 to 17, at minimum, the donor child and/or one parent must consent/assent to participation. If that minimum is not met, the family will be excluded
• Donor or non-donor siblings who do not live in the same household as the recipient at lease half of the time

• Unable to consent/assent or complete a phone interview in English

  • Parents may consent/assent in Spanish
• No access to a telephone

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

6

Cohorts and Interventions

Group / Cohort
Sibling pediatric donors; Donors who are donating to a sibling
Sibling recipients and caregivers; Recipients who are receiving a transplant from a sibling
Non-donor sibling; From the donor-recipient families
Non-donor siblings; Of patients receiving unrelated transplants
Healthy comparison; A matched sample
Parents; Parent/caregiver of study participating donor.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HRQoL (Health Related Quality of Life)	To longitudinally and quantitatively describe the HRQoL of a diverse nation-wide cohort of sibling pediatric Hematopoietic Stem Cell (HSC) donors (Peripheral Blood Stem Cell or Bone Marrow) and to compare their HRQoL to that of (1) healthy non-donor sibling from the same family, (2) siblings of children with similar diseases who receive alternate treatments (e.g., unrelated cord blood transplants), and (3) healthy age, gender, and race/ethnicity-matched controls.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
1 year post-donation	To identify and examine donor HRQoL trajectories from pre- through 1 year post-donation.	1 year
Donor characteristics	To determine which donor characteristics (e.g., demographic, psychosocial, and donation-related), recipient characteristics (e.g., recipient disease, transplant complications and outcomes), family characteristics (e.g., composition, cohesiveness, stress), and transplant center characteristics (e.g., presence of a donor advocate) most strongly predict membership in trajectory classes with poor HRQoL among donors.	1 year

Collaborators and Investigators

• Sponsor: Center for International Blood and Marrow Transplant Research
• Collaborators: University of Pittsburgh
• Investigators: Principal Investigator: Galen Switzer, Ph.D, University of Pittsburgh

Study record dates

Study Major Dates

• Study Start (Actual): April 3, 2019
• Primary Completion (Estimated): August 31, 2024
• Study Completion (Estimated): August 31, 2024

Study Registration Dates

• First Submitted: October 23, 2018
• First Submitted That Met QC Criteria: October 23, 2018
• First Posted (Actual): October 24, 2018

Study Record Updates

• Last Update Posted (Actual): December 6, 2023
• Last Update Submitted That Met QC Criteria: December 5, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: 17-SIBS

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No