Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies
1 other identifier
observational
340
1 country
32
Brief Summary
This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric HCT for hematologic malignancies. This study is a collaboration between the Pediatric Blood and Marrow Transplant Consortium (PBMTC), the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Transplant Program (NMDP) and the Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI-BMT) of the CIBMTR. The study will enroll pediatric patients who undergo myeloablative HCT for hematologic malignancies at PBMTC sites.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Mar 2015
Longer than P75 for all trials
32 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 12, 2015
CompletedFirst Posted
Study publicly available on registry
January 14, 2015
CompletedStudy Start
First participant enrolled
March 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2023
CompletedFebruary 8, 2023
February 1, 2023
5.5 years
January 12, 2015
February 7, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
To report the incidence of chronic kidney disease (CKD), metabolic syndrome, and osteopenia
Baseline to 1 and 2 years following allogeneic HCT for hematologic malignancy
Secondary Outcomes (9)
To identify prognostic risk factors for the development and progression of post-HCT CKD, metabolic syndrome, and osteopenia
Baseline to 1 and 2 years following HCT
To investigate potential associations of systemic hypertension as measured with intermittent blood pressure assessment with proteinuria, acute kidney injury, and CKD
Baseline to 100 days, and at 1 and 2 years following HCT
To compare the results of GFR estimating equations based on serum cystatin C levels or serum creatinine to GFR measured by nuclear medicine GFR and/or 24-hour creatinine clearance
Baseline to 180 days, and at 1 and 2 years following HCT
To explore potential association of the protein biomarker elafin in the urine at with the development of CKD
Baseline to 180 days, and at 1 and 2 years following HCT
To report levels of fasting triglycerides, low-density lipoprotein, high-density lipoprotein, insulin, and glucose levels
Baseline to 100 days, and at 1 and 2 years following HCT
- +4 more secondary outcomes
Eligibility Criteria
This study will enroll is a nonrandomized prospective cohort undergoing HCT for treatment of childhood leukemia and myelodysplasia.
You may qualify if:
- Age less than 22 years at admission for HCT
- Planned allogeneic HCT from any donor and stem cell source. There are no study-specific criteria for HLA-matching
- Disease and disease status criteria
- Acute lymphoblastic leukemia/lymphoma in complete morphologic remission defined as a M1 marrow (\<5% blasts) with no evidence of active extramedullary disease within 30 days of the start of the conditioning regimen; OR
- Myelodysplasia (regardless of subtype) with less than 10% marrow blasts within 30 days of the start of the conditioning regimen; OR
- Acute myelogenous leukemia in complete morphologic remission defined as an M1 marrow (\<5% blasts) with no evidence of extramedullary disease within 30 days of the start of the conditioning regimen; OR
- Juvenile myelomonocytic leukemia; OR
- Chronic myelogenous leukemia excluding refractory blast crisis.
- Planned myeloablative conditioning regimen, defined as a regimen including one of the following as a backbone agent:
- Busulfan ≥ 12.8 mg/kg total dose (IV or PO). PK-based dosing allowed, if the intent is total overall dose ≥ 12.8 mg/kg; OR
- Total Body Irradiation ≥ 1200 cGy fractionated; OR
- Treosulfan ≥ 30 g/m2 total dose IV
- Enrollment in the following NMDP research protocols:
- Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxicity Injuries
- Protocol for a Research Sample Repository for Allogeneic Hematopoietic Stem Cell Transplantation and Marrow Toxic Injuries
- +1 more criteria
You may not qualify if:
- Prior allogeneic or autologous HCT
- Patients with renal disease prior to the start of HCT conditioning requiring the use of dialysis at the time of enrollment and/or GFR \< 60 mL/min/1.73 m2
- Patients with osteopenia or osteoporosis treated with a bisphosphonate medication at any time prior to enrollment
- Patients with preexisting diabetes or hyperglycemia treated with insulin or oral hypoglycemic medication at the time of enrollment
- Patients with uncontrolled viral, bacterial, fungal or protozoal infection at the time of study enrollment
- Karnofsky performance score or Lansky Play-Performance Scale Score \<60 at the time of study enrollment
- Known inherited or constitutional predisposition to cancer including, but not limited to Down Syndrome, Li-Fraumeni syndrome, Fanconi Anemia, and patients with BRCA1 and BRCA2 mutations
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (32)
Mayo Clinic
Scottsdale, Arizona, 85259-5499, United States
University of Arizona Medical Center
Tucson, Arizona, 85724, United States
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
UCLA Center for Health Sciences
Los Angeles, California, 90095, United States
Children's Hospital & Research Center - Oakland
Oakland, California, 94609, United States
University of California San Francisco Medical Center
San Francisco, California, 94143, United States
University of Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
All Children's Hospital
St. Petersburg, Florida, 33701, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60601, United States
Indiana University Hospital/Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
Dana Farber Cancer Institute - Pediatrics
Boston, Massachusetts, 02215, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
The Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
Westchester Medical Center
Valhalla, New York, 10595, United States
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27599, United States
Levine Children's Hospital
Charlotte, North Carolina, 28203, United States
Duke University Medical Center - Pediatrics
Durham, North Carolina, 27705, United States
University Hospitals Case Medical Center
Cleveland, Ohio, 44106, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Science University - Doernbecher Children's Hospital
Portland, Oregon, 97239, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37235, United States
Children's Medical Center Dallas
Dallas, Texas, 75235, United States
Texas Transplant Institute
San Antonio, Texas, 78229, United States
Primary Children's Hospital
Salt Lake City, Utah, 84111, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Christine Duncan, MD
Dana-Farber Cancer Institute
- STUDY CHAIR
K. Scott Baker, MD
Fred Hutchinson Cancer Center
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 12, 2015
First Posted
January 14, 2015
Study Start
March 1, 2015
Primary Completion
September 1, 2020
Study Completion
December 1, 2023
Last Updated
February 8, 2023
Record last verified: 2023-02