Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies

Inclusion Criteria:

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
Written informed consent document signed by patient if the age is greater than or equal to 18 years and the patient is developmentally able to provide consent. The informed consent document is to be signed by the parent or legal guardian if the patient's age is less than 18 years or if the patient is older than 18 years, but developmentally unable to provide consent. Assent will be obtained according to the guidelines of the patient's transplant institution.

Exclusion Criteria:

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