This is a multi-institutional phase II haploidentical T cell replete bone marrow transplant (BMT) study in children with high-risk leukemia. The myeloablative conditioning regimen prescribed will be Total body irradiation (TBI)-based for lymphoid leukemia and busulfan-based for myeloid leukemia. Our goal is to establish an easily exportable, inexpensive platform for haplotransplantation that has a safety profile equivalent to matched related and unrelated BMTs. The primary objective will be to estimate the incidence of 6-month non-relapse mortality (NRM), hypothesizing that NRM is \< 18%.
This is a phase II prospective study designed to evaluate the incidence of 6 month non- relapse mortality, safety, and feasibility of haploidentical bone marrow transplantation (BMT) after myeloablative conditioning with post-transplant Cy. Conditioning regimens include a total body irradiation (TBI)-based prep for lymphoid leukemias and a chemotherapy based prep for myeloid leukemias. To estimate the incidence of non-relapse mortality at 180 days following myeloablative haploidentical BMT for children and young adults with high risk hematologic malignancies.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
Chemotherapy administration
Radiation Therapy
Chemotherapy Administered
Bone Marrow Transplant
Immunosuppressive Drug Administered
Immunosuppressive Drug Administered
Children's Hospital of Colorado
Aurora, Colorado, United States
Nemours Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
All Children's Hospital Johns Hopkins Medicine
St. Petersburg, Florida, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Washington University in St. Louis
St Louis, Missouri, United States
Levine Cancer Center
Charlotte, North Carolina, United States
Medical University of South Carolina
Charleston, South Carolina, United States
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Hospital for Sick Children
Toronto, Ontario, Canada
Cumulative Incidence of Non-relapse Mortality
Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies.
Time frame: Day 180
Number of Participants With Donor Cell Engraftment
Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT.
Time frame: Day 60
Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4
Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe).
Time frame: 100 days
Cumulative Incidence of Chronic GVHD
Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD).
Time frame: 2 years
Primary and Secondary Graft Failure
Incidence (measured as a percentage) of primary and secondary graft failure.
Time frame: 2 years
Steroid and Non-steroid Immunosuppressants
Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD.
Time frame: Two Years
Steroid and Non-steroid Immunosuppressants Use Duration
Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD.
Time frame: Two Years
Survival
Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage.
Time frame: up to 1 years
Survival
Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage.
Time frame: up to 2 years
Immune Reconstitution
Characterize immune reconstitution post myeloablative haploidentical BMT with Post transplantation cyclophosphamide (PT/Cy).
Time frame: Two Years
Time to Neutrophil and Platelet Recovery
Time to neutrophil and platelet recovery in median days
Time frame: 100 days
Incidence of Donor Cell Engraftment
Incidence of donor cell engraftment measured as the percentage of donor cell engraftment.
Time frame: 60 days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.