Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)

Study Overview
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Brief Summary
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Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Good outcomes can be achieved with alkylating agents only as a preparative regimen. This plan will use a combination of busulfan and cyclophosphamide (Bu/Cy) with or without antithymocyte globulin (ATG) to reduce the late toxicities of therapy that includes TBI.

Official Title
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Treatment Plan for Hematologic Malignancies Using Intravenous Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide to Examine Results, Success and Side Effects of Treatment With Chemotherapy Only, as a Preparative Therapy for Patients With Cord Blood Transplants

Conditions
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Acute Leukemias, Chronic Leukemias, Myelodysplastic Syndrome, Juvenile Myelomonocytic Leukemia

Intervention / Treatment
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* Drug: Busulfan/Cyclophosphamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Suitable cord blood from matched unrelated or related donor. Cardiac (Echo/EKG): shortening fraction ≥ 27% Electrolytes within normal CCHMC limits. Pulmonary function tests: DLCO ≥ 50% Renal: creatinine clearance/GFR ≥ 60 ml/minute/1.73m2 Lumbar puncture: no leukemic infiltrate. CBC: ANC ≥ 1000ml and unsupported platelet count of ≥ 50,000/ml Documented HSV and CMV titers, Hepatitis B surface antigen, Hepatitis C by serology, HIV by serology: all negative. Hepatic transaminases < 2.5x normal; Total bilirubin < 2 mg/dl Patients who do not meet above organ function criteria (liver, cardiac, renal), due to the presence of a tumor compromising these organs, may have exception made for these criteria and remain eligible for treatment plan after consultation with Program Director of Blood and Marrow Transplant Exclusion Criteria: Patients with neoplastic or non-neoplastic disease of any major organ system that would compromise their ability to withstand the pre-transplant conditioning regimen. Patients with uncontrolled (culture or biopsy positive) infections requiring intravenous antivirals, antibiotics, or antifungals. Patients on prolonged antifungal therapy with a history of fungal infection should be considered for a non-myeloablative protocol. Patients who are pregnant or lactating. Patients of childbearing potential must practice an effective method of birth control while participating on this treatment plan. HIV seropositive patients

Ages Eligible for Study
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Maximum Age: 21 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Busulfan/Cyclophosphamide<br> | Drug: Busulfan/Cyclophosphamide<br>* Conditioning regimen: Cyclophosphamide 50mg/kg/day for 4 days + Busulfan 0.8-1.0mg/kg/day for 4 days.<br>* Other names: Cytoxan;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | Five years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease free survival |  | Five years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Stem-cells, Transplantation, Busulfex, Cyclophosphamide, Total-Body-Ittadiation