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

Tel Aviv Sourasky Medical Center

Status and phase

Unknown

Phase 4

Conditions

Acute Leukemias

Chronic Leukemias

Juvenile Myelomonocytic Leukemia

Myelodysplastic Syndrome

Treatments

Drug: Busulfan/Cyclophosphamide

Study type

Interventional

Funder types

Other

Identifiers

NCT01339988

TASMC-11-MB-442-CTIL

Details and patient eligibility

About

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.

Enrollment

10 estimated patients

Sex

Male

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

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