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A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children with Juvenile Myelomonocytic Leukemia (JMML)
Johns Hopkins Kimmel Cancer Center in Baltimore
This randomized phase II trial studies how well giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before donor hematopoietic cell transplant works in treating younger patients with juvenile myelomonocytic leukemia. Giving chemotherapy before a donor hematopoietic transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving busulfan, cyclophosphamide, and melphalan or busulfan and fludarabine phosphate before a donor stem cell transplant is more effective in treating juvenile myelomonocytic leukemia.
Ages Eligible for Study: 3 Months to 18 Years Genders Eligible for Study: Both Accepts Healthy Volunteers: No CriteriaInclusion Criteria:•Patients must have a strong clinical suspicion of JMML, based on a modified category 1 of the revised diagnostic criteria; specifically, eligible patients must have all of the following:?Splenomegaly?Absolute monocyte count (AMC) greater than 1000/uL?Blasts in peripheral blood (PB)/bone marrow (BM) less than 20%•For the 7-10% of patients without splenomegaly, the diagnostic entry criteria must include all other features described above and at least 2 of the following criteria:?Circulating myeloid precursors?White blood cell (WBC) greater than 10,000/uL?Increased fetal hemoglobin (HgbF) for age?Sargramostim (GM-CSF) hypersensitivity•Patients must be previously untreated with HCTExclusion Criteria:•Patients with a known germline mutation of Noonan's Syndrome (PTPN11) are not eligible•Patients with a history of Neurofibromatosis type 1 (NF1) and either?A history of a tumor of the central nervous system (astrocytoma or optic glioma), or?A malignant peripheral nerve sheath tumor with a complete remission of less than 1 year are not eligible
Experimental: BU-CY-MEL (busulfan, cyclophosphamide, melphalan) HCT CONDITIONING REGIMEN: Patients receive busulfan IV QD, BID, or QID (total daily dose of 3.2 - 4 mg/kg based on weight and age, and adjusted by pharmacokinetics) over 2-3 hours on days -8 to -5, cyclophosphamide 60 mg/kg IV QD over 60 minutes on days -4 and -3, and melphalan 140 mg/m2 IV over 15-30 minutes on day -1.TRANSPLANT: Patients undergo allogeneic hematopoietic stem cell transplantation no sooner than 24 hours after the last dose of chemotherapy.Patients receive tacrolimus IV then PO on days -1 to 98 (related donor) or 180 (unrelated donor) and mycophenolate mofetil IV over 2 hours TID then PO on days 1-30 (related donor) or 45 (unrelated donor).
10/01/2016 05:03 AM