Overview

Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia

Status:
Completed
Trial end date:
2017-12-31
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Children's Oncology Group
Collaborator:
National Cancer Institute (NCI)
Treatments:
Busulfan
Cyclophosphamide
Fludarabine
Fludarabine phosphate
Mechlorethamine
Melphalan
Mycophenolate mofetil
Mycophenolic Acid
Nitrogen Mustard Compounds
Tacrolimus
Vidarabine
Criteria
Inclusion 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) > 1000/uL

- Blasts in peripheral blood (PB)/bone marrow (BM) < 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) > 10,000/uL

- Increased fetal hemoglobin (HgbF) for age

- Sargramostim (GM-CSF) hypersensitivity OR, patients must have been previously
diagnosed with JMML

- Patients must be previously untreated with HCT

- All patients and/or their parents or legal guardians must sign a written informed
consent

- All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met

Exclusion Criteria:

- Patients with a known germline mutation of PTPN11 (Noonan?s Syndrome) are not eligible

- Patients with a known history of NF1 (Neurofibromatosis Type 1) 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 < 1 year
are not eligible

- Human immunodeficiency virus (HIV) positive patients are not eligible