A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Description

This is a single arm, pilot study to determine if sequential cycles of maintenance therapy with decitabine and filgrastim post allogenic hematopoietic cell transplant (HCT) are feasible and effective in preventing relapse after HCT in pediatric and young adult patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and treatment related myeloid malignancies (tAML/MDS) with either idiopathic disease or underlying germline disorders, including a subset that may be at higher risk for toxicity from treatment.

Decitabine in combination with filgrastim after stem cell transplant has been shown to be effective in a large study that included mainly adult patients with acute myeloid leukemia.

The research study procedures include screening for eligibility, study treatment including evaluations and follow up visits and blood tests. Bone marrow biopsies and aspirates will be performed as standard of care.

Participants will receive study treatment for 6 months if tolerated and will be followed for 24 months after stem cell transplant.

It is expected about 37 people will take part in this research study.

This research study is supported by Dana-Farber Cancer Institute philanthropy and institutional grants from the Dana-Farber Cancer Institute and Boston Children’s Hospital.