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for people ages up to 29 years
at San Francisco, California and other locations
study started
estimated completion:
Principal Investigator



The goal of this study is to determine whether post-transplant consolidation with azacitidine combined with donor lymphocyte infusion (DLI) is a safe and effective approach for the prevention of relapse in pediatric and young adult patients with hematologic malignancies who have undergone hematopoietic stem cell transplantation (HSCT).

Official Title

A Phase II Study of Risk-adapted Donor Lymphocyte Infusion and Azacitidine for the Prevention of Hematologic Malignancy Relapse Following Allogeneic Stem Cell Transplantation


This is a phase II single-arm trial of azacitidine (IV or SC) in combination with escalating donor lymphocyte infusion (DLI). Patients will be enrolled on the study by day +28 +/- 7 post-transplant, prior to withdrawal of immunosuppression or administration of donor lymphocyte infusion (DLI). They will have donor chimerism and minimal residual disease (MRD) testing from peripheral blood (PB) and bone marrow (BM) on day +28 ± 7. Patients will be stratified according to risk categories (low, standard and high), defined by GVHD status, mixed versus full donor chimerism, and positive versus negative MRD results. Depending on risk assessment, immunosuppression will be tapered according to standard or fast schedules, and patients (with the exception of low-risk ALL patients) will receive one cycle of low-dose azacitidine (40mg/m2 IV/SC daily x 4 days). After tapering immunosuppression, chimerism will be repeated and patients will receive up to 6 additional cycles of low-dose azacitidine, depending on risk assessment. For patients who meet criteria for high risk of relapse, azacitidine will be combined with escalating doses of DLI for a maximum of 7 cycles in total. Risk and safety assessments, including routine laboratory parameters, donor chimerism, minimal residual disease, and GHVD activity will be assessed following each cycle. Chimerism and minimal residual disease testing will be repeated every cycle by peripheral blood (PB), and bone marrow (BM) will be tested every other cycle. Patients will be followed by laboratory monitoring and physician evaluation prior to each cycle, and will be followed for two years post-transplant to study toxicity and GVHD outcomes.


Acute Myelogenous Leukemia Acute Lymphoid Leukemia Juvenile Myelomonocytic Leukemia Myelodysplastic Syndrome pediatric leukemia myelodysplastic syndrome azacitidine Azacitidine


You can join if…

Open to people ages up to 29 years

  • Patients age 0 - 29.9 years undergoing allogeneic peripheral blood stem cell transplant
  • Patients with acute myelogenous leukemia (AML) or acute lymphoblastic leukemia (ALL)
  • Patients with juvenile myelomonocytic leukemia (JMML)
  • Patients with myelodysplastic syndrome (MDS)

You CAN'T join if...

  • Patients who have had a prior transplant.
  • Patients with Fanconi anemia or other cancer-predisposition syndromes
  • Patients with expected survival <12 weeks
  • Lansky score <60%


  • Lucile Packard Children's Hospital not yet accepting patients
    Palo Alto, California, 94304, USA
  • Children's Hospital of Orange County not yet accepting patients
    Orange, California, 92868, USA


accepting new patients
Start Date
Completion Date
University of California, San Francisco
Phase 2
Lead Scientists
Justin Wahlstrom
Biljana Horn
Study Type
Last Updated
May 2016
I'm interested in this study!