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Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Phase 1/Phase 2
18 Years
Open (Enrolling)
Juvenile Myelomonocytic Leukemia

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Trial Information

Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six
times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two
days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide,
and melphalan are given to destroy the subject's leukemia. As well, these drugs will
destroy the subject's own immune system to help ensure the new bone marrow takes and grows
after transplantation.

On the day of transplantation, bone marrow or umbilical cord blood from the donor will
arrive to the bone marrow transplant unit and be transfused via venous line. These new
cells will replace the subject's bone marrow.

Inclusion Criteria:

- Patients must have a diagnosis of JMML and fulfill these minimal criteria
(International diagnostic criteria for JMML):

- Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)

- The presence of immature myeloid cells in the peripheral blood

- Less than 30% marrow blasts

- Absence of t(9:22) or BCR-ABL transcript

- Adequate major organ function including:

- Cardiac: ejection fraction > 45%

- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy,

- Karnofsky performance status > 70% or Lansky score > 50%

- Creatinine must be < 2 x normal for age

- Written informed consent.

Exclusion Criteria:

- Active uncontrolled infection within one week of HCT.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Determine probability of long-term disease free survival in JMML

Outcome Time Frame:

at 1 year after transplant

Safety Issue:


Principal Investigator

Margaret MacMillan, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota


United States: Institutional Review Board

Study ID:




Start Date:

December 1999

Completion Date:

May 2016

Related Keywords:

  • Juvenile Myelomonocytic Leukemia
  • Stem cell transplant
  • long term survival
  • retinoic acid
  • Leukemia
  • Leukemia, Myelomonocytic, Acute
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Juvenile



Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota  55455