MT1999-20 Hematopoietic cell transplantation in children with juvenile myelomonocytic leukemia


To determine the probability of long-term disease-free survival in patients with JMML treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation.

I'm interested

up to 18 Years old
This study is NOT accepting healthy volunteers
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, ascites)
• 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.

Biological: Stem Cell Transplant, Drug: Preparative Regimen

Juvenile Myelomonocytic Leukemia

Stem cell transplant, long term survival, retinoic acid

Kim Nelson -
Margaret MacMillan, MD
Phase 1/Phase 2
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