Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

• Status: Completed
• Trial end date: 2021-07-28
• Target enrollment: 0
• Participant gender: All

Summary

The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Criteria

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.