Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Masonic Cancer Center, University of Minnesota15 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Juvenile Myelomonocytic Leukemia

Interventions

Stem Cell TransplantBIOLOGICAL

Transplantation on Day 0.

Preparative RegimenDRUG

* Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

Determine Probability of Long-term Disease Free Survival in JMML

Time frame: at 1 year after transplant

Secondary Outcomes

Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.

Measured by AGVHD Grades 2 - 4.

Time frame: at 1 year after transplant

Data from ClinicalTrials.gov

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