N/A
Completed N=52
Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome
leukemia · Childhood Myelodysplastic Syndromes · de Novo Myelodysplastic Syndromes · Essential Thrombocythemia
Source: ClinicalTrials.gov NCT00445744 ↗
Enrolled (actual)
52
Serious AEs
36.5%
Results posted
Jan 2018
Primary outcomePrimary: Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity — 8 Participants
Summary
This trial is studying the side effects and how well giving cyclophosphamide and busulfan followed by donor stem cell transplant works in treating patients with myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome. Giving chemotherapy, such as cyclophosphamide and busulfan, before a donor stem cell transplant helps stops the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and methotrexate after the transplant may stop this from happening
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity |
8 | — |
| PRIMARY Non-relapse Mortality (NRM) (Patients With AML/MDS) |
17 | — |
Eligibility Criteria
Inclusion Criteria
- Idiopathic myelofibrosis (CIMF)
- Myelofibrosis developing with polycythemia vera or essential thrombocythemia
- Acute myeloid leukemia with or without antecedent hematologic disorder, at any disease stage (complete remission, minimal residual disease, or relapsed leukemia)
- Myelodysplastic syndrome of any World Health Organization (WHO) or French-American-British (FAB) category, at any disease stage
- Less than 61 years of age if transplanted from an unrelated donor, or less than 66 years of age if transplanted from a related donor
- Receiving unmanipulated peripheral blood stem cells from an human leukocyte antigen (HLA)-identical or 1-allele-mismatched related or unrelated donor, or receiving G-CSF-stimulated bone marrow if co-enrolled on Fred Hutchinson Cancer Research Center (FHCRC) protocol 2250
- With a Karnofsky Performance score of > 70% at the time of pre-transplant evaluation
- Able to give informed consent (if >= 18 years of age), or with a legal guardian capable of giving consent (if 80%
- DONOR: Able to give informed consent (if >= 18 years of age), or with a legal guardian able to give informed consent (if 3)
- Women who are pregnant or lactating
- With known hypersensitivity to BU or CY
- With hepatic dysfunction as evidenced by total bilirubin or AST > 2x the upper limit of normal, or evidence of synthetic dysfunction or cirrhosis
- With impaired renal function, as evidenced by creatinine clearance 2x the upper limit of normal, or dialysis dependence
- With impaired pulmonary function, as evidenced by pO2 = 18 years of age), or without a legal guardian able to give informed consent (if <18 years of age)
Data sourced from ClinicalTrials.gov (NCT00445744). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.