Phase 2
Completed N=96
Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia
leukemia · Childhood Myelodysplastic Syndromes · Chronic myelomonocytic leukemia · de Novo Myelodysplastic Syndromes
Source: ClinicalTrials.gov NCT00860574 ↗
Enrolled (actual)
96
Serious AEs
17.7%
Results posted
Feb 2021
Primary outcomePrimary: Relapse Incidence — 18 Participants
Summary
This phase II trial is studying how well giving treosulfan together with fludarabine phosphate and total-body irradiation followed by donor stem cell transplant works in treating patients with high-risk acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia. Giving chemotherapy, such as treosulfan and fludarabine phosphate, and total-body irradiation before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells. It may also 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 before and after transplant may stop this from happening
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Relapse Incidence |
18 | — |
| PRIMARY Non Relapse Mortality (NRM) Incidence |
6 | — |
| SECONDARY Non Relapse Mortality Incidence |
6 | — |
| SECONDARY Overall Survival (OS) |
71 | — |
| SECONDARY Relapse-free Survival |
62 | — |
| SECONDARY Incidence of Grades II-IV Acute GVHD |
57 | — |
| SECONDARY Incidence of Chronic GVHD |
20 | — |
| SECONDARY Median Donor CD3 + T Lymphocyte Chimerism in Peripheral Blood |
100 | — |
Eligibility Criteria
Inclusion Criteria
- Acute myeloid leukemia (AML):
- All AML patients beyond 1st remission;
- Intermediate or high risk AML patients (based on South West Oncology Group [SWOG] cytogenetic criteria) in 1st complete remission
- Myelodysplastic syndrome (MDS)
- Other myeloid malignancies as chronic myelogenous leukemia (CML), CML accelerated phase, CML blast crisis, chronic myelomonocytic leukemia (CMML) (to be approved by patient care conference [PCC])
- With Karnofsky Index or Lansky Play-Performance Scale > 70% on pre-transplant evaluation
- Able to give informed consent (if > 18 years), or with a legal guardian capable of giving informed consent (if 90%
- Able to give informed consent (if > 18 years), or with a legal guardian capable of giving informed consent (if 2x upper normal limit or dialysis-dependent
- With hepatic dysfunction as evidenced by total bilirubin or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2.0 x upper normal limit or evidence of synthetic dysfunction or severe cirrhosis
- With active infectious disease requiring deferral of conditioning, as recommended by an Infectious Disease specialist
- With human immunodeficiency virus (HIV)-positivity or active infectious hepatitis because of possible risk of lethal infection when treated with immunosuppressive therapy
- With central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy and/or cranial radiation prior to initiating conditioning (day -6)
- With life expectancy severely limited by diseases other than malignancy
- Women who are pregnant or lactating because of possible risk to the fetus or infant
- With known hypersensitivity to treosulfan and/or fludarabine
- Receiving another experimental drug within 4 weeks before initiation of conditioning (day -6)
- Unable to give informed consent (if > 18 years) or with a legal guardian (if 18 years) or with a legal guardian (if < 18 years) unable to give informed consent
Data sourced from ClinicalTrials.gov (NCT00860574). 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.