Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia in Remission

This study has been withdrawn prior to enrollment.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: January 7, 2005
Last updated: July 6, 2006
Last verified: July 2006

RATIONALE: A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving total-body irradiation together with fludarabine, thiotepa, and antithymocyte globulin before transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well a donor stem cell transplant works in treating patients with acute myeloid leukemia in remission.

Condition Intervention Phase
Adult Acute Erythroid Leukemia
Adult Acute Monoblastic and Acute Monocytic Leukemia
Adult Acute Myeloid Leukemia
Drug: anti-thymocyte globulin
Drug: fludarabine phosphate
Drug: thiotepa
Procedure: biological therapy
Procedure: bone marrow ablation with stem cell support
Procedure: chemotherapy
Procedure: non-specific immune-modulator therapy
Procedure: peripheral blood stem cell transplantation
Procedure: radiation therapy
Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Phase II Study of Haploidentical Allogeneic Peripheral Blood Stem Cell Transplantation in Patients With High-Risk Acute Myeloid Leukemia in First Remission

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Detailed Description:



  • Determine the safety and antileukemia activity of haploidentical allogeneic peripheral blood stem cell transplantation in patients with high-risk acute myeloid leukemia in first remission.


  • Determine the early treatment-related mortality (before day 100) of patients treated with this regimen.
  • Determine the incidence of acute graft-versus-host disease in patients treated with this regimen.
  • Determine the incidence of graft failure in patients treated with this regimen.
  • Correlate a mismatch in the expression of the natural killer cell inhibitory receptors CD158a and CD158b with engraftment and disease recurrence in patients treated with this regimen.

OUTLINE: This is a multicenter study.

Patients receive a preparative regimen comprising total-body irradiation twice on day –8; fludarabine IV over 30 minutes on days –7 to –3; thiotepa IV over 2 hours twice on day –7; and antithymocyte globulin IV over 4-6 hours on days –5 to –2. Patients undergo haploidentical allogeneic peripheral blood stem cell transplantation on day 0.

Patients are followed at day 100, at least monthly for 2 years, and then periodically for 3 years.

PROJECTED ACCRUAL: A total of 44 patients will be accrued for this study within 2.2 years.


Ages Eligible for Study:   18 Years to 59 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Morphologically confirmed acute myeloid leukemia of 1 of the following subtypes:
  • Acute myeloblastic leukemia (M0, M1, M2)
  • Acute myelomonocytic leukemia (M4)
  • Acute monocytic leukemia (M5)
  • Acute erythroleukemia (M6)
  • Acute megakaryocytic leukemia (M7)
  • Must have 1 of the following karyotypic abnormalities at the time of diagnosis:
  • Complex cytogenetic abnormalities (≥ 3 cytogenetic clones)
  • Abnormalities of chromosome 5 [-5 or del(5q)]
  • Abnormalities of the long (q) arm of chromosome 3, 9, 11, 20, or 21
  • Abnormalities of the short (p) arm of chromosome 17, monosomy 7, t(9;22), or t(6;9) (8)
  • In morphologic first complete remission*, as evidenced by all of the following for ≥ 4 weeks before study entry:
  • Absolute neutrophil count > 1,000/mm^3
  • Platelet count > 100,000/mm^3
  • Leukemic blasts not present in the peripheral blood
  • Cellularity of bone marrow biopsy > 20% with maturation of all cell lines
  • Less than 5% blasts by bone marrow biopsy
  • No extramedullary leukemia, such as CNS or soft tissue involvement NOTE: *Reduced hemoglobin concentration or hematocrit has no bearing on remission status
  • Haploidentical (3/6 or 4/6 antigen matched [A, B, and DR]) family donor available



  • 18 to 59

Performance status

  • ECOG 0-2

Life expectancy

  • Not specified


  • See Disease Characteristics


  • Bilirubin ≤ 2.0 mg/dL
  • AST < 2 times upper limit of normal


  • Creatinine ≤ 1.5 mg/dL


  • Ejection fraction > 40% by MUGA or echocardiogram
  • None of the following within the past 3 months:
  • Myocardial infarction
  • Significant congestive heart failure
  • Significant cardiac arrhythmia


  • FEV_1 and DLCO > 50% of predicted


  • HIV negative
  • No active or unresolved infection
  • No evidence of invasive fungal infection (e.g., positive blood or deep tissue cultures or stains)


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No organ damage
  • No other medical problem that would preclude study participation
  • No other currently active tumor that would likely interfere with study treatment or that would likely compromise the patient’s morbidity or mortality


Biologic therapy

  • No concurrent routine use of filgrastim (G-CSF) or sargramostim (GM-CSF) to accelerate hematopoietic recovery post-transplantation


  • More than 4 weeks since prior chemotherapy

Endocrine therapy

  • Not specified


  • More than 4 weeks since prior radiotherapy


  • Not specified
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00101140

Sponsors and Collaborators
Eastern Cooperative Oncology Group
Study Chair: Mark R. Litzow, MD Mayo Clinic
Investigator: Jacob M. Rowe, MD Rambam Health Care Campus
  More Information

No publications provided Identifier: NCT00101140     History of Changes
Other Study ID Numbers: CDR0000405838, ECOG-E1903
Study First Received: January 7, 2005
Last Updated: July 6, 2006
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
adult erythroleukemia (M6a)
adult acute megakaryoblastic leukemia (M7)
adult acute minimally differentiated myeloid leukemia (M0)
adult acute monocytic leukemia (M5b)
adult acute myeloblastic leukemia with maturation (M2)
adult acute myeloblastic leukemia without maturation (M1)
adult acute myeloid leukemia in remission
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with t(8;21)(q22;q22)
adult acute myelomonocytic leukemia (M4)

Additional relevant MeSH terms:
Leukemia, Erythroblastic, Acute
Leukemia, Monocytic, Acute
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Bone Marrow Diseases
Hematologic Diseases
Myeloproliferative Disorders
Neoplasms by Histologic Type
Antilymphocyte Serum
Fludarabine phosphate
Antimetabolites, Antineoplastic
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on November 25, 2014