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The Comparison of Transplantation From Family-mismatched/Haploidentical Donors With Matched Unrelated Donors in Adult Patients With Acute Myeloid Leukemia

Phase 3
17 Years
65 Years
Open (Enrolling)
Acute Myeloid Leukemia

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Trial Information

The Comparison of Transplantation From Family-mismatched/Haploidentical Donors With Matched Unrelated Donors in Adult Patients With Acute Myeloid Leukemia

For patients lacking an HLA-identical sibling, 8/8-matched unrelated donors are currently
the "gold standard" for a donor, since outcomes after HLA-identical sibling have been
compared to 8/8-matched unrelated donors. Currently, there are three alternative graft
sources, including mismatched unrelated donors, familial mismatch/haploidentical donors, and
umbilical cord bloods. Compared with other sources, transplants from familial
mismatch/haploidentical donors (FMT) have the benefit of an immediate availability of a
donor, particularly for those patients who urgently need transplantation. Initial reports
had characterized FMT to a poor engraftment and a high incidence of graft-versus-host
disease. However, outcomes of FMT have significantly improved over the past decade in the
optimization of conditioning regimen and graft selection to allow a stable engraftment
across major HLA barriers, with promising leukemia-free survival in adults with acute
leukemia. Despite the encouraging results and potential benefit of FMT, there have been few
studies comparing clinical outcomes of FMT with other donor types, particularly in acute
myeloid leukemia (AML) as a single disease. Since August 2008, we have been continuously
performing FMT using unmanipulated donor cells and a less aggressive conditioning regimen in
high-risk AML lacking an HLA-identical sibling, 8/8 or 7/8-matched unrelated donors. We
reported the feasibility of FMT using our novel reduced-intensity regimen without ex vivo
T-cell depletion, showing early results similar to outcomes of transplant from 8/8-matched
unrelated donors (MUT). This study will test the hypothesis that overall survival at 3 years
after FMT is similar to overall survival after MUT.

Inclusion Criteria

Inclusion Criteria

- Patients with AML aged from 18 to 65 years

- Eastern Cooperative Oncology Group (ECOG) performance < 2

- High risk group for relapse

1. Complete remission (CR) 1 with unfavorable prognostic factor; presenting white
blood cell > 100,000/microliter or prior myelodysplastic syndrome (MDS) or
myeloproliferative neoplasm (MPN) or MDS/MPN or cytogenetics & molecular
features (intermediate and adverse)

2. CR2 or CR3 at transplantation

- No HLA-matched sibling and unrelated donor (HLA-A, -B, -C, and -DRB1)

- Acceptable organ function defined as serum creatinine < 2 mg/dl, unless considered
due to leukemia and serum bilirubin < 3 mg/dl, unless considered due to leukemia

- Written informed consent form

Exclusion Criteria

- Active uncontrolled infections

- Corrected pulmonary diffusion capacity of <40%

- Cardiac ejection fraction of <35%

- ECOG performance status :2, 3, 4

- Active central nervous system involvement of disease

- Serological evidence of infection with HIV

- Pregnancy or breastfeeding

- Patient who are not suitable for the trial in accordance with principal
investigator's decision

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall survival

Outcome Description:

Overall survival is defined as the time interval between date of enrollment and death from any cause or for surviving patients, to last follow-up

Outcome Time Frame:

annually through 3 years

Safety Issue:


Principal Investigator

Hee-Je Kim, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, The Catholic University of Korea


Korea: Food and Drug Administration

Study ID:




Start Date:

January 2013

Completion Date:

December 2017

Related Keywords:

  • Acute Myeloid Leukemia
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid