Clinical trial NCT03337568: Allogeneic HCT Using Uniform Conditioning Regimen Regardless of Donors (MS, MU, or HF) for AML in Remission
Official title: Allogeneic HCT From Donor-sources of Matched-sibling, Matched-unrelated, or Haploidentical-family Donors Using Uniform Conditioning Regimen of Busulfan, Fludarabine, and Antithymocyte Globulin for AML in Remission - an Observational Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Myeloid Leukemia
Keywords: AML CR
allogeneic hematopoietic cell transplantation
busulfan
fludarabine
ATG
Lead sponsor: Asan Medical Center
Sponsor class: OTHER
Collaborators: name: Korea Research Institute of Bioscience & Biotechnology
First posted: 2017-11-09
Enrollment: 110
Sex: ALL
Minimum age (years): 16
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This is a single center, observational study in a patient population with non-promyelocytic AML who have achieved complete remission after induction chemotherapy. The patients will be enrolled consecutively.

Brief summary: The purpose of this study is to evaluate the effect of various clinical variables including HLA-disparity and NK cell-related variables, upon outcomes of allogeneic hematopoietic cell transplantation (HCT) using uniform conditioning regimen including busulfan, fludarabine, and antithymocyte globulin (ATG) in patients with acute myeloid leukemia (AML) in the first complete remission (CR). The donors for allogeneic HCT include HLA-matched siblings, matched unrelated donors, and haploidentical family donors.

Therefore, the endpoints of the study are engraftment, secondary graft failure, acute and chronic graft-versus-host disease (GVHD), immune recovery, infections, leukemia recurrence, non-relapse mortality, and relapse-free (RFS) and overall survival (OS) of patients.


Eligibility criteria: Inclusion Criteria:

* Patients with non-promyelocytic AML (intermediate-risk or high-risk diseases by NCCN guideline 2016) in the first CR
* Patients should be 16 years of age or more and 75 years of age or less
* The performance status of the patients should be 70 or over by Karnofsky performance scale
* Patients should have adequate hepatic function (bilirubin less than 2.0 mg/dl, AST less than three times the upper normal limit)
* Patients should have adequate renal function (creatinine less than 2.0 mg/dl)
* Patients should have adequate cardiac function (ejection fraction \> 40% on MUGA scan)
* Patients and stem cell donors must sign informed consent
* For hematopoietic cell donor, if a patient has an HLA-matched sibling (65 years or younger), that sibling will be a cell donor. If a patient does not have an HLA-matched sibling but an HLA-A, B, C, DRB1 7-8/8 matched unrelated donor, the unrelated donor will be a cell donor. If a patient has neither HLA-matched sibling nor unrelated donor, an HLA-haploidentical familial donor will be a cell donor.

Primary outcomes:
measure: leukemia recurrence
timeFrame: from HCT (day of donor cell infusion) to leukemia recurrence at 2 years after HCT
measure: engraftment
timeFrame: from HCT to neutrophil count over >500/uL at 30 days after HCT
measure: GVHD, acute and chronic
timeFrame: from HCT to the occurrence of GVHD at 2 years after HCT
measure: Non-relapse mortality
timeFrame: from HCT to the occurrence of death without leukemia recurrence at 2 years after HCT
Secondary outcomes:
measure: relapse free survival
timeFrame: from HCT to last the follow-up, leukemia recurrence, or death at 2 years after HCT
measure: overall survival
timeFrame: from HCT to the last follow-up or death at 2 years after HCT
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
Study officials:
affiliation: University of Ulsan, Asan Medical Center
name: Kyoo-Hyung Lee, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Lee KH, Lee JH, Lee JH, Kim DY, Seol M, Lee YS, Kang YA, Jeon M, Hwang HJ, Jung AR, Kim SH, Yun SC, Shin HJ. Reduced-intensity conditioning therapy with busulfan, fludarabine, and antithymocyte globulin for HLA-haploidentical hematopoietic cell transplantation in acute leukemia and myelodysplastic syndrome. Blood. 2011 Sep 1;118(9):2609-17. doi: 10.1182/blood-2011-02-339838. Epub 2011 Jun 28.
citation: Lee KH, Lee JH, Lee JH, Kim DY, Park HS, Choi EJ, Ko SH, Seol M, Lee YS, Kang YA, Jeon M, Baek S, Kang YL, Kim SH, Yun SC, Kim H, Jo JC, Choi Y, Joo YD, Lim SN. Reduced-Intensity Conditioning with Busulfan, Fludarabine, and Antithymocyte Globulin for Hematopoietic Cell Transplantation from Unrelated or Haploidentical Family Donors in Patients with Acute Myeloid Leukemia in Remission. Biol Blood Marrow Transplant. 2017 Sep;23(9):1555-1566. doi: 10.1016/j.bbmt.2017.05.025. Epub 2017 May 25.