The purpose of this research study is to look at overall health status and how acute myeloid leukemia (AML) responds to a stem cell transplant when followed with cyclophosphamide. Some participants enrolling in this study may receive a transplant from a sibling, some may receive a transplant from a matched unrelated donor, and some may receive what is called a haploidentical transplant. A haploidentical stem cell transplant is a type of transplant that occurs when a person who needs a transplant cannot find a donor who exactly matches their tissue type (either among family members or through a matched unrelated donor). When no matched donor is available, half-matched related (haploidentical) donors may be used. Haploidentical donors are first degree relatives such as siblings, children, or parents. People who undergo a stem cell transplant can experience complications such as rejection of the stem cell transplant or severe graft-versus-host disease (GVHD). GVHD occurs when some of the cells from the donor attack the recipient's tissues, resulting in mild, moderate, or even life-threatening side effects to the recipient's skin, stomach, intestines, and liver. However, recent research has shown that receiving cyclophosphamide after stem cell transplant can improve the outcomes of the transplant, and that is the purpose of this study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Washington University School of Medicine
St Louis, Missouri, United States
Event Free Survival (EFS) rate
The time from date of first dose of the preparative regimen until failure to engraft, treatment failure, disease progression/relapse, or death from any cause (whichever occurs first). Rates will be calculated with exact 95% confidence intervals.
Time frame: Assessed at 100 days post-transplant
Overall Survival (OS)
The time from the date of day 0 until death from any cause. Rates will be calculated with exact 95% confidence intervals.
Time frame: Assessed at 1 year post-transplant
Rate of Leukemia Free Survival (LFS)
The time from date of day 0 until disease progression or death from any cause (whichever occurs first). Rates will be calculated with exact 95% confidence intervals. For patients who achieve CR, CRc, or CRi only.
Time frame: Assessed at 100 days post-transplant
Transplant Related Mortality (TRM) Rate
Death from causes other than disease relapse or progression prior to Day +100 visit. Rates will be calculated with exact 95% confidence intervals.
Time frame: Assessed at 100 days post-transplant
Time to neutrophil engraftment
Defined as an untransfused platelet measurement \> 20,000/ x10\^9/L x 3 consecutive days. Kaplan-Meier models will be used to estimate median time to platelet engraftment and plot a time to event curve. Cox proportional hazards models may be used to explore the effect of covariates on time to platelet engraftment.
Time frame: Assessed up to day 30
Incidence of acute GVHD
The incidence and severity of acute GVHD will be determined. Rates will be calculated with exact 95% confidence intervals.
Time frame: Up to 100 days post-transplant
Incidence of chronic GVHD
The incidence and severity of chronic GVHD will be determined. Rates will be calculated with exact 95% confidence intervals.
Time frame: 1 year post-transplant starting at day +100
Time to platelet engraftment
Defined as an untransfused platelet measurement \> 20,000/ x10\^9/L x 3 consecutive days. Kaplan-Meier models will be used to estimate median time to platelet engraftment and plot a time to event curve. Cox proportional hazards models may be used to explore the effect of covariates on time to platelet engraftment.
Time frame: Assessed up to day 100
Rate of CRS with and without tocilizumab prophylaxis
To investigate the rate of cytokine release syndrome with and without tocilizumab prophylaxis in patients with active AML who undergo haploidentical transplantation
Time frame: Assessed up to day 7
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