Busulfan and Cyclophosphamide Followed By ALLO BMT

• Sponsor: Masonic Cancer Center, University of Minnesota (Other)
• Overall Status: Terminated
• CT.gov ID: NCT01685411
• Collaborator: (none)
• Enrollment: 5
• Location: 1
• Arm: 1
• Duration (Months): 85.3
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

This is a treatment guideline to allow routine clinical data to be collected and maintained in Oncore (clinical database) and the University of Minnesota Blood and Marrow Database as part of the historical database maintained by the department.

Condition or Disease	Intervention/Treatment	Phase
Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Myelodysplastic Syndrome	Drug: Allopurinol; Drug: Keppra; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Biological: Allogeneic hematopoietic stem cell transplant; Biological: Filgrastim; Biological: antithymocyte globulin	N/A

Detailed Description

This is a single arm trial to evaluate the efficacy of busulfan and cyclophosphamide followed by an allogeneic hematopoietic stem cell transplant (HSCT) in the treatment of hematological malignancies. The intent of this study is to provide a protocol that will use unmanipulated allogeneic hematopoietic stem cells from related and unrelated donors after a common preparative regimen.

Study Design

• Study Type: Interventional
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Busulfan and Cyclophosphamide Followed By Allogeneic Hematopoietic Cell Transplantation In Patients With Hematological Malignancies
• Study Start Date: Jan 1, 2013
• Actual Primary Completion Date: Feb 10, 2020
• Actual Study Completion Date: Feb 10, 2020

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Allogeneic Hematopoietic Stem Cell Transplant Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.

Drug: Allopurinol; Day -8 (prior to transplant): Per institutional guidelines; Other Names: Lopurin Zyloprim ; Drug: Keppra; Day -8 (prior to transplant): Per institutional guidelines; Other Names: Levetiracetam ; Drug: Busulfan; Days -7 through -4 (prior to transplant): given intravenously (IV) infusion over 2 hours every 6 hours following dose, administration and pharmacokinetic monitoring per University of Minnesota institutional guidelines.; Other Names: Myleran ; Drug: Cyclophosphamide; Days -3 and -2 (prior to transplantation): given as a 2 hour intravenous infusion with a high volume fluid flush and mesna per institutional guidelines. Dosing is based on actual body weight.; Other Names: Cytoxan ; Drug: Tacrolimus; All patients (regardless of allograft source) will receive tacrolimus therapy beginning on day -3. Dosing will be monitored and altered as clinically appropriate per institutional pharmacy guidelines. Dose adjustments will be made on the basis of toxicity and/or low tacrolimus levels. Taper at day +100 for matched sibling donor (MSD) recipients, and day +180 for non-MSD recipients. Taper to zero by 10% weekly dose reduction over approximately 10 weeks.; Drug: Mycophenolate mofetil; Day -3 (prior to transplant): Recipients of umbilical cord blood will given a dose of 3 gm/day every 8 or 12 hours (> or = 40 kg) or 15 mg/kg 3 times per day (< 40 kg) for up to 30 days unless no engraftment.; Other Names: MMF ; Biological: Allogeneic hematopoietic stem cell transplant; Day 0 (or Day+1/+2 to accommodate weekdays): Infusion of cells from related or unrelated donor bone marrow or single or double unrelated donor umbilical cord blood.; Biological: Filgrastim; Beginning Day +1: Intravenously (IV) 5 mcg/kg once daily and continuing until the absolute neutrophil count is >2500 x 10^9/L or per institutional guidelines.; Other Names: G-CSF Granulocyte-colony stimulating factor ; Biological: antithymocyte globulin; Administered per institutional guidelines for recipients of umbilical cord blood transplant.; Other Names: ATG

Outcome Measures

Primary Outcome Measures

1. Counts of Participants With Disease Free Survival [2 Years]

   The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

2. Count of Participants With Disease Free Survival [5 Years]

   The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

3. Count of Participants With Disease Free Survival [7 Years]

   The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

Secondary Outcome Measures

1. Count of Participants Who Achieved Neutrophil Engraftment [By Day 42]

   Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater.

2. Percentage of Participants With Acute Graft-Versus-Host Disease by Grade [Day 100]

   Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

3. Percentage of Participants With Chronic Graft-Versus-Host Disease [6 Months]

   Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

4. Percentage of Participants With Chronic Graft-Versus-Host Disease [1 Year]

   Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

5. Percentage of Participants With Treatment-Related Toxicity [6 Months]

   In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

6. Percentage of Participants With Treatment-Related Toxicity [1 year]

   In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

7. Percentage of Participants With Relapse [1 Year]

   The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

8. Percentage of Participants With Relapse [2 Years]

   The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.

9. Percentage of Participants With Engraftment Failure [Day 42]

   Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.

10. Number of Participant Who Were Alive at 2 Years Post Transplant [2 Years]

    Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

11. Number of Participant Who Were Alive at 5 Years Post Transplant [5 Years]

    Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

12. Number of Participant Who Were Alive at 7 Years Post Transplant [7 Years]

    Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 44 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Diagnosis of acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and current in complete remission meeting one of the following:

• <45 years of age who are at least 6 months after initial hematopoietic stem cell transplant (HSCT)

• <45 years of age and have received sufficient radiation treatment to be ineligible for total body irradiation (TBI) containing preparative therapy

• Karnofsky performance status >70% or if <16 years of age, a Lansky play score >50

• Adequate major organ function including:

• cardiac: left ventricular ejection fraction >45% by echocardiogram (ECHO/MUGA)

• renal: creatinine clearance >40 mL/min/1.73m^2

• hepatic: no clinical evidence of hepatic failure (e.g., coagulopathy, ascites)

• An acceptable source of stem cells according to current University of Minnesota Bone

Marrow Transplant program guidelines. Acceptable stem cell sources include:

• HLA-matched related or unrelated donor bone marrow (6/6 or 5/6 antigen match)

• HLA-matched related or unrelated donor peripheral blood stem cells

• related or single or double unrelated donor umbilical cord blood (6/6, 5/6 or 4/6 match)

• Women of childbearing age must have a negative pregnancy test and all sexually active participants must agree to use effective contraception during study treatment

• Written consent (adult or parent/guardian)

Exclusion Criteria:

• eligible for TBI containing preparative regimen

• active uncontrolled infection within one week of study enrollment

• pregnant or lactating female

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Masonic Cancer Center, University of Minnesota	Minneapolis	Minnesota	United States	55455

Sponsors and Collaborators

• Masonic Cancer Center, University of Minnesota

Investigators

• Principal Investigator: Margaret L. MacMillan, M.D., Masonic Cancer Center, University of Minnesota

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Apr 19, 2018

More Information

Publications

None provided.

• Responsible Party: Masonic Cancer Center, University of Minnesota
• ClinicalTrials.gov Identifier: NCT01685411
• Other Study ID Numbers: 2011OC139; MT2011-20C
• First Posted: Sep 14, 2012
• Last Update Posted: Apr 13, 2021
• Last Verified: Mar 1, 2021

• Additional relevant MeSH terms: Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myelodysplastic Syndromes; Mycophenolic Acid; Cyclophosphamide; Busulfan; Allopurinol; Levetiracetam; Tacrolimus; Antilymphocyte Serum; Lenograstim

Study Results

Participant Flow

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Period Title: Overall Study
STARTED	5
COMPLETED	5
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion
Overall Participants	5
Age (Count of Participants)
<=18 years	5 100%
Between 18 and 65 years	0 0%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	2 40%
Male	3 60%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	5 100%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	5 100%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	5 100%

Outcome Measures

1. Primary Outcome

Title	Counts of Participants With Disease Free Survival
Description	The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.
Time Frame	2 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	3 60%

2. Primary Outcome

Title	Count of Participants With Disease Free Survival
Description	The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.
Time Frame	5 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	1 20%

3. Primary Outcome

Title	Count of Participants With Disease Free Survival
Description	The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.
Time Frame	7 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	1 20%

4. Secondary Outcome

Title	Count of Participants Who Achieved Neutrophil Engraftment
Description	Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater.
Time Frame	By Day 42

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	5 100%

5. Secondary Outcome

Title	Percentage of Participants With Acute Graft-Versus-Host Disease by Grade
Description	Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Time Frame	Day 100

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Grade 2-4	40 800%
Grade 3-4	20 400%

6. Secondary Outcome

Title	Percentage of Participants With Chronic Graft-Versus-Host Disease
Description	Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Time Frame	6 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	0 0%

7. Secondary Outcome

Title	Percentage of Participants With Chronic Graft-Versus-Host Disease
Description	Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Time Frame	1 Year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	0 0%

8. Secondary Outcome

Title	Percentage of Participants With Treatment-Related Toxicity
Description	In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
Time Frame	6 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	0 0%

9. Secondary Outcome

Title	Percentage of Participants With Treatment-Related Toxicity
Description	In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	0 0%

10. Secondary Outcome

Title	Percentage of Participants With Relapse
Description	The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.
Time Frame	1 Year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	40 800%

11. Secondary Outcome

Title	Percentage of Participants With Relapse
Description	The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated.
Time Frame	2 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	40 800%

12. Secondary Outcome

Title	Percentage of Participants With Engraftment Failure
Description	Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.
Time Frame	Day 42

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Number (95% Confidence Interval) [percentage of participants]	0 0%

13. Secondary Outcome

Title	Number of Participant Who Were Alive at 2 Years Post Transplant
Description	Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.
Time Frame	2 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	4 80%

14. Secondary Outcome

Title	Number of Participant Who Were Alive at 5 Years Post Transplant
Description	Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.
Time Frame	5 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	3 60%

15. Secondary Outcome

Title	Number of Participant Who Were Alive at 7 Years Post Transplant
Description	Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive.
Time Frame	7 Years

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
Measure Participants	5
Count of Participants [Participants]	1 20%

Adverse Events

Time Frame	7 years
Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Patients treated with Allopurinol, Keppra, Busulfan, Cyclophosphamide, Filgrastim, antithymocyte globulin, Tacrolimus, Mycophenolate mofetil and allogeneic hematopoietic stem cell transplant infusion.
All Cause Mortality
	Allogeneic Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	4/5 (80%)
Serious Adverse Events
	Allogeneic Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	0/5 (0%)
Other (Not Including Serious) Adverse Events
	Allogeneic Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	5/5 (100%)
Blood and lymphatic system disorders
CSA induced hypertension	2/5 (40%)	2
Transplant-associated thrombotic microangiopathy	1/5 (20%)	1
Cardiac disorders
Cardiac dysfunction	1/5 (20%)	1
Pericardial effusion	1/5 (20%)	1
Endocrine disorders
Adrenal insufficiency	1/5 (20%)	1
Eye disorders
Cataracts	1/5 (20%)	1
Gastrointestinal disorders
Ascites	1/5 (20%)	1
GI bleeding	1/5 (20%)	1
General disorders
Engraftment syndrome	1/5 (20%)	1
Infections and infestations
Bacterial infection	4/5 (80%)	26
Fungal infection	1/5 (20%)	3
Pneumonia	3/5 (60%)	6
Viral infection	2/5 (40%)	11
Metabolism and nutrition disorders
Hyperglycemia	1/5 (20%)	1
Musculoskeletal and connective tissue disorders
Osteopenia	1/5 (20%)	1
Nervous system disorders
Brain infarction	1/5 (20%)	1
Multifactorial delirium	1/5 (20%)	1
Neurotoxicity	1/5 (20%)	2
Seizure	1/5 (20%)	1
Renal and urinary disorders
Renal failure	1/5 (20%)	2
Respiratory, thoracic and mediastinal disorders
Veno-occlusive disease (VOD)	1/5 (20%)	1
Intubation	1/5 (20%)	3
Respiratory failure	1/5 (20%)	1
Shortness of breath	1/5 (20%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.