Trial Outcomes & Findings for Donor Stem Cell Transplant or Donor White Blood Cell Infusions in Treating Patients With Hematologic Cancer (NCT NCT00281879)

Last Updated: 2017-09-27

Results Overview

Determine the effectiveness of unrelated donor allogeneic hematopoietic stem cells for transplantation after conditioning for the treatment of high-risk hematopoietic malignancies. Disease-free survival: 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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

200 participants

Primary outcome timeframe

Duration of the study; Up to 2 years

Results posted on

2017-09-27

Participant Flow

Participant milestones

Participant milestones
Measure	Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy.	Busulfan and Cyclophosphamide (Cytoxan) On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI).	BEAM Regimen On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI).	Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein.	ATG For Cord Blood Transplants If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion.	Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells.
Overall Study STARTED	128	36	20	6	9	1
Overall Study COMPLETED	0	0	0	0	0	0
Overall Study NOT COMPLETED	128	36	20	6	9	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Donor Stem Cell Transplant or Donor White Blood Cell Infusions in Treating Patients With Hematologic Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) n=128 Participants On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy.	Busulfan and Cyclophosphamide (Cytoxan) n=36 Participants On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI).	BEAM Regimen n=20 Participants On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI).	Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells.	Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) n=6 Participants On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein.	ATG For Cord Blood Transplants n=9 Participants If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion.	DLI (Donor Leukocyte Infusion) Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's.	Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) n=1 Participants On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells.	Total n=200 Participants Total of all reporting groups
Age, Categorical <=18 years	27 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	—	6 Participants n=36 Participants	7 Participants n=10 Participants	—	1 Participants n=40 Participants	42 Participants n=8 Participants
Age, Categorical Between 18 and 65 years	101 Participants n=93 Participants	35 Participants n=4 Participants	20 Participants n=27 Participants	—	0 Participants n=36 Participants	0 Participants n=10 Participants	—	0 Participants n=40 Participants	156 Participants n=8 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	—	0 Participants n=36 Participants	2 Participants n=10 Participants	—	0 Participants n=40 Participants	2 Participants n=8 Participants
Age, Continuous	32.033 years STANDARD_DEVIATION 14.923 • n=93 Participants	48.33 years STANDARD_DEVIATION 11.67 • n=4 Participants	46.187 years STANDARD_DEVIATION 12.87 • n=27 Participants	—	12.22 years STANDARD_DEVIATION 7.90 • n=36 Participants	11.77 years STANDARD_DEVIATION 10.725 • n=10 Participants	—	5.42 years STANDARD_DEVIATION 0 • n=40 Participants	34.743 years STANDARD_DEVIATION 16.818 • n=8 Participants
Sex: Female, Male Female	56 Participants n=93 Participants	13 Participants n=4 Participants	3 Participants n=27 Participants	—	4 Participants n=36 Participants	5 Participants n=10 Participants	—	1 Participants n=40 Participants	82 Participants n=8 Participants
Sex: Female, Male Male	72 Participants n=93 Participants	23 Participants n=4 Participants	17 Participants n=27 Participants	—	2 Participants n=36 Participants	4 Participants n=10 Participants	—	0 Participants n=40 Participants	118 Participants n=8 Participants
Region of Enrollment United States	128 participants n=93 Participants	36 participants n=4 Participants	20 participants n=27 Participants	—	6 participants n=36 Participants	9 participants n=10 Participants	—	1 participants n=40 Participants	200 participants n=8 Participants

PRIMARY outcome

Timeframe: Duration of the study; Up to 2 years

Outcome measures

Outcome data not reported

Adverse Events

Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI)

Serious events: 101 serious events

Other events: 19 other events

Deaths: 0 deaths

Busulfan and Cyclophosphamide (Cytoxan)

Serious events: 25 serious events

Other events: 0 other events

Deaths: 0 deaths

BEAM Regimen

Serious events: 17 serious events

Other events: 2 other events

Deaths: 0 deaths

Low-Dose Fludarabine and TBI(for Second Stem Cell Donation)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only)

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

ATG For Cord Blood Transplants

Serious events: 6 serious events

Other events: 0 other events

Deaths: 0 deaths

DLI (Donor Leukocyte Infusion)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only)

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) n=128 participants at risk On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy.	Busulfan and Cyclophosphamide (Cytoxan) n=36 participants at risk On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI).	BEAM Regimen n=20 participants at risk On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI).	Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells.	Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) n=6 participants at risk On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein.	ATG For Cord Blood Transplants n=9 participants at risk If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion.	DLI (Donor Leukocyte Infusion) Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's.	Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) n=1 participants at risk On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells.
Blood and lymphatic system disorders Staph Sepsis	2.3% 3/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Psychiatric disorders Altered mental status	0.78% 1/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pneumonia	3.9% 5/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Hypoxia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
General disorders Death	78.9% 101/128	69.4% 25/36	85.0% 17/20	— 0/0	83.3% 5/6	66.7% 6/9	— 0/0	100.0% 1/1 • Number of events 1
Immune system disorders Fever	0.00% 0/128	0.00% 0/36	5.0% 1/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Gastrointestinal disorders Graft versus host disease (GVHD)	13.3% 17/128	36.1% 13/36	15.0% 3/20	— 0/0	16.7% 1/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Acute Respiratory Distress Syndrome (ARDS)	2.3% 3/128	5.6% 2/36	10.0% 2/20	— 0/0	16.7% 1/6	0.00% 0/9	— 0/0	100.0% 1/1
Cardiac disorders Cardiac Problems	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Blood and lymphatic system disorders Thrombocytopenia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
General disorders Diarrhea	3.1% 4/128	5.6% 2/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Blood and lymphatic system disorders Orthostatic Hypertension	2.3% 3/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Renal and urinary disorders acute renal failure	0.78% 1/128	2.8% 1/36	5.0% 1/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	100.0% 1/1
Vascular disorders Intraparenchymal Hemorrhage	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pulmonary Hemorrhage	1.6% 2/128	0.00% 0/36	0.00% 0/20	— 0/0	16.7% 1/6	0.00% 0/9	— 0/0	0.00% 0/1
Immune system disorders Rigors	2.3% 3/128	5.6% 2/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Immune system disorders Pneutropenic Fever	2.3% 3/128	5.6% 2/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Musculoskeletal and connective tissue disorders Back pain	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Renal and urinary disorders Kidney Stones	0.00% 0/128	8.3% 3/36	10.0% 2/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Infections and infestations Encephalopathy	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Infections and infestations Viral encephalitis	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pulmonary aspergillus	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Musculoskeletal and connective tissue disorders Avascular necrosis	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Psychiatric disorders Anorexia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
General disorders Dehydration	2.3% 3/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Alveolar hemorrhage	11.7% 15/128	8.3% 3/36	10.0% 2/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Bronchopneumonia	3.1% 4/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Bacterial pneumonia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
General disorders Seizures	1.6% 2/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Cytomegalovirus pneumonitis	2.3% 3/128	0.00% 0/36	5.0% 1/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Cardiac disorders Tachycardia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Blood and lymphatic system disorders Positive blood cultures	1.6% 2/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Gastrointestinal disorders Gastroenteritis	1.6% 2/128	2.8% 1/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Hepatobiliary disorders Liver failure	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Hepatobiliary disorders Hyperbilirubinemia	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
Infections and infestations Fungal infection	8.6% 11/128	5.6% 2/36	10.0% 2/20	— 0/0	0.00% 0/6	11.1% 1/9	— 0/0	0.00% 0/1
Psychiatric disorders Steroid-induced psychoses	0.78% 1/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	11.1% 1/9	— 0/0	0.00% 0/1

Other adverse events

Other adverse events
Measure	Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) n=128 participants at risk On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy.	Busulfan and Cyclophosphamide (Cytoxan) n=36 participants at risk On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI).	BEAM Regimen n=20 participants at risk On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI).	Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells.	Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) n=6 participants at risk On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein.	ATG For Cord Blood Transplants n=9 participants at risk If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion.	DLI (Donor Leukocyte Infusion) Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's.	Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) n=1 participants at risk On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells.
General disorders Headache	6.2% 8/128	0.00% 0/36	10.0% 2/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1
General disorders Nausea	8.6% 11/128	0.00% 0/36	0.00% 0/20	— 0/0	0.00% 0/6	0.00% 0/9	— 0/0	0.00% 0/1

Additional Information

Dr. Richard Maziarz

OHSU Knight Cancer Institute

Phone: 503-494-1551

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place