Trial Outcomes & Findings for Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (NCT NCT00578292)

NCT ID: NCT00578292

Last Updated: 2020-05-01

Results Overview

Engraftment is defined as an absolute neutrophil count (ANC) \>500/microL x 3 days.

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 10 participants
• Primary outcome timeframe: up to 30 days
• Results posted on: 2020-05-01

Participant Flow

Participant milestones

Measure	Bone Marrow or Stem Cell Infusion Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Overall Study STARTED	10
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Bone Marrow or Stem Cell Infusion Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Overall Study Death	1
Overall Study Graft failure	2

Baseline Characteristics

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Baseline characteristics by cohort

Measure	Bone Marrow or Stem Cell Infusion n=10 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Age, Continuous	6 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	7 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 30 days

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) from unrelated donors.

Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=10 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Engraftment Rate After Transplant

1 proportion of participants Interval 0.69 to 1.0

PRIMARY outcome

Timeframe: 1 year post-transplant

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) from unrelated donors and had mixed chimerism (MC). Six participants were not evaluable at the time point of assessment for this outcome measure due to death or graft failure or full donor.

Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=4 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)

3 Participants

PRIMARY outcome

Timeframe: 1 year post-transplant

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) from unrelated donors and had mixed chimerism (MC). Six participants were not evaluable at the time point of assessment for this outcome measure due to death or graft failure or full donor.

Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=4 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)

0 Participants

PRIMARY outcome

Timeframe: up to day 100

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) from unrelated donors.

All AEs and SAEs (including infections) will be collected for evaluation of infectious complications.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=10 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Number of Participants With Infectious Complications

7 Participants

PRIMARY outcome

Timeframe: 1 year post-transplant

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) and were evaluable for the outcome measure. Two participants were not evaluated at the time point of assessment for this outcome measure due to death or graft failure.

Hematopoietic: defined as transfusion independence.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=8 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Hematopoietic Reconstitution

7 Participants

PRIMARY outcome

Timeframe: 1 year post-transplant

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) and were evaluable for the outcome measure. Two participants were not evaluated at the time point of assessment for this outcome measure due to death or graft failure.

Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=8 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Immune Reconstitution

8 Participants

PRIMARY outcome

Timeframe: Assessed weekly from Day 0 to day 100

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) and were evaluable for acute GVHD. A participant is evaluable for acute GVHD if he/she engrafted and either completed 100 days observation after transplant or experienced acute GVHD.

Acute GVHD is graded by the method of Przepiorka D. et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD

Outcome measures

Measure	Bone Marrow or Stem Cell Infusion n=10 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Number of Participants With ACUTE GVHD Grade 0	4 Participants
Number of Participants With ACUTE GVHD Grade I	4 Participants
Number of Participants With ACUTE GVHD Grade II-IV	2 Participants

PRIMARY outcome

Timeframe: Assessed monthly from month 3 to month 12

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) and were evaluable for chronic GVHD. A participant is evaluable for chronic GVHD if he/she engrafted and survived or remained in the study for more than 100 days after transplant.

Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=8 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Number of Participants With CHRONIC GVHD

1 Participants

PRIMARY outcome

Timeframe: up to 2 years post transplant

Population: The analysis included all participants who underwent allogeneic hematopoietic stem cell transplantation (SCT) from unrelated donors.

Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause.

Outcome measures

Measure

Bone Marrow or Stem Cell Infusion n=10 Participants Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2

Event-free Survival

0.7 probability of event-free survival Interval 0.33 to 0.89

Adverse Events

Bone Marrow or Stem Cell Infusion

Serious events: 4 serious events

Other events: 9 other events

Deaths: 1 deaths

Serious adverse events

Measure	Bone Marrow or Stem Cell Infusion n=10 participants at risk Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Infections and infestations Catheter related Infection	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Diarrhea	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Hematuria	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Aspartate aminotransferase increased	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Alanine aminotransferase increased	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.

Other adverse events

Measure	Bone Marrow or Stem Cell Infusion n=10 participants at risk Mesna, Cyclophosphamide, Busulfan, Fludarabine, Campath 1H Bone Marrow or Stem Cell infusion with pre-meds to take place on Day 0. Bone marrow dose/stem cell dose: To ensure the probability for bone marrow engraftment, 4 x 10e8 nucleated cells/kg patient weight or 5 x 10e6/kg of CD34+ cells/kg patient weight if the product is mobilized peripheral blood, will be the target to be obtained from the unrelated donor. Busulfan: 4.0 mg/kg/day divided into four doses daily for four days; total dose = 16 mg/kg Days -9 through -6 Fludarabine: 30mg/m2 Day -5 through Day -2 Campath 1H: Per institutional guidelines Days -5 through -2 Cyclophosphamide: 50 mg/kg Days -5 through -2 MESNA: 10 mg/kg x 5 Days -5 through -2
Gastrointestinal disorders Abdominal Pain	20.0% 2/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Acidosis	10.0% 1/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Alkalosis	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Anorexia	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Ascites	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Blood bilirubin increased	40.0% 4/10 • Number of events 6 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Cardiac disorders Cardiac disorders-Other: Cardiomegaly	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Catheter Related Infection	30.0% 3/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Diarrhea	20.0% 2/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Dysuria (painful urination)	30.0% 3/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Respiratory, thoracic and mediastinal disorders Epistaxis	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations GGT increased	50.0% 5/10 • Number of events 20 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Gastric ulcer	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Gastritis	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Psychiatric disorders Hallucinations	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Nervous system disorders Headache	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Hematuria	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Hemoglobinuria	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Cystitis noninfective	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Hepatobiliary disorders Hepatobiliary disorders-Other: GVHD	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Hepatobiliary disorders Hepatobiliary disorders-Other: hepatic VOD	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hyperkalemia	20.0% 2/10 • Number of events 4 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypermagnesemia	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Vascular disorders Hypertension	30.0% 3/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypertriglyceridemia	50.0% 5/10 • Number of events 11 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypoalbuminemia	60.0% 6/10 • Number of events 30 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypocalcemia	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypokalemia	30.0% 3/10 • Number of events 10 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypomagnesemia	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hyponatremia	30.0% 3/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Metabolism and nutrition disorders Hypophosphatemia	30.0% 3/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Respiratory, thoracic and mediastinal disorders Hypoxia	10.0% 1/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Infections and infestations - Other: Parainfluenza virus	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Infections and infestations - Other: CMV reactivation	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Infections and infestations - Other: Gram negative rods	20.0% 2/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Infections and infestations - Other: BK virus	30.0% 3/10 • Number of events 4 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Infections and infestations - Other: HSV	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Lipase	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Anal hemorrhage	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Nausea	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Nervous system disorders Nervous system disorders-Other: Altered mental status	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Eye disorders Eye disorders -Other: PRES	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
General disorders Pain	10.0% 1/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Partial thromboplastin time (PTT)	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
General disorders Pelvic Pain	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Respiratory, thoracic and mediastinal disorders Pleural Effusion	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Proteinuria	40.0% 4/10 • Number of events 10 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Prothrombin time (PT)	20.0% 2/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Skin and subcutaneous tissue disorders Rash	20.0% 2/10 • Number of events 3 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Renal Failure	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Renal and urinary disorders-Other: Nitrite in urine	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Aspartate aminotransferase increased	80.0% 8/10 • Number of events 31 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Investigations Alanine aminotransferase increased	60.0% 6/10 • Number of events 31 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Cardiac disorders Sinus tachycardia	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Stomatitis/Pharyngitis (oral/pharyngeal mucositis)	20.0% 2/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Renal and urinary disorders Urine discoloration	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Gastrointestinal disorders Vomiting	10.0% 1/10 • Number of events 2 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.
Infections and infestations Wound-Infectious	10.0% 1/10 • Number of events 1 • Primary BMT studies have a critical toxicity period of 30 days after BMT day 0. During the critical toxicity period the most severe grade of all AE's is captured. Grade 1 and 2 AEs, hematological toxicities, and fevers are excluded. Serious Adverse Events/Unanticipated Problems are reported (excluding hematological toxicities and fevers) until 100 days after BMT day 0. All adverse events were collected using CTCAE 2.0 for the study. For reporting purpose, adverse event terms were converted using CTCAE v4.0.

Additional Information

Dr. Tami D. John

Baylor College of Medicine/Texas Children's Hospital

Phone: 832-824-4723

Email: tdjohn@texaschildrens.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place