Trial Outcomes & Findings for HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies (NCT NCT00145626)

NCT ID: NCT00145626

Last Updated: 2017-06-19

Results Overview

The one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a total body irradiation (TBI)-excluding conditioning regimen followed by an HLA-nonidentical family donor hematopoietic stem cell (HSC) graft depleted of T cells ex vivo using the CliniMACS CD34+ selection system, with a subsequent infusion of donor NK cells purified ex vivo using the CliniMACS CD3+ depletion and CD56+ enrichment system. The Kaplan-Meier estimate for one-year survival is reported.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 40 participants
• Primary outcome timeframe: One year after transplant
• Results posted on: 2017-06-19

Participant Flow

19 participants and 21 stem cell donors were enrolled between October 2006 and June 2011. The study was temporarily closed to accrual in June 2011 due to unavailability of study drug. The study was formally closed March 2015 because of continued unavailability of study drug. The 21 donors are excluded from this report.

19 stem cell recipients were enrolled, and 5 were excluded. Two participants did not have natural killer cell infusions due to donor was unable to donate enough CD34+ cells or CD56+ cells for infusion, 1 participant became ineligible because they turned 2 years old prior to start of therapy, 1 withdrew and 1 expired.

Participant milestones

Measure	Study Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days post-transplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.
Overall Study STARTED	19
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Measure	Study Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days post-transplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.
Overall Study Did not have NK infusions	2
Overall Study Turned 2 years old before treatment	1
Overall Study Death	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies

Baseline characteristics by cohort

Measure	Alive n=7 Participants Group of participants who survived to at least one year post HSCT. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Total n=14 Participants Total of all reporting groups
Age, Continuous	1.0 years n=5 Participants	0.8 years n=7 Participants	0.9 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Race/Ethnicity, Customized Black	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized White	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: One year after transplant

The one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a total body irradiation (TBI)-excluding conditioning regimen followed by an HLA-nonidentical family donor hematopoietic stem cell (HSC) graft depleted of T cells ex vivo using the CliniMACS CD34+ selection system, with a subsequent infusion of donor NK cells purified ex vivo using the CliniMACS CD3+ depletion and CD56+ enrichment system.

The Kaplan-Meier estimate for one-year survival is reported.

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired Those participants who did not survive to at least one year post HSCT.	Study Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
One-year Survival	50 percentage of participants	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: 100 days post-transplantation

The cumulative incidences of regimen-related mortality will be estimated using method of Kalbfleisch and Prentice.

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired Those participants who did not survive to at least one year post HSCT.	Study Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Number of Transplant-Related Adverse Outcomes: Regimen-Related Mortality	3 participants	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: 100 days post-transplantation

Engraftment failure is defined as <10% donor cell chimerism at any time point between 28 and 100 days after transplant with no evidence of disease relapse or requiring stem cell boost.

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired Those participants who did not survive to at least one year post HSCT.	Study Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Number of Transplant-Related Adverse Outcomes: Engraftment Failure	0.286 proportion of engraftment failures Interval 0.084 to 0.581	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: 100 days post-transplantation

The cumulative incidence estimate for occurrence of fatal acute GVHD by the end of the first 100 days post-transplant was calculated using method of Kalbfleisch and Prentice.

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired Those participants who did not survive to at least one year post HSCT.	Study Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Number of Transplant-Related Adverse Outcomes: Fatal Acute Graft-Versus Host Disease (GVHD)	0 Number of Deaths	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to 5 years after transplant

Chronic GVHD was graded according to Seattle Criteria: limited or extensive. Limited is defined as localized skin and/or hepatic dysfunction. Extensive is defined as one or more of the following:

• generalized skin involvement
• liver histology showing chronic aggressive hepatitis, bridging necrosis or cirrhosis
• eye dryness with Schirmer's test <5 mm wetting
• oral: involvement of salivary glands or oral mucosa
• other: another target organ involvement

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired Those participants who did not survive to at least one year post HSCT.	Study Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Number of Incidences of Chronic GVHD. Extensive chronic GVHD	0 Participants	—	—	—	—	—	—	—
Number of Incidences of Chronic GVHD. Limited chronic GVHD	1 Participants	—	—	—	—	—	—	—
Number of Incidences of Chronic GVHD. No chronic GHVHD	13 Participants	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Median Age of Donor at HSCT	21.5 Years Interval 20.1 to 36.3	27.2 Years Interval 19.4 to 34.9	25.73 Years Interval 19.4 to 36.3	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Median Dose of CD34	35.2 CD34 X 10^6/kg Interval 16.2 to 49.8	38.3 CD34 X 10^6/kg Interval 16.1 to 49.6	37.8 CD34 X 10^6/kg Interval 16.1 to 49.8	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Median Dose of NK Cells	40.2 NKcells X 10^6/kg Interval 10.4 to 102.5	37.6 NKcells X 10^6/kg Interval 9.8 to 74.1	38.9 NKcells X 10^6/kg Interval 9.8 to 102.5	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Disease Status at HSCT Active Disease	0 participants	1 participants	1 participants	—	—	—	—	—
Factors Affecting One-year Survival: Disease Status at HSCT Complete Remission-1	6 participants	2 participants	8 participants	—	—	—	—	—
Factors Affecting One-year Survival: Disease Status at HSCT Complete Remission-2	0 participants	1 participants	1 participants	—	—	—	—	—
Factors Affecting One-year Survival: Disease Status at HSCT Progressive Disease	1 participants	0 participants	1 participants	—	—	—	—	—
Factors Affecting One-year Survival: Disease Status at HSCT Relapse	0 participants	3 participants	3 participants	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Donor Type Father	2 participants	4 participants	6 participants	—	—	—	—	—
Factors Affecting One-year Survival: Donor Type Mother	5 participants	2 participants	7 participants	—	—	—	—	—
Factors Affecting One-year Survival: Donor Type Uncle	0 participants	1 participants	1 participants	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

HLA typing determined the degree of match by looking at 6 different HLA loci. The results indicate the number of the 6 loci that matched for each participant. Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model).

Outcome measures

Measure	Study Participants n=7 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=7 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Match N/6 HLA Loci 3/6 HLA Loci	6 participants	3 participants	9 participants	—	—	—	—	—
Factors Affecting One-year Survival: Match N/6 HLA Loci 4/6 HLA Loci	1 participants	4 participants	5 participants	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to one year after transplant

Population: Only four of the 14 participants had MRD measured at the one-year time point.

Detection of leukemia blasts in bone marrow by flow cytometry

Outcome measures

Measure	Study Participants n=3 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=1 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=4 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Factors Affecting One-year Survival: Minimal Residual Disease (MRD) Negative for MRD	2 participants	1 participants	3 participants	—	—	—	—	—
Factors Affecting One-year Survival: Minimal Residual Disease (MRD) Positive for MRD	1 participants	0 participants	1 participants	—	—	—	—	—

SECONDARY outcome

Timeframe: Up to 5 Years after transplant

Population: There was not enough data available after 1 year post-transplant to evaluate long term outcomes on this study.

The organ dysfunction will be summarized using summary statistics and assessed in a longitudinal manner and analyzed accordingly.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 5 Years after transplant

Population: There was not enough data available after 1 year post-transplant to evaluate long term outcomes on this study.

The long-term neurocognitive deficit will be summarized using summary statistics and assessed in a longitudinal manner and analyzed accordingly.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline before HSCT, 1 year post HSCT, and up to 5 years post HSCT

Population: MRD data was collected on only four participants during at least one time point.

The presence or absence of MRD before and after the bone marrow transplant (BMT) and its frequency distribution will be obtained for each time point separately.

Outcome measures

Measure	Study Participants n=4 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=4 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=4 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT Study participants as previously described.	1-2 Years After HSCT Study participants as previously described.	2-3 Years After HSCT Study participants as previously described.	3-4 Years After HSCT Study participants as previously described.	4-5 Years After HSCT Study participants as previously described.
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 1 · Negative MRD	1 Participants	1 Participants	1 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 1 · Positive MRD	0 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 1 · Data Not Collected	0 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 2 · Negative MRD	0 Participants	0 Participants	1 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 2 · Positive MRD	0 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 2 · Data Not Collected	1 Participants	1 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 3 · Negative MRD	1 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 3 · Positive MRD	0 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 3 · Data Not Collected	0 Participants	1 Participants	1 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 4 · Negative MRD	0 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 4 · Positive MRD	1 Participants	0 Participants	0 Participants	—	—	—	—	—
Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation Patient 4 · Data Not Collected	0 Participants	1 Participants	1 Participants	—	—	—	—	—

SECONDARY outcome

Timeframe: From 0-3 months after HSCT through 4-5 years after HSCT

Population: Data was not available for analysis for all patients at all time points.

The lymphohematopoietic reconstitution will be summarized using summary statistics and assessed in a longitudinal manner and analyzed accordingly.

Outcome measures

Measure	Study Participants n=14 Participants Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	Expired n=14 Participants Those participants who did not survive to at least one year post HSCT.	Study Participants n=14 Participants Fourteen study participants were evaluable for the outcome measures. Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days posttransplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.	9-12 Months After HSCT n=14 Participants Study participants as previously described.	1-2 Years After HSCT n=14 Participants Study participants as previously described.	2-3 Years After HSCT n=14 Participants Study participants as previously described.	3-4 Years After HSCT n=14 Participants Study participants as previously described.	4-5 Years After HSCT n=14 Participants Study participants as previously described.
Kinetics of Lymphohematopoietic Reconstitution CD3 Lymphocyte	0.22 cells *10^3/µl Interval 0.0 to 1.41	0.57 cells *10^3/µl Interval 0.34 to 0.86	1.15 cells *10^3/µl Interval 0.92 to 1.5	2.24 cells *10^3/µl Interval 0.34 to 2.5	1.65 cells *10^3/µl Interval 1.41 to 3.98	2.88 cells *10^3/µl Interval 2.0 to 3.76	2.65 cells *10^3/µl Interval 1.73 to 4.02	1.87 cells *10^3/µl Interval 1.75 to 3.02
Kinetics of Lymphohematopoietic Reconstitution CD3 Gamma Delta	0.00 cells *10^3/µl Interval 0.0 to 0.04	0.04 cells *10^3/µl Interval 0.04 to 0.04	0.08 cells *10^3/µl Interval 0.08 to 0.08	0.09 cells *10^3/µl Interval 0.09 to 0.09	0.70 cells *10^3/µl Interval 0.3 to 1.11	0.24 cells *10^3/µl Interval 0.11 to 0.36	0.21 cells *10^3/µl Interval 0.11 to 0.48	0.38 cells *10^3/µl Interval 0.14 to 0.43
Kinetics of Lymphohematopoietic Reconstitution CD4 Lymphocyte	0.13 cells *10^3/µl Interval 0.0 to 0.8	0.42 cells *10^3/µl Interval 0.27 to 0.53	0.92 cells *10^3/µl Interval 0.36 to 1.11	1.37 cells *10^3/µl Interval 0.17 to 2.17	0.96 cells *10^3/µl Interval 0.66 to 3.16	1.56 cells *10^3/µl Interval 0.78 to 2.33	1.33 cells *10^3/µl Interval 0.74 to 2.42	1.94 cells *10^3/µl Interval 0.77 to 77.0
Kinetics of Lymphohematopoietic Reconstitution CD8 Lymphocyte	0.01 cells *10^3/µl Interval 0.0 to 0.57	0.09 cells *10^3/µl Interval 0.01 to 0.43	0.29 cells *10^3/µl Interval 0.05 to 0.45	0.36 cells *10^3/µl Interval 0.08 to 0.86	0.61 cells *10^3/µl Interval 0.45 to 0.71	1.05 cells *10^3/µl Interval 0.83 to 1.28	1.10 cells *10^3/µl Interval 0.55 to 1.38	0.70 cells *10^3/µl Interval 0.58 to 1.04
Kinetics of Lymphohematopoietic Reconstitution CD19 Lymphocyte	0.26 cells *10^3/µl Interval 0.0 to 0.76	0.48 cells *10^3/µl Interval 0.04 to 0.66	0.61 cells *10^3/µl Interval 0.02 to 4.6	0.52 cells *10^3/µl Interval 0.0 to 1.06	0.45 cells *10^3/µl Interval 0.33 to 0.97	0.56 cells *10^3/µl Interval 0.39 to 0.73	0.56 cells *10^3/µl Interval 0.42 to 0.99	0.43 cells *10^3/µl Interval 0.34 to 0.77
Kinetics of Lymphohematopoietic Reconstitution CD56 Lymphocyte	0.36 cells *10^3/µl Interval 0.0 to 1.91	0.33 cells *10^3/µl Interval 0.09 to 0.82	0.20 cells *10^3/µl Interval 0.14 to 0.38	0.19 cells *10^3/µl Interval 0.08 to 2.75	0.22 cells *10^3/µl Interval 0.07 to 0.98	0.32 cells *10^3/µl Interval 0.21 to 0.43	0.34 cells *10^3/µl Interval 0.13 to 0.89	0.19 cells *10^3/µl Interval 0.17 to 0.72
Kinetics of Lymphohematopoietic Reconstitution CD4/CD8 Ratio	2.60 cells *10^3/µl Interval 0.0 to 34.0	5.53 cells *10^3/µl Interval 0.63 to 71.5	3.36 cells *10^3/µl Interval 0.8 to 28.28	1.90 cells *10^3/µl Interval 1.9 to 8.1	1.88 cells *10^3/µl Interval 0.82 to 4.4	1.38 cells *10^3/µl Interval 0.9 to 1.85	1.30 cells *10^3/µl Interval 1.2 to 1.8	1.30 cells *10^3/µl Interval 1.1 to 1.9
Kinetics of Lymphohematopoietic Reconstitution Absolute Lymphocyte Value	0.88 cells *10^3/µl Interval 0.0 to 4.1	1.29 cells *10^3/µl Interval 0.4 to 2.37	1.95 cells *10^3/µl Interval 1.32 to 2.58	2.90 cells *10^3/µl Interval 0.53 to 6.05	2.59 cells *10^3/µl Interval 1.8 to 5.1	3.76 cells *10^3/µl Interval 2.6 to 4.93	3.65 cells *10^3/µl Interval 2.4 to 5.5	2.40 cells *10^3/µl Interval 2.4 to 4.5

Adverse Events

Study Participants

Serious events: 15 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Measure	Study Participants n=16 participants at risk Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days post-transplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.
Blood and lymphatic system disorders Graft Failure	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Graft Rejection	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Leukocytosis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Post-Transplant Lymphoproliferative Disease	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Cardiac Tamponade	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Fever without Neutropenia	56.2% 9/16 • Number of events 21 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Colitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hemorrhage, Brain	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Hyperbilirubinemia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Veno-Occlusive Disease, Hepatic	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Febrile Neutropenia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Fever Without Neutropenia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Aspergillus, Central Nervous System	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Methicillin Resistant Staphylococcus Aureus	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, RSV, Pneumonitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Staphylococcus Aureus, Blood	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Staphylococcus Epidermidis, Blood	6.2% 1/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Stenotrophomonas, Blood	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Metabolism and nutrition disorders Hypernatremia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Nervous system disorders Seizure	12.5% 2/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Hypoxia	18.8% 3/16 • Number of events 4 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Respiratory Distress	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Renal and urinary disorders Failure, Renal	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Engraftment Syndrome	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Gastrointestinal Perforation	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.

Other adverse events

Measure	Study Participants n=16 participants at risk Study participants received a non-TBI based preparative regimen consisting of Cyclophosphamide, fludarabine, thiotepa, melphalan, and muromonab-CD3 (OKT3) followed by an infusion of a T-lymphocyte depleted haploidentical hematopoietic stem cell graft. Seven days post-transplant, participants received an infusion of additional donor derived cells called natural killer (NK) cells. Stem cells were obtained from donors using the Miltenyi Biotec CliniMACS stem cell selection device.
Skin and subcutaneous tissue disorders Erythema, Perianal	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Hyperpigmentation, Peri-Rectal	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Petechiae, Eyes	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Allergic Drug Reaction, Defibrotide	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Allergic Drug Reaction, OKT3	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Allergic Drug Reaction, Thiotepa	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Allergic Reaction, Pure Red Blood Cells	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Allergic Reaction, Platelet Transfusion	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Immune system disorders Rash, Generalized	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Eosinophilia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Prolonged QTc	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Heart block (incomplete right bundle branch block)	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Tachycardia	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema of hand	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema, Anasarca	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema, Facial	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema, Feet	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema, Generalized	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Edema, Lower Extremity	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Hypertension	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Hypotension	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Murmur	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Cardiac disorders Pulmonary Edema	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Coagulopathy	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Coagulopathy, Elevated Bleeding Times	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Fever Without Neutrophenia	37.5% 6/16 • Number of events 8 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Weight Loss	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Abscess, Longissimus Colli, left	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Erythema, Face	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Erythema, Genitourinary	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Erythema, Labia, Perianal Region	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Erythema, Neck	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Diaper	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Face	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Generalized	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Labia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Lower Left Leg	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Palms and Feet	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Perineum	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Scaly, Non-Erythematous	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Rash, Thorax	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Skin Lesion	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Skin and subcutaneous tissue disorders Toxic Epidermal Necrolysis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Colitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Diarrhea	18.8% 3/16 • Number of events 4 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Hemorrhage, Gastrointestinal, Oral	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Loss of Appetite	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Mucositis	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Nausea	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Stomatitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Gastrointestinal disorders Vomiting	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Bilateral Subdural hematomas	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Epistaxis	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Gastrointestinal Hemorrhage	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hematemesis	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hematoma, Scalp	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hematuria	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hemorrhage, Biopsy Site	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hemorrhage, Secretions from Endotracheal Tube, Respiratory Tract	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Hemorrhage, Vasocath Site	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Petechiae, Lower Extremity	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Failure, Hepatic	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Hepatomegaly	18.8% 3/16 • Number of events 3 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Hypoalbuminemia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Splenomegaly	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Hepatobiliary disorders Veno-Occlusive Disease, Hepatic	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Febrile Neutropenia	62.5% 10/16 • Number of events 12 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Fever Without Neutropenia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection With Neutropenia, Klebsiella and Escherichia Coli, Blood	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection Without Neutropenia, Enterococcus Faecalis, Blood	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection With Neutropenia, Gamma Hemolytic Streptococcus, Skin	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Adenovirus, Stool	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Aspergillus Terreus, Lungs	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Aspergillus, Retina	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Candida, Diaper Area with Rash	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Candidiasis, Mouth	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Coagulase Negative Staph, Blood	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Coagulase Negative Staphylococcus, Wrist	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Influenza Virus A	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Klebsiella Pneumoniae, Hickman Line	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Legionella, Mucous	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Pantoea Species, Blood	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Parainfluenza Virus 3	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Pseudomonas Aeruginosa, Blood	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Staphylococcus Aureus, Skin	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Stomatococcus Mucilaginosus, Blood	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Stool, Vancomycin Resistant Enterococci	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Infections and infestations Infection, Streptococcus Mitis, Hickman Line	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Blood and lymphatic system disorders Splenic Infarction	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Metabolism and nutrition disorders Hypernatremia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Metabolism and nutrition disorders Hyperuricemia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Metabolism and nutrition disorders Hypophosphatemia	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Nervous system disorders Meningoencephalitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Nervous system disorders Mood Alteration - Anxiety/Agitation	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Eye disorders Exposure Keratitis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Eye disorders Glaucoma, Right Eye	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Eye disorders Hyphema, Right Eye	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Pain, Abdominal	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Pain, Generalized, Multiple Sites	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Pain, Gums	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Pain, Throat	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Atelectasis	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Cough	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Hemorrhage, Pulmonary	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Interstitial Lung Disease	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Nodule, Pulmonary	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Pulmonary Airspace Disease, Diffuse, Bilateral	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Respiratory Distress	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Respiratory, thoracic and mediastinal disorders Tachypnea	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Renal and urinary disorders Renal Disease	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Renal and urinary disorders Renal Failure	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Capillary Leak Syndrome	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Engraftment Syndrome	12.5% 2/16 • Number of events 2 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Abdominal Compartment Syndrome	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
Vascular disorders Systemic Inflammatory Response Syndrome	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.
General disorders Cytokine Release Syndrome, Rituximab	6.2% 1/16 • Number of events 1 • Adverse events are reported from the on-study date through April 2015 (up to one year post-transplant). All 16 patients who were enrolled and proceeded to transplant are included.

Additional Information

Brandon Triplett, MD

St. Jude Children's Research Hospital

Phone: 866-278-5833

Email: referralinfo@stjude.org

Results disclosure agreements

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