Trial Outcomes & Findings for Gut Decontamination In Pediatric Allogeneic Hematopoietic (NCT NCT02641236)

Last Updated: 2023-09-15

Results Overview

Shannon diversity index (range: 0-6), measured at 2 weeks post stem cell transplant. Shannon diversity is a way to calculate biodiversity in a community, and assumes that all species are represented in a sample and that they are randomly sampled. Shannon Diversity is a measurement sensitive to the loss of rare taxa (34 in the JCI Insight manuscript, Konopinski MK, PeerJ. 2020;8:e9391) and estimates microbial richness (e.g., the number of species) and evenness (e.g., the relative abundance of organisms within a sample). Formula 1: H = -∑\[(pi) \* ln(pi)\], * H: Shannon diversity index (H = 0 indicates that a community has only one species) pi: Proportion of individuals of i-th species in a whole community Formula 2: pi = n / N, * n: individuals of a given type/species * N: total number of individuals in a community

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

2 Weeks post HSCT

Results posted on

2023-09-15

Participant Flow

Participant milestones

Participant milestones
Measure	Gut Decontamination With Vancopoly * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B starting on Day -5, relative to stem cell infusion (Day 0) and continue through neutrophil engraftment. Vancomycin-polymyxin B dosing is based off of body surface area (bsa): * Subjects with a bsa \< 0.5 m2 will take 1 capsule, administered orally (PO), three times a day (TID). * Subjects with a bsa of 0.5-0.99 m2 will take 2 capsules, PO, TID. * Subjects with a bsa of 1-1.49 m2 will take 3 capsules, PO, TID. * Subjects with a bsa \> or =1.5 m2 will take 4 capsules, PO, TID.	No Gut Decontamination * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.	Healthy Donor Control Group Eligible subjects will be registered to the study. Healthy donors will not receive any treatment on the study. Healthy donors will provide a stool sample around the time of the donor evaluation visit or donor bone marrow harvest. Additional stool samples may be collected at the 6 months post-transplant and 1 year post-transplant timepoints. Donor stool samples will be compared to their corresponding recipient's stool samples. Additionally, healthy donors will provide a one time peripheral blood sample at the time of the evaluation visit or donor bone marrow harvest. The volume of blood will not exceed 30ml or 3ml/kg, whichever is less.
Overall Study STARTED	10	10	4
Overall Study COMPLETED	5	3	4
Overall Study NOT COMPLETED	5	7	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Healthy donor control group did not receive transplant, so 'Age at Transplant' were not capable to be collected for this group.

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: 2 Weeks post HSCT

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Gut Microbiome Description	2.4 Shannon diversity index Interval 0.03 to 5.32	3.1 Shannon diversity index Interval 2.1 to 3.7

SECONDARY outcome

Timeframe: Participants were followed 7 days after HSCT.

The number of daily bowel movements during the first 7 days post-HSCT is charted by floor nurses and/or clinical assistants and will be obtained from within each patient's electronic medical record in PowerChart. Diarrhea frequency is defined the proportion of participants who had greater than 3 bowel movements per day.

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Diarrhea Frequency	0.4 proportion of participants Interval 0.15 to 0.7	0.4 proportion of participants Interval 0.15 to 0.7

SECONDARY outcome

Timeframe: Performed at the post-transplant time points (1,2,3,6,9,12 months post-transplant)

Population: Excluding two patients with graft failure.

CD4+ Tregs were defined as CD3+CD4+CD25med-hiCD127lo, CD4+; Tcon as CD3+CD4+CD25neg-lo CD127med-hi, B cells as CD19+, cytotoxic T cells as CD8+, and natural killer cells as CD56+CD3-. Within CD4+ Tregs and CD4+ Tcon, subsets were defined as follows: naive T cells (CD45RO-CD62L+), central memory (CD45RO+CD62L+), and effector memory (CD45RO+CD62L-).

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=9 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=9 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 1 month	80.95 number of cells per microliter Interval 3.72 to 195.49	119.78 number of cells per microliter Interval 5.99 to 725.56
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 2 months	99.89 number of cells per microliter Interval 59.01 to 190.76	81.92 number of cells per microliter Interval 8.31 to 285.83
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 3 months	126.54 number of cells per microliter Interval 23.72 to 246.35	182.09 number of cells per microliter Interval 9.97 to 312.63
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 6 months	206.50 number of cells per microliter Interval 123.18 to 675.64	248.24 number of cells per microliter Interval 44.29 to 739.08
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 9 months	230.28 number of cells per microliter Interval 161.54 to 565.65	335.43 number of cells per microliter Interval 37.39 to 458.98
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ at 12 months	380.22 number of cells per microliter Interval 200.19 to 459.49	429.53 number of cells per microliter Interval 6.74 to 524.61
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at 1 month	0.10 number of cells per microliter Interval 0.04 to 0.55	0.12 number of cells per microliter Interval 0.06 to 0.43
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at 2 months	0.10 number of cells per microliter Interval 0.02 to 0.43	0.12 number of cells per microliter Interval 0.01 to 0.14
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at at 3 months	0.091 number of cells per microliter Interval 0.021 to 0.255	0.098 number of cells per microliter Interval 0.013 to 0.13
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at 6 months	0.088 number of cells per microliter Interval 0.02 to 0.295	0.098 number of cells per microliter Interval 0.043 to 0.181
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at at 9 months	0.094 number of cells per microliter Interval 0.062 to 0.176	0.072 number of cells per microliter Interval 0.035 to 0.218
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry Treg/Tcon at 12 months	0.080 number of cells per microliter Interval 0.071 to 0.298	0.088 number of cells per microliter Interval 0.046 to 0.115
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 1 month	126.44 number of cells per microliter Interval 3.56 to 408.7	39.40 number of cells per microliter Interval 3.06 to 751.53
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 2 months	89.12 number of cells per microliter Interval 53.44 to 681.27	111.19 number of cells per microliter Interval 36.31 to 1191.26
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 3 months	401.16 number of cells per microliter Interval 20.64 to 1432.25	191.32 number of cells per microliter Interval 13.9 to 1158.49
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 6 months	331.87 number of cells per microliter Interval 63.76 to 820.64	270.11 number of cells per microliter Interval 93.85 to 2895.98
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 9 months	302.13 number of cells per microliter Interval 137.93 to 455.02	206.93 number of cells per microliter Interval 40.67 to 3131.85
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD8+ at 12 months	317.51 number of cells per microliter Interval 182.15 to 618.93	394.30 number of cells per microliter Interval 11.64 to 2919.11
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 1 month	0.08 number of cells per microliter Interval 0.0 to 0.44	0.04 number of cells per microliter Interval 0.01 to 0.57
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 2 months	0.17 number of cells per microliter Interval 0.04 to 0.35	0.02 number of cells per microliter Interval 0.003 to 0.18
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 3 months	0.09 number of cells per microliter Interval 0.02 to 0.42	0.05 number of cells per microliter Interval 0.02 to 0.26
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 6 months	0.11 number of cells per microliter Interval 0.04 to 0.7	0.11 number of cells per microliter Interval 0.002 to 0.33
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 9 months	0.32 number of cells per microliter Interval 0.09 to 0.86	0.05 number of cells per microliter Interval 0.02 to 0.57
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry CD4+ Tcon naive at 12 months	0.41 number of cells per microliter Interval 0.37 to 0.54	0.31 number of cells per microliter Interval 0.05 to 0.61
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 1 month	2.06 number of cells per microliter Interval 0.0 to 25.02	2.75 number of cells per microliter Interval 0.55 to 55.25
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 2 months	14.26 number of cells per microliter Interval 0.0 to 84.63	1.85 number of cells per microliter Interval 0.77 to 258.41
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 3 months	60.39 number of cells per microliter Interval 1.94 to 161.1	8.07 number of cells per microliter Interval 0.55 to 126.58
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 6 months	261.38 number of cells per microliter Interval 29.75 to 418.49	103.25 number of cells per microliter Interval 1.43 to 199.0
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 9 months	617.48 number of cells per microliter Interval 0.81 to 928.48	250.69 number of cells per microliter Interval 1.83 to 357.38
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry B-cells at 12 months	903.44 number of cells per microliter Interval 579.11 to 1195.48	223.92 number of cells per microliter Interval 0.04 to 460.35
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 1 month	46.92 number of cells per microliter Interval 5.39 to 406.13	79.89 number of cells per microliter Interval 23.28 to 1264.25
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 2 months	104.42 number of cells per microliter Interval 57.68 to 270.51	123.61 number of cells per microliter Interval 14.07 to 295.87
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 3 months	109.50 number of cells per microliter Interval 20.43 to 276.17	136.75 number of cells per microliter Interval 26.35 to 230.76
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 6 months	209.84 number of cells per microliter Interval 117.32 to 346.88	111.80 number of cells per microliter Interval 36.17 to 293.05
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 9 months	187.37 number of cells per microliter Interval 32.95 to 333.75	137.90 number of cells per microliter Interval 61.41 to 239.08
Median Absolute Cell Numbers of T-, B-, NK- and Dendritic Cell Subsets by Flow Cytometry NK cells at 12 months	193.19 number of cells per microliter Interval 37.87 to 338.91	100.42 number of cells per microliter Interval 9.86 to 215.03

SECONDARY outcome

Timeframe: Each stool collection time point after neutrophil engraftment until day +100

Grade 2 acute GVHD was defined as skin stage 3 or GI stage 1 or liver stage 1. Skin stage 3 was defined as maculopapular rash \>50% of body surface or generalized erythroderma; GI stage 1 was defined as adults: 500 - 1000 mL/day, children: 10 - 19.9 mL/kg/day or nausea, anorexia or vomiting with biopsy (EGD) confirmation of upper GI GVHD; liver stage 1 was defined as bilirubin 2.1-3 mg/dL. Grade 3 acute GVHD was defined as GI stage 2-4 or liver stage 2-3. GI stage 2-4 was defined as \>1001 mL/day (adults), \>30 ml/kg/day(children) or large volume stool with severe abdominal pain with our whiteout ileus or stool with frank blook or melena; liver stage 2-3 was defined as bilirubin 3.1-15mg/dL. Grade 4 acute GVHD was defined as skin stage 4 or liver stage 4. Skin stage 4 was defined as generalized erythroderma with bullous formation and desquamation; liver stage 4 was defined as bilirubin \> 15mg/dL.

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Incidence of Acute GVHD (Grade 2-4) Grade 2	0.10 proportion of participants Interval 0.003 to 0.45	0 proportion of participants Interval 0.0 to 0.31
Incidence of Acute GVHD (Grade 2-4) Grade 3-4	0 proportion of participants Interval 0.0 to 0.31	0.30 proportion of participants Interval 0.07 to 0.65

SECONDARY outcome

Timeframe: All participants were followed for 1 years after study entry.

OS12 is the proportion of participants alive at 12 months after study entry.

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Overall Survival Rate at 12 Months (OS12)	1 proportion of participants Interval 0.69 to 1.0	1 proportion of participants Interval 0.69 to 1.0

SECONDARY outcome

Timeframe: Patients were followed for 12 months.

Population: The analysis is comprised of participants with a malignancy.

Relapse free rate at 12 months was defined as the proportion of patients surviving without any signs or symptoms of that cancer after 12 months.

Outcome measures

Outcome measures
Measure	Gut Decontamination With Vancopoly n=7 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=8 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.
Relapse Free Rate at 12 Months	0.57 proportion of participants Interval 0.18 to 0.9	0.75 proportion of participants Interval 0.35 to 0.97

Adverse Events

Gut Decontamination With Vancopoly

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

No Gut Decontamination

Serious events: 3 serious events

Other events: 2 other events

Deaths: 0 deaths

Healthy Donor Control Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Gut Decontamination With Vancopoly n=10 participants at risk * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice.	No Gut Decontamination n=10 participants at risk * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.	Healthy Donor Control Group -Healthy individuals, ages 4 years old and older and toilet trained, who have been identified as 9/10 or 10/10 matched, bone marrow donors for transplantation. Healthy donors may be related or unrelated to the bone marrow recipient.
Blood and lymphatic system disorders Blood Bilirubin Increased	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Blood and lymphatic system disorders ALT Increase	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	20.0% 2/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Blood and lymphatic system disorders AST Increased	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Hepatobiliary disorders Hepatobiliary disorders	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Cardiac disorders Hypotension	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Blood and lymphatic system disorders Hypokalemia	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.
Blood and lymphatic system disorders GGT Increased	0.00% 0/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	10.0% 1/10 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.	— 0/0 • Adverse events were collected from the time of consent until the subject reached 24 months post-transplant or until they were taken off study, whichever came first. Subjects in the Healthy Donor Control Group were not monitored/ for Adverse Events. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with treatment attribution of possibly, probably or definitely and grade 3 or higher per CTCAEv4. All remaining events regardless of treatment attribution were classified as Other AEs. Per the source vocabulary (CTCAEv4.0) there is a category "Other, specify" for each system organ class. No further data is available to specify classification beyond this general term.

Other adverse events

Additional Information

Dr. Leslie Lehmann

Dana-Farber Cancer Institute

Phone: 617-632-4882

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Gut Decontamination With Vancopoly n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will receive non-absorbable, oral vancomycin-polymyxin B as per our institutional standard practice. Vancomycin-polymyxin B	No Gut Decontamination n=10 Participants * All eligible participants will be randomized to either Arm A: "Gut Decontamination" or Arm B: "No Gut Decontamination". * Participants assigned to this arm will not receive oral vancomycin-polymyxin B, but all other HSCT supportive care will be the same as for patients in Arm A.	Healthy Donor Control Group n=4 Participants -Healthy individuals, ages 4 years old and older and toilet trained, who have been identified as 9/10 or 10/10 matched, bone marrow donors for transplantation. Healthy donors may be related or unrelated to the bone marrow recipient.	Total n=24 Participants Total of all reporting groups
Age, Customized Age at transplant	13.4 years n=10 Participants • Healthy donor control group did not receive transplant, so 'Age at Transplant' were not capable to be collected for this group.	18.7 years n=10 Participants • Healthy donor control group did not receive transplant, so 'Age at Transplant' were not capable to be collected for this group.	—	15.2 years n=20 Participants • Healthy donor control group did not receive transplant, so 'Age at Transplant' were not capable to be collected for this group.
Sex: Female, Male Female	7 Participants n=10 Participants	3 Participants n=10 Participants	2 Participants n=4 Participants	12 Participants n=24 Participants
Sex: Female, Male Male	3 Participants n=10 Participants	7 Participants n=10 Participants	2 Participants n=4 Participants	12 Participants n=24 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=10 Participants	2 Participants n=10 Participants	0 Participants n=4 Participants	6 Participants n=24 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=10 Participants	8 Participants n=10 Participants	4 Participants n=4 Participants	17 Participants n=24 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	1 Participants n=24 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Asian	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Black or African American	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Race (NIH/OMB) White	10 Participants n=10 Participants	10 Participants n=10 Participants	4 Participants n=4 Participants	24 Participants n=24 Participants
Race (NIH/OMB) More than one race	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=4 Participants	0 Participants n=24 Participants
Primary disease Acute lymphocytic leukemia (ALL)	4 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	4 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	0 Participants Healthy Donor Control Group subjects do not have a Primary Disease.	8 Participants n=20 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.
Primary disease Acute myeloid leukemia (AML)	1 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	4 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	0 Participants Healthy Donor Control Group subjects do not have a Primary Disease.	5 Participants n=20 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.
Primary disease Myelodysplastic syndromes (MDS)	2 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	0 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	0 Participants Healthy Donor Control Group subjects do not have a Primary Disease.	2 Participants n=20 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.
Primary disease Anemia/red blood cell disorder	3 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	2 Participants n=10 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.	0 Participants Healthy Donor Control Group subjects do not have a Primary Disease.	5 Participants n=20 Participants • Healthy Donor Control Group subjects do not have a Primary Disease.
Conditioning regimen intensity Myeloablative	8 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	9 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	17 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Conditioning regimen intensity Nonmyeloablative	2 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	1 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	3 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Human leukocyte antigen (HLA) molecular typing Matched related	5 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	1 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	6 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Human leukocyte antigen (HLA) molecular typing Mismatch related	0 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	1 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	1 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Human leukocyte antigen (HLA) molecular typing Matched unrelated	2 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	5 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	7 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Human leukocyte antigen (HLA) molecular typing Mismatch unrelated	3 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	3 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	6 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Patient or donor serostatus Positive	5 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	8 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	13 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Patient or donor serostatus Negative	5 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	2 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	7 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Patient/donor sex mismatch F to M	2 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	2 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	4 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Patient/donor sex mismatch Other	8 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	8 Participants n=10 Participants • Data not collected from Healthy Donor Control Group.	0 Participants Data not collected from Healthy Donor Control Group.	16 Participants n=20 Participants • Data not collected from Healthy Donor Control Group.
Graft source Bone marrow	9 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	10 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	19 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Graft source Cord	1 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	1 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Graft Versus Host Disease (GVHD) prophylaxis Cyclosporine (CsA)	1 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	1 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Graft Versus Host Disease (GVHD) prophylaxis CsA/ Methotrexate (MTX) +/- methylprednisone	8 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	9 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	17 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.
Graft Versus Host Disease (GVHD) prophylaxis CsA/ Mycophenolate mofetil (MMF)	1 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	1 Participants n=10 Participants • Healthy Donor Control subjects did not receive transplants.	0 Participants Healthy Donor Control subjects did not receive transplants.	2 Participants n=20 Participants • Healthy Donor Control subjects did not receive transplants.