Trial Outcomes & Findings for Donor Bone Marrow Transplant With or Without G-CSF in Treating Young Patients With Hematologic Cancer or Other Diseases (NCT NCT00450450)

Last Updated: 2022-04-28

Results Overview

EFS is defined as relapse or treatment-related mortality (TRM). relapse is defined by either morphological or cytogenetic evidence of ALL consistent with pre-transplant features.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

27 participants

Primary outcome timeframe

at 2 years

Results posted on

2022-04-28

Participant Flow

Participant milestones

Participant milestones
Measure	Control BM Arm Conventional bone marrow transplant (BM)	Experimental G-BM Arm G-CSF (filgrastim) stimulated bone marrow (G-BM)
Overall Study STARTED	13	14
Overall Study COMPLETED	8	11
Overall Study NOT COMPLETED	5	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Control BM Arm Conventional bone marrow transplant (BM)	Experimental G-BM Arm G-CSF (filgrastim) stimulated bone marrow (G-BM)
Overall Study Relapse	2	0
Overall Study Missing Data	2	0
Overall Study Death	1	3

Baseline Characteristics

Donor Bone Marrow Transplant With or Without G-CSF in Treating Young Patients With Hematologic Cancer or Other Diseases

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=14 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)	Total n=27 Participants Total of all reporting groups
Age, Continuous	12 Whole year n=5 Participants	11 Whole year n=7 Participants	12 Whole year n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	9 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	5 Participants n=7 Participants	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=5 Participants	11 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	12 Participants n=5 Participants	14 Participants n=7 Participants	26 Participants n=5 Participants
Region of Enrollment Australia	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: at 2 years

Population: Early terminated study. One patient is inevaluable for EFS on experimental arm and is excluded from analysis.

EFS is defined as relapse or treatment-related mortality (TRM). relapse is defined by either morphological or cytogenetic evidence of ALL consistent with pre-transplant features.

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated Two-year Event-free Survival (EFS)	66 Percentage of patients Interval 32.0 to 86.0	83 Percentage of patients Interval 47.0 to 96.0

SECONDARY outcome

Timeframe: Up to 10 years

Population: One patient is inevaluable for EFS on experimental arm and is excluded from analysis.

Primary graft failure is defined as the failure to achieve an absolute neutrophil count of more than 5000 per cubic millimeter for at least three consecutive days by Day +42.

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated Graft Failure Rate	0 Percentage of patients	0 Percentage of patients

SECONDARY outcome

Timeframe: Up to 3 months

Population: One patient is inevaluable and is excluded from analysis.

Stage III-IV aGVHD is defined as: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI; OR Stage 4 skin, liver or GI involvement.

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated Incidence of Grade III-IV Acute Graft-versus-host Disease (aGVHD)	7.69 Percentage of patients	15.38 Percentage of patients

SECONDARY outcome

Timeframe: 100 days

Population: One patient is inevaluable and is excluded from analysis.

Death in a patient after transplant due to protocol treatment is defined as an TRM.

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated 100-day Transplant Related Mortality (TRM) Percentage	0 percentage of patients	7.69 percentage of patients

SECONDARY outcome

Timeframe: 18 months post-transplant

Population: Included only patients survived beyond 100 days.

cGVHD definition is based on BMT CTN MOP SEPT. 2005; outlined in Protocol Appendix III.

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=12 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated Percentage of Chronic Graft-versus-host Disease (cGVHD)	7.69 percentage of patients	0 percentage of patients

SECONDARY outcome

Timeframe: Up to 10 years

Population: One patient was inevaluable and excluded from the analysis.

Median Time from transplant to neutrophil engraftment

Outcome measures

Outcome measures
Measure	Control BM Arm n=13 Participants Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 Participants G-CSF (filgrastim) stimulated bone marrow (G-BM)
Estimated Median Time to Neutrophil Engraftment	23 Days Interval 20.0 to 25.0	20 Days Interval 16.0 to 23.0

SECONDARY outcome

Timeframe: Up to 10 years

Population: Data regarding length of Initial Hospitalization are not collected for this study according to Study Chair.

Estimated and compared between randomization arms using the Wilcoxon rank-sum test.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 1 year

Population: Data are not collected for this study.

Summarized graphically. Generalized estimating equation will be used to model the levels as a function of time and randomization assignment and to test the impact of G-CSF stimulation on immune reconstruction.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 10 years

Population: Data are not collected for this study.

Compared using the Wilcoxon rank-sum test.

Outcome measures

Outcome data not reported

Adverse Events

Control BM Arm

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Experimental G-BM Arm

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Control BM Arm n=13 participants at risk Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 participants at risk G-CSF (filgrastim) stimulated bone marrow (G-BM)
Respiratory, thoracic and mediastinal disorders Dyspnea	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Respiratory, thoracic and mediastinal disorders Pneumonitis	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Respiratory, thoracic and mediastinal disorders Pneumothorax	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.

Other adverse events

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-447-0064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Must obtain prior Sponsor approval.
Publication restrictions are in place

Restriction type: OTHER

Measure	Control BM Arm n=13 participants at risk Conventional bone marrow transplant (BM)	Experimental G-BM Arm n=13 participants at risk G-CSF (filgrastim) stimulated bone marrow (G-BM)
Gastrointestinal disorders Abdominal pain	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Blood and lymphatic system disorders Anemia	30.8% 4/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Metabolism and nutrition disorders Anorexia	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Immune system disorders Autoimmune disorder	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Bladder infection	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Investigations Blood bilirubin increased	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
General disorders Death NOS	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
General disorders Edema face	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Renal and urinary disorders Hematuria	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Hepatobiliary disorders Hepatobiliary disorders - Other, specify	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Metabolism and nutrition disorders Hyperglycemia	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Metabolism and nutrition disorders Hypokalemia	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Metabolism and nutrition disorders Hyponatremia	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Infections and infestations - Other, specify	38.5% 5/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Lung infection	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Investigations Lymphocyte count decreased	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Gastrointestinal disorders Mucositis oral	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	23.1% 3/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
General disorders Multi-organ failure	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Gastrointestinal disorders Nausea	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Investigations Neutrophil count decreased	38.5% 5/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	23.1% 3/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Gastrointestinal disorders Oral pain	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Peripheral nerve infection	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Pharyngitis	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Investigations Platelet count decreased	38.5% 5/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	38.5% 5/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Gastrointestinal disorders Rectal mucositis	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Renal and urinary disorders Renal and urinary disorders - Other, specify	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Infections and infestations Skin infection	0.00% 0/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	7.7% 1/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.
Investigations White blood cell decreased	38.5% 5/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.	15.4% 2/13 Group 2 enrollment total is 14, patient 774353 enrolled on study, but was not randomized and didn't contribute any data. Group 2 AE total is 13.