Trial Outcomes & Findings for Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies (NCT NCT00795132)

NCT ID: NCT00795132

Last Updated: 2013-07-30

Results Overview

Assessed donor engraftment in very high risk pediatric patients.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 47 participants
• Primary outcome timeframe: 24 months
• Results posted on: 2013-07-30

Participant Flow

Participant milestones

Measure	All Study Participants This group contains data from all groups (Related Bone marrow Peripheral Blood Stem Cell (BM PBSC), Unrelated Peripheral Blood Stem Cell (PBSC), and Unrelated Cord blood.
Overall Study STARTED	47
Overall Study COMPLETED	44
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	All Study Participants This group contains data from all groups (Related Bone marrow Peripheral Blood Stem Cell (BM PBSC), Unrelated Peripheral Blood Stem Cell (PBSC), and Unrelated Cord blood.
Overall Study Death	1
Overall Study Relapse	2

Baseline Characteristics

Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies

Baseline characteristics by cohort

Measure	All Study Participants n=47 Participants This group contains data from all groups (Related Bone marrow Peripheral Blood Stem Cell (BM PBSC), Unrelated Peripheral Blood Stem Cell (PBSC), and Unrelated Cord blood.
Age, Categorical <=18 years	35 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	13 years STANDARD_DEVIATION 9.899 • n=5 Participants
Sex: Female, Male Female	22 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants
Region of Enrollment United States	35 participants n=5 Participants
Region of Enrollment Canada	8 participants n=5 Participants
Region of Enrollment Australia	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 months

Population: All enrolled patients were analyzed except for the 3 patients who relapsed or died prior to engraftment.

Assessed donor engraftment in very high risk pediatric patients.

Outcome measures

Measure	Related BM PBSC n=16 Participants Related donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated BM PBSC n=18 Participants Unrelated donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated Cord Blood n=10 Participants Unrelated donor: Cord Blood
Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments	16 participants	18 participants	10 participants

SECONDARY outcome

Timeframe: 24 months

Population: All enrolled participants were analyzed.

The probability that a given patient will be alive two years after transplantation.

Outcome measures

Measure	Related BM PBSC n=16 Participants Related donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated BM PBSC n=19 Participants Unrelated donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated Cord Blood n=12 Participants Unrelated donor: Cord Blood
Two Year Overall Survival	0.501 probability Standard Error 0.141	0.395 probability Standard Error 0.120	0.438 probability Standard Error 0.165

SECONDARY outcome

Timeframe: 24 months

Population: All incidences of enrolled patients were analyzed.

Cumulative incidence transplantation-related mortality (TRM)

Outcome measures

Measure	Related BM PBSC n=16 Participants Related donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated BM PBSC n=19 Participants Unrelated donor: bone marrow or peripheral blood stem cell (PBSC)	Unrelated Cord Blood n=12 Participants Unrelated donor: Cord Blood
Number of Participants Who Experienced Transplantation-related Mortality (TRM)	0 participants	5 participants	1 participants

Adverse Events

All Study Participants

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Michael Pulsipher

University of Utah

Phone: 801-588-2680

Email: michael.pulsipher@hsc.utah.edu

Results disclosure agreements

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