Trial Outcomes & Findings for Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial (NCT NCT00072761)
NCT ID: NCT00072761
Last Updated: 2016-02-08
Results Overview
The primary end point was the recurrence of infarct or hemorrhage as determined by neuroimaging, clinical evidence of permanent neurologic injury, or both. A new infarct had to meet the criteria for a silent cerebral infarction; an enlarged silent cerebral infarct was defined as a previously identified silent cerebral infarct that increased by at least 3 mm along any linear dimension in any plane on MRI.
COMPLETED
PHASE3
196 participants
From study entry to study exit
2016-02-08
Participant Flow
Recruitment began in December, 2004 and ended May, 2010. Among the 1074 children screened with a MRI of the brain, 1.9% (20 of 1074) had strokes and 35.2% (379 of 1074) had infarct-like lesions. 196 participants completed all pre-randomization procedures and were successfully randomly allocated.
Participant milestones
| Measure |
Transfusion Group
The transfusion group received blood transfusion therapy every 4-6 weeks for 36 months.
|
Observation Group
The observation group received standard care therapy and quarterly physical examination by a study hematologist for 36 months.
|
|---|---|---|
|
Overall Study
STARTED
|
99
|
97
|
|
Overall Study
COMPLETED
|
90
|
95
|
|
Overall Study
NOT COMPLETED
|
9
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial
Baseline characteristics by cohort
| Measure |
Transfusion Group
n=99 Participants
The transfusion Group received blood transfusion therapy every 4-6 weeks for 36 months.
|
Observation Group
n=97 Participants
The observation group received standard care therapy and quarterly physical examination by a study hematologist for 36 months.
|
Total
n=196 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
99 Participants
n=99 Participants
|
97 Participants
n=107 Participants
|
196 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
10 years
n=99 Participants
|
10 years
n=107 Participants
|
10 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
40 Participants
n=99 Participants
|
45 Participants
n=107 Participants
|
85 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
59 Participants
n=99 Participants
|
52 Participants
n=107 Participants
|
111 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
91 Participants
n=99 Participants
|
90 Participants
n=107 Participants
|
181 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
13 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: From study entry to study exitPopulation: Randomization assignments were provided by the statistical data coordinating center with the use of a permuted block design, with stratification according to site, age, and sex. Participants were assigned in a 1:1 ratio to the observation or transfusion group and were followed until study exit or study endpoint.
The primary end point was the recurrence of infarct or hemorrhage as determined by neuroimaging, clinical evidence of permanent neurologic injury, or both. A new infarct had to meet the criteria for a silent cerebral infarction; an enlarged silent cerebral infarct was defined as a previously identified silent cerebral infarct that increased by at least 3 mm along any linear dimension in any plane on MRI.
Outcome measures
| Measure |
Transfusion Group
n=99 Participants
The transfusion Group received blood transfusion therapy every 4-6 weeks for 36 months.
|
Observation Group
n=97 Participants
The observation group received standard care therapy and quarterly physical examination by a study hematologist for 36 months.
|
|---|---|---|
|
Recurrence of an Infarct, Defined as a Stroke or a New or Enlarged Silent Cerebral Infarct
|
2.0 infarct recurrence per 100 person years
|
4.8 infarct recurrence per 100 person years
|
Adverse Events
Transfusion Group
Observation Group
Serious adverse events
| Measure |
Transfusion Group
n=99 participants at risk
The transfusion Group received blood transfusion therapy every 4-6 weeks for 36 months.
|
Observation Group
n=97 participants at risk
The observation Group received standard care therapy and quarterly physical examination by a study hematologist for 36 months.
|
|---|---|---|
|
General disorders
Stroke
|
1.0%
1/99
|
7.2%
7/97
|
|
General disorders
Silent Cerebral Infarct
|
5.1%
5/99
|
7.2%
7/97
|
|
General disorders
Transient Ischemic Attack
|
0.00%
0/99
|
2.1%
2/97
|
Other adverse events
| Measure |
Transfusion Group
n=99 participants at risk
The transfusion Group received blood transfusion therapy every 4-6 weeks for 36 months.
|
Observation Group
n=97 participants at risk
The observation Group received standard care therapy and quarterly physical examination by a study hematologist for 36 months.
|
|---|---|---|
|
General disorders
Vaso-Occlusive Pain
|
32.3%
32/99 • Number of events 126
|
57.7%
56/97 • Number of events 295
|
|
Respiratory, thoracic and mediastinal disorders
Acute Chest Syndrome
|
5.1%
5/99 • Number of events 5
|
24.7%
24/97 • Number of events 41
|
|
General disorders
Priapism
|
1.7%
1/59 • Number of events 1
|
13.5%
7/52 • Number of events 10
|
|
General disorders
Symptomatic avascular necrosis of the hip
|
1.0%
1/99 • Number of events 1
|
6.2%
6/97 • Number of events 6
|
|
General disorders
Headache
|
24.2%
24/99 • Number of events 76
|
30.9%
30/97 • Number of events 93
|
|
General disorders
Blood-transfusion reaction
|
16.7%
15/90 • Number of events 24
|
3.2%
1/31 • Number of events 1
|
|
General disorders
Ferritin > 1500 ng/ml
|
84.4%
76/90 • Number of events 1479
|
9.7%
3/31 • Number of events 33
|
Additional Information
Michael R. DeBaun, MD, MPH
Vanderbilt University School of Medicine
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place