Trial Outcomes & Findings for Age of Blood in Sickle Cell Transfusion (NCT NCT03704922)
NCT ID: NCT03704922
Last Updated: 2024-11-19
Results Overview
The investigators will compare the transfusions provided to the two groups (the proportion of biochemically old units when stored ≥30 days compared to when stored ≤10 days) using a Fisher exact test at an alpha of 0.05.
COMPLETED
PHASE2/PHASE3
26 participants
through third transfusion, an average of 18 weeks
2024-11-19
Participant Flow
Participant milestones
| Measure |
≤10 Day Blood
Subjects in this group will receive only blood stored ≤10 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
≥30 Day Blood
Subjects in this group will receive only blood stored ≥30 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
13
|
|
Overall Study
COMPLETED
|
10
|
10
|
|
Overall Study
NOT COMPLETED
|
3
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Age of Blood in Sickle Cell Transfusion
Baseline characteristics by cohort
| Measure |
≤10 Day Blood
n=13 Participants
Subjects in this group will receive only blood stored ≤10 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
≥30 Day Blood
n=13 Participants
Subjects in this group will receive only blood stored ≥30 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
Total
n=26 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
30.2 years
STANDARD_DEVIATION 8.5 • n=99 Participants
|
29.8 years
STANDARD_DEVIATION 11.2 • n=107 Participants
|
30.0 years
STANDARD_DEVIATION 9.7 • n=206 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
26 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 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
|
13 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
26 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 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
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
13 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
26 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: through third transfusion, an average of 18 weeksThe investigators will compare the transfusions provided to the two groups (the proportion of biochemically old units when stored ≥30 days compared to when stored ≤10 days) using a Fisher exact test at an alpha of 0.05.
Outcome measures
| Measure |
≤10 Day Blood
n=13 Participants
Subjects in this group will receive only blood stored ≤10 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
≥30 Day Blood
n=13 Participants
Subjects in this group will receive only blood stored ≥30 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
|---|---|---|
|
Proportion of Biochemically Old Red Cell Units
|
0.71 proportion biologically old units
|
0.86 proportion biologically old units
|
OTHER_PRE_SPECIFIED outcome
Timeframe: through third transfusion, an average of 18 weeksChange in CD62Lcirculating monocyte/macrophage mean fluorescence intensity (MFI) at 2 hours post-transfusion compared to the subject pre-transfusion. The investigators will compare the two groups using a two sample two-sided t-test of the log at an alpha of 0.05. Power: In addition, we will use a generalized linear model to include biochemically old units transfused,regardless of unit age, as a covariate. Other co-variates will include free heme, cell free hemoglobin, and non-transferrin bound iron (NTBI) from the transfused unit. We will similarly compare secondary activation endpoints: activation markers for neutrophils and measured cytokine concentrations. When possible, donor and recipient red cells will be differentiated in S-antigen negative patients who are by chance provided S-positive heterozygous or homozygous donor red cells.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: through fourth transfusion, an average of 24 weeksThe primary endpoint is the percentage of infections in adults who receive ≥30 day-old units as compared to ≤10 day-old units. The presence of an indwelling catheter will be used as a key co-variate in this analysis. The investigators will further explore the relationship of blood age and transfusion of biochemically old red cell units on the change in hemoglobin (Hb) and hemoglobin S (HbS)% over time, daily pain scores, opioid use and dose, emergency department (ED) and hospitalization rate, infection symptoms, new alloantibody formation, and antibiotic use during the 3-month study period. The investigators will compare groups using a Fisher exact test. Power: A difference of 20% will be of clinical interest. The investigators do not expect to have adequate power for this pilot study but at an alpha of 0.05 with 20 subjects in each group the investigators will be able to detect a difference of 47% between the proportions.
Outcome measures
Outcome data not reported
Adverse Events
≤10 Day Blood
≥30 Day Blood
Serious adverse events
| Measure |
≤10 Day Blood
n=13 participants at risk
Subjects in this group will receive only blood stored ≤10 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
≥30 Day Blood
n=13 participants at risk
Subjects in this group will receive only blood stored ≥30 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
|---|---|---|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
7.7%
1/13 • Number of events 2 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Infections and infestations
Sepsis
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
General disorders
Vaso-occlusive Pain
|
15.4%
2/13 • Number of events 5 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
23.1%
3/13 • Number of events 4 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Acute Chest Syndrome
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Skin and subcutaneous tissue disorders
Fistula Swelling
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
Other adverse events
| Measure |
≤10 Day Blood
n=13 participants at risk
Subjects in this group will receive only blood stored ≤10 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
≥30 Day Blood
n=13 participants at risk
Subjects in this group will receive only blood stored ≥30 days for 3 consecutive transfusion events.
Transfusion: Red cell units for transfusion
|
|---|---|---|
|
General disorders
Vaso-occlusive pain
|
15.4%
2/13 • Number of events 2 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
30.8%
4/13 • Number of events 25 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
General disorders
Penile pain
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
7.7%
1/13 • Number of events 8 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
General disorders
Injury due to Fall
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Skin and subcutaneous tissue disorders
Fistula Swelling
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
|
Infections and infestations
Influenza
|
7.7%
1/13 • Number of events 1 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
0.00%
0/13 • From the time of signing informed consent through study completion, a total of up to 24 weeks.
|
Additional Information
Matthew S. Karafin MD MS
University of North Carolina at Chapel Hill
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place