Trial Outcomes & Findings for ECMO Hemostatic Transfusions in Children (NCT NCT05796557)

NCT ID: NCT05796557

Last Updated: 2025-05-31

Results Overview

The total dose (in mL/kg/day) will be computed by the research team, by dividing the total platelet transfusion volume by the patient's weight at admission and the number of days of intervention.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 50 participants
• Primary outcome timeframe: up to day 21
• Results posted on: 2025-05-31

Participant Flow

Participant milestones

Measure	Higher Platelet Transfusion Strategy Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Overall Study STARTED	25	25
Overall Study COMPLETED	25	25
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

ECMO Hemostatic Transfusions in Children

Baseline characteristics by cohort

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.	Total n=50 Participants Total of all reporting groups
Age, Continuous	0.1 years n=5 Participants	0.2 years n=7 Participants	0.2 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	15 Participants n=7 Participants	28 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	10 Participants n=7 Participants	22 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	0 Participants n=7 Participants	0 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	7 Participants n=5 Participants	4 Participants n=7 Participants	11 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	18 Participants n=7 Participants	27 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants n=5 Participants	3 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=5 Participants	3 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants	20 Participants n=7 Participants	35 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Region of Enrollment United States	23 Participants n=5 Participants	24 Participants n=7 Participants	47 Participants n=5 Participants
Region of Enrollment Israel	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Cannulation Type Veno-venous	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Cannulation Type Veno-arterial	20 Participants n=5 Participants	24 Participants n=7 Participants	44 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to day 21

The total dose (in mL/kg/day) will be computed by the research team, by dividing the total platelet transfusion volume by the patient's weight at admission and the number of days of intervention.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Total Platelet Transfusion Dose	7.4 mL/kg/day Interval 0.0 to 16.6	0 mL/kg/day Interval 0.0 to 8.4

PRIMARY outcome

Timeframe: During intervention

Pre-transfusion platelet count, during intervention

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Pre-transfusion Platelet Count	79 10^9 cells/L Interval 70.0 to 86.0	43 10^9 cells/L Interval 39.0 to 47.0

SECONDARY outcome

Timeframe: At screening

Population: Number of eligible patients among the patients that were screened.

Feasibility will be assessed by the number of eligible participants that were screened.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=159 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Feasibility Assessed by the Screening Rate	123 Participants	—

SECONDARY outcome

Timeframe: At screening

Population: Number of eligible patients who were enrolled.

Feasibility will be assessed by the number of eligible participants that were enrolled.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=123 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Feasibility Assessed by the Inclusion Rate	50 participants	—

SECONDARY outcome

Timeframe: At screening

Population: Number of approached patients who consented.

Feasibility will be assessed by the number of participants that sign consent within the first 24 hours after ECMO cannulation.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=80 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Feasibility Assessed by the Number of Informed Consents Signed in the First 24 Hours Post Cannulation.	50 participants	—

SECONDARY outcome

Timeframe: up to Day 21

Population: Number of transfusion decisions (either to transfuse or not to transfuse, based on platelet count).

The proportion of transfusions that were given for platelet counts below the arm threshold will be computed to assess compliance.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=319 Transfusion Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=389 Transfusion Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Compliance With Transfusion Thresholds	316 Compliant transfusion	386 Compliant transfusion

SECONDARY outcome

Timeframe: up to Day 21

The number of participants who required at least one temporary suspension during ECMO.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Participants With at Least One Temporary Suspension	6 Participants	8 Participants

SECONDARY outcome

Timeframe: up to Day 21

The investigators will collect information on the duration of each suspension.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Duration for Temporary Suspensions	7.5 Hours Interval 1.5 to 19.6	13.5 Hours Interval 1.8 to 23.7

SECONDARY outcome

Timeframe: up to Day 21

The investigators will collect the proportion of participants who progress to a composite outcome of severe bleeding and/or severe thrombosis. The outcome will be adjudicated by an external review committee, blinded to the allocation arm.

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Progression to Composite Outcome of Severe Bleeding and/or Severe Thrombotic Event	4 Participants	5 Participants

SECONDARY outcome

Timeframe: Up to 90 days

Number of patients who withdraw from the study and/or are lost to follow-up (i.e., withdraw and/or are missing 90-day mortality assessment)

Outcome measures

Measure	Higher Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 Participants Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Number of Participants Who Were Withdrawn and/or Lost to Follow-up	2 Participants	2 Participants

Adverse Events

Higher Platelet Transfusion Strategy

Serious events: 4 serious events

Other events: 5 other events

Deaths: 1 deaths

Lower Platelet Transfusion Strategy

Serious events: 5 serious events

Other events: 5 other events

Deaths: 2 deaths

Serious adverse events

Measure	Higher Platelet Transfusion Strategy n=25 participants at risk Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 participants at risk Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Blood and lymphatic system disorders Bleeding	12.0% 3/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.	16.0% 4/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.
Blood and lymphatic system disorders Clotting	4.0% 1/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.	4.0% 1/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.

Other adverse events

Measure	Higher Platelet Transfusion Strategy n=25 participants at risk Participants randomized to this arm will be transfused if the platelet count is \< 90 x 10e9 cells/L.	Lower Platelet Transfusion Strategy n=25 participants at risk Participants randomized to this arm will be transfused if the platelet count is \< 50 x 10e9 cells/L.
Blood and lymphatic system disorders Bleeding	12.0% 3/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.	16.0% 4/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.
Blood and lymphatic system disorders Clotting	8.0% 2/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.	0.00% 0/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.
Surgical and medical procedures Accidental Removal of Vascular Access	0.00% 0/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.	4.0% 1/25 • From randomization up to 24 hours after the end of the intervention, up to 90 days Serious adverse events, as evaluated by medical monitor.

Additional Information

Oliver Karam

Yale

Phone: 203-785-4651

Email: oliver.karam@yale.edu

Results disclosure agreements

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