Trial Outcomes & Findings for Management of Platelet Transfusion Therapy in Patients With Blood Cancer or Treatment-Induced Thrombocytopenia (NCT NCT03195010)

NCT ID: NCT03195010

Last Updated: 2019-10-21

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Up to 1 year
• Results posted on: 2019-10-21

Participant Flow

Participant milestones

Measure	Group I (Lower Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 30 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo lower dose platelet transfusion	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Overall Study STARTED	2	2
Overall Study COMPLETED	2	2
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Management of Platelet Transfusion Therapy in Patients With Blood Cancer or Treatment-Induced Thrombocytopenia

Baseline characteristics by cohort

Measure	Group I (Lower Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 30 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo lower dose platelet transfusion	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion	Total n=4 Participants Total of all reporting groups
Age, Continuous	42 years n=5 Participants	35 years n=7 Participants	39 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 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	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 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	2 Participants n=7 Participants	4 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 1 year

Outcome measures

Measure	Approached Patients n=4 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Eligible Patients Approached for the Study	4 Participants	—

PRIMARY outcome

Timeframe: Up to 1 year

Reasons for ineligibility will be reported qualitatively in order to inform future studies.

Outcome measures

Measure	Approached Patients n=4 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Patients Approached for But Refusing Consent	0 Participants	—

PRIMARY outcome

Timeframe: Up to 1 year

Outcome measures

Measure	Approached Patients n=4 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Patients Consenting to Enrollment	4 Participants	—

PRIMARY outcome

Timeframe: Up to 1 year

Outcome measures

Measure	Approached Patients n=7 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Patients Eligible	4 Participants	—

PRIMARY outcome

Timeframe: Up to 1 year

Reasons for ineligibility will be reported qualitatively in order to inform future studies.

Outcome measures

Measure	Approached Patients n=7 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Patients Screened and Deemed Ineligible	3 Participants	—

PRIMARY outcome

Timeframe: Up to 1 year

Will evaluate the number of patients successfully following protocol, defined as receiving transfusions 'on protocol' at the end of the study period.

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Patients Successfully Following Protocol	2 Participants	2 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Will evaluate the incidence of hemorrhagic events (World Health Organization grade 2 or greater).

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Incidence of Hemorrhagic Events (World Health Organization Grade 2 or Greater)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Will evaluate the major bleeds (World Health Organization grade 3 or 4).

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Major Bleeds (World Health Organization Grade 3 or 4)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Number of Platelet Transfusions Per Patient During the Study Period	2 platelet transfusions Interval 2.0 to 2.0	9 platelet transfusions Interval 5.0 to 13.0

SECONDARY outcome

Timeframe: Up to 1 year

The frequency with which transfusions are given despite a platelet count above the determined threshold will be documented, as will the frequency with which transfusions are not administered within 24 hours after a platelet count below the determined threshold.

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Percent of Days on Which Subjects Are Transfused (or Transfusion Are Not Given)	0 percentage of study days	3.8 percentage of study days

SECONDARY outcome

Timeframe: Up to 1 year

Total number of transfusion reactions, patients experiencing alloimmunization and volume overload will be reported.

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Platelet Transfusion Related Complications	0 Platelet transfusion complication	2 Platelet transfusion complication

SECONDARY outcome

Timeframe: Up to 1 year

Will evaluate the progression or new arterial thromboembolism by either documented acute electrocardiographic changes compatible with myocardial injury and/or serum biochemical changes diagnostic of myocardial infarction, or documented imaging (computed tomography or magnetic resonance imaging) changes compatible with infarct due to embolism in the presence of a new neurological deficit, or imaging demonstrated intraluminal filling defects in an arterial distribution accompanied by symptoms of acute ischemia (acute onset pain, pallor, loss of pulses or other end-organ damage).

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Progressive or New Arterial Thromboembolism	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 1 year

Will evaluate the progressive or new venous thromboembolic. Will require imaging confirmation, defined as intraluminal filling defect(s) on contrast-enhanced computed tomography or incompressible venous segment(s) on ultrasonography.

Outcome measures

Measure	Approached Patients n=2 Participants Eligible patients approached for participation	Group II (Higher Dose Platelet Transfusion) n=2 Participants Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Progressive or New Venous Thromboembolic	0 Participants	0 Participants

Adverse Events

Group I (Lower Dose Platelet Transfusion)

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Group II (Higher Dose Platelet Transfusion)

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Group I (Lower Dose Platelet Transfusion) n=2 participants at risk Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 30 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo lower dose platelet transfusion	Group II (Higher Dose Platelet Transfusion) n=2 participants at risk Patients undergo platelet transfusion on all days when the morning platelet count is below the threshold 50 x 10\^9/L for up to 30 days or until the platelet count spontaneously recovers to \> 50 x 10\^9 for 3 consecutive days in the absence of transfusions. Platelet Transfusion: Undergo higher dose platelet transfusion
Gastrointestinal disorders mucositis	50.0% 1/2 • Number of events 1 • On study time (varied between subjects)	100.0% 2/2 • Number of events 2 • On study time (varied between subjects)
Gastrointestinal disorders transaminitis	0.00% 0/2 • On study time (varied between subjects)	50.0% 1/2 • Number of events 1 • On study time (varied between subjects)

Additional Information

Dr Bethany Samuelson Bannow

FHCRC

Phone: 5034949925

Email: samuelsb@ohsu.edu

Results disclosure agreements

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