Outpatient Platelet Transfusions in Myelodysplastic Syndromes and Leukemia: The OPTIMAL Pilot
A local study nurse will use a web-based randomization system (permuted random blocks of two
or four patients) to allocate consenting patients. Medical and research staff and
investigators will be blinded to randomization scheme.
Study Duration and Follow-up:
All patients will be assigned to either a therapeutic or prophylactic platelet transfusion
for a 6-month period. This period of time will allow us to assess our primary feasibility
outcomes of enrollment, compliance with transfusion protocols, and completion of bleeding
evaluations and quality of life questionnaires by patients.
Patients in both groups will have their CBC measured at least weekly while on study. More
frequent monitoring of the platelet count may be performed at the discretion of the treating
physician. The research team will clinically assess patients within 1 week (+/- 3 days) of
randomization, and monthly thereafter. Patients will be asked to report any non-cutaneous
grade 2 or greater bleeding immediately to their treating physicians and the study team. All
the self-assessments of bleeding will be reviewed at each patient visit to ensure that all
clinically relevant bleeding episodes have been captured.
Patients whose platelet count recovers to greater than 20 x 109/L for at least six weeks
will be taken off the weekly platelet count monitoring as reflects clinical practice. They
will continue to be monitored for the duration of the study. If their platelet count falls
to 10 x 109/L or below then they will be restarted on the monitoring and previous
Baseline Data - The following clinical and laboratory data will be collected at the time of
enrolment: (1) demographic data, (2) diagnosis including date and disease stage, (3) prior
chemotherapy, (4) ECOG performance status, (5) comorbidites, (6) previous platelet and red
cell transfusions, (7) red cell transfusion history, (8) prior bleeding events, (9) quality
of life (EQ5D) (10) routine bloodwork
All platelet and red cell transfusions will be recorded including date and number of units.
For platelet transfusions, the type of platelet product (e.g. apheresis or buffy coat) and
ABO compatibility will recorded. Additionally, the indication for all transfusions
(prophylactic or to treat bleeding) will be obtained from the attending physician.
Participants will be asked to complete a simple bleeding questionnaire on a daily basis
which has been previously tested and used in outpatients with thrombocytopenia. Patients
will receive a short training session and written material on how to complete the form. A
bleeding assessment will be performed at all study follow-up visits (interview, physical
exam and review of daily bleeding forms). Bleeding events will be assigned a bleeding grade
by the study personnel. A final bleeding grade will be assigned by an adjudication panel of
2 blinded physicians who will independently assign bleeding scores. Any discrepancies in
bleeding scores will be resolved by consensus.
Quality of Life:
Quality of life will be measured using the EQ-5D. The EQ-5D is a validated tool for
measurement of health-related quality of life there is a precedent for its use in
transfusion medicine trials. Quality of life assessments will be performed at baseline and
at all subsequent patient visits.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment
Overall enrollment, off protocol transfusions per each randomized group, total number of platelet transfusions per group and patient compliance with daily self assessment of bleeding will be evaluated.
Alan Tinmouth, MD, MSc
Ottawa Hospital Research Institute
Canada: Ethics Review Committee