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A randomised controlled trial of prophylactic vs non-prophylactic platelet transfusions in patients with haematological malignancies

Dates:  2011-2012
Funding: NHS Blood and Transplant (NHSBT)
Collaborators: Dr Simon Stanworth, NHSBT Oxford Centre
Information: Liz Stokes

Platelets are the second most frequent blood component used by the NHS, and over 50% of all platelet transfusions are given as prophylaxis to patients with haematological malignancies.  There is however a lack of evidence and considerable uncertainty about the benefits of prophylactic platelet transfusions for the prevention of thrombocytopenic bleeding in patients with bone marrow failure.  The aim of the TOPPS multi-centre trial is to assess whether therapeutic transfusion of platelets in patients with haematological malignancies and thrombocytopenia is as safe and effective as prophylactic transfusion.  The primary outcome measure is the proportion of patients who have a major bleed up to 30 days.

No formal health economic data capture was incorporated within the TOPPS trial, which completed recruitment in 2011.  However, it was decided that it would be feasible to generate an estimate of the net cost impact of moving from prophylactic to therapeutic transfusion.  Data will be available on key resources consumed up to 30 days, and the costs in each arm of the trial will be presented alongside the primary and secondary clinical endpoints. 

The number of blood products transfused to patients will be one of the key resources measured.  In addition to the costs of the blood products per se, the cost of administering those products, in particular nursing time, is likely to be sizeable but has not previously been quantified.  A detailed costing study is being conducted at haematology day units at two of the TOPPS trial centres to measure and cost the nursing inputs and consumables required to give transfusions of platelets and red blood cells; final cost estimates which will be used in the trial analysis.  Given the increasing number of studies being conducted around blood product transfusion, it is anticipated that these estimates will be of interest to the wider blood transfusion and research community.


Campbell, HE, Estcourt, LJ, Stokes, EA, Llewelyn, CA, Murphy, MF, Wood, EM, and Stanworth, SJ (2014). Prophylactic platelet transfusions in patients with blood malignancies: cost analysis of a randomized trial. Transfusion, DOI: 10.1111/trf.12697.