TOPPS
The TOPPS study is now complete.
Chief investigators: Dr Simon Stanworth and Dr Erica Wood
Primary sponsor: NHSBT
Funder: NHSBT and the Australian Red Cross Blood Service (for the Australian arm of the trial)
Summary
This study is a randomised controlled trial comparing prophylactic platelet use with no-prophylaxis in adult thrombocytopenic patients with haematological malignancies.
Study hypothesis
This two-stage, randomised, non-inferiority study is to determine whether a platelet transfusion policy with no prophylaxis is as clinically effective and safe as the prophylactic transfusion policy currently practised (based on a threshold peripheral blood platelet count of less than 10 x 109/L).
Study design
The trial is a two-stage randomised trial: after a minimum of 100 patients have been randomised (and 50 patients have completed their period of observation in the control arm) there will be an internal pilot study to review sample size and feasibility.
It is anticipated that around 300 patients will need to be randomly allocated to each arm of the trial over the entire duration of the trial. Patients will be initially enrolled through four UK centres.
Findings
This multicentre study has not shown that a no-prophylaxis platelet transfusion policy is non-inferior to prophylaxis. Patients in the no-prophylaxis group had more days with a WHO grade 2 or above bleed, and a shorter time to first bleed.
Further analysis is needed to determine whether these findings apply to all subgroups of patients. Despite a role for prophylactic platelet transfusions, rates of bleeding in patients remain high.
Publications
Stanworth SJ, Dyer C, Choo L, Bakrania L, Copplestone A, Llewelyn C, Norfolk D, Powter G, Littlewood T, Wood EM, Murphy M. Do all patients with hematologic malignancies and severe thrombocytopenia need prophylactic platelet transfusions?: Background, rationale and design of a clinical trial (trial of platelet prophylaxis) to assess the effectiveness of prophylactic platelet transfusions. Transfusion Medicine Reviews 2009;24:163-171.
Stanworth SJ, Estcourt LJ, Powter GP, Kahan BC, Dyer C, Choo L, Bakrania L, Llewelyn C, Littlewood T, Soutar R,Norfolk D, Copplestone A, Smith N, Kerr P, Jones G, Raj K, Westerman D, Szer J, Jackson N, Bardy P, Plews D, Lyons S, Bielby L, Wood EM and Murphy MB. A no-prophylaxis platelet-transfusion strategy for hematologic cancers. New England Journal of Medicine 2013; 368(19):1771-80 [E Pub 09 May 2013 doi:10.1056/NEJMoa1212772]
Stanworth SJ, Estcourt LJ, Llewelyn CA, Murphy MF, Wodd EM; for the TOPPS Study Investigators. Impact of prophylactic platelet transfusions on bleeding events in patients with haematological malignancies;a sub-group analysis of a randomised trial. Transfusion, 2014 [E Pub 14 April 2014 doi:10.1111/trf.12646]
Campbell HE, Estcourt LJ, Stokes EA, Llewelyn CA, Murphy MF, Wood, EM, Stanworth SJ. Prophylactic platelet transfusions in patients with blood malignancies: cost analysis of a randomized trial. Transfusion [E Pub 15 May 2014 doi:10.1111trf.12697]
Stanworth, SJ, Hudson CL, Estcourt LJ, Johnson RJ, Wood EM. Risk of Bleeding and use of platelet transfusions in patients with hematologic malignancies: recurrent event analysis. Haematologica 2015 published online: doi:10.3324/haematol.2014.118075