A No-Prophylaxis Platelet-Transfusion Strategy for Hematologic Cancers

Simon J. Stanworth*, Lise J. Estcourt, Gillian Powter, Brennan C. Kahan, Claire Dyer, Louise Choo, Lekha Bakrania, Charlotte Llewelyn, Timothy Littlewood, Richard Soutar, Derek Norfolk, Adrian Copplestone, Neil Smith, Paul Kerr, Gail Jones, Kavita Raj, David A. Westerman, Jeffrey Szer, Nicholas Jackson, Peter G. BardyDianne Plews, Simon Lyons, Linley Bielby, Erica M. Wood, Michael F. Murphy, TOPPS Investigators

*Corresponding author for this work

    Research output: Contribution to journalArticlepeer-review

    350 Citations (Scopus)



    The effectiveness of platelet transfusions to prevent bleeding in patients with hematologic cancers remains unclear. This trial assessed whether a policy of not giving prophylactic platelet transfusions was as effective and safe as a policy of providing prophylaxis.


    We conducted this randomized, open-label, noninferiority trial at 14 centers in the United Kingdom and Australia. Patients were randomly assigned to receive, or not to receive, prophylactic platelet transfusions when morning platelet counts were less than 10x10(9) per liter. Eligible patients were persons 16 years of age or older who were receiving chemotherapy or undergoing stem-cell transplantation and who had or were expected to have thrombocytopenia. The primary end point was bleeding of World Health Organization (WHO) grade 2, 3, or 4 up to 30 days after randomization.


    A total of 600 patients (301 in the no-prophylaxis group and 299 in the prophylaxis group) underwent randomization between 2006 and 2011. Bleeding of WHO grade 2, 3, or 4 occurred in 151 of 300 patients (50%) in the no-prophylaxis group, as compared with 128 of 298 (43%) in the prophylaxis group (adjusted difference in proportions, 8.4 percentage points; 90% confidence interval, 1.7 to 15.2; P = 0.06 for noninferiority). Patients in the no-prophylaxis group had more days with bleeding and a shorter time to the first bleeding episode than did patients in the prophylaxis group. Platelet use was markedly reduced in the no-prophylaxis group. A prespecified subgroup analysis identified similar rates of bleeding in the two study groups among patients undergoing autologous stem-cell transplantation.


    The results of our study support the need for the continued use of prophylaxis with platelet transfusion and show the benefit of such prophylaxis for reducing bleeding, as compared with no prophylaxis. A significant number of patients had bleeding despite prophylaxis.

    Original languageEnglish
    Pages (from-to)1771-1780
    Number of pages10
    JournalNew England Journal of Medicine
    Issue number19
    Publication statusPublished - 9 May 2013


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