A Randomised Controlled Trial Of Prophylactic Vs No-Prophylactic Platelet transfusions in Patients with Haematological malignancies

When do we need to use platelet transfusions for people with low platelet counts?

What was this study about?

The trial team wanted to find out more about how best to use platelets – the cells that help blood to clot and so help the body to stop bleeding.

Previously, it was standard practice to give platelets to patients with low platelet counts whether they were bleeding or not. However, platelet transfusions are not without risk, and it was not clear whether platelet transfusions are really necessary when the platelet count is low but there are no signs or symptoms of bleeding.  Therefore, the TOPPS trial was designed.

Patients who consented were divided randomly into two groups. Patients in one group were allocated to receive platelet transfusions only when they needed them to treat bleeding. Patients in the other group received platelet transfusions not only when they needed them for bleeding, but also when their platelet count dropped below a certain low level. Although both ways of using platelet transfusions had been used before, we were not certain which is better. Patients could also be given additional platelets by their doctor whenever he/she thought it was appropriate, regardless of which group they were in.

The aim of this study was to compare the benefits of different ways of using platelet transfusions, and the information from this study will help us to improve our use of platelets in this patient group. This is important because platelet transfusions are a scarce resource in all countries including the UK.

What difference did this study make?

The TOPPS trial conducted at UK and Australian centres assessed whether a no-prophylaxis platelet transfusion strategy was as effective and safe as giving prophylactic (preventive) platelet transfusions. It demonstrated the overall benefits of continuing to give prophylactic platelet transfusions to blood cancer patients with low platelet counts to reduce the rate of clinically significant bleeding as opposed to "therapeutic-only" platelet transfusions (i.e. only giving platelets to treat, rather than prevent bleeding). The results also showed that there is still a high burden of bleeding in many of these patients despite being given prophylactive platelets suggesting that alternative strategies to prevent bleeding should be investigated.

The results are already influencing practice, as they demonstrate the benefit of continuing to give prophylactic platelets to these patients and it has also led to a new trial (TREATT) being run by the NHSBT Clinical Trials Unit. This will look at whether a drug called tranexamic acid can help prevent bleeding in the same group of patients.

Type of study

Randomised trial

Contact details

Who funded the study?

The National Blood Service, part of NHS Blood and Transplant.

When did it take place?

Recruitment opened in August 2006 and closed in August 2011.

Where did it take place?

8 hospitals within the UK.

Who was included?

Adult patients with a confirmed diagnosis of a haematological malignancy who either were or were expected to become thrombocytopaenic with a platelet count of less than 50x109/L for at least 5 days.