"Prophylactic Platelet Transfusions in Hematologic Cancers".The New England Journal of Medicine. 2013.

Clinical Question

Does a policy of no prophylactic platelet transfusions in thrombocytopenic patients with hematologic cancers reduce WHO grade 2 or higher bleeding events as effectively as a standard prophylactic transfusion policy?

Bottom Line

A strategy of omitting prophylactic platelet transfusions was inferior to the standard practice of prophylactic transfusions in preventing bleeding events of WHO grade 2, 3, or 4 in patients with hematologic cancers and thrombocytopenia.

Major Points

The open-label, noninferiority randomized trial involved 600 patients who were assigned either to a no-prophylaxis policy or to receive standard prophylactic platelet transfusions when platelet counts were less than 10×10^9 per liter. The primary outcome was the occurrence of WHO grade 2 or higher bleeding events up to 30 days after randomization.

The trial found that 50% of patients in the no-prophylaxis group experienced grade 2, 3, or 4 bleeding events, compared to 43% in the prophylaxis group, demonstrating the superiority of prophylactic platelet transfusions in reducing bleeding events. Platelet use was markedly reduced in the no-prophylaxis group, although patients also experienced more days with bleeding. Subgroup analysis showed similar rates of bleeding among patients undergoing autologous stem-cell transplantation, irrespective of prophylaxis policy.

Guidelines

Current guidelines recommend prophylactic platelet transfusions for patients with hematologic cancers, especially for those undergoing chemotherapy or allogeneic hematopoietic stem-cell transplantation.

Design

Multicenter, open-label, parallel-group, randomized, noninferiority trial conducted at 14 centers in the UK and Australia.

Population

- Inclusion Criteria: Patients ≥16 years of age with hematologic cancers undergoing chemotherapy or stem-cell transplantation, with existing or expected thrombocytopenia (platelet count <50×10^9 per liter).
- Exclusion Criteria: Previous WHO grade 3 or 4 bleeding events, WHO grade 2 bleeding during current admission, inherited hemostatic or thrombotic disorders, need for therapeutic anticoagulants, acute promyelocytic leukemia, known HLA antibodies, prior study participation.

Interventions

- No Prophylaxis Group: Did not receive prophylactic platelet transfusions unless platelet counts were less than 10×10^9 per liter.
- Prophylaxis Group: Received standard prophylactic platelet transfusions when platelet count was less than 10×10^9 per liter.

Outcomes

- Primary Outcome: Percentage of patients with bleeding events of WHO grade 2, 3, or 4 up to 30 days post-randomization.
- Secondary Outcomes: Days with WHO grade 2, 3, or 4 bleeding; time to first grade 2, 3, or 4 bleeding episode; number of platelet and red-cell transfusions; days with platelet count less than 20×10^9 per liter; time until recovery from thrombocytopenia.

Criticisms

- The primary bleeding assessment relies on patient and staff reporting, which could introduce variance between study centers.
- The trial was not powered to specifically determine whether withholding prophylactic transfusions in patients undergoing autologous stem-cell transplantation is safe and effective.

Funding

National Health Service Blood and Transplant Research and Development Committee, Australian Red Cross Blood Service.

Further Reading

The full text of this article is available with additional supplementary material at NEJM.org.