Chu, T., Tang, Y., Wang, H., Qi, J., Zhao, Y., Pan, T., . . . Han, Y. (2017). Efficacy of recombinant factor VIIa for severe bleeding complicated by platelet transfusion refractoriness in patients with hematologic malignancies. Thrombosis Research, 160, 14–18.

The purpose of this study was to evaluate the effect of recombinant factor VIIa (rFVIIa) upon severe bleeding in patients with platelet transfusion refractoriness.

Patients in the intervention group received 60 ug/kg of recombinant factor VIIa intravenously, with or without conventional treatment. Those in the control group received only conventional treatment. Dosing of rFVIIa was dependent on clinical characteristics and response to treatment. Conventional treatments included transfusion of platelets, plasma, or cryoprecipitate, or medications such as hemocoagulase atrox or carbazochrome sodium sulfonate.

• N = 64   
• AGE: Mean age was 35 years.
• MALES: Not available  
• FEMALES: Not available 
• CURRENT TREATMENT: Other
• KEY DISEASE CHARACTERISTICS: Hematologic malignancies

• SITE: Single site   
• SETTING TYPE: Inpatient    
• LOCATION: University Hospital in Soochow, China

PHASE OF CARE: Active antitumor treatment

This was a controlled prospective study. Randomization was not stated.

Bleeding severity was graded according to criteria established by Nevo et al. (1999): 0 (no bleeding) to 4 (massive bleeding leading to hemodynamic compromise or bleeding into a vital organ). Major bleeding was graded according to criteria from the International Society on Thrombosis and Hemostasis.

Response rates to hemostatic treatment were significantly higher in the intervention group receiving rFVIIa at 24 hours (p = 0.014) and 48 hours (p = 0.020) when compared to the control group. Patients achieving a complete remission were also significantly higher in the intervention group at 24 hours (p = 0.031), 48 hours (p = 0.039), and 72 hours (p = 0.021). Bleeding score and time to control bleeding were significantly reduced in the intervention group (p = 0.029 and p = 0.034).

Administration of rfVIIa was significantly more likely to lead to control of bleeding and decreased time to control bleeding in patients with hematologic malignancies with severe thrombocytopenia, who were refractory to platelet transfusions. In addition, patients receiving rFVIIa were significantly more likely to have a complete remission than those who did not receive rFVIIa.

• Small sample (< 100)
• Risk of bias (no blinding)
• Risk of bias (no random assignment)
• Other limitations/explanation: There were no standard number of doses received or threshold when given, except for clinical assessment (median number was 3, range was 1-15).

The administration of rFVIIa may be considered an effective adjunct to conventional therapy to reduce bleeding in patients with hematologic malignancies who are refractory to platelet transfusions, along with use of HLA-matched or cross-matched platelets, to improve outcomes.