Cryopreserved platelet (CPP) transfusions appear to be safe and effective in bleeding patients with thrombocytopenia, according to a study published in Transfusion.
The phase 1 trial was a multicenter, randomized, open-label, dose-escalation study. The patient population included 28 hospitalized hematology-oncology patients with thrombocytopenia and a mean age of 51.9 ± 15.8 years (39.3% female, 60.7% male; 75% white). All patients had active World Health Organization bleeding scores of grade 2 (organ system bleeding, 14 patients), grade 3 (requiring red blood cell transfusion, 7 patients), or grade 4 (life-threatening bleeding, 7 patients).
The 28 patients were divided into 5 cohorts: 4 were sequential CPP cohorts that received escalating doses of CPP transfusions — 0.5, 1, 2, or 3 units (24 patients) — while the fifth cohort received 1 unit of standard liquid-stored platelets (LSP; 4 patients). Patients were monitored for adverse events, coagulation markers, platelet responses, and hemostatic efficacy for 6 days.
Bleeding scores improved in 58% (14/24) and 50% (2/4) of patients that received CPPs and LSPs, respectively. The platelet count increment in patients receiving CPPs increased in a dose-dependent manner (1.20×109 ± 3.10×109 platelets/L for 0.5-unit to 13.2×109 ± 6.38×109 platelets/L for 3-unit transfusions) but was much smaller than the count increment in patients receiving LSPs (>25.0×109 platelets/L). No evidence showed a dose response based on bleeding reduction and the number of CPP units transfused.
Three patients experienced mild transfusion-related adverse events, which included chill and fever (1 patients), transient increased respiratory rate (1 patient), DMSO-related skin odor (2 patient), and headache (1 patient).
The investigators concluded, “CPP transfusions appear to be safe and effective, and a phase 2 efficacy and safety trial in cardiopulmonary bypass surgery patients with postsurgical bleeding is planned.”
Slichter SJ, Dumont LJ, Cancelas JA, et al. Safety and efficacy of cryopreserved platelets in bleeding patients with thrombocytopenia. Transfusion. 2018;58(9):2129-2138. doi:10.1111/trf.14780