Freeze-Dried Platelets promising alternative in bleeding thrombocytopenic patients: Study

USA: In a new study, it was shown that Freeze-Dried Platelets may be a viable alternative to traditional platelets in the treatment of bleeding. The following study was conducted by Maro Ohanian and the team, results of which were published in the American Journal of Hematology on 8th November 2021.

Thrombosomes are freeze-dried, trehalose-stabilized group O platelets having a 3-year shelf life. They may be stored in large quantities, quickly reconstituted, and injected regardless of the recipient's blood type. 

The current study looked at the safety and possible early signs of the effectiveness of thrombosomes in patients with bleeding thrombocytopenia. For this study, researchers conducted an open-label, phase 1 research of single doses of allogeneic Thrombosomes at three dosage levels in three cohorts of eight patients each with hematologic malignancies, thrombocytopenia, and bleeding. Adverse events, dose-limiting toxicities (DLTs), bleeding ratings from the World Health Organization (WHO), and hematological parameters were also evaluated.

The average age was 59 years (24-71). The majority of patients had AML (58%) or ALL (29%), followed by MDS (8%), and myeloproliferative neoplasm (4% ). Through day 6, the WHO scores of 22 patients who were actively bleeding at a total of 27 locations at baseline either improved or stabilized. Twelve patients (50%) experienced a clinically meaningful rise in platelet count 24 hours after infusion.

5 patients exhibited a clinically meaningful rise in platelet count of 5000 platelets/L and two achieved platelet count stabilization after receiving no platelet transfusions for six days following Thrombosomes infusion. In 24 bleeding, thrombocytopenic patients with hematological malignancies, thrombosome dosages up to 3.78 x 108 particles/kg were shown to be safe.

In conclusion, the data from the study clearly states the potential efficiency of Thrombosomes in bleeding treatment and further studies with various modifications can be done in the future to strengthen the findings and come to a rock-solid result.

Reference:

Ohanian, M., Cancelas, J. A., Davenport, R., Pullarkat, V., Hervig, T., Broome, C., Marek, K., Kelly, M., Gul, Z., Rugg, N., Nestheide, S., Kinne, B., Szczepiorkowski, Z., Kantarjian, H., Pehta, J., Biehl, R., Yu, A., Aung, F., Antebi, B., & Fitzpatrick, G. M. (2021). Freeze‐Dried Platelets Are a Promising Alternative in Bleeding Thrombocytopenic Patients with Hematological Malignancies. In American Journal of Hematology. Wiley. https://doi.org/10.1002/ajh.26403