Freeze-dried platelets are a promising alternative in bleeding thrombocytopenic patients with hematological malignancies.

Autor: Ohanian M; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Cancelas JA; Hoxworth Blood Center, Cincinnati, Ohio, USA., Davenport R; University of Michigan Hospitals, Ann Arbor, Michigan, USA., Pullarkat V; City of Hope Comprehensive Cancer Center, Duarte, California, USA., Hervig T; Haukeland Universitetssjukehus, Bergen, Norway., Broome C; MedStar Georgetown University Hospital, Washington, District of Columbia, USA., Marek K; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Kelly M; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Gul Z; Division of Hematology/Oncology, Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA., Rugg N; Hoxworth Blood Center, Cincinnati, Ohio, USA., Nestheide S; Hoxworth Blood Center, Cincinnati, Ohio, USA., Kinne B; Division of Hematology/Oncology, Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA., Szczepiorkowski Z; Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire, USA., Kantarjian H; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Pehta J; The Alpha Bio Group, New Canaan, Connecticut, USA., Biehl R; Cellphire Therapeutics, Rockville, Maryland, USA., Yu A; Cellphire Therapeutics, Rockville, Maryland, USA., Aung F; Department of Transfusion Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Antebi B; Cellphire Therapeutics, Rockville, Maryland, USA., Fitzpatrick GM; Cellphire Therapeutics, Rockville, Maryland, USA.
Jazyk: angličtina
Zdroj: American journal of hematology [Am J Hematol] 2022 Mar 01; Vol. 97 (3), pp. 256-266. Date of Electronic Publication: 2021 Dec 23.
DOI: 10.1002/ajh.26403
Abstrakt: Thrombosomes are trehalose-stabilized, freeze-dried group O platelets with a 3-year shelf life. They can be stockpiled, rapidly reconstituted, and infused regardless of the recipient's blood type. Thrombosomes thus represent a potential alternative platelet transfusion strategy. The present study assessed the safety and potential early signals of efficacy of Thrombosomes in bleeding thrombocytopenic patients. We performed an open-label, phase 1 study of single doses of allogeneic Thrombosomes at three dose levels in three cohorts, each consisting of eight patients who had hematologic malignancies, thrombocytopenia, and bleeding. Adverse events, dose-limiting toxicities (DLTs), World Health Organization (WHO) bleeding scores, and hematology values were assessed. No DLTs were reported. The median age was 59 years (24-71). Most patients had AML (58%) or ALL (29%), followed by MDS (8%) and myeloproliferative neoplasm (4%). The WHO scores of 22 patients who were actively bleeding at a total of 27 sites at baseline either improved (n = 17 [63%]) or stabilized (n = 10 [37%]) through day 6. Twenty-four hours after infusion, 12 patients (50%) had a clinically significant platelet count increase. Of eight patients who received no platelet transfusions for 6 days after Thrombosomes infusion, 5 had a clinically significant increase in platelet count of ≥5000 platelets/μL and 2 had platelet count normalization. Thrombosomes doses up to 3.78 × 10 8 particles/kg demonstrated safety in 24 bleeding, thrombocytopenic patients with hematological malignancies. Thrombosomes may represent an alternative to conventional platelets to treat bleeding. A phase 2 clinical trial in a similar patient population is underway.
(© 2021 Wiley Periodicals LLC.)
Databáze: MEDLINE
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