Annals of hematology
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Annals of hematology · Nov 2020
Randomized Controlled Trial Comparative Study Clinical TrialDirect comparison of two extended half-life PEGylated recombinant FVIII products: a randomized, crossover pharmacokinetic study in patients with severe hemophilia A.
An open-label, crossover randomized study was performed to compare the pharmacokinetics (PK) of damoctocog alfa pegol and rurioctocog alfa pegol, two recombinant factor VIII (FVIII) products indicated in patients with hemophilia A, both conjugated to polyethylene glycol to reduce clearance and extend time in circulation. Adult patients (N = 18) with severe hemophilia A (FVIII < 1 IU/dL), previously treated with any FVIII product for ≥ 150 exposure days, were randomized to receive a single 50 IU/kg infusion of damoctocog alfa pegol followed by rurioctocog alfa pegol, or vice versa, with ≥ 7-day washout between doses. FVIII activity was measured using the one-stage clotting assay. ⋯ Overall, damoctocog alfa pegol had a superior PK profile versus rurioctocog alfa pegol. Trial registration number: NCT04015492 ( ClinicalTrials.gov identifier). Date of registration: July 9, 2019.
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Annals of hematology · Nov 2020
Clinical TrialTreatment outcomes and chronicity predictors for primary immune thrombocytopenia: 10-year data from an academic center.
Primary immune thrombocytopenia (ITP) is an intriguing autoimmune disease characterized by autoantibodies against platelets and megakaryocytes. Clinical outcomes, response to treatment, and chronicity predictors were investigated. Patients with newly diagnosed primary ITP treated at a hematology referral center from 2008 to 2018 with complete medical and recent medication history were stratified by age as children < 16 years and adults > 16 years. ⋯ Platelets ≥ 20 × 109/L and age ≥ 6 years were risk factors for chronic ITP in the univariate analysis in the adult and children groups, respectively. Clinical course and treatment outcomes for ITP are considerably heterogeneous. Higher platelet counts at diagnosis in adults and age ≥ 6 years in children were associated with an increased risk of chronicity.