epoetin alfa (GerEpo / epoetin alfa, NCPC)

Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A.

Kessler Craig M CM, Mannucci Pier M PM, Jiménez-Yuste Victor V, Frenzel Laurent L et al.

Introduction Prophylaxis with factor VIII (FVIII) is the standard of care for individuals with severe hemophilia A, but it is associated with a substantial economic burden. This analysis compared the efficacy, dosing, and annual treatment costs of simoctocog alfa (Nuwiq, a recombinant FVIII [rFVIII]) with five extended half-life (EHL) rFVIII concentrate products. Methods Matching-adjusted indirect comparisons (MAICs) were performed to compare simoctocog alfa with efanesoctocog alfa (ALTUVIIIO) and turoctocog alfa pegol (Esperoct), and results were integrated with a previously published MAIC comparing simoctocog alfa with efmoroctocog alfa (ELOCTATE), damoctocog alfa pegol (JIVI), and rurioctocog alfa pegol (ADYNOVATE). These MAIC-adjusted populations were used to develop a cost model from a United States payer perspective. Results Annual total costs per person were $147,058 to $492,090 lower with simoctocog alfa compared with EHL rFVIII products, corresponding to a 21% to 46% cost reduction, primarily driven by lower drug acquisition costs. Outcomes were comparable or favorable to simoctocog alfa versus turoctocog alfa pegol, efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol. In comparison with efanesoctocog alfa (Group A), no significant differences were observed in the proportion of individuals with zero bleeds, whereas treated total annualized bleeding rate (ABR) and treated spontaneous ABR favored efanesoctocog alfa. Conclusion This indirect comparison suggests that personalized prophylaxis with simoctocog alfa may offer economic advantages versus EHL rFVIII products in individuals with severe hemophilia A. Clinical outcomes were broadly comparable across comparators, although treated total and spontaneous ABRs were significantly lower with efanesoctocog alfa (Group A).

Ropeginterferon alfa-2b in mycosis fungoides: A multicentre cohort.

Nikolaou Vasiliki V, Koumprentziotis Ioannis-Alexios IA, Konstantinou Iliana I, Iliakis Theodoros T et al.

Reversal Is Not Hemostasis: What a Trauma Model Teaches Us about Andexanet Alfa and Four-Factor Prothrombin Complex Concentrate in Cardiac Surgery.

Erdoes Gabor G, Koster Andreas A, Frere Corinne C

pgRNA-m6A levels associate with HBV replication and liver function.

Yang Chaoqi C, Qin Shanshan S, Gao Rui-Huan RH, Li Anling A et al.

m6A modification has been shown to play a role in regulating HBV RNA expression. However, it remains unclear whether the m6A levels of HBV pregenomic RNA (pgRNA) are related to HBV replication and liver function. Serum HBV pgRNA-m6A levels were determined by T3 DNA ligase assay. HBV DNA copy numbers and gene expression were detected by TaqMan assay and RT-qPCR, respectively. Liver function was evaluated using clinical laboratory indicators. The TET-off stable HBV-producing cell line HepAD38 and targeted pgRNA demethylation by SunTag system (TRADES) were used for the site-specific m6A editing. Flow cytometry was used to measure the cytokine levels in the supernatant of cell culture. Serum HBV pgRNA-m6A levels were positively correlated with HBV DNA copy number, pgRNA expression and liver function indicators (all p < 0.05). Both pgRNA-m6A levels and pgRNA expression were significantly increased in patients with high virus load (HBV DNA > 1.0 × 107 IU/mL). Site-specific demethylation of pgRNA1907-m6A significantly reduced HBV DNA, hepatitis B e antigen (HBeAg) and HBV pgRNA, while induced levels of interferon alfa 2 (IFN-α2) and apolipoprotein B mRNA editing enzyme catalytic subunit 3A (APOBEC3A). pgRNA-m6A modification of HBV associates with viral replication and liver function indicators. Site-specific m6A demethylation of pgRNA provides novel insight into anti-HBV treatment.