Optimization of Romiplostim Biosimilar Efficacy Trial Using In Silico Clinical Trial Approach for Patients With Immune Thrombocytopenia.

During biosimilar drug development, conducting a clinical trial of biosimilar efficacy in patients may become necessary in the presence of residual uncertainty regarding the biosimilarity of the drugs. In the development of the biosimilar romiplostim GP40141, we aimed to use a model-based in silico clinical trial (ISCT) approach to optimize the planned biosimilar efficacy trial in patients with immune thrombocytopenia. The population pharmacokinetic/pharmacodynamic model for healthy volunteers was modified and validated to describe platelet dynamics in patients with immune thrombocytopenia.

Active spread of β-thalassemia beyond the thalassemia belt: A study on a Russian population.

β-Thalassemia is a disease traditionally associated with thalassemia belt countries. Nonetheless, as global migration intensifies, β-thalassemia-causing variants spread far from their origin. We investigated this process to detect some patterns underlying its course.

TiCTMXene as surface-enhanced Raman scattering substrate.

The electromagnetic field enhancement mechanisms leading to surface-enhanced Raman scattering (SERS) of R6G molecules near TiCTMXene flakes of different shapes and sizes are analyzed theoretically in this paper. In COMSOL simulations for the enhancement factor (EF) of SERS, the dye molecule is modeled as a small sphere with polarizability spectrum based on experimental data. It is demonstrated, for the first time, that in the wavelength range of500 nm-1000 nm, the enhancement of Raman signals is largely conditioned by quadrupole surface plasmon (QSP) oscillations that induce a strong polarization of the MXene substrate.

Sustained response off-treatment in eltrombopag-treated adult patients with ITP who are refractory or relapsed after first-line steroids: Primary, final, and ad-hoc analyses of the Phase II TAPER trial.

Immune thrombocytopenia (ITP) is characterized by reduced platelet count due to increased destruction and is categorized according to the time following diagnosis (newly diagnosed, persistent, chronic). First-line corticosteroid therapy is associated with transient response, high relapse rates, and considerable toxicity. TAPER (NCT03524612) is a Phase II, prospective, single-arm trial investigating whether eltrombopag can induce a sustained response off-treatment (SRoT) in adult patients with ITP after first-line corticosteroid failure.

Surgical treatment of epilepsy in children with focal cortical dysplasia in central gyri.

Unlabelled: Focal cortical dysplasias are known to be the most frequent and furtive lesions leading to intractable epilepsy in children. Epilepsy surgery in central gyri, been effective in 60-70% of cases, is still significantly challenging due to the high risk of postoperative permanent neurological impairment.

Study Aims: Assessment of the outcome after epilepsy surgery in children with FCD in central lobules.