Effective targeting of somatic cancer mutations to enhance the efficacy of cancer immunotherapy requires an individualized approach. Autogene cevumeran is a uridine messenger RNA lipoplex-based individualized neoantigen-specific immunotherapy designed from tumor-specific somatic mutation data obtained from tumor tissue of each individual patient to stimulate T cell responses against up to 20 neoantigens. This ongoing phase 1 study evaluated autogene cevumeran as monotherapy (n = 30) and in combination with atezolizumab (n = 183) in pretreated patients with advanced solid tumors.
View Article and Find Full Text PDFObjectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.
Methods: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82).
Background: The Mobility Scale for Acute Stroke Patients (MSAS) was developed to discriminate between the lower levels of mobility in acute stroke patients in the first two weeks post-onset.
Objective: The present study aims to develop and validate an Italian version of the MSAS.
Methods: The English version of the MSAS was translated into Italian according to international guidelines.
Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases.
Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE.