Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.

Background: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.

Initial pig developmental stage influences intestinal organoid growth but not cellular composition.

Background: Intestinal organoids are promising tools in the context of animal experiment reduction but a thorough characterization of the impact of the origin of intestinal stem cells (ISC) on organoid phenotype is needed to routinely use this cellular model. Our objective was to evaluate the effect of ISC donor age on the growth, morphology and cellular composition of intestinal organoids derived from pig.

Methods: Organoids were derived from jejunal and colonic ISC obtained from 1-, 7-, 28-, 36- and 180-day-old pigs and passaged three times.

Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors.

Introduction: The phase 3 XTEND-1 trial (NCT04161495) demonstrated that efanesoctocog alfa prophylaxis provided superior bleed protection compared with pre-trial factor VIII (FVIII) prophylaxis in patients with severe haemophilia A. The aim of this study was to indirectly compare the efficacy of efanesoctocog alfa with non-factor replacement therapy emicizumab in adolescent and adult patients with severe haemophilia A without inhibitors.

Methods: A systematic literature review was conducted to identify phase 3 trials of emicizumab.

Non-inferiority of Cataract Surgery in a Stand-Alone Unidirectional Airflow System Versus in a Conventional Operating Room: A Retrospective Study.

Purpose: To demonstrate the non-inferiority of a stand-alone unidirectional airflow system, the SurgiCube (Surgi-Cube International BV), for cataract surgery compared to a conventional operating theater (OT).

Methods: This was a retrospective and comparative cross-sectional study conducted at the Rothschild Foundation, Paris, France. All patients who underwent cataract surgery using the SurgiCube between February 2020 and February 2023 were included and compared to a separate group of patients with cataract surgery performed in a conventional OT and under topical anesthesia during the same period.