Efgartigimod efficacy and safety in refractory myasthenia gravis: UK's first real-world experience.

Background: We report our experience of patients with generalised myasthenia gravis (gMG) treated with efgartigimod, an neonatal Fc receptor antagonist, under the Early Access to Medicine Scheme (EAMS) in the UK.

Methods: Data from all UK patients treated with efgartigimod under the EAMS July 2022 to July 2023 were collected retrospectively. Efgartigimod was administered as per the ADAPT protocol (consisting of a treatment cycle of four infusions at weekly intervals with further cycles given according to clinical need).

Post hoc analysis: 6 Months immunogenicity after third dose of BNT162b2 vs JNJ-78436735 After Two Doses of BNT162b2 vaccine in Solid Organ Transplant Recipients.

Introduction: In Solid Organ Transplant (SOT) recipients, due to immunosuppression, the immunogenicity after COVID-19 vaccination is suboptimal and its durability is unknown.

Methods: We conducted a post-hoc analysis of a patient-blinded, single center, randomized controlled trial comparing BNT162b2 vs JNJ-78436735 as the third dose after two doses of BNT162b2 in adult SOT recipients with active graft to compare long-term immunogenicity.

Results: Forty-one recipients were analyzed.

Impact of the SARS-CoV-2/COVID-19 pandemic on the patient journeys of those with a newly diagnosed paediatric brain tumour in the UK: a qualitative study.

Objectives: To explore the impact of the SARS-CoV-2/COVID-19 pandemic on the diagnosis, management and patient journey for children and young people with a newly diagnosed brain tumour in the UK.

Design: Exploratory qualitative study focused on patient journeys from multiple perspectives, conducted as part of a wider mixed-methods study.

Setting: Three paediatric oncology tertiary centres in the UK.

Stratified Medicine Paediatrics: Cell free DNA and serial tumour sequencing identifies subtype specific cancer evolution and epigenetic states.

We profiled a large heterogenous cohort of matched diagnostic-relapse tumour tissue and paired plasma-derived cell free DNA (cfDNA) from patients with relapsed and progressive solid tumours of childhood. Tissue and cfDNA sequencing results were concordant, with a wider spectrum of mutant alleles and higher degree of intra-tumour heterogeneity captured by the latter, if sufficient circulating tumour-derived DNA (ctDNA) was present. Serial tumour sequencing identified putative drivers of relapse, with alterations in epigenetic drivers being a common feature.