Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK.

With the first gene therapies for haemophilia approved by the European Commission, the US Food and Drug Administration, and the Medicines and Healthcare products Regulatory Agency, it is important to consider the remaining unmet needs and challenges that may arise throughout patients' treatment journeys. We discuss existing unmet needs and important considerations prior to, during, and following haemophilia gene therapy treatment in the UK, and propose potential next steps. Key areas for attention are education, psychological support, and guidance on implementation. Strategies are urgently required to fulfil these needs. An immediate priority for information providers should be comprehensive education for people with haemophilia (PWH) and healthcare professionals (HCPs). Greater access to resources and training in psychological services will be required throughout the treatment pathway. More specific guidance is required to define the implementation model, criteria for accreditation, and responsibilities of care centres. Furthermore, PWH may revisit discussions with HCPs multiple times pre-infusion, thus the patient journey is unlikely to be linear. Consideration of these challenges, and of potential strategies to address them, will be integral to optimising the future success of this promising therapy.

Safety profile of plasma for fractionation donated in the United Kingdom, with respect to variant Creutzfeldt-Jakob disease.

Plasma-derived medicinal products (PDMPs) are life-saving and life-improving therapies, but the raw material is in short supply: Europe depends on importation from countries including the United States. Plasma from donors resident in the United Kingdom has not been fractionated since 1999 when a precautionary measure was introduced in response to the outbreak of variant Creutzfeldt-Jakob disease (vCJD). Cases of vCJD have been far fewer than originally predicted in the 1990s. Since the introduction of leucodepletion in 1999, and accounting for the incubation period, more than 40 million UK-derived blood components have been issued with no reports of TT vCJD. In February 2021, the UK Government authorized manufacture of immunoglobulin from UK plasma. Following separate reviews concluding no significant difference in the risk posed, the United States, Australia, Ireland and Hong Kong also lifted their deferrals of blood donors with a history of living in the United Kingdom. Other countries are actively reviewing their position. Demand is rising for PDMPs, and Europe faces a threat of supply shortages. Industry and patient groups are clear that using UK plasma would bring significant immediate benefits to patients and to the resilience of the European supply chain. From this scientific review, we conclude that UK plasma is safe for fractionation and urge blood regulators and operators to take account of this safety profile when considering fractionation of UK plasma, and to revise their guidelines on the deferral of donors who have lived in, or received a transfusion in, the United Kingdom.

Expediting the management of cauda equina syndrome in the emergency department through clinical pathway design.

Introduction: Cauda equina syndrome (CES) is a neurosurgical emergency. Early diagnosis with MRI and subsequent surgical decompression surgery can prevent permanent neurological dysfunction. Charing Cross Hospital (CXH) is a tertiary neurosurgical referral centre where in the emergency department (ED), current practice mandated a neurosurgery review prior to requesting MRI. Hypothesis: It was hypothesised that a new clinical pathway, with better coordination from the ED, radiology and neurosurgical teams could reduce the time of presentation to diagnosis or exclusion of CES. Method: Retrospective case-note analysis of patients presenting with back pain to CXH ED over a 3-month period was performed. The primary outcome was the time interval between the patient's arrival to the ED and the MRI preliminary report. Results: The baseline primary outcome was recorded at 8 hours and 16 min (n=30). A new clinical pathway was designed empowering ED senior decision makers to order MRIs prior to neurosurgical review. Two Plan-Do-Study-Act (PDSA) cycles were performed, each measured over a 2-month period. The first PDSA cycle was performed after the pathway was initially launched (n=17), while the second PDSA cycle measured the effect of staff education and active promotion of the pathway (n=17). MRI was requested earlier, waiting and reporting time for MRI were reduced. The exclusion or diagnosis of CES was reduced to 5 hours and 54 min in PDSA 1 and 5 hours 17 min in PDSA 2, a 29% and 36% reduction (p=0.048 and p=0.012, respectively). Conclusion: The clinical protocol was a cost-neutral and sustainable intervention that effectively reduced the time taken to diagnose or exclude CES and ED waiting times.