BACKGROUND:Haemolytic disease of the foetus and newborn (HDFN) is an immune disorder driven by maternal alloimmunisation against foetal/newborn red blood cell antigens. HDFN can cause significant morbidity and mortality, with symptoms in the foetus ranging from mild anaemia to hydrops fetalis. While in newborns, HDFN can lead to severe forms of neonatal hyperbilirubinaemia and kernicterus. This systematic review (SR) aimed to identify and summarise real-world evidence (RWE) related to the patient burden/experience and economic burden of HDFN. METHODS:Electronic database searches supplemented by handsearching of grey literature, were conducted to identify studies that reported the clinical patient burden/experience, and economic burden of HDFN in Europe, the Middle East, and Africa (EMEA). Data from eligible studies were summarised in a narrative synthesis due to heterogeneity between studies. RESULTS:A total of 26 relevant publications were identified for inclusion in the SR, consisting of one study that directly measured Health Related Quality of Life, 9 studies reporting on proxy outcomes for patient burden and 18 studies reporting on economic burden (this includes two double-counted studies reporting more than one outcome type). Neurodevelopment, academic development, behaviour and personality were assessed as proxy outcomes for patient burden given the limited identification of patient-reported outcome data. These studies suggested potential neurodevelopmental impairments in children with HDFN. Despite these indirect insights into patient burden, identified data were limited and results should be interpreted with consideration of the inherent heterogeneity in design and endpoints assessed across RWE studies. Economic burden data were primarily limited to healthcare resource use outcomes, with limited reported data on healthcare costs, it is difficult to draw notable conclusions on the true economic burden of HDFN. CONCLUSIONS:The current SR provides a clear summary of the available evidence for the patient experience and economic burden of HDFN. While the limited evidence indicates that HDFN does confer a significant burden on patients, the review identifies the need for further well-powered and representative observational studies using well-defined outcome measures to aid a greater understanding of the burden and experience of HDFN. TRIAL REGISTRATION:The protocol for this systematic review was registered in PROSPERO CRD42022328444.

Background Myasthenia gravis (MG) is a rare autoimmune disease characterised by muscle weakness, and progression from ocular (oMG) to generalised (gMG) symptoms results in a substantial negative impact on quality of life (QoL). This systematic review aimed to provide an overview of the patient burden experienced by people living with gMG. Methods Electronic database searches (conducted March 2022), supplemented by interrogation of grey literature, were conducted to identify studies reporting patient burden outcomes in patients with gMG in Europe, the Middle East and Africa. Results were synthesised narratively due to the heterogeneity across trials. Results In total, 39 patient burden publications (representing 38 unique studies) were identified as relevant for inclusion in the systematic review, consisting of 37 publications reporting formal patient-reported outcome measures (PROMs), and two publications describing alternative qualitative assessments of patient experience. The studies included a variety of measures including generic and disease-specific PROMs, as well as symptom-specific PROMs focusing on key comorbidities including depression, anxiety, fatigue and sleep disturbance. The findings showed some variation across studies and PROMs; however, in general there was evidence for worse QoL in patients with gMG than in healthy controls or in patients with oMG, and a trend for worsening QoL with increasing MG severity. Conclusions This review highlights the importance of considering patient QoL when developing and assessing treatment and management plans for patients with gMG. However, the heterogeneity identified across studies illustrates the need for further representative and well-powered studies in large cohorts administering consistent, validated questionnaires. Trial registration The protocol for this systematic review was registered in PROSPERO: CRD42022328444.

Generalized Myasthenia Gravis (gMG) is a chronic, auto-antibody, neuromuscular disease. Diagnosis can be difficult as symptoms, such as fatigue, are often mistaken for a range of other disorders. A timely diagnosis is important to effectively manage the disease, reduce patient anxiety, improve patient quality of life, and avoid unnecessary healthcare resource use. The Adelphi MG Disease Specific Programme (DSP™) collected point-in-time data from a cross-sectional cohort of physicians and their patients with gMG (defined as MGFA class II-V) across France, Germany, Italy, Spain and the UK between March – September 2020. Physicians provided data including demographics, diagnostic pathway and their perception of disease impact. Patients were invited to complete a follow-up form, paired to their physicians, which included the MG-QoL-15r PRO tool. 191 physicians provided data for 387 gMG patients with a known diagnosis date. 53.9% were female, mean age was 52.5 (SD±15.69) and mean time from diagnosis to survey was 4.2 years (SD±5.66). Mean time from symptom onset to gMG diagnosis was 1.0 years (SD±1.43). 105 patients (27.1%) received a gMG diagnosis more than a year after the onset of symptoms. At the time of survey, these patients were more likely to experience moderate or higher levels of fatigue (78.1%) and anxiety (75.2%) than those diagnosed within a year (64.7% and 56.2% respectively). 117 patients completed the MG-QoL-15r. Those with a delayed diagnosis (n=43) had higher impairment (14.4; SD±5.50) than those diagnosed within a year (12.6; SD±7.84). Physicians reported patients with diagnoses taking longer than a year experienced more fatigue, anxiety, and prolonged burden on health-related quality of life, leading to higher unnecessary health care resource utilization in patients with gMG. These findings underscore the importance of a timely diagnosis of gMG after symptom onset and the need to properly educate all stakeholders on optimal disease management strategies.

Generalized Myasthenia Gravis (gMG) is a chronic, autoantibody condition causing muscle weakness. There is no cure, however a range of pharmacological treatments are currently prescribed, typically acetylcholinesterase inhibitors (AChEIs), corticosteroids, and non-steroidal immunosuppressants (NS-ISTs).

Background: Readmission rates are often seen as a marker of quality of hospital care.We wanted to establish how many patients, admitted to our Care of the Elderly wards, were readmitted within 28 days and if these were avoidable.Furthermore, particularly in light of recent data (Clark D et al.Palliative Medicine: 2014, 28: 474-479) showing 29% of adult hospital inpatients were dead by 1 year, we wanted to establish the proportion of our patients in their last year of life to establish if the unit should be more actively encouraging anticipatory care planning.Sampling methods: 100 consecutive admissions, in June/July 2011, to Acute Elderly Care wards at Wishaw General Hospital were reviewed using the TRAKcare patient management system.Data was gathered on length of stay, readmissions and death.Casenotes,

extra and in our own time, with outside funding required to support larger studies. It is our opinion that as a minimum standard, laboratories should select a reference interval that is supported by findings in the peer-reviewed literature relating specifically to the assay they are using. Routine laboratories can verify that their assay measures ‘normal’ troponin concentrations that are below the 99th percentile URL by using blood samples from young males and females, e.g. medical students. As a minimum standard for imprecision testing, routine laboratories should verify the withinand between-run precision capability of their assay over the troponin measuring range concentration, e.g. CLSI guideline EP15-A2. The Study Group on Biomarkers in Cardiology of the European Society of Cardiology Working Group on Acute Cardiac Care recommends determination of at least one level close to the clinical decision limit for troponin, which may be the 99th percentile of a reference population value distribution. This enables verification of the limit of quantitation that is clinically relevant. Adequate long-term monitoring of troponin imprecision is also essential to avoid wrong results in clinical samples and to confirm troponin transferability and accuracy across different reagent lots. Ultimately it is buyer beware. A doctor using a pathology laboratory relies on them to produce quality results. When all is considered, it is the responsibility of each laboratory to put out meaningful, quality troponin data.