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Abstract Disease-modifying therapeutics in the α-synucleinopathies multiple system atrophy (MSA) and Parkinson’s Disease (PD) are in early phases of clinical testing. Involving patients’ preferences including therapy-associated risk willingness in initial stages of therapy development has been increasingly pursued in regulatory approval processes. In our study with 49 MSA and 38 PD patients, therapy-associated risk willingness was quantified using validated standard gamble scenarios for varying severities of potential drug or surgical side effects. Demonstrating a non-gaussian distribution, risk willingness varied markedly within, and between groups. MSA patients accepted a median 1% risk [interquartile range: 0.001–25%] of sudden death for a 99% [interquartile range: 99.999–75%] chance of cure, while PD patients reported a median 0.055% risk [interquartile range: 0.001–5%]. Contrary to our hypothesis, a considerable proportion of MSA patients, despite their substantially impaired quality of life, were not willing to accept increased therapy-associated risks. Satisfaction with life situation, emotional, and nonmotor disease burden were associated with MSA patients’ risk willingness in contrast to PD patients, for whom age, and disease duration were associated factors. An individual approach towards MSA and PD patients is crucial as direct inference from disease (stage) to therapy-associated risk willingness is not feasible. Such studies may be considered by regulatory agencies in their approval processes assisting with the weighting of safety aspects in a patient-centric manner. A systematic quantitative assessment of patients’ risk willingness and associated features may assist physicians in conducting individual consultations with patients who have MSA or PD by facilitating communication of risks and benefits of a treatment option.

Abstract Background Parkinson’s disease (PD) significantly impacts the health-related quality of life of affected individuals and their relatives. In order to support the affected individuals and their families in coping with PD, it is essential to offer comprehensive information about their experiences. A comprehensive understanding of their lived experiences with the disease, the healthcare system, applied self-management strategies and their needs is considered crucial for developing a PD support program. Therefore, we aimed to explore the lived experiences and support needs of individuals with PD and their relatives in Germany. Methods This non-interventional, qualitative study conducted an explorative status quo and needs assessment. It generated knowledge through semi-structured focus groups and interviews with individuals with PD at various disease stages and their relatives. The interviews were digitally recorded, transcribed verbatim, and analysed using content analysis. Results Fifty-two individuals with PD and 29 relatives participated in eight focus groups and 13 paired and 13 individual interviews. Four themes with corresponding subthemes emerged: (1) experiences, revealing individuals’ experiences around their diagnosis and with disease-specific care provision; (2) management support offers, clarifying who provides support and the type of support offered; (3) self-management, including comprehensibility, meaningfulness and manageability; and (4) future needs, differentiating between deficits and needs. Most participants expressed a sense of abandonment when obtaining self-management strategies and mastering their lives with PD, often referred to as ‘life 2.0’. They identified the lack of structured and adequate provision of information, system orientation and social awareness. Conclusions In Germany, there is an urgent need for a comprehensive PD care program that addresses the needs of individuals with PD and their relatives from the start of their care trajectory. It could assist individuals in gaining a comprehensive understanding of the disease, obtaining self-management strategies, building a support network, and becoming experts in self-managing their disease. Moreover, it may positively influence their care trajectory and reduce burdens, such as overburdening, fear of progression, and health anxiety. Trial Registration German Clinical Studies Register ( https://www.drks.de/DRKS00030090 , No. DRKS00030090, Date of registration: 15.12.2022).

Background: The overall level of physical and psychological symptom burden of advanced cancer patients (ACP) in an outpatient setting is notoriously difficult to assess. Therefore, more efficient and objective assessment is needed to accomplish this important task. Objectives: The aim of this study was to compare the physical and psychological symptom burden rated by palliative care nurse (PCN) versus patient?s self-rating. Design: This retrospective German cohort study analyzed symptom burden using an electronic patient-reported outcome measure (ePROM). After referral to an outpatient specialized palliative care team, a PCN assessed the patient?s symptoms both up to three months before initial presentation (IP) and at IP. Group differences were identified using analyses of variance (ANOVA). Further descriptive analysis of patient characteristics was used. Subjects: The study enrolled 164 ACP who were referred to a specialized palliative care (SPC) team. Mean age was 62 (? 12.6) years. Gastrointestinal (n = 46; 28.0%), lung (n = 32; 19.5%), and breast cancer (n = 34; 20.7%) were the most common entities. Results: Most frequent reasons for referral were pain (n = 55; 33.5%) and social care problems (n = 36; 22.0%). Patients reported significantly higher grades on depression (n = 144; Z = ?2.8, p = 0.005), anxiety (n = 144; Z = ?2.376, p = 0.018), and worsened general condition (n = 139; Z = ?7.005, p < 0.001). Conclusion: ACP in an outpatient setting were more frequently referred to SPC for pain management and assistance with social problems with regard to the cancer and its limitations. Psychological distress was underrated by the PCN in comparison with patient self-reporting through ePROM. This underlines the importance of self-reported outcome measurement.

Abstract Background Pre-hospital endotracheal intubation (ETI) is a sophisticated procedure with a comparatively high failure rate. Especially, ETI in confined spaces may result in higher difficulty, longer times, and a higher failure rate. This study analyses if Helicopter Emergency Medical Services (HEMS) intubation (time-to) success are influenced by noise, light, and restricted space in comparison to ground intubation. Available literature reporting these parameters was very limited, thus the reported differences between ETI in helicopter vs. ground by confronting parameters such as time to secure airway, first pass success rate and Cormack-Lehane Score were analysed. Methods A systematic review and meta-analysis were conducted using PUBMED, EMBASE, Cochrane Library, and Ovid on October 15th, 2022. The database search provided 2322 studies and 6 studies met inclusion and quality criteria. The research was registered with the International Prospective Register of Systematic Reviews (CRD42022361793). Results A total of six studies were selected and analysed as part of the systematic review and meta-analysis. The first pass success rate of ETI was more likely to fail in the helicopter setting as compared to the ground (82,4% vs. 87,3%), but the final success rate was similar between the two settings (96,8% vs. 97,8%). The success rate of intubation in literature was reported higher in physician-staffed HEMS than in paramedic-staffed HEMS. The impact of aircraft type and location inside the vehicle on intubation success rates was inconclusive across studies. The meta-analysis revealed inconsistent results for the mean duration of intubation, with one study reporting shorter intubation times in helicopters (13,0s vs.15,5s), another reporting no significant differences (16,5s vs. 16,8s), and a third reporting longer intubation times in helicopters (16,1s vs. 15,0s). Conclusion Further research is needed to assess the impact of environmental factors on the quality of ETI on HEMS. While the success rate of endotracheal intubation in helicopters vs. on the ground is not significantly different, the duration and time to secure the airway, and Cormack-Lehane Score may be influenced by environmental factors. However, the limited number of studies reporting on these factors highlights the need for further research in this area.

Abstract Flexible endoscopic evaluation of swallowing (FEES) is one of the most important methods for instrumental swallowing evaluation. The most challenging part of the examination consists in the interpretation of the various observations encountered during endoscopy and in the deduction of clinical consequences. This review proposes the framework for an integrated FEES-report that systematically moves from salient findings of FEES to more advanced domains such as dysphagia severity, phenotypes of swallowing impairment and pathomechanisms. Validated scales and scores are used to enhance the diagnostic yield. In the concluding part of the report, FEES-findings are put into the perspective of the clinical context. The potential etiology of dysphagia and conceivable differential diagnoses are considered, further diagnostic steps are proposed, treatment options are evaluated, and a timeframe for re-assessment is suggested. This framework is designed to be adaptable and open to continuous evolution. Additional items, such as novel FEES protocols, pathophysiological observations, advancements in disease-related knowledge, and new treatment options, can be easily incorporated. Moreover, there is potential for customizing this approach to report on FEES in structural dysphagia.

Levodopa is the gold standard for the symptomatic treatment of Parkinson's disease (PD). There are well documented motor and non-motor fluctuations, however, that occur almost inevitably once levodopa is started after a variable period in people with PD. Whilst brain neurodegenerative processes play a part in the pathogenesis of these fluctuations, a range of barriers across the gastrointestinal (GI) tract can alter levodopa pharmacokinetics, ultimately contributing to non-optimal levodopa response and symptoms fluctuations. GI barriers to levodopa transport and absorption include dysphagia, delayed gastric emptying, constipation, Helicobacter pylori infection, small intestinal bacterial overgrowth and gut dysbiosis. In addition, a protein-rich diet and concomitant medication intake can further alter levodopa pharmacokinetics. This can result in unpredictable or sub-optimal levodopa response, 'delayed on' or 'no on' phenomena. In this narrative review, we provided an overview on the plethora of GI obstacles to levodopa transport and absorption in PD and their implications on levodopa pharmacokinetics and development of motor fluctuations. In addition, management strategies to address GI dysfunction in PD are highlighted, including use of non-oral therapies to bypass the GI tract.

IntroductionTreatment with chimeric antigen receptor T (CAR-T) cells involves a large number of interdisciplinary stakeholders and is associated with complex processes ranging from patient-specific production to follow-up care. Due to the complexity, maximum process optimization is required in order to avoid efficiency losses. This study aimed at systematically determining the preconditions for a frictionless flow of the CAR-T process by surveying the stakeholders involved.MethodsA Group Concept Mapping (GCM) analysis, a mixed-methods participatory research, was conducted. CAR-T experts from different professional backgrounds went through three steps: 1) Brainstorming relevant aspects (statements) for a frictionless process, 2) Sorting the collected statements based on their similarity, and 3) Rating the importance and feasibility of each statement. A cluster map reflecting the overarching topics was derived, and mean ratings per statement and cluster were calculated.ResultsOverall, 20 CAR-T experts participated. A total of 80 statements were collected, resulting in a map of the following 10 clusters (mean importance/feasibility): Information for patients and physicians (4.16/3.77), Supportive network (4.03/3.53), Eligibility of patients (4.41/3.63), Evidence, transparency and communication (4.01/3.33), Paperwork (4.1/2.52), Interface with pharmaceutical manufacturer (4.03/2.85), Reimbursement (4.29/2.31), Quality Management (4.17/3.18), Infrastructure of CAR-T clinics (4.1/2.93), and Patient-oriented processes (4.46/3.32).DiscussionThe 80 statements underlined the complex and manifold nature of the CAR-T treatment process. Our results reflect the first step in overcoming hurdles: identifying potential hurdles and required preconditions. Decision-makers and stakeholders can use the results to derive strategies and measures to further promote a frictionless process.

Abstract Background The Comprehensive Geriatric Assessment (CGA) records geriatric syndromes in a standardized manner, allowing individualized treatment tailored to the patient’s needs and resources. Its use has shown a beneficial effect on the functional outcome and survival of geriatric patients. A recently published German S1 guideline for level 2 CGA provides recommendations for the use of a broad variety of different assessment instruments for each geriatric syndrome. However, the actual use of assessment instruments in routine geriatric clinical practice and its consistency with the guideline and the current state of literature has not been investigated to date. Methods An online survey was developed by an expert group of geriatricians and sent to all licenced geriatricians (n = 569) within Germany. The survey included the following geriatric syndromes: motor function and self-help capability, cognition, depression, pain, dysphagia and nutrition, social status and comorbidity, pressure ulcers, language and speech, delirium, and frailty. Respondents were asked to report which geriatric assessment instruments are used to assess the respective syndromes. Results A total of 122 clinicians participated in the survey (response rate: 21%); after data cleaning, 76 data sets remained for analysis. All participants regularly used assessment instruments in the following categories: motor function, self-help capability, cognition, depression, and pain. The most frequently used instruments in these categories were the Timed Up and Go (TUG), the Barthel Index (BI), the Mini Mental State Examination (MMSE), the Geriatric Depression Scale (GDS), and the Visual Analogue Scale (VAS). Limited or heterogenous assessments are used in the following categories: delirium, frailty and social status. Conclusions Our results show that the assessment of motor function, self-help capability, cognition, depression, pain, and dysphagia and nutrition is consistent with the recommendations of the S1 guideline for level 2 CGA. Instruments recommended for more frequent use include the Short Physical Performance Battery (SPPB), the Montreal Cognitive Assessment (MoCA), and the WHO-5 (depression). There is a particular need for standardized assessment of delirium, frailty and social status. The harmonization of assessment instruments throughout geriatric departments shall enable more effective treatment and prevention of age-related diseases and syndromes.