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Background: Anorectal malformations (ARMs) are congenital anomalies of the anorectum and the genitourinary system that result in a broad spectrum of hindgut anomalies. Despite surgical correction patients continue to have late postoperative genitourinary and colorectal dysfunction that have significant impact on quality of life., Aim: This paper will review the current evidence and discuss the evaluation and management of postoperative patients with ARMs who present with persistent defecation disorder., Methods: A literature search was conducted using PubMed/MEDLINE/EMBASE databases applying the following terms: ARMs, imperforate anus, constipation, faecal incontinence, neurogenic bowel, posterior sagittal anorectoplasty., Results: Patients who present with postoperative defecation disorders require timely diagnostic and surgical evaluation for anatomic abnormalities prior to initiation of bowel management. Goals of management are to avoid constipation in young children, achieve faecal continence in early childhood and facilitate independence in older children and adolescents. Treatment options vary from high dose stimulant laxatives to high-volume retrograde and antegrade enemas that facilitate mechanical colonic emptying., Conclusions: Appropriate diagnostic work-up and implementation of treatment can decrease long-term morbidity and improve quality of life in postoperative patients with ARMs who presents with defecation disorders., (© 2024 John Wiley & Sons Ltd.)

Heart failure (HF) is common and associated with a poor prognosis, despite advances in treatment. Over the last decade cardiovascular outcome trials with sodium–glucose co‐transporter 2 (SGLT2) inhibitors in patients with type 2 diabetes mellitus have demonstrated beneficial effects for three SGLT2 inhibitors (empagliflozin, canagliflozin and dapagliflozin) in reducing hospitalisations for HF. More recently, dapagliflozin reduced the risk of worsening HF or death from cardiovascular causes in patients with chronic HF with reduced left ventricular ejection fraction, with or without type 2 diabetes mellitus. A number of additional trials in HF patients with reduced and/or preserved left ventricular ejection fraction are ongoing and/or about to be reported. The present position paper summarises recent clinical trial evidence and discusses the role of SGLT2 inhibitors in the treatment of HF, pending the results of ongoing trials in different populations of patients with HF. [ABSTRACT FROM AUTHOR]

Heart failure (HF) is a long‐term clinical syndrome, with increasing prevalence and considerable healthcare costs that are further expected to increase dramatically. Despite significant advances in therapy and prevention, mortality and morbidity remain high and quality of life poor. Epidemiological data, that is, prevalence, incidence, mortality, and morbidity, show geographical variations across the European countries, depending on differences in aetiology, clinical characteristics, and treatment. However, data on the prevalence of the disease are scarce, as are those on quality of life. For these reasons, the ESC‐HFA has developed a position paper to comprehensively assess our understanding of the burden of HF in Europe, in order to guide future policies for this syndrome. This manuscript will discuss the available epidemiological data on HF prevalence, outcomes, and human costs—in terms of quality of life—in European countries. [ABSTRACT FROM AUTHOR]

Evidence exists regarding the impact of flourishing in individuals living with chronic pain, but there are currently no reviews which collate the literature on flourishing in adolescents living with chronic pain and their parents. Therefore, the aim of this scoping review was to map and review the current literature, to document how flourishing is defined and understood in the literature, and to identify gaps in the field. Six databases were searched (Web of Science, Medline, Embase, APA PsycNet and the Cochrane Central Register of Controlled Trials). In addition, a limited gray literature search was conducted. The resulting data were collated and reported in relation to the review questions, by examining the included papers to search for the presence of flourishing. Database searches resulted in 7326 papers after duplicate removal, with eight remaining papers being assessed for full‐text eligibility. Following full‐text screening, a final four papers were included in the review. Within the papers, flourishing was defined in relation to commonalities of benefit finding, enhanced maturity and growth, and social support. Gaps in the literature and directions for future research are considered. This review suggests that there is a dearth of knowledge and research regarding flourishing among adolescents living with chronic pain and their parents, despite aspects of flourishing identified in limited literature. This warrants further investigation. [ABSTRACT FROM AUTHOR]

Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder, in which rituximab (RTX) induces the best long-term effect among recommended second-line treatments. Nevertheless, the optimal regimen of RTX remains unclear. We herein conducted a prospective, multicenter, open-label, randomized controlled trial to compare the efficacy and safety of RTX at two different dosage regimens in patients with corticosteroid-resistant or relapsed ITP. Recruited patients were randomly assigned (1:1) to receive either RTX at a repeated low dose (100 mg weekly for 4 weeks, LD-RTX) or at a single dose (375 mg/m 2 , S-RTX). Overall response was achieved in 64.3% of patients who received LD-RTX versus 67.4% of those receiving S-RTX (p = .759). The complete response (CR) rate was 23.8% after LD-RTX and 28.3% after S-RTX (p = .635). In health-related quality of life, S-RTX improved patients' psychological status, quality of life, social activities, and work compared with LD-RTX. Furthermore, S-RTX significantly reduced physician visits without compromising efficacy. Our findings demonstrate that a S-RTX is comparable to LD-RTX in effectiveness and safety for treatment of corticosteroid-resistant or relapsed ITP. The single-dosage regimen optimizes the use of medical resources, improves the cost-effectiveness of RTX, and represents a promising and more convenient replacement for LD-RTX in ITP. This study has been completed and is registered with ClinicalTrials.gov, number NCT03258866., (© 2022 Wiley Periodicals LLC.)

Background: A person‐centred approach in the context of health services delivery implies a biopsychosocial model focusing on all factors that influence the person's health and functioning. Those wishing to monitor change should consider this perspective when they develop and use guidelines to stimulate active consideration of the person's needs, preferences and participation in goal setting, intervention selection and the use of appropriate outcome measures. Objective: To develop a position paper that promotes a person‐centred approach in guideline development and implementation. Design, setting and participants: We used three narrative discussion formats to collect data for achieving consensus: a nominal group technique for the Allied Health Steering Group, an Internet discussion board and a workshop at the annual G‐I‐N conference. We analysed the data for relevant themes to draft recommendations. Results: We built the position paper on the values of the biopsychosocial model. Four key themes for enhancing a person‐centred approach in clinical guidelines emerged: (i) use a joint definition of health‐related quality of life as an essential component of intervention goals, (ii) incorporate the International Classification of Functioning, Disability and Health (ICF) as a framework for considering all domains related to health, (iii) adopt a shared decision‐making method, and (iv) incorporate patient‐reported health outcome measures. The position statement includes 14 recommendations for guideline developers, implementers and users. Conclusion: This position paper describes essential elements for incorporating a person‐centred approach in clinical guidelines. The consensus process provided information about barriers and facilitators that might help us develop strategies for implementing person‐centred care. [ABSTRACT FROM AUTHOR]

Thanks to advances in technology such as Internet-based mapping and the development of special inks, papers and global positioning systems, map making for vision impaired people can provide a range of products (e.g., tactile, haptic and audio-tactile maps) that can improve their independence, self-confidence and everyday life. The applications of tactile maps include learning spatial concepts and geography; audio-tactile maps combined with access to geospatial information can enhance mobility and independence. In 1998, the Mapping Services Branch (MSB) of the Earth Sciences Sector of Natural Resources Canada initiated a tactile mapping program. Now referred to as the Government on Line—Mapping for the Visually Impaired Project (Natural Resources Canada 2003a), it aims to serve the community with special needs, with emphasis on those who are blind or visually impaired, their teachers and mobility instructors. This technical paper discusses the various types of tactile and audio-tactile maps of Canada developed by the MSB in cooperation with its partners and describe the current research and development activities carried out within the project. It also leads the reader to further information on audio-tactile maps and touch- and sound-based computer interfaces. [ABSTRACT FROM AUTHOR]

The White Paper on Chinese population policy, entitled "Family Planning in China," was issued by the Chinese Information Office of the State Council on August 23, 1995. The population problem is an important question that touches upon the survival and development of the Chinese nation, the success or failure of China's modernization drive as well as the coordinated and sustained development between the population on one hand and the economy, society, resources and environment on the other. It is a natural choice that the Chinese government has made to implement family planning, control population growth and improve the life quality of the population a basic state policy on the basis of a wish to make the state strong and powerful, the nation prosperous and the people happy. In the 1960s, the country's population entered its second peak birth period. From 1962 to 1972, the annual number of births in China averaged 26.69 million, totaling 300 million. In the practice of carrying out family planning programme, whilst persistently proceeding from its reality and taking into full account and observing principles and regulations concerning population and family planning formulated by international institutions and organizations, the Chinese government has gradually set up guiding principles, policies, measures and methods that reflect the basic interests and various rights and interests of the people and has continuously improved these as the actual situations change, so as to better safeguard the right to subsistence and development of the Chinese region.

Introduction: This paper provides a critical reflection from both the researcher and public contributor (PC) perspective on the benefits and the learnings taken from involving PCs in research related to Parkinson's. Approach to Patient and Public Involvement (PPI): This paper reports on how PCs shaped the design and development of the PRIME‐UK research programme study materials through input into information leaflets, consent forms and other patient‐facing documents used across three studies within the PRIME‐UK research programme. The PRIME‐UK research programme is designed to improve the quality of life of people with Parkinson's and this project included three studies: a cross‐sectional study, a randomised control trial and a qualitative study. We captured these impacts using Public Involvement Impact Logs, which provide a framework allowing researchers and PCs to report on the learnings, immediate outcomes and impacts from PPI. For this project, the impact logs enabled us to provide reflections from PCs and researchers on the process of involving 'the public' in Parkinson's research. Findings: This paper builds on existing evidence of the range of benefits and challenges that emerge from working with patients and the public in Parkinson's research; this includes reflecting on the changes made to the study materials and benefits for the people involved. Four themes emerged from the reflections that were common to the researchers and PCs; these were the importance of providing a supportive environment; recognition of the benefit of the evaluation of the impact of PPI; acknowledgement that engagement of PPI can make a positive difference to the research process and that timely communication and the use of face‐to‐face communication, where available, is key. Furthermore, we demonstrate how impact logs provide a useful and straightforward tool for evaluating public involvement practices and supporting the feedback process. Conclusion: We offer key recommendations for involving patients and the public in Parkinson's research and suggest approaches that could be implemented to capture the impacts of public involvement. Public Contribution: Public contributors (PCs) were involved in the design and development of the participant information leaflets, consent forms and other patient‐facing documents used for studies within the PRIME‐UK research programme. In addition, PCs evaluated their involvement using impact logs and co‐authored this paper. [ABSTRACT FROM AUTHOR]

Objectives: The aim of the present study was to follow the daily course of patients with olfactory dysfunction and healthy controls and to assess (i) how many times a day, (ii) at which time, and (iii) in which aspect of daily life participants are conscious about their sense of smell. Methods: In this longitudinal study, 49 patients with smell loss and 30 healthy participants were enrolled. Olfactory function was assessed using the Sniffin' Sticks. All participants received paper diaries designed for a 14-day period, featuring 12 rows representing 12 daily hours and six columns for various daily life aspects. They were instructed to mark their awareness of smell by indicating the relevant row and column in the diary. Following the return of the diaries, a second olfactory test was conducted within the patient group. Results: On average, patients were consciously aware of their sense of smell around 8 times daily, while healthy participants noted it about 6.5 times a day. Both groups primarily focused on their sense of smell during activities related to "eating," followed by considerations in "social life" and "personal hygiene." Interestingly, distinct patterns emerged: patients peaked in awareness at 8 a.m. and 7 p.m., whereas healthy individuals showed peaks at 6 a.m., 12 p.m., and 7 p.m. Despite regular diary use, we observed no improvement in patients' olfactory function or related quality of life. Conclusion: The olfactory diary is a valuable tool unveiling individual smell awareness patterns in patients with smell loss, aiding in counseling and patient management. [ABSTRACT FROM AUTHOR]

Traditionally, the main indication for cardiopulmonary exercise testing (CPET) in heart failure (HF) was for the selection of candidates to heart transplantation: CPET was mainly performed in middle-aged male patients with HF and reduced left ventricular ejection fraction. Today, CPET is used in broader patients' populations, including women, elderly, patients with co-morbidities, those with preserved ejection fraction, or left ventricular assistance device recipients, i.e. individuals with different responses to incremental exercise and markedly different prognosis. Moreover, the diagnostic and prognostic utility of symptom-limited CPET parameters derived from submaximal tests is more and more considered, since many patients are unable to achieve maximal aerobic power. Repeated tests are also being used for risk stratification and evaluation of intervention, so that these data are now available. Finally, patients, physicians and healthcare decision makers are increasingly considering how treatments might impact morbidity and quality of life rather than focusing more exclusively on hard endpoints (such as mortality) as was often the case in the past. Innovative prognostic flowcharts, with CPET at their core, that help optimize risk stratification and the selection of management options in HF patients, have been developed. [ABSTRACT FROM AUTHOR]

Aim: To gain a greater understanding of how compression therapy affects quality of life, this systematic review appraised existing published studies measuring the impact of compression therapy on health quality of life (HRQoL), and pain, among people with venous leg ulcers (VLU). Method: Five databases were searched, and two authors extracted data and appraised the quality of selected papers using the RevMan risk of bias tool. Due to heterogeneity in the types of compression and instruments used to evaluate HRQoL, meta‐analysis was not appropriate; thus, a narrative synthesis of findings was undertaken. Results: Ten studies were included, 9 RCTs and one before‐after study. The studies employed nine different HRQoL tools to measure the impact of a variety of compression therapy systems, with or without an additional exercise programme, versus other compression systems or usual care, and the results are mixed. With the use of the Cardiff Cardiff Wound Impact Schedule, the SF‐8 and the SF‐12, study authors found no differences in QoL scores between the study groups. This is similar to one study using QUALYs (Iglesias et al., 2004). Conversely, for studies using EuroQol‐5D, VEINES‐QOL, SF‐36 and CIVIQ‐20 differences in QoL scores between the study groups were noted, in favour of the study intervention groups. Two further studies using QUALYs found results that favoured a two‐layer cohesive compression bandage and the TLCCB group, respectively. Results for the five studies that assessed pain are also mixed, with one study finding no difference between study groups, one finding that pain increased over the study period and three studies finding that pain reduced in the intervention groups. All studies were assessed as being at risk of bias in one or more domains. Conclusion: Results were varied, reflecting uncertainty in determining the impact of compression therapy on quality of life and pain among people with a venous leg ulcer. The heterogeneity of the compression systems and the measures used to evaluate HRQoL make it a challenge to interpret the overall evidence. Further studies should strive for homogeneity in design, interventions and comparators to enhance both internal and external validity. [ABSTRACT FROM AUTHOR]

Scars are fibrous tissues that replace normal tissue during the wound healing process. Scarring can lead to low self‐esteem, social impairment, depression, anxiety, and other psychiatric and psychological distress, necessitating a comprehensive understanding of the latest perspectives, topical research, and directions in scarring‐mental health. This is a biblioshiny and VOSviewer based bibliometric analysis study. All data were obtained from the Web of Science, and a total of 664 articles from 2003 to 2022 met the criteria. The last 7 years have been a period of rapid growth in the field, with 2022 having the highest number of articles. The United States is the core country with the highest production and citation rate. The most cited literature was written in 2003 by Van Loey NE et al. Van Loey NE is the most prolific and influential author in this field. The top five popular keywords include "quality of life", "depression", "management", "anxiety", and "prevalence". The paper concludes that the current focus of scholars in the field is on the treatment of scars and that multidisciplinary treatment of such patients is worth exploring. These findings provide relevant researchers with the current state of research and possible future directions in this field. [ABSTRACT FROM AUTHOR]

Aim: To describe the life situation of spouses having a partner with heart disease and adolescents living at home. Design: Qualitative inductive design. Method: Participants (n = 22) were included from three Scandinavian countries. Semi‐structured interviews were analysed using thematic analysis with an inductive and latent approach. Results: Three themes were derived. 'Being in spousal and parental role transition' described how daily life had been affected and parental responsibilities had been doubled due to their partner's heart disease. 'Living with unpredictability and insecurity' included how the unpredictable illness trajectory caused worries and affected the well‐being of the family. 'Managing a challenging life situation' highlights how spouses coped with their partners' heart disease and adapted to a new life situation. Conclusion: Young spouses' life situation was greatly affected by their partner's heart disease, resulting in increased responsibilities and double parenthood. Having a positive attitude and mindset towards life was used as a strategy to cope with the changed life situation and find a new way of life. Implications for the Profession and/or Patient Care: All family members are affected by heart disease. Spouses needed additional professional support and guidance on how to involve the children when a parent is ill. Impacts: This study highlights how young spouses, with adolescents living at home, experience their life situation.The life situation is unpredictable due to the partner's heart disease, as they must handle both caring for their partner and taking on double parenthood.Research involving family members can improve person‐ and family‐centred care and treatment outcomes in health care and society. Reporting Method: COREQ checklist was used preparing the manuscript. Patient or Public Contribution: Data collection included interviews with spouse. What Does This Paper Contribute to the Wider Global Clinical Community?: By highlighting the spouses changed life situation due to heart disease and the importance of including them in health care. [ABSTRACT FROM AUTHOR]

Background: Merkel cell carcinoma is a rare skin cancer associated with poor survival. Based on a previous Phase II trial of adults with advanced Merkel cell carcinoma by Kim and colleagues (2022), there is now a strong rationale for combination therapy (i.e., nivolumab and ipilimumab) to become a treatment option for patients with advanced Merkel cell carcinoma. The goal of this paper was to report on the secondary outcome of quality of life (QOL) among patients on this trial. Methods: Patients receiving combined nivolumab and ipilimumab, with or without stereotactic body radiation therapy (SBRT), completed the European Organisation for Research and Treatment of Cancer (EORTC) QLQ‐C30 prior to starting treatment and every 2 weeks thereafter. Changes in QOL during treatment and post‐treatment were evaluated using piecewise random‐effects mixed models. Exploratory analyses compared changes in QOL between study arms. The original trial was registered with ClinicalTrials.gov (NCT03071406). Results: Study participants (n = 50) reported no changes in overall QOL (ps > 0.05), but emotional functioning improved during treatment (p = 0.01). Cognitive and social functioning worsened post‐treatment (ps < 0.01). In general, patients treated with combination therapy only (n = 25) reported no change in QOL over time, whereas patients also treated with SBRT (n = 25) consistently demonstrated worsening QOL post‐treatment. Conclusion: QOL is generally preserved in patients treated with combination therapy, but the addition of SBRT may worsen QOL. Combined with clinical efficacy data published previously, results support the use of combination therapy with nivolumab and ipilimumab as a treatment option for patients with advanced Merkel cell carcinoma. [ABSTRACT FROM AUTHOR]

Objective: Although Ireland has one of the highest levels of well‐being in Europe, having a health condition has been found to have a direct negative impact. The aim of this study is to evaluate the current situation and the experiences of patients with epidermolysis bullosa (EB), a rare genetic skin disease, and their relatives living in Ireland, with a focus on burdens and helpful practices. Methods and Measures: In a mixed‐methods design, a series of standardised questionnaires were combined with open‐ended questions. Via an online survey, data from n = 59 EB patients and relatives of EB patients living in Ireland were collected. Results: EB affects both the patients and their relatives. Burdens were found in relation to the visibility of EB, the degree of severity, the current health status, reduced mobility, the financial impact of EB, the psychosocial impact and personal and social resources. The paper also analyses existing resources and highlights opportunities for support and needs of improvement. Conclusion: Quality of life with EB is influenced by somatic symptoms and the psychosocial burden. Individual helpful practices in dealing with this rare disease can be considered as mediators, but they need to be supported by structural and healthcare improvements. Patient or Public Contribution: The perspective of EB patients, their relatives and EB experts were taken into account in the development of the study design via two feedback loops with the EB patient organisations DEBRA Ireland and DEBRA Austria. The design was adapted accordingly. Additionally, by including open‐ended questions, patients and relatives could contribute their individual perspectives and add insights into their lives with EB that might not have been captured with the structured online survey alone. [ABSTRACT FROM AUTHOR]

Background: Motor neuron disease (MND) (also known as amyotrophic lateral sclerosis) is a life‐limiting neurodegenerative condition. In up to 20% of people with MND, a pathogenic variant associated with autosomal dominant inheritance can be identified. Children of people carrying a pathogenic variant have a 50% chance of inheriting this and a higher, although harder to predict, chance of developing the disease compared to the general adult population. This paper explores the experience of living with the genetic risk of MND. Methods: We undertook a UK‐based interview study with 35 individuals, including: 7 people living with genetically‐mediated forms of MND; 24 asymptomatic relatives, the majority of whom had an increased risk of developing the disease; and 4 unrelated partners. Results: We explore how individuals make sense of genetic risk, unpacking the interplay between genetic knowledge, personal perception, experiences of the disease in the family, age and life stage and the implications that living with risk has for different aspects of their lives. We balance an emphasis on the emotional and psychological impact described by participants, with a recognition that the salience of risk fluctuates over time. Furthermore, we highlight the diverse strategies and approaches people employ to live well in the face of uncertainty and the complex ways they engage with the possibility of developing symptoms in the future. Finally, we outline the need for open‐ended, tailored support and information provision. Conclusions: Drawing on wider literature on genetic risk, we foreground how knowledge of MND risk can disrupt individuals' taken‐for‐granted assumptions on life and perceptions of the future, but also its contextuality, whereby its relevance becomes more prominent at critical junctures. This research has been used in the development of a public‐facing resource on the healthtalk.org website. Patient or Public Contribution: People with experience of living with genetic risk were involved throughout the design and conduct of the study and advised on aspects including the topic guide, sampling and recruitment and the developing analysis. Two patient and public involvement contributors joined a formal advisory panel. [ABSTRACT FROM AUTHOR]

Aim: This paper describes the trajectory during 1 year of four patient‐reported outcomes (PROs), namely, sleep, depressive symptoms, health‐related quality of life (HrQoL), and well‐being, in patients with heart failure (HF), their relationship and the patient characteristics associated with changes in these PROs. Methods and results: Data analyses of PROs from 603 patients (mean age 67 years; 29% female, 60% NYHA II) enrolled in the HF‐Wii study. On short term, between baseline and 3 months, 16% of the patients experienced continuing poor sleep, 11% had sustained depressive symptoms, 13% had consistent poor HrQoL, and 13% consistent poor well‐being. Across the entire 1‐year period only 21% of the patients had good PRO scores at all timepoints (baseline, 3, 6, and 12 months). All others had at least one low score in any of the PROs at some timepoint during the study. Over the 12 months, 17% had consistently poor sleep, 17% had sustained symptoms of depression, 15% consistently rated a poor HrQoL, and 13% poor well‐being. Different patient characteristics per PRO were associated with a poor outcomes across the 12 months. Age, education, New York Heart Association, and length of disease were related to two PRO domains and submaximal exercise capacity (6 min test), co‐morbidity, and poor physical activity to one. Conclusion: In total, 79% of the patients with HF encountered problems related to sleep, depressive symptoms, HrQoL, and well‐being at least once during a 1‐year period. This underscores the need for continuous monitoring and follow‐up of patients with HF and the need for dynamic adjustments in treatment and care regularly throughout the HF trajectory. [ABSTRACT FROM AUTHOR]

Background: People with motor neuron disease (pwMND) are routinely offered gastrostomy feeding tube placement and (non‐invasive and invasive) ventilation to manage the functional decline associated with the disease. This study aimed to synthesise the findings from the qualitative literature to understand how individual, clinical team and organisational factors influence pwMND decisions about these interventions. Methods: The study design was guided by the enhancing transparency in reporting the synthesis of qualitative research (ENTREC) statement. The search of five bibliography databases and an extensive supplementary search strategy identified 27 papers that included qualitative accounts of pwMND, caregivers and healthcare professionals' (HCPs) experiences of making decisions about gastrostomy and ventilation. The findings from each study were included in a thematic synthesis. Findings: Making decisions about interventions is an emotional rather than simply a functional issue for pwMND. The interventions can signal an end to normality, and increasing dependence, where pwMND consider the balance between quality of life and extending survival. Interactions with multiple HCPs and caregivers can influence the process of decision‐making and the decisions made. These interactions contribute to the autonomy pwMND are able to exert during decision‐making. HCPs can both promote and threaten pwMND perceived agency over decisions through how they approach discussions about these interventions. Though there is uncertainty over the timing of interventions, pwMND who agree to interventions report reaching a tipping point where they accept the need for change. Conclusion: Discussion of gastrostomy and ventilation options generate an emotional response in pwMND. Decisions are the consequence of interactions with multiple external agents, including HCPs treading a complex ethical path when trying to improve health outcomes while respecting pwMND right to autonomy. Future decision support interventions that address the emotional response and seek to support autonomy have the potential to enable pwMND to make informed and timely decisions about gastrostomy placement and ventilation. Patient or Public Contribution: The lead author collaborated with several patient and participant involvement (PPI) groups with regards to the conceptualisation and design of this project. Decisions that have been influenced by discussions with multiple PPI panels include widening the scope of decisions about ventilation in addition to gastrostomy placement and the perceptions of all stakeholders involved (i.e., pwMND, caregivers and HCPs). [ABSTRACT FROM AUTHOR]

Aims: The aim of this study was to analyse the status, hotspots and trends of research on oral care for the elderly in mainland China in the past 20 years through bibliometrics, which aims to provide new ideas and targets for future clinical work and research. Design: Bibliometric analysis. Methods: Relevant literature was retrieved from China National Knowledge Infrastructure, Wanfang, China Science and Technology Journal Database, Web of Science and PubMed. NoteExpress, Co‐Occurrence and CiteSpace were used to analyse bibliometric features in terms of year of publication, journal of publication, authors, institution and keywords. Results: A total of 716 related articles were obtained. The number of publications shows an increasing trend over time, only 2017–2021; 309 papers were published, accounting for 43.2% of the total number of publications. A total of 238 articles were published in Science Citation Index journals or Chinese core journals, accounting for 33.2% of the total number of articles. The study of oral health‐related quality of life in the elderly is a hot research topic. There is a lack of research on the elderly living in elderly care facilities. [ABSTRACT FROM AUTHOR]

The aim of this review is to revisit the meaning of common concepts and frameworks promoted to capture subjective outcomes of patients, the content of their corresponding measurements, and the preferred sources of the information of interest. This is important because conceptualizations of 'health' and the subject evaluations thereof continue to evolve. Related but distinct concepts like quality of life (QoL), health‐related QoL (HRQoL), functional status, health status, and well‐being are often used indiscriminately to assess clinical impacts of interventions and to influence decisions about patient care and policymaking. The discussion addresses and illustrates the following issues: (1) the required features of effective and valid health‐related concepts; (2) understanding underlying factors that often create confusion about QoL and HRQoL; and (3) how these concepts provide insight into, and promote, health in the context of populations with neurodisability. The hope is to illustrate how a combination of a clear research question, a hypothesis, conceptualization of the required outcomes, and operational definitions of the domains and items of interest, including item mapping, can help to achieve robust methodology and valid findings beyond the required psychometric properties. What this paper adds: The language, content, and the source of perceived health and life issues are clarified.Using the same terms for different constructs, or different terms for the same constructs, creates confusion and hinders outcome research.The challenges of using patient‐reported outcomes in neurodisability are addressed. What this paper adds: The language, content, and the source of perceived health and life issues are clarified.Using the same terms for different constructs, or different terms for the same constructs, creates confusion and hinders outcome research.The challenges of using patient‐reported outcomes in neurodisability are addressed. [ABSTRACT FROM AUTHOR]

This paper provides a general framework for conceptualizing triage for intensive care unit admissions in public health emergencies such as the COVID‐19 pandemic. It applies this framework to some of the guidelines issued during the pandemic and addresses some controversial issues, including the role of age, the use of lives or life years, and the relevance of quality of life considerations. The paper defends a view on which triage protocols for public health emergencies should aim to maximize the number of life years saved, may take into account age as a proxy, and should ignore quality of life considerations. [ABSTRACT FROM AUTHOR]

Aim: The aim of this research is to identify the main approaches and domains of palliative care quality assessment through three questionnaires used for this purpose. Design: Systematic review. Methods: The proposed analysis process consists of three stages from 2000 to 2020: (i) massive literature search, (ii) text mining and (iii) systematic reviews carried out on the QLQ C30, Zarit Burden Interview and FAMCARE questionnaires. The Preferred Reporting Items for Systematic Reviews (PRISMA‐P) have guided our research. Results: Sixteen papers were included in our study. The main findings have been summarised using a descriptive narrative synthesis approach. Systematic reviews evidenced that such tools present variable factor structures or latent domains. The results obtained are generally representative of the evidence supporting the factor structure of the QLQ‐C30 in the general cancer population. The factor structure of the Zarit Burden Interview remains ambiguous, although the idea of a unifactorial structure predominates. In the case of FAMCARE, most of the factor structures differ from the initial proposal of Kristjanson. The categorisation of the main subjective assessment approaches could be useful for the construction of a coherent system of indicators to be used in nursing practice. For its part, the variability in the latent dimensionality of the questionnaires analysed could be due to: (i) the characteristics of the sample, (ii) the population studied, (iii) cross‐cultural variability, (iv) the design of the questionnaire and (v) the analysis techniques employed. [ABSTRACT FROM AUTHOR]

There is a drastic increase in urbanization over the past few years, which requires energy‐efficient and optimized solutions for transportation, governance, quality of life in a smart city among all the citizens. The Internet‐of‐Energy (IoE) ecosystem offers many sophisticated and ubiquitous applications for smart cities. The energy demand of IoE applications is increased while IoE devices continue to grow. Therefore, smart city solutions must have the ability to utilize energy and handle the associated challenges efficiently. Moreover, energy Optimization (EO) techniques can be used to reduce energy consumption to meet the sustainability goals in IoE. Different techniques have been proposed for EO in various fields by researchers worldwide. Computing systems also need energy optimization. The energy consumption in the data center, clouds, and blockchain (BC)‐based architectures are a point of concern at the current time. Due to the enormous energy demands of these systems, we cannot take advantage of the latest technologies to their fullest. Due to the emergence of new technologies and some limitations of proposed techniques, we can still not optimize energy usage more than some extent. There is minimal exploration done in energy optimization in BC‐based systems. In this paper, we have proposed a survey on the energy optimization techniques in various systems, including the optimization techniques in BC‐based systems. We have proposed a taxonomy that classifies energy optimization techniques. We have also proposed an energy‐efficient consensus mechanism, Proof‐of‐High Performance optimization (named as PoHPo), for High‐Performance Computing (HPC) based ecosystems. The open issues and challenges are then discussed in EO. The survey intends to propose future directions for industry professionals, green‐energy stakeholders, and researchers worldwide to explore this topic further. [ABSTRACT FROM AUTHOR]

Aim: To test the efficacy of an integrated microfinance/livelihood and community‐based rehabilitation (IMCBR) programme in improving health‐related quality of life (HRQoL) and motor function of children with cerebral palsy (CP) and gain in social capital to their ultra‐poor families in rural Bangladesh. Method: This was an open‐label cluster randomized control trial. Children with CP aged 5 years or under were randomly allocated to three arms; Arm A: IMCBR; Arm B: community‐based rehabilitation (CBR); and Arm C: care‐as‐usual. The CBR was modified with phone follow‐up followed by home‐based CBR at 2.5 months post‐enrolment because of the COVID‐19 pandemic. Intention‐to‐treat analysis was performed. Results: Twenty‐four clusters constituting 251 children–primary caregivers' dyads were assigned to three arms (Arm A = 80; Arm B = 82; Arm C = 89). Between baseline and endline, the percentage mean change in the physical functioning domain of HRQoL was highest in Arm A (30.0%) with a significant mean difference between Arm A and Arm B (p = 0.015). Improvement in the mean social capital score was significantly higher in Arm A compared to Arm C (p < 0.001). Interpretation: The findings suggest that IMCBR could improve the HRQoL of children with CP and the social capital of their ultra‐poor families. Long‐term follow‐up of the trial participants and future exploration of such interventions are essential. The integrated livelihood and CBR programme holds potential to improve health and well‐being of children with CP and their ultra‐poor families. What this paper adds: Half of the families who received livelihoods were impacted by a cold‐wave, suggesting the need for a more disaster‐resilient livelihood asset.The integration of livelihood with community‐based rehabilitation programme helps to improve health‐related quality of life of children with cerebral palsy and the social capital of their ultra‐poor families. What this paper adds: Half of the families who received livelihoods were impacted by a cold‐wave, suggesting the need for a more disaster‐resilient livelihood asset.The integration of livelihood with community‐based rehabilitation programme helps to improve health‐related quality of life of children with cerebral palsy and the social capital of their ultra‐poor families. This original article is commented on by Fianto on pages 727–728 of this issue. [ABSTRACT FROM AUTHOR]

Aim: To develop a core set of outcome domains to be measured in clinical studies on lower limb orthopaedic surgery for ambulant children with cerebral palsy (CP) that represents the priorities of an international multi‐stakeholder group (children, parent/carers, and health professionals). Method: Potential outcome domains were identified through literature review and qualitative interviews with key stakeholders. These were scored in an international two‐round Delphi survey, using a 9‐point Likert scale. A final consensus meeting with key stakeholders agreed on the most important outcome domains and refined the core outcome set (COS). Results: One hundred and sixty‐one health professionals and 36 individuals with CP and their parents/carers rated 21 of 41 outcomes as important in the Delphi survey. The final consensus group agreed 19 outcomes within eight domains to be included in the final COS: pain and fatigue, lower limb structure, motor function, mobility (daily life activities), gait‐related outcomes, physical activity, independence, and quality of life. Interpretation: A COS for lower limb orthopaedic surgery for children with CP was developed. Incorporating this in the design of future clinical studies will provide a more holistic assessment of the impact of treatment while allowing meaningful comparisons and future synthesis of results from primary studies. What this paper adds: Eight core outcome domains were identified as important to measure in future clinical research.Key stakeholders perceived pain, balance and fall, and independence as very important outcomes.Six contextual factors were identified as essential in surgical decision‐making. What this paper adds: Eight core outcome domains were identified as important to measure in future clinical research.Key stakeholders perceived pain, balance and fall, and independence as very important outcomes.Six contextual factors were identified as essential in surgical decision‐making. This original article is commented on by Shrader on pages 153–154 of this issue. [ABSTRACT FROM AUTHOR]

Introduction: The American College of Sports Medicine provided guidelines for exercise prescriptions in cancer survivors for specific cancer‐ and treatment‐related health outcomes. However, there was insufficient evidence to generate exercise prescriptions for 10 health outcomes of cancer treatment. We sought to update the state of evidence. Methods: We conducted a systematic review of these 10 understudied health outcomes (bone health, sleep, cardiovascular function, chemotherapy‐induced peripheral neuropathy (CIPN), cognitive function, falls and balance, nausea, pain, sexual function, and treatment tolerance) and provided an update of evidence. Results: While the evidence base for each outcome has increased, there remains insufficient evidence to generate exercise prescriptions. Common limitations observed across outcomes included: variability in type and quality of outcome measurement tools, variability in definitions of the health outcomes, a lack of phase III trials, and a majority of trials investigating breast or prostate cancer survivors only. Conclusion: We identified progress in the field of exercise oncology for several understudied cancer‐ and treatment‐related health outcomes. However, we were not able to generate exercise prescriptions due to continued insufficient evidence base. More work is needed to prescribe exercise as medicine for these understudied health outcomes, and our review highlights several strategies to aid in research acceleration within these areas of exercise oncology. Marked progress has been made in a short amount of time on understudied cancer‐ and treatment‐related health outcomes. However, compared to the breadth of evidence previously used to generate FITT (frequency, intensity, time, type) prescriptions for anxiety, depressive symptoms, fatigue, health‐related quality of life, lymphedema, and physical function, rigorous research is still warranted. In this paper, we accomplished three goals: (1) presented the state of the evidence for bone health, sleep, cardiovascular function, CIPN, cognitive function, falls and balance, nausea, pain, sexual function, and treatment tolerance; (2) identified common tools used in exercise oncology to assess these specific understudied outcomes, and (3) suggested knowledge gaps that currently limit our ability to provide clinical guidance, or FITT prescriptions, on these understudied outcomes for specialists, patients and caregivers. [ABSTRACT FROM AUTHOR]

In the last two decades, genital transplants have emerged as another type of quality‐of‐life transplants. Successful allogenic transplantations of the uterus, ovary, testicle, and penis have all been reported. Yet, there is no discussion of clitoris transplantation in the medical literature, mass media, and everywhere else I searched. This surgery could be used for cisgender women who have a clitoral injury or disease or who have undergone female genital cutting. I examine the gender norms regarding sexuality and reproduction to show how they shape surgical advancements. My point in this paper is not to take a normative position on status of current genital transplantations. Rather, I highlight that their existence is due, at least in part, because they align with dominant gender norms: penis and testicle transplantations reinforce the importance of men's virility and the existence of "normal" male genitalia, whereas uterus and ovary transplantations uphold the conflation of women and reproduction and the strong valuing of women's fertility. That medical advances reflect cultural values is not a new claim. What is new in this paper is the discussion of how sexism norms—regarding the invisibility of the clitoris and the devaluing of women's sexual pleasure— has engendered various types of genital transplants, but not clitoris transplantation. [ABSTRACT FROM AUTHOR]

Rationale and Aim: Advanced Practice Nurse (APN) is a specialist who has acquired clinical skills to make complex decisions for a better professional practice. In the United States, this figure has been developed in different ways, but in some European countries, it is not yet fully developed, although it may imply a significant advance in terms of continuity and quality of care in patients with chronic or multiple pathologies, including cardiac ones and, more specifically, heart failure (HF). The follow‐up of HF patients in many countries has focused on the medical management of the process, neglecting all the other comprehensive health aspects that contribute to decompensation of HF, worsening quality indicators or patient satisfaction, and there are not updated reviews to clarify the relevance of APN in HF, comparing the results of APN interventions with doctors clinical practice, since the complexity of care that HF patients need makes it difficult to control the disease through regular treatment. For this reason, this systematic review was proposed in order to update the available knowledge on the effectiveness of APN interventions in HF patients, analysing four PICO questions (Patients, Interventions, Comparison and Outcomes): whether APN implies a reduction in the number of hospital readmissions, if it reduces mortality, if it has a positive cost‐benefit relationship and if it implies any improvement in the quality of life of HF patients. Design and Methods: A systematic review was performed based on the PRISMA statement, searching at four databases: PubMed, CINAHL, Scopus and Cuiden. Articles were selected based on the following criteria: English/Spanish language, up to 6 years since publication, and original quantitative studies of experimental, quasi‐experimental or observational character. Papers were excluded if they do not comply with CONSORT or STROBE checklists, and if they had not been published in journals indexed in JCR and/or SJR. For the analysis, two separate researchers used the Cochrane Handbook form for systematic reviews of intervention, collecting authorship variables, study methods, risks of bias, intervention and comparison groups, results obtained, PICO question or questions answered, and the main conclusions. Results: A total of 43,754 patients participated in the 11 included studies for the development of this review, mostly from United States and non‐European countries, with a clearly visible lack of European publications. Regarding the results related to first PICO question, researches reviewed proved that APN implied a reduction in the number of hospital readmissions in patients with heart failure (up to 33%). Regarding the second question, mortality was always lower in groups assisted by APN versus in control groups (up to 7.8% vs. 17.7%). Regarding the third question, APN was cost‐effective in this type of patient as the cost reduction was eventually calculated in 1.9 million euros. Regarding the last question, quality of life of patients who have been cared for by an APN had notoriously improved, although one of the papers concluded that no significant differences were found. All the questions addressed obtained a positive answer; therefore, APN is a practice that reduced hospital readmissions and mortality in HF patients. The cost‐effectiveness is much better with APN than with usual care, and although the quality of life of HF patients seems to improve with APN, more studies are needed to support this focused on this. [ABSTRACT FROM AUTHOR]

The demand for vehicles in India is on a continuous rise, primarily attributed to increasing income levels. The Indian government has initiated steps to decarbonize the transport sector, offering incentives for alternate vehicles' adoption. This study introduces a qualitative framework, employing a causal structure to comprehend the dynamics of vehicle demand. The framework takes into account critical feedback loops that influence the demand for alternate vehicles and, consequently, the overall vehicle demand. The causal loop diagram for vehicle demand encompasses five key domains: socio‐economic development, infrastructure development, environmental awareness, fuel supply market and alternate vehicle technology. Notably, economic activity, urban sprawl, vehicle demand, alternate vehicle technology improvement, and the sustainability and fuel constraints linked with internal combustion engine vehicles emerge as the critical aspects governing the dynamics of the model. The leverage and intervention points influencing the vehicle demand are also identified. Further, based on the evolving interaction of the various reinforcing and balancing loops, this work identifies three distinct phases of the evolution of vehicle demand dynamics in India, namely, the emerging phase (2020–2040), the growth phase (2040–2060) and the stagnation phase (2060–2080). The paper discusses the crucial features of these phases. [ABSTRACT FROM AUTHOR]

Aim: To summarize the current knowledge on brain involvement in spinal muscular atrophy (SMA) type 1, focusing on brain pathology, cognition, and speech/language development. Method: A scoping review was performed using the methodology of the Joanna Briggs Institute. Five databases and references from relevant articles were searched up to December 2019. Articles were screened on the basis of titles and abstracts. Full‐text papers published in peer‐reviewed journals in English were selected. Results: Nineteen articles met eligibility criteria. Eight case series/reports on brain pathology showed abnormalities in few SMA type 0/1 cases, supported by findings in three post‐mortem examinations in mice. Four studies (three case–control, one cross‐sectional) on cognition reported contradictory results, with impaired cognitive performances in recent, small groups with SMA type 1. Four studies (three cross‐sectional, one observational) on speech/language showed that untreated SMA type 1 patients rarely achieve functional and intelligible speech, with data limited to parent reports/non‐formal evaluations. Interpretation: Brain involvement is an under‐investigated aspect of SMA type 1, requiring further exploration in longitudinal studies. A deeper knowledge of brain involvement would improve the interpretation of clinical phenotypes and the personalization of rehabilitation programmes supporting patients' autonomies and quality of life. Additionally, it may help to define further outcome measures testing the efficacy of current and new developing drugs on this domain. What this paper adds: Brain involvement is under‐investigated in spinal muscular atrophy (SMA) type 1.Neuropathological data suggest progressive brain involvement in severe forms of SMA.Impaired cognitive performances are reported in small groups with SMA type 1.Data on language in those with SMA type 1 are limited to parent reports and non‐formal assessments. What this paper adds: Brain involvement is under‐investigated in spinal muscular atrophy (SMA) type 1.Neuropathological data suggest progressive brain involvement in severe forms of SMA.Impaired cognitive performances are reported in small groups with SMA type 1.Data on language in those with SMA type 1 are limited to parent reports and non‐formal assessments. [ABSTRACT FROM AUTHOR]

Objective To conduct a systematic appraisal of the published literature reviews related to the impact of cancer on everyday life. This theme was identified as the top priority area for research by participants in the Macmillan Listening Study, which was the first UK-wide public consultation exercise to identify patients' priorities for cancer research. Search strategy The top priority area was divided into ten sub-themes, and a modified systematic review was undertaken for each sub-theme using electronic searches. Inclusion criteria Literature review papers were included if they were written in English, involved patients diagnosed ≥18 years, any cancer types and published between 2000 and 2006. Data extraction and synthesis Two thousand and two hundred and fifty-two potentially eligible papers were identified. Abstracts were read by the first author and selected for inclusion in the review. Twenty percentages of the papers were also read independently by other authors. Sixty-two review papers were finally selected. Main results The systematic appraisal revealed that some sub-themes of the patient-identified priority area (e.g., how to support family members of cancer patients) were under-researched, while other sub-themes (e.g., anxiety and depression experienced by cancer patients) had been explored to some extent. Certain areas of research interest to patients were found to have been explored; however, their significance was limited by the quality of the research. Conclusion The systematic appraisal highlights important areas for future research and the need for more effective dissemination of study findings to wider audiences, including service users. This study also indicates the need for further research to enhance the generalizability and/or significance of findings. [ABSTRACT FROM AUTHOR]

The purpose of this study was to explore the literature on the psychosocial aspects of Marfan syndrome ( MFS), to critically appraise and to synthesize relevant literature. A mixed-method systematic review was performed by searching the published literature databases using available medical, psychological, pedagogical and social databases and other sources. All studies that addressed psychosocial aspects of MFS, published in peer-reviewed journals were assessed. Of 81 search results, 15 articles (four articles based on same study population) satisfied the eligibility criteria. All studies were cross-sectional; no intervention or randomized controlled trial ( RCT) studies were found. Most studies were of small sample sizes, had low response rate or participants without a verified diagnosis. Despite these limitations, all studies described, that MFS has a significant impact on the psychosocial aspects of people's lives: Decreased quality of life; challenges in education, work and family life, depression and anxiety. Some studies indicated that the subjective perception of discomfort did not necessarily match the medical severity of a disease. The research of the psychosocial aspects of MFS is limited in size and quality. More research is needed on the psychosocial aspects of MFS in samples with a verified diagnosis to develop evidence-based knowledge and appropriate guidelines. [ABSTRACT FROM AUTHOR]

Aim: Careful study and accurate description of behaviour are important to understand developmental challenges for individuals with Cornelia de Lange syndrome (CdLS). Here we present a systematic review of current understanding of behaviour in CdLS.Method: A systematic search was performed for articles published between January 1946 and December 2015 evaluating autism, self-injury, and/or cognition in CdLS. After study-selection, 43 papers were included. The Cochrane quality criteria were adjusted to assign quality scores to the included studies.Results: Participants were mostly categorized in the severe/profound developmental level. Methodology and quality were very heterogeneous, as well as reporting occurrence of autism. Self-injurious behaviour was reported in 15 papers. Physical conditions were reported in 21 studies, mostly related to hearing and vision. Only nine studies mentioned details about medication.Interpretation: Comparison of presented results was hindered by heterogeneous assessment methods. Improving our understanding of behavioural characteristics in CdLS requires more uniform methodology. We propose a criterion standard of instruments that can ideally be used in assessment of behaviour and development. This will improve understanding of behaviour in the context of developmental level and daily functioning. [ABSTRACT FROM AUTHOR]

Radiation‐induced dermatitis is one of the most prevalent complications in patients undergoing cancer radiotherapy and poses a significant challenge to cancer therapy. The symptoms include erythema, dry desquamation, and moist desquamation, which are frequently observed in patients with breast, head and neck, anal, and vulvar cancers. Early skin reactions typically manifest within 2–4 weeks following the initiation of radiotherapy. In severe cases, acute dermatitis can cause radiotherapy interruptions, prolong treatment time, and ultimately affect patient outcomes and quality of life. Currently, there are numerous guidelines on radiation dermatitis, including the Multinational Association of Supportive Care in Cancer (MASCC), British Columbia Cancer Agency (BCCA), Oncology Nursing Society (ONS), and UK Society of Radiographers (SCoR) guidelines. In China, dermatology experts have drafted a consensus. However, due to the differing backgrounds of experts, recommendations among guidelines vary. These guidelines were first developed by Chinese radiation oncologists. The evidence‐based guideline in this paper fully considers and adopts China's national conditions; hence, it can be easily applied in daily practice. [ABSTRACT FROM AUTHOR]

In recent years, the use of topical morphine gel has increased in the palliative care setting to reduce pain in chronic wounds and fungating tumours. However, there is a paucity of evidence to support its effectiveness. Epidermolysis bullosa (EB) is a rare genetic skin fragility disorder characterised by painful chronic wounds. Adequate control of wound pain can be challenging in this patient group due to other complexities associated with the more severe sub‐types of the disease. Topical morphine gel has been used as an adjunct therapy in a small number of EB patients in our tertiary centre in an attempt to improve pain control and quality of life. The purpose of this paper is to demonstrate the efficacy of topical morphine gel used in a variety of EB wounds as well as patient reported reduction in pain through a series of case studies. The case studies suggest a positive effect of topical morphine gel on painful wounds across a spectrum of EB subtypes. [ABSTRACT FROM AUTHOR]

Young children in the East Asia and the Pacific region1 are failing to thrive, in large numbers, as indicated by stagnation in the decline of stunting, wasting, and micronutrient deficiencies and the fastest growing rates of overweight in the world. Eliminating the triple burden of malnutrition is essential to ensure that, as a matter of right, all children reach their full physical growth and development potential and actively contribute to equitable prosperity and the sustainable development of their communities and nations. Ending all forms of malnutrition will only be achieved through the implementation of effective policies and programmes soundly based on an understanding of the leading contextual drivers of child malnutrition. To address the lack of data on these drivers in the region, the UNICEF regional office for East Asia and the Pacific commissioned a series of papers in 2017–2019 to fill gaps in the current body of evidence on the triple burden of maternal and child malnutrition. This series includes analyses of the determinants of child malnutrition including maternal nutrition status, dietary quality of children, inequity, and poverty. Additionally, policy and programmatic actions associated with improved coverage and quality of nutrition interventions are reviewed. This overview paper summarizes the findings of these analyses and presents recommendations for the direction of future advocacy, policy, and programmatic actions to address the triple burden of malnutrition in East Asia and the Pacific. [ABSTRACT FROM AUTHOR]

Background: Immune checkpoint inhibitors (ICIs) have significantly improved survival in advanced melanoma but are associated with immune‐related adverse events (irAEs). This single center, cross‐sectional survey aimed to describe the long‐term symptom burden and impact on health‐related quality of life (HRQL) of advanced melanoma patients with sustained disease control following treatment with ICIs. Methods: Advanced melanoma patients (stage IIB, III or IV, AJCCv8), treated with anti‐PD1‐based ICIs, who were off‐treatment and had at least 6 months follow‐up from their last infusion with an ongoing response in the metastatic setting or no evidence of disease recurrence in the adjuvant setting. A paper‐based questionnaire, consisting of the EORTC QLQ‐C30, EORTC QLQ‐FA12, and the PRO‐CTCAE was administered. Results: Of 90 participants, 61 (68%) completed the questionnaire; 40 received single‐agent anti‐PD1, and 21 anti‐PD1/anti‐CTLA4. Thirty‐three (54%) were treated in the adjuvant setting. At the time of enrolment, 31 (51%) participants had active treatment for a previous irAE. Overall, 18/61 (30%) participants reported long‐term symptoms and trouble in physical and emotional functioning. Physical fatigue was common and interfered with daily activities (n = 12, 20%). In the PRO‐CTCAE questionnaire, muscle ache (n = 12, 20%) and joint ache (n = 9, 15%) were commonly reported. Despite this, participants reported overall good health (6.00, range 2.00–7.00) and reasonable level of HRQL (6.00, range 3.00–7.00). Discussion: Melanoma survivors experience long‐term symptoms in physical and psychosocial HRQL domains after ICI treatment. These results underline the importance to address existing gaps in survivorship care, implement these findings in clinical practice and increase awareness for long‐term symptoms in these patients. [ABSTRACT FROM AUTHOR]

Objective: This paper reports the study design, participant characteristics, and recruitment results of By‐Band‐Sleeve, which investigated the clinical and cost‐effectiveness of gastric bypass, gastric banding, and sleeve gastrectomy in adults with severe obesity in the UK. Methods: A pragmatic open adaptive noninferiority trial with 3‐year follow‐up was conducted. Participants were randomly assigned to bypass or band initially and to sleeve after the adaptation. Co‐primary end points are weight loss and health‐related quality of life assessed using the EQ‐5D utility index. Results: Between December 2012 and August 2015, the study recruited into two groups and, after the adaptation, into three groups until September 2019. The study screened 6960 patients; 4732 (68%) were eligible and 1351 (29%) were randomized; 5 subsequently withdrew consent to use data, leaving 462, 464, and 420 assigned to bypass, band, and sleeve, respectively. Baseline data showed high levels of obesity (mean BMI = 46.4 kg/m2; SD: 6.9) and comorbidities (e.g., 31% diabetes), low scores for health‐related quality of life, and high levels of anxiety and depression (e.g., 25% abnormal scores). Nutritional parameters were poor, and the average equivalized household income was low (£16,667). Conclusions: By‐Band‐Sleeve fully recruited. Participant characteristics are consistent with contemporary patients having bariatric surgery, and therefore the results will be generalizable. [ABSTRACT FROM AUTHOR]

Background: Fragmented and complex healthcare systems make it difficult to provide continuity of care for patients with advanced cancer near the end of life. Nurse‐based cross‐sectoral navigation support has the potential to increase patients' quality of life. The objective of this paper was to evaluate associations between navigation support and health care utilization, and the associated costs of care. Methods: The evaluation is based on claims data from 37 statutory health insurance funds. Non‐randomized recruitment of the intervention group (IG) took place between 2018 and 2019 in four German hospitals. The comparison group (CG) was defined ex post. It comprises nonparticipating clients of the involved health insurance funds matched on age, gender, and diagnosis in a 1:4 ratio to the IG. Healthcare resource utilization was compared using incident rate ratios (IRRs) based on negative binomial regression models. Linear mixed models were performed to compare differences in lengths of hospital stays and costs between groups. Results: A total of 717 patients were included (IG: 149, CG: 568). IG patients showed shorter average lengths of hospital stays (IG: 11 days [95% CI: 10, 13] vs. CG: 15 days [95% CI: 14, 16], p < 0.001). In the IG, 21% fewer medications were prescribed and there were on average 15% fewer outpatient doctor contacts per month. Average billed costs in the IG were 23% lower than in the CG (IG: 6754 EUR [95% CI: 5702, 8000] vs. CG: 8816 EUR [95% CI: 8153, 9533], p < 0.001). Conclusions: The intervention was associated with decreased costs mainly as a result of a non‐intended navigation effect. The social care nurses had navigated patients within the hospital early, needs‐oriented and effectively but interpreted their function less cross‐sectorally. Linkage of hospital‐based navigators with the outpatient care sector needs further exploration. [ABSTRACT FROM AUTHOR]

Objective. To determine whether e/Tablets (wireless tablet computers used in community oncology clinics to collect review of systems information at point of care) are feasible, acceptable, and valid for collecting research-quality data in academic oncology. Data/Setting. Primary/Duke Breast Cancer Clinic. Design. Pilot study enrolling sample of 66 breast cancer patients. Methods. Data were collected using paper- and e/Tablet-based surveys: Functional Assessment of Cancer Therapy General, Functional Assessment of Cancer Therapy-Breast, MD Anderson Symptom Inventory, Functional Assessment of Chronic Illness Therapy (FACIT), Self-Efficacy; and two questionnaires: feasibility, satisfaction. Principal Findings. Patients supported e/Tablets as: easy to read (94 percent), easy to respond to (98 percent), comfortable weight (87 percent). Generally, electronic responses validly reflected responses provided by standard paper data collection on nearly all subscales tested. Conclusions. e/Tablets offer a valid, feasible, acceptable method for collecting research-quality, patient-reported outcomes data in outpatient academic oncology. [ABSTRACT FROM AUTHOR]

Aim: To determine the efficacy of fenfluramine on seizure frequency in patients with Sunflower syndrome. Secondary endpoints were changes in electroencephalogram (EEG) characteristics, cognitive functioning, executive functioning, and quality of life. Method: In this open‐label study, patients underwent a 4‐week baseline period, followed by 3 months of treatment. An oral solution of fenfluramine was administered twice daily for 3 months. The dose was titrated up to a maximum dose of 0.7mg/kg/day or 26mg/day. Cardiac safety was monitored by transthoracic echocardiogram and electrocardiogram. EEGs, abbreviated neuropsychological testing, and questionnaires were administered before starting the study medication and again at the end of the treatment period. Results: Ten patients (eight females, two males; mean age 13y 4mo [SD 4y 11mo], range 7–24y) were enrolled in the study. Nine of the 10 patients completed the core study, eight of whom met the primary endpoint. There were no observations of cardiac valvulopathy or pulmonary hypertension during the study. Interpretation: Treatment with low‐dose fenfluramine resulted in a clinically significant reduction in seizure frequency, including hand‐waving episodes. Fenfluramine may be an effective treatment option for patients with Sunflower syndrome. What this paper addsNine patients with Sunflower syndrome were treated with fenfluramine.Eight patients were responders, displaying a ≥30% reduction in seizure activity.Six patients experienced a ≥70% reduction in hand‐waving episodes.Improvements on electroencephalogram were observed after treatment with fenfluramine.None of the patients developed evidence of cardiac valvulopathy or pulmonary hypertension. What this paper adds: Nine patients with Sunflower syndrome were treated with fenfluramine.Eight patients were responders, displaying a ≥30% reduction in seizure activity.Six patients experienced a ≥70% reduction in hand‐waving episodes.Improvements on electroencephalogram were observed after treatment with fenfluramine.None of the patients developed evidence of cardiac valvulopathy or pulmonary hypertension. [ABSTRACT FROM AUTHOR]

Aim: To examine the impact of clinical factors, cognitive deficits, and sleepiness on health‐related quality of life (HRQoL) among young people with craniopharyngioma. Method: Seventy‐eight patients (67% White; 41 males, 37 females; mean age 10y 8mo, SD 3y 11mo, range 6–20y) with craniopharyngioma were assessed for tumor extent and diabetes insipidus. All patients underwent overnight polysomnography and multiple sleep latency tests after surgical resection. Executive functioning was assessed using parent‐reported measures. Patients and their parents completed measures of HRQoL. None had a history of previous radiation therapy. Results: Path analysis was used to test hypothesized relations while controlling for demographic and disease characteristics. Analyses revealed poorer parent‐reported HRQoL among young people with greater executive functioning symptoms (estimate −0.83; p<0.001). Direct and indirect effects were found among diabetes insipidus, executive functioning, and parent‐reported HRQoL. Diabetes insipidus directly predicted greater global executive functioning impairment (estimate 5.15; p=0.04) and indirectly predicted lower HRQoL through executive functioning impairment (estimate −4.25; p=0.049). No significant effects were found between excessive daytime sleepiness, tumor hypothalamic involvement, diabetes insipidus, executive functioning, and patient‐reported HRQoL. Interpretation: These findings suggest that young people with craniopharyngioma presenting with diabetes insipidus may benefit from targeted neurocognitive and psychosocial screening to inform interventions. What this paper addsChildren with craniopharyngioma and executive functioning impairment are more likely to have poorer health‐related quality of life (HRQoL).Diabetes insipidus, a complication associated with surgery, predicted greater executive functioning impairment.Diabetes insipidus indirectly predicted lower parent‐reported HRQoL through executive functioning impairment. What this paper adds: Children with craniopharyngioma and executive functioning impairment are more likely to have poorer health‐related quality of life (HRQoL).Diabetes insipidus, a complication associated with surgery, predicted greater executive functioning impairment.Diabetes insipidus indirectly predicted lower parent‐reported HRQoL through executive functioning impairment. This article is commented on by Cheetham and Wood on page 895 of this issue. [ABSTRACT FROM AUTHOR]

Aim: To gain insight into parents' perspectives about their decision‐making process concerning nusinersen treatment for their child, including perceived needs and concerns, and to explore factors that influence this process. Method: This was an exploratory qualitative interview study among parents of children with spinal muscular atrophy types 1 to 3. Data were analysed using inductive thematic analysis. Results: Nineteen parents of 16 children representing 13 families participated. A wide variety of perspectives was reported ranging from a biomedical approach, which focused on battling the disease, to a holistic approach, which aimed for a good quality of life for their child. The most important factors that helped parents to decide were honest and neutral communication with their physician and access to available information. Interpretation: It is important physicians understand that there are different perspectives influencing the decision‐making process. Physicians should create an environment that allows parents to accept or reject treatment by communicating honestly and openly with them and by discussing both options extensively. Clear information about pros and cons, recent developments in research, and the experiences of other parents should be made available to enable parents to make an informed decision. What this paper addsParents perceived different needs and concerns about nusinersen treatment, which emphasized individual differences.Parents' perspectives varied from battling the disease to preserving quality of life.Life expectancy, stopping deterioration, and improving quality of life were the perceived benefits of nusinersen treatment.Open communication about the pros and cons of treatment with clinicians facilitated decision‐making.Clear and honest information facilitated the alignment of values and goals. What this paper adds: Parents perceived different needs and concerns about nusinersen treatment, which emphasized individual differences.Parents' perspectives varied from battling the disease to preserving quality of life.Life expectancy, stopping deterioration, and improving quality of life were the perceived benefits of nusinersen treatment.Open communication about the pros and cons of treatment with clinicians facilitated decision‐making.Clear and honest information facilitated the alignment of values and goals. [ABSTRACT FROM AUTHOR]

Aim: To assess the association between epilepsy characteristics and proxy‐reported health‐related quality of life (HRQoL) in children and young people with non‐ambulatory cerebral palsy (CP) and seizures. Method: This was a cross‐sectional study of 164 children and young people (74 females, 90 males; mean age 10 years 6 months, range 2–21 years, SD 5 years 5 months). Caregivers completed the Child Health Index of Life with Disabilities (CPCHILD) in an outpatient setting. We utilized univariable linear regression and multivariable modeling to study relationships between variables and CPCHILD scores. Results: Gross Motor Function Classification System levels were 37% IV and 63% V. Sociodemographic factors included the Child Opportunity Index (median 51, interquartile range [IQR] 25–80). A median of 2 (IQR 1–3) antiseizure medications (ASMs) were used, and days with seizures ranged from 0 (30%) to 28 (20%) days in the previous 4 weeks. Total CPCHILD scores decreased 2.3 points for each ASM (95% confidence interval [CI] –4.1 to −0.42). Compared to persons with focal epilepsy, those with generalized epilepsy had lower total CPCHILD scores (−5.7; 95% CI –11 to −0.55). Number of days with seizures was not associated with total CPCHILD scores. Interpretation: Proxy‐reported HRQoL was affected by epilepsy‐specific features in children and young people with severe CP. What this paper adds: Health‐related quality of life (HRQoL) was lower with increasing numbers of antiseizure medications.Overall quality of life (QoL) scores were lower by a similar amount, independent of seizure frequency.HRQoL was lower in persons with recent hospital admissions for epilepsy. [ABSTRACT FROM AUTHOR]

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This paper describes a holistic, yet simple and comprehensible, ecosystem model to deal with multiple and complex challenges in eyecare. It aims at producing the best possible wellbeing and eyesight with the available resources. When targeting to improve the real-world cost-effectiveness, what gets done in everyday practice needs be measured routinely, efficiently and unselectively. Collection of all real-world data of all patients will enable evaluation and comparison of eyecare systems and departments between themselves nationally and internationally. The concept advocates a strategy to optimize real-life effectiveness, sustainability and outcomes of the service delivery in ophthalmology. The model consists of three components: (1) resource-governing principles (i.e., to deal with increasing demand and limited resources), (2) real-world monitoring (i.e., to collect structured real-world data utilizing automation and visualization of clinical parameters, health-related quality of life and costs), and (3) digital innovation strategy (i.e., to evaluate and benchmark real-world outcomes and cost-effectiveness). The core value and strength of the model lies in the consensus and collaboration of all Finnish university eye clinics to collect and evaluate the uniformly structured real-world outcomes data. In addition to ophthalmology, the approach is adaptable to any medical discipline to efficiently generate real-world insights and resilience in health systems., (© 2021 The Authors. Acta Ophthalmologica published by John Wiley & Sons Ltd on behalf of Acta Ophthalmologica Scandinavica Foundation.)