Search

Objectives : In families with a parent diagnosed with amyotrophic lateral sclerosis (ALS), children's adaptation depends among others on how their parents communicate with them about the disease and its trajectory. The aim of this study was to explore parents' and children's perceptions of ALS-related family communication. Methods : A qualitative analysis using a conventional content analysis approach was applied to interview data previously collected from 21 parents (8 with ALS) and 15 children (age 13-23 years) about their experiences living with ALS. Results : Three themes emerged from the interviews: communication topics, styles and timing. Communication topics include facts about disease and prognosis, feelings, care and equipment, and the end. Although most parents perceived the familial communication style concerning ALS as open, the interviews revealed that both parents and children sometimes avoid interactions about ALS, because they do not know what to say or how to open the dialogue, are afraid to burden other family members, or are unwilling to discuss. Communication timing is directed by changes in the disease trajectory and/or questions of children. A family-level analysis showed that ALS-related family communication is sometimes perceived differently by parents and children. Conclusions : The study provides a better understanding of what, how and when parents and children in families living with ALS communicate about the disease. Most families opened the dialogue about ALS yet encountered challenges which may hamper good familial communication. Through addressing those challenges, healthcare professionals may facilitate better communication and adaptation in families with a parent with ALS.

Background: Gastrostomy is recommended in amyotrophic lateral sclerosis for long-term nutritional support, however, people with amyotrophic lateral sclerosis and healthcare professionals perceive decision-making as complex., Method: To explore their perspectives on decision-making regarding gastrostomy, we used semi-structured interviews with people with amyotrophic lateral sclerosis, who had made a decision, and their caregivers; healthcare professionals were interviewed separately. Interviews were transcribed and analyzed thematically., Results: In 14 cases, 13 people with amyotrophic lateral sclerosis and 12 caregivers were interviewed; and in 10 of these cases, 5 healthcare professionals. Participants described decision-making on gastrostomy as a continuous process of weighing (future) clinical need against their values and beliefs in coming to a decision to accept or reject gastrostomy, or to postpone decision-making, while being supported by loved ones and healthcare professionals. Participants described gastrostomy as inevitable, but retained agency through control over the timing of decision-making. They said physical necessity, experiences of loss and identity, and expectations about gastrostomy placement were important factors in decision-making. Decision-making was described as a family affair, with caregivers supporting patient choice. healthcare professionals supported people with amyotrophic lateral sclerosis during the decision-making process and respected their autonomy and values. People with amyotrophic lateral sclerosis stressed the importance of adequate information on the procedure and the benefits., Conclusion: People with amyotrophic lateral sclerosis feel in control of decision-making on gastrostomy if they are able to make their own choice at their own pace, supported by loved ones and healthcare professionals. Person-centered decision-making on gastrostomy requires early information exchange and repeated discussions with people with amyotrophic lateral sclerosis and their caregivers, incorporating their values and respecting patient choice., Competing Interests: The authors have declared that no competing interests exist., (Copyright: © 2023 van Eenennaam et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.)

Background and Objectives: Current scales used in amyotrophic lateral sclerosis (ALS) attempt to summarize different functional domains or "dimensions" into 1 overall score, which may not accurately characterize the individual patient's disease severity or prognosis. The use of composite score risks declaring treatments ineffective if not all dimensions of ALS disease progression are affected equally. We aimed to develop the ALS Impairment Multidomain Scale (AIMS) to comprehensively characterize disease progression and increase the likelihood of identifying effective treatments., Methods: The Revised ALS Functional Rating Scale (ALSFRS-R) and a preliminary questionnaire, based on literature review and patient input, were completed online by patients from the Netherlands ALS registry at bimonthly intervals over a period of 12 months. A 2-week test-retest, factor analysis, Rasch analysis, and a signal-to-noise optimization strategy were performed to create a multidomain scale. Reliability, longitudinal decline, and associations with survival were evaluated. The sample size required to detect a 35% reduction in progression rate over 6 or 12 months was assessed for a clinical trial that defines the ALSFRS-R or AIMS subscales as a primary endpoint family., Results: The preliminary questionnaire, consisting of 110 questions, was completed by 367 patients. Three unidimensional subscales were identified, and a multidomain scale was constructed with 7 bulbar, 11 motor, and 5 respiratory questions. Subscales fulfilled Rasch model requirements, with excellent test-retest reliability of 0.91-0.94 and a strong relationship with survival ( p < 0.001). Compared with the ALSFRS-R, signal-to-noise ratios were higher as patients declined more uniformly per subscale. Consequently, the estimated sample size reductions achieved with the AIMS compared with those achieved with the ALSFRS-R were 16.3% and 25.9% for 6-month and 12-month clinical trials, respectively., Discussion: We developed the AIMS, consisting of unidimensional bulbar, motor, and respiratory subscales, which may characterize disease severity better than a total score. AIMS subscales have high test-retest reliability, are optimized to measure disease progression, and are strongly related to survival time. The AIMS can be easily administered and may increase the likelihood of identifying effective treatments in ALS clinical trials., (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.)

Competing Interests: Declaration of interests We declare no competing interests.

Background: Amyotrophic lateral sclerosis is a progressive and lethal neurodegenerative disease that is at the forefront of debates on regulation of assisted dying. Since 2002, when euthanasia was legally regulated in the Netherlands, the frequency of this end-of-life practice has increased substantially from 1·7% of all deaths in 1990 and 2005 to 4·5% in 2015. We aimed to investigate whether the frequency of euthanasia in patients with amyotrophic lateral sclerosis had similarly increased since 2002, and to assess the factors associated with end-of-life practices and the quality of end-of-life care in patients with this disease., Methods: Using data from the Netherlands ALS registry, we did a population-based cohort study of clinicians and informal caregivers of patients with amyotrophic lateral sclerosis to assess factors associated with end-of-life decision making and the quality of end-of-life care. We included individuals who were diagnosed with amyotrophic lateral sclerosis according to the revised El-Escorial criteria, and who died between Jan 1, 2014, and Dec 31, 2016. We calculated the frequency of euthanasia in patients with amyotrophic lateral sclerosis from reports made to euthanasia review committees (ERCs) between 2012 and 2020. Results were compared with clinic-based survey studies conducted in 1994-2005. End-of-life practices were end-of-life decisions by a clinician when hastening of death was considered as the potential, probable, or definite effect comprising euthanasia, physician-assisted suicide, ending of life without explicit request, forgoing life-prolonging treatment, and intensified alleviation of symptoms., Findings: Between Jan 1, 2012, and Dec 31, 2020, 4130 reports of death from amyotrophic lateral sclerosis were made to ERCs, of which 1014 were from euthanasia or physician-assisted suicide (mean frequency 25% [SD 3] per year). Sex and gender data were unavailable from the ERC registry. Of 884 patients with amyotrophic lateral sclerosis who died between Jan 1, 2014, and Dec 31, 2016, their treating clinician was identified for 731 and a caregiver was identified for 741, of whom 356 (49%) and 450 (61%), respectively, agreed to participate in the population-based survey study. According to clinicians, end-of-life practices were chosen by 280 (79%) of 356 patients with amyotrophic lateral sclerosis who died. The frequency of euthanasia in patients with amyotrophic lateral sclerosis in 2014-16 (141 [40%] of 356 deaths in patients with amyotrophic lateral sclerosis) was higher than in 1994-98 (35 [17%] of 203) and 2000-05 (33 [16%] of 209). Median survival of patients with amyotrophic lateral sclerosis from diagnosis was 15·9 months (95% CI 12·6-17·6) for those who chose euthanasia and 16·1 months (13·4-19·1) for those who did not choose euthanasia (hazard ratio 1·07, 95% CI 0·85-1·34; p=0·58). According to caregivers, compared with other end-of-life practices, patients with amyotrophic lateral sclerosis choosing euthanasia commonly reported reasons to hasten death as no chance of improvement (53 [56%] of 94 patients who chose euthanasia vs 28 [39%] of 72 patients who chose other end-of-life practices), loss of dignity (47 [50%] vs 15 [21%]), dependency (34 [36%] vs five [7%]), and fatigue or extreme weakness (41 [44%] vs 14 [20%]). According to caregivers, people with amyotrophic lateral sclerosis-whether they chose euthanasia or did not-were satisfied with the general quality (83 [93%] of 89 patients who chose euthanasia vs 73 [86%] of 85 patients who did not) and availability (85 [97%] of 88 vs 81 [91%] of 90) of end-of-life care., Interpretation: The proportion of patients with amyotrophic lateral sclerosis who chose euthanasia in the Netherlands has increased since 2002. The choice of euthanasia was not associated with disease or patient characteristics, depression or hopelessness, or the availability or quality of end-of-life care. The choice of euthanasia had no effect on overall survival. Future studies could focus on the effect of discussing end-of-life options on quality of life as part of multidisciplinary care throughout the course of the disease, to reduce feelings of loss of autonomy and dignity in patients living with amyotrophic lateral sclerosis., Funding: Netherlands ALS Foundation., Competing Interests: Declaration of interests We declare no competing interests., (Copyright © 2023 Elsevier Ltd. All rights reserved.)

Objective: Previous work suggests that stigma negatively impacts quality of life in people living with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA). This study aimed to explore experiences of enacted stigma (experienced discrimination) and felt stigma (shame, fear of exclusion) among Dutch ALS/PMA patients and their caregivers. A secondary aim was to assess associated factors of enacted/felt stigma among patients., Methods: A two-phase mixed-methods study was conducted, comprising cross-sectional surveys among 193 ALS/PMA patients and 87 caregivers, and semi-structured interviews with 8 ALS/PMA patients and 11 family caregivers. Descriptive and multivariable regression analyses along with qualitative content analysis were used to analyze survey and interview data., Results: Survey findings indicate that patients and caregivers experience enacted and felt stigma. Interviews with both patients and caregivers revealed two manifestations of enacted stigma, including social exclusion (e.g. relationship distancing) and stigmatizing attitudes/behaviors displayed by others (e.g. staring), and three manifestations of felt stigma, including alienation (e.g. shame/embarrassment), perceived discrimination (e.g. feeling judged) and anticipated stigma (e.g. fear of exclusion). Patients and caregivers engaged in concealing and resisting responses to stigma. More bulbar symptoms, King's clinical stage, younger age and living without a partner were significantly associated with enacted/felt stigma among patients., Conclusions: Our findings reveal a range of perceptions and experiences underlying enacted/felt stigma among ALS/PMA patients and their caregivers that may serve as conversation topics in clinical practice. Future research may shed more light on the determinants as well as the consequences of stigmatizing experiences among patients and caregivers.

Introduction: To capture the patient's attitude toward remote monitoring of motor neuron disease (MND) in care and clinical trials, and their concerns and preferences regarding the use of digital technology., Methods: We performed an international multi-centre survey study in three MND clinics in The Netherlands, the United Kingdom, and Australia. The survey was co-developed by investigators and patients with MND, and sent to patients by e-mail or postal-mail. The main topics included: patients' attitude towards remote care, participating in decentralized clinical trials, and preferences for and concerns with digital technology use., Results: In total, 332 patients with MND participated. A majority of patients indicated they would be happy to self-monitor their health from home (69%), be remotely monitored by a multidisciplinary care team (75%), and would be willing to participate in clinical trials from home (65%). Patients considered respiratory function and muscle strength most valuable for home-monitoring. The majority of patients considered the use of at least three devices/apps (75%) once a week (61%) to be acceptable for home-monitoring. Fifteen percent of patients indicated they would not wish to perform home-measurements; reporting concerns about the burden and distress of home-monitoring, privacy and data security., Conclusion: Most patients with MND exhibited a positive attitude toward the use of digital technology in both care and clinical trial settings. A subgroup of patients reported concerns with home-monitoring, which should be addressed in order to improve widespread adoption of remote digital technology in clinical MND care., (© 2022. The Author(s).)

Objective: Uniform data collection is fundamental for multicentre clinical trials. We aim to determine the variability, between ALS trial centers, in the prevalence of unexpected or implausible improvements in the revised ALS functional rating scale (ALSFRS-R) score, and its associations with individual patient and item characteristics. Methods: We used data from two multicentre studies to estimate the prevalence of an unexpected increase or implausible improvement in the ALSFRS-R score, defined as an increase of 5 points or more between two consecutive, monthly visits. For each patient with a 5-point or more increase, we evaluated the individual contribution of each ALSFRS-R item. Results: Longitudinal ALSFRS-R scores, originating from 114 trial centers enrolling a total of 1,240 patients, were analyzed. A 5-point or more increase in ALSFRS-R total score was found in 151 (12.2%) patients, with prevalence per study center ranging from 0% to 83%. Bulbar onset, faster disease progression at enrollment, and a lower ALSFRS-R score at baseline were associated with a sudden 5-point or more increase in the ALSFRS-R total score. ALSFRS-R items 2 (saliva), 9 (stairs), 10 (dyspnea), and 11 (orthopnea) were the primary drivers when a 5-point or more increase occurred. Conclusions: Sudden 5-point or more increases in ALSFRS-R total scores between two consecutive visits are relatively common. These sudden increases were not found to occur with equal frequency in trial centers; which underscores the need for amending existing standard operating procedures toward a universal version and monitoring of data quality during the study, in multicentre research.

Background: Home-monitoring of spirometry has the potential to improve care for patients with a motor neuron disease (MND) by enabling early detection of respiratory dysfunction and reducing travel burden. Our aim was to evaluate the validity and feasibility of home-monitoring vital capacity (VC) in patients with MND., Methods: We included 33 patients with amyotrophic lateral sclerosis, progressive muscular atrophy or primary lateral sclerosis who completed a 12-week home-monitoring protocol, consisting of 4-weekly unsupervised home assessments of VC and a functional rating scale. At baseline, during a home visit, patients/caregivers were trained in performing a VC test, and the investigator performed a supervised VC test, which was repeated at final follow-up during a second home visit. Validity of the unsupervised VC tests was evaluated by the differences between supervised and unsupervised VC tests, and through Bland-Altman 95% limits-of-agreement. Feasibility was assessed by means of a survey of user-experiences., Results: The 95% limits-of-agreement were [- 14.3; 11.7] %predicted VC, and 88% of unsupervised VC tests fell within 10%predicted of supervised VC. 88% of patients experienced VC testing as easy and not burdensome, however, 15% patients did not think their VC test was performed as well as in the clinic. 94% of patients would like home-monitoring of VC in MND care., Discussion: Unsupervised VC testing at home, with prior face-to-face training, is a valid and time-efficient method for the remote monitoring of respiratory function, and well-accepted by patients with MND and their caregivers., (© 2022. The Author(s).)

Objective: To describe current practices and barriers and support needs in gastrostomy indication and decision-making amongst rehabilitation physicians of ALS care teams in the Netherlands., Methods: Cross-sectional online survey of rehabilitation physicians of ALS care teams in the Netherlands. Survey items covered current practices in timing of indication (i.e. indicators and criteria), goals , initiating discussion about gastrostomy , and criteria for preferred method of placement ; and barriers and support needs in indication and decision-making. Descriptive analysis was used for quantitative responses, thematic, and content analysis for qualitative data., Results: Twenty-nine physicians (41%) of 27 ALS care teams (71%) responded. Timing of indication : physicians agreed on important indicators but not cutoff values/criteria. Goals : optimizing nutritional status (100%), ensuring safe food-intake (72%), and reducing effort of meals (59%). Initiating discussion about gastrostomy : 52% introduces the topic early after diagnosis, 48% at indication. Criteria for method of placement included physician preference (69%), availability of service (21%), lower complication risk (17%), contraindication (59%), and patient preference (24%). Reported barriers (69% of respondents) were: patient readiness (52%), timing of indication (31%), and organizational barriers (18%). Support needs (62%): evidence-based timing of indication (35%) and tailored patient education (31%)., Conclusions: There is practice variation in the timing of first introduction of gastrostomy and preferred method of placement, but agreement on goals and indicators . More evidence on optimal timing of gastrostomy placement is needed. However, until then early and regular discussion of the topic of gastrostomy and better patient information may promote patient readiness and support patient choice.

Background: Amyotrophic lateral sclerosis (ALS), progressive muscular atrophy (PMA) and primary lateral sclerosis (PLS), together referred to as ALS, are life-limiting diagnoses affecting not only patients but also the families surrounding them, especially when dependent children are involved. Despite previous research highlighting the vulnerability of children in these families, they are, as yet, often overlooked in healthcare. Efforts are needed to better support children in families living with ALS, both directly and through strengthening parents in their parental role. This study sought to gain a better understanding of parental and children's experiences, struggles and support needs in families living with ALS., Methods: Semi-structured interviews were conducted with 8 parents with ALS, 13 well parents and 15 children, together representing 17 families. Interview data were analyzed using qualitative content analysis., Results: Three major themes were identified relating to (1) ALS-related transformations in families' homes, activities, roles and relationships, that trigger (2) distress among families, which, in turn, evokes (3) emotional, psychological, educational and practical support needs. For emotional and practical support, parents and children mainly rely on their own family and social network, whereas they seek educational and psychological support from healthcare professionals., Conclusions: Our findings imply that ALS care professionals may foster family adjustment to living with ALS, most notably through encouraging parents to engage in a dialogue with their children about the many transformations, struggles and needs imposed by ALS and teaching them how to start the dialogue., (© 2022. The Author(s).)

Background: Personalized ENCALS survival prediction model reliably estimates the personalized prognosis of patients with amyotrophic lateral sclerosis. Concerns were raised on discussing personalized prognosis without causing anxiety and destroying hope. Tailoring communication to patient readiness and patient needs mediates the impact of prognostic disclosure. We developed a communication guide to support physicians in discussing personalized prognosis tailored to individual needs and preferences of people with ALS and their families., Methods: A multidisciplinary working group of neurologists, rehabilitation physicians, and healthcare researchers A) identified relevant topics for guidance, B) conducted a systematic review on needs of patients regarding prognostic discussion in life-limiting disease, C) drafted recommendations based on evidence and expert opinion, and refined and finalized these recommendations in consensus rounds, based on feedback of an expert advisory panel (patients, family member, ethicist, and spiritual counsellor)., Results: A) Topics identified for guidance were 1) filling in the ENCALS survival model, and interpreting outcomes and uncertainty, and 2) tailoring discussion to individual needs and preferences of patients (information needs, role and needs of family, severe cognitive impairment or frontotemporal dementia, and non-western patients). B) 17 studies were included in the systematic review. C) Consensus procedures on drafted recommendations focused on selection of outcomes, uncertainty about estimated survival, culturally sensitive communication, and lack of decisional capacity. Recommendations for discussing the prognosis include the following: discuss prognosis based on the prognostic groups and their median survival, or, if more precise information is desired, on the interquartile range of the survival probability. Investigate needs and preferences of the patients and their families for prognostic disclosure, regardless of cultural background. If the patient does not want to know their prognosis, with patient permission discuss the prognosis with their family. If the patient is judged to lack decisional capacity, ask the family if they want to discuss the prognosis. Tailor prognostic disclosure step by step, discuss it in terms of time range, and emphasize uncertainty of individual survival time., Conclusion: This communication guide supports physicians in tailoring discussion of personalized prognosis to the individual needs and preferences of people with ALS and their families.

Background: Poor monitoring of respiratory function may lead to late initiation of non-invasive ventilation (NIV) in patients with motor neuron diseases (MND). Monitoring could be improved by remotely assessing hypoventilation symptoms between clinic visits. We aimed to determine which patient-reported hypoventilation symptoms are best for screening reduced respiratory function in patients with MND, and compared them to the respiratory domain of the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R)., Methods: This prospective multi-center study included 100 patients with MND, who were able to perform a supine vital capacity test. Reduced respiratory function was defined as a predicted supine vital capacity ≤ 80%. We developed a 14-item hypoventilation symptom questionnaire (HYSQ) based on guidelines, expert opinion and think-aloud interviews with patients. Symptoms of the HYSQ were related to dyspnea, sleep quality, sleepiness/fatigue and pneumonia. The diagnostic performances of these symptoms and the ALSFRS-R respiratory domain were determined from the receiver operating characteristic (ROC) curves, area under the curve (AUC), sensitivity, specificity, predictive values and accuracy., Results: Dyspnea-related symptoms (dyspnea while eating/talking, while lying flat and during light activity) were combined into the MND Dyspnea Scale (MND-DS). ROC curves showed that the MND-DS had the best diagnostic performance, with the highest AUC = 0.72, sensitivity = 75% and accuracy = 71%. Sleep-quality symptoms, sleepiness/fatigue-related symptoms and the ALSFRS-R respiratory domain showed weak diagnostic performance., Conclusion: The diagnostic performance of the MND-DS was better than the respiratory domain of the ALSFRS-R for screening reduced respiratory function in patients with MND, and is, therefore, the preferred method for (remotely) monitoring respiratory function.

Objective: To evaluate whether a blended (face-to-face and online) psychosocial support program for caregivers of patients with Amyotrophic Lateral Sclerosis (ALS) and Progressive Muscular Atrophy (PMA), aimed at enhancing feeling of control over caregiving, reduces psychological distress. Methods: A randomized controlled trial using a wait-list control design was conducted. Caregiver-patient dyads were randomly assigned to either the support program ( n  = 74) or to a wait-list control group ( n  = 74). The support program, based on Acceptance and Commitment Therapy, consists of 1 face-to-face contact, 6 online guided modules and 1 telephone contact. Participants filled in questionnaires at baseline, 3 and 6 months. Caregivers' feeling of control over caregiving was assessed using two self-efficacy measures. Primary outcome was caregivers' psychological distress. Secondary outcomes included caregiver burden, caregiver quality of life and patients' quality of life and psychological distress. Intention-to-treat and per-protocol analyses were performed using linear mixed models. Results: The support program had no effect on the primary or secondary outcomes, despite a significant positive effect on the intervening variable self-efficacy with regard to control over thoughts. Almost half of the caregivers did not complete the intervention with the most frequently reported reason being lack of time. Caregivers who completed the intervention evaluated the support program positively. Conclusions: The support program did not reduce distress of partners of patients with ALS/PMA but may be beneficial by increasing feeling of control over the caregiving situation. The high level of intervention drop outs may have limited the ability to detect an intervention effect. Trial registration: Netherlands Trial Registry NTR5734, registered 28 March 2016.

Objective : We aimed to provide an overview of telehealth used in the care for patients with amyotrophic lateral sclerosis (ALS), and identify the barriers to and facilitators of its implementation. Methods : We searched Pubmed and Embase to identify relevant articles. Full-text articles with original research reporting on the use of telehealth in ALS care, were included. Data were synthesized using the Consolidation Framework for Implementation Research. Two authors independently screened articles based on the inclusion criteria. Results : Sixteen articles were included that investigated three types of telehealth: Videoconferencing, home-based self-monitoring and remote NIV monitoring. Telehealth was mainly used by patients with respiratory impairment and focused on monitoring respiratory function. Facilitators for telehealth implementation were a positive attitude of patients (and caregivers) toward telehealth and the provision of training and ongoing support. Healthcare professionals were more likely to have a negative attitude toward telehealth, due to the lack of personal evaluation/contact and technical issues; this was a known barrier. Other important barriers to telehealth were lack of reimbursement and cost-effectiveness analyses. Barriers and facilitators identified in this review correspond to known determinants found in other healthcare settings. Conclusions : Our findings show that telehealth in ALS care is well-received by patients and their caregivers. Healthcare professionals, however, show mixed experiences and perceive barriers to telehealth use. Challenges related to finance and legislation may hinder telehealth implementation in ALS care. Future research should report the barriers and facilitators of implementation and determine the cost-effectiveness of telehealth.

Objective: To evaluate the use of telehealth as part of specialized care for patients with amyotrophic lateral sclerosis (ALS) and the user experiences of patients and healthcare professionals. Methods: Fifty patients with ALS were recruited from a single specialist center and used telehealth, consisting of an ALS-app for self-monitoring and messaging, alerts for symptom-worsening, and nurse practitioner follow-up. Patients self-monitored their well-being (daily report), body weight (weekly) and functional status (monthly). The use of the telehealth service was evaluated through adoption rate, dropout rate and adherence to self-monitoring. User-experiences were collected through online surveys among 23 patients and nine healthcare professionals, and interviews with 12 patients. Results: The adoption rate was 80%, dropout rate 4% and median follow-up was 11 months. Good adherence was seen in 49% of patients for well-being, 83% for body weight and 87% for functional assessment. For patients who discontinued using telehealth due to the end-of-life phase, median time between last measurement and death was 19 days. The majority of patients experienced using telehealth as easy, helpful, not burdensome, and reported satisfaction with flexible clinic visits and the continuity of care. Healthcare professionals reported that telehealth was of added value in ALS-care. Conclusions: ALS-care supplemented by home-monitoring and nurse practitioner follow-up was shown to be suitable and widely accepted by patients and healthcare professionals in our ALS clinic. Success factors were low self-monitoring burden, a user-friendly platform and the provision of personalized feedback. Further research is needed to replicate these findings in other ALS clinics.

Caregivers of Amyotrophic Lateral Sclerosis (ALS) and Progressive Muscular Atrophy (PMA) patients often experience psychological distress. Yet, it is unclear which factors explain the variance in psychological distress. This study seeks to evaluate how care demands and perceived control over caregiving influence psychological distress using moderation and mediation analysis. Data were collected as part of a RCT and 148 partners of patients with ALS or PMA were included. Psychological distress was assessed using the Hospital Anxiety and Depression Scale. Care demands were operationalized as physical functioning (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) and behavioural changes of the patient (Amyotrophic Lateral Sclerosis-Frontotemporal Dementia-Questionnaire). Perceived control over caregiving was assessed using items adapted from the Job Content Questionnaire. Results showed that more behavioural changes and lower perceived control over caregiving were associated with higher levels of psychological distress in caregivers. Patients' physical functioning was not significantly related to caregivers' psychological distress. No moderation or mediation effects were found of perceived control over caregiving on the relationship between demand and psychological distress. Monitoring, psychoeducation and caregiver support with regard to behavioural changes in patients, seem to be important for the wellbeing of caregivers. Caregivers' perceived control might be a target for future interventions.

Background: Partners are often the main caregivers in the care for patients with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA). Providing care during the progressive and fatal disease course of these patients is challenging and many caregivers experience feelings of distress. A blended psychosocial support program based on Acceptance and Commitment Therapy was developed to support partners of patients with ALS and PMA. The aim of this qualitative study is to gather insight into experiences with different components of the support program (program evaluation) and to discover what caregivers gained from following the program (mechanisms of impact)., Methods: Individual in-depth interviews, about caregivers' experiences with the support program were conducted with 23 caregivers of ALS/PMA patients enrolled in a randomized controlled trial designed to measure the effectiveness of the blended psychosocial support program. The program, performed under the guidance of a psychologist, consists of psychoeducation, psychological and mindfulness exercises, practical tips and information, and options for peer contact. Interviews were audio-recorded, transcribed verbatim and analyzed thematically., Results: The program evaluation showed that caregivers perceived each component of the program as beneficial but ambivalent reactions were expressed about the mindfulness exercises and peer contact functions. Caregivers expressed the need for a more personalized program with respect to the order and timing of the modules and wanted to continue the support program for a longer time. The main mechanism of impact of the program that caregivers reported was that they became more aware of their own situation. They further indicated that the program helped them to perceive control over the caregiving situation, to accept negative emotions and thoughts, to be there for their partner and feel acknowledged., Conclusions: The blended psychosocial support program for caregivers of patients with ALS/PMA is valued by caregivers for enhancing self-reflection on their challenging situation which stimulated them to make choices in line with their own needs and increased their feeling of control over caregiving. The different components of the program were overall appreciated by caregivers, but the mindfulness and peer support components should be further adapted to the needs of the caregivers., Trial Registration: Dutch Trialregister NTR5734 , registered 28 March 2016.

Background: Informal caregivers of patients with Amyotrophic Lateral Sclerosis (ALS) or Progressive Muscular Atrophy (PMA) face stressful demands due to severe impairments and prospect of early death of the patients they care for. Caregivers often experience feelings of psychological distress and caregiver burden, but supportive interventions are lacking. The objective of this study is to investigate the effectiveness of a psychosocial support program aimed at enhancing feelings of control over caregiving tasks and reducing psychological distress. This support program is based on an existing program for adult partners of people with cancer and is adapted to meet the needs of ALS caregivers., Methods: This study is a randomized controlled trial using a wait-list control design. One hundred and forty caregiver-patient dyads, recruited from a nationwide database and through the website of the Dutch ALS Center, will be either randomized to a support program or a wait-list control group. The blended intervention is based on Acceptance and Commitment Therapy and consists of 1 face-to-face contact, 6 online guided modules and 1 telephone contact. The intervention can be worked through in 8 weeks. The effectiveness and the participants' satisfaction with the intervention will be evaluated using a mixed method design. Caregivers and patients will be asked to fill in questionnaires on 4 occasions during the study: baseline, 3 months, 6 months and 9 months. The main study outcome is the psychological distress of the caregiver assessed with the Hospital Anxiety and Depression Scale. Secondary outcomes are caregiver burden, caregiver quality of life, quality of life of the patient and psychological distress of the patient. Group differences in primary and secondary outcomes at 6 months will be compared with linear mixed model analysis. In a subgroup of caregivers we will explore experiences with the support program through semi-structured interviews. Usage of the online modules will be logged., Discussion: The study will provide insights into the effectiveness of a blended psychosocial support program on psychological distress of caregivers of patients with ALS or PMA, as well as into indirect relations with patients' wellbeing., Trial Registration: Netherlands Trial Registry NTR5734 , registered 28 March 2016.

Background: Informal caregivers of patients with amyotrophic lateral sclerosis experience increased levels of caregiver burden as the disease progresses. Insight in the factors related to caregiver burden is needed in order to develop supportive interventions., Aim: To evaluate the evidence on patient and caregiver factors associated with caregiver burden in amyotrophic lateral sclerosis informal caregivers., Design: A systematic review., Data Sources: Four electronic databases were searched up to 2017. Studies that investigated quantitative relations between patient or caregiver factors and caregiver burden were included. The overall quality of evidence for factors was assessed using the Grading of Recommendations Assessment, Development and Evaluation approach., Results: A total of 25 articles were included. High quality of evidence was found for the relation between caregiver burden and the factor "behavioral impairments." Moderate quality of evidence was found for the relations between caregiver burden and the factors "feelings of depression" of the caregiver and "physical functioning" of the patient. The remaining rated caregiver factors-"feelings of anxiety," "distress," "social support," "family functioning," and "age"-and patient factors-"bulbar function," "motor function," "respiratory function," "disease duration," "disinhibition," "executive functioning," "cognitive functioning," "feelings of depression," and "age"-showed low to very low quality of evidence for their association with caregiver burden., Conclusion: Higher caregiver burden is associated with greater behavioral and physical impairment of the patient and with more depressive feelings of the caregiver. This knowledge enables the identification of caregivers at risk for caregiver burden and guides the development of interventions to diminish caregiver burden.

Objective: To examine the longitudinal associations between caregiver strain and patients' clinical and psychosocial characteristics as well as caregivers' psychosocial characteristics., Methods: At 4-month intervals during the 12 months study period, longitudinal data on caregiver strain and patient and caregiver factors potentially associated with caregiver strain were collected from 126 couples, who participated in a randomised controlled trial on the effectiveness of case management in amyotrophic lateral sclerosis (ALS). Caregiver strain was assessed with the Caregiver Strain Index (CSI). Patient and caregiver factors included sociodemographic characteristics, distress, coping style and perceived quality of care, as well as the patient's functional status and emotional functioning. Multilevel regression analyses were performed., Results: Caregiver strain increased significantly during the study period (β=0.315 points/months, p<0.001) and was significantly associated with patient time-dependent factors functional status (β=-0.131 points/months, p<0.001) and emotional functioning (β=0.022 points/months, p=0.03), and caregiver time-dependent factors passive coping style (β=0.152 points/months, p=0.03), symptoms of anxiety (β=0.186 points/months, p<0.001) and perceived quality of care for the caregiver (β=-0.452 points/months, p<0.001)., Conclusions: Our study has identified that apart from the patient's physical disability and emotional well-being, a passive coping style of the caregiver, increased symptoms of anxiety and feeling less supported by the ALS-team impact on caregiver strain. The multidisciplinary teams involved with the care of patients with ALS need to be aware of these factors and increase their attention for the caregiver. This will help guide the development of evidence-based supportive interventions that focus on caregiver's coping style and avoiding distress., Trial Registration Number: Netherlands Trial Register, number NTR1270., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/)

Our objective was to explore the needs and value of case management according to patients with amyotrophic lateral sclerosis (ALS), their spousal caregivers, and health care professionals in the context of multidisciplinary ALS care. We undertook semi-structured interviews with 10 patients with ALS, their caregivers (n = 10) and their ALS health care professionals (n = 10), and held a focus group (n = 20). We transcribed the audio-taped interviews and analysed all data thematically. Participants indicated that in certain circumstances case management can have an added value. They identified factors for receptiveness to case management: adequacy of usual care, rate of disease progression, and degree of social network support and personal factors of patients and spousal caregivers. Participants valued the time for consultation, house calls and proactive approach of the case manager. Patients with ALS and caregivers appreciated emotional support, whereas professionals did not mention the importance of emotional support by the case manager. In conclusion, ALS teams can consider implementation of valued aspects of case management (accessibility, ample time, proactive approach, emotional support) in the usual multidisciplinary ALS care. Additional support might be provided to patients with rapidly progressive disease course, passive coping style and small social network.

The progressive course of amyotrophic lateral sclerosis (ALS) results in an ever-changing spectrum of the care needs of patients with ALS. Knowledge of prognostic factors for the functional course of ALS may enhance clinical prediction and improve the timing of appropriate interventions. Our objective was to systematically review the evidence regarding prognostic factors for the rate of functional decline of patients with ALS, assessed with versions of the ALS Functional Rating Scale (ALSFRS). Two reviewers independently assessed the methodological quality of the thirteen included studies using the Quality in Prognosis Studies (QUIPS) tool. The overall quality of evidence for each prognostic factor was assessed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, considering risk of bias, imprecision, inconsistency, indirectness, and publication bias. The quality of evidence for the prognostic value of age at onset, site of onset, time from symptom onset to diagnosis, and ALSFRS-Revised baseline score was low, mainly due to the limited data and inconsistency of results in the small number of studies included. The prognostic value of initial rate of disease progression, age at diagnosis, forced vital capacity, frontotemporal dementia, body mass index, and comorbidity remains unclear. We conclude that the current evidence on prognostic factors for functional decline in ALS is insufficient to allow the development of a prediction tool that can support clinical decisions. Given the limited data, future prognostic studies may need to focus on factors that have a predictive value for a decline in ALSFRS(-R) domain scores, preferably based on internationally collected and shared data.

The timely provision of assistive devices and home adaptations (ADHA) is crucial in the management of patients with amyotrophic lateral sclerosis (ALS) in order to maintain their independence and relieve their caregivers. Our objective was to study the experiences of patients with ALS during the process of procuring ADHA. We sent a cross-sectional questionnaire survey addressing issues concerning the application for and provision process of ADHA to 239 patients with ALS registered at one of the three tertiary academic diagnostic centres within the Netherlands ALS Centre. One hundred and fifty-nine (89%) of the 179 responding patients (response rate 75%) had experience with the procurement process and 93 (58%) of them indicated problems in obtaining ADHA. The most reported problems were delay (42%) and the authorities' lack of disease knowledge (24%). Patients viewed these issues as the most prominent requiring improvement. In conclusion, the main problems perceived by patients indicate that increasing awareness of ALS and promoting a proactive attitude among ALS care professionals towards the application for ADHA may contribute positively to the quality of ALS care.

Objectives: To study the effect of case management on quality of life, caregiver strain, and perceived quality of care (QOC) in patients with amyotrophic lateral sclerosis (ALS) and their caregivers., Methods: We conducted a multicenter cluster randomized controlled trial with the multidisciplinary ALS care team as the unit of randomization. During 12 months, patients with ALS and their caregivers received case management plus usual care or usual care alone. Outcome measures were the 40-item ALS Assessment Questionnaire (ALSAQ-40), Emotional Functioning domain (EF); the Caregiver Strain Index (CSI); and the QOC score. These measures were assessed at baseline and at 4, 8, and 12 months., Results: Case management resulted in no changes in ALSAQ-40 EF, CSI, or QOC from baseline to 12 months. ALSAQ-40 EF scores in both groups were similar at baseline and did not change over time (p = 0.331). CSI scores in both groups increased significantly (p < 0.0001). Patients with ALS from both groups rated their perceived QOC at baseline with a median score of 8, which did not change significantly during follow-up., Conclusion: Within the context of multidisciplinary ALS care teams, case management appears to confer no benefit for patients with ALS or their caregivers., Classification of Evidence: This study provides Class III evidence that case management in addition to multidisciplinary ALS care does not significantly improve health-related quality of life of patients with ALS.

Objective: The aim of this study was to investigate unidimensionality and differential item functioning of the SF-36 physical functioning scale (PF10) in patients with various neurological disorders., Patients: PATIENTS post-stroke (n = 198), with multiple sclerosis (n = 151) and amyotrophic lateral sclerosis (n = 193) participated., Methods: Unidimensionality of the PF10 within the patient groups was investigated by performing a separate Rasch analysis for each group. Differential item functioning was investigated in a pooled Rasch analysis of the 3 groups., Results: Within each group, all items fitted the Rasch model, except the "bathing/dressing" item in the amyotrophic lateral sclerosis group. The pooled analysis showed inadequate fit to the Rasch model for one item ("walking several hundred metres"). Of the other 9 fitting items, 5 showed differential item functioning for stroke vs multiple sclerosis and amyotrophic lateral sclerosis, while no differential item functioning was found between multiple sclerosis and amyotrophic lateral sclerosis., Conclusion: All items of the PF10, except one for the amyotrophic lateral sclerosis group, form a unidimensional scale, supporting the use of a sum score as a measure of physical functioning within these diagnostic groups. When comparing the data of patients after stroke, with that of patients with multiple sclerosis and/or amyotrophic lateral sclerosis patients, adjustments for differential item functioning are required.

To determine the feasibility, reliability, and sensitivity of remotely monitoring muscle strength loss of knee extensors using a novel portable fixed dynamometer (PFD) in patients with amyotrophic lateral sclerosis (ALS). We conducted a pilot study with a newly developed device to measure knee extension strength. Patients performed unsupervised PFD measurements, biweekly, for 6 months at home. We evaluated feasibility using adherence and a device-specific questionnaire. Reliability was assessed by (1) comparing unsupervised and supervised measurements to identify systematic bias, and (2) comparing consecutive unsupervised measurements to determine test-retest reliability expressed as intraclass correlation coefficient (ICC) and standard error of measurement (SEM). Sensitivity to detect longitudinal change was described using linear mixed-effects models. We enrolled 18 patients with ALS. Adherence was 86%, where all patients found that the device suitable to measure muscle strength at home; 4 patients (24%) found the measurements burdensome. The correlation between (un)supervised measurements was excellent (Pearson's r 0.97, 95%CI; 0.94 − 0.99) and no systematic bias was present (mean difference 0.13, 95%CI; −2.22 − 2.48, p = 0.91). Unsupervised measurements had excellent test-retest reliability with an average ICC of 0.97 (95%CI: 0.94 − 0.99) and SEM of 5.8% (95%CI: 4.8 − 7.0). Muscle strength declined monthly by 1.9 %predicted points (95%CI; −3.0 to −0.9, p = 0.001). Using the PFD, it proved feasible to perform knee extension strength measurements at home which were reliable and sensitive for detecting muscle strength loss. Larger studies are warranted to compare the device with conventional outcomes. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to explore the support needs of Dutch informal caregivers of patients with amyotrophic lateral sclerosis (ALS).Method: Individual semi-structured interviews were conducted with 21 caregivers of ALS patients. Audio-taped interviews were transcribed and data were analyzed thematically.Result: A total of four global support needs emerged: "more personal time", "assistance in applying for resources", "counseling", and "peer contact". Despite their needs, caregivers are reluctant to apply for and accept support. They saw their own needs as secondary to the needs of the patients.Significance Of Results: ALS seems to lead to an intensive caregiving situation with multiple needs emerging in a short period. This study offers targets for the development of supportive interventions. A proactive approach seems essential, acknowledging the importance of the role of the caregivers in the care process at an early stage, informing them about the risk of burden, monitoring their wellbeing, and repeatedly offering support opportunities. Using e-health may help tailor interventions to the caregivers' support needs. [ABSTRACT FROM AUTHOR]

The ENCALS survival prediction model offers patients with amyotrophic lateral sclerosis (ALS) the opportunity to receive a personalized prognosis of survival at the time of diagnosis. We explored experiences of patients with ALS, caregivers, and physicians with discussing personalized prognosis through interviews with patients and their caregivers, and in a focus group of physicians. Thematic analysis revealed four themes with seven subthemes; these were recognized by the focus group. First, tailored communication: physician's communication style and information provision mediated emotional impact and increased satisfaction with communication. Second, personal factors: coping style, illness experiences, and information needs affected patient and caregiver coping with the prognosis. Third, emotional impact ranged from happy and reassuring to regret. Fourth, regaining control over the future: participants found it helpful in looking towards the future, and emphasized the importance of quality over quantity of life. Personalized prognosis can be discussed with minimal adverse emotional impact. How it is communicated—i.e., tailored to individual needs—is as important as what is communicated—i.e., a good or poor prognosis. Discussing personalized prognosis may help patients with ALS and their caregivers regain control over the future and facilitate planning of the future (care). For many patients, quality of life matters more than quantity of time remaining. [ABSTRACT FROM AUTHOR]

Despite recent and potent technological advances, the real-world implementation of remote digital health technology in the care and monitoring of patients with motor neuron disease has not yet been realized. Digital health technology may increase the accessibility to and personalization of care, whereas remote biosensors could optimize the collection of vital clinical parameters, irrespective of patients' ability to visit the clinic. To facilitate the wide-scale adoption of digital health care technology and to align current initiatives, we outline a road map that will identify clinically relevant digital parameters; mediate the development of benefit-to-burden criteria for innovative technology; and direct the validation, harmonization, and adoption of digital health care technology in real-world settings. We define two key end products of the road map: (1) a set of reliable digital parameters to capture data collected under free-living conditions that reflect patient-centric measures and facilitate clinical decision making and (2) an integrated, open-source system that provides personalized feedback to patients, health care providers, clinical researchers, and caregivers and is linked to a flexible and adaptable platform that integrates patient data in real time. Given the ever-changing care needs of patients and the relentless progression rate of motor neuron disease, the adoption of digital health care technology will significantly benefit the delivery of care and accelerate the development of effective treatments. [ABSTRACT FROM AUTHOR]

AbstractObjective: This proof-of-concept study aimed to explore the acceptability and potential benefit of a self-guided online self-compassion intervention to aid resilient coping and reduce emotional distress among patients and caregivers living with ALS. Methods: A single-arm pilot study was conducted in 20 adults living with ALS either as a patient or as a caregiver. Acceptability was examined using questionnaires (n = 20) and semi-structured interviews (n = 9). Potential benefit was assessed as changes in self-compassion, self-criticism and emotional distress, determined using psychological questionnaires at 3 and 6 weeks. Questionnaires were analyzed using linear mixed-effects models and interview data using inductive thematic analysis. Results: Out of 20 participants who started the intervention, 16 completed the study (80%). The majority of study completers (12/16) were satisfied with the intervention, but the data suggest room for improvement in terms of personalization. Qualitative data revealed multiple psychological benefits of using the intervention, including self-kindness, emotional self-awareness and savoring. Although not statistically significant, quantitative data showed positive trends with increased self-compassion (mean difference: 2.07; 95% CI: −.5.76 − 1.63) and reduced self-criticism (mean difference: −2.62; 95% CI: −.1.97 − 7.23) and emotional distress (mean difference: −2.49; 95% CI: −.51 − 5.50) at week 6 compared to baseline. Conclusions: The findings suggest that a self-compassion intervention is acceptable to people living with ALS, but its beneficial effects and the mechanisms involved have yet to be established in larger and more diverse samples, using controlled designs. [ABSTRACT FROM AUTHOR]

Amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) is a systemic and fatal neurodegenerative condition for which there is currently no cure. Informal caregivers play a vital role in supporting the person with ALS, and it is essential to support their wellbeing. This multi-centre, mixed methods descriptive exploratory study describes the complexity of burden and self-defined difficulties as described by the caregivers themselves. Quantitative and qualitative data were collected during face-to-face interviews with informal caregivers from centres in the Netherlands, England, and Ireland. Standardised measures assessed burden, quality of life, and psychological distress; furthermore, an open-ended question was asked about difficult aspects of caregiving. Most caregivers were female, spouse/partners, and lived with the person with ALS for whom they provided care. Significant differences between national cohorts were identified for burden, quality of life, and anxiety. Among the difficulties described were the practical issues associated with the caregiver role and emotional factors such as witnessing a patient's health decline, relationship change, and their own distress. The mixed-methods approach allows for a more nuanced understanding of the burden and difficulties experienced. It is important to generate an evidence base to support the psychosocial wellbeing and brain health of informal caregivers. [ABSTRACT FROM AUTHOR]