Chronic inflammatory neuropathies and their impact on activities and participation

Background: Chronic inflammatory demyelinating polyneuropathy (CIDP) and multifocal motor neuropathy (MMN) are inflammatory neuropathies that can lead to considerable limitations in daily activities and in social participation. However, systematic evaluation of these self-reported limitations is lacking in the currently available studies. Understanding the impact of these diagnoses on patients' life is important to optimize management strategies.Aim: To systematically assess the self-reported limitations in activities and participation and determine associated factors.Methods: A survey study was conducted in 2021 in a cohort of patients with CIDP (n = 257) and MMN (n = 148) from a university hospital. The survey included the Rasch-built Overall Disability Scale and the Utrecht Scale for Evaluation of Rehabilitation-Participation, questions addressing personal and disease-related factors and treatment. Multivariate linear regression analysis was used to determine associations with disease-related and personal factors.Results: A total of 147 CIDP and 103 MMN patients responded. Limitations in activities were reported by 70.7% CIDP and 52.2% MMN patients with moderate to severe limitations in 22.4% and 5.9% patients, respectively. Participation restrictions were reported by 50% of CIDP and 40% of MMN patients, nevertheless satisfaction with participation was high. Fatigue, pain and resilience were independently associated with limitations in activities and satisfaction with participation in CIDP patients.Conclusions: Activity limitations and restrictions in participation are common in CIDP patients and to a lesser extent in MMN patients. Fatigue, pain and resilience independently contributed to perceived limitations in CIDP patients. Referral to a rehabilitation physician is warranted to address these limitations appropriately.</p

Telehealth as part of specialized ALS care: feasibility and user experiences with "ALS home-monitoring and coaching"

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

Current practices and barriers in gastrostomy indication in amyotrophic lateral sclerosis: a survey of ALS care teams in The Netherlands

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

Control in the absence of choice: A qualitative study on decision-making about gastrostomy in people with amyotrophic lateral sclerosis, caregivers, and healthcare professionals

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

Using patient-reported symptoms of dyspnea for screening reduced respiratory function in patients with motor neuron diseases

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

Discussing personalized prognosis in amyotrophic lateral sclerosis: development of a communication guide

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

A validation study of the Caregiver Mastery Scale for partners of patients with acquired brain injury

OBJECTIVE: To validate the Caregiver Mastery Scale for partners of patients with acquired brain injury. DESIGN: The score distributions, internal consistency and convergent validity of the Caregiver Mastery Scale were determined. SUBJECTS: A total of 92 partners (53% male, age 62 years) of patients with acquired brain injury (91% stroke) discharged from inpatient rehabilitation (time since injury 32 months). MAIN MEASURES: Outcome measure: Caregiver Mastery Scale. Reference measures: Caregiver Strain Index, Hospital Anxiety and Depression Scale and CarerQoL. RESULTS: The Caregiver Mastery Scale has a normal distribution, with no floor or ceiling effects. Its internal consistency is acceptable (Cronbach's alpha: 0.75). The convergent validity analyses confirmed our hypothesis that higher scores on the Caregiver Mastery Scale correlate with less burden, lower levels of anxiety and depression and greater well-being. Furthermore, partners scoring high on the Caregiver Mastery Scale mostly scored below the clinical cut-off scores on the Caregiver Strain Index and the anxiety and depression subscales of the Hospital Anxiety and Depression Scale, whereas partners scoring low on the Caregiver Mastery Scale were more likely to score above the cut-off points. CONCLUSION: The Caregiver Mastery Scale is a valid instrument to assess the caregiver mastery of partners of patients with acquired brain injury

A validation study of the Caregiver Mastery Scale for partners of patients with acquired brain injury

OBJECTIVE: To validate the Caregiver Mastery Scale for partners of patients with acquired brain injury. DESIGN: The score distributions, internal consistency and convergent validity of the Caregiver Mastery Scale were determined. SUBJECTS: A total of 92 partners (53% male, age 62 years) of patients with acquired brain injury (91% stroke) discharged from inpatient rehabilitation (time since injury 32 months). MAIN MEASURES: Outcome measure: Caregiver Mastery Scale. Reference measures: Caregiver Strain Index, Hospital Anxiety and Depression Scale and CarerQoL. RESULTS: The Caregiver Mastery Scale has a normal distribution, with no floor or ceiling effects. Its internal consistency is acceptable (Cronbach's alpha: 0.75). The convergent validity analyses confirmed our hypothesis that higher scores on the Caregiver Mastery Scale correlate with less burden, lower levels of anxiety and depression and greater well-being. Furthermore, partners scoring high on the Caregiver Mastery Scale mostly scored below the clinical cut-off scores on the Caregiver Strain Index and the anxiety and depression subscales of the Hospital Anxiety and Depression Scale, whereas partners scoring low on the Caregiver Mastery Scale were more likely to score above the cut-off points. CONCLUSION: The Caregiver Mastery Scale is a valid instrument to assess the caregiver mastery of partners of patients with acquired brain injury