Predicting Ambulatory Capacity in Parkinson's Disease to Analyze Progression, Biomarkers, and Trial Design.

BACKGROUND: In Parkinson's disease (PD), gait and balance is impaired, relatively resistant to available treatment and associated with falls and disability. Predictive models of ambulatory progression could enhance understanding of gait/balance disturbances and aid in trial design. OBJECTIVES: To predict trajectories of ambulatory abilities from baseline clinical data in early PD, relate trajectories to clinical milestones, compare biomarkers, and evaluate trajectories for enrichment of clinical trials. METHODS: Data from two multicenter, longitudinal, observational studies were used for model training (Tracking Parkinson's, n = 1598) and external testing (Parkinson's Progression Markers Initiative, n = 407). Models were trained and validated to predict individuals as having a "Progressive" or "Stable" trajectory based on changes of ambulatory capacity scores from the Movement Disorders Society Unified Parkinson's Disease Rating Scale parts II and III. Survival analyses compared time-to-clinical milestones and trial outcomes between predicted trajectories. RESULTS: On external evaluation, a support vector machine model predicted Progressive trajectories using baseline clinical data with an accuracy, weighted-F1 (proportionally weighted harmonic mean of precision and sensitivity), and sensitivity/specificity of 0.735, 0.799, and 0.688/0.739, respectively. Over 4 years, the predicted Progressive trajectory was more likely to experience impaired balance, loss of independence, impaired function and cognition. Baseline dopamine transporter imaging and select biomarkers of neurodegeneration were significantly different between predicted trajectory groups. For an 18-month, randomized (1:1) clinical trial, sample size savings up to 30% were possible when enrollment was enriched for the Progressive trajectory versus no enrichment. CONCLUSIONS: It is possible to predict ambulatory abilities from clinical data that are associated with meaningful outcomes in people with early PD. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Pilot trial of The Living Well Toolkit: qualitative analysis and implications for refinement and future implementation.

BACKGROUND: Following a neurological event, people's long-term health and well-being is hampered by a system that struggles to deliver person-centred communication and coordinated care and fails to harness individual and family capability to live well with the condition. We aimed to implement and evaluate a toolkit package to support these processes for people with long-term neurological conditions. METHODS: This is a multi-phased study drawing on the principles of participatory research. In this pilot phase, the toolkit package was introduced to clinicians, who introduced it to clients in four neurorehabilitation settings (inpatient and community-based). Individual and focus group interviews were carried out with clients (n = 10) and clinicians (n = 9). Data were categorised by the four components of Normalisation Process Theory (NPT), and data within each component was then coded inductively. This analysis was used to inform revisions to the toolkit package and wider implementation processes. RESULTS: There was widespread support for the principles underpinning the toolkit package from clients and clinicians. However, it was less clear how the client toolkit could support these principles in clinical practice which impacted buy-in. The flexibility of use of the client toolkit, which we encouraged, made it difficult for clinicians and clients to be clear about its purpose and for clinicians to operationalise in practice. Clinicians and clients identified a number of barriers that limited the time, energy and work users were able or prepared to invest, to the extent that uptake of the toolkit package was modest. Use of the toolkit package appeared more likely when clinicians perceived it to augment existing processes (e.g. goal setting) rather than detract from 'doing' therapy. This analysis was used to inform revisions to the toolkit package, including simplification of the client toolkit, development of videos with examples of use and a modular and reflective training package for clinical services. The refinements were intended to improve sense-making and minimise the cognitive barriers associated with implementation of a new intervention. CONCLUSION: Understanding how supporting the client toolkit could add value to the therapeutic encounter was necessary for clinicians to invest time and perceive the worth of the toolkit package. TRIAL REGISTRATION: ANZCTR: ACTRN12614000537651. Registered 21 May, 2014.

Measuring Neurobehavioral Functioning in People With Traumatic Brain Injury: Rasch Analysis of Neurobehavioral Functioning Inventory.

OBJECTIVE: To examine internal construct validity of the Neurobehavioral Functioning Inventory (NFI) by applying Rasch analysis. SETTING: An outpatient rehabilitation program trial in New Zealand employing a goal-setting intervention in people with traumatic brain injury (TBI). PARTICIPANTS: One hundred eight people (mean age = 46 years; 73% male) between 6 months and 5 years post-TBI. DESIGN: Rasch analysis of the NFI (Partial Credit Model). RESULTS: Three NFI subscales were not unidimensional and at least 4 items in each subscale had disordered response categories. Two items showed differential item functioning by age, 1 item by educational attainment, and 2 items were found to misfit the overall construct. These items were excluded from the total score calculation. The revised scale fit the Rasch model and supported the internal construct validity of the NFI. CONCLUSIONS: Current scoring of the NFI subscales for people with TBI in New Zealand does not meet the requirements of the Rasch model. The revised version of NFI can improve the interpretation of scores but should be further tested with people with TBI in other settings.

A personalised activity programme helps reduce fatigue in people after Guillain-Barré Syndrome in a replicated single system design.

PURPOSE: We evaluated the effectiveness of an online activity-focussed programme to decrease fatigue in people with prior Guillain-Barré Syndrome and explored the perspectives of participants regarding the acceptability and feasibility of the programme. MATERIALS AND METHODS: We recruited eight people diagnosed with Guillain-Barré Syndrome more than two years previously who still had fatigue limiting daily activity. We used mixed methods with a replicated single system design using repeated outcome measures across the three phases (baseline, intervention, follow-up) to evaluate the impact of the intervention on fatigue, activity, wellbeing and confidence to exercise. We used qualitative interviews to explore participants' perspectives of the programme. RESULTS: All participants developed a personalized plan to manage fatigue using goals and feedback, which was effective in reducing fatigue for most participants. Participants were positive about what they had learnt about fatigue, themselves and strategies to manage fatigue. Some participants also experienced improvements in activity, exercise confidence and health and wellbeing. Not all changes were sustained past the follow-up period, which reflects participants' differing levels of confidence to continue with their plan. CONCLUSION: Graduated physical activity in association with developing a personalised plan were key features effective in managing fatigue after Guillain-Barré Syndrome.

Carefully graded activity can improve fatigue for people with Guillain-Barré Syndrome.Telehealth is a useful medium for delivering a fatigue management programme as it removes the need to travel and improves access for those who live remotely.Collaboratively developing a fatigue management programme that is individualized to a person's context is probable to contribute to their sense of ownership and likelihood to sustain the plan.Although people may have the skills and knowledge to set goals, use feedback and change their plan, many appreciate the accountability they perceive from ongoing sessions for fatigue management.

Randomised pragmatic waitlist trial with process evaluation investigating the effectiveness of peer support after brain injury: protocol.

INTRODUCTION: Traumatic brain injury (TBI) is an important global health problem. Formal service provision fails to address the ongoing needs of people with TBI and their family in the context of a social and relational process of learning to live with and adapt to life after TBI. Our feasibility study reported peer support after TBI is acceptable to both mentors and mentees with reported benefits indicating a high potential for effectiveness and likelihood of improving outcomes for both mentees and their mentors. OBJECTIVES: To (a) test the effectiveness of a peer support intervention for improving participation, health and well-being outcomes after TBI and (b) determine key process variables relating to intervention, context and implementation to underpin an evidence-based framework for ongoing service provision. METHODS AND ANALYSIS: A randomised pragmatic waitlist trial with process evaluation. Mentee participants (n=46) will be included if they have moderate or severe TBI and are no more than 18 months post-injury. Mentor participants (n=18) will be people with TBI up to 6 years after injury, who were discharged from inpatient rehabilitation at least 1 year prior. The primary outcome will be mentee participation, measured using the Impact on Participation and Autonomy questionnaire after 22 weeks. Primary analysis of the continuous variables will be analysis of covariance with baseline measurement as a covariate and randomised treatment as the main explanatory predictor variable at 22 weeks. Process evaluation will include analysis of intervention-related data and qualitative data collected from mentors and service coordinators. Data synthesis will inform the development of a service framework for future implementation. ETHICS AND DISSEMINATION: Ethics approval has been obtained from the New Zealand Health and Disability Ethics Committee (19/NTB/82) and Auckland University of Technology Ethics Committee (19/345). Dissemination of findings will be via traditional academic routes including publication in internationally recognised peer-reviewed journals. TRIAL REGISTRATION NUMBER: ACTRN12619001002178.

Developing better digital health measures of Parkinson's disease using free living data and a crowdsourced data analysis challenge.

One of the promising opportunities of digital health is its potential to lead to more holistic understandings of diseases by interacting with the daily life of patients and through the collection of large amounts of real-world data. Validating and benchmarking indicators of disease severity in the home setting is difficult, however, given the large number of confounders present in the real world and the challenges in collecting ground truth data in the home. Here we leverage two datasets collected from patients with Parkinson's disease, which couples continuous wrist-worn accelerometer data with frequent symptom reports in the home setting, to develop digital biomarkers of symptom severity. Using these data, we performed a public benchmarking challenge in which participants were asked to build measures of severity across 3 symptoms (on/off medication, dyskinesia, and tremor). 42 teams participated and performance was improved over baseline models for each subchallenge. Additional ensemble modeling across submissions further improved performance, and the top models validated in a subset of patients whose symptoms were observed and rated by trained clinicians.

Embedding the living well toolkit into service delivery - A complex process.

Objective: To embed the Living Well Toolkit package and to understand how it was implemented at each site and to explore the experiences of users. Methods: The toolkit package was introduced in four rehabilitation settings using a tailored implementation process negotiated with each site. The varied data sources were analysed drawing on directed content analysis. Results: Clients with neurological conditions and clinicians initially weighed the merits of the toolkit package. A positive weighing up was prerequisite for deciding to use. Clinicians described considerable thought and planning to make the toolkit package fit and flow in clinical practice. Users of the toolkit package described ways in which it shaped their thinking. Conclusion: Implementation of the toolkit package was a complex process for clinicians and services, involving ongoing work to optimise its impact relative to the client and context. Clinicians and clients who used the toolkit package described positive changes, congruent with person-centred communication. Innovation: The Living Well Toolkit is freely available for all to use. Clinicians who used reflective and responsive thinking to make the toolkit package work found it provided them with a broader perspective of the client.

"It's Always Been a Second Class Cancer": An Exploration of the Experiences and Journeys of Bereaved Family Carers of People with Sarcoma.

Sarcomas are a group of rare and aggressive cancers, which develop in bones and connective tissue throughout the body. Sarcomas account for only 1-2% of all cancers worldwide; however, mortality rates for sarcoma are high with approximately two in four sarcoma patients dying following a diagnosis. Delays in diagnosis, poor management of symptoms, patients' high symptom loads and high carer burden are all associated with carer distress, which may lead to complications after bereavement. The experience of having a family member referred for palliative care is also distressing for carers, with the realisation that their family member is dying. This study aimed to explore the experiences of bereaved family carers of people diagnosed with sarcoma. A qualitative descriptive design using a social constructionist framework was adopted. Interviews were conducted with sixteen participants, and thematic analysis was used to identify patterns in the data. Four overarching themes emerged: beginning the journey; moving through treatment; transitioning to palliative care; and experiencing bereavement. The narratives were coherent and potent, and people reflected on their journeys. Interventions and supports for bereaved carers could include opportunities for counselling to support reflections, supports for developing a narrative such as writing therapy, and preparation for the death of the family member.

Incidence and outcomes of major trauma in New Zealand: findings from a feasibility study of New Zealand's first national trauma registry.

AIMS: The aim of the study was to pilot the feasibility of long-term outcomes data collection from adult major trauma survivors in New Zealand. This initial paper aims to characterise the New Zealand major trauma population in terms of long-term disability and functional outcomes after major trauma. METHODS: A prospective cohort study of adults who had survived major trauma was conducted between June 2015 and December 2016 at two major trauma centres in Auckland. RESULTS: Of 256 trauma referrals, 112 (44%) were confirmed eligible and consented. One hundred completed the survey at six months and 83 at 12 months. A majority of the study sample were male (72%), under 65 years (84%), with a disproportionally higher number of Maori in the sample (23%). At six months post-injury, the majority of participants were categorised as experiencing either moderate disability (37%) or good recovery (42%). Half of the participants experienced moderate pain at both 6 and 12 months post-injury (50% and 52% respectively), and problems with their usual activities at six months post-injury (51%). CONCLUSIONS: Most study participants made a good recovery, but there was still a large group of people experiencing disability, pain and not in paid employment at 12 months post-injury.

Development of a toolkit to enhance care processes for people with a long-term neurological condition: a qualitative descriptive study.

OBJECTIVE: To (A) explore perspectives of people with a long-term neurological condition, and of their family, clinicians and other stakeholders on three key processes: two-way communication, self-management and coordination of long-term care; and (B) use these data to develop a 'Living Well Toolkit', a structural support aiming to enhance the quality of these care processes. DESIGN: This qualitative descriptive study drew on the principles of participatory research. Data from interviews and focus groups with participants (n=25) recruited from five hospital, rehabilitation and community settings in New Zealand were analysed using conventional content analysis. Consultation with a knowledge-user group (n=4) and an implementation champion group (n=4) provided additional operational knowledge important to toolkit development and its integration into clinical practice. RESULTS: Four main, and one overarching, themes were constructed: (1) tailoring care:referring to getting to know the person and their individual circumstances; (2) involving others: representing the importance of negotiating the involvement of others in the person's long-term management process; (3) exchanging knowledge: referring to acknowledging patient expertise; and (4) enabling: highlighting the importance of empowering relationships and processes. The overarching theme was: assume nothing. These themes informed the development of a toolkit comprising of two parts: one to support the person with the long-term neurological condition, and one targeted at clinicians to guide interaction and support their engagement with patients. CONCLUSION: Perspectives of healthcare users, clinicians and other stakeholders were fundamental to the development of the Living Well Toolkit. The findings were used to frame toolkit specifications and highlighted potential operational issues that could prove key to its success. Further research to evaluate its use is now underway.

Making sense of recovery after traumatic brain injury through a peer mentoring intervention: a qualitative exploration.

OBJECTIVE: To explore the acceptability of peer mentoring for people with a traumatic brain injury (TBI) in New Zealand. DESIGN: This is a qualitative descriptive study exploring the experiences reported by mentees and mentors taking part in a feasibility study of peer mentoring. Interviews with five mentees and six mentors were carried out. Data were analysed using conventional content analysis. SETTING: The first mentoring session took place predischarge from the rehabilitation unit. The remaining five sessions took place in mentees' homes or community as preferred. PARTICIPANTS: Twelve people with TBI took part: six mentees (with moderate to severe TBI; aged 18-46) paired with six mentors (moderate to severe TBI >12 months previously; aged 21-59). Pairing occurred before mentee discharge from postacute inpatient brain injury rehabilitation. Mentors had been discharged from rehabilitation following a TBI between 1 and 5 years previously. INTERVENTION: The peer mentoring programme consisted of up to six face-to-face sessions between a mentee and a mentor over a 6-month period. The sessions focused on building rapport, exploring hopes for and supporting participation after discharge through further meetings and supported community activities. RESULTS: Data were synthesised into one overarching theme: making sense of recovery. This occurred through the sharing of experiences and stories; was pivotal to the mentoring relationship; and appeared to benefit both mentees and mentors. Mentors were perceived as valued experts because of their personal experience of injury and recovery, and could provide support in ways that were different from that provided by clinicians or family members. Mentors required support to manage the uncertainties inherent in the role. CONCLUSIONS: The insight mentors developed through their own lived experience established them as a trusted and credible source of hope and support for people re-engaging in the community post-TBI. These findings indicate the potential for mentoring to result in positive outcomes.