The GOAL-Hem journey: Shared decision making and patient-centred outcomes.

INTRODUCTION: GOAL-Hem is a novel, haemophilia-specific, patient-centred outcome measure (PCOM) based on goal attainment scaling, allowing people with haemophilia (PwH) to set and monitor the attainment of individualized goals for treatment. AIM: To provide a thorough overview of the creation, validation, and development of GOAL-Hem. METHODS: Clinician workshops were held to develop a haemophilia-specific goal menu. Qualitative data from semistructured interviews with PwH and their caregivers guided further revisions to the goal menu (i.e., goal domains and descriptors). A feasibility study was performed including a 12-week, prospective, noninterventional evaluation involving clinicians and PwH at four US haemophilia treatment centres. Finally, the Patient Voice Study gathered feedback from PwH and their caregivers via an online survey, interviews, and a focus group. RESULTS: The feasibility study validated GOAL-Hem with successful outcomes in construct/content validity and responsiveness, including a large effect in patient- and clinician-rated goal attainments. The Patient Voice Study led to significant refinement of GOAL-Hem goals and descriptors, resulting in a more straightforward and relatable menu for PwH and their caregivers. Overall, GOAL-Hem captured qualitative data in areas important to PwH and employed quantitative methods to evaluate meaningful changes in those areas. The individualized tool was well equipped to handle the complex and chronic nature of haemophilia and was endorsed by PwH, their caregivers, and clinicians. CONCLUSION: The GOAL-Hem development journey may serve as a roadmap for other PCOMs in a variety of settings, including clinical studies, haemophilia treatment centres for care planning, and as a tool to gather real-world evidence.

Incorporating the patient voice and patient engagement in GOAL-Hem: Advancing patient-centric hemophilia care.

BACKGROUND: Goal Attainment Scaling for Hemophilia (GOAL-Hem) is a novel, hemophilia-specific, validated patient engagement tool and patient-reported outcome instrument. OBJECTIVE: We evaluated the degree to which the language of GOAL-Hem was patient-centric and the content valuable and relevant for people with hemophilia (PWH) and/or their caregivers. PATIENTS/METHODS: Patients and caregivers participated in one of three investigations: an online survey, one-on-one patient interviews, or a focus group. The survey and interviews assessed the clarity and relevance of the GOAL-Hem menu items. Interviews were semistructured, audio recorded, and transcribed verbatim. Feedback from interviews was coded as "clear," "unclear," "remove," or "add." The focus group explored participants' experience of GOAL-Hem and elicited recommendations for implementation. Quotations from focus group and interview transcripts were indexed and charted to emergent themes for analysis. RESULTS: Participants comprised 19 adults with hemophilia and 19 caregivers of children with hemophilia (survey, n = 20; interview, n = 12; focus group, n = 6). After their feedback, 32% (15/48) of goals were retained unchanged. Further feedback resulted in the removal of 45% (286/635) of the goal descriptors, and 30% (193/635) of the retained descriptors were modified. Three new (total = 38) goals and 42 descriptors (total = 368) were added to the menu. Thematic analysis indicated that participants were enthusiastic about patient-centric language, empowered through the goal-setting process, and recognized GOAL-Hem could measure clinically meaningful change. CONCLUSION: By listening closely to patients and caregivers, we refined GOAL-Hem to better capture the experiences of PWH, enhance content validity, and augment implementation strategies. Incorporating the patient voice is integral to developing patient-centered outcome measures.

Patterns of Symptom Tracking by Caregivers and Patients With Dementia and Mild Cognitive Impairment: Cross-sectional Study.

BACKGROUND: Individuals with dementia and mild cognitive impairment (MCI) experience a wide variety of symptoms and challenges that trouble them. To address this heterogeneity, numerous standardized tests are used for diagnosis and prognosis. myGoalNav Dementia is a web-based tool that allows individuals with impairments and their caregivers to identify and track outcomes of greatest importance to them, which may be a less arbitrary and more sensitive way of capturing meaningful change. OBJECTIVE: We aim to explore the most frequent and important symptoms and challenges reported by caregivers and people with dementia and MCI and how this varies according to disease severity. METHODS: This cross-sectional study involved 3909 web-based myGoalNav users (mostly caregivers of people with dementia or MCI) who completed symptom profiles between 2006 and 2019. To make a symptom profile, users selected their most personally meaningful or troublesome dementia-related symptoms to track over time. Users were also asked to rank their chosen symptoms from least to most important, which we called the symptom potency. As the stage of disease for these web-based users was unknown, we applied a supervised staging algorithm, previously trained on clinician-derived data, to classify each profile into 1 of 4 stages: MCI and mild, moderate, and severe dementia. Across these stages, we compared symptom tracking frequency, symptom potency, and the relationship between frequency and potency. RESULTS: Applying the staging algorithm to the 3909 user profiles resulted in 917 (23.46%) MCI, 1596 (40.83%) mild dementia, 514 (13.15%) moderate dementia, and 882 (22.56%) severe dementia profiles. We found that the most frequent symptoms in MCI and mild dementia profiles were similar and comprised early hallmarks of dementia (eg, recent memory and language difficulty). As the stage increased to moderate and severe, the most frequent symptoms were characteristic of loss of independent function (eg, incontinence) and behavioral problems (eg, aggression). The most potent symptoms were similar between stages and generally reflected disruptions in everyday life (eg, problems with hobbies or games, travel, and looking after grandchildren). Symptom frequency was negatively correlated with potency at all stages, and the strength of this relationship increased with increasing disease severity. CONCLUSIONS: Our results emphasize the importance of patient-centricity in MCI and dementia studies and illustrate the valuable real-world evidence that can be collected with digital tools. Here, the most frequent symptoms across the stages reflected our understanding of the typical disease progression. However, the symptoms that were ranked as most personally important by users were generally among the least frequently selected. Through individualization, patient-centered instruments such as myGoalNav can complement standardized measures by capturing these infrequent but potent outcomes.

Impact of Cognitive Impairment on Adults with Multiple Sclerosis and Their Family Caregivers.

BACKGROUND: Cognitive impairment is a prominent feature of multiple sclerosis (MS). However, the impact that cognitive symptoms have on daily life, and the effect this has on persons with MS and their relationships with spouses/partners, remains unclear. This qualitative study sought information on the nature of cognitive impairments experienced and the impact of cognitive impairments on the daily lives of adults with MS and their partners to gain further insights into how health care professionals can best support families. METHODS: Fifteen persons with MS and their spouses/partners participated in separate semistructured telephone interviews. RESULTS: Six themes and several subthemes were identified: the social impact of cognitive impairments in MS, changes to daily living, relationship quality, communication, ways of coping with MS, and the desire for help in managing MS. CONCLUSIONS: These results identified types of support that couples needed and wanted; ways that MS affects couples' social lives; that there are difficulties negotiating changes in roles due to cognitive challenges; that there are difficulties coping with the impact of cognitive challenges on daily living; that couples often have difficulty communicating about the impact of cognitive changes on daily living and related issues, which also contributes to relationship strain; and finally, that most participants felt that they did not understand enough about the cognitive symptoms of MS. We outline key areas to address these identified needs.

The Clinician's Interview-Based Impression of Change (Plus caregiver input) and goal attainment in two dementia drug trials: Clinical meaningfulness and the initial treatment response.

INTRODUCTION: The Clinician's Interview-Based Impression of Change Plus caregiver input (CIBIC-Plus) has been widely used in dementia drug trials to evaluate cognition, behavior, and function. New trials of symptomatic drugs forecast renewed interest in this measure. METHODS: To test its clinical meaningfulness, we examined how CIBIC-Plus performed in two cholinesterase inhibitor trials compared to goal attainment scaling Scale (GAS) scores, a patient-reported outcome measure. RESULTS: Net goal attainment was seen for all but one GAS domains in subjects who improved on the CIBIC-Plus. Subjects who improved initially on CIBIC-Plus scores were likely to remain improved across all other outcomes for each trial's duration, except for Disability Assessment for Dementia scores. DISCUSSION: The initial response to treatment, as assessed by CIBIC-Plus, remained stable for most outcome measures. Even small CIBIC-Plus improvement changes are associated with clinically meaningful change as assessed by GAS. Other tests detect decline better than improvement.

A Patient-Centric Tool to Facilitate Goal Attainment Scaling in Neurogenic Bladder and Bowel Dysfunction: Path to Individualization.

OBJECTIVES: People with neurogenic bladder and/or bowel dysfunction experience diverse challenges that can be difficult to evaluate with standardized outcome measures. Goal attainment scaling (GAS) is an individualized, patient-centric outcome measure that enables patients/caregivers to identify and track their own treatment goals. Because creating goals de novo can be cumbersome, we aimed to develop a neurogenic bladder/bowel dysfunction goal menu to facilitate goal attainment scaling uptake and use. METHODS: We conducted a workshop with 6 expert clinicians to develop an initial menu. Individual interviews with 12 people living with neurogenic bladder and/or bowel dysfunction and 2 clinician panels with 5 additional experts aided us in refining the menu. A thematic framework analysis identified emergent themes for analysis and reporting. RESULTS: Interview participants were adults (median = 36 years, range 25-58), most with spinal cord injury (75%; 9/12). Of 24 goals identified initially, 2 (8%) were not endorsed and were removed, and 3 goals were added. Most participants listed "Impact on Life" goals (eg, Exercise, Emotional Well-Being) among their 5 most important goals (58%; 35/60). Three main themes emerged: challenges posed by incontinence, limitations on everyday life, and need for personalized care. CONCLUSIONS: We developed a clinical outcome assessment tool following a multistep process of representative stakeholder engagement. This patient-centric tool consists of 25 goals specific to people living with neurogenic bladder and/or bowel dysfunction. Asking people what matters most to them can identify important constructs that clinicians might have overlooked.

Development of a symptom menu to facilitate Goal Attainment Scaling in adults with Down syndrome-associated Alzheimer's disease: a qualitative study to identify meaningful symptoms.

BACKGROUND: As life expectancy of people with Down syndrome (DS) increases, so does the risk of Alzheimer's disease (AD). Identifying symptoms and tracking disease progression is especially challenging whenever levels of function vary before the onset of dementia. Goal Attainment Scaling (GAS), an individualized patient-reported outcome, can aid in monitoring disease progression and treatment effectiveness in adults with DS. Here, with clinical input, a validated dementia symptom menu was revised to facilitate GAS in adults living with Down Syndrome-associated Alzheimer's disease (DS-AD). METHODS: Four clinicians with expertise in DS-AD and ten caregivers of adults living with DS-AD participated in semi-structured interviews to review the menu. Each participant reviewed 9-15 goal areas to assess their clarity and comprehensiveness. Responses were systematically and independently coded by two researchers as 'clear', 'modify', 'remove' or 'new'. Caregivers were encouraged to suggest additional items and recommend changes to clarify items. RESULTS: Median caregiver age was 65 years (range 54-77). Most were female (9/10) with ≥15 years of education (10/10). Adults with DS-AD had a median age of 58 years (range 52-61) and either a formal diagnosis (6/10) or clinical suspicion (4/10) of dementia. The initial symptom menu consisted of 67 symptoms each with 2-12 descriptors (589 total). The clinicians' adaptation yielded 58 symptoms each with 4-17 descriptors (580 total). Of these 580 descriptors, caregivers identified 37 (6%) as unclear; these were reworded, and one goal area (4 descriptors) was removed. A further 47 descriptors and one goal area were added to include caregiver-identified concepts. The final menu contained 58 goal areas, each with 7-17 descriptors (623 total). CONCLUSIONS: A comprehensive symptom menu for adults living with DS-AD was developed to facilitate GAS. Incorporating expert clinician opinion and input from caregivers of adults with DS-AD identified meaningful items that incorporate patient/caregiver perspectives.

Use of Patient-Reported Symptoms from an Online Symptom Tracking Tool for Dementia Severity Staging: Development and Validation of a Machine Learning Approach.

BACKGROUND: SymptomGuide Dementia (DGI Clinical Inc) is a publicly available online symptom tracking tool to support caregivers of persons living with dementia. The value of such data are enhanced when the specific dementia stage is identified. OBJECTIVE: We aimed to develop a supervised machine learning algorithm to classify dementia stages based on tracked symptoms. METHODS: We employed clinical data from 717 people from 3 sources: (1) a memory clinic; (2) long-term care; and (3) an open-label trial of donepezil in vascular and mixed dementia (VASPECT). Symptoms were captured with SymptomGuide Dementia. A clinician classified participants into 4 groups using either the Functional Assessment Staging Test or the Global Deterioration Scale as mild cognitive impairment, mild dementia, moderate dementia, or severe dementia. Individualized symptom profiles from the pooled data were used to train machine learning models to predict dementia severity. Models trained with 6 different machine learning algorithms were compared using nested cross-validation to identify the best performing model. Model performance was assessed using measures of balanced accuracy, precision, recall, Cohen κ, area under the receiver operating characteristic curve (AUROC), and area under the precision-recall curve (AUPRC). The best performing algorithm was used to train a model optimized for balanced accuracy. RESULTS: The study population was mostly female (424/717, 59.1%), older adults (mean 77.3 years, SD 10.6, range 40-100) with mild to moderate dementia (332/717, 46.3%). Age, duration of symptoms, 37 unique dementia symptoms, and 10 symptom-derived variables were used to distinguish dementia stages. A model trained with a support vector machine learning algorithm using a one-versus-rest approach showed the best performance. The correct dementia stage was identified with 83% balanced accuracy (Cohen κ=0.81, AUPRC 0.91, AUROC 0.96). The best performance was seen when classifying severe dementia (AUROC 0.99). CONCLUSIONS: A supervised machine learning algorithm exhibited excellent performance in identifying dementia stages based on dementia symptoms reported in an online environment. This novel dementia staging algorithm can be used to describe dementia stage based on user-reported symptoms. This type of symptom recording offers real-world data that reflect important symptoms in people with dementia.

The Symptoms Targeted for Monitoring in a Web-Based Tracking Tool by Caregivers of People With Dementia and Agitation: Cross-Sectional Study.

BACKGROUND: In people with dementia, neuropsychiatric symptoms (NPSs), especially agitation, are associated with worse quality of life and caregiver burden. As NPSs may vary with illness severity, knowledge of how people with dementia and their caregivers describe and rate the importance of agitation symptoms can improve the understanding of the clinical meaningfulness of the manifestations of agitation. The internet provides new opportunities to better understand patient experiences, as patients and caregivers increasingly look to Web-based platforms as a means of managing symptoms. OBJECTIVE: The aim of this study was to examine Web-based reports from a dementia symptom website to better understand the symptoms of agitation and explore how they are being targeted for monitoring by caregivers of people with dementia. METHODS: The Dementia Guide website hosts a Web-based database used by caregivers (97%) and people with dementia (3%). From its 61 dementia symptoms, users can select relevant symptoms that they deem important to monitor or track the effects of treatment. We employed a staging algorithm to determine if individuals had mild cognitive impairment (MCI) or mild, moderate, or severe dementia. Agitation was defined using terms consistent with the International Psychogeriatrics Association's provisional consensus definition. We compared the proportion of people with NPSs and agitation across stages of dementia severity and studied how many agitation-defining descriptors were selected, and how often they occurred, by stage. RESULTS: As of March 2017, 4121 people had used the tracking tool, of whom 2577 provided sufficient data to allow disease severity staging. NPSs were tracked by 2127/2577 (82.54%) and agitation by 1898/2577 (73.65%). The proportion in whom agitation was tracked increased with increasing cognitive impairment: 68.5% (491/717) in people with MCI, and 72.50% (754/1040), 73.3% (378/516), and 90.5% (275/304) in mild, moderate, and severe dementia, respectively (χ23=54.9; P<.001). The number of NPS and agitation descriptors selected also increased with severity (median number of NPSs=1, 2, 2, and 3 for MCI, mild, moderate, and severe dementia, respectively, Kruskal-Wallis H Test H3=250.47; P<.001; median number of agitation descriptors=1, 2, 3, and 4, H3=146.11; P<.001). CONCLUSIONS: NPSs and agitation are common targets for tracking over the course of dementia and appear more frequently with increasing disease severity. These common and distressing symptoms represent clinically meaningful targets in treating people with dementia.

Characterizing the symptom of misplacing objects in people with dementia: findings from an online tracking tool.

OBJECTIVES: Misplacing objects is often reported as a clinically important symptom in dementia. Here we explored misplacing objects in relation to dementia type and stage in an online sample of individuals with dementia and their caregivers. METHODS: Participants were recruited from www.dementiaguide.com, a web-based tracker for common dementia symptoms. Users provided information about symptoms that they selected as important for monitoring. We analysed cross-sectional data from respondents who tracked at least three symptoms, which allowed for staging dementia severity. RESULTS: Of 2,775 users with three-plus symptoms, 787 (28%) identified misplacing objects for symptom tracking. Misplacing objects was monitored by users across all stages of dementia, but was more prevalent in mild and severe dementia. Three common clinical subtypes of misplacing were investigated: lost & found (forgetting the location of items), hidden away (hiding items so others would not find them), and odd places (putting items in usual spots). Of the 787, 96% targeted lost & found, the most frequent type. Odd places (targeted in 56%) significantly increased with dementia severity (p < 0.001). Misplacing objects was most strongly associated with the symptoms of interaction with strangers (OR 4.60, 95% CI: 3.20-6.62), reading (3.68: 2.86-4.73), shopping (3.55: 2.73-4.61) and travel/vacationing (3.31: 2.54-4.31). CONCLUSIONS: Misplacing objects was most often selected for tracking in mild and severe stages of dementia. As disease advances, misplacing more often reflects odd placement of objects rather than their simple loss. Misplacing objects may be a clinically important therapeutic target for improving patients' quality of life and lessening caregiver burden.