Characteristics, Symptom Severity, and Experiences of Patients Reporting Chronic Kidney Disease in the PatientsLikeMe Online Health Community: Retrospective and Qualitative Study.

BACKGROUND: Chronic kidney disease (CKD) is a major global health burden, and is associated with increased adverse outcomes, poor quality of life, and substantial health care costs. While there is an increasing need to build patient-centered pathways for improving CKD management in clinical care, data in this field are scarce. OBJECTIVE: The aim of this study was to understand patient-reported experiences, symptoms, outcomes, and treatment journeys among patients with CKD through a retrospective and qualitative approach based on data available through PatientsLikeMe (PLM), an online community where patients can connect and share experiences. METHODS: Adult members (aged ≥18 years) with self-reported CKD within 30 days of enrollment, who were not on dialysis, and registered between 2011 and 2018 in the PLM community were eligible for the retrospective study. Patient demographics and disease characteristics/symptoms were collected from this retrospective data set. Qualitative data were collected prospectively through semistructured phone interviews in a subset of patients, and questions were oriented to better understand patients' experiences with CKD and its management. RESULTS: The retrospective data set included 1848 eligible patients with CKD, and median age was 56 years. The majority of patients were female (1217/1841, 66.11%) and most were US residents (1450/1661, 87.30%). Of the patients who reported comorbidities (n=1374), the most common were type 2 diabetes (783/1374, 56.99%), hypertension (664/1374, 48.33%), hypercholesterolemia (439/1374, 31.95%), and diabetic neuropathy (376/1374, 27.37%). The most commonly reported severe or moderate symptoms in patients reporting these symptoms were fatigue (347/484, 71.7%) and pain (278/476, 58.4%). In the qualitative study, 18 eligible patients (13 females) with a median age of 60 years and who were mainly US residents were interviewed. Three key concepts were identified by patients to be important to optimal care and management: listening to patient needs, coordinating health care across providers, and managing clinical care. CONCLUSIONS: This study provides a unique source of real-world information on the patient experience of CKD and its management by utilizing the PLM network. The results reveal the challenges these patients face living with an array of symptoms, and report key concepts identified by patients that can be used to further improve clinical care and management and inform future CKD studies.

In search of a daily physical activity "sweet spot": Piloting a digital tracking intervention for people with multiple sclerosis.

OBJECTIVE: This pilot study tested a course-based intervention to help people with multiple sclerosis (MS) match their daily activity to symptom severity ("sweet spot") using wearable activity trackers. METHODS: This two-phase study recruited online research network members reporting MS and who were utilizing Fitbit One™ activity trackers. In the first phase, participant interviews assessed demand based on physical activity and the use of behavior-change techniques. The second phase assessed the demand, limited efficacy, acceptability, and practicality of a "Wearables 101" course that integrated behavior change and self-experimentation principles. Tracker data were used to determine the percent of matches between daily symptom-based step goals and step counts. RESULTS: Participants expressed demand in the form of interest in gaining insights about a possible "sweet spot" behavioral target, if a system could be produced to support that. Limited efficacy results were mixed, with approximately one-third of participants dropping out and only half matching their daily target goals for at least 50% of days. In terms of practicality, participants commented on the burden of daily measurement and the need for a longer baseline period. Participants noted that tracking helped support an understanding of the link between activities and symptom severity, suggesting acceptability. CONCLUSIONS: Results suggested that the intervention demand and acceptability criteria were demonstrated more strongly than limited efficacy and practicality. The matching intervention tested in this study will require refinement in baseline measurement, goal definition, and reduced data-gathering burden. Such changes may improve efficacy and practicality requirements and, by extension, later impact of the intervention on MS outcomes. Overall, these results provide justification for additional work on refining the intervention to increase practicality and efficacy.

Patient perspectives on the linkage of health data for research: Insights from an online patient community questionnaire.

OBJECTIVE: To examine the patient perspective on the risks and benefits of linking existing data sources for research. MATERIALS AND METHODS: Between December 2015 and February 2016, we fielded a questionnaire in PatientsLikeMe, an online patient community representing over 2500 health conditions. The questionnaire was developed using subject matter expertise and patient feedback from a concept elicitation phase (N = 57 patients). The final questionnaire consisted of 37 items. RESULTS: Of n = 5741 who opened the email invitation, n = 3516 respondents completed the questionnaire (61.2%). Of these, 73.8% were women, 86.4% were Caucasian, 14.5% were 65 or older, and 44.9% had completed college or post-graduate education. Questionnaire respondents indicated that the most important benefits of sharing data were "helping my doctor make better decisions about my health" (94%) and "helping make new therapies available faster" (94%). The most important data sharing risk identified was health data being "stolen by hackers" (87%). Of 693 patients who were not comfortable with researchers accessing their de-identified data, most reported that their comfort levels would increase if they were able to learn how their data was protected (84%). In general, responders felt more comfortable when unique identifiers such as social security number (90%) and insurance ID (82%) were removed from the data for linkage and research use. DISCUSSION: The majority of patients in a US-based online community are comfortable with researchers accessing their de-identified data for research purposes. CONCLUSIONS: Developing methods to link databases minimizing the exposure of unique identifiers may improve patient comfort levels with linking data for research purposes.

Attribute Development Using Continuous Stakeholder Engagement to Prioritize Treatment Decisions: A Framework for Patient-Centered Research.

OBJECTIVES: To develop a methodological approach for selecting, validating, and prioritizing attributes for health care decision making. METHODS: Participants (n = 48) were recruited from community support groups if they had a child aged 26 years or younger diagnosed with a coexisting mental health condition and cognitive impairment. Six in-depth interviews eliciting care management experiences were transcribed and coded into themes following the principles of grounded theory and the constant comparative method. Six focus groups involving 42 participants assessed the relevance, priority, and meaning and inter-relationship among the themes. The positive predictive value and sensitivity assessed agreement on thematic meaning. A final list was selected from the top priorities with good agreement as candidate attributes. Attribute levels reflecting the range of experiences in care management decisions emerged from the verbatim passages within each coded theme. RESULTS: Participants were the child's mother (73%), white (77%), married (69%), and on average 48 years old. The children were on average 14 years old; 44% had an intellectual disability, 25% had autism, and more than half had anxiety or attention-deficit/hyperactivity disorder. All 14 attributes identified from the in-depth interviews were deemed relevant. The positive predictive value exceeded 90%, and the sensitivity ranged from 64% to 89%. The final set of attributes formed the framework for care management decisions consisting of six attributes (medication, behavior, services, social, treatment effects, and school) each with three levels. CONCLUSIONS: A systematic approach grounded in qualitative methods produced a framework of relevant, important, and actionable attributes representing competing alternatives in clinical decisions.

Understanding preferences for type 2 diabetes mellitus self-management support through a patient-centered approach: a 2-phase mixed-methods study.

BACKGROUND: Patients with type 2 diabetes mellitus (T2DM) who participate in diabetes management programs have been shown to have better glycemic control and slower disease progression, although program participation remains low. In the USA, increasing participation in diabetes management support programs may also directly impact provider reimbursement, as payments are increasingly based on patient-centered measures. However, little is known about factors that may enhance patient participation. This study aimed at further understanding what is important in diabetes management support from the patients' perspective and at assessing the utilization of various types of diabetes-management programs. METHODS: A two-phase mixed-methods study was conducted of adult US members of PatientsLikeMe®, an online research network of patients. Phase 1 comprised qualitative interviews with 10 individuals to inform the online survey's contents, aided by literature review. During phase 2, this online survey was completed by 294 participants who reported on their diabetes goals and preferences for T2DM self-management support programs. RESULTS: The majority of the respondents were not participating in any program (65 %), but most had goals of improving diet (77 %), weight loss (71 %), and achieving stable blood glucose levels (71 %). Among those currently participating in programs, clinic, hospital-based, or other health-care professional programs were the most commonly used (51 %). The most preferred type of support was diet/weight-loss support (62 %), while doctors or nurses (61 %) and dietitians (55 %) were the most preferred sources of diabetes support. CONCLUSIONS: The low participation in diabetes self-management programs revealed in this study underscores the need for strategies to improve patient engagement. The results revealed support types and formats that patients with T2DM prefer and need. These findings may help improve patient engagement by guiding the future design of more effective diabetes management support programs.

An Innovative Approach to Informing Research: Gathering Perspectives on Diabetes Care Challenges From an Online Patient Community.

BACKGROUND: Funding agencies and researchers increasingly recognize the importance of patient stakeholder engagement in research. Despite calls for greater patient engagement, few studies have engaged a broad-based online community of patient stakeholders in the early stages of the research development process. OBJECTIVE: The objective of our study was to inform a research priority-setting agenda by using a Web-based survey to gather perceptions of important and difficult aspects of diabetes care from patient members of a social networking site-based community. METHODS: Invitations to participate in a Web-based survey were sent by email to members of the PatientsLikeMe online diabetes community. The survey asked both quantitative and qualitative questions addressing individuals' level of difficulty with diabetes care, provider communication, medication management, diet and exercise, and relationships with others. Qualitative responses were analyzed using content analysis. RESULTS: Of 6219 PatientsLikeMe members with diabetes who were sent survey invitations, 1044 (16.79%) opened the invitation and 320 (5.15% of 6219; 30.65% of 1044) completed the survey within 23 days. Of the 320 respondents, 33 (10.3%) reported having Type 1 diabetes; 107 (33.4%), Type 2 diabetes and taking insulin; and 180 (56.3%), Type 2 diabetes and taking oral agents or controlling their diabetes with lifestyle modifications. Compared to 2005-2010 National Health and Nutrition Examination Survey data for individuals with diabetes, our respondents were younger (mean age 55.8 years, SD 9.9 vs 59.4 years, SE 0.5); less likely to be male (111/320, 34.6% vs 48.4%); and less likely to be a racial or ethnic minority (40/312, 12.8% vs 37.5%). Of 29 potential challenges in diabetes care, 19 were categorized as difficult by 20% or more of respondents. Both quantitative and qualitative results indicated that top patient challenges were lifestyle concerns (diet, physical activity, weight, and stress) and interpersonal concerns (trying not to be a burden to others, getting support from family/friends). In our quantitative analysis, similar concerns were expressed across patient subgroups. CONCLUSIONS: Lifestyle and interpersonal factors were particularly challenging for our online sample of adults with Type 1 or Type 2 diabetes. Our study demonstrates the innovative use of social networking sites and online communities to gather rapid, meaningful, and relevant patient perspectives that can be used to inform the development of research agendas.

Development and content validity of a patient reported outcomes measure to assess symptoms of major depressive disorder.

BACKGROUND: Although many symptoms of Major Depressive Disorder (MDD) are assessed through patient-report, there are currently no patient-reported outcome (PRO) instruments that incorporate documented evidence of patient input in PRO instrument development. A review of existing PROs used in MDD suggested the need to conduct qualitative research with patients with MDD to better understand their experience of MDD and develop an evaluative instrument with content validity. The aim of this study was to develop a disease-specific questionnaire to assess symptoms important and relevant to adult MDD patients. METHODS: The questionnaire development involved qualitative interviews for concept elicitation, instrument development, and cognitive interviews to support content validity. For concept elicitation, ten MDD severity-specific focus group interviews with thirty-eight patients having clinician-confirmed diagnoses of MDD were conducted in January 2009. A semi-structured discussion guide was used to elicit patients' spontaneous descriptions of MDD symptoms. Verbatim transcripts of focus groups were coded and analyzed to develop a conceptual framework to describe MDD. A PRO instrument was developed by operationalizing concepts elicited in the conceptual framework. Cognitive interviews were carried out in patients (n = 20) to refine and test the content validity of the instrument in terms of item relevance and comprehension, instructions, recall period, and response categories. RESULTS: Concept elicitation focus groups identified thirty-five unique concepts falling into several domains: i) emotional, ii) cognitive, iii) motivation, iv) work, v) sleep, vi) appetite, vii) social, viii) activities of daily living, ix) tired/fatigue, x) body pain, and xi) suicidality. Concept saturation, the point at which no new relevant information emerges in later interviews, was achieved for each of the concepts. Based on the qualitative findings, the PRO instrument developed had 15 daily and 20 weekly items. The cognitive interviews confirmed that the instructions, item content, and response scales were understood by the patients. CONCLUSIONS: Rigorous qualitative research resulted in the development of a PRO measure for MDD with supported content validity. The MDD PRO can assist in understanding and assessing MDD symptoms from patients' perspectives as well as evaluating treatment benefit of new targeted therapies.

Hearing conservation program for agricultural students: short-term outcomes from a cluster-randomized trial with planned long-term follow-up.

OBJECTIVES: (1) To conduct a contemporary analysis of historical data on short-term efficacy of a 3-year hearing conservation program conducted from 1992 to 1996 in Wisconsin, USA, with 753 high school students actively involved in farm work; (2) to establish procedures for assessment of hearing loss for use in a recently funded follow-up of this same hearing conservation program cohort. METHODS: We analyzed a pragmatic cluster-randomized controlled trial, with schools as the unit of randomization. Thirty-four rural schools were recruited and randomized to intervention or control. The intervention included classroom instruction, distribution of hearing protection devices, direct mailings, noise level assessments, and yearly audiometric testing. The control group received the audiometric testing. RESULTS: Students exposed to the hearing conservation program reported more frequent use of hearing protection devices, but there was no evidence of reduced levels of noise-induced hearing loss (NIHL). CONCLUSION: Our analysis suggests that, since NIHL is cumulative, a 3-year study was likely not long enough to evaluate the efficacy of this intervention. While improvements in reported use of hearing protection devices were noted, the lasting impact of these behaviors is unknown and the finding merits corroboration by longer term objective hearing tests. A follow-up study of the cohort has recently been started.