Sophia Jan

BACKGROUND Long-term care (LTC) planning is challenging for individuals with intellectual and/or developmental disabilities (IDD) and their caregivers. It also lacks a concrete framework. The Preparation for Future Care (PFC) model has been used to describe advanced care planning for older adults and may apply to individuals with IDD. OBJECTIVE This study uses the PFC model to understand LTC planning for this population and applies this understanding to development of an LTC digital planning tool to support family caregivers. METHODS We conducted semi-structured focus groups in five states with family caregivers of individuals with IDD regarding LTC planning. We applied the Framework Method analysis using PFC as potential nodes. Informed by focus groups and a pre-existing care planning website for seniors, we developed a planning tool to support LTC planning for this population. We then conducted a pilot study to assess the feasibility and acceptability of the digital planning tool. RESULTS A total of 61 family caregivers participated in focus groups. Analysis revealed that LTC planning progressed in accordance with PFC. Four sub-nodes (advocate, finances, housing, and medical management) were identified, which served as a foundation for website development. A pilot study (n=4) was conducted to assess feasibility. Average total usability (measured by the SUS) was high (M=80.6, SD=16.7, range 57.5-97.5) indicating that participants found the tool easy to use. CONCLUSIONS The PFC model was found to be an appropriate framework for understanding LTC planning and intervention development. The framework was further used to build the Map Our Life website. Once established a formative usability evaluation deemed the digital planning tool to be feasible and acceptable by caregivers of individuals with IDD.

Importance: Young adults with sickle cell disease (SCD) experience challenges transitioning from pediatric to adult care, leading to increased morbidity and mortality. Objective: To evaluate the effectiveness of community health worker (CHW) support or a mobile health application (mHealth) compared with enhanced usual care (EUC) in improving health-related quality of life (HRQOL) for young adults with SCD transitioning to adult care. Design, Setting, and Participants: The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care study was an observer-blinded, multicenter, randomized clinical trial performed at 5 US children’s hospitals with a recruitment period from January 15, 2019, to December 31, 2022, and data analysis performed from September 30, 2024, to June 30, 2025. Participants were 17 years or older with SCD. Interventions: Participants were randomized 1:1:1 to 6 months of EUC, CHW plus EUC, or mHealth plus EUC. Both interventions included goal setting, self-management, skill development, symptom tracking, and transition support. The CHW plus EUC intervention provided weekly synchronous support primarily via phone calls, while mHealth plus EUC offered virtual peer support via interaction with discussion boards. The EUC group received standard care consisting of a transition checklist for consistency across sites. Main Outcomes and Measures: The main outcome was HRQOL, assessed using the Pediatric Quality of Life Inventory (PedsQL) for SCD module. Clinically meaningful improvement was prespecified as a 10-point change. Secondary outcomes included SCD knowledge, transition readiness, and social support. All outcomes were collected at baseline and follow-ups at 6, 12, and 18 months. Results: Of the 700 eligible patients across the 5 sites, 405 were enrolled, and 375 participants with SCD were randomized, 191 (51.5%) of whom were women. The mean (SD) age was 18.9 (1.9) years; the median age was 18.0 (IQR, 17-20) years. Baseline demographic data, clinical characteristics, and markers of disease severity were comparable across the study groups. At 6 months, the CHW plus EUC group showed modest improvements in HRQOL compared with the EUC group at 2.67 (95% CI, 0.25-5.09) at 6 months; there was no change for the mHealth plus EUC group at 0.73 (95% CI, −1.48 to 2.93) at 6 months; and the EUC group had a decline of 2.58 (95% CI, −4.67 to −0.49). CHW support demonstrated the greatest improvement in PedsQL scores compared with EUC at 6 (5.25 [95% CI, 2.05-8.45] points), 12 (5.56 [95% CI, 1.52-9.61] points), and 18 (6.14 [95% CI, 1.75-10.54] points) months. The mHealth plus EUC intervention demonstrated improvement in PedsQL scores at 6 months only (3.31 [95% CI, 0.27-6.35] points). Throughout the study, the HRQOL for the EUC group declined. No significant differences were found in secondary outcomes. Conclusions and Relevance: Although neither intervention met the prespecified 10-point threshold for a large clinical effect, the CHW intervention produced a significant and durable improvement in HRQOL that halted the decline observed in EUC. This sustained effect during the 18 months of follow-up suggests that CHW support provides a clinically relevant benefit for young adults with SCD during their transition to adult care. Integrating such programs into routine care could improve outcomes for this vulnerable population. Trial Registration: ClinicalTrials.gov Identifier: NCT03648710

Background: Nearly three-quarters of the estimated 4.9 million people with intellectual and developmental disabilities (IDDs) in the United States live with family caregivers, 25% of whom are aged older than 60 years. People with IDDs experience disproportionately high rates of mortality, morbidity, and acute care use, often due to a lack of accessible, tailored resources to support them throughout the lifespan. Few caregivers engage in long-term care (LTC) planning, often due to a lack of information and support. Objective: This protocol describes the design and methods of the Future Planning and Well-Being for Individuals with Intellectual Disabilities and Family Caregivers study, a randomized controlled trial evaluating the effectiveness of the Map Our Life web-based LTC planning tool compared to an attention control website on caregiver-reported burden, caregiver-reported well-being, and progress in and communication of future plans for the individual with an IDD. Methods: The Future Planning and Well-Being for Individuals with Intellectual Disabilities and Family Caregivers study is a national, multisite, randomized controlled clinical trial with a target enrollment of 1050 family caregivers of individuals with IDDs at 6 sites across the United States. Participants are randomly assigned (1:1) to either the Map Our Life web-based LTC planning tool plus enhanced usual care or an attention control group consisting of a Centers for Disease Control and Prevention–sponsored health promotion website for people with disabilities plus enhanced usual care. Primary outcomes, including caregiver-reported burden, caregiver-reported well-being, and LTC planning behaviors, are assessed at baseline and at 1, 6, and 18 months. Data will be analyzed using mixed-effects models to accommodate the repeated measures design. Results: This study was funded in July 2022, received initial Institutional Review Board approval in August 2023, and was registered on ClinicalTrials.gov in December 2023. Recruitment began in December 2023 and is scheduled to conclude in December 2025. Primary outcome analyses will commence immediately following the completion of final follow-up surveys. Conclusions: LTC planning is an important component of addressing health disparities among individuals with disabilities and their family caregivers. By focusing on using an innovative and accessible tool for LTC planning, the study addresses a critical gap in available resources with the potential to improve quality of life and reduce caregiver burden. Trial Registration: ClinicalTrials.gov NCT06065527; https://clinicaltrials.gov/study/NCT06065527 International Registered Report Identifier (IRRID): DERR1-10.2196/77184

Background: Transitioning from pediatric to adult sickle cell disease (SCD) care is challenging for emerging adults (aged 17-25 years). This period is marked by a 7-fold increase in mortality rates and has the highest rates of hospitalizations, emergency room visits, and hospital readmissions compared with children living with SCD. These challenges are exacerbated by fragmented care coordination, difficulty navigating adult health care systems, and increased self-management responsibilities. Objective: This study aims to compare the effectiveness of 2 interventions designed to support emerging adults living with SCD during this transition: a mobile health (mHealth) app and community health worker (CHW) support to standard care. Methods: The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care (COMETS) trial is an ongoing multicenter, 3-arm, open-label randomized controlled trial; 375 emerging adults (aged 17-25 years) are being enrolled and randomized 1:1:1 to (1) a 6-month CHW intervention focused on self-management skills, symptom tracking, care coordination, and transition planning; (2) a 6-month mHealth self-management program (enhanced iManage application) with tailored SMS text messaging (THRIVE [Texting Health-Related Resources to Inform, Motivate, and Engage] 2.0); or (3) enhanced usual care (control). Participants are followed for 18 months. The primary outcome is the change in self-reported health-related quality of life assessed using the PedsQL SCD module. Secondary outcomes include acute care use (hospitalizations and emergency department visits), patient activation, self-management behavior, and successful transfer to adult hematology care. Results: The institutional review board at Children’s Hospital of Philadelphia approved this study in June 2018. Recruitment began in January 2019 and ended in December 2022; we completed data collection in November 2024. We have enrolled a total of 405 participants. Conclusions: This trial addresses a critical gap in transition intervention research for young adults with SCD. It will provide evidence on the comparative effectiveness of 2 promising interventions (CHW and mHealth) and inform the development of scalable and sustainable transition support programs. Findings will have implications for improving health-related quality of life, reducing acute care use, and promoting successful transition to adult-centered SCD care for this vulnerable population. Trial Registration: ClinicalTrials.gov NCT03648710; https://clinicaltrials.gov/study/NCT03648710 International Registered Report Identifier (IRRID): DERR1-10.2196/69239

Journal of Medical Internet Research - International Scientific Journal for Medical Research, Information and Communication on the Internet #Preprint #PeerReviewMe: Warning: This is a unreviewed preprint. Readers are warned that the document has not been peer-reviewed by expert/patient reviewers or an academic editor, may contain misleading claims, and is likely to undergo changes before final publication, if accepted, or may have been rejected/withdrawn. Readers with interest and expertise are encouraged to sign up as peer-reviewer, if the paper is within an open peer-review period. Please cite this preprint only for review purposes or for grant applications and CVs (if you are the author). Background: Nearly three-quarters of the estimated 4.9 million people with intellectual and developmental disabilities (IDDs) in the United States live with family caregivers, 25% of whom are aged older than 60 years. People with IDDs experience disproportionately high rates of mortality, morbidity, and acute care use, often due to a lack of accessible, tailored resources to support them throughout the lifespan. Few caregivers engage in long-term care (LTC) planning, often due to a lack of information and support. Objective: This protocol describes the design and methods of the Future Planning and Well-Being for Individuals with Intellectual Disabilities and Family Caregivers study, a randomized controlled trial evaluating the effectiveness of the Map Our Life web-based LTC planning tool compared to an attention control website on caregiver-reported burden, caregiver-reported well-being, and progress in and communication of future plans for the individual with an IDD. Methods: The Future Planning and Well-Being for Individuals with Intellectual Disabilities and Family Caregivers study is a national, multisite, randomized controlled clinical trial with a target enrollment of 1050 family caregivers of individuals with IDDs at 6 sites across the United States. Participants are randomly assigned (1:1) to either the Map Our Life web-based LTC planning tool plus enhanced usual care or an attention control group consisting of a Centers for Disease Control and Prevention–sponsored health promotion website for people with disabilities plus enhanced usual care. Primary outcomes, including caregiver-reported burden, caregiver-reported well-being, and LTC planning behaviors, are assessed at baseline and at 1, 6, and 18 months. Data will be analyzed using mixed-effects models to accommodate the repeated measures design. Results: This study was funded in July 2022, received initial Institutional Review Board approval in August 2023, and was registered on ClinicalTrials.gov in December 2023. Recruitment began in December 2023 and is scheduled to conclude in December 2025. Primary outcome analyses will commence immediately following the completion of final follow-up surveys. Conclusions: LTC planning is an important component of addressing health disparities among individuals with disabilities and their family caregivers. By focusing on using an innovative and accessible tool for LTC planning, the study addresses a critical gap in available resources with the potential to improve quality of life and reduce caregiver burden. Trial Registration: ClinicalTrials.gov NCT06065527; https://clinicaltrials.gov/study/NCT06065527

BACKGROUND Transitioning from pediatric to adult sickle cell disease (SCD) care is challenging for emerging adults (aged 17-25 years). This period is marked by a 7-fold increase in mortality rates and has the highest rates of hospitalizations, emergency room visits, and hospital readmissions compared with children living with SCD. These challenges are exacerbated by fragmented care coordination, difficulty navigating adult health care systems, and increased self-management responsibilities. OBJECTIVE This study aims to compare the effectiveness of 2 interventions designed to support emerging adults living with SCD during this transition: a mobile health (mHealth) app and community health worker (CHW) support to standard care. METHODS The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care (COMETS) trial is an ongoing multicenter, 3-arm, open-label randomized controlled trial; 375 emerging adults (aged 17-25 years) are being enrolled and randomized 1:1:1 to (1) a 6-month CHW intervention focused on self-management skills, symptom tracking, care coordination, and transition planning; (2) a 6-month mHealth self-management program (enhanced iManage application) with tailored SMS text messaging (THRIVE [Texting Health-Related Resources to Inform, Motivate, and Engage] 2.0); or (3) enhanced usual care (control). Participants are followed for 18 months. The primary outcome is the change in self-reported health-related quality of life assessed using the PedsQL SCD module. Secondary outcomes include acute care use (hospitalizations and emergency department visits), patient activation, self-management behavior, and successful transfer to adult hematology care. RESULTS The institutional review board at Children’s Hospital of Philadelphia approved this study in June 2018. Recruitment began in January 2019 and ended in December 2022; we completed data collection in November 2024. We have enrolled a total of 405 participants. CONCLUSIONS This trial addresses a critical gap in transition intervention research for young adults with SCD. It will provide evidence on the comparative effectiveness of 2 promising interventions (CHW and mHealth) and inform the development of scalable and sustainable transition support programs. Findings will have implications for improving health-related quality of life, reducing acute care use, and promoting successful transition to adult-centered SCD care for this vulnerable population. CLINICALTRIAL ClinicalTrials.gov NCT03648710; https://clinicaltrials.gov/study/NCT03648710 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/69239