Funding Source
Patient Centered Outcome Research Institute (PCORI)

Project Period: 08/01/2025 – 07/31/2031

Team Members
Patient and caregiver partners: Katrina Byrd, David Garland, Deborah Garland, Raziel Green, William O’Sullivan, Linda O’Sullivan, Sridhar Tummala

Site PIs: Mark Garret, MD (Dartmouth Health, Lebanon NH), Stephen Johnson, MD (Mayo Clinic, Scottsdale AZ); Björn Oskarsson, MD (Mayo Clinic, Jacksonville FL); Zachary Simmons, MD (Penn State Health Milton S. Hershey Medical Center, Hershey PA); Tawfiq Al-Lahham, MD ( University of Pittsburgh Medical Center, Pittsburgh PA); Kelly Gwathmey, MD (Virginia Commonwealth University, Richmond VA); Colin Kreple, MD (University of Wisconsin School of Medicine and Public Health, Madison WI); Reed Bratches, PhD (University of Alabama at Birmingham, Birmingham AL).

Co-Is: James O’Malley, PhD (Geisel School of Medicine at Dartmouth, Lebanon NH); Renata Yen, PhD, CTBH Geisel School of Medicine at Dartmouth, Lebanon NH); Alejandra Martinez, PhD (CTBH, Geisel School of Medicine at Dartmouth, Lebanon NH); Elizabeth Carpenter-Song, PhD (Dept. of Anthropology, Dartmouth College, Hanover NH); Elijah Stommel, MD (Dartmouth Health, Lebanon NH); Timothy Burdick, MD (Dartmouth Health, Lebanon NH); David Lacomis, MD (University of Pittsburgh Medical Center, Pittsburgh PA).

BACKGROUND AND SIGNIFICANCE

Amyotrophic lateral sclerosis (ALS) is a fatal, rare neurodegenerative disease affecting 30,000 people in the United States. People with ALS (pwALS) and their caregivers, attend 3 – 5 hour long multidisciplinary clinics (MDC) every 3 months where they meet with up to 12 specialists. The goal of treatment is to maintain pwALS quality of life (QOL). Optimal information from MDCs is necessary for the intensive at-home management which gets more complex as ALS progresses. Yet 40% of recommendations are forgotten by pwALS negatively impacting QOL, while caregivers report information deficits increasing caregiving burden. Patient portal access to MDC notes is standard, and can improve recall and health outcomes, yet concerns exist about their layout, comprehensiveness and challenges for people with low health literacy. An underutilized strategy to share MDC information is video recording. Systematic reviews find recordings improve health outcomes, and the use of video in an ALS context may be particularly helpful given complicated at-home management. We plan to discover what is the most effective approach to communicating MDC information to facilitate optimal delivery of care for pwALS and their caregivers

STUDY AIMS

Aim 1. Assess the comparative effectiveness of written MDC visit information provided to pwALS and their caregivers via the patient portal (NOTES) versus NOTES plus visit video recording (VIDEO) on patient and caregiver quality of life.

Aim 2. Determine whether patient and caregiver health literacy and rapid ALS disease progression are modifying factors on the comparative effectiveness of NOTES versus VIDEO on primary outcomes.

Aim 3. Identify system and patient-level factors that impact the implementation and use of multimodal strategies of MDC information exchange by a diverse sample of pwALS, caregivers, clinicians and health system leaders.

Hypothesis (Main Effect): Compared to those receiving NOTES, pwALS randomized to also receive routine VIDEO recordings of their MDC visit will report better QOL via improved care-management (i.e., treatment adherence), self-management ability, satisfaction with communication and reduced healthcare utilization at 12 months; there will be larger relative benefits for pwALS, and caregivers, with low health literacy and rapid disease progression. We also hypothesize that compared to NOTES alone, caregivers in the VIDEO arm will report better QOL, improved preparedness and less burden at 12 months

STUDY DESCRIPTION

Overall study design

We will conduct a multisite, two-arm, parallel group, patient-randomized, controlled, comparative effectiveness trial with 12-month follow-up. We will adopt a Hybrid Type 1 design, and examine implementation barriers and facilitators using interviews guided by the Consolidated Framework for Implementation Research Framework 2.0.

Comparators

Comparator 1 (NOTES): After the initial MDC we will provide a 15-minute training to orient participants to the portal and how to access visit notes.

Comparator 2 (AUDIO): All MDC visits will be video-recorded over 12 months, using HealthPAL, an NIH-funded HIPAA-compliant personal health library developed by PI Barr. Participants in the VIDEO arm will receive orientation to the patient portal and HealthPAL.

Following each scheduled MDC, pwALS and caregivers in both groups will receive emails reminding them to watch the visit video recording and/or review their note: 1) within 48 hours of the visit to remind them of the visit discussion and self-management tasks they may have; and 2) 3 days before their next MDC visit to prepare for their next MDC visit.

Study Population

We will recruit 400 dyads (400 pwALS and their 400 caregivers) from eight MDC sites (∼50 dyads per site): Dartmouth Health (Lebanon, NH), Massachusetts General Hospital (Boston, MA), Mayo Clinics (Scottsdale, AZ, and Jacksonville, FL), Penn State Health (Hershey, PA), University of Pittsburgh Medical Center (Pittsburgh, PA), University of Wisconsin (Madison, WI) and Virginia Commonwealth University (Richmond, VA). We will include pwALS, age ≥18 years, who plan to attend MDC visits for 12-months, speak English or Spanish, have ≥12 months projected survival, and internet access and their primary caregivers (family member or friend), ≥18 years who attends MDC visits or assist with their care.

Outcomes

Primary outcomes: QOL is the primary outcome for both patients (ALSAQ-40) and for caregivers (PROMIS Global 10); Secondary outcomes: exercise adherence, medication adherence, adherence to MDC recommendations, self-management ability, patient satisfaction with communication, caregiver visit engagement, preparedness for caregiving, and caregiver burden. Exploratory outcome: healthcare utilization.

Timeframe

Twelve-month follow-up for all outcomes.

Stakeholders: Katherine Burke, PT, DPT (Massachusetts General Hospital, Boston MA); Chelsey Carter, PhD (Yale School of Public Health, New Haven CT); Thomas Cochrane, MD, MBA (Harvard Medical School Center for Bioethics, Boston MA); Jason Cohen, JD (Hart Wagner LLP, Portland OR); Jeffrey Cohen, MD (Dartmouth Health, Lebanon NH); Raman Kaur, MSN, AGNP-C (Health Insurance); Carrie Reichwein, MS, CCC-SLP (Penn State Health Milton S. Hershey Medical Center, Hershey PA); Joshua Sharfstein, MD (Johns Hopkins Bloomberg School of Public Health, Baltimore MD); Stephen Spaulding, MA (ALS United Mid-Atlantic, Philadelphia PA); Amber Stalker, MEd (ALS Adovcate)