Tailoring an Online Platform to Promote Evidence-Based Care in Children and Adults with Neurofibromatosis 1 to Address Medical and Social Barriers to Care
Abstract
Neurofibromatosis type 1 (NF1) is a complex multisystem disorder that predisposes people to multiple types of benign and malignant tumors, as well as cognitive, behavioral, bone, vascular, and other medical issues. Regular medical evaluations are critical to identify NF1-related problems as early as possible and offer appropriate treatment. Professional medical societies in the United States (U.S.) have issued guidelines about what routine medical evaluations are needed by NF1 patients, and we previously showed these guidelines are strongly supported by NF specialists. But, in the U.S., a majority of NF1 patients don’t go to specialty NF clinics for their care. Our preliminary research shows that these patients who don’t go to specialty NF clinics are significantly less likely to get recommended NF1-related medical evaluations than patients who do go to these clinics. We hypothesize that if we could help primary care providers (PCPs) perform routine NF1-related medical evaluations, we could significantly improve the quality of care for patients who don’t go to specialty NF clinics, and ultimately, improve their overall health and wellbeing. To achieve this goal, we propose to adapt an innovative online platform that has already successfully improved care for people with Down Syndrome (DS). The Down Syndrome Clinic to You platform consists of a website where parents/caregivers fill out questions about their loved one’s health. Based on the answers to those questions, the website automatically generates a list of personalized medical recommendations for the person with DS, such as whether the person is due for an eye exam, should be tested for sleep apnea, or needs another medical evaluation. These recommendations get automatically formatted into two letters – one written for parents/caregivers and one written for PCPs. Parents can then share the PCP letter with their loved one’s doctor at their next visit, so that the PCP knows what evaluations the person with DS needs and why. Our proposed study will adapt this online platform to suit the unique needs of people with NF1. We will test whether our new NF Guidelines to You platform can increase evidenced-based healthcare for people with NF1 by providing personalized care recommendations directly to NF1 patients/parents and their PCPs across the U.S. This project will have three stages. In the first stage, we will ask adults with NF1, parents of children with NF1, and PCPs who care for individuals with NF1 about their primary care visits and the problems they have seeking out or delivering NF1-related medical evaluations during these visits. In the second stage, we will create the NF Guidelines to You website based on recommendations from individuals in stage 1 and with the help of an advisory board of NF1 patients/parents, PCPs, and scientific/medical experts. We will also create tailored educational resources as a means of empowering individuals with NF1 to advocate for their medical care needs. These resources will be tested to be as widely accessible as possible, including for people who speak Spanish, have learning disabilities, or are young adults managing their care for the first time. In the third stage, we will test whether using NF Guidelines to You is helpful for adults with NF1, parents of children with NF1, and PCPs across the U.S. We will focus on whether the website is easy to use and if people find the letters and educational resources useful so that we can make any final improvements as needed. We will also preliminarily look at whether patients who use the platform receive more guideline-recommended NF1 care and are more satisfied with their care. At the end of this process, we will have optimized the NF Guidelines to You platform and will be ready to test if it is better than the current standard of care in a large clinical trial. This platform could help the roughly 63,000 people with NF1 who do not attend specialty NF cl
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310457
Entities
People
- Vanessa Merker
Organizations
- Massachusetts General Hospital
- United States Army