Sickle cell disease (SCD) is a serious condition that requires coordinated treatment from experts well-versed in the disease. As it affects an estimated 100,000 Americans, there’s a paucity of providers who are trained to provide preventive therapies and interventions and to manage the complications that arise, including in children.
Pediatric-trained hematologists typically practice only at academic medical centers, which are few and far between across much of New England. And the knowledge base in the pediatric primary care or family medicine setting is often limited to the few patients these providers cared for while in medical school or residency training. This limited access problem is compounded by the social determinants of health for many children with sickle cell disease, who tend to come from communities that face higher rates of poverty and whose families may have cultural differences or health literacy issues that limit their capacity to navigate or communicate with their healthcare providers.
Decreasing as many barriers as possible is crucial for delivering the best care to these children. As part of the efforts to improve access and outcomes, clinicians and investigators have joined a system of telementoring called Project ECHO, which brings expert input to community-based healthcare providers to empower clinicians to bring expert care to all these families.
The access problem with sickle cell disease
Youth with SCD benefit the most from extensive collaboration between hematology, primary care providers, and other subspecialists — which may involve doctors in cardiology, nephrology, endocrine, pulmonology, neurology, ophthalmology, and developmental and behavioral pediatrics. A pediatric hematologist typically leads the charge, coordinating with a child’s medical home and family in terms of setting treatment goals, providing medications, and making referrals to prevent or diagnose complications of sickle cell disease.
For families of children with SCD, keeping up with so many appointments can be a significant burden on their resources. They may have to travel long distances or navigate multiple forms of public transportation to reach their specialists. This can lead to missed appointments or settling for care with less-experienced clinicians, both of which can diminish the impact of preventive or supportive treatments and could lead to more ER visits, hospitalizations, disability, and even death.
Crowdsourcing expertise with Project ECHO
Project ECHO was originally developed by Sanjeev Arora, MD to leverage the concentration of specialists at the University of New Mexico Medical Center to the far-flung population of patients across the state who struggled with hepatitis. The program model was an immediate success, and today, many other ECHO programs exist across the country to address diverse mental health and medical issues.
The Pediatric Sickle Cell ECHO follows the hub-and-spoke model to bring together teams of SCD consultants who offer interactive sessions to physicians, nurses, nurse practitioners, social workers, and other healthcare professionals, who then help manage their patients’ condition locally.
The barriers to visiting a specialist can lead to missed appointments or settling for care with less-experienced clinicians, both of which could lead to more ER visits, hospitalizations, disability, and mortality.
The online sessions follow a standardized format of presenting case-based discussion through video conferencing. A clinician presents a clinical conundrum in the outpatient setting, emergency department, or inpatient setting and frames clinical questions for the content experts and other providers to discuss, debate, and comment. In the case of sickle cell disease, the case discussions may revolve around issues such as pain management, medication compliance, or the risks and benefits of chronic transfusion therapy. At the end of each session, a short didactic presentation grounds the discussion in the latest literature.
Many methods used to manage sickle cell disease are based on experience and expert opinion rather than large clinical trials because it's a rare disease. ECHO's digital platform allows providers to manage patients with SCD better because of the rich discussion provided by specialists.
Moving in the right direction to manage sickle cell
The best approach to managing sickle cell disease involves a multidisciplinary, team-based approach that engages a patient and their family in formulating best practices and goals. Project ECHO is one way to bring that team closer using available technology.
The Pediatric Sickle Cell ECHO project as an important step toward a longer-term goal. While our research colleagues work apace on providing improved medications to prevent complications and find a universal cure for this condition, we will work to connect our peers across New England with experts dedicated to helping them care for these patients.