by Julie Kanter, M.D., Temeia D. Martin, M.D., W. Scott Russell, M.D., Greg A. Hall, M.D. and Kimberly McGhee
Illustration by Emma Vought
On completion of this article, readers should be able to:
- Recognize that young adults with sickle cell disease are optimally co-managed by a sickle cell specialist and primary care provider
- Describe why a seamless transition from pediatric to adult care is crucial
- Discuss the importance of early aggressive opioid therapy in managing pain associated with vaso-occlusive episodes
Sickle cell disease (SCD) was once considered a pediatric disease because patients rarely lived beyond childhood. Due to early diagnosis and improved medical management, many now live into adulthood. The health care system has struggled to serve this new adult SCD population, leading to an erosion of trust between patients and providers, but it is beginning to evolve to better meet their needs.
Young adults with SCD are particularly vulnerable to being lost to care as they transition from pediatric to adult providers. This is unfortunate because they are more likely to have chronic organ complications, including damage to the brain (stroke, silent infarcts), the heart (pulmonary hypertension), the lungs (fibrosis), the eyes (retinopathy), the bones (avascular necrosis) and the kidneys (renal failure).1
However, primary care physicians (PCPs) and hematologists willing and able to care for adult patients with SCD have been in short supply, causing affected adults to rely on emergency departments (EDs) for their care. According to an analysis of acute care utilization for patients with SCD, young adults aged 18 to 30 relied most heavily on EDs.2 A 2013 needs assessment in South Carolina found that more than 70 percent of patients aged 18 to 35 years seen in the ED or hospital returned within 30 days.3 Such episodic care is suboptimal and comes with a hefty price tag. In South Carolina, where the SCD burden is almost twice the national average (29.6 vs. 15.5 per 1,000 infants),4 the cost was estimated at $43 million in fiscal year 20105 and $100 million in fiscal year 2014.
Bettering the lives of young adults with SCD in South Carolina while controlling costs will require a multi-pronged approach that includes informed and compassionate care by ED staff, robust transition counseling by pediatricians, enhanced training of adult providers in evidence-based best practices for SCD and innovative health care delivery models that will enable access to sickle cell specialists and closer collaboration between specialists and PCPs in the provision of coordinated care.
Most adolescents and young adults with SCD arrive at the ED in search of pain relief from vaso-occlusive episodes (VOE), which are the hallmark of SCD. VOE occur when the sickled red blood cells, along with white blood cells and platelets, block the microcapillaries and prevent blood flow and oxygen from reaching tissues. These blockages cause intense pain but also injure the affected tissue and, over time, can cause organ damage and premature death.
Aggressive opioid management, ideally administered in accordance with an individualized pain plan, is strongly recommended for treatment of VOE-associated pain by the 2014 National Heart, Lung and Blood Institute (NHLBI)–sponsored guidelines for the management of SCD.6 However, the growing opioid epidemic has left ED staff wary of requests for narcotics, leading in some cases to those with SCD being mistaken for drug seekers.
“A review of the literature makes it very clear that those with sickle cell disease have a baseline level of pain that they live with that none of us can fully understand,” says W. Scott Russell, M.D., medical director of pediatric emergency medicine at MUSC Children’s Health.
The pediatric and adult emergency medicine teams have worked closely with hematologist Julie Kanter, M.D., who also serves as the director of sickle cell research, and her team to develop specific order sets and treatment protocols for these patients. “We train our staff to trust those protocols to ensure these patients receive timely pain relief,” adds Russell.
Communication between the ED and the patient’s PCP and SCD specialist will also be crucial to ensuring continuity of care for these patients. “The electronic medical record offers all providers the opportunity to communicate quickly so that young adults can continue the care they have been receiving up until age 18,” says Greg A. Hall, M.D., medical director of the adult ED.
Improving the transition from pediatric to adult care
For adolescents with SCD, the transition from pediatric to adult care can be both terrifying and confusing. They must leave behind a long relationship with a trusted physician to venture forward into what can seem the confusing maze of adult health care. To help allay their fears and prepare them for the change, pediatricians should begin discussing transition early with patients and their families, certainly by the time the child is 12 or 13, and continue throughout the adolescent years. Topics should include differences between pediatric and adult care systems, strategies for navigating and paying for adult care, self-management skills and the importance of self-advocacy. Tours of adult care facilities can remove fear of the unknown. Ideally, the pediatric hematologist should assist in locating an adult provider and should “co-manage” the affected adolescent initially to enable him or her to become more familiar and comfortable with adult care.
Adult primary care practices can act as a patient-centered medical home (PCMH) for young adults with SCD, providing them a personal and ongoing relationship with an adult PCP that can ease the anxiety accompanying the transition from pediatric care. A PCMH can closely monitor young adults and offer them primary and secondary preventive strategies to improve their overall outcomes, reduce their risk of complications and decrease their reliance on the ED. A PCMH can help coordinate care with subspecialists (including a hematologist), ED physicians and in-patient physicians to provide seamless care. For example, the PCMH for SCD at MUSC Health, directed by Temeia D. Martin, M.D., prescribes and monitors specialized disease therapy, including hydroxyurea (HU) and iron chelation therapy, and supervises long-term transfusion and anticoagulation programs in collaboration with pharmacy specialists. Additionally, it provides management for acute pain and other complications of disease, including access to intravenous narcotics, hydration and other treatments at a fraction of the cost of ED visits.
SCD-specific specialty clinics that treat all ages are another approach to ensuring seamless care for young adults with SCD. This type of clinic allows affected patients to remain in the same outpatient setting with continuity of care provided by nurses, social workers and case managers. One example is MUSC Health’s Lifespan Comprehensive Sickle Cell Center, which provides access to long-term transfusion therapy, intravenous pain management, hydration and disease-modifying therapy in a specialty environment. “This is one of the only clinics that treats patients with SCD from cradle to grave in one place in an effort to decrease loss to follow-up and care,” explains Kanter, who serves as its director.
Whether accomplished via improved transitional counseling, establishment of PCMHs or access to an SCD-specific clinic that accepts all ages, successful transition to adult care is key to better outcomes for young adults with SCD and for reducing their ED use.
Better educating adult providers
Transition programs can only be successful if there are adult providers willing and able to provide SCD care. Young adults with SCD who receive preventive care by a knowledgeable PCP in close collaboration with a sickle cell specialist are less likely to rely on the ED for their care. Indeed, a recent study showed that those who received such comprehensive care had half the number of ED visits and hospitalizations as those without it.8
Preventive care comprises immunizations, regular screening and HU treatment for eligible patients.6 Adult patients with SCD should receive a single dose of Haemophilus influenzae type b vaccine, two doses of the meningococcal ACWY vaccine 8 to 12 weeks apart, the 13-valent pneumococcal conjugate vaccine and the 23-valent pneumococcal polysaccharide vaccine (with revaccination five years later). They should also undergo an annual dilated eye examination to screen for retinopathy, an annual echocardiogram to screen for pulmonary hypertension, regular monitoring of bones for evidence of avascular necrosis and regular screening for iron overload.
The 2014 NHLBI guidelines recommend HU therapy in all adults with sickle cell anemia (hbSS or hbSB0).6 The only FDA-approved therapy that changes the clinical course of SCD, HU is a daily oral medication that has been shown to increase levels of fetal hemoglobin, reduce the frequency of VOE and improve survival.9,10 Currently, HU remains underutilized,5 in part because of concern for side effects and because some PCPs may be uncomfortable with their ability to prescribe and monitor it appropriately.
With proper monitoring and in collaboration with a sickle cell specialist, HU therapy can be safely implemented in primary care. Because HU can harm the developing fetus and can be passed through breast milk, it should not be prescribed in pregnant or lactating women, and both male and female patients receiving HU should practice contraception. HU should be discontinued at least 90 days prior to attempts to start a family. HU can cause a decrease in the number of blood cells in the bone marrow and so requires monitoring of blood cell counts, but no HU-related infections have been reported. Patients should be screened with full blood panels and renal and liver function tests before beginning therapy and monitored monthly for the first year for dosage titration. If absolute neutrophil counts drop below 2000/ L and platelet counts below 80,000/ L, HU therapy should be temporarily discontinued until levels return to normal and then resumed at a dose reduction of 5 mg/kg/d.6
New models of health care delivery
Co-management by an adult PCP and a sickle cell specialist is optimal for those with SCD, but specialists tend to be located in urban centers while many patients and their providers, especially in South Carolina, are located in rural regions. Project ECHO, developed at the University of New Mexico for hepatitis C, adopts a telementoring model for training more front-line providers in specialty care. Following the ECHO model, Kanter’s Lifespan clinic serves as the hub of the (SC)2 network (sc2.org), which extends across the state. Each of the spokes is initiating a sickle cell center with a PCP trained in SCD care as well as an infusion center to work with Kanter via telemedicine to provide disease-specific management for affected adults. This innovative health delivery system promises to make evidence-based, coordinated care for SCD a reality in the state by overcoming issues of access and training a new generation of providers.
1 Kanter J, Kruse-Jarres R. Blood Rev 2013 Nov;27(6):279-87.
2 Brousseau DC, et al. JAMA 2010 Apr 7; 303(13):1288-1294.
3 Schlenz AM, et al. Public Health Rep 2016 Jan-Feb;131(1):108-116.
4 Ojodu J, et al. MMWR December 12, 2014: 63(49):1155-1158.
5 Lòpez-De Fede A., et al. Sickle cell disease and SC Medicaid recipients: SFY 2010 factsheet. Columbia, SC: University of South Carolina. 2012.
6 Yawn BP, et al. JAMA 2014;312(10):1033-1048.
7 DeBaun MR and Telfair J. Pediatrics 2012;130:926-935.
8 Wolfson JA, et al. Pediatr Blood Cancer 2012;58:66-73.
9 Charache S, et al, N Engl J Med 1995 May 18;332(20):1317-1322.
10 Le PQ, et al. Pediatr Blood Cancer 2015; 62: 1956-1961.