Lifespan care in SCD: Whom to transition, the patients or the health care system?
Authors: Caterina P. Minniti, Elliott Vichinsky
Journal: American Journal of Hematology (2017)

The process of becoming an adult for patients affected by congenital or chronic illnesses is a complex and difficult one. Societal and family expectations vary by country of origin and ethnicity. This compounds the difficulty in defining how, when and what is the right time to transition from pediatric to adult care, and how to go about it. In resource-rich countries one hundred years after the description of the first patient with sickle cell disease (SCD), a combination of government programs (e.g. the Sickle Cell Treatment Act), universal neonatal screening, antibiotic prophylaxis, improved supportive care, and successful research trials have resulted in the vast majority of children with SCD reaching adulthood.1-3 This remarkable accomplishment has de facto: (1) shifted the burden of care to adult providers and to the adult health care system and (2) changed the disease's paradigm from a pediatric illness, punctuated by acute exacerbations, to a chronic medical condition with progressive end-organ damage. Unfortunately, while mortality continues to decline in the pediatric age group, mortality rates for adults with SCD have not experienced the same levels of success.4 The medical community has recognized that specialized comprehensive care centers can decrease the morbidity and mortality associated with certain chronic diseases. For example, the benefits and requirements of specialized centers for hemophilia is promoted by the Centers for Disease Control (CDC), who reports there are approximately 20,000 patients with hemophilia in the United States—with 70% of pediatric and adult patients receiving care in the 134 multidisciplinary hemophilia treatment centers. Patients treated within these hemophilia centers have a lower mortality rate and a 40% decreased hospitalization rate for bleeding complications compared to those not treated within the centers (http://hemophilia.org). Similarly, the approximately 30,000 cystic fibrosis patients in the United States are recognized to require comprehensive care which is delivered within 120 accredited treatment centers and treatment affiliate sites (www.cff.org). Access to these services is credited in part for the significant improvement in survival of the cystic fibrosis population.5 Strong and active involvement of the respective patient communities — alongside the will of the medical community — was instrumental in the creation of such specialized centers. Unfortunately, the sickle cell community, their families and advocates have not been that successful to date. In the United States, young adult patients with SCD ages 18-25 are particularly vulnerable to medical complications and have higher rates of health care utilization (i.e., emergency room, inpatient admissions and re-admissions) and mortality than children < 18 years of age.3, 6 This is in contrast with recent data from Belgium that reported no such increase.3 Belgium, like other European nations that have reported better outcomes for their SCD patients,2 has a national health care system that offers the same level of access to care to patients regardless of the age and has a small and close hematologist network that shares common standards of care. SCD, which affects over 100,000 individuals in the United States and millions across the globe, largely does not have comprehensive treatment centers that can accommodate pediatric and adult patients. There are many possible explanations for why the health care system has been unwilling or unable to develop adult SCD treatment centers—including discrimination based on ethnicity, socioeconomic status, poor insurance status, and lack of effective advocacy from the sickle cell patient communities. In an effort to improve care for adult patients with SCD as an alternative to a national mandate for treatment centers, some have embraced the concept of a “medical home.” In this model, a primary care physician working with a support network assumes responsibility for the patient's global health needs. Equally important to this model is the development of pediatric-to-adult transition programs that prepare the young adult for a successful transition. Both a medical home and a transition program are worthy endeavors, but they are not a substitute for comprehensive care. Without access to comprehensive care, this model may actually increase patient morbidity.7, 8 There is little optimism among many patients and providers that this approach will provide the access to knowledgeable care needed by those with this complex, serious disease. Most surveys of primary care providers indicate they do not feel comfortable caring for adult patients with SCD, nor do they have the required resources or time to provide such care.9, 10 The primary care community is also under significant financial and practice stress and is severely challenged to assume primary care for our transitioning young adults. The American Society of Hematology (ASH) sponsored a State of Sickle Cell Disease report card, published in the fall of 2016 (www.scdcoalition.org). The report gave a 3.7 out of 10 score to “access to care,” citing factors such as health insurance status, availability of knowledgeable health care providers, provider experience, and geography. The same report gave a 3.2 out of 10 to the state of training and professional education, writing that “many family physicians felt they do not have adequate background in SCD management.” Furthermore, much of the care provided to adult patients is not in the primary care physician's office, but in the hospital by hospitalists11 and frequently in the emergency room. This phenomenon could also explain why patient utilization of hydroxyurea is still low in the United States despite an aggressive campaign from the hematology community and the National Institutes of Health (NIH). The transitioning of young adult patients to a health care system that does not have an integrated inpatient-outpatient environment is a serious problem. Studies suggest that even when patients are successfully transitioned to adult programs, they appear to have a marked increase in emergency room use, and their visits may be associated with significantly longer waiting times.6 Stehouwer analyzed the time to treatment in emergency rooms for patients with SCD in transition programs and compared the waiting time at both pediatric and adult sites; disappointingly, the time to treatment after transfer often doubled or tripled.12 There is likely a negative outcome in transitioning young adults from pediatric sickle cell treatment center to ill-equipped adult care models, resulting in frustrations and negative attitudes from patients, primary care physicians and emergency room departments. Unless major, comprehensive infrastructure changes occur within the primary care model, there will be increased emergency room visits, waiting times, healthcare costs and poor patient outcomes.13 A meaningful public health program should increase access to comprehensive care, including validated methods that decrease morbidity such as day hospitals with dedicated staff. These programs decrease hospitalization fivefold and improve outcomes.14, 15 Much has been written on the topic of transition. The American Academy of Pediatrics has released guidelines for transitioning to adult care for individuals needing complex care.16 Transition “passports” have been developed and a great deal of information is available for the physicians taking care of children and their families. The emphasis has been placed on education and empowerment of the patients—certainly an essential component of health care maintenance for any patient with a chronic illness. Indeed, patient readiness is a much utilized word when we talk about transition. While we cannot dispute the importance of educating young adults (with or without SCD) to be able to take care of themselves, navigate the complexities of the health care system, and take ownership of their lives, we have to recognize that much still has to be accomplished to develop an equally involved and well-equipped health care team on the receiving side of the transition — the “adult side.” We are asking our young patients to acknowledge, accept and manage their disease — a tall order for any teenager and for the population of patients with SCD specifically. The reality that settles in is that their disease often takes a turn for the worse in early adulthood, providers are often unfamiliar with the everyday and acute management of this disease, and end organ damage starts to manifest itself — demanding input from a vast array of subspecialists and increased need for coordination of care. Additionally, the fractionation of adult care with silos between emergency department, inpatient and outpatient care, the rise in hospitalist care, and the demise of the primary care attending following their patients in the hospital setting disrupts the providing of care in the moment when the patient needs a familiar face more than ever. It is not surprising that adults do not have similar outcomes as children with SCD. Moreover, fewer and fewer adult hematologists choose to care for a complex and often under-insured patient population. We witness that young patients with SCD often enter young adulthood with limited psychological, practical, emotional, and monetary support from their families. This is in stark contrast to the expectations in other serious chronic illnesses (including cancer) where families are expected to take an active role in helping patients navigate the complexity of the system and offer material and emotional support. While we need to promote adult self-management skills, family engagement improves adherence and quality of life.17 The current model of care relies on episodic, encounter focused, Relative Value Units (RVU)-driven, individual care with much of the responsibility for management of the myriad SCD related complications placed on the shoulders of the patient and little crosstalk between outpatient, inpatient and community. It is no surprise that providers and patients alike become overwhelmed by the care of these patients. What if we switch focus and concentrate our energies on developing a system of lifespan care, instead of looking for ways to transition patients from one group of providers to another? SCD pathophysiology does not change as patients grow older, but the effects of continuous end organ damage, ischemia-reperfusion injury, oxidative damage, and chronic pain are additive and often not visible until the third decade of life — exactly when patients transition out of pediatrics. Therefore when we think about transition, we may want to consider that it is not the patients who need to transition, but it is the health care system that needs to transition its model from an acute, episodic approach to taking care of disease symptoms, to a comprehensive and compassionate approach to the care of the patient in totality and for his/her lifespan. Furthermore, patients with SCD and the health care system have to work as a team as opposed to against each other. There is a clear need to expand the SCD “adult” work force and we could do so by developing a short, focused, post-graduate educational curriculum for physicians interested in “lifespan care” for SCD. Those willing to operate in the context of a comprehensive approach to care, with the support of “classic” adult providers such as internists, cardiologists, pulmonologists, etc. should be engaged, recruited and educated about how they could integrate into this model of care. Too often the patient with SCD receives no primary care, as he/she is seen only by the hematologist or the emergency room physician. In summary, comprehensive management of chronic, “lifespan” conditions such as SCD is challenging for patients, their providers, and health care systems. Current models of care are inadequate to solve this problem but much can be achieved by applying and perfecting other models of chronic illness care to SCD. In doing so, SCD providers are poised to become pioneers in “lifespan care,” creating a model of care that will be essential as many of our century's serious illnesses transition from acute, deadly problems (i.e. cancer, cardiovascular diseases, diabetes, opioids use) to chronic conditions in which prevention and lifestyle choices, patient engagement and comprehensive care become progressively more important in determining outcomes.