Standardizing elements of care in pediatric sickle cell disease centers: The road toward health equity

In this issue of Pediatric Blood and Cancer, Hulbert et al. present a consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease (SCD) center in the United States,1 similarly to what has been done for adults in the same country by Kanter et al.2 The authors utilized a modified Delphi consensus-seeking process involving 19 SCD pediatric experts from large academic centers to set defined standards of SCD care within the United States. They propose essential (n = 26), optimal (n = 10), and suggested (n = 5) elements of a pediatric SCD center which are, respectively, defined as required for comprehensive care and evidence-based or guidelines-based, beneficial for comprehensive care but not required for guidelines-based care and likely to enhance treatment but not required for guidelines-based care. Among the essential elements, the consensus identified not only staff/teammembers with specific expertise (SCD physician expert, pediatric hematologist, outpatient nursing staff, case manager/care coordinator, social worker, education liason), but also detailed processes (such as procedures for newborn screening follow-up, protocols for emergency department care and guidelines for acute and chronic complications, qualitymanagement and improvement processes, formal process for patient/family input), SCD-specific diagnostic procedures, or therapeutic opportunities (on site transcranial Doppler, iron overload magnetic resonance imaging, hydroxyurea utilization, erythrocytapheresis for acute and chronic complications, and annual individualized pain plan) and collaborations with different experts or teams (partnership with hematopoietic stem cell transplantation, with adult SCD providers for transition, subspecialists, neuropsychologist, mental health specialist, etc.). Optimal elements included staff/teammembers, dedicated physical spaces, availability of clinical trial enrollment, and collaborationwith community-basedorganizations, while suggested elements were staff/teammembers (clinical pharmacist, primary care physician, genetic counselor, nutritionist) and a single process (written business plan). The authors conclude that the consensus and identification of the elements of care of a pediatric SCD center among experts is just the first step toward defining an accreditation process for comprehensive pediatric SCD centers in order to pursue federal funding and ensure quality of care and access to care for all children living with SCD. They aim at applying to SCD the model already in use with other rare and complex disorders of childhood such as cystic fibrosis and hemophilia for which a similar model has granted patients with those diseases, good quality care, and an improved survival with better quality of life.1 In fact, as the authors note, in spite of the scientific advancements and the isolated National Sickle Cell Disease Control Act, in the past 30 years the limited funding and investments for SCD have resulted in reducedaccess to comprehensive care, lackof improvementof theestimated median survival—which remained more or less the same in the past 20 years—and lack of development of implementation research. Overall, this has led to health disparities and limited achievements in health equity. Health equity is “the state in which everyone has the opportunity to attain full health potential and no one is disadvantaged from achieving this potential because of social position or any other socially defined circumstance.”3,4 In general, health equity is considered the principle, goal, or process thatmotivates or underpins efforts to eliminate health disparities.While there has been a general underachievement in reaching health equity in children overall and the knowledge of health equity and health disparities in the pediatric population is much more limited compared to adults,3,4 the lag in pediatric hematology, and especially in SCD, is even more striking. As the Strategic Plan and Blueprint for Action recently highlighted,5 while scientific advancements for the molecular understanding and pathophysiology have improved, the development of interventions to improve the quality of life for these individuals, aswell as the organization of health care systems to deliver appropriate care, has lagged resulting in comprehensive care being delivered unevenly across geographic areas, institutions, providers. In addition, there is substantial evidence that thosewith SCDmay receive poorer quality of care and therefore SCD continues to pose a high burden on affected individuals and their families.6