A special report published in Pediatric blood and cancer offers a list of essential, optimal, and suggested components of pediatric sickle cell disease (SCD) therapy that may help optimize outcomes for these patients in the United States.
“Although SCD is the most common childhood genetic disease in the United States, SCD treatment, care infrastructure, and research have been underfunded compared to cystic fibrosis and hemophilia,” wrote the authors. “The funding disparity has produced limited treatments, few guidelines or standards of care, and inadequate reimbursement compared to other complex pediatric conditions.”
Early treatment of sickle cell disease, a rare inherited blood disorder, can significantly alter the course of the disease and minimize the lifelong symptom burden for patients. Preventative care with hydroxyurea, for example, can help minimize the irreversible organ damage that occurs over time in patients with SCD. Therefore, a collaborative multidisciplinary care team with expertise in SCD therapy is crucial to optimize outcomes.
While newborn screening in the United States identifies people with SCD, there is a lack of formal long-term follow-up of these cases. Varying aftercare processes among states affect access to care for patients with sickle cell disease, and there are currently no standardized guidelines for pediatric treatment of sickle cell disease. Progress in the development of specific therapies for SCD has also stagnated in recent decades, and there is currently no federal mechanism supporting comprehensive SCD centers in the United States.
The new report, authored by Hulbert et al., used a modified Delphi consensus-building process involving 19 pediatric SCD experts to develop similar guidelines for the pediatric SCD population. This effort is intended to inform future accreditation processes for comprehensive pediatric SCD centers. .
Team members deemed essential for a comprehensive pediatric SCD center include an expert physician in SCD, a pediatric hematology team, ambulatory nursing staff with SCD experience, a social worker, a care coordinator and a liaison officer with patient education. These team members would ensure that essential processes are carried out efficiently and in collaboration with the clinicians and programs involved.
Standardized processes for pediatric treatment of sickle cell disease are also needed, including follow-up for newborn screening and timely initiation of prophylactic treatment and preventive care. A defined transition process from childhood to adult care is also an essential aspect of maintaining access to hematology and improving patient outcomes. Patients with sickle cell disease are prone to episodes of acute pain, warranting additional management guidelines including emergency department protocol. Formal quality assessment and improvements, as well as a formal patient and family feedback process, are also essential aspects of a comprehensive care center.
The authors also proposed essential diagnostic tests and therapies. The prescription of hydroxyurea from 9-12 months is recommended to prevent acute chest syndrome, stroke, hospitalizations and the need for transfusions in children with hemoglobin (Hb) SS/S-β0 thalassemia. Screening with transcranial Doppler ultrasound, or screening for sickle cell disease, is also recommended in children with Hb SS/S-β0 thalassemia. Screening at the same SCD center or hospital could help reduce barriers to care, the authors wrote. Annual reviews of each patient’s pain management strategy are also essential to personalize treatment and achieve optimal results.
The optimal, but non-essential elements are specific to the SCD but should not necessarily be included in the guidelines. Optimally, care teams would include a pain management specialist, advanced practice providers to increase overall team capacity, dedicated inpatient nursing staff, and physical therapists. Partnering with community organizations is another optimal step for comprehensive centers that can expand support systems for sickle cell patients and their families.
A dedicated inpatient area, a day hospital or infusion center, and a dedicated outpatient clinic are all considered optimal physical spaces within a comprehensive clinic. Making clinical trials accessible to patients is another optimal aspect of comprehensive care that would promote access to new treatments. Patients treated at clinics with active trials in other diseases also showed better outcomes, even if they weren’t enrolled in the trials.
Suggested elements include a written business plan that includes funding sources, financial impact and sources of support; and additional members of the care team, including a clinical pharmacist, primary care physician, genetic counselor and nutritionist.
Identifying essential, optimal, and suggested elements is only the first step toward standardizing pediatric SCD care. Accredited comprehensive care centers such as those for conditions like cystic fibrosis and hemophilia are still needed to reduce disparities in treatment availability. It might even be possible to use existing pediatric hematology resources from other programs, the authors noted.
“Care disparities between SCD and other childhood illnesses have been driven by lack of federal funding and exacerbated by structural racism, and a standardized definition of comprehensive SCD care and center accreditation would be a step toward mitigating these disparities,” the authors concluded. These essential elements of care will also need to be updated as new management strategies and treatments are developed.
Hulbert ML, Manwani D, Meier ER, et al. Consensus definition of the essential, optimal and suggested components of a pediatric sickle cell centre. Pediatric blood cancer. Published online September 12, 2022. doi:10.1002/pbc.29961