Year: 2024
Type: editorial
Abstract: Recent developments suggest that we have finally entered an era of optimism about the outcomes of children in the United States living with sickle cell anemia (SCA).In the last decade, 4 disease-modifying medications have been approved, in contrast to none in the previous century.1-4 Children are living longer, including some into their eighth decade.5,6 Gene therapy and improvements in donor-based stem cell transplantation allow children living with SCA and their families to envision the possibility of living without it.7-10 However, in this issue of Pediatrics, data are presented that dampen this optimism and justify the need for thoughtful reflection; we are, in fact, still failing children with SCA.Using data from the Sickle Cell Disease Collection Programs in California and Georgia from 2010 to 2019, the authors of this study assessed the receipt of 2 nationally endorsed quality measures for children with SCA: antibiotic prophylaxis and annual stroke risk screening.Notably, these measures must be delivered to all children with SCA to prevent severe and life-threatening complications (eg, sepsis and stroke) and are driven by data from high-quality, randomized clinical trials published decades ago.11,12 Unlike many studies that rely on diagnostic claims codes to identify children with SCA, the authors of this study used a laboratory diagnosis of SCA (defined as Hemoglobin SS or Hemoglobin Sb 0 thalassemia genotypes).Identified children were linked to Medicaid eligibility and claims data to determine the proportion of children with SCA aged 3 months to 4 years who were dispensed $300 days of prophylactic antibiotics each year and the proportion of children aged 2 to 15 years who received a transcranial Doppler ultrasound (TCD) each year.Dismally, <1 in 4 children received enough antibiotics to cover the majority of the year, and only one-half received a TCD screen.This analysis is strong in its method of identifying the population of interest (eg, those with SCA), rather than the broader sickle cell disease population that includes additional genotypes (ie, Hemoglobin SC) that are not included in the quality metrics.However, the conclusion that younger age, living in a rural setting, and hematology prescribing are associated with increased receipt of services should be interpreted with caution because of confounding or masking of effects by unadjusted variables.Despite this limitation, the findings are consistent with multiple other studies evaluating these measures in the last 20 years: the receipt of antibiotic prophylaxis and TCD screening among children with SCA remains abysmally low and unchanged.13-16 Given these large and persistent gaps in care, actions for meaningful change must be taken.Several quality improvement initiatives have been associated with improved receipt of these important services.17-19 However, these initiatives are focused on increasing preventive services for children engaged in care at comprehensive sickle cell clinics.Previous work within the Sickle Cell Data Collection program revealed that 24% (California) and 13% (Georgia) of children with sickle cell disease did not have any encounters with a hematologist in a 3-year period.20 Therefore, quality initiatives clearly need to be expanded beyond
Source: PEDIATRICS
Authors: Susan Creary, Sarah Reeves
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: bronze
OpenAlex ID: W4392516385