Trends in quality of care among children with sickle cell anemia

ImportanceYouths with sickle cell anemia (SCA) are at risk of pain crises, stroke, and early death. Complications can be reduced by the oral disease-modifying medication hydroxyurea, and in 2014, the National Heart, Lung, and Blood Institute published revised guidelines that hydroxyurea should be offered to youths aged 9 months and older with SCA regardless of disease severity.ObjectiveTo describe changes in hydroxyurea use among youths with SCA before and after release of the National Heart, Lung, and Blood Institute guidelines.Design, Setting, and ParticipantsThis cross-sectional study was conducted using administrative data from 2010 to 2018 from Michigan and New York State (NYS) Medicaid programs. The study population included youths aged 1 to 17 years with SCA enrolled in the Michigan or NYS Medicaid programs for at least 1 year (Michigan: 2010-2018; NYS: 2012-2018). Youths with SCA were identified using validated claims–based definitions. Data were analyzed from June to October 2020.Main Outcomes and MeasuresThe main outcome was hydroxyurea use characterized as mean annual counts of days’ supply of filled hydroxyurea prescriptions. Rates of hydroxyurea use over time were assessed using regression models (Michigan: zero-inflated negative binomial; NYS: negative binomial). Models included indicators for periods before and after guideline release.ResultsA total of 4302 youths with SCA (2236 males [52.0%]; 2676 born 2005-2017 [62.2%]; 150 Hispanic [3.5%], 2929 non-Hispanic Black [68.0%], and 389 non-Hispanic White [9.0%]) contributed 12 565 person-years. The mean (SD) annual days’ supply of hydroxyurea was 47.2 (93.6) days per youth in Michigan and 97.4 (137.0) days per youth in NYS. In Michigan, there was an increase in the odds of having nonzero days’ supply after the guidelines were released (odds ratio, 1.52; 95% CI, 1.07-2.14). In NYS, no change was seen in the mean days’ supply of filled hydroxyurea.Conclusions and RelevanceThese findings suggest that hydroxyurea was substantially underused among youths with SCA, despite establishment as the primary disease-modifying therapy for SCA, and that there was incomplete clinician or patient uptake of newly released guidelines. Results suggest that expanding use of hydroxyurea may require a multifaceted approach that includes addressing multiple system- and patient-level barriers.

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“…Hydroxyurea use was characterized as the count of the annual days' supply. 36 Annual days' supply was capped at 365 days.…”

Section: Outcome: Hydroxyurea Usementioning

confidence: 99%

Changes in Hydroxyurea Use Among Youths Enrolled in Medicaid With Sickle Cell Anemia After 2014 Revision of Clinical Guidelines

et al. 2023

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System's failure: Poor adherence to guidelines for children with sickle cell disease

Sampino

McGann

2021

Pediatric Blood & Cancer

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Pneumococcal infections in children with sickle cell disease before and after pneumococcal conjugate vaccines

Adamkiewicz,

Yee,

Thomas

et al. 2023

Blood Advances

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Children with sickle cell disease (SCD) are at increased risk of invasive pneumococcal infections (IPD). Over 25 years the Georgia Emerging Infections Program/CDC Active Bacterial Core Surveillance network identified 104 IPD episodes among 3707 children with hemoglobin SS (HbSS) or HbSC aged <10 years, representing 6% of IPD in Black or African American children residing in Metropolitan Atlanta (reference population). Children with IPD and HbSS/SC were older than those with IPD in the reference population (p<0.001). From 1994-1999 to 2010-2018, IPD declined by 87% in children with HbSS 0-4 years old, and by 80% in those aged 5-9. However, IPD incidence rate ratios comparing children with SCD with the reference population increased from 20.2 to 29.2 over these periods. Among children with HbSS and IPD, death declined from 14% to 3% after 2002, and meningitis declined from 16% to 8%. Penicillin resistance was more prevalent in children with SCD prior to PCV7 licensure. After 2010, all IPD serotypes were not included in the 13-valent pneumococcal conjugate vaccine (PCV13). Within 3 years of vaccination, effectiveness of the 23-valent pneumococcal polysaccharide vaccine (PPSV23) against non-PCV13 serotypes included in PPSV23 plus 15A/15C was 92% (95% confidence-interval: 40.8, 99.0, p=0.014; indirect-cohort effect adjusted for age and hydroxyurea). PPSV23 would cover 62% of non-PCV13 serotype IPD in children with SCD, while PCV15, PCV20 and PCV21/V116 (in development) could cover 16%, 51% and 92% respectively. Although less frequent, IPD remains a life-threatening risk in children with SCD. Effective vaccines with broader coverage could benefit these children.

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Trends in quality of care among children with sickle cell anemia

Cited by 5 publications

References 35 publications

Changes in Hydroxyurea Use Among Youths Enrolled in Medicaid With Sickle Cell Anemia After 2014 Revision of Clinical Guidelines

Changes in Hydroxyurea Use Among Youths Enrolled in Medicaid With Sickle Cell Anemia After 2014 Revision of Clinical Guidelines

System's failure: Poor adherence to guidelines for children with sickle cell disease

Pneumococcal infections in children with sickle cell disease before and after pneumococcal conjugate vaccines

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