Patricia L. Kavanagh

Sickle Cell Disease–Related Pediatric Medical Expenditures in the U.S.

BACKGROUND Although it is known that people with sickle cell disease (SCD) have relatively high utilization of medical care, most previous estimates of SCD-attributable expenditures have been limited to either inpatient care or single-state data. PURPOSE To extend known findings by measuring the attributable or incremental expenditures per child with SCD compared to children without this illness and to thereby estimate SCD-attributable expenditures among children in the U.S. METHODS MarketScan Medicaid and Commercial Claims databases for 2005 were used to estimate total medical expenditures of children with and without SCD. Expenditures attributable to SCD were calculated as the difference in age-adjusted mean expenditures during 2005 for children with SCD relative to children without SCD in the two databases. RESULTS Children with SCD incurred medical expenditures that were

Quality-of-Care Indicators for Children With Sickle Cell Disease

9369 and

Health care utilization and expenditures for privately and publicly insured children with sickle cell disease in the United States.

13,469 higher than those of children without SCD enrolled in Medicaid and private insurance, respectively. In other words, expenditures of children with SCD were 6 and 11 times those of children without SCD enrolled in Medicaid and private insurance, respectively. CONCLUSIONS Using a large, multistate, multipayer patient sample, SCD-attributable medical expenditures in children were conservatively and approximately estimated at

Communication of Positive Newborn Screening Results for Sickle Cell Disease and Sickle Cell Trait: Variation Across States

335 million in 2005.

Medication adherence among pediatric patients with sickle cell disease: A systematic review

OBJECTIVE: To develop a set of quality-of-care indicators for the management of children with sickle cell disease (SCD) who are cared for in a variety of settings by addressing the broad spectrum of complications relevant to their illness. METHODS: We used the Rand/University of California Los Angeles appropriateness method, a modified Delphi method, to develop the indicators. The process included a comprehensive literature review with ratings of the evidence and 2 rounds of anonymous ratings by an expert panel (nominated by leaders of various US academic societies and the National Heart, Lung, and Blood Institute). The panelists met face-to-face to discuss each indicator in between the 2 rounds. RESULTS: The panel recommended 41 indicators that cover 18 topics; 17 indicators described routine health care maintenance, 15 described acute or subacute care, and 9 described chronic care. The panel identified 8 indicators most likely to have a large positive effect on improving quality of life and/or health outcomes for children with SCD, which covered 6 topics: timely assessment and treatment of pain and fever; comprehensive planning; penicillin prophylaxis; transfusion; and the transition to adult care. CONCLUSIONS: Children with SCD are at risk for serious morbidities and early mortality, yet efforts to assess and improve the quality of their care have been limited compared with other chronic childhood conditions. This set of 41 indicators can be used to assess quality of care and provide a starting point for quality-improvement efforts.

Pasteurized Human Donor Milk Use among US Level 3 Neonatal Intensive Care Units

There are no current national estimates on health care utilization and expenditures for US children with sickle cell disease (SCD).

Management of Children With Sickle Cell Disease: A Comprehensive Review of the Literature

In the US, all states and the District of Columbia have universal newborn screening (NBS) programs for sickle cell disease (SCD), which also identify sickle cell trait (trait). In this project, we surveyed follow‐up coordinators, including one in the District of Columbia and two in Georgia, about protocols for stakeholder notification for SCD and trait. The primary outcomes were total number and type of stakeholder informed of a positive screen. We received 52 completed surveys (100% response). Primary care providers (PCPs) (100%), hematologists (81%), hospitals (73%), and families (40%) were the most commonly notified stakeholders of positive SCD screens, while PCPs (88%), hospitals (63%), and families (37%) were most commonly notified for trait. On average, 3.4 stakeholders were notified for a positive screening for SCD, compared to 2.4 stakeholders for sickle cell trait (P < 0.001). In multivariate analyses for SCD, we found a 2.9% increase in stakeholders notified for each additional year of universal screening mandated in a state (95% CI: 1.4–4.4%). For trait, we found an 8.5% increase in stakeholders notified for each additional follow‐up staff (95% CI: 1.3–15.7%), and a 1.3% increase for each additional percent of black births in the state (95% CI: 0.1–2.5%). Wide variation exists in stakeholder notification by NBS programs of positive screenings for SCD and trait. This variation may alter the effectiveness of NBS programs by location of birth.

Improving the Management of Vaso-Occlusive Episodes in the Pediatric Emergency Department.

OBJECTIVES: Describe rates of adherence for sickle cell disease (SCD) medications, identify patient and medication characteristics associated with nonadherence, and determine the effect of nonadherence and moderate adherence (defined as taking 60%–80% of doses) on clinical outcomes. METHODS: In February 2012 we systematically searched 6 databases for peer-reviewed articles published after 1940. We identified articles evaluating medication adherence among patients <25 years old with SCD. Two authors reviewed each article to determine whether it should be included. Two authors extracted data, including medication studied, adherence measures used, rates of adherence, and barriers to adherence. RESULTS: Of 24 articles in the final review, 23 focused on 1 medication type: antibiotic prophylaxis (13 articles), iron chelation (5 articles), or hydroxyurea (5 articles). Adherence rates ranged from 16% to 89%; most reported moderate adherence. Medication factors contributed to adherence. For example, prophylactic antibiotic adherence was better with intramuscular than oral administration. Barriers included fear of side effects, incorrect dosing, and forgetting. Nonadherence was associated with more vaso-occlusive crises and hospitalizations. The limited data available on moderate adherence to iron chelation and hydroxyurea indicates some clinical benefit. CONCLUSIONS: Moderate adherence is typical among pediatric patients with SCD. Multicomponent interventions are needed to optimally deliver life-changing medications to these children and should include routine monitoring of adherence, support to prevent mistakes, and education to improve understanding of medication risks and benefits.

Sickle Cell Trait—Neglected Opportunities in the Era of Genomic Medicine

Background: Pasteurized human donor milk (DM) is recommended by the World Health Organization and American Academy of Pediatrics for preterm infants when mother’s own milk is unavailable, yet the extent and predictors of use and criteria for use in US neonatal intensive care units (NICUs) are unknown. Objective: This study aimed to evaluate current DM use in US level 3 NICUs and predictors and criteria of use. Methods: We sent mail surveys to 302 US level 3 NICU directors. We used multivariable logistic regression to analyze predictors of DM use. Results: Survey response rate was 60%, and 76 of 182 (42%) directors reported DM use. Among DM users, 30% have used DM < 2 years and 55% for 2 to 5 years. Among nonusers, 63% were uncertain of turnaround time when ordering DM, 36% were unclear what guidelines milk banks followed, and 31% were unsure of parent receptiveness. In multivariate analyses, > 800 annual admissions (odds ratio [OR], 4.11; 95% confidence interval [CI], 1.43-11.82; reference ≤ 400 admissions) and location in the Midwest (OR, 3.02; 95% CI, 1.17-7.76) and West (OR, 6.33; 95% CI, 2.28-15.57; reference Northeast) were positively associated with DM use; safety-net hospitals (> 75% Medicaid insurance) were negatively associated (OR, 0.35; 95% CI, 0.14-0.89). Conclusion: Pasteurized human donor milk use is rapidly emerging among US level 3 NICUs. Larger NICUs and those in the West and Midwest were more likely to use DM, while safety-net hospitals were less likely to use DM. Lack of knowledge by medical directors of accessibility, safety, and parental receptiveness may be barriers to DM use.

Weight Status of Children With Sickle Cell Disease

OBJECTIVE: Sickle cell disease (SCD) affects 70 000 to 100 000 people in the United States, and 2000 infants are born with the disease each year. The purpose of this study was to review the quality of the literature for preventive interventions and treatment of complications for children with SCD to facilitate the use of evidence-based medicine in clinical practice and identify areas in need of additional research. METHODS: We searched the Ovid Medline database and the Cochrane Library for articles published between January 1995 and April 2010 for English-language abstracts on 28 topics thought to be important for the care of children with SCD. We also added pertinent references cited by studies identified in our search. Each abstract was reviewed independently by 2 authors. Data from articles retrieved for full review were abstracted by using a common form. RESULTS: There were 3188 abstracts screened, and 321 articles underwent full review. Twenty-six articles (<1% of abstracts initially screened), which consisted of 25 randomized controlled trials and 1 meta-analysis, were rated as having level I evidence. Eighteen of the 28 topics selected for this review did not have level I evidence studies published. The management and prevention of pain episodes accounted for more than one-third of the level I studies. CONCLUSIONS: Although significant strides have been made in the care of children with SCD in the past 2 decades, more research needs to be performed, especially for acute events associated with SCD, to ensure that the health and well-being of children with SCD continues to improve.