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For Black children with sickle cell disease (SCD) and their families, high disease stigmatization and pervasive racism increase susceptibility to discrimination in healthcare settings. Childhood experiences of discrimination can result in medical nonadherence, mistrust of healthcare providers, and poorer health outcomes across the lifespan. Caregivers and medical providers are essential to childhood SCD management and are therefore well-positioned to provide insight into discrimination in the context of pediatric SCD. This mixed-methods study sought caregivers’ and providers’ perspectives on processes underlying discrimination and potential solutions to mitigate the negative effects of perceived discrimination among children with SCD. Caregivers (N = 27) of children with SCD (≤ 12 years old) and providers from their hematology clinics (N = 11) participated in individual semi-structured interviews exploring experiences of discrimination and daily SCD management and completed a quantitative measure of discrimination. Qualitative data were collected until themes reached saturation and subsequently transcribed verbatim, coded, and analyzed using applied thematic analysis. Quantitative and qualitative data converged to suggest the pervasiveness of discrimination in healthcare settings. Three qualitative themes emerged: (1) healthcare system factors underlie discrimination, (2) families’ challenging interactions with providers lead to perceptions of discrimination, and (3) experiences of discrimination impact caregiver-provider interactions. Both caregivers and providers highlighted building trusting patient-provider relationships and encouraging patients’ self-advocacy as means to reduce experiences and impacts of discrimination. These findings offer potential approaches to tangibly mitigate occurrences of discrimination in pediatric healthcare settings by trust building, accountability keeping, and fostering rapport to improve quality of care and pediatric SCD health outcomes.

For Black children with sickle cell disease (SCD) and their families, high disease stigmatization and pervasive racism increase susceptibility to discrimination in healthcare settings. Childhood experiences of discrimination can result in medical nonadherence, mistrust of healthcare providers, and poorer health outcomes across the lifespan. Caregivers and medical providers are essential to childhood SCD management and are therefore well-positioned to provide insight into discrimination in the context of pediatric SCD. This mixed-methods study sought caregivers' and providers' perspectives on processes underlying discrimination and potential solutions to mitigate the negative effects of perceived discrimination among children with SCD. Caregivers (N = 27) of children with SCD (≤ 12 years old) and providers from their hematology clinics (N = 11) participated in individual semi-structured interviews exploring experiences of discrimination and daily SCD management and completed a quantitative measure of discrimination. Qualitative data were collected until themes reached saturation and subsequently transcribed verbatim, coded, and analyzed using applied thematic analysis. Quantitative and qualitative data converged to suggest the pervasiveness of discrimination in healthcare settings. Three qualitative themes emerged: (1) healthcare system factors underlie discrimination, (2) families' challenging interactions with providers lead to perceptions of discrimination, and (3) experiences of discrimination impact caregiver-provider interactions. Both caregivers and providers highlighted building trusting patient-provider relationships and encouraging patients' self-advocacy as means to reduce experiences and impacts of discrimination. These findings offer potential approaches to tangibly mitigate occurrences of discrimination in pediatric healthcare settings by trust building, accountability keeping, and fostering rapport to improve quality of care and pediatric SCD health outcomes.

Objective Our aim was to develop an efficient search strategy for prognostic studies and clinical prediction guides (CPGs), optimally balancing sensitivity and precision while independent of MeSH terms, as relying on them may miss the most current literature. Materials and Methods We combined 2 Hedges-based search strategies, modified to remove MeSH terms for overall prognostic studies and CPGs, and ran the search on 269 journals. We read abstracts from a random subset of retrieved references until ≥ 20 per journal were reviewed and classified them as positive when fulfilling standardized quality criteria, thereby assembling a standard dataset used to calibrate the search strategy. We determined performance characteristics of our new search strategy against the Hedges standard and performance characteristics of published search strategies against the standard dataset. Results Our search strategy retrieved 16 089 references from 269 journals during our study period. One hundred fifty-four journals yielded ≥ 20 references and ≥ 1 prognostic study or CPG. Against the Hedges standard, the new search strategy had sensitivity/specificity/precision/accuracy of 84%/80%/2%/80%, respectively. Existing published strategies tested against our standard dataset had sensitivities of 36%–94% and precision of 5%–10%. Discussion We developed a new search strategy to identify overall prognosis studies and CPGs independent of MeSH terms. These studies are important for medical decision-making, as they identify specific populations and individuals who may benefit from interventions. Conclusion Our results may benefit literature surveillance and clinical guideline efforts, as our search strategy performs as well as published search strategies while capturing literature at the time of publication.

Objectives: Contraception use reduces teen pregnancy, and long-acting reversible contraception is recommended as first-line treatment. Since many adolescents use the emergency department (ED) as a ...

Purpose Adolescents and young adults (AYA) with sickle cell disease (SCD) are at risk for serious complications including increased morbidity and early mortality during their transition from pediatric to adult care. Self-management support may help improve transition outcomes in this vulnerable population. Interventions based on teaching problem solving skills have been shown to improve adherence to therapy for AYA with other chronic disease and is a promising intervention in SCD. We sought patient and parent perspectives on improving self-management and input on the development of a problem-solving education (PSE) intervention. Design and methods We held focus groups with AYA with SCD and caregivers to discuss barriers and facilitators of self-management, acceptability of PSE and intervention content and delivery. Results Five focus groups were held with AYA (n = 17) and caregivers (n = 15). Groups participated jointly to discuss self-management and then separately to discuss PSE. Data were analyzed using grounded theory and double-coded until thematic saturation was achieved. Conclusions Both groups endorsed PSE as an acceptable intervention. Barriers to self-management included wanting to fit in with peers (AYA) and trouble letting go (parents); facilitators included having a regular routine (AYA) and sharing responsibility (parents). Participants suggested meeting in small groups for PSE sessions rather than individually and adding group sessions for parents. Practical implications Understanding AYA and caregivers' perceptions of barriers and facilitators of transition in SCD can help us better prepare AYA for transition. The specifics both groups identified as helpful will guide intervention development.

Background The purpose of this study was to assess pediatric hematology clinic staff’s perspectives regarding barriers and facilitators in addressing unmet basic needs for children with sickle cell disease (SCD). Methodology Six focus groups were held at four urban pediatric hematology clinics in the Northeastern region of the United States from November to December 2019. Discussion questions were developed to align with the integrated Promoting Action on Research Implementation in Health Services (i-PARIHS) implementation science framework, focusing on the domains of context and recipient and how clinics address adverse social determinants of health (SDoH) in their patient populations. A summative content analytical approach was taken to identify major themes in the data. Results We discerned the following themes: (1) families of children with SCD experience numerous unmet basic needs; (2) clinic staff believed they had a role to play in addressing these unmet basic needs; (3) staff felt their ability to address families’ unmet basic needs depended upon caregivers’ capacity to act on staff’s recommendations; and (4) clinic staff’s ability to address these needs was limited by organizational and systemic factors beyond their control. Conclusions These findings have important implications for how best to address adverse SDoH for this vulnerable pediatric population so that urban-based pediatric hematology clinics can more equitably support families.

The purpose of this study was to assess pediatric hematology clinic staff’s perspectives regarding barriers and facilitators in addressing unmet basic needs for children with sickle cell disease (SCD). Six focus groups were held at four urban pediatric hematology clinics in New England from November to December 2019. Discussion questions were developed to align with the integrated Promoting Action on Research Implementation in Health Services (i-PARIHS) implementation science framework, focusing on the domains of context and recipient and how clinics address adverse social determinants of health (SDoH) in their patient populations. A summative content analytical approach was taken to identify major themes in the data. We discerned the following themes: (1) families of children with SCD experience numerous unmet basic needs; (2) clinic staff believed they had a role to play in addressing these unmet basic needs; (3) staff felt their ability to address families’ unmet basic needs depended upon caregivers’ capacity to act on staff’s recommendations; and (4) clinic staff’s ability to address these needs was limited by organizational and systemic factors beyond their control. These findings have important implications for how best to address adverse SDoH for this vulnerable pediatric population so that urban-based pediatric hematology clinics can more equitably support families.

ObjectivesCoordinated measurement strategies are needed to inform collaborative approaches to improve access to and quality of care for persons with sickle cell disease (SCD). The objective of our study was to develop a multilevel measurement strategy to assess improvements in access to and quality of care for persons with SCD in 4 US regions.MethodsFrom 2014 through 2017, regional grantees in the Sickle Cell Disease Treatment Demonstration Program collected administrative and patient-level electronic health record (EHR) data to assess quality improvement initiatives. Four grantees—covering 29 US states and territories and an SCD population of 56 720—used a collective impact model to organize their work. The grantees collected administrative data from state Medicaid and Medicaid managed care organizations (MCOs) at multiple points during 2014-2017 to assess improvements at the population level, and local patient-level data were abstracted from site-level EHRs at regular intervals to track improvements over time.ResultsAdministrative data were an important source of understanding population-level improvements but were delayed, whereas patient-level data were more sensitive to small-scale quality improvements.ConclusionsWe established a shared measurement approach in partnership with Medicaid and Medicaid MCO stakeholders that can be leveraged to effectively support quality improvement initiatives for persons with SCD in the United States.

Objectives Painful vaso-occlusive episodes (VOE) are the most common reason for emergency department (ED) visits experienced by patients with sickle cell disease (SCD). The National Heart, Lung and Blood Institute (NHLBI) evidence-based recommendations for VOE treatment are based primarily on expert opinion. In this randomized controlled trial (RCT), we will compare changes in pain scores between patients randomized to a patient-specific analgesic protocol versus those randomized to a weight-based analgesic protocol, as recommended by the NHLBI guidelines. Methods We report the rationale and design of a multi-site, phase III, single-blinded, RCT to be conducted in six EDs in the United States. Eligible participants will be randomized after providing consent, anticipating 50% of those randomized would have an ED visit during the enrollment period. A total of 230 participants with one VOE ED visit provides sufficient power to detect a clinically significant difference in pain score reductions of 14 between groups with 0.05 type I error. Uniquely, this trial randomizes participants in a larger population than the study population, given the impossibility of consenting and randomizing participants during emergencies. The primary endpoint is the change in pain scores in the ED from time of placement in treatment area to time of disposition (hospitalization, discharged home, or assigned to observation status) or a maximum treatment duration of 6 hours. Additional outcomes include hospitalizations and ED visits seven days post enrollment, side effects, and safety assessments. Conclusions The COMPARE-VOE study design will provide high-level evidence to support the NHLBI VOE treatment guidelines.

OBJECTIVES: Vaso-occlusive episodes (VOEs) account for the majority of emergency department (ED) visits for children with sickle cell disease (SCD). We hypothesized that addressing key barriers to VOE care would improve receipt of analgesics and outcomes. METHODS: A quality improvement (QI) initiative was conducted from September 2010 to April 2014 to streamline VOE care in an urban pediatric ED. Four interventions were used: a standardized time-specific VOE protocol; intranasal fentanyl as the first parenteral pain medication; an SCD pain medication calculator; and provider and patient/family education. Data were collected for 3 outcome measures (mean time from triage to first parenteral opioid and admission/discharge decision, and proportion discharged from the ED); 1 process measure (mean time from triage to initiation of patient-controlled analgesia); and 4 balancing measures (mean time from triage to second intravenous opioid dose, 24-hour ED readmission, respiratory depression, and length of stay). RESULTS: There were 289 ED visits in the study period. Improvements were seen in mean time to: first dose of parenteral opioid (56 to 23 minutes); second opiate intravenous dose (106 to 83 minutes); admission and discharge decisions (163 to 109 minutes and 271 to 178 minutes, respectively); and initiation of patient-controlled analgesia (216 to 141 minutes). The proportion discharged from the ED increased from 32% to 48% (χ2 = 6.5402, P = .01). No increase in 24-hour readmission, respiratory depression, or inpatient length of stay was observed. CONCLUSIONS: Using VOE-specific interventions, we significantly improved VOE care for children. Studies are needed to determine if these results can be replicated.

Background Children with sickle cell disease (SCD) are at increased risk of complications from influenza. However, despite widespread recommendations that these patients receive an annual influenza immunization, reported vaccination rates remain very low at under 50%. Procedure Our aim was to increase the influenza vaccination rate among our pediatric patients with SCD aged 6 months to 21 years over two influenza seasons, 2012–2013 and 2013–2014, to 80%, consistent with the Health People 2020 goal. We used multiple quality improvement methods, based on the literature and our previous experience in other aspects of SCD care, including parent and provider education, enhancement of our EHR, use of a SCD patient registry and reminder and recall done by a patient navigator. Results We vaccinated 80% of our pediatric patients with SCD for influenza during the 2012–2013 season and 90% of patients in 2013–2014. Our early season vaccination rates were nearly double that of those for the general population. Conclusions Use of quality improvement methods can increase rates of influenza vaccination for this high-risk population, suggesting that less health care utilization and lower cost might result. Pediatr Blood Cancer 2015;62:654–657. © 2014 Wiley Periodicals, Inc.

In children with Sickle Cell Disease, the combination of risk stratification with Transcranial Doppler Ultrasound (TCD) and selective chronic red cell transfusion (CRCT—the STOP Protocol) is one of the most effective stroke prevention strategies in medicine. How fully it is being implemented is unclear.

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.

Despite the recent advances made in the care of children with sickle cell disease (SCD), premature mortality, especially among older children and young adults, remains a hallmark of this disease. The lack of survival gains highlights the translational gap of implementing innovations found efficacious in the controlled trial setting into routine clinical practice. Health services research (HSR) examines the most effective ways to finance, organize, and deliver high quality care in an equitable manner. To date, HSR has been underutilized as a means to improve the outcomes for children with SCD. Emerging national priorities in health care delivery, new sources of funding, and evolving electronic data collection systems for patients with SCD have provided a unique opportunity to overcome the translational gap in pediatric SCD. The purpose of this article is to provide a comprehensive HSR agenda to create patient-specific evidence of clinical effectiveness for interventions used in the routine care setting, understand the barriers faced by clinicians to providing high quality care, assess and improve the interactions of patients with the health care system, and measure the quality of care delivered to increase survival for all children and young adults with SCD.

OBJECTIVE. In this we study explored the relationship between food insecurity and compensatory maternal feeding practices that may be perceived as buffers against periodic food shortages among urban black families.METHODS. We interviewed a convenience sample of black mothers of children aged 2 to 13 years. Food-security status (predictor) was determined at the household level. Five maternal feeding practices (outcomes) were assessed. Two were based on Birch's Child Feeding Questionnaire (restricting access to certain foods and pressuring a child to eat), and 3 were derived from investigators' clinical experience (use of high-energy supplements, added sugar in beverages, and perceived appetite stimulants). Anthropometric data were collected from mothers and children.RESULTS. A total of 278 mother–child dyads were analyzed, and 28% of these mothers reported being food insecure. Use of Child Feeding Questionnaire feeding practices was defined as the top quartile of responses. Use of nutritional supplements, defined as “at least 1 to 2 times monthly,” ranged from 13% to 25%. In logistic regression models adjusted for child age, weight status, and ethnicity and maternal weight status, mothers from food-insecure households were significantly more likely to use high-energy supplements and appetite stimulants. The odds of using the remaining compensatory feeding practices (adding sugars to beverages, pressuring a child to eat, and restricting access to certain foods) were elevated among food-insecure households but did not reach statistical significance.CONCLUSIONS. Household food insecurity was independently associated with 2 of the 5 maternal compensatory feeding practices studied, and such practices may alter the feeding environment. Longitudinal research is necessary to determine how the relationship between food security and compensatory maternal feeding practices may affect child weight trajectories.

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 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.

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.

OBJECTIVE: Historically, many children and adolescents with sickle cell disease (SCD) were underweight. Treatment advances like hydroxyurea have been associated with improved growth. We hypothesized that increased hemoglobin (Hb) levels would be associated with increased weight status of children with SCD. METHODS: Investigators at 6 institutions conducted a retrospective chart review of all patients aged 2 to 19 years of age for the calendar years 2007–2009. Height, weight, baseline Hb levels, demographic information, and select comorbidities were recorded from the most recent clinic visit. Overweight and obesity were defined as ≥85th and ≥95th BMI percentiles for age and gender, respectively, and underweight was defined as RESULTS: Data were collected on 675 children and adolescents in 3 New England states. In this sample, 22.4% were overweight or obese, whereas only 6.7% were underweight. Overweight or obese status was associated with sickle genotypes other than Hb SS or Hb Sβ0 disease, and were associated with higher baseline Hb levels. Underweight individuals were more likely to be male, older, and have had at least 1 SCD-related complication. After adjusting for demographic factors, any SCD-related complication, SCD-directed treatments, and obesity-related conditions, there was a 36% increased odds of overweight/obesity for each 1 g/dL increase in baseline Hb levels. CONCLUSIONS: Nearly one-quarter of children and adolescents with SCD in New England are overweight or obese. Longitudinal studies are needed to determine the impact of elevated BMI on the morbidity and mortality of both children and adults with SCD.

Objective Despite recent scientific advances, children with sickle cell disease (SCD) continue to experience high mortality and significant morbidity, in part due to variations in the care provided. We sought to identify and compare drivers for quality improvement among clinical staff and parents of children with SCD. Methods We interviewed clinical staff across care settings in an urban teaching hospital to elicit their perspectives on improving care for children with SCD. Concurrently, we invited parents of children with SCD to participate in focus groups to identify their needs. Findings are reported according to Consolidated Criteria for Reporting Qualitative Research guidelines. Results We conducted 29 interviews with clinical staff and 4 focus groups with parents. Both groups identified the need for effective communication of relevant patient information across disciplines as a key area for improvement. Clinical staff cited standardization of care delivery as a top priority through increased accessibility of pertinent clinical information, enhanced pain assessment and management, and improved availability of clinical decision-making tools. Parents listed the need for increased community awareness about SCD, including school and day care staff, enhanced parental education and peer support, and self-management skills for their children as opportunities to improve pediatric SCD care. Conclusions Identifying drivers for quality improvement is a critical first step in transforming the care provided to children with SCD. Using a systematic approach that includes eliciting the perspectives of both clinicians and parents may significantly enhance the development of a patient-centered quality improvement agenda.

Background: Pain is the most common complication of sickle cell disease (SCD), and pain diaries are an effective way to capture pain experienced by both children and adults for many conditions, including SCD. The most recent data on pain experienced by children with SCD outside the clinical trial setting, particularly those with Hemoglobin (Hb) SS disease, were collected prior to the publication of the Baby-HUG trial in 2011, which found hydroxyurea to be safe for use in early childhood. One study found that school-aged children with SCD had at least one pain episode per month; another longitudinal study reported that SCD-related pain was experienced by children on 8% of pain diary days. In the era of hydroxyurea use in children, the occurrence of SCD-related pain episodes in children has not been well studied, especially among those with Hb SS disease. Methods: Parent/child dyads were recruited for a 6-month pilot study of the use of home pain plans and pain diaries from a pediatric hematology clinic based in a safety net institution. The study ran from February to November 2014. Dyads were eligible if the child with SCD was aged 5-15 years and had at least one episode of pain in the year prior to enrollment, by parent report. Parents and children were asked to fill out the diary each day. They received weekly phone calls for the first month, then monthly phone calls until the close of the study, to remind them to complete the daily pain diary and mail it back monthly; $20 gift cards were given for each diary returned. We excluded data from dyads that recorded less than 4 weeks of pain diary data. Results: Thirty-six parent/child dyads were approached and 30 (83%) consented to enroll in the study. Twenty-six (87%) dyads recorded at least four weeks of data. The average age of the child with SCD was 10.3 ± 3.0 years and 20 (77%) were insured through Medicaid. Twenty-two (85%) children had Hb SS disease, of whom 21 were prescribed hydroxyurea. Data were recorded per dyad on average 135 ± 46 diary days. One child with Hb SS disease reported no pain during the study period. The remaining 25 children had any pain and SCD-related pain an average of 8% and 4% diary days, respectively. Nearly all children reporting non-SCD-related pain had headache or abdominal pain. Ten (38%) children missed at least one day of school during the study period. Conclusions: In thissample of children with SCD, composed predominantly of those with Hb SS disease prescribed hydroxyurea, pain was an infrequent occurrence and was attributed to SCD only half the time. This study suggests that SCD-related pain experienced by children may be lower than previously reported. Further study is warranted, especially among those prescribed hydroxyurea. Disclosures No relevant conflicts of interest to declare.

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 ( 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.

Despite the recent advances made in the care of children with sickle cell disease (SCD), premature mortality, especially among older children and young adults, remains a hallmark of this disease. The lack of survival gains highlights the translational gap of implementing innovations found efficacious in the controlled trial setting into routine clinical practice. Health services research (HSR) examines the most effective ways to finance, organize, and deliver high quality care in an equitable manner. To date, HSR has been underutilized as a means to improve the outcomes for children with SCD. Emerging national priorities in health care delivery, new sources of funding, and evolving electronic data collection systems for patients with SCD have provided a unique opportunity to overcome the translational gap in pediatric SCD. The purpose of this article is to provide a comprehensive HSR agenda to create patient-specific evidence of clinical effectiveness for interventions used in the routine care setting, understand the barriers faced by clinicians to providing high quality care, assess and improve the interactions of patients with the health care system, and measure the quality of care delivered to increase survival for all children and young adults with SCD.

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 $9369 and $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 $335 million in 2005.

Background There are no current national estimates on health care utilization and expenditures for US children with sickle cell disease (SCD). Procedure We used the MarketScan® Medicaid Database and the MarketScan® Commercial Claims and Encounters Database for 2005 to estimate health services use and expenditures. The final samples consisted of 2,428 Medicaid-enrolled and 621 privately insured children with SCD. Results The percentage of children with SCD enrolled in Medicaid with an inpatient admission was higher compared to those privately insured (43% vs. 38%), yet mean expenditures per admission were 35% lower ($6,469 vs. $10,013). The mean number of emergency department (ED) visits was 49% higher for Medicaid-enrolled children compared to those with private insurance (1.36 vs. 0.91), but mean expenditures per ED visit were 28% lower. The mean number of non-ED outpatient visits was similar (12.6 vs. 11.5) but mean expenditures were 40% lower for the Medicaid-enrolled children ($3,557 vs. $5,908). The mean expenditures on drug claims were higher among those with Medicaid than private insurance ($1,049 vs. $531). Mean total expenditures for children with SCD enrolled in Medicaid were 25% lower than for privately insured children ($11,075 vs. $14,722). The samples were comparable with respect to SCD-related inpatient discharge diagnoses and use of outpatient blood transfusions. Conclusions Children with SCD enrolled in Medicaid had lower expenditures than privately insured children, despite higher utilization of medical care, which indicates lower average reimbursements. Research is needed to assess the quality of care delivered to Medicaid-enrolled children with SCD and its relation to health outcomes. Pediatr Blood Cancer 2009;53:642–646. Published 2009 Wiley-Liss, Inc.

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.

Background: Vaso-occlusive episodes (VOE) are the leading cause of hospitalizations & emergency department (ED) visits in sickle cell disease (SCD), & are associated with increased mortality. Intranasal fentanyl (INF) provides rapid & powerful parenteral analgesia, with an onset of action of 5-10 minutes, & peak effect in 30 minutes. INF is a safe & effective method of pain management for children in the ED & other settings, yet it is underutilized in SCD. Objective: To study the impact of the addition of INF to standard ED management for SCD VOE on time to parenteral opioid, pain scores, ED length of stay (LOS) & admission rate. Methods: This quality improvement initiative was conducted at a tertiary pediatric ED at Children's Healthcare of Atlanta from November 2013-May 2014. Patients with all genotypes of SCD ages ≥2 years old who presented with VOE & pain score >6/10 were offered INF within 15 minutes of triage. 2 INF doses (1.5mcg/kg/dose; max 100mcg/dose) were given 5 minutes apart. Patients then continued on institutional standard protocol treatment for VOE (±PO hydrocodone, ±IV ketorolac, & ± IV morphine or IV hydromorphone). Pain scores were documented at frequent intervals by nursing staff. Patient/parent satisfaction data were obtained using a satisfaction questionnaire. Outcomes included: Time from arrival to 1st parenteral opioid, pain scores, ED LOS (time from arrival to disposition), admission rates & patient/parent satisfaction. We compared patients in this study to those who did not receive INF during the study period (n=48) & historical controls from Jan-Dec 2012 (n=231) for the 1st three outcomes. Results: 248 visits for VOE met inclusion criteria, of whom 228 received parenteral opioids (92%) & 180/228 (79 %) received INF. Mean age was 11.7±4.5 years. The majority were female (56%), 65% HbSS, 14% HbSC & 21% had other genotype. 48 patients did not receive INF; 36 were not offered INF without explanation for protocol deviation &12 patients refused. They had similar gender & Hb genotype, but were older than INF+ patients (13.5±4.0 years, p=0.01). The mean time from arrival to 1st parenteral opioid decreased significantly in the INF+ group compared to historical controls, and was remarkably shorter than those not treated with INF. There was minimal difference in LOS between INF+ group & historical controls, but LOS was shorter in the group that did not receive INF. Admission rates were significantly higher in those who did not receive INF when compared to those treated with INF & historical controls. (See Table 1) Table 1 INF+(n =180) INF-(n =48) Historical Data (n=231) P-value Time to 1st Parenteral Opioid (minutes) 29 ±15 77 ±44 35±18 p < 0.0001 LOS (minutes) 215± 86 197± 67 231±95 p = 0.028 Admission Rate (%) 48% 71% 45% p = 0.004 Mean baseline pain score was 8.5±1.5, 1st reassessment pain score after INF was 7.8±2.4 & last ED pain score prior to disposition was 5.5±3.4. Pain scores were similar in all groups. 98 families completed the patient satisfaction questionnaire, 79 (81%) of whom received INF. 65% of patients who received INF were satisfied & would like to receive the treatment again in the ED. Conclusions: The addition of INF to the management of SCD VOE significantly improved time to receipt of 1st parenteral analgesia & was well tolerated. Admission rates were significantly higher in patients who did not receive INF during our study period. The associated delay in time to receipt of 1st parenteral analgesia may have contributed to the increased admission rate. However, causality of INF on admission rates cannot be determined without further study. INF did not impact ED LOS compared to historical controls, however rapid admission turn-around time likely decreased LOS in the INF- group. INF is a safe & effective strategy to provide rapid pain relief in children with SCD & VOE. Disclosures Off Label Use: Intranasal Fentanyl (INF). Widely accepted as off label use in pediatric ED's and a variety of other clinical settings for the management of pain. It is a synthetic narcotic analgesic with a rapid onset and short duration of action. Current evidence suggests that INF is a safe and effective method of pain management for children in a variety of clinical settings..

Objective To assess the economic efficiency of adding troglitazone to sulfonylurea therapy to improve glycemic control. Background Despite the high prevalence of type 2 diabetes, existing treatment strategies often fail. New oral agents give a wider segment of the population with type 2 diabetes hope of achieving near-normal blood-glucose levels. Troglitazone, a novel chemical entity, is one promising new agent. Methods We conducted an economic analysis based on glycemic-control data from a randomized clinical trial comparing troglitazone with placebo, each added to glyburide. A patient simulation model was used to translate these data to long-term outcomes associated with diabetes. Patients had poorly controlled type 2 diabetes mellitus despite glyburide therapy. Risk functions of developing and progressing through nephropathy, retinopathy, neuropathy, hypoglycemia, and macrovascular disease were developed from the Diabetes Control and Complications Trial and large epidemiologic studies. Cost estimates were based on data from 5 states, all payor databases, surveys, and literature. The main outcomes of the model were cost-consequences, number of patients developing each type of complication, mean time to development of the complication, cost per life-year gained (LYG), and cost per quality-adjusted life-year. Results The model predicts that for every 1000 patients treated with troglitazone, the improved glycemic control could mean that 95 to 140 fewer patients would experience one of the most severe diabetic complications (eg, blindness, end-stage renal disease, amputation), which may increase life expectancy by 2.0 years. These benefits are obtained at an additional $2100 per LYG (undiscounted). The ratio remains Conclusions The clinical trial demonstrated that troglitazone + glyburide improves glycemic control compared with glyburide alone. Based on these results, the model estimates fewer diabetic complications at a cost well below accepted cost-effective thresholds.

OBJECTIVE To estimate direct medical costs of managing the complications of type 2 diabetes. RESEARCH DESIGN AND METHODS Costs were estimated for 15 diabetic complications by applying unit costs to typical resource-use profiles. Resource use and unit costs were estimated from many sources, including acute care discharge databases, clinical guidelines, government reports, fee schedules, and peer-reviewed literature. For each complication, the event costs are those associated with resource use that is specific to the acute episode and any subsequent care occurring in the 1st year. State costs are the annual costs of continued management. All costs are expressed in 1996 U.S. dollars. RESULTS As expected, the more severe or debilitating events, such as acute myocardial infarction ($27,630 event cost; $2,185 state cost), generate a greater financial burden than do early-stage complications, such as microalbuminuria ($62 event cost; $14 state cost). Yet, complications that are initially relatively low in cost (e.g., microalbuminuria) can progress to more costly advanced stages (e.g., end-stage renal disease, $53,659 state cost); therefore, minor complications should also be considered in any economic analysis of diabetes. CONCLUSIONS The recent literature has lacked cost estimates that may be readily translated into patient-level cost inputs for an economic model. Emerging therapies that may reduce the incidence of some diabetic complications will need to be scrutinized economically in today's cost-conscious environment. The cost estimates from this study provide one piece of the economic analysis needed to evaluate these new interventional therapies.