Development of a Hydroxyurea Decision Aid for Parents of Children With Sickle Cell Anemia.

National evidence-based guidelines recommend offering hydroxyurea to patients with sickle cell anemia 9 months of age and older using shared decision making, but offer no strategies to aid implementation. We developed a hydroxyurea multicomponent decision aid via a needs assessment, clinic observations, and iterative feedback to address parent decision needs and promote a discussion between clinicians and parents. A total of 75 parents and 28 clinicians participated across all phases. The decision aid was rated as useful. Hydroxyurea knowledge improved and decisional conflict decreased supporting the potential for use to facilitate shared decision making in pediatric sickle cell anemia.

Association of silent infarcts in sickle cell anemia with decreased annexin A5 resistance.

BACKGROUND: Sickle cell anemia (SCA) is characterized by abnormally shaped, adhesive RBCs that interact with white blood cells and the endothelium, leading to chronic hemolysis, vasculopathy and a prothrombotic state. About 10% of subjects with a thrombotic event in the general population will have an associated antiphospholipid (aPL) antibody. One proposed mechanism for the thrombophilic nature of aPL antibodies is the disruption of the potent anticoagulant annexin A5 or Annexin A5 resistance (A5R). We designed a pilot study assessing the presence of aPL antibodies and disruption of A5R in pediatric sickle cell subjects. METHODS: 39 subjects with SCA participated in this study. A5R, DRVVT, anti-ß2GP1, anti-ß2GP1, anti-phosphatidylserine and anti-cardiolipin antibody assays were performed. RESULTS: There was a high prevalence of abnormal A5R despite a low prevalence of antiphospholipid antibodies. Multivariate logistic regression analyses showed an association with silent infarcts (p=0.015), lower hemoglobin (p=0.037), older age (p=0.047) and abnormal A5R. CONCLUSION: We report an association between annexin A5 resistance and presence of silent infarct, low hemoglobin, and older age in a subgroup of SCA subjects. A potential role for perturbed A5R in the pathophysiology of SCA needs to be evaluated further.

Mortality of New York children with sickle cell disease identified through newborn screening.

PURPOSE: Long-term follow-up of newborn screening for conditions such as sickle cell disease can be conducted using linkages to population-based data. We sought to estimate childhood sickle cell disease mortality and risk factors among a statewide birth cohort with sickle cell disease identified through newborn screening. METHODS: Children with sickle cell disease identified by newborn screening and born to New York residents in 2000-2008 were matched to birth and death certificates. Mortality rates were calculated (using numbers of deaths and observed person-years at risk) and compared with mortality rates for all New York children by maternal race/ethnicity. Stratified analyses were conducted to examine associations between selected factors and mortality. RESULTS: Among 1,911 infants with sickle cell disease matched to birth certificates, 21 deaths were identified. All-cause mortality following diagnosis was 3.8 per 1,000 person-years in the first 2 years of life and 1.0 per 1,000 person-years at ages 2-9 years. The mortality rate was significantly lower among children of foreign-born mothers and was significantly higher among preterm infants with low birth weight. The mortality rates were not significantly higher for infants after 28 days with sickle cell disease than for all New York births, but they were 2.7-8.4 times higher for children 1 through 9 years old with homozygous sickle cell disease than for those of all non-Hispanic black or Hispanic children born to New York residents. CONCLUSION: Estimated mortality risk in children with homozygous sickle cell disease remains elevated even after adjustment for maternal race/ethnicity. These results provide evidence regarding the current burden of child mortality among children with sickle cell disease despite newborn screening.Genet Med 17 6, 452-459.

Association between Hospital Volume and Within-Hospital Intensive Care Unit Transfer for Sickle Cell Disease in Children's Hospitals.

OBJECTIVE: To assess the relationship between hospital volume and intensive care unit (ICU) transfer among hospitalized children with sickle cell disease (SCD). STUDY DESIGN: We conducted a retrospective cohort study of 83,477 SCD-related hospitalizations at children's hospitals (2009-2012) using the Pediatric Health Information System database. Hospital-level all-cause and SCD-specific volumes were dichotomized (low vs high). Outcomes were within-hospital ICU transfer (primary) and length of stay (LOS) total (secondary). Multivariable logistic/linear regressions assessed the association of hospital volumes with ICU transfer and LOS. RESULTS: Of 83,477 eligible hospitalizations, 1741 (2.1%) involving 1432 unique children were complicated by ICU transfer. High SCD-specific volume (OR 0.77, 95% CI 0.64-0.91) was associated with lower odds of ICU transfer while high all-cause hospital volume was not (OR 0.87, 95% CI 0.73-1.04). A statistically significant interaction was found between all-cause and SCD-specific volumes. When results were stratified according to all-cause volume, high SCD-specific volume was associated with lower odds of ICU transfer at low all-cause volume (OR 0.46, 95% CI 0.38-0.55). High hospital volumes, both all-cause (OR 0.94, 95% CI 0.92-0.97) and SCD-specific (OR 0.86, 95% CI 0.84-0.88), were associated with shorter LOS. CONCLUSIONS: Children's hospitals vary substantially in their transfer of children with SCD to the ICU according to hospital volumes. Understanding the practices used by different institutions may help explain the variability in ICU transfer among hospitals caring for children with SCD.

Pediatric hematology providers on referral for transplant evaluation for sickle cell disease: a regional perspective.

Hematology referral for evaluation is a key step for hematopoietic stem cell transplantation for sickle cell disease (SCD). Pediatric SCD providers in the US Northeast (New York-Mid-Atlantic and New England regions) were surveyed anonymously for perspectives and practices regarding transplant referral and compared by whether they practiced at SCD transplant centers. Data were analyzed using the Fisher exact test, χ test, and logistic regression. Half of the respondents practiced primarily at transplant sites. Most (79%) were enthusiastic about transplant for SCD and 78% had recently referred ≥1 child for evaluation. Overall, 77% limited referral to certain sickle hemoglobinopathies and 44% preferred referral for ß-thalassemia to SCD. Indications selected for referral resembled current transplant criteria, plus family request or poor response to therapy. Referral for children on chronic transfusions predicted enthusiasm and prior referral. Many (66%) referred children with multiple SCD complications, even without matched sibling donors, 37% with sibling donors despite limited disease. Practitioners at transplant centers more commonly accepted event-free survival rates of ≤90% (P=0.002). Northeastern providers expressed varying enthusiasm for referral for evaluation based on eligibility, donor availability, and acceptable risk, with modestly more interest from practitioners at transplant centers. Differing provider perspectives may affect patient referral for transplant consideration.

Material Hardship, Protective Factors, Children's Special Health Care Needs, and the Health of Mothers and Fathers.

BACKGROUND: Parents of children with special health care needs (CSHCN) are at risk of poorer health outcomes. Material hardships also pose significant health risks to parents. Little is known about how protective factors may mitigate these risks and if effects are similar between mothers and fathers. METHODS: This was a cross-sectional survey study conducted using the US 2018/2019 National Survey of Children's Health, including parents of children 0 to 17 with income <200% of the federal poverty level. Separately, for parents of children with and without special health care needs (N-CSHCN), weighted logistic regression measured associations between material hardship, protective factors (family resilience, neighborhood cohesion, and receipt of family-centered care), and 2 outcomes: mental and physical health of mothers and fathers. Interactions were assessed between special health care needs status, material hardship, and protective factors. RESULTS: Sample consisted of parents of 16,777 children; 4440 were parents of CSHCN. Most outcomes showed similar associations for both mothers and fathers of CSHCN and N-CSHCN: material hardship was associated with poorer health outcomes, and family resilience and neighborhood cohesion associated with better parental health outcomes. Family-centered care was associated with better health of mothers but not fathers. Interaction testing showed that the protective effects of family resilience were lower among fathers of CSHCN experiencing material hardship. CONCLUSIONS: Family resilience and neighborhood cohesion are associated with better health outcomes for all parents, though these effects may vary by experience of special health care needs, parent gender, and material hardship.

Sickle cell disease incidence among newborns in New York State by maternal race/ethnicity and nativity.

PURPOSE: Sickle cell disease is estimated to occur in 1:300-400 African-American births, with higher rates among immigrants from Africa and the Caribbean, and is less common among Hispanic births. This study determined sickle cell disease incidence among New York State newborns stratified by maternal race/ethnicity and nativity. METHODS: Newborns with confirmed sickle cell disease born to New York State residents were identified by the New York State newborn screening program for the years 2000-2008 and matched to birth records to obtain birth and maternal information. Annual incidence rates were computed and bivariate analyses were conducted to examine associations with maternal race/ethnicity and nativity. RESULTS: From 2000 to 2008, 1,911 New York State newborns were diagnosed with sickle cell disease and matched to the birth certificate files. One in every 1,146 live births was diagnosed with sickle cell disease. Newborns of non-Hispanic black mothers accounted for 86% of sickle cell disease cases whereas newborns of Hispanic mothers accounted for 12% of cases. The estimated incidence was 1:230 live births for non-Hispanic black mothers, 1:2,320 births for Hispanic mothers, and 1:41,647 births for non-Hispanic white mothers. Newborns of foreign-born non-Hispanic black mothers had a twofold higher incidence of sickle cell disease than those born to US-born non-Hispanic black mothers (P < 0.001). CONCLUSION: This study provides the first US estimates of sickle cell disease incidence by maternal nativity. Women born outside the United States account for the majority of children with sickle cell disease born in New York State. Such findings identify at-risk populations and inform outreach activities that promote ongoing, high-quality medical management to affected children.

Trends in blood transfusion among hospitalized children with sickle cell disease.

BACKGROUND: Blood transfusions represent a major therapeutic option in acute management of sickle cell disease (SCD). Few data exist documenting trends in transfusion among children with SCD, particularly during hospitalization. PROCEDURE: This was an analysis of cross-sectional data of hospital discharges within the Kid's Inpatient Database (years 1997, 2000, 2003, 2006, 2009). Hospitalizations for children (0-18 years) with a primary or secondary SCD-related diagnosis were examined. The primary outcome was blood transfusion. Trends in transfusion were assessed using weighted multivariate logistic regression in a merged dataset with year as the primary independent variable. Co-variables consisted of child and hospital characteristics. Multivariate logistic regression was conducted for 2009 data to assess child and hospital-level factors associated with transfusion. RESULTS: From 1997 to 2009, the percentage of SCD-related hospitalizations with transfusion increased from 14.2% to 28.8% (P < 0.0001). Among all SCD-related hospitalizations, the odds of transfusion increased over 20% for each successive study interval. Hospitalizations with vaso-occlusive pain crisis (OR 1.35, 95% CI 1.27-1.43) or acute chest syndrome/pneumonia (OR 1.24, 95% CI 1.13-1.35) as the primary diagnoses had the highest odds of transfusion for each consecutive study interval. Older age and male gender were associated with higher odds of transfusion. CONCLUSIONS: Blood transfusion is increasing over time among hospitalized children with SCD. Further study is warranted to identify indications contributing to the rise in transfusions and if transfusions in the inpatient setting have been used appropriately. Future studies should also assess the impact of rising trends on morbidity, mortality, and other health-related outcomes.

Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease.

BACKGROUND: Hydroxyurea (HU) is highly effective treatment for sickle cell disease (SCD). While pediatric use of HU is accepted clinical practice, barriers to use may impede its potential benefit. PROCEDURE: A survey of parents of children ages 5-17 years with SCD was performed across five institutions to assess factors associated with HU use. RESULTS: Of the 173 parent responses, 65 (38%) had children currently taking HU. Among parents of children not taking HU, the most commonly cited reasons were that their hematology provider had not offered it, their child was not sufficiently symptomatic and concerns about potential side effects. Even parents of HU users reported widespread concern about effectiveness, long-term safety, and off-label use. In bivariate analyses, children's ages, parental demographics such as education level, or travel time to their hematology provider were not correlated with HU use. Bivariate analysis and multivariate logistic regression revealed three significant factors associated with current HU use: better parental knowledge about its major therapeutic effects (P < 0.001), sickle genotype (P = 0.005), and institution of clinical care (P = 0.04). CONCLUSIONS: Pervasive concerns about HU safety exist, even among parents of current users. Varying knowledge among parents appears to be independent of their demographics, and is associated with HU use. Inter-institutional variability in parental knowledge and drug uptake highlights potentially potent site-specific influences on likelihood of HU use. Overall, these survey data underscore the need for strategies to bolster parental understanding about benefits of HU and address concerns about its safety.

Family, Neighborhood and Parent Resilience Are Inversely Associated With Reported Depression in Adolescents Exposed to ACEs.

OBJECTIVE: To examine the association of 1) extrinsic resilience factors and 2) adverse childhood experiences (ACEs) with a caregiver reported diagnosis of depression in a nationally representative sample of adolescents. METHODS: A cross sectional analysis of the 2016-2017 National Survey of Children's Health, restricted to adolescents 12 to 17 years old was conducted. The dependent variable was caregiver reported depression: no current diagnosis vs. current diagnosis of depression. Independent variables were reported ACEs dichotomized as lower (0-3) or higher (4 or more), and specific resilience factors: family resilience, neighborhood cohesion and caregiver emotional support. Resilience factors were analyzed as a composite score dichotomized as lower (0-3) or higher (4 or more) and individually. Purposeful selection multivariable logistic regression model building was used to estimate the associations between reported diagnosis of depression, ACEs and resilience factors adjusting for demographic covariates. RESULTS: Study sample consisted of 29,617 (weighted N = 24,834,232) adolescents, 6% with current reported diagnosis of depression, 8% with higher ACEs and 91% with higher resilience. Family resilience, neighborhood cohesion and caregiver emotional supports were each independently associated with lower odds of reported diagnosis of depression. However, with all resilience factors in the model, only family resilience and neighborhood cohesion (specifically school safety) remained significantly associated with lower odds of reported diagnosis of depression. CONCLUSION(S): In this nationally representative sample, family resilience and neighborhood cohesion were associated with lower odds of a reported diagnosis of depression even with confounding ACEs exposure. These factors may be important targets for future intervention.

Shorter hospitalization trends among children with sickle cell disease.

BACKGROUND: Vaso-occlusive crises (VOC) contribute to frequent hospitalizations among children with sickle cell disease (SCD). The objective of this study was to determine whether length of stay (LOS) has decreased for VOC hospitalizations between 1997 and 2009. PROCEDURE: We analyzed pediatric discharges (aged 0-18) with a primary or secondary diagnosis of SCD with crisis from the Kid's Inpatient Database (years 1997, 2003, and 2009), a nationally representative sample of pediatric hospital discharges. We conducted bivariate and multivariate, sample-weighted linear regression analyses to determine associations between independent variables (patient demographics, hospital characteristics, co-diagnoses, and procedures) and LOS. RESULTS: Both the number (22,661-21,741) and proportion of VOC hospitalizations (0.34-0.29%) among all pediatric hospitalizations marginally decreased between 1997 and 2009 (P < 0.01). Mean LOS decreased from 4.59 to 4.21 days (P < 0.01). For all study years, older age was the only socio-demographic variable associated with longer LOS, controlling for other factors. Between 1997 and 2009, LOS decreased for all age categories, with the largest statistically significant reduction occurring among adolescents (5.69-4.76 days). CONCLUSIONS: Nationally representative hospital data indicate modest but meaningful reductions in LOS for children with VOC over a 12-year period. Adolescents who typically have the greatest disease severity showed the largest reduction in LOS. However, adolescents continue to account for a large proportion of inpatient stays for VOC. These findings illustrate that the adolescent period is a critical time in the lifespan for targeted intervention.

Transition Navigator Intervention Improves Transition Readiness to Adult Care for Youth With Sickle Cell Disease.

OBJECTIVE: Adolescents and young adults (AYA) with sickle cell disease (SCD) experience high rates of acute care utilization and increased morbidity. At this high-risk time, they also face the need to transition from pediatric to adult services, which, if poorly coordinated, adds to heightened morbidity and acute care utilization. The study objective was to characterize the feasibility, acceptability, and short-term efficacy of a protocolized transition navigator (TN) intervention in AYA with SCD. METHODS: We developed a protocolized TN intervention that used ecological assessment and motivational interviewing to assess transition readiness, identify goals, and remove barriers to transition, and to provide disease and pain management education and skills to AYAs with SCD. RESULTS: Ninety-three percent (56/60) of enrolled individuals completed the intervention. Participation in the TN program was associated with significant improvement in mean transition readiness scores (3.58-4.15, P < .0001), disease knowledge scale (8.91-10.13, P < .0001), Adolescent Medication Barriers Scale (40.05-35.39, P = .003) and confidence in both disease (22.5-23.96, P = .048) and pain management (25.07-26.61, P = .003) for youth with SCD. CONCLUSION: The TN intervention was acceptable to youth with SCD, feasible to implement at an urban academic medical center, and addressed barriers to transition identified by the youth. Longer-term assessment is needed to determine if the TN intervention improved successful transfer to and retention in adult care.

Pediatric surgical site infection (SSI) following ambulatory surgery: Incidence, risk factors and patient outcomes.

BACKGROUND: Inpatient surgical site infections (SSIs) cause morbidity in children. The SSI rate among pediatric ambulatory surgery patients is less clear. To fill this gap, we conducted a multiple-institution, retrospective epidemiologic study to identify incidence, risk factors, and outcomes. METHODS: We identified patients aged <22 years with ambulatory visits between October 2010 and September 2015 via electronic queries at 3 medical centers. We performed sample chart reviews to confirm ambulatory surgery and adjudicate SSIs. Weighted Poisson incidence rates were calculated. Separately, we used case-control methodology using multivariate backward logistical regression to assess risk-factor association with SSI. RESULTS: In total, 65,056 patients were identified by queries, and we performed complete chart reviews for 13,795 patients; we identified 45 SSIs following ambulatory surgery. The weighted SSI incidence following pediatric ambulatory surgery was 2.00 SSI per 1,000 ambulatory surgeries (95% confidence interval [CI], 1.37-3.00). Integumentary surgeries had the highest weighted SSI incidence, 3.24 per 1,000 ambulatory surgeries (95% CI, 0.32-12). The following variables carried significantly increased odds of infection: clean contaminated or contaminated wound class compared to clean (odds ratio [OR], 9.8; 95% CI, 2.0-48), other insurance type compared to private (OR, 4.0; 95% CI, 1.6-9.8), and surgery on weekend day compared to weekday (OR, 30; 95% CI, 2.9-315). Of the 45 instances of SSI following pediatric ambulatory surgery, 40% of patients were admitted to the hospital and 36% required a new operative procedure or bedside incision and drainage. CONCLUSIONS: Our findings suggest that morbidity is associated with SSI following ambulatory surgery in children, and we also identified possible targets for intervention.

Pediatric Ambulatory Central Line-Associated Bloodstream Infections.

BACKGROUND: Inpatient pediatric central line-associated bloodstream infections (CLABSIs) cause morbidity and increased health care use. Minimal information exists for ambulatory CLABSIs despite ambulatory central line (CL) use in children. In this study, we identified ambulatory pediatric CLABSI incidence density, risk factors, and outcomes. METHODS: Retrospective cohort with nested case-control study at 5 sites from 2010 through 2015. Electronic queries were used to identify potential cases on the basis of administrative and laboratory data. Chart review was used to confirm ambulatory CL use and adjudicated CLABSIs. Bivariate followed by multivariable backward logistic regression was used to identify ambulatory CLABSI risk factors. RESULTS: Queries identified 4600 potentially at-risk children; 1658 (36%) had ambulatory CLs. In total, 247 (15%) patients experienced 466 ambulatory CLABSIs with an incidence density of 0.97 CLABSIs per 1000 CL days. Incidence density was highest among patients with tunneled externalized catheters versus peripherally inserted central catheters and totally implanted devices: 2.58 CLABSIs per 1000 CL days versus 1.46 vs 0.23, respectively (P < .001). In a multivariable model, clinic visit (odds ratio [OR] 2.8; 95% confidence interval [CI]: 1.4-5.5) and low albumin (OR 2.3; 95% CI: 1.2-4.3) were positively associated with CLABSI, and prophylactic antimicrobial agents for underlying conditions within the preceding 30 days (OR 0.22; 95% CI: 0.12-0.40) and operating room CL placement (OR 0.36; 95% CI: 0.16-0.79) were inversely associated with CLABSI. A total of 396 patients (85%) were hospitalized because of ambulatory CLABSI with an 8-day median length of stay (interquartile range 5-13). CONCLUSIONS: Ambulatory pediatric CLABSI incidence density is appreciable and associated with health care use. CL type, patients with low albumin, prophylactic antimicrobial agents, and placement setting may be targets for reduction efforts.

Central Venous Catheter Salvage in Ambulatory Central Line-Associated Bloodstream Infections.

BACKGROUND: Guidelines for treatment of central line-associated bloodstream infection (CLABSI) recommend removing central venous catheters (CVCs) in many cases. Clinicians must balance these recommendations with the difficulty of obtaining alternate access and subjecting patients to additional procedures. In this study, we evaluated CVC salvage in pediatric patients with ambulatory CLABSI and associated risk factors for treatment failure. METHODS: This study was a secondary analysis of 466 ambulatory CLABSIs in patients <22 years old who presented to 5 pediatric medical centers from 2010 to 2015. We defined attempted CVC salvage as a CVC left in place ≥3 days after a positive blood culture result. Salvage failure was removal of the CVC ≥3 days after CLABSI. Successful salvage was treatment of CLABSI without removal of the CVC. Bivariate and multivariable logistic regression analyses were used to test associations between risk factors and attempted and successful salvage. RESULTS: A total of 460 ambulatory CLABSIs were included in our analysis. CVC salvage was attempted in 379 (82.3%) cases. Underlying diagnosis, CVC type, number of lumens, and absence of candidemia were associated with attempted salvage. Salvage was successful in 287 (75.7%) attempted cases. Underlying diagnosis, CVC type, number of lumens, and absence of candidemia were associated with successful salvage. In patients with malignancy, neutropenia within 30 days before CLABSI was significantly associated with both attempted salvage and successful salvage. CONCLUSIONS: CVC salvage was often attempted and was frequently successful in ambulatory pediatric patients presenting with CLABSI.

Primary care clinicians' knowledge and confidence about newborn screening for sickle cell disease: randomized assessment of educational strategies.

OBJECTIVE: In Massachusetts, primary care clinicians receive and act upon hemoglobinopathy newborn screening results. We assessed clinicians' knowledge, confidence, and practices regarding hemoglobinopathy newborn screening, and the effect of mailed educational materials vs interactive seminar on knowledge and confidence. METHODS: A randomized educational intervention trial was performed at 15 community health sites. Practices were randomized to determine the order in which the educational interventions were administered: mailed educational materials first or interactive seminars on the management of hemoglobinopathy newborn screening results first. Clinicians' demographics, knowledge, confidence, and practices were assessed by a survey. Posttests were administered soon after the intervention. RESULTS: Responses came from 85 of 170 eligible providers (50%). Twenty-nine percent of respondents provided both pretests and posttests. In respondents with paired data, knowledge on a 5-point scale improved by 1.4 +/- 0.4 (mean +/- standard error of the mean, p = .003), while self-efficacy on a 16-point scale increased by 1.3 +/- 0.3, p = .002. There were no significant differences between seminar and mailed-materials groups. CONCLUSIONS: Both educational strategies led to modest improvements in knowledge about newborn screening for hemoglobin disorders. Enhancing knowledge and confidence about newborn screening-related tasks may improve clinicians' capacity to act upon newborn screening results for hemoglobinopathies.

Relationships Between Material Hardship, Resilience, and Health Care Use.

BACKGROUND: Material hardship has been associated with adverse health care use patterns for children with special health care needs (CSHCN). In this study, we assessed if resilience factors were associated with lower emergency department (ED) visits and unmet health care needs and if they buffered associations between material hardship and health care use for CSHCN and children without special health care needs. METHODS: A cross-sectional study using the 2016 National Survey of Children's Health, restricted to low-income participants (<200% federal poverty level). Separately, for CSHCN and children without special health care needs, weighted logistic regression was used to measure associations between material hardship, 2 resilience factors (family resilience and neighborhood cohesion), and 2 measures of use. Moderation was assessed using interaction terms. Mediation was assessed using structural equation models. RESULTS: The sample consisted of 11 543 children (weighted: n = 28 465 581); 26% were CSHCN. Material hardship was associated with higher odds of ED visits and unmet health care needs for all children. Resilience factors were associated with lower odds of unmet health care needs for CSHCN (family resilience adjusted odds ratio: 0.58; 95% confidence interval: 0.36-0.94; neighborhood cohesion adjusted odds ratio: 0.53; 95% confidence interval: 0.32-0.88). For CSHCN, lower material hardship mediated associations between resilience factors and unmet health care needs. Neighborhood cohesion moderated the association between material hardship and ED visits (interaction term: P = .02). CONCLUSIONS: Among low-income CSHCN, resilience factors may buffer the effects of material hardship on health care use. Future research should evaluate how resilience factors can be incorporated into programs to support CSHCN.

A National Measurement Framework to Assess and Improve Sickle Cell Care in 4 US Regions.

OBJECTIVES: Coordinated 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. METHODS: From 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. RESULTS: Administrative 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. CONCLUSIONS: We 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.

Pediatric ambulatory catheter-associated urinary tract infections (CAUTIs): Incidence, risk factors, and patient outcomes.

OBJECTIVE: Catheter-associated urinary tract infections (CAUTIs) occur frequently in pediatric inpatients, and they are associated with increased morbidity and cost. Few studies have investigated ambulatory CAUTIs, despite at-risk children utilizing home urinary catheterization. This retrospective cohort and case-control study determined incidence, risk factors, and outcomes of pediatric patients with ambulatory CAUTI. DESIGN: Broad electronic queries identified potential patients with ambulatory urinary catheters, and direct chart review confirmed catheters and adjudicated whether ambulatory CAUTI occurred. CAUTI definitions included clean intermittent catheterization (CIC). Our matched case-control analysis assessed risk factors. SETTING: Five urban, academic medical centers, part of the New York City Clinical Data Research Network. PATIENTS: Potential patients were age <22 years who were seen between October 2010 and September 2015. RESULTS: In total, 3,598 eligible patients were identified; 359 of these used ambulatory catheterization (representing186,616 ambulatory catheter days). Of these, 63 patients (18%) experienced 95 ambulatory CAUTIs. The overall ambulatory CAUTI incidence was 0.51 infections per 1,000 catheter days (1.35 for indwelling catheters and 0.47 for CIC; incidence rate ratio, 2.88). Patients with nonprivate medical insurance (odds ratio, 2.5; 95% confidence interval, 1.1-6.3) were significantly more likely to have ambulatory CAUTIs in bivariate models but not multivariable models. Also, 45% of ambulatory CAUTI resulted in hospitalization (median duration, 3 days); 5% resulted in intensive care admission; 47% underwent imaging; and 88% were treated with antibiotics. CONCLUSIONS: Pediatric ambulatory CAUTIs occur in 18% of patients with catheters; they are associated with morbidity and healthcare utilization. Ambulatory indwelling catheter CAUTI incidence exceeded national inpatient incidence. Future quality improvement research to reduce these harmful infections is warranted.

Costs of ambulatory pediatric healthcare-associated infections: Central-line-associated bloodstream infection (CLABSIs), catheter-associated urinary tract infection (CAUTIs), and surgical site infections (SSIs).

OBJECTIVE: Ambulatory healthcare-associated infections (HAIs) occur frequently in children and are associated with morbidity. Less is known about ambulatory HAI costs. This study estimated additional costs associated with pediatric ambulatory central-line-associated bloodstream infections (CLABSIs), catheter-associated urinary tract infections (CAUTI), and surgical site infections (SSIs) following ambulatory surgery. DESIGN: Retrospective case-control study. SETTING: Four academic medical centers. PATIENTS: Children aged 0-22 years seen between 2010 and 2015 and at risk for HAI as identified by electronic queries. METHODS: Chart review adjudicated HAIs. Charges were obtained for patients with HAIs and matched controls 30 days before HAI, on the day of, and 30 days after HAI. Charges were converted to costs and 2015 USD. Mixed-effects linear regression was used to estimate the difference-in-differences of HAI case versus control costs in 2 models: unrecorded charge values considered missing and a sensitivity analysis with unrecorded charge considered $0. RESULTS: Our search identified 177 patients with ambulatory CLABSIs, 53 with ambulatory CAUTIs, and 26 with SSIs following ambulatory surgery who were matched with 382, 110, and 75 controls, respectively. Additional cost associated with an ambulatory CLABSI was $5,684 (95% confidence interval [CI], $1,005-$10,362) and $6,502 (95% CI, $2,261-$10,744) in the 2 models; cost associated with a CAUTI was $6,660 (95% CI, $1,055, $12,145) and $2,661 (95% CI, -$431 to $5,753); cost associated with an SSI following ambulatory surgery at 1 institution only was $6,370 (95% CI, $4,022-$8,719). CONCLUSIONS: Ambulatory HAI in pediatric patients are associated with significant additional costs. Further work is needed to reduce ambulatory HAIs.