Pediatric Sickle Cell Disease Clinic Research Articles

Pain screening in youth with sickle cell disease: A quality improvement study.

Youth with sickle cell disease (SCD) face several challenges as they age, including increased pain frequency, duration, and interference. The purpose of this study was to (i) determine the feasibility of routine pain screening; (ii) identify and describe various clinical pain presentations; and (iii) understand preferences/resources related to engaging in integrative health and medicine (IHM) modalities within an outpatient pediatric SCD clinic. During routine outpatient visits, patients aged 8-18 completed measures of pain frequency, duration, and chronic pain risk (Pediatric Pain Screening Tool [PPST]). Participants screening positive for (i) persistent or chronic pain or (ii) medium or high risk for persistent symptoms and disability on the PPST were asked to complete measures of pain interference, pain catastrophizing, and interest in/resources for engaging in IHM modalities. Between March 2022 and May 2023, 104/141 (73.8%) patients who attended at least one outpatient visit were screened. Of these 104 (mean age 12.46, 53.8% female, 63.5% HbSS), 34 (32.7%) reported persistent or chronic pain, and 48 (46.2%) reported medium or high risk for persistent symptoms and disability. Patients completing subsequent pain screening measures reported a mean pain interference T-score of 53.2±8.8 and a mean pain catastrophizing total score of 24.3±10.2. Patients expressed highest interest in music (55.6%) and art therapy (51.9%) and preferred in-person (81.5%) over virtual programming (22.2%). Comprehensive pain screening is feasible within pediatric SCD care. Classifying patients by PPST risk may provide a means of triaging patients to appropriate services to address pain-related psychosocial factors.

Neighborhood Disadvantage and Healthcare Utilization in Pediatric Sickle Cell Patients

Background: Sickle cell disease (SCD) is a complex heritable blood disorder associated with higher acute healthcare utilization further complicated by psychosocial and socioeconomic factors. Other studies have indicated a relationship between neighborhood disadvantage and increased healthcare utilization. We hypothesize that those with greater neighborhood disadvantage will have greater acute healthcare utilization.
 Methods: Data was collected retrospectively from the electronic medical record between 9/30/2021 and 7/11/2023 for patients followed at Riley Hospital for Children’s Pediatric SCD Clinic who completed a psychosocial needs assessment (PAT). Number of emergency department (ED) visits, hospitalizations, and missed clinic visits were collected from the time the PAT was completed up to 1 year after (or the end of the data collection period). Data regarding area deprivation index (ADI) and childhood opportunity index (COI) were collected utilizing patient home address. Descriptive statistics were performed on all data above, as well as t-tests and chi square tests for univariate analyses.
 Results: 142 patients completed a PAT during the study period. 88% had a primary care provider documented, and 73% had public insurance. The median state and national ADI (6, 76.5) and COI (2, 2) demonstrated greater neighborhood disadvantage and less childhood opportunity. There was no statistically significant difference between ADI or COI and number of ED encounters or hospitalizations. However, patients who missed 1 or more SCD visits had a higher median state ADI than those who did not miss a visit (7.5 vs 5, p=0.002). Median state COI was also lower in this group than those who did not miss a visit (1 vs 2, p=0.001).
 Conclusion: Those with higher ADI and lower COI could benefit from more directed support to improve access to preventative care visits. Further analysis accounting for comorbidities (seizures, asthma, depression) could disambiguate a relationship between ADI or COI and acute care utilization.

Impact of Neighborhood Opportunity on Missed Clinic Visits and Acute Care Utilization in Individuals with Sickle Cell Disease

Background: Sickle cell disease (SCD) is a complex inherited blood disorder associated with high acute healthcare utilization, further complicated by psychosocial and socioeconomic factors. Social determinants of health (SDOH) have a known impact on healthcare access and health outcomes, particularly in minoritized communities. The Area Deprivation Index (ADI) is a composite measure of neighborhood socioeconomic disadvantage. The Childhood Opportunity Index (COI) is a validated approach for estimating multi-dimensional SDOH leading to inequitable distribution of opportunity across neighborhoods. Both have been associated with acute care utilization in pediatric populations other than SCD. We hypothesize that ADI and COI will be associated with acute care utilization and care access in SCD, as well. Methods: Data were collected retrospectively from the electronic medical record between September 2021 and July 2023 for patients followed at a Midwestern Pediatric SCD Clinic who completed a Psychosocial Assessment Tool (PAT) at an annual comprehensive visit. The PAT is a validated quantitative caregiver survey used to screen for psychosocial risk factors and resiliency, with subcategories such as social support, child problems, structure/resources, and family beliefs. Number of emergency department (ED) visits, hospitalizations, and missed clinic visits were collected from the time the PAT was completed up to 1 year after (or the end of the data collection period). The area deprivation index (ADI) and childhood opportunity index (COI) were determined utilizing census block data for the patient at the time the PAT was completed. Descriptive statistics were performed on all data above, as well as t-tests and chi square tests for univariate analyses. Results: One hundred seventy-four unique patients completed a PAT during the study period. The median age of the child with SCD was 11.5 years (range 4-22 years), with the majority having Hb SS disease (61%). Eighty-nine percent had a primary care provider documented and 75% had public insurance. The median state ADI (6) and COI (2) for the cohort demonstrated significant neighborhood disadvantage/SDOH for all patients (See Figure). Ninety-two unique patients had 239 ED encounters. Fifty-eight unique patients accounted for 149 hospitalizations. Forty-seven percent of those hospitalized had a PAT in the Targeted or Clinical range (i.e. increased psychosocial stressors) compared to 30% of those who had not been hospitalized ( p=0.04). There was no statistically significant difference between ADI or COI and number of ED encounters or hospitalizations. However, patients who missed 1 or more SCD visits (n=79) had a higher median state ADI than those who did not miss a visit (8 vs 6, p=0.009). Median state COI was also lower in this group than those who did not miss a visit (1 vs 2, p=0.008). Forty-eight percent of those who missed 1 or more SCD visit had a PAT in the Targeted or Clinical category versus 27% of those who did not miss any ( p=0.009). Conclusion: While those with significant SDOH and neighborhood disadvantage did not have higher rates of acute care utilization in this cohort, there was a significant contribution to missed preventative SCD care visits, further emphasizing the need for psychosocial support and resources as essential elements to SCD programs, as well as alternative forms of care delivery, such as telemedicine, to improve access to SCD specialists. Further studies are needed to understand the impact comprehensive resources such as these may have on care burden, as well as disease outcomes.

Implementing a Structured Health Care Transition (HCT) Process for Sickle Cell Disease Using Quality Improvement: Results from the Sickle Cell Trevor Thompson Transition Project HCT Learning Collaborative

Ensuring consistent healthcare during transition is challenging for sickle cell disease (SCD). Emerging adults with SCD (EASCD) experience triple the mortality rates and double the acute care utilization rates compared to younger and older ages. The transition period is also plagued by reduced quality of life (QOL) with increased missed school days per year and worse standardized test scores. The Sickle Cell Trevor Thompson Transition (ST3P-UP) study aimed to improve acute care utilization and QOL for EASCD by standardizing the transition process while engaging the community as an equal partner. A prior regional stakeholder needs assessment identified care standardization, establishing individual emergency care plans (ECPs), and community engagement as critical success factors for improved access to and engagement in care during transition. The ST3P-UP SCD health hare transition (HCT) learning collaborative engaged 14 pediatric and adult paired sites that included a partner community-based organization (CBO) using the concept of a site “Triad” as the critical framework of each sites transition team. The collaborative aim statement established at a kickoff meeting was “ to improve the health of EASCD by implementing an education-based transition program in pediatric and adult SCD clinics to be accomplished by 24 months, using the Model for Improvement and the Got Transition Six Core Elements of Transition (6CE) model. ” Using the 6CE framework established by Got Transition® and the Model for Improvement QI methodology, we established a SCD HCT Process Measurement Tool (PMT) and 2 outcome measures for the transition learning collaborative namely: transition readiness assessment (RA), and an individualized emergency care plan (ECP). Monthly virtual meetings provided coaching on HCT recommendations, use of Plan-Do-Study-Act cycles, and practical guidance on implementing the 6CE. Sites were given a change package that included provider and patient education materials, resources to establish and strengthen a QI team, transition billing and coding resources etc.) to improve their transition processes. Implementation of 6CE was measured across 14 clinical sites, each comprised of both pediatric and adult clinics, who cared for 1625 EASCD aged 16-25. Clinical programs varied: 12 urban, 2 rural; 12 academic, 2 non-academic; 6 small, 8 large. The HCT-PMT assessed 6CE implementation adherence using iterative QI strategies at baseline (2018) and every six months thereafter for 54mo (2022). Pre-post results were compared for the overall group and by practice size. All 14 sites made substantial progress towards implementing a structured HCT process within 24 months. Overall HCT-PMT scores improved by 402% by the end of the 54mo collaborative (19.4 ± 14.1 to 97.4 ± 5.2). PMT scores improved significantly by 312% (24 to 99) for pediatrics and 539% (15 to 96 for adult sites. Pediatric PMT scores were higher at each assessment interval vs. adult scores, however adult sites achieved a higher degree of improvement. Smaller sites achieved a higher degree of improvement vs. large sites. Large site PMT scores improved 362% (21 to 97); small site PMT scores improved 444% (18 to 98). Additionally, sites demonstrated substantial improvements with having current ECPs for EASCD annually (completed and reviewed within the past year). At baseline, only 20% of pediatric and 3% of adult EASCD had a current ECP; only 38% of pediatric and 20% of adult EASCD had a current RA. Run charts demonstrated significant improvement with implementing the ECP measure. Pediatric sites had significant and sustained improvement in adherence to the ECP measure for >6mo. Adult sites started with a lower baseline score. Although they met their goal of showing process capability, they have not yet demonstrated sustained improvement. By Dec. 2022, site adherence with current ECP improved to 84% and 70% for pediatric and adult sites respectively. Adherence with current RA also improved to 76% and 70% for pediatric and adult sites respectively. All 14 sites showed significant improvement with implementing a structured HCT process aligned with the 6CE using QI confirming its feasibility in establishing a standardized and measurable transition process that can be applied to various chronic conditions.

Associations between Sickle Cell Disease, Pica and Enuresis

Introduction: Sickle cell disease (SCD) is a genetic disorder that impacts the shape and functional capacity of red blood cells. Beyond altered cerebral perfusion, individuals with SCD have higher rates of neurodevelopmental disorders (NDDs), including attention deficit hyperactivity disorder (ADHD), intellectual disability, and specific learning disabilities. Past research has shown that in adolescent populations with either SCD or NDDs, pica, the act of eating non-food items, and enuresis, or involuntary urination, commonly occur. Both pica and enuresis are associated with biopsychosocial issues in typically developing children. However, the etiology of both of these disorders, as well as the confluence of NDDs and SCD on their occurrence, remains unclear. Hypothesis: The objective of this study was to determine the rates of pica and enuresis in a local pediatric SCD clinic and determine if these conditions present differently and/or have different associations in SCD than in the general population. We hypothesized that NDDs would serve as risk factors for both pica and enuresis with both conditions being associated with increased sickle cell severity. Methods: To test these hypotheses, we performed a retrospective chart review on 275 SCD patients. Using Epic, patients who at point visited either Kennedy Krieger Institute or John Hopkins Medical Center for SCD and were in an existing database were queried. Patients in the database ranged from 1 to 17 years old and were predominantly Black, non-Hispanic individuals. Primary keywords searched included: “enuresis, pica, bedwetting, diurnal, nocturnal, eating, non-food, peeing, urination, incontinence.” Results: Pica was reported in 9% (Table 1), while enuresis was reported in 27% of patient charts reviewed (Table 2). Both enuresis (Chi-squared test, p = 0.0050) and pica (Chi-squared test, p = 0.0190) were significantly linked to NDD occurrence, with pica being nearly three times as likely to occur alongside NDDs and/or enuresis. Notably, SCD patients with pica most commonly ate paper or wood, different from the general population with pica primarily eating rocks or dirt. Through the Wilcoxon rank sum test, we also noted no associations between pica and median hemoglobin (p=0.9572), ferritin (p=0.6209), iron serum (p=0.9500), total iron-binding capacity (p=0.1598), iron saturation (p=0.6292), or transferrin (p=0.1765) levels. Beyond this, our data also validate prior results demonstrating that enuresis is associated with both SCD type and hydroxyurea usage. There were no significant differences in rates of enuresis between males and females, unlike what is seen in the general population. Sleep apnea was associated with enuresis, at a higher rate than in the general population (Chi-squared post-hoc residual test, p = 0.0004). Patients with SCD and enuresis had higher mean reticulocyte counts and lower mean hemoglobin levels than patients with SCD without enuresis, consistent with increased hemolysis and worsening SCD severity. Conclusion: These results suggest etiologies for pica and enuresis in SCD patients may differ from the general population. As pica and enuresis commonly occur together with NDDs, there is a clear need for increased standardized screening for these conditions in clinics. Understanding why the risk factors for these conditions in SCD differ from the general population requires further investigation into their respective mechanisms of disease.

Hemolysis scavenger proteins and renal function marker in children with sickle cell disease at steady state: A cross-sectional study.

Hemolysis is a fundamental feature of sickle cell disease (SCD) contributing to the vaso-occlusive crisis of patients. The objectives of the study were to assess the link between hemolysis proteins and hematological parameters, and to validate cystatin C(CYS C) as a potent renal marker in diagnoising SCD. Here, a cross-sectional study carried out at the pediatric SCD clinic of the Komfo Anokye Teaching Hospital comprised 90 SCDchildren (HbSC, HbSF, and HbSS). ANOVA, t-test, and Spearman's rank correlation analysis were done. Elevated proteins levels were compared to standard values; alpha-1 microglobulin (A1M) (1.8-65 µg/L), CYS C (0.1-4.5 µmol/L), and haemopexin(HPX) (500-1500 µg/mL). The mean (standard deviation) age of participants was 9.830 (±0.3217) years, and 46% of them were males. From simple descriptive analysis, we observed that all but one patient had their HPX level below the reference range (<500 µg/mL). Here, A1M levels were shown to be within the appropriate reference range for all the patients except few patients. CYS C levels were also all within the required reference values. A Spearman's rank correlation test between full blood count and HPX generally suggested a weak but positive correlation; RBC (coef. = 0.2448;p = 0.0248), HGB (coef. = 0.2310;p = 0.030), hematocrit(coef. = 0.2509; p = 0.020), and platelet(coef. = 0.1545;p = 0.160). Mean corpuscular volume(coef. = -0.5645;p = 0.610) had a stronger but negative correlation with HPX. This study depicts a positive and stronger association between CYS C and HPX levels (coef. = 0.9996;p < 0.0001), validating the use of CYS C as a useful marker of renal function in persons with SCDs. In the present study, we show that A1M levels were normal for most of the patients, hence CYS C levels are not alarming in this study. Further, there exists a correlation between hemolysis scavenger proteins and hematological parameters.

Intraindividual pain variability metrics for youth with sickle cell disease: Relations to health outcomes.

While the majority of pediatric sickle cell disease (SCD) research has used mean pain intensity as the only pain metric, recent evidence suggests this metric alone is inadequate in describing the intraindividual variability in SCD pain experiences and subsequent impact. There is limited information on other intraindividual pain metrics in youth with SCD, or how they relate to health outcomes in this population. The aims of this study were to describe differing patterns of intraindividual pain metrics derived from ecological momentary assessments (EMAs) of youth with SCD and to characterize the unique relationships between these metrics and health outcomes. Eighty-eight youth with SCD, aged 8-17 (mean age=11.6), were recruited from three regional pediatric SCD clinics in the United States. At baseline, youth and their guardians reported on demographic and disease information. Then youth completed twice daily EMAs for up to 4weeks. Pain metrics derived from EMA data were calculated including mean daily pain intensity (DP), SD-DP (standard deviation of DP), proportion of pain days (PPD), and 90th percentile of DP (p90). Pearson correlations were calculated between pain metrics and health outcomes. High DP and SD-DP were correlated with more anxiety symptoms, while high SD-DP and p90 were correlated with more depression symptoms. High SD-DP was correlated with low self-esteem, and high DP and PPD were correlated with low sickle cell self-efficacy. For healthcare utilization due to pain, high p90 was correlated with more emergency department visits, while high DP, p90, and PPD were correlated with more healthcare contacts. There are distinct associations between pain variability metrics beyond DP and health outcomes. Collectively, the patterns of associations suggest the utility of these pain metrics for determining risk in relation to specific health outcomes for youth with SCD.

P-017: POINT-OF-CARE DIAGNOSTIC FOR QUANTIFICATION OF FOETAL HAEMOGLOBIN (HB F) LEVELS IN MONITORING HYDROXYUREA THERAPY FOR CHILDREN WITH SICKLE CELL DISEASE (SCD) IN GHANA

Purpose: Sickle cell disease (SCD) potentially causes numerous acute and chronic complications across the lifespan. The majority of patients with SCD live in sub-Saharan Africa and 1 in 50 Ghanaian newborns are affected. Foetal hemoglobin (Hb F) level is a known modulator of the severity of SCD. High levels of Hb F inhibit sickling and correlate with reduced disease morbidity and mortality. Hb F monitoring is also used to assess adherence and response to disease modifying (hydroxyurea) therapy. Standard hemoglobin electrophoretic techniques are unable to quantify Hb F. In resource-limited settings like Ghana, the use of high performance liquid chromatography (HPLC) for Hb F quantification is often challenging due to high cost and unavailability of laboratory equipment and skilled technicians. We report on the performance of a low-cost, point-of-care, microchip based cellulose acetate electrophoresis “Gazelle” compared to HPLC for Hb F quantification in Ghanaian children with SCD. GazelleTM is a fast (<8 minutes), easy-to-use test which can be performed by minimally trained personnel using only a finger-prick volume of blood. Materials and methods: The study was conducted at the paediatric SCD Clinic in Korle Bu Teaching Hospital, Accra, Ghana. Children (age ≥ 1 year to 16 years) known to have sickle cell disease who are on hydroxyurea therapy, and followed at the paediatric sickle cell clinic were enrolled in the study. The study is still ongoing. Results: A total of 110 children were included in the analysis. Gazelle showed a correlation of 0.93 when compared to HPLC (Figure 1). Conclusion: Haemoglobin variants are quantified by Gazelle offering the ability to monitor disease modifying therapy in SCD such as hydroxyurea, where quantification of HbF levels is the key to assessing treatment adherence and determining response to therapy. Gazelle has the potential to be utilized as a POC test for quantification of foetal haemoglobin (Hb F) levels in monitoring hydroxyurea therapy for children with SCD.Hb Quantification Performance of Gazelle compared to HPLC C. SEGBEFIA declares a conflict of interest: Consultancy, Expert: Novartis - Advisory Board / Steering Committee Member Global Blood Therapeutics - DSMB Member P. THOTA declares a conflict of interest: Other: Employee of Hemex Health

Functional Connectivity in Pediatric Sickle Cell Disease

Children with sickle cell disease (SCD) have a high risk of developing cerebrovascular complications, such as stroke and silent cerebral infarction (SCI). SCI is associated with increased risk of future infarction as well as neurocognitive deficits related to brain injury location and size; however, neurocognitive impairment may occur in the absence of neuroimaging abnormalities. Resting state functional magnetic resonance imaging (RS-fMRI) measures blood oxygen level dependent (BOLD) signal during rest to evaluate functional connectivity between brain regions. Functional connectivity is the temporal correlation between the BOLD signal in spatially distant brain regions, which reflects synchronous activity. For this study, we hypothesized that participants with SCI would have lower functional connectivity than participants without SCI and that specific resting state networks would be associated with specific cognitive tests in SCD and control participants.We recruited 26 participants from the local pediatric hematology and SCD clinics. Children with SCD were included in the study if they had a SCD diagnosis confirmed by laboratory studies and no known prior history of overt stroke or seizure. We obtained clinical history, laboratory tests, neuropsychological testing scores, and RS-fMRI scans in 21 participants with SCD and 5 control participants without SCD, 2 of who had sickle cell trait. Each participant received a resting state functional connectivity scan using a 3T MR scanner. Participants were asked to remain still, stay awake, and keep their eyes open during the resting state scan. The MRI study protocol included a BOLD scan and a T1-weighted magnetization-prepared rapid gradient-echo sequence (MPRAGE) with a scan duration of 8 minutes. We performed standard image pre-processing steps, including realignment, normalization to Montreal Neurologic Institute (MNI) standard brain space via MPRAGE image, spatial smoothing, and slice timing correction.Table 1 shows the characteristics of the study participants. Eight participants with SCD had SCI diagnosed as an incidental finding during the study. The average connectivity within 7 resting state networks (control, default mode, dorsal attention, limbic, salience ventral attention, somato-motor, and visual networks) was compared between all (both SCD and control) participants with SCI and without SCI (Table 2). Participants with SCI had significantly lower functional connectivity in the control network (p = 0.0231, 95% CI: 0.073- 0.144) in comparison to participants without SCI. We also analyzed the relationship between 4 clinical variables and functional connectivity within each resting state network for all of the participants, with and without SCD. After adjusting for age and sex, there was a significant association between 3 resting state networks (control, salience ventral attention, and visual networks) and both hemoglobin and hematocrit (Table 3). There was a significant association between functional connectivity in the visual network and hemoglobin when adjusting for age and sex among just the participants with SCD (p = 0.045, 95% CI: 0.001-0.082). We analyzed the relationship between functional connectivity within each resting state network and neuropsychological test scores and found multiple significant associations between control, default mode, dorsal attention, salience ventral attention, and visual networks and attention/executive functioning test scores for all participants as well as just participants with SCD.Our findings suggest that children with SCD and SCI have decreased functional connectivity in the control network in comparison to children with and without SCD without SCI, which may indicate abnormalities in brain regions underlying executive dysfunction. Our data also established a relationship between the degree of anemia and functional connectivity, showing increased functional connectivity in the control, salience ventral attention, and visual network in participants with higher hemoglobin and hematocrit levels. Neuropsychological data shows that select test scores are associated with changes in functional connectivity in resting state networks primarily involved with attention and executive functioning. This research supports the utility of RS-fMRI as an adjunct analysis for investigating neurocognitive abnormalities in pediatric SCD. [Display omitted] DisclosuresLanzkron: Novartis: Research Funding; Imara: Research Funding; CSL Behring: Research Funding; Bluebird Bio: Consultancy; Shire: Research Funding; Novo Nordisk: Consultancy; Pfizer: Current holder of individual stocks in a privately-held company; Teva: Current holder of individual stocks in a privately-held company; GBT: Research Funding. Mirro: NOUS Imaging: Current Employment, Current holder of stock options in a privately-held company. Fields: Global Blood Therapeutics: Consultancy; Proclara Biosciences: Current equity holder in publicly-traded company. Lance: Novartis: Other: participated in research advisory board in 2020.

Pediatric sickle cell pain-sleep relationships: The roles of positive and negative affect.

The current study investigated the role of affect in the daily sleep quality-pain severity relationship in youth with sickle cell disease (SCD). Previous investigations have not allowed researchers to examine whether positive and negative affect work differentially in their relations with pediatric SCD pain, sleep, and the sleep-pain relationship. The current study focused on examining the possible mediating and moderating roles positive and negative affect have in the sleep-pain relationship for youth with SCD. Eighty-eight youth with SCD (aged 8-17 years), and their guardians were recruited from three regional pediatric SCD clinics. Youth completed a twice daily ecological momentary assessment, where they reported on their daily pain severity, sleep quality, positive affect and negative affect. Multilevel models were calculated to examine the relationship between negative affect, positive affect, and the sleep-pain relationship. Multilevel mediation analyses indicated that low positive affect mediated the daily cyclic relationships between poor sleep and high pain. Moderation analyses indicated that high negative affect strengthened the relationship between high pain severity and poor sleep quality that night. Further research is needed to examine the differential roles of positive and negative affect in other pain and adult SCD populations. Research examining the mechanisms by which positive and negative affect may influence the sleep-pain relationship is needed to inform future interventions to improve sleep and pain in the pediatric SCD population. (PsycInfo Database Record (c) 2021 APA, all rights reserved).

Pediatric Pain Screening Identifies Youth at Risk for Chronic Pain in Sickle Cell Disease

▪Introduction: Chronic pain related to pediatric sickle cell disease (SCD) contributes to high healthcare use and poor quality of life. Factors that contribute to the transition from acute to chronic pain in SCD remain largely unknown. Thus, chronic SCD pain remains a challenging problem that is difficult to manage due to the complex interaction of biological, psychological, and social determinants that contribute to its onset and maintenance. Common sequelae of chronic SCD pain in children and adolescents include functional impairment, frequent school absences, elevated depressive symptoms, and high levels of anxiety or catastrophic thinking. Early identification of youth who are at risk of developing chronic SCD pain is needed to help prevent the onset and exacerbation of chronic pain. This study aimed to identify youth at increased risk for chronic SCD pain and its sequelae through the integration of a brief psychosocial pain screening tool. Children and adolescents with SCD identified as “high risk” on the pain screening tool were expected to demonstrate a psychosocial profile consistent with chronic SCD pain, including increased pain frequency and duration, high levels of functional impairment, elevated anxiety and depressive symptoms, and reduced quality of life.Methods: Children and adolescents with SCD presenting to a pediatric SCD clinic completed the Pediatric Pain Screening Tool, a brief 9-item self-report questionnaire developed for rapid identification of risk in youth with pain complaints. Youth also completed a battery of surveys about their pain characteristics (pain frequency and duration), pain burden (SCD Pain Burden Interview for Youth), functional impairment (PROMIS Pain Interference), anxiety (Pain Catastrophizing Scale-Child; Fear of Pain Questionnaire), depressive symptoms (Children's Depression Inventory -2), and quality of life (Pediatric Quality of Life-SCD Module).Results: Youth (n=45) were on average 13.5 years old (SD = 4.4), 89% Black or African American, 80% HbSS, 85.5% prescribed hydroxyurea, and 16.7% receiving chronic transfusions. Youth were classified into Low (n=15, 33.3%), Medium (n=15, 33.3%), and High (n=15, 33.3%) risk groups based on pain screening. Risk groups did not significantly differ by age, race, hemoglobin type, or treatment with hydroxyurea or chronic transfusions. Multivariate Analysis of Variance (MANOVA) showed significant risk group differences on pain characteristics, pain burden, functional impairment, anxiety and depressive symptoms, and quality of life (Wilks' λ = 0.13, F16, 54 = 6.02, p < .001). Post-hoc univariate tests revealed that youth in the High Risk group had significantly higher pain frequency (M=15.2 days/month, SD= 2.2) and duration (over 1 year) compared to the Low (M=1.8, SD= 2.3; duration 1.2 months) and Medium (M=7.6, SD= 2.3; duration 2.5 months) Risk groups. The High Risk group also had significantly higher pain burden, functional impairment, catastrophic thinking, depressive symptoms, and poorer quality of health compared to the Low and Medium Risk groups. The High Risk group had significantly higher levels of fear of pain relative to the Low Risk group, but there was no significant difference in fear of pain between the Medium and High Risk groups.Conclusions: Brief psychosocial pain screening may be a helpful tool to support the early identification of youth at risk for chronic SCD pain. Youth classified as High Risk based on pain screening demonstrated a psychosocial profile consistent with other pediatric chronic pain conditions. Additionally, the High Risk group reported high pain frequency and duration that is consistent with the primary diagnostic criteria for chronic SCD pain (i.e., ≥15 days of pain per month, lasting ≥ 6 months). Further validation with a larger sample of children and adolescents with SCD is needed to support the utility of the Pediatric Pain Screening Tool in identifying youth at risk of transitioning from acute to chronic pain.Supported by NCATS Award UL1TR000454. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. DisclosuresNo relevant conflicts of interest to declare.

Health Care Utilization Trajectories and Associated Factors for Transitioning Adolescents/Young Adults with Sickle Cell Disease

BackgroundLimited research had explored health care utilization (HCU) in transitioning adolescent/young adults (AYAs) with sickle cell disease (SCD). Major limitations of the current studies include the use of cross-sectional designs and lack of longitudinal exploration of HCU trajectories and the factors that might contribute to the increased HCU. The purposes of this longitudinal database study were to describe HCU trajectories of transitioning AYAs with SCD and examine factors associated with these trajectories.MethodsAYAs (12-27 years) at one urban center with a comprehensive SCD program were included if they had SCD (SS, SC, Sβ+, Sβ° genotypes) and at least two pediatric SCD clinic encounters between 01/01/1989 and 07/30/2015. We used latent class growth modeling to identify HCU (clinic, Emergency department (ED), hospitalizations) trajectories and ordinal logistic regression to examine factors associated with HCU, including individual (demographic; disease measures including severity, comorbidities, and medications), contextual (distance to clinic), and transfer-related factors (transfer age and continuity of care). We also calculated age-related ED reliance (EDR)scores. Significance was set at p<0.05.Results339 AYAs (55% male; 96% black; mean age=24.5 years, SD=5.38) had 10848 clinic encounters. Of these, 240 AYAs (71%) had 3840 hospitalizations and 197 (58%) had 3152 ED encounters. Low, medium, and high utilization groups were identified, with the majority of AYAs having low clinic (n=206, 60.77%), low hospital (n=171, 71.25%), and/or low ED (n=146, 74.11%) utilization trajectories (Figures 1-3). Mean age-related EDR scores were < 0.33 and increased with age (figure 4). AYAs transferred at mean age 19 and the majority had at least one additional adult clinic encounter.Factors significantly associated with higher clinic utilization trajectories included iron overload (OR=36.6; p <0.0001; 95% CI= 11.657-114.979), lower extremity ulcers (OR=8.5; p= 0.0174; 95% CI=1.457- 49.906), cerebral infarct/vasculopathy (OR=7.2; p <0.0001; 95% CI= 3.599-14.51), hydroxyurea (OR=3.2; p= 0.0007; 95% CI= 1.626-6.198), attention deficit disorders (OR=11.5; p= 0.0003; 95% CI= 3.033-43.505), and distance to clinic (OR=0.98; p <0.0001; 95%CI= 0.970-0.989).Factors significantly associated with higher hospitalization trajecotries included chronic pain (OR=3.3; p <0.0001; 95% CI=1.916-5.522), long-acting narcotics (OR=2.3; p= 0.0254; 95% CI= 1.11- 4.933), hydroxyurea (OR= 1.9; p= 0.0343; 95% CI= 1.047-3.341), intellectual disabilities (OR=7.96; p= 0.0129; 95% CI=1.551-40.858), and depression (OR=2.2; p= 0.0180; 95% CI= 1.146- 4.271).Factors significantly associated with higher ED utilization trajectories included hip/shoulder avascular necrosis (OR=28.9; p <.0001; 95% CI=5.921-140.731), long-acting narcotics (OR=32.5; p <0.0001; 95% CI=7.717-136.927), major depression (OR=22.5; p <0.0001; 95% CI= 4.952-102.591), and distance to clinic (OR=0.95; p <0.0001; 95% CI=0.923-0.974).SCD genotype and SCD severity scores were not associated with group membership in any of the HCU trajectories.ConclusionsDifferent factors were associated with different HCU trajectories, underscoring the complexity of HCU during transition. Understanding the factors associated with HCU might help identify AYAs with SCD at risk for high utilization. [Display omitted] DisclosuresShah:Alexion: Other: Speaker; Novartis: Other: Speaker.

Maintenance of a High Influenza Vaccination Rate and Improvement in Health Outcomes in a Pediatric Sickle Cell Disease Clinic.

Children with sickle cell disease (SCD) are at high-risk of complications from influenza and should receive an influenza vaccination seasonally. Despite this recommendation, vaccination rates remain suboptimal. Boston Medical Center (BMC) previously achieved high influenza vaccination rates among its pediatric patients with SCD. The purpose of this study was to determine whether this high vaccination rate has been maintained and whether it has influenced outcome measures. A retrospective chart review was conducted in the hematology clinic at an urban, academic medical center. Fisher's exact test and the independent samples t test were used to determine if there were any significant differences in characteristics between patients with influenza and patients without influenza, as well as between vaccinated and unvaccinated patients. Influenza vaccination rate, influenza-related hospitalization rate, and influenza-positive rate were collected and compared with reported rates. Data from 124 pediatric patients with SCD were examined. The influenza vaccination rate for pediatric patients with SCD at BMC (90.32%) was higher than previous studies that were not conducted at BMC, while BMC's influenza-related hospitalization rate (0) and influenza-positive rate (4.84%) were lower than other studies. Subjects who contracted influenza were younger than those who did not (4.67 vs. 10.03 y, P=0.005). BMC has maintained a high influenza vaccination rate among pediatric patients with SCD. BMC's vaccination strategy has been successful at improving outcome measures including rates of influenza and influenza hospitalizations without requiring additional staff. Such efforts should be replicated at other centers.

Feasibility and acceptability of early infant screening for sickle cell disease in Lagos, Nigeria-A pilot study.

In Nigeria, about 150000 babies are born annually with sickle cell disease (SCD), and this figure has been estimated to increase by 100% by the year 2050 without effective and sustainable control strategies. Despite the high prevalence, newborn screening for SCD which allows for early prophylactic treatment, education of parents/guardians and comprehensive management is not yet available. This study explored a strategy for screening in early infancy during the first and second immunization visits, determined the prevalence, feasibility and acceptability of early infant screening for SCD and the evaluation of the HemoTypeSC diagnostic test as compared to the high-performance liquid chromatography (HPLC) gold standard. A cross-sectional study was conducted in two selected primary health care centres in Somolu local government area (LGA) in Lagos, Nigeria. Two hundred and ninety-one mother-infant pairs who presented for the first or second immunization visit were consecutively enrolled in the study following written informed consent. The haemoglobin genotype of mother-infant pairs was determined using the HemoTypeSC rapid test kit. Confirmation of the infants' Hb genotype was done with HPLC. Data were analysed with SPSS version 22. Validity and Predictive value of HemotypeSC rapid screening test were also calculated. Infant screening for SCD was acceptable to 86% of mothers presenting to the immunization clinics. The prevalence of SCD among the infant cohort was 0.8%. The infants diagnosed with SCD were immediately enrolled in the paediatric SCD clinic for disease-specific care. The HemoTypeSC test had 100% sensitivity and specificity for sickle cell disease in early infancy compared to HPLC. This study affirms that it is feasible and acceptable for mothers to implement a SCD screening intervention program in early infancy in Lagos State. The study also demonstrates the utility of the HemotypeSC rapid testing for ease and reduced cost of screening infants for SCD.

Food Insecurity Is a Common Problem Affecting Dietary Quality in a Clinic-Based Pediatric Sickle Cell Disease Sample

Background: Understanding the effect of food insecurity on diet of children with sickle cell disease (SCD) is necessary to improve child dietary quality and optimize overall health. Objective: To characterize the association between food insecurity and dietary quality of children with SCD. Methods: A cross-sectional study of 100 youth aged 1-20 years with SCD and their parents was conducted from January-June 2018 at a pediatric SCD center at an academic medical center in New York City (NYC). Participating SCD patients were recruited from a convenient clinic-based sample. Food insecurity over the prior year, defined as a disruption in eating patterns due to lack of resources, was reported by adults ≥ 18 years using the validated 18-item U.S. Department of Agriculture Food Security Survey. Dietary quality in the prior 30 days was parent-reported for children &amp;lt;11 years and self-reported for children ≥11 years using a 15-item food frequency questionnaire adapted from the validated Dietary Screener Questionnaire (DSQ).1 Good dietary quality was defined as higher daily intake frequency of fruit, vegetable, protein and dairy and a lower frequency of pizza. Patient characteristics measured included SCD type, transfusion history, emergency room visits, housing hardship (defined as ≥2 moves in the prior year), and enrollment in federal food assistance programs (Supplemental Nutrition Assistance Program (SNAP), Special Supplemental Nutrition Program for Women, Infants, and Children (WIC), and the National School Lunch program). Multiple regression models adjusted for a priori covariates associated with food insecurity and nutrition: age, sex, and housing hardship. Results: Of the sample, 55% were male and 50% Caribbean Hispanic (Table). Overall, 85% of children had HbSS, 13% HbSC, and 3% HbSβ0. Mean hemoglobin level was 9.0 ± 1.7 mg/dL, including 11% on chronic transfusion therapy. Participants had a median of 2.2 emergency room visits (interquartile range (IQR): 3) over the prior year. Among families, 80% lived in lower-income neighborhoods in northern Manhattan and the Bronx, 22% had housing hardship and 80% received at least one federal food assistance benefit. Among all families, 30% reported food insecurity. Children with and without food insecurity were not significantly different by age (median (IQR): 14 (7) vs. 10 (10) years, p=0.6), male sex (53% vs. 55%, p=0.9), and housing hardship (13% vs. 24%, p=0.2). Food-insecure children had significantly lower mean daily frequency of fruit (0.5 ±0.2 vs. 0.7 ±0.6, p=0.04) and vegetable intake (0.3 ±0.2 vs. 0.5 ±0.5, p=0.04) compared to those without food insecurity (Figure). Similarly, among the 80% of children receiving federal food assistance, those with food insecurity had lower mean daily frequency of vegetable intake compared to children without food insecurity (0.3 ±0.4 vs. 0.5 ±0.3 respectively, p=0.02). Food insecurity was significantly associated with increased frequency of pizza intake among all children (β=0.2 ±0.1, 95% confidence interval 0.01-0.29). Conclusions: In this clinic-based urban sample of children with SCD, 30% experienced food insecurity and 1 in 5 experienced housing hardship. This was higher than reported NYC-wide food insecurity rate of 13% and the Bronx rate of 26% in 2018.2 Food insecurity was significantly associated with a poorer diet quality - lower mean daily frequency of fruit and vegetable intake, even among those receiving a federal food assistance benefit, and increased frequency of pizza intake. Screening for food insecurity and other health-related social needs in pediatric SCD clinic may identify many families needing assistance for basic food and housing needs, and may inform efforts to improve dietary quality and overall health in these children. This research was conducted due to support from a NIH Loan Repayment Program for Health Disparities Research Award (NIMHD), Dr. Fernández, PI.

Feasibility and preliminary outcomes of an integrated pediatric sickle cell disease and pulmonary care clinic for children with sickle cell disease.

In children with sickle cell disease (SCD), comorbid asthma is associated with increased disease severity and morbidity, but it remains underdiagnosed and optimal management paradigms are not well defined. The purpose of this study was to determine the feasibility and preliminary outcomes of an integrated pediatric SCD and pulmonary care clinic in children with SCD. We implemented a pre-post quality improvement (QI) project in our pediatric hematology clinic between 2017 and 2019. Guided by the chronic care model, patients who were ages 2-18years, diagnosed with SCD and suspected pulmonary comorbidities, received care in an interdisciplinary clinic. We examined feasibility and compared clinical outcomes to 24months prior (2015-2017) to the implementation of the integrated care model. Twenty-four patients were included in the QI project: 88% (n=21) received pulmonary function testing, 92% (n=22) were diagnosed with asthma, and 33% (n=8) with obstructive sleep apnea. Adherence to pulmonary appointments was increased by 81% (mean difference [MD]=1.3, 95% confidence interval [CI]=0.71-1.92; P<.001). Unplanned acute health care utilization was reduced by 59% (MD=2.9, 95% CI=1.14-4.69; P<.01) and packed red blood cell transfusion was reduced by 81% (MD=1.38, 95% CI=0.71-2.04; P<.001). Asthma is prevalent in children with SCD, and interdisciplinary clinics can improve access to subspecialty pulmonary care and reduce unplanned acute care. Additional patients and a longer follow-up period are required to determine the true treatment effect.

Sleep Moderating the Relationship Between Pain and Health Care Use in Youth With Sickle Cell Disease.

The purpose of the current study was to investigate the influence of sleep on the relationship between pain and health care use (HCU) in youth with sickle cell disease (SCD). It was hypothesized that poor sleep would be related to higher HCU and would strengthen the relationship between high pain frequency and more HCU among youth with SCD. Ninety-six youth with SCD (aged 8 to 17 y) and their guardians were recruited from 3 regional pediatric SCD clinics. Guardians reported on the youth's pain frequency and HCU using the Structured Pain Interview for parents, and youth wore a sleep actigraph for up to 2 weeks to assess sleep duration and sleep efficiency. A series of regression models were calculated with the following outcomes: emergency department visits, hospitalizations, and health care provider contacts. Inconsistent with hypotheses, poor sleep was not directly related to HCU. Also, higher sleep duration appeared to strengthen the relationship between high pain frequency and more emergency department visits. Findings suggest that good sleep may serve as a protective factor for better matching pain to HCU. Results should be interpreted in the context of study limitations. Research is needed to investigate possible mechanisms linking sleep duration to HCU in response to pain and to ascertain if sleep patterns influence the relationship between pain and other functional outcomes in youth with SCD. Clinically, these findings support the need to acknowledge and address the role that sleep plays in responding to SCD pain in pediatric populations.

Mental Health Screening in a Pediatric Sickle Cell Disease Clinic: Acceptability and Impact on Mental Health Discussions, Referrals, and Patient Satisfaction

Introduction: Mental health (MH) problems are relatively common in children with sickle cell disease (SCD). Psychological factors are known to directly and indirectly affect pain perception, functional impairment, and other patient-reported health outcomes. Therefore, it is essential to consider these domains in order to provide comprehensive, evidence-based care to youth with SCD. However, it can be challenging for hematology providers without substantive training in MH assessment to recognize symptoms and discuss impressions and treatment recommendations with families. We examined the acceptability and outcomes of a MH screening program in a pediatric SCD clinic. Methods: During an initial data collection period ("Phase A"), patients' parents (n = 57) were interviewed after hematology clinic visits, providing details about any MH discussions that occurred during the visit and completing Press Ganey® patient satisfaction questions. During a secondary phase ("Phase B"), patients (ages 8-17) and parents (of children ages 5-17) completed the PROMIS Pediatric Profile-25, which assesses 6 domains (fatigue, anxiety, depression, pain interference, mobility, and peer relationships), via iPad in the clinic waiting room. Raw scores were entered into an electronic form, which calculated T scores and generated tailored MH recommendations for hematologists to review before seeing each patient. Parents (n = 32) completed the same post-visit interviews. Results: At baseline (Phase A), 83% of parents reported that their child's hematologist asked questions about MH during the visit. Across Phases A and B, 97% of parents who reported that their child's hematologist asked about MH reported that they liked that these questions were asked. Of the parents who said MH was not discussed, 27% said that they wished their hematologist would have asked about it. All parents reported that the 25-item PROMIS Pediatric Profile was an appropriate length and that the screening survey was easy to complete on an iPad. Additionally, 100% of parents reported feeling that it was a good idea to ask the screening questions on an iPad prior to the hematology visit. Most parents (94%) agreed or strongly agreed that the survey questions were appropriate for the care of their child. In Phase B, hematologists stated that they referred to the MH screening summary to guide part of their patient visit 90% of the time and they described the screening summary as "Very Useful" 92% of the time. Parent reports of the frequency of MH discussions were not significantly different between Phase A (no screening) and Phase B (screening) (84% to 81%; p = .772). However, when asking hematologists whether specific issues were discussed during the visit, significant increases were observed in the rates of discussions about the impact of pain on daily activity (72% to 94%; χ2(1) = 8.87, p = .004), anxiety (4% to 36%; χ2(1) = 17.08, p &lt; .001), and depression (6% to 30%; χ2(1) = 10.57, p = .001). Hematologists reported a nonsignificant increase in the rate of referral for MH services in Phase B (9% to 14%; p = .548). In regards to patient/parent satisfaction, there was a statistically significant increase in the proportion of "Very Good" ratings on the item asking about satisfaction with the degree to which the physician talked using words the parent could understand (88% to 100%; χ2(1) = 4.27, p = .046), and clinically meaningful but statistically nonsignificant increases on items about satisfaction with the amount of time the physician spent with the patient (88% to 97%; p = .250) and the likelihood of the parent recommending the physician to others (84% to 94%; p = .315). Conclusions: This study demonstrated the acceptability of MH screening in a pediatric SCD clinic. Although hematologists already discussed MH frequently prior to implementing the screening program, increases in discussions about anxiety, depression, and the functional impact of pain were noted after introducing MH screenings. Rates of referrals for MH treatment rose following implementation of the screening program, but this increase was not statistically significant. Hematologists found a brief MH screening summary to be useful and certain aspects of patient/parent satisfaction showed improvement. Pediatric SCD clinics could integrate MH screenings to increase consideration of the role of psychological factors in patients' presentations and positively affect patient/parent satisfaction. Disclosures No relevant conflicts of interest to declare.

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

An Analysis of Racial and Ethnic Backgrounds within the Casire International Cohort of Sickle Cell Disease Patients: Implications for Disease Phenotype and Clinical Research

Investigating ‘No Shows’ at a Hospital-Based Pediatric Sickle Cell Disease Clinic

BACKGROUND: In sickle cell disease (SCD), preventing all pain crises or other serious complications is not possible. When consistently followed by a health provider, some complications are avoidable. Patients lose vital opportunities for health monitoring/education when appointments are missed, increasing risk of hospitalization or mortality. Forgetfulness, disease risk/symptom severity perception, transportation, long clinic wait times, scheduling and work/school commitments are examples of factors keeping patients from appointments. An understanding of relevant benefits of and barriers to appointment keeping is essential to overcoming the barriers and promoting the benefits. Appointment keeping behaviors are multi-dimensional and often related to many of the social determinants of health. The Health Belief Model (HBM) is a theoretical framework previously used to investigate appointment keeping. Exploring an individual's general health motivations, perceived disease risks and the role of specific behaviors to reduce risks can be informative in this context. OBJECTIVE: The purpose of this research was to explore how health beliefs affect the appointment keeping behavior of pediatric SCD patients and caregivers at a hospital-based outpatient clinic in central Illinois. METHODS: A 53 question multiple-choice questionnaire was utilized to assess health beliefs. This modified version of a validated HBM questionnaire was changed with permission from the creator (Mirotznik et al., 1998) to better represent the symptoms/complications of SCD. Questions determined participants' general health motivation (GHM), perceived susceptibility to the complications of SCD (PSC), perceived severity of SCD (PS), perceived benefits of doctor visits (PB), perceived costs of doctor visits (PC) (i.e. finding transportation, the energy or time to keep visit, wait at clinic, having blood drawn, talking multiple people at one visit, etc.) and intent to keep doctor appointments (INT). The Cronbach alpha coefficient was .87, suggesting good internal consistency reliability. Participants were recruited through convenience sampling at scheduled clinic appointments. A chart review measured overall appointment keeping. RESULTS: In total, 32 individuals (caregiver or patient ≥14 years) completed the questionnaire. There was a significant difference in 'no show' appointment rates for males versus females (Mean= 5.4% 'no show' rate for males vs Mean=17.6% for females, p=.008, two-tailed t-test). There was not a significant difference in 'no show' rates based on the gender of the child (&lt;14 years), participant's city of residence or child's SCD type. There was a small, positive correlation between the age of the child and 'no show' rate (r =.175), but it was not statistically significant. There were statistically significant correlations between INT and GHM (r =.523), PC (r = -.584), and 'no show' rates (r = -.426). PSC had statistically significant correlations with PS (r =.670), PB (r = -.370), and PC (r = .378). PB had a statistically significant correlation with PC (r = -.370). A regression analysis for INT with GHM, PSC, PS, PB and PC as variables explained 66.8% of the variance in intent. Of all the variables, PC made the largest unique contribution (beta = -.69). PS and GHM also made statistically significant contributions (beta = -.35 and .25, respectively). CONCLUSIONS: Three HBM dimensions were uniquely associated with INT. GHM was positively associated, PS negatively associated and PC negatively associated. Participants' PSC and PS play an important role in the perception of the benefits and costs to appointment attendance. Future work to improve appointment keeping will require a larger focus on minimizing costs of doctor visits. Furthermore, effectively enhancing GHM could also improve attendance, potentially lessening the disease process and improving overall wellness. PS negatively correlates with INT. Although counterintuitive, these findings are congruent with those found in lupus patients by Mirotznik et al. Patients with higher PS may have acute medical visits more often, leaving them unable to attend additional scheduled preventative visits because of the costs. Additionally, if some preventative care tasks are added to acute visits when appropriate (as in our institution), the patients/caregivers may see less benefit in attending the next scheduled comprehensive visit. Table Disclosures No relevant conflicts of interest to declare.