Impact of an Individualized Pain Plan to Treat Sickle Cell Disease Vaso-occlusive Episodes in the Emergency Department.

To address acute vaso-occlusive episodes (VOEs), the leading cause of Emergency Department (ED) visits among individuals with sickle cell disease (SCD), we conducted the clinical study, An Individualized Pain Plan with Patient and Provider Access for Emergency Department care of SCD (ALIGN), across eight sites. We hypothesized an improvement of 0.5 standard deviations in perceived quality of ED pain treatment of a VOE after implementing individualized pain plans (IPPs) accessible to both patients and providers. Patients with SCD were 18-45, owned a cell phone, and had an ED VOE visit within 90 days prior. Patients completed perceived quality of care surveys at baseline and within 96 hours after a VOE ED visit. Providers completed surveys regarding comfort managing VOEs at baseline and after managing an enrolled patient. Most of the 153 patients were African American (95.4%), female (64.7%) and had Hb SS/Sß0 genotype (71.9%). The perceived quality of ED pain treatment was high at both baseline and post implementation of IPPs; our primary outcome hypothesis was not met, as no statistically significant change in patient perceived quality ED treatment ocurred. A total of 135 providers completed baseline and follow-up surveys. On a scale of 1-7, with 7 being extremely comfortable managing VOEs, 60.5% reported a score ≥6 post IPP implementation vs. 57.8% at baseline. Almost all (97.6%) ordered the recommended medication, and 94.7% intend to use IPPs. In this implementation protocol, all sites successfully implemented IPPs . Patients and ED providers both endorsed the use of IPPs.

Association of hydroxyurea adherence with transcranial Doppler screenings in children with sickle cell disease.

BACKGROUND: National sickle cell disease (SCD) guidelines recommend oral hydroxyurea (HU) starting at 9 months of age, and annual transcranial Doppler (TCD) screenings to identify stroke risk in children aged 2-16 years. We examined prevalence and proportion of TCD screenings in North Carolina Medicaid enrollees to identify associations with sociodemographic factors and HU adherence over 3 years. STUDY DESIGN: We conducted a longitudinal study with children ages 2-16 years with SCD enrolled in NC Medicaid from years 2016-2019. Prevalence of TCD screening claims was calculated for 3 years, and proportion was calculated for 12, 24, and 36 months of Medicaid enrollment. Enrollee HU adherence was categorized using HU proportion of days covered. Multivariable Poisson regression assessed for TCD screening rates by HU adherence, controlling for age, sex, and rurality. RESULTS: The prevalence of annual TCD screening was between 39.5% and 40.1%. Of those with 12-month enrollment, 77.8% had no TCD claims, compared to 22.2% who had one or higher TCD claims. Inversely, in children with 36 months of enrollment, 36.7% had no TCD claims compared to 63.3% who had one or higher TCD claims. The proportion of children with two or higher TCD claims increased with longer enrollment (10.5% at 12 months, 33.7% at 24 months, and 52.6% at 36 months). Children with good HU adherence were 2.48 (p < .0001) times more likely to have TCD claims than children with poor HU adherence. CONCLUSION: While overall TCD screening prevalence was low, children with better HU adherence and longer Medicaid enrollment had more TCD screenings. Multilevel interventions are needed to engage healthcare providers and families to improve both evidence-based care and annual TCD screenings in children with SCD.

Epigenetic Aging Associations With Psychoneurological Symptoms and Social Functioning in Adults With Sickle Cell Disease.

Objective: Sickle cell disease (SCD), the most common inherited blood disorder in the United States, is associated with severe psychoneurological symptoms. While epigenetic age acceleration has been linked to psychoneurological symptom burden in other diseases, this connection is unexplored in SCD. This study aimed to assess the association between epigenetic age acceleration and psychoneurological symptom burden in SCD. Methods: In this cross-sectional study, emotional impact, pain impact, sleep impact, social functioning, and cognitive function were assessed in 87 adults living with SCD. DNA methylation data were generated from blood specimens and used to calculate epigenetic age using five clocks (Horvath, Hannum, PhenoAge, GrimAge, & DunedinPACE). Associations between epigenetic age acceleration and symptoms were assessed. Results: The sample (N = 87) had a mean (SD) chronologic age was 30.6 (8.1) years. Epigenetic age acceleration was associated with several symptom outcomes. GrimAge age acceleration (ß = -0.49, p = .03) and increased DunedinPACE (ß = -2.23, p = .004) were associated with worse emotional impact scores. PhenoAge (ß = -0.32, p = .04) and the GrimAge (ß = -0.48, p = .05) age acceleration were associated with worse pain impact scores. Increased DunedinPACE (ß = -2.07 p = .04) were associated with worse sleep impact scores. Increased DunedinPACE (ß = -2.87, p = .005) was associated with worse social functioning scores. We did not find associations between epigenetic age acceleration and cognitive function in this sample. Conclusion: Epigenetic age acceleration was associated with worse symptom experiences, suggesting the potential for epigenetic age acceleration as a biomarker to aid in risk stratification or targets for intervention to mitigate symptom burden in SCD.

Opioid Use Among Children and Adults With Sickle Cell Disease in North Carolina Medicaid Enrollees in the Era of Opioid Harm Reduction.

Adults and children with sickle cell disease (SCD) are predominantly African American, with pain-related health disparities. We examined opioid prescription fill patterns in adults and children with SCD and compared factors associated with fills in North Carolina Medicaid enrollees. Our retrospective cohort study included 955 enrollees diagnosed with SCD having at least one opioid fill. Associations were measured between two cohorts (12 and 24 mo of continuous enrollment) for the following characteristics: sex, age, enrollee residence, hydroxyurea adherence, comanagement, enrollment in Community Care North Carolina, prescription for short versus short and long-acting opioids, and emergency department reliance. The majority of individuals did not have an opioid claim over a 12 or 24-month period. Claims increased at ages 10 to 17, peaking at ages 18 to 30. The increased number of claims was associated with the following factors: increasing age, male, short versus long-acting opioids, and Medicaid enrollment for 24 versus 12 months. Community Care North Carolina enrollees in the 12-month cohort had higher opioid days of supply per month; the inverse was true of the 24-month cohort.

Diabetes distress in Veterans with type 2 diabetes mellitus: Qualitative descriptive study.

Diabetes distress (DD) is a negative psychosocial response to living with type 2 diabetes mellitus (T2DM). We sought insight into Veterans' experiences with DD in the context of T2DM self-management. The four domains in the Diabetes Distress Scale (i.e. regimen, emotional, interpersonal, healthcare provider) informed the interview guide and analysis (structural coding using thematic analysis). The mean age of the cohort (n = 36) was 59.1 years (SD 10.4); 8.3% of patients were female and 63.9% were Black or Mixed Race; mean A1C was 8.8% (SD 2.0); and mean DDS score was 2.4 (SD 1.1), indicating moderate distress. Veterans described DD and challenges to T2DM self-management across the four domains in the Diabetes Distress Scale. We found that (1) Veterans' challenges with their T2DM self-management routines influenced DD and (2) Veterans experienced DD across a wide range of domains, indicating that clinical interventions should take a "whole-person" approach.Trial Registration: NCT04587336.

Nurse practitioner satisfaction with in-person versus telehealth chronic care delivery.

BACKGROUND: The widespread use of telehealth and regulatory changes that enhanced nurse practitioner (NP) practice authority because of the SARS-CoV-2 pandemic offers an opportunity to assess postpandemic NP satisfaction with telehealth care delivery and perceptions of its feasibility compared with in-person visits. PURPOSE: Outpatient chronic care delivery satisfaction and preference were compared among NPs who provide care to adults through in-person and/or telehealth visits and examined NP demographic and clinical characteristics associated with overall satisfaction by care delivery type. METHODOLOGY: Data were collected using a cross-sectional, descriptive design through online dissemination of The Care Delivery Satisfaction Survey to a nationally representative sample of 586 NPs. RESULTS: Compared with NPs using both visit types to deliver care, NPs delivering care in-person only had significantly lower satisfaction scores for interpersonal manner ( p = .0076) and communication ( p = .0108). NPs using telehealth only had significantly higher overall satisfaction and satisfaction subscale scores (all p < .01) compared with NPs using both visit types. Overall, 77% of NPs using both visit types preferred in-person delivery. CONCLUSIONS/IMPLICATIONS: NPs delivering telehealth care only were more satisfied with chronic care delivery than NPs using both delivery types. NPs using both types were more satisfied with interpersonal manner and communication compared with NPs delivering in-person care only. Most NPs using both types preferred in-person care delivery. Given increased telehealth use, health systems, academic institutions, and insurance companies can use these study findings to inform policy on telehealth resources and infrastructure.

Characterizing epigenetic aging in an adult sickle cell disease cohort.

ABSTRACT: Sickle cell disease (SCD) affects ∼100 000 predominantly African American individuals in the United States, causing significant cellular damage, increased disease complications, and premature death. However, the contribution of epigenetic factors to SCD pathophysiology remains relatively unexplored. DNA methylation (DNAm), a primary epigenetic mechanism for regulating gene expression in response to the environment, is an important driver of normal cellular aging. Several DNAm epigenetic clocks have been developed to serve as a proxy for cellular aging. We calculated the epigenetic ages of 89 adults with SCD (mean age, 30.64 years; 60.64% female) using 5 published epigenetic clocks: Horvath, Hannum, PhenoAge, GrimAge, and DunedinPACE. We hypothesized that in chronic disease, such as SCD, individuals would demonstrate epigenetic age acceleration, but the results differed depending on the clock used. Recently developed clocks more consistently demonstrated acceleration (GrimAge, DunedinPACE). Additional demographic and clinical phenotypes were analyzed to explore their association with epigenetic age estimates. Chronological age was significantly correlated with epigenetic age in all clocks (Horvath, r = 0.88; Hannum, r = 0.89; PhenoAge, r = 0.85; GrimAge, r = 0.88; DunedinPACE, r = 0.34). The SCD genotype was associated with 2 clocks (PhenoAge, P = .02; DunedinPACE, P < .001). Genetic ancestry, biological sex, ß-globin haplotypes, BCL11A rs11886868, and SCD severity were not associated. These findings, among the first to interrogate epigenetic aging in adults with SCD, demonstrate epigenetic age acceleration with recently developed epigenetic clocks but not older-generation clocks. Further development of epigenetic clocks may improve their predictive ability and utility for chronic diseases such as SCD.

"The project did not come to us with a solution": Perspectives of research teams on implementing a study about electronic health record-embedded individualized pain plans for emergency department treatment of vaso-occlusive episodes in adults with sickle cell disease.

BACKGROUND: This study aimed to capture the implementation process of the ALIGN Study, (An individualized Pain Plan with Patient and Provider Access for Emergency Department care of Sickle Cell Disease). ALIGN aimed to embed Individualized Pain Plans in the electronic health record (E-IPP) and provide access to the plan for both adult patients with sickle cell disease (SCD) and emergency department providers when a person with SCD comes to the emergency department in vaso-occlusive crises. METHODS: Semi-structured interviews were conducted with research teams from the 8 participating sites from the ALIGN study. Seventeen participants (principal investigators and study coordinators) shared their perspectives about the implementation of ALIGN in their sites. Data were analyzed in three phases using open coding steps adapted from grounded theory and qualitative content analysis. RESULTS: A total of seven overarching themes were identified: (1) the E-IPP structure (location and upkeep) and collaboration with the informatics team, (2) the role of ED champion, (3) the role of research coordinators, (4) research team communication, and communication between research team and clinical team, (5) challenges with the study protocol, (6) provider feedback: addressing over-utilizers, patient mistrust, and the positive feedback about the intervention, and (7) COVID-19 and its effects on study implementation. CONCLUSIONS: Findings from this study contribute to learning how to implement E-IPPs for adult patients with SCD in ED. The study findings highlight the importance of early engagement with different team members, a champion from the emergency department, study coordinators with different skills and enhancement of communication and trust among team members. Further recommendations are outlined for hospitals aiming to implement E-IPP for patients with SCD in ED.

A comparison of the effect of patient-specific versus weight-based protocols to treat vaso-occlusive episodes in the emergency department.

BACKGROUND: Vaso-occlusive crises (VOCs) cause debilitating pain and are a common cause of emergency department (ED) visits, for people with sickle cell disease (SCD). Strategies for achieving optimal pain control vary widely despite evidence-based guidelines. We tested existing guidelines and hypothesized that a patient-specific pain protocol (PSP) written by their SCD provider may be more effective than weight-based (WB) dosing of parenteral opiate medication, in relieving pain. METHODS: This study was a prospective, randomized controlled trial comparing a PSP versus WB protocol for patients presenting with VOCs to six EDs. Patients were randomized to a PSP or WB protocol prior to an ED visit. The SCD provider wrote their protocol and placed it in the electronic health record for future ED visits with VOC exclusion criteria that included preexisting PSP excluding parenteral opioid analgesia or outpatient use of buprenorphine or methadone or highly suspected for COVID-19. Pain intensity scores, side effects, and safety were obtained every 30 min for up to 6 h post-ED bed placement. The primary outcome was change in pain intensity score from placement in an ED space to disposition or 6 h. RESULTS: A total of 328 subjects were randomized; 104 participants enrolled (ED visit, target n = 230) with complete data for 96 visits. The study was unable to reach the target sample size and stopped early due to the impact of COVID-19. We found no significant differences between groups in the primary outcome; patients randomized to a PSP had a shorter ED length of stay (p = 0.008), and the prevalence of side effects was low in both groups. Subjects in both groups experienced both a clinically meaningful and a statistically significant decrease in pain (27 mm on a 0- to 100-mm scale). CONCLUSIONS: We found a shorter ED length of stay for patients assigned to a PSP. Patients in both groups experienced good pain relief without significant side effects.

Infertility and treatment-seeking practices among females and males with sickle cell disease in the Sickle Cell Disease Implementation Consortium registry.

OBJECTIVE: To describe the prevalence of infertility and infertility treatment seeking among people enrolled in the Sickle Cell Disease Implementation Consortium (SCDIC) registry and identify sociodemographic and clinical correlates of infertility. DESIGN: Cross-sectional. PARTICIPANTS: The study population included 2108 women and men (≥18 years of age) enrolled in the SCDIC registry who completed the fertility questionnaire. RESULTS: All participants who completed the infertility-specific questions were included in the analysis (1224 females; 884 males). Of these, 16.9% of males and 23.7% of females reported infertility, in contrast to rates in the general population (12% of males; 11% of females). Only 22.8% of this subgroup had sought a fertility consultation; of these, 41% received infertility testing and 58% received advice, yet only a few received specific treatment: ovulation medication (19.1%), fallopian tubal surgery (4.8%), other female treatment (17.5%), varicocelectomy (8.1%), or other male treatment (10.8%). Increasing age, employment status, and interaction between gender and single marital status are associated with reported infertility. We did not observe differences between groups relative to sickle cell disease (SCD) genotype, a broad category of self-reported hydroxyurea use any time during life, type of medical insurance, income, or education. CONCLUSION: To our knowledge, this is the first study to examine self-reported identification of and treatment for infertility among a large sample of people with SCD. These findings suggest that (a) infertility occurs at a higher rate, but fertility care treatment seeking is less frequent than in the general public; and (b) sociodemographic and clinical differences between individuals who report experiencing infertility and those who do not did not emerge in this study.

Time to pain relief: A randomized controlled trial in the emergency department during vaso-occlusive episodes in sickle cell disease.

OBJECTIVE: Compare time to pain relief (minimum of a 13 mm and 30% reduction) during an Emergency Department (ED) visit among patients with sickle cell disease (SCD) experiencing severe pain associated with a vaso-occlusive episode who were randomized to receive either an individualized or weight-based pain protocol. METHODS: A randomized controlled trial in two EDs. Adults with sickle cell disease. Research staff recorded pain scores every 30 min during an ED visit (up to 6 h in the ED) using a 0-100 mm visual analogue scale. Analysis included 122 visits, representing 49 patients (individualized: 61 visits, 25 patients; standard: 61 visits, 24 patients). RESULTS: Pain reduction across 6-h was greater for the individualized compared to the standard protocol (protocol-by-time: p = .02; 6-h adjusted pain score comparison: Individualized: M = 29.2, SD = 38.8, standard: M = 45.3, SD = 35.6; p = .03, Cohen d = 0.43). Hazards models indicated a greater probability of 13 mm (HR = 1.54, 95% CI = 1.05, 2.27, p = .03) and 30% (HR = 1.71, 95% CI = 1.11, 2.63, p = .01) reduction in the individualized relative to the standard protocol. CONCLUSIONS: Patients who received treatment with an individualized protocol experienced a more rapid reduction in pain, including a 13 mm and 30% reduction in pain scores when compared to those that received weight-based dosing.

Stigma and quality of life in adults with sickle cell disease in Jamaica and the United States.

Sickle cell disease (SCD) is the most common inherited blood disorder in both Jamaica and the United States and is characterized by poor quality of life and debilitating complications, with the hallmark symptom being pain caused by acute and chronic conditions. Individuals with SCD often experience stigma due to their disease status, opioid use, and race. This study sought to understand the influence of perceived stigma and demographic/clinical characteristics on quality of life in adults with SCD in Jamaica (n = 50) and the United States (n = 50). Participants completed interviewer-administered surveys including demographic/clinical characteristics; the Measure of Sickle Cell Stigma (MoSCS); and the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me). A set of general linear models for each country was built to examine the influence of explanatory variables on the quality of life outcomes. Overall, stigma scores were low for both countries, with the exception of the MoSCS disclosure concerns and expected discrimination subscales, where scores averaged medium and high, respectively. In both countries, being employed was associated with better quality of life; and reports of stigma (internalized stigma and expected discrimination) was associated with worse quality of life. These findings have several implications for healthcare providers caring for individuals with SCD, policy makers, and researchers. Specifically, findings can be used to advocate for improved access to mental health care for individuals with SCD and inform stigma reduction intervention approaches in SCD.

Characterising the prevalence of overweight and obese status among adults with sickle cell disease.

Individuals with sickle cell disease (SCD) have historically been considered underweight. Despite increasing body mass index (BMI) in the general population, the prevalence of overweight and obese status remains unclear in the adult SCD population. Our primary aim was to determine the prevalence of overweight and obese status and to identify associations between BMI, demographic, and clinical characteristics. We conducted an analysis of abstracted electronic health record data and patient-reported outcomes from the Sickle Cell Disease Implementation Consortium registry; individuals aged 20-45 years were included. The median (interquartile range) BMI for the 1664 adults in this analysis was 23.9 (21.1-28) kg/m2 . In this cohort, 42.9% had a BMI of >25 kg/m2 (Centers for Disease Control and Prevention definition of overweight/obese). In multivariable analysis, higher odds of being overweight or obese were associated with female gender, older age, college education, private insurance, and hypertension diagnosis. Higher odds of a BMI of >25 kg/m2 were observed in individuals with HbSC or HbSß+ thalassaemia regardless of hydroxycarbamide (hydroxyurea) exposure (odds ratio [OR] 3.4, p < 0.0001) and HbSS or HbSß0 thalassaemia exposed to hydroxycarbamide (OR 1.6, p = 0.0003) compared to those with HbSS or HbSß0 thalassaemia with no hydroxycarbamide exposure. These data highlight the importance of early identification, prevention, and intervention for increasing BMI to reduce obesity-related complications that may impact SCD-related complications.

Evaluating Associations between Average Pain Intensity and Genetic Variation in People with Sickle Cell Disease: An Exploratory Study.

BACKGROUND: Pain is one of the most common and deleterious symptoms experienced by individuals with sickle cell disease (SCD). There is a paucity of studies identifying potential genetic mechanisms of pain in this population. AIM: Examine associations between 11 functional single nucleotide polymorphisms in 9 candidate genes with reports of average pain intensity in individuals with sickle cell disease. METHOD: Cross-sectional analyses were performed on data and blood samples collected through the Duke SCD Implementation Consortium Registry. Participants were asked to rate their pain "on the average" using an 11-point numeric rating scale (0 = no pain; 10 = pain as bad as you can imagine). We genotyped 11 single nucleotide polymorphisms in 9 pain-related genes using TaqMan® Genotyping Assays. Associations between each polymorphism and reports of average pain were evaluated. RESULTS: The 86 participants (mean age: 28.7 years; 64% female) included in this study reported moderate pain on average (Mean = 4, Standard Deviation = 2.4). ICAM1 rs1799969 was the only genetic polymorphism that was significantly associated with pain (p = .01). Individuals with one or more minor alleles had lower average pain (Mean = 1.25, Standard Deviation = 1.50) than individuals without a minor allele (Mean = 4.13, Standard Deviation = 2.25). The effect size for ICAM1 rs1799969 was 1.30, which is considered large. The effect sizes for all other single nucleotide polymorphisms ranged from small to medium (range: 0-0.3). CONCLUSIONS: Our findings provide preliminary evidence that the minor allele in ICAM1 rs1799969 had protective effects against experiencing more severe pain in sickle cell disease.

Perspectives of individuals with sickle cell disease on barriers to care.

INTRODUCTION: Sickle cell disease (SCD) is an inherited hemoglobinopathy that predominantly affects African Americans in the United States. The disease is associated with complications leading to high healthcare utilization rates, including emergency department (ED) visits and hospitalizations. Optimal SCD care requires a multidisciplinary approach involving SCD specialists to ensure preventive care, minimize complications and prevent unnecessary ED visits and hospitalizations. However, most individuals with SCD receive sub-optimal care or are unaffiliated with care (have not seen an SCD specialist). We aimed to identify barriers to care from the perspective of individuals with SCD in a multi-state sample. METHODS: We performed a multiple methods study consisting of surveys and interviews in three comprehensive SCD centers from March to June 2018. Interviews were transcribed and coded, exploring themes around barriers to care. Survey questions on the specific themes identified in the interviews were analyzed using summary statistics. RESULTS: We administered surveys to 208 individuals and conducted 44 in-depth interviews. Barriers to care were identified and classified according to ecological level (i.e., individual, family/interpersonal, provider, and socio-environmental/organizational level). Individual-level barriers included lack of knowledge in self-management and disease severity. Family/interpersonal level barriers were inadequate caregiver support and competing life demands. Provider level barriers were limited provider knowledge, provider inexperience, poor provider-patient relationship, being treated differently, and the provider's lack of appreciation of the patient's SCD knowledge. Socio-environmental/organizational level barriers included limited transportation, lack of insurance, administrative barriers, poor care coordination, and reduced access to care due to limited clinic availability, services provided or clinic refusal to provide SCD care. CONCLUSION: Participants reported several multilevel barriers to SCD care. Strategies tailored towards reducing these barriers are warranted. Our findings may also inform interventions aiming to locate and link unaffiliated individuals to care.

Severe Persistent Pain and Inflammatory Biomarkers in Sickle Cell Disease: An Exploratory Study.

BACKGROUND: Severe pain is among the most common and deleterious symptoms experienced by individuals with sickle cell disease (SCD), of whom more than 50% report chronic pain. Despite this, the understanding of the biological contributors to persistent severe SCD pain is limited. This exploratory study sought to describe pain phenotypes based on frequency of severe pain experienced over 6 months and identify inflammatory biomarkers associated with pain phenotypes among individuals with SCD. METHODS: This study used self-report and electronic health record data collected from 74 individuals enrolled in the Duke Sickle Cell Disease Implementation Consortium Registry. Plasma from previously collected blood specimens was used to generate inflammatory biomarker data using the Inflammation 20-plex ProcartaPlexTM panel. Descriptive statistics were used to describe the occurrence of severe pain over the past 6 months, and bi-variate analyses were used to evaluate the relationship between inflammatory biomarkers and pain phenotypes. RESULTS: Among the 74 participants included in this study, 33.8% reported severe pain occurring never or rarely, 40.5% reported severe pain occurring sometimes, and 25.7% reported severe pain occurring often or always. Soluble E-selectin (sE-selectin) was the only inflammatory biomarker significantly associated with the pain phenotype groups (p = 0.049). Post hoc comparisons identified that participants in the often/always severe pain group had significantly higher plasma concentrations of sE-selectin compared to those in the sometimes severe pain group (p = 0.040). CONCLUSIONS: Our findings provide preliminary evidence of the frequent occurrence of severe pain and that sE-selectin may be an objective biomarker for the frequent occurrence of severe pain in this population.

Identifying barriers to evidence-based care for sickle cell disease: results from the Sickle Cell Disease Implementation Consortium cross-sectional survey of healthcare providers in the USA.

OBJECTIVES: Sickle cell disease (SCD) leads to chronic and acute complications that require specialised care to manage symptoms and optimise clinical results. The National Heart Lung and Blood Institute (NHLBI) evidence-based guidelines assist providers in caring for individuals with SCD, but adoption of these guidelines by providers has not been optimal. The objective of this study was to identify barriers to treating individuals with SCD. METHODS: The SCD Implementation Consortium aimed to investigate the perception and level of comfort of providers regarding evidence-based care by surveying providers in the regions of six clinical centres across the USA, focusing on non-emergency care from the providers' perspective. RESULTS: Respondents included 105 providers delivering clinical care for individuals with SCD. Areas of practice were most frequently paediatrics (24%) or haematology/SCD specialist (24%). The majority (77%) reported that they were comfortable managing acute pain episodes while 63% expressed comfort with managing chronic pain. Haematologists and SCD specialists showed higher comfort levels prescribing opioids (100% vs 67%, p=0.004) and managing care with hydroxyurea (90% vs 51%, p=0.005) compared with non-haematology providers. Approximately 33% of providers were unaware of the 2014 NHLBI guidelines. Nearly 63% of providers felt patients' medical needs were addressed while only 22% felt their mental health needs were met. CONCLUSIONS: A substantial number of providers did not know about NHLBI's SCD care guidelines. Barriers to providing care for patients with SCD were influenced by providers' specialty, training and practice setting. Increasing provider knowledge could improve hydroxyurea utilisation, pain management and mental health support.

Awareness and Use of the Sickle Cell Disease Toolbox by Primary Care Providers in North Carolina.

BACKGROUND: Sickle cell disease (SCD) is a complex chronic blood disorder characterized by severe disease complications ideally managed by both hematologists and primary care providers (PCP's). PCP's report knowledge gaps and discomfort with SCD management. Our team developed and a decision support tool for SCD management (SCD Toolbox) based on the National Heart, Lung, and Blood Institute's SCD guidelines. We surveyed PCPs in North Carolina (NC) prior to formal dissemination to determine current co-management practices, assess toolbox acceptability, use, format preferences, and understand which algorithms would be most helpful. METHOD: A 23-item baseline needs assessment survey was disseminated to PCPs throughout NC. RESULTS: A total of 63 medical providers responded to the survey and of these respondents, 64% reported caring for 1 to 10 patients with SCD. Only 39% of PCPs reported regular communication with an SCD specialist. Providers reported the highest level of awareness of the pediatric and adult health maintenance tools (41% and 39% respectively) and highest use of the pediatric (26%) and adult (28%) health maintenance tools. Respondents also expressed a desire to have access to multiple toolbox formats (37%) (website, mobile app and/or paper). LIMITATIONS: The use of a convenience sample and low survey response are study limitations which hinder generalizability. CONCLUSIONS: PCPs rarely co-managed with a specialist, had low awareness and use of SCD toolbox, and requested multiple formats for the toolbox.

Sex-based differences in the manifestations and complications of sickle cell disease: Report from the Sickle Cell Disease Implementation Consortium.

INTRODUCTION: Sex-based clinical outcome differences in sickle cell disease (SCD) remain largely unknown despite evidence that female sex is associated with an increased lifespan. To better characterize sex-based differences in SCD, we assessed pain, treatment characteristics, laboratory measures and complications among males and females currently enrolled in the Sickle Cell Disease Implementation Consortium (SCDIC) registry. METHODS: The SCDIC consists of eight comprehensive SCD centers and one data coordinating center that received funding from the National Heart Lung and Blood Institute to improve outcomes for individuals with SCD. Eligibility criteria included: 15 to 45 years of age and a confirmed diagnosis of SCD. Self-report surveys were completed and data were also abstracted from the participants' medical records. RESULTS: A total of 2,124 participants were included (mean age: 27.8 years; 56% female). The majority had hemoglobin SS SCD genotype. Females had worse reports of pain severity (mean (SD) T-score 51.6 (9.6) vs 49.3 (10), p<0.001), more vaso-occlusive episodes (p = 0.01) and a higher occurrence of 3 or more hospital admissions in the past year (30.9% vs. 25.5, p = 0.03). On multivariable analysis, males had higher odds of acute chest syndrome (odds ratio (OR) 1.4, p = 0.002), cardiovascular (OR 1.70, p<0.001) and musculoskeletal (OR 1.33, p = 0.0034) complications and lower odds of depression (OR 0.77, p = 0.0381). Females had higher fetal hemoglobin levels with and without hydroxyurea use (9.6% vs 8.5%, p = 0.03 and 3% vs 2.2%, p = 0.0005, respectively). CONCLUSION: Our data suggests that sex differences in clinical outcomes do occur among individuals with SCD. Future research needs to explore the mechanisms underlying these differences.