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BACKGROUND: Sickle cell disease (SCD), an inherited red blood cell disorder, primarily affects African Americans in the United States. Adolescents and young adults with SCD (AYA-SCD) are at risk of high morbidity and mortality when transitioning from pediatric to adult care. The goal of this qualitative study was to understand factors associated with optimal implementation of the AYA-SCD transition. METHODS: Participants were recruited from a large hospital system and the community. Interview guides included topics on access to primary and specialized care, beliefs and practices related to pain control, transition from pediatric to adult care, and patient experiences in the emergency department (ED). Data were coded and analyzed using an inductive thematic coding approach in combination with a deductive coding approach using domains from the Consolidated Framework for Implementation Research (CFIR). RESULTS: Fifty-nine participants, including twenty-one AYA-SCD from both the pediatric and adult clinics, seventeen caregivers, nine pediatric SCD providers, six adult SCD providers, and six ED providers, completed 11 focus groups and 5 semi-structured interviews. Results identified multiple factors within the domains of CFIR including the outer setting, inner setting, individual characteristics, and intervention characteristics. Results were incorporated into a transition framework to inform local practice improvement. CONCLUSION: Our study highlights the importance of multi-level barriers and facilitators for AYA-SCD transition from pediatric to adult care. Future studies could use implementation science frameworks to understand local context and identify strategies and intervention characteristics to improve transition programming. These efforts will ultimately reduce health disparities and ensure health equity.

Background Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/β0 thalassemia) aged as early as 9 months. However, the uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or tools, especially those that provide medical evidence and consider caregivers’ preferences and values, to facilitate a shared discussion with caregivers. Objective The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences). Methods We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0-5 years) who were offered and prescribed hydroxyurea and the resultant health outcomes. Results The Ethics Committee of the Cincinnati Children’s Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants. Conclusions The long-term objective of this study is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA can make informed and shared decisions about their health. Trial Registration ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114 International Registered Report Identifier (IRRID) DERR1-10.2196/27650

BACKGROUND Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/β0 thalassemia) aged as early as 9 months. However, the uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or tools, especially those that provide medical evidence and consider caregivers’ preferences and values, to facilitate a shared discussion with caregivers. OBJECTIVE The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences). METHODS We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0-5 years) who were offered and prescribed hydroxyurea and the resultant health outcomes. RESULTS The Ethics Committee of the Cincinnati Children’s Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants. CONCLUSIONS The long-term objective of this study is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA can make informed and shared decisions about their health. CLINICALTRIAL ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/27650

Background Hydroxyurea prevents disease complications among patients with sickle cell disease (SCD). Although its efficacy has been endorsed by the National Health Lung and Blood Institute evidence-based guidelines, its adoption is low, both by patients with SCD and providers. Mobile health (mHealth) apps provide benefits in improving medication adherence and self-efficacy among patients with chronic diseases and have facilitated prescription among medical providers. However, mHealth has not been systematically tested as a tool to increase hydroxyurea adherence nor has the combination of mHealth been assessed at both patient and provider levels to increase hydroxyurea utilization. Objective This study aims to increase hydroxyurea utilization through a combined two-level mHealth intervention for both patients with SCD and their providers with the goals of increasing adherence to hydroxyurea among patients and improve hydroxyurea prescribing behavior among providers. Methods We will test the efficacy of 2 mHealth interventions to increase both patient and provider utilization and knowledge of hydroxyurea in 8 clinical sites of the NHLBI-funded Sickle Cell Disease Implementation Consortium (SCDIC). The patient mHealth intervention, InCharge Health, includes multiple components that address memory, motivation, and knowledge barriers to hydroxyurea use. The provider mHealth intervention, Hydroxyurea Toolbox (HU Toolbox), addresses the clinical knowledge barriers in prescribing and monitoring hydroxyurea. The primary hypothesis is that among adolescents and adults with SCD, adherence to hydroxyurea, as measured by the proportion of days covered (the ratio of the number of days the patient is covered by the medication to the number of days in the treatment period), will increase by at least 20% after 24 weeks of receiving the InCharge Health app, compared with their adherence at baseline. As secondary objectives, we will (1) examine the change in health-related quality of life, acute disease complications, perceived health literacy, and perceived self-efficacy in taking hydroxyurea among patients who use InCharge Health and (2) examine potential increases in the awareness of hydroxyurea benefits and risks, appropriate prescribing, and perceived self-efficacy to correctly administer hydroxyurea therapy among SCD providers between baseline and 9 months of using the HU Toolbox app. We will measure the reach, adoption, implementation, and maintenance of both the InCharge Health and the HU Toolbox apps using the reach, effectiveness, adoption, implementation, and maintenance framework and qualitatively evaluate the implementation of both mHealth interventions. Results The study is currently enrolling study participants. Recruitment is anticipated to be completed by mid-2021. Conclusions If this two-level intervention, that is, the combined use of InCharge Health and HU Toolbox apps, demonstrates efficacy in increasing adherence to hydroxyurea and prescribing behavior in patients with SCD and their providers, respectively, both apps will be offered to other institutions outside the SCDIC through a future large-scale implementation-effectiveness study. Trial Registration ClinicalTrials.gov NCT04080167; https://clinicaltrials.gov/ct2/show/NCT04080167 International Registered Report Identifier (IRRID) DERR1-10.2196/16319

Hydroxyurea is an efficacious treatment for sickle cell disease (SCD), but adoption is low among individuals with SCD. The objective of this study was to examine barriers to patients’ adherence to hydroxyurea use regimens by using the intentional and unintentional medication nonadherence framework. We interviewed individuals with SCD age 15 to 49.9 years who were participants in the Sickle Cell Disease Implementation Consortium (SCDIC) Needs Assessment. The intentional and unintentional medication nonadherence framework explains barriers to using hydroxyurea and adds granularity to the understanding of medication adherence barriers unique to the SCD population. In total, 90 semi-structured interviews were completed across 5 of the 8 SCDIC sites. Among interviewed participants, 57.8% (n = 52) were currently taking hydroxyurea, 28.9% (n = 26) were former hydroxyurea users at the time of the interview, and 13.3% (n = 12) had never used hydroxyurea but were familiar with the medication. Using a constructivist grounded theory approach, we discovered important themes that contributed to nonadherence to hydroxyurea, which were categorized under unintentional (eg, Forgetfulness, External Influencers) and intentional (Negative Perceptions of Hydroxyurea, Aversion to Taking Any Medications) nonadherence types. Participants more frequently endorsed adherence barriers that fell into the unintentional nonadherence type (70%) vs intentional nonadherence type (30%). Results from this study will help SCD health care providers understand patient choices and decisions as being either unintentional or intentional, guide tailored clinical discussions regarding hydroxyurea therapy, and develop specific, more nuanced interventions to address nonadherence factors.

Background: Sickle cell disease (SCD) is a genetic disorder that causes significant medical and neurologic morbidity in children. Hydroxyurea (HU) is the primary medication used to prevent these complications. National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering HU to children as young as 9 months of age with SCD (HbSS or HbSB0 thalassemia) using a shared decision-making approach. Although HU has proven efficacious it remains underutilized and caregivers report that they are not always actively involved in the decision to initiate this therapy. Reasons for limited HU uptake likely include lack of clinician knowledge and training and negative caregiver perceptions. Thus, we developed the Engage-HU trial as a novel approach to address HU utilization barriers. A critical consideration for this trial was that SCD primarily affects individuals of African and Hispanic/Latino descent. In these minority populations, intervention trials are sometimes terminated early because of recruitment difficulties related to mistrust of research, caregiver burden, and transportation issues. As such, the Engage-HU trial design included best-practice strategies for recruiting people of color in research. This study describes these strategies, the initial recruitment plan, preliminary recruitment outcomes and strategies, and our procedural adaptations. Study Design and Methods: Engage-HU is a randomized control trial (NCT03442114) to assess how clinicians can engage caregivers in a shared discussion that considers their values and preferences and includes evidence that supports HU. Engage-HU compares two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD: 1) the American Society of Hematology Pocket Guide, and 2) the HU Shared-Decision Making (H-SDM) Toolkit. The study aims to recruit 174 caregivers and evaluate the effectiveness of the dissemination methods on patient-centered outcomes (caregiver confidence in decision-making and perceptions of experiencing shared decision-making) as well as HU uptake and child health outcomes. Eligible children are aged 0 to 5 years, candidates for HU, and their caregiver has not made a decision about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a stepped-wedge design. Data will be analyzed based on the intent-to-treat principle. All participants will remain in the arm of the study to which they were randomized, regardless of whether or not they receive the assigned dissemination method. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using a linear mixed effects regression model with a robust variance estimator and maximum likelihood estimation with observations clustered within site. The Engage-HU trial includes adaptations to increase recruitment such as tailored messaging, a relational recruitment approach, streamlined data collection, and a Stakeholder Advisory Committee. However, even with these adaptations, the first 6-months of the trial yielded lower than anticipated recruitment. Rather than terminate the trial or accept low enrollment, the research team implemented a series of recruitment strategies to address barriers including helping to improve research coordinator knowledge of the study purpose and adjusting no-show and follow-up procedures (e.g., calls to families after missed appointments and reminder calls before appointments). Site clinicians and clinic staff were provided with additional training so they could give more context about Engage-HU to caregivers and the study principal investigator led monthly "all coordinator" calls to provide support by sharing updates and experiences about successful recruitment. Implementation of these strategies resulted in triple the number of enrollments over the next 7-months compared to the previous 6-months (Table 1). Our goal in sharing this information is to provide lessons learned that can be implemented in future trials with the systematically underserved SCD population. It is also anticipated that methods described here may also inform clinical approaches to better engage caregivers of young children around critical clinical conversations, such as initiating medications like HU. Disclosures King: Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Bioline: Consultancy; RiverVest: Consultancy; Novimmune: Research Funding; Celgene: Consultancy; Tioma Therapuetics: Consultancy; Amphivena Therapeutics: Research Funding; WUGEN: Current equity holder in private company; Cell Works: Consultancy; Incyte: Consultancy. Smith-Whitley:Prime: Other: Education material; Celgene: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Neumayr:Emmaus: Consultancy; Bayer: Consultancy; CTD Holdings: Consultancy; Pfizer: Consultancy; ApoPharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; Micelle: Other: Site principal investigator; GBT: Other: Site principal investigator; PCORI: Other: site principal investigator; Novartis: Other: co-investigator; Bluebird Bio: Other: co-investigator; Sangamo Therapeutics: Other; Silarus: Other; Celgene: Other; La Jolla Pharmaceuticals: Other; Forma: Other; Imara: Other; National Heart, Lung, and Blood Institute: Other; Health Resources and Services Administration: Other; Centers for Disease Control and Prevention: Other; Seattle Children's Research: Other. Yates:Novartis: Research Funding. Thompson:Novartis: Consultancy, Honoraria, Research Funding; CRISPR/Vertex: Research Funding; BMS: Consultancy, Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; bluebird bio, Inc.: Consultancy, Research Funding.

Objectives: To report the laboratory investigation of a case of severe combined immunodeficiency (SCID) with maternal T-cell engraftment, focusing on the interference of human leukocyte antigen (HLA) typing by blood chimerism. Methods: HLA typing was performed with three different methods, including sequence-specific primer (SSP), sequence-specific oligonucleotide, and Sanger sequencing on peripheral blood leukocytes and buccal cells, from a 3-month-old boy and peripheral blood leukocytes from his parents. Short tandem repeat (STR) testing was performed in parallel. Results: HLA typing of the patient’s peripheral blood leukocytes using the SSP method demonstrated three different alleles for each of the HLA-B and HLA-C loci, with both maternal alleles present at each locus. Typing results from the patient’s buccal cells showed a normal pattern of inheritance for paternal and maternal haplotypes. STR enrichment testing of the patient’s CD3+ T lymphocytes and CD15+ myeloid cells confirmed maternal T-cell engraftment, while the myeloid cell profile matched the patient’s buccal cells. Conclusions: Maternal T-cell engraftment may interfere with HLA typing in patients with SCID. Selection of the appropriate typing methods and specimens is critical for accurate HLA typing and immunologic assessment before allogeneic hematopoietic stem cell transplantation.

Key Points Question What are the barriers to care experienced by adolescents and adults living with sickle cell disease in the United States? Findings This survey study conducted by the Sickle Cell Disease Implementation Consortium enrolled 440 adolescents and adults in 7 different states and found that most respondents were pleased with their usual care physicians but had negative experiences in acute care settings. Pain and the frequency of pain episodes were associated with patient-reported self-efficacy, further emphasizing the association of severe pain with poor outcomes in this population. Meaning A negative perception of care in the emergency department setting may be a barrier for seeking care among adolescents and adults living with sickle cell disease., Importance Sickle cell disease (SCD) is the most common inherited red blood cell disorder in the United States, and previous studies have shown that individuals with SCD are affected by multiple health disparities, including stigmatization, inequities in funding, and worse health outcomes, which may preclude their ability to access quality health care. This needs assessment was performed as part of the Sickle Cell Disease Implementation Consortium (SCDIC) to assess barriers to care that may be faced by individuals with SCD. Objective To assess the SCD-related medical care experience of adolescents and adults with SCD. Design, Setting, and Participants This one-time survey study evaluated pain interference, quality of health care, and self-efficacy of 440 adults and adolescents (aged 15 to 50 years) with SCD of all genotypes and assessed how these variables were associated with their perceptions of outpatient and emergency department (ED) care. The surveys were administered once during office visits by trained study coordinators at 7 of 8 SCDIC sites in 2018. Results The SCDIC sites did not report the number of individuals approached to participate in this study; thus, a response rate could not be calculated. In addition, respondents were not required to answer every question in the survey; thus, the response rate per question differed for each variable. Of 440 individuals with SCD, participants were primarily female (245 [55.7%]) and African American (428 [97.3%]) individuals, with a mean (SD) age of 27.8 (8.6) years. The majority of participants (306 of 435 [70.3%]) had hemoglobin SS or hemoglobin S β0-thalassemia. Most respondents (361 of 437 [82.6%]) reported access to nonacute (usual) SCD care, and the majority of respondents (382 of 413 [92.1%]) noted satisfaction with their usual care physician. Of 435 participants, 287 (66.0%) reported requiring an ED visit for acute pain in the previous year. Respondents were less pleased with their ED care than their usual care clinician, with approximately half (146 of 287 [50.9%]) being satisfied with or perceiving having adequate quality care in the ED. Participants also noted that when they experienced severe pain or clinician lack of empathy, this was associated with a negative quality of care. Age group was associated with ED satisfaction, with younger patients (, This survey study evaluates pain interference, quality of health care, and disease-specific self-efficacy to assess perceived barriers for seeking care among adults and adolescents with sickle cell disease..

BACKGROUND: The transition from the pediatric setting to adult care is a well-described period of morbidity and mortality for persons with sickle cell disease (SCD). We sought to measure the feasibility and effectiveness of providing skill-based educational handouts on improving self-management and transition readiness in adolescents with SCD. METHODS: This was a single center study in which participants completed a self-assessment, the Adolescent Autonomy Checklist (AAC), to assess transition readiness and self-management skills at baseline. After results were reviewed by the study coordinator, participants were provided with skill-based handouts on noted areas of deficit. The AAC was subsequently completed at a follow-up visit. All data were stored electronically and transferred into SAS for statistical analyses. RESULTS: Sixty-one patients completed the AAC at baseline and post-intervention. At baseline, patients reported needing the most help with skills in money management, living arrangements, vocational skills, and emergency and healthcare skills. Post-intervention, statistically significant improvements (p < 0.05) occurred in skills related to laundry, housekeeping, healthcare, and sexual development. A regression model exploring the time to follow-up showed that most improvements could not be attributed to maturation alone. CONCLUSION: This study showed that educational handouts are a readily implementable and well-accepted intervention among adolescents with SCD who identify challenges with skills necessary to successfully transition to adult care. Distinguishing which transition needs are best improved with this type of intervention will help to strengthen the multidisciplinary approach necessary to support adolescents and young adults with SCD as they matriculate to adult care.

Sickle cell disease (SCD) is the most common genetic condition in the world and disproportionately affects African Americans in families with lower household incomes. SCD is characterized by a variety of complications including episodes of severe pain, chronic anemia, and end-organ damage. Morbidity from SCD begins in infancy and increases in frequency and severity with age. Complications during childhood and adolescence, both critical learning periods for youth, substantially impact educational attainment and life outcomes. SCD-related hospitalizations are associated with social determinants of health, such as socioeconomic status (SES), depression, health literacy, and educational outcomes. In youth with SCD, family and neighborhood SES are predictors of pain level, pain frequency, and overall quality of life. In addition to the physiological impacts of SCD, individuals with SCD experience emotional and stress related effects of the disease that may impact daily quality of life and frequency and severity of pain. Studies have found that hospital admission frequency has limited or no impact on academic outcomes in youth with SCD. Few studies have explicitly examined the relationship between SCD-related pain and educational, socioeconomic, and mental health outcomes. This is a cross-sectional study of patient survey data from a single site in the Sickle Cell Disease Implementation Science Consortium (SCDIC). The primary objective was to identify a relationship between educational attainment, employment status, mental health, and the frequency, severity, or length of pain crises for individuals with SCD. Multivariate analysis was used to assess the impact of patients' educational attainment, employment status, annual household income (low = less than $25,000, high = $75,001 and above), and self-reported depression on the frequency, length, and severity of SCD-related pain. Our central hypothesis was that individuals with a history of depression, lower educational attainment, periods of unemployment, and lower incomes experience more frequent, more severe, and longer pain crises. A total of 307 participants were included. The mean age was 27.4 years (range 15 to 45), 58.3% were female, and 99% were African American. Sixty-two percent had Hgb SS, the most severe form of SCD. About half of all patients (50.5%) reported they take pain medication every day for SCD and majority were on some form of disease modification (64.2% on hydroxyurea (HU), 20.2% on chronic blood transfusion). Slightly less than half (48.9%) reported their highest level of education as a high school diploma or lower. Most were unemployed (15.3%), students (22.8%), or disabled (21.5%), and 59.2% reported an average annual household income less than $25,000. Univariate analysis revealed statistically significant associations between employment status as unemployed or disabled and frequency of pain (p < .001), employment status as unemployed or disabled and severity of pain (p < .001), and employment status as disabled and length of pain > 4 days. Relationships between depression and frequency and severity of pain were statistically significant at the p < .001 level, and between depression and length of pain > 1 week at the p < .01 level. Multivariate analysis revealed positive statistically significant relationships between depression and high pain frequency (p < .001), employment status as disabled and severe pain (p < .01), depression and severe pain (p < .01), and employment status as disabled and length of pain >4 days (p < .05), Table 1. Educational attainment did not demonstrate statistically significant relationships with pain outcomes. No variables demonstrated statistically significant relationships with length of pain > 1 week and length of pain > 2 weeks. The only significant association with pain outcomes was that HU users were less likely to take daily opioids. Individuals with SCD who are disabled or have a history of depression are more likely to report more severe and frequent pain. No relationship emerged between educational attainment and pain outcomes. As the results are limited to the cross-sectional design, we cannot make statements of causality. For now, we know that people with SCD and these risk factors need further study for interventions. We plan to further assess study participants across all eight SCDIC sites in the next phase of this work. Disclosures King: Bioline: Consultancy; Amphivena Therapeutics: Research Funding; Incyte: Consultancy; Cell Works: Consultancy; Celgene: Consultancy; Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novimmune: Research Funding; RiverVest: Consultancy; Tioma Therapeutics (formerly Vasculox, Inc.):: Consultancy; WUGEN: Equity Ownership.

Introduction In the United States 36% of adults have limited health literacy, which is associated with poor health outcomes (Kutner et al. National Assessment of Adult Literacy.2006). Health literacy is defined as "the capacity to obtain, process and understand basic health information and services needed to make appropriate health decisions" (Nielsen-Bohlman et al. National Academies Press.2004). Health literacy is understudied in sickle cell disease (SCD), a genetic disease affecting a minority population in the United States. SCD Preventive treatment requires patient engagement, therefore health literacy is an important determinant of health outcomes in SCD. Previous studies reported Health literacy in adolescents with SCD is suboptimal (Perry et al. J Pediatr Nur.2017), but few studies have investigated factors influencing health literacy in this population. Health literacy can be impacted by many factors such as cognition, socioeconomic status, education level and gender. This study evaluated health literacy level and factors that influenced Health literacy in adolescents and adults with SCD. In addition, we assessed the relationship of health literacy level and Hydroxyurea therapy use, one of only two FDA-approved disease-modifying therapies for SCD. Methods This was a cross- sectional study of adolescents and adults with SCD performed at St. Jude Children's Research hospital and St. Louis Children's hospital. Adolescents and adults ages 15 to 45 years completed a health literacy assessment using the newest vital sign (NVS). NVS is a validated tool with 6 questions that tests reading, mathematics and comprehension based on a nutrition label (Weiss et al. Ann Fam Med.2005). Score of 0-1 suggests high likelihood of limited health literacy, 2-3 indicates possibility of limited health literacy and 4-6 almost always suggests adequate health literacy. Self-reported demographic information was collected such as educational level, household income, age, sex, race. Additionally, SCD genotypes and hydroxyurea utilization were confirmed through chart review. Results A random sample of 125 adolescents and adults with SCD was evaluated (Table 1): 34 (28.1%) had Hb SC or Hb Sβ+-thalassemia, 87(71.9%) had Hgb SS or Hgb Sβ0-thalassemia, and 4 participants had unknown or other genotypes. Limited health literacy was prevalent, only 40 (32%) of subjects had adequate literacy, median NVS score was 2 for all participants. Participants with Hgb SS/Hgb Sβ0-thalassemia were more likely to be on hydroxyurea (p Conclusion This study shows a low prevalence of adequate health literacy amongst adolescents and adults with SCD. Our data corroborate previous literature which has reported limited health literacy in this population as well adds granularity to the factors that may influence this suboptimal outcome. There was a statistically significant association between health literacy, income level and education level in adolescents and young adults with SCD. Further, health literacy was not significantly associated with sickle cell genotype or hydroxyurea use. This study highlights the social determinants of health and specific need to address health literacy in SCD patients. To optimize the care of adolescents and young adults with SCD, targeted multimodal interventions that are tailored to low health literacy levels should be employed to reduce morbidity and mortality in this vulnerable population. Disclosures Kang: MBIO: Other: St. Jude Children's Research Hospital has an existing exclusive license and ongoing partnership with Mustang Bio for the further clinical development and commercialization of this XSCID gene therapy. King:Amphivena Therapeutics: Research Funding; Bioline: Consultancy; Celgene: Consultancy; Cell Works: Consultancy; Incyte: Consultancy; Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novimmune: Research Funding; RiverVest: Consultancy; Tioma Therapeutics (formerly Vasculox, Inc.):: Consultancy; WUGEN: Equity Ownership. Zhao:MBIO: Other: St. Jude Children's Research Hospital has an existing exclusive license and ongoing partnership with Mustang Bio for the further clinical development and commercialization of this XSCID gene therapy. Hankins:NHLBI: Research Funding; National Committee for Quality Assurance: Consultancy; Global Blood Therapeutics: Research Funding; NHLBI: Honoraria; ASPHO: Honoraria; LYNKS Foundation: Research Funding; Novartis: Research Funding; Bluebird Bio: Consultancy.

Background Sickle cell disease (SCD) is one of the most common genetic conditions worldwide. In the United States it affects over 100,000 people, primarily African Americans, with increasing prevalence. With the widespread implementation of prophylactic penicillin, pneumococcal vaccinations and the use of disease modifying therapies such as Hydroxyurea, survival amongst children with SCD has improved greatly. Nonetheless, the life expectancy for adults with SCD remains 20 years lower than African Americans without SCD. The adolescent and young adult period is well described as time of increased healthcare utilization, high morbidity and mortality for patients with SCD. As a result, increased efforts to understand and improve the transition from pediatric to adult care are ongoing. Previous literature has eluded to the importance of patients' disease specific knowledge in improving this transition. However, there is limited literature that examines knowledge gaps across multiple domains. The goal of this study was to use qualitative methods to understand the role of health knowledge in the successful transition from pediatric to adult care for patients with SCD. Methods Participants were recruited using a purposeful recruitment approach from a large hospital system and local community. Stratified qualitative focus groups with patients (ages 15-25 years with any genotype of SCD) and their parents; and providers who cared for SCD patients were held. Semi-structured interviews with emergency department (ED) providers were also conducted. An interview guide was developed that specifically addressed faciltators and barriers to essential healthcare and educational attainment for young adults with SCD. Using an inductive and emergent coding approach, field notes and transcripts were analyzed for themes/subthemes. A focused sub-analysis looking at health knowledge was then conducted. Transcripts were double-coded by trained coders, discrepancies were resolved by consensus. Results 62 participants (23 patients, 20 caregivers, 20 providers) completed a total of 11 focus groups (8 patient-caregiver dyad focus groups, 3 provider focus groups) and 5 semi-structured interviews with ED providers. Analysis showed knowledge gaps across four major domains: patients, caregivers, educators and in the occupational setting. Patients expressed the desire to know more about treatment options for SCD. They noted specific challenges with self-management as new symptoms of SCD developed in adolescence and disease management increased in complexity. Patients noted that increased disease education and earlier transition planning may be helpful to improve this gap. Caregivers noted challenges with supporting their loved ones during the transition period due to limited knowledge about disease complications and management as they age. Patients and caregivers noted that this was mediated by patient-provider communication. Patients expressed educators' lack of knowledge about SCD as a barrier in the school setting. Participants noted that the implementation of formalized educational plans as well as educating teachers improved their academic experiences. Similarly, participants expressed that the lack of understanding about SCD related disease complications was a barrier to success in the occupational setting. Conclusions Multi-level knowledge gaps exist when examining the transition from pediatric to adult care. This study supports previous literature that highlights the need for patient education to improve disease specific knowledge, health literacy and self-management for young adults transitioning to adult care. This data also suggests that educational interventions across multiple settings may optimize this process. Focused interventions designed to increase SCD specific knowledge amongst caregivers, educators and in the occupational setting represent a feasible and readily implementable point of impact to improve patient-provider communication and overall outcomes in adolescents and young adults with SCD. The successful transition from pediatric to adult care requires a multidisciplinary approach in which the onus is not simply on the patient, but all involved in the care of the patient. Closing knowledge gaps across multiple domains is essential in improving care, quality of life and educational attainment in this vulnerable population. Disclosures No relevant conflicts of interest to declare.

Higher Prevalence of Hydroxyurea Use Is Associated with Lower Hospitalization Rate in a Population of Children with Sickle Cell Disease Cecelia L. Calhoun, MD; Ryan Colvin, MPH; Sherry Lassa-Claxton, MS; Arti Verlekar MS; F. Sessions Cole, MD; Monica L. Hulbert, MD Background Individuals with sickle cell disease (SCD) treated with hydroxyurea (HU) in clinical trials have had fewer hospitalizations and vaso-occlusive pain episodes than those on placebo. Since 2014, HU has been recommended for all children with Hemoglobin SS/S-β0 thalassemia, and many SCD centers have increased HU utilization. The effect of expanded HU treatment on hospitalization rate has not been reported in a population of children clinically treated with HU. In a pediatric SCD center cohort, we tested the hypothesis that increasing prevalence of HU treatment was associated with reduced hospitalization rate per patient-year and in total hospitalizations. Methods PedsNet, a PCORI-funded clinical research initiative, was queried for all patients with inpatient or outpatient encounters for SCD at St. Louis Children's Hospital (SLCH) from 2010 through 2015. Data from patients with ICD-9 codes indicating any genotype of SCD were included. Patients were considered to be receiving HU when it was prescribed on 2 or more outpatient encounters. Exclusion criteria were lack of SCD diagnosis confirmation, lack of outpatient visits at SLCH, and presence of a significant unrelated medical condition. Patients were censored at their 21st birthday or date of hematopoietic stem cell transplant. Data generated at the adult hospital were excluded. All hospitalizations in the final cohort were included for analysis, regardless of diagnosis code. For comparisons, patients were grouped as having a severe genotype (SS, S-β0 thalassemia, SD) or less-severe genotype (SC, S-β+ thalassemia). Statistical analysis was performed using SAS 9.4. Logistic and Poisson regression were used to compare proportions and rates between groups, respectively. Generalized Estimating Equations were used to account for within patient correlation. Results The PedsNet query identified 646 patients. Sixty patients were excluded due to lack of SCD diagnosis, 78 due to lack of outpatient care, and 4 due to severe unrelated disease (3 with congenital disease/malformation, 1 with cancer), leaving 504 who met all inclusion criteria (51.6% male). Hematopoietic stem cell transplant was performed in 23 patients during the study period. Of patients with a severe genotype, 28 (20.1%) received HU in 2010 compared with 111 (65.6%) in 2015 (p Conclusions In this clinical cohort of children with SCD, increasing prevalence of HU treatment was associated with significantly fewer total hospitalizations and a lower hospitalization rate per patient-year. The constant hospitalization rate in the less-severe genotype group suggests that the reduction is attributable to HU, rather than to other changes in clinical practice affecting patients with all genotypes. Hospitalization rates were higher among HU-treated patients, suggesting that more symptomatic children remain more likely to receive HU despite published recommendations to treat all children with severe genotypes. Families and clinicians may be reluctant to initiate HU for primary prevention of pain and hospitalizations in asymptomatic children. Future work should examine HU effects on specific SCD complications and on costs of care. Family and clinician interventions are needed to promote HU utilization in targeted patient groups. Table. Table. Disclosures Hulbert: Pfizer, Inc.: Other: spouse employment.

Background: The transition from the pediatric setting to adult care is a challenge for many adolescents with chronic disease. Patients with sickle cell disease (SCD) represent a unique cohort as the timing of psychosocial development of adolescence often coincides with worsening end organ damage. Previously, we used the Adolescent Autonomy Checklist (AAC) modified to include SCD specific tasks that patients with SCD need to practice in order to transition to adult healthcare and independent living. This study sought to use the AAC to measure the effects of skill based educational handouts on improving self-management and transition readiness in adolescents with SCD. Methods: This was a single center, retrospective study approved by the Washington University Institutional Review Board. Inclusion criteria were patients with SCD, age 13-21 years, and completion of pre and post assessments. As standard care, patients from a pediatric hematology clinic completed the AAC-SCD. The AAC-SCD assesses skill level in twelve domains (Table). The tool includes 100 items, and users check "can do already" or "needs practice" for each item. After review with the coordinator, participants were given skill-based handouts based on up to five noted deficits. Patients completed the AAC-SCD at the subsequent clinic visit. In addition to baseline and follow up AAC-SCD data, medical and demographic data were collected via chart abstraction. All data were entered into SPSS for statistical analysis, including descriptives, paired sample T-tests, and bivariate Pearson's correlations. Results: A total of 61 patients completed baseline and follow up. Of those participants, 49.2% were female. The mean age was 15.4 (+ 2.2) years. The genotypic distribution was as follows: 67.2% HbSS, 19.7% HbSC, 3.3% HbS-beta-thal+ and 9.8% HbS-beta-thal0. The majority of patients received healthcare coverage via Medicaid (52.5%), private insurance (45.6%) and 1.6% had no insurance coverage. Twenty-five patients (42.0 %) had a history of stroke or silent cerebral infarct and 34 (55.7%) were currently taking or were previously prescribed hydroxyurea. Formal academic support (IEP or 504 Plan) was reported for 20 (32.8%) of patients. At baseline, patients needed the most help with skills in the kitchen, housekeeping, personal care and leisure. Statistically significant improvements (p< 0.05) occurred in skills related to laundry, housekeeping, healthcare, sexual development and living arrangements. Modest sized and statistically significant correlation between the receipt of the educational handouts and decreased number of items marked "needs help" occurred in the areas of money management (r=-0.27, p=0.044), vocational skills (r=-0.27, p=0.046;) and laundry (r=0.32, p=0.015). A post hoc analysis by age groups 13-15 (n= 34),16-18 (n=24) and 19-21 (n=3) showed a decreased amount of items marked "needs help" in the areas of sexual development for both 13-15 year olds (r=0.42, p=0.024) and 16-18 year olds (r=0.93, p=0.001) as well. Conclusion: Transition skills improved over time among adolescents with SCD. While we cannot say for certain if gains in knowledge occur with age as development progresses or if a formal transition program can be credited, providing educational materials on transition related skills within a clinic setting was associated with significant improvements in three of the domains. Our preliminary data offers insight into what skill deficits may be most amenable to educational interventions based on age group. As is the case with medical management, the development of a multimodal intervention is needed to prepare adolescents with SCD to transition to adult care and independent living. Clinic based education is a simple intervention that could be one component of future approaches to transition. Disclosures No relevant conflicts of interest to declare.