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Individuals with sickle cell disease (SCD) experience neurocognitive decline, low medication adherence, increased unemployment, and difficulty with instrumental activities of daily living (IADL). The relationship between self-perceived cognitive difficulties and IADLs, including employment, school enrollment, independence, engagement in leisure activities, and medication adherence is unknown. We hypothesized that self-reported difficulties across neurocognitive areas would predict lower IADL skills. Adolescent and adult participants of the multi-site Sickle Cell Disease Implementation Consortium (SCDIC) (n = 2436) completed patient-reported outcome (PRO) measures of attention, executive functioning, processing speed, learning, and comprehension. Cognitive symptoms were analyzed as predictors in multivariable modeling. Outcome variables included 1) an IADL composite that consisted of employment, participation in school, reliance on others, and leisure pursuits, and 2) hydroxyurea adherence. Participants reported cognitive difficulty across areas of attention (55%), executive functioning (51%), processing speed (57%), and reading comprehension (65%). Executive dysfunction (p < 0.001) and sometimes or often experiencing learning difficulties (p < 0.001 and p = 0.04) and poor comprehension (p = 0.000 and p = 0.001), controlled for age (p < 0.001), pain (p < 0.001), and hydroxyurea use (p = 0.001), were associated with poor IADL skills. Executive functioning difficulties (p = 0.021), controlled for age (p = 0.013 for ages 25-34), genotype (p = 0.001), and hemoglobin (p = 0.004), predicted hydroxyurea non-adherence. Analysis of PRO measures indicated that cognitive dysfunction is prevalent in adolescents and adults with SCD. Cognitive dysfunction translated into clinically meaningful outcomes. PRO of cognitive symptoms can be used as an important adjunct clinical tool to monitor symptoms that impact functional skills, including engagement in societal activities and medication adherence., (© 2021 Wiley Periodicals LLC.)

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., (©Anna M Hood, Heather Strong, Cara Nwankwo, Yolanda Johnson, James Peugh, Constance A Mara, Lisa M Shook, William B Brinkman, Francis J Real, Melissa D Klein, Rogelle Hackworth, Sherif M Badawy, Alexis A Thompson, Jean L Raphael, Amber M Yates, Kim Smith-Whitley, Allison A King, Cecelia Calhoun, Susan E Creary, Connie M Piccone, Aimee K Hildenbrand, Steven K Reader, Lynne Neumayr, Emily R Meier, Amy E Sobota, Sohail Rana, Maria Britto, Kay L Saving, Marsha Treadwell, Charles T Quinn, Russell E Ware, Lori E Crosby. Originally published in JMIR Research Protocols (https://www.researchprotocols.org), 21.05.2021.)

Background: Individuals living with sickle cell disease often require aggressive treatment of pain associated with vaso-occlusive episodes in the emergency department. Frequently, pain relief is poor. The 2014 National Heart, Lung, and Blood Institute evidence-based guidelines recommended an individualized treatment and monitoring protocol to improve pain management of vaso-occlusive episodes., Objective: This study will implement an electronic health record-embedded individualized pain plan with provider and patient access in the emergency departments of 8 US academic centers to improve pain treatment for adult patients with sickle cell disease. This study will assess the overall effects of electronic health record-embedded individualized pain plans on improving patient and provider outcomes associated with pain treatment in the emergency department setting and explore barriers and facilitators to the implementation process., Methods: A preimplementation and postimplementation study is being conducted by all 8 sites that are members of the National Heart, Lung, and Blood Institute-funded Sickle Cell Disease Implementation Consortium. Adults with sickle cell disease aged 18 to 45 years who had a visit to a participating emergency department for vaso-occlusive episodes within 90 days prior to enrollment will be eligible for inclusion. Patients will be enrolled in the clinic or remotely. The target analytical sample size of this study is 160 patient participants (20 per site) who have had an emergency department visit for vaso-occlusive episode treatment at participating emergency departments during the study period. Each site is expected to enroll approximately 40 participants to reach the analytical sample size. The electronic health record-embedded individualized pain plans will be written by the patient's sickle cell disease provider, and sites will work with the local informatics team to identify the best method to build the electronic health record-embedded individualized pain plan with patient and provider access. Each site will adopt required patient and provider implementation strategies and can choose to adopt optional strategies to improve the uptake and sustainability of the intervention. The study is informed by the Technology Acceptance Model 2 and the Reach, Effectiveness, Adoption, Implementation, and Maintenance framework. Provider and patient baseline survey, follow-up survey within 96 hours of an emergency department vaso-occlusive episode visit, and selected qualitative interviews within 2 weeks of an emergency department visit will be performed to assess the primary outcome, patient-perceived quality of emergency department pain treatment, and additional implementation and intervention outcomes. Electronic health record data will be used to analyze individualized pain plan adherence and additional secondary outcomes, such as hospital admission and readmission rates., Results: The study is currently enrolling study participants. The active implementation period is 18 months., Conclusions: This study proposes a structured, framework-informed approach to implement electronic health record-embedded individualized pain plans with both patient and provider access in routine emergency department practice. The results of the study will inform the implementation of electronic health record-embedded individualized pain plans at a larger scale outside of Sickle Cell Disease Implementation Consortium centers., Trial Registration: ClinicalTrials.gov NCT04584528; https://clinicaltrials.gov/ct2/show/NCT04584528., International Registered Report Identifier (irrid): DERR1-10.2196/24818., (©Lingzi Luo, Allison A King, Yvonne Carroll, Ana A Baumann, Donald Brambilla, Christopher R Carpenter, Joseph Colla, Robert W Gibson, Siera Gollan, Greg Hall, Lisa Klesges, Abdullah Kutlar, Matthew Lyon, Cathy L Melvin, Sarah Norell, Martina Mueller, Michael B Potter, Rachel Richesson, Lynne D Richardson, Gery Ryan, Lauren Siewny, Marsha Treadwell, Leslie Zun, Janelle Armstrong-Brown, Lisa Cox, Paula Tanabe. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 16.04.2021.)

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., (© 2020 by The American Society of Hematology.)

Understanding patient experiences, quality of life, and treatment needs in individuals with sickle cell disease (SCD) is essential in promoting health and well-being. We used measures from the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), Patient Reported Outcomes Measurement Information System (PROMIS), and Quality of Life in Neurological Disorders (NeuroQol) to evaluate pain impact, sleep impact, social functioning, depressive symptoms, tiredness, and cognitive function (collectively, patient reported outcomes [PROs]) and to identify associated demographic and clinical characteristics. Participants (n = 2201) between 18 and 45 years were recruited through the eight Sickle Cell Disease Implementation Consortium (SCDIC) sites. In multivariate models, PROs were significantly associated with one another. Pain impact was associated with age, education, employment, time since last pain attack, hydroxyurea use, opioid use, sleep impact, social functioning, and cognitive function (F = 88.74, P < .0001). Sleep impact was associated with household income, opioid use, pain impact, social functioning, depressive symptoms, and tiredness (F = 101.40, P < .0001). Social functioning was associated with employment, pain attacks in the past year, autoimmune/inflammatory comorbidities, pain impact, sleep impact, depressive symptoms, tiredness, and cognitive function (F = 121.73, P < .0001). Depressive symptoms were associated with sex, sleep impact, social functioning, tiredness, and cognitive function (F = 239.51, P < .0001). Tiredness was associated with sex, education, sleep impact, social functioning, depressive symptoms, and cognitive function (F = 129.13, P < .0001). These findings reflect the baseline PRO assessments among SCDIC registry participants. Further research is needed to better understand these outcomes and new targets for interventions to improve quality of life and function in people with SCD., (© 2020 Wiley Periodicals LLC.)

Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. It is a medically and socially complex, multisystem illness that affects individuals throughout the lifespan. Given improvements in care, most children with SCD survive into adulthood. However, access to adult sickle cell care is poor in many parts of the United States, resulting in increased acute care utilization, disjointed care delivery, and early mortality for patients. A dearth of nonmalignant hematology providers, the lack of a national SCD registry, and the absence of a centralized infrastructure to facilitate comparative quality assessment compounds these issues. As part of a workshop designed to train health care professionals in the skills necessary to establish clinical centers focused on the management of adults living with SCD, we defined an SCD center, elucidated required elements of a comprehensive adult SCD center, and discussed different models of care. There are also important economic impacts of these centers at an institutional and health system level. As more clinicians are trained in providing adult-focused SCD care, center designation will enhance the ability to undertake quality improvement and compare outcomes between SCD centers. Activities will include an assessment of the clinical effectiveness of expanded access to care, the implementation of SCD guidelines, and the efficacy of newly approved targeted medications. Details of this effort are provided., (© 2020 by The American Society of Hematology.)

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., (©Jane S Hankins, Nirmish Shah, Lisa DiMartino, Donald Brambilla, Maria E Fernandez, Robert W Gibson, Victor R Gordeuk, Richard Lottenberg, Abdullah Kutlar, Cathy Melvin, Jena Simon, Ted Wun, Marsha Treadwell, Cecelia Calhoun, Ana Baumann, Michael B Potter, Lisa Klesges, Hayden Bosworth, Sickle Cell Disease Implementation Consortium. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 14.07.2020.)

Purpose: Sickle cell disease (SCD) is associated with high acute healthcare utilization. The purpose of this study was to examine whether Medicaid expansion in California increased Medicaid enrollment, increased hydroxyurea prescriptions filled, and decreased acute healthcare utilization in SCD., Methods: Individuals with SCD (≤65 years and enrolled in Medicaid for ≥6 total calendar months any year between 2011 and 2016) were identified in a multisource database maintained by the California Sickle Cell Data Collection Program. We describe trends and changes in Medicaid enrollment, hydroxyurea prescriptions filled, and emergency department (ED) visits and hospital admissions before (2011-2013) and after (2014-2016) Medicaid expansion in California., Results: The cohort included 3635 individuals. Enrollment was highest in 2014 and lowest in 2016 with a 2.8% annual decease postexpansion. Although <20% of the cohort had a hydroxyurea prescription filled, the percentage increased by 5.2% annually after 2014. The ED visit rate was highest in 2014 and decreased slightly in 2016, decreasing by 1.1% annually postexpansion. Hospital admission rates were similar during the pre- and postexpansion periods. Young adults and adults had higher ED and hospital admission rates than children and adolescents., Conclusions: Medicaid expansion does not appear to have improved enrollment or acute healthcare utilization among individuals with SCD in California. Future studies should explore whether individuals with SCD transitioned to other insurance plans or became uninsured postexpansion, the underlying reasons for low hydroxyurea utilization, and the lack of effect on hospital admissions despite a modest effect on ED visits., (© 2020 Wiley Periodicals, Inc.)

Objectives: To evaluate knowledge, perceptions and the effectiveness of different sources of information about sickle cell trait (SCT) and sickle cell disease (SCD); to determine individual knowledge of SCT status., Methods: 28 individuals participated in three focus groups (healthcare providers, people affected by SCD or SCT, and community members). Surveyors interviewed 282 respondents within their neighborhoods., Results: Common themes across the focus groups included the limited general awareness of SCD and SCT, the emphasis on the benign nature of SCT rather than on future implications, and the need for public health education campaigns about SCD and SCT involving media strategies. The majority of community survey respondents (n = 243, 86.2%) had correct general knowledge about the genetic basis and severity of SCD, but only 16% (n = 45) knew their own trait status. When respondents had received information about SCD from friends and acquaintances, they were three times more likely to know their SCT status, compared with respondents who had not received information from a personal source (p < 0.01)., Conclusions: Despite a screening history in the 1970s fraught with controversy, sickle cell disease management and detection can be a model for the empowerment of communities in making informed decisions about theirs and their families' futures, given the burgeoning of genetic information.