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Abstract: Guidelines recommend transfer to adult health care within 6 months of completing pediatric care; however, this has not been studied in sickle cell disease (SCD). We hypothesized that longer transfer gaps are associated with increased resource utilization. Transfer gaps were defined as the time between the last pediatric and first adult visits. We estimated the association between varying transfer gaps and the rates of inpatient, emergency department (ED), and outpatient visits, using negative binomial regression. Health care utilization was evaluated in a mid-south comprehensive program for a follow-up period of up to 8 years (2012-2020) and was restricted to the first 2 years of adult health care. In total, 183 young adults (YAs) with SCD (51% male, 67% HbSS/HbSβ0-thalassemia) were transferred to adult health care between 2012 and 2018. YAs with transfer gaps ≥6 months compared with <2 months had 2.01 (95% confidence interval [CI], 1.31-3.11) times the rate of hospitalizations in the 8-year follow-up and 1.89 (95% CI, 1.17-3.04) when restricted to the first 2 years of adult health care. In the first 2 years of adult care, those with transfer gaps ≥6 months compared with <2 months, had 1.75 (95% CI, 1.10-2.80) times the rate of ED encounters. Those with gaps ≥2 to <6 months compared with <2 months had 0.71 (95 % CI, 0.53-0.95) times the rate of outpatient visits. Among YAs with SCD, a longer transfer gap was associated with increased inpatient and decreased outpatient encounters in adult health care and more ED encounters in the first 2 years of adult health care. Strategies to reduce the transfer gaps are needed., (© 2024 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.)

Sickle cell disease (SCD) remains a public health priority in the United States because of its association with complex health needs, reduced life expectancy, lifelong disabilities, and high cost of care. A cross-sectional analysis was conducted to calculate the crude and race-specific birth prevalence for SCD using state newborn screening program records during 2016-2020 from 11 Sickle Cell Data Collection program states. The percentage distribution of birth mother residence within Social Vulnerability Index quartiles was derived. Among 3,305 newborns with confirmed SCD (including 57% with homozygous hemoglobin S or sickle β-null thalassemia across 11 states, 90% of whom were Black or African American [Black], and 4% of whom were Hispanic or Latino), the crude SCD birth prevalence was 4.83 per 10,000 (one in every 2,070) live births and 28.54 per 10,000 (one in every 350) non-Hispanic Black newborns. Approximately two thirds (67%) of mothers of newborns with SCD lived in counties with high or very high levels of social vulnerability; most mothers lived in counties with high or very high levels of vulnerability for racial and ethnic minority status (89%) and housing type and transportation (64%) themes. These findings can guide public health, health care systems, and community program planning and implementation that address social determinants of health for infants with SCD. Implementation of tailored interventions, including increasing access to transportation, improving housing, and advancing equity in high vulnerability areas, could facilitate care and improve health outcomes for children with SCD., Competing Interests: All authors have completed and submitted the International Committee of Medical Journal Editors form for disclosure of potential conflicts of interest. Sophia S. Horiuchi reports ownership of Natera stock and stock options and of Roche stock by her spouse. Mariam Kayle reports institutional support from Agios Medical Education Program and Novo Nordisk Independent Medical Education Activity to support the 11th Annual Sickle Cell Conference, September 8–9, 2023; receipt of consulting fees from Loyola University; receipt of honorarium for being an ad hoc reviewer from the National Institutes of Health (NIH), Center for Scientific Review, Social and Environmental Determinants of Health Study Section; and uncompensated membership in the American Society of Hematology Research Collaborative Data Hub Oversight Group, Sickle Cell Disease Subcommittee. Carri S. Polick reports support from the Veterans Administration and Duke University Clinical and Translational Science Institute. Ashima Singh reports institutional support from NIH, National Heart, Lung, and Blood Institute, the National Institute of Neurological Disorders and Stroke, and the Health Resources and Services Administration (HRSA) Sickle Cell Treatment Demonstration Program. John J. Strouse reports institutional support from the HRSA Southeast Region Coordinating Center Sickle Cell Treatment Demonstration Program and the North Carolina State Department of Health Sickle Cell Syndrome Program for the Duke Adult and Pediatric Sickle Cell Program; royalties from UpToDate for preparation of guidance on the use of hydroxyurea for sickle cell disease (SCD); consulting fees for GLG telephone consultation on SCD and Guidepoint telephone consultation on SCD; receipt of honoraria from the University of Rochester-Equity in Sickle Cell Disease Care; payment for medical legal expert testimony from Emory University in a case involving emergency department care of an adult with sickle beta thalassemia; travel support from the American Society of Hematology Sickle Cell Learning Community Meeting and Sickle Cell Disease Association of America Meeting to speak on behalf of the American Society of Hematology and travel support from the American Thrombosis Hemostasis Network Data Summit as an invited speaker on gene therapy for SCD: Design for Equity; payment for participation on a data safety monitoring board for disc medicines for phase 1 trial planning for bitopertin for SCD; and service as vice president of the Sickle Cell Adult Provider Network. No other potential conflicts of interest were disclosed.

Competing Interests: JSH receives consultancy fees from Global Blood Therapeutics, CVS Health and Forma Therapeutics during the conduct of this study. JSP received consultancy fees from Forma Therapeutics and funding from the National Heart Lung and Blood Institute K01 HL125495 during the conduct of this study. All the other authors have no conflicts of interest to disclose.

Objective: Population-level data on sickle cell disease (SCD) are sparse in the United States. The Centers for Disease Control and Prevention (CDC) is addressing the need for SCD surveillance through state-level Sickle Cell Data Collection Programs (SCDC). The SCDC developed a pilot common informatics infrastructure to standardize processes across states., Materials and Methods: We describe the process for establishing and maintaining the proposed common informatics infrastructure for a rare disease, starting with a common data model and identify key data elements for public health SCD reporting., Results: The proposed model is constructed to allow pooling of table shells across states for comparison. Core Surveillance Data reports are compiled based on aggregate data provided by states to CDC annually., Discussion and Conclusion: We successfully implemented a pilot SCDC common informatics infrastructure to strengthen our distributed data network and provide a blueprint for similar initiatives in other rare diseases., Competing Interests: Dr Matthew Smeltzer has worked as a paid research consultant for the Association of Community Cancer Centers. The findings and conclusions in this publication are those of the authors and do not necessarily represent the views of the North Carolina Department of Health and Human Services, Division of Public Health. No other authors have competing interests to declare., (© The Author(s) 2023. Published by Oxford University Press on behalf of the American Medical Informatics Association.)

Sickle cell disease (SCD) state level surveillance data are limited. We performed a retrospective review of emergency department (ED) visits and hospitalizations from individuals with SCD in Illinois (2016-2020) using the Illinois Health and Hospital Association's Comparative Health Care and Hospital Data Reporting Services. There were 48,094 outpatient ED visits and 31,686 hospitalizations. Most visits (67%) occurred in Cook County, were covered by public insurance (77%) and were from individuals with medium high (40.3%) or high (36.1%) poverty levels. SCD healthcare utilization remains high and surveillance data may inform SCD program development and resource allocation at the state level.AbbreviationsCDCCenters for Disease Control and PreventionEDEmergency DepartmentFDAFood & Drug AdministrationICDInternational Classification of DiseasesILIllinoisSCDSickle cell disease.

Purpose: Clean intermittent catheterization (CIC) responsibility among youths with spina bifida is not well studied. We sought to determine longitudinal trajectories of CIC responsibility to examine the transition of CIC responsibility from caregiver-CIC to self-CIC., Materials and Methods: We performed a secondary analysis of a prospective cohort study of youths with spina bifida. Participants aged 8-15 years originally recruited from 4 hospitals and a statewide spina bifida association were followed every 2 years. Participants who required CIC were included. Group-based trajectory modeling was used to isolate distinct trajectories of CIC responsibility, which was the primary outcome and was graded from caregiver-CIC to shared-CIC to self-CIC. Predictors of trajectory group membership were entered into multivariate logistic regression models and included various demographic, clinical and psychosocial characteristics such as CIC adherence and CIC mastery., Results: Of 140 youths in the original cohort study, 89 met eligibility criteria for this study. Mean age was 11 years at enrollment and 93% of patients had myelomeningocele. Two distinct trajectory groups emerged: 17% of patients had a low-flat trajectory and 83% had a high-increasing trajectory of CIC responsibility, with shared-CIC by age 8-9 years and increasing self-CIC responsibility thereafter. Significant predictors of group membership in the high-increasing trajectory group included less severe spinal lesion levels, higher CIC mastery and lower CIC adherence., Conclusions: Nearly 1 in 5 youths with spina bifida in our cohort persistently required caregiver-CIC over time, while the remainder achieved shared-CIC responsibility by age 8-9 years, with increasing self-CIC responsibility thereafter.

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.)

Objective: To determine if there are distinct developmental trajectories of medical responsibility in youth with spina bifida (SB) across ages 8-17 years and to identify condition-related, parental, and family systems predictors of membership in these trajectory groups., Methods: Participants were 140 youth with SB and their parents who participated in four waves of a longitudinal study across 6 years (ages 8-15 years at Time 1). Multi-method (questionnaires and observed family interactions) and multi-respondent assessments were conducted during home visits., Results: Findings revealed that there were two distinct developmental trajectories that characterized this sample, with one being labeled "high increasing" (two thirds of the sample) and one labeled "low increasing" (one third of the sample). Most predictor variables were significantly associated with trajectory group membership, with the exception of ethnicity, SES, and measures of conflict. When all significant univariate predictors were included in the same model, only intelligence quotient (IQ), family stress, and gender were retained as significant., Conclusions: Most youth exhibited relatively rapid increases in responsibility over the course of late childhood and adolescence, but there was a smaller portion of the sample that did not exhibit this type of developmental trajectory. The magnitude of the IQ effect on group differentiation appeared to attenuate the effects of most other predictors. It will be important for clinicians working with youth with SB to recognize that the transfer of medical responsibility from parent to child cannot be expected to unfold in the same manner for all families of youth with SB., (© The Author(s) 2020. Published by Oxford University Press on behalf of the Society of Pediatric Psychology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

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.)

Background: Sickle cell disease (SCD) is a chronic blood disorder in which mortality has increased for adolescents and young adults (AYA)., Procedure: A longitudinal analysis of medical records was conducted to describe the clinical course among AYAs (ages 12-27 years) during transition to adult care. Measures included sociodemographic, complications, SCD severity (modified pediatric SCD severity index), comorbidities, and transfer. Group-based trajectory modeling (GBTM) to identify subgroups with distinct severity trajectories and chi-square and unpaired Student t test to explore subgroup differences were used., Results: Overall, 339 AYAs (97% black, 56% male, 69% hemoglobin SS) had 10 848 clinic, 3840 hospital, and 3152 emergency department visits. Complications included vaso-occlusive crises (80%) and acute chest syndrome (41%). Comorbidities included depression (19%) and anxiety (14%). Most AYAs transferred to adult care (n = 220) at 19 years. Fourteen AYAs died, 10 within seven years from transfer. GBTM identified both stable and increasing severity trajectory groups: stable-low (n = 31, 23%), stable-medium (n = 61, 46%), stable-high (n = 6, 4.5%), low-increasing (n = 13, 10%), and medium-increasing (n = 22, 17%). AYAs with increasing severity (25%) were older, lived closer to the clinic, and had higher risk for SCD complications and comorbidities. They had fewer pediatric clinic visits; however, they were more likely to transfer and remain longer in adult SCD care., Conclusions: Whereas most AYAs had stable severity, nearly a quarter had increasing severity, over time. AYAs with increasing severity had more complications, were more likely to transfer to adult care, and demonstrated higher and longer adult SCD care utilization compared with AYAs with stable severity., (© 2018 Wiley Periodicals, Inc.)

This study explored the challenges faced by adolescents with sickle cell disease (SCD) and their parents and the work they engage in to progressively shift from parent management to independent adolescent self-management., Design and Methods: A qualitative descriptive focus-group design with semi-structured interviews was used with adolescents (11-18 years) with SCD (HbSS genotype) and their parents/primary caregivers. Interviews were analyzed using content analysis., Results: Two adolescent focus groups, with a total of 14 adolescents, and two parent focus groups, with a total of 15 parents, described adaptive challenges. Adolescents' adaptive challenges included mastering complex symptom management, communicating about SCD and symptoms, and maintaining control. Parents' adaptive challenges included giving over the complex management, communicating the management with the adolescent, balancing protection against risk with fostering independence, changing a comfortable rhythm, and releasing the adolescent into an "SCD-naive" world. Adolescents' adaptive work included pushing back at parents, defaulting back to parental care, stepping up with time, learning how SCD affects them, and educating friends about SCD. Parents' adaptive work included engaging the adolescent in open dialogue and co-managing with the adolescent., Conclusions: Shifting management responsibility from parents to adolescents imposes adaptive challenges for both. Future research is needed to develop and test interventions that improve adaptive capacity in adolescents and parents., Practice Implications: Health care providers need to assess the parent-child relationship and their progress in shifting the management responsibility, facilitate discussions to arrive at a shared understanding of the challenges, and collaborate on adaptive work to address these challenges., (Copyright © 2016 Elsevier Inc. All rights reserved.)

Sickle cell disease (SCD) is a complex multisystem debilitating disease. Despite its complexity, health care providers who are not SCD experts receive little formal education on SCD. An open-access, educational website, "Emergency Department Sickle Cell Disease: Crisis Management and Beyond," was created to provide education about SCD to emergency department (ED) providers who are not SCD experts but who provide care for patients with SCD. Electronic surveys were used to conduct a formal evaluation of the accuracy and relevance of the website's content, as well as the effectiveness of the education modules in improving knowledge among health care providers. The evaluation consisted of (1) individual module pre- and post-knowledge assessment, (2) content validity assessment of educational modules, (3) overall website content assessment, and (4) overall website assessment (Health on the Net core principles). A convenient sample of ED providers, accelerated bachelor of science in nursing students, SCD experts, and website experts completed the anonymous surveys. Descriptive statistics and paired t tests were used to compare mean difference in post- minus pre-knowledge test scores. Knowledge scores statistically improved for nursing students (p value less than 0.0001). Emergency department providers showed a mean improvement of 3.2 points on the eight-item knowledge assessment. Both SCD experts and ED providers agreed that the module content was clear and easy to understand, accurate, comprehensive, relevant, and met module objectives. Participants agreed that the website was clear, easy to navigate, and visually appealing. Website experts stated that the website met much of the Health on the Net criteria. The website is a useful resource for providers and nursing students, especially those who serve or plan to serve in EDs.

Objectives: Using a Life Course Health Development framework, this article summarizes what is known about the impact of cancer and its treatment on the biopsychosocial world of the adolescent and young adult., Data Sources: Published peer reviewed literature, web-based resources, and cancer-related professional organizations' resources., Conclusion: Adolescents and young adults with cancer, between 15 and 29 years of age, have emerged as a distinct group requiring specialized care. The demands of cancer and its treatment are often directly counter to the developmental needs of this age group and often alter those life course experiences that contribute to resilience, thriving, and flourishing., Implications for Nursing Practice: Providing high-quality care to this age group requires a depth of understanding of the complexity of factors that merge to influence the developmental life course., (Copyright © 2015 Elsevier Inc. All rights reserved.)

Patients with sickle cell disease (SCD) often seek care in the emergency department (ED) for pain associated with vaso-occlusive crises. Research has shown that negative provider attitudes serve as a barrier to care in this patient population. Our aim was to validate a survey that measures attitudes toward SCD patients among ED providers (nurses and physicians) and to compare differences in attitude scores between provider types. We administered the general perceptions about Sickle Cell Disease Patients Scale, previously validated among internal medicine providers, and the Medical Condition Regard Scale (MCRS) to ED nurses and physicians from two EDs. A total of 215 surveys were returned (63% response rate, 200 with attitude items completed). Three subscales were identified: Negative Attitudes, Uneasiness With Care, and Positive Attitudes. Cronbach's αs exceeded 0.81 for each subscale. Mean (SD) scores for the Negative, Uneasiness, and Positive subscales and MCRS were 61.5 (20.3), 66.1 (17.1), 41.2 (17.8), and 42.2 (8.9), respectively. Compared with physicians, nurses had significantly higher mean Negative Attitude scores and lower Uneasiness scores. A slightly modified version of the general perceptions about Sickle Cell Disease Patients Scale appears to be a valid measure of ED provider attitudes toward SCD patients. Among ED providers, this scale identified a dimension not observed in research with the original instrument among internal medicine providers. Provider attitudes influence patient-provider interactions and quality of care. The scale we present here has major clinical implications, particularly for advanced practice nurses, who can use the scale not only to assess providers' attitudes toward SCD patients but also to determine the effectiveness of tailored interventions to improve those attitudes.

Background: To explore physicians' opinions and attitudes regarding resuscitation of extremely premature infants (EPIs) in a developing country with suboptimal resources., Methods: A survey was developed, revised, and pilot-tested. All 964 paediatricians registered in the Lebanese Order of Physicians were contacted; physicians involved in resuscitation of EPIs were eligible. Between February and April of 2009, anonymous surveys were mailed to consenting participants., Results: Three hundred twenty-eight eligible physicians agreed to participate. One hundred twenty (36%) returned the survey, 45.3% of which were neonatologists. The vast majority agreed that parents would like to be informed and to participate in the resuscitation decision of an EPI. The majority of physicians considered infants at gestational age of ≤25 weeks (78%) or ≤800 g (89%) as non-viable. Physician's age, years of practice, and practising neonatal intensive care unit level were significantly associated with the choice of birthweight at which infants were considered non-viable., Conclusions: The majority of surveyed physicians consider infants at gestational age less than or equal to 25 weeks gestation or 800 g at birth as non-viable, and therefore would not attempt their resuscitation. Factors influencing threshold of viability in developing countries need to be addressed and explored further., (© 2014 John Wiley & Sons Ltd.)