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Objective: To test the hypothesis that caregivers' or adult participants' low ratings of provider communication are associated with more hospital admissions among adults and children with sickle cell disease (SCD), respectively. Secondarily, we determined whether there was an association between the caregivers' or participants' health literacy and rating of providers' communication. Methods: Primary data were collected from participants through surveys between 2014 and 2016, across six sickle cell centers throughout the U.S. In this cross-sectional cohort study, 211 adults with SCD and 331 caregivers of children with SCD completed surveys evaluating provider communication using the Consumer Assessment of Healthcare Providers and Systems (CAHPS), healthcare utilization, health literacy, and other sociodemographic and behavioral variables. Analyses included descriptive statistics, bivariate analyses, and logistic regression. Results: Participants with better ratings of provider communication were less likely to be hospitalized (odds ratio (OR) = 0.54, 95% confidence interval (CI) = [0.35, 0.83]). Positive ratings of provider communication were associated with fewer readmissions for children (OR = 0.23, 95% CI = [0.09, 0.57]). Participants with better ratings of provider communication were less likely to rate their health literacy as lower (regression coefficient (B) = -0.28, 95% CI = [-0.46, -0.10]). Conclusions: Low ratings of provider communication were associated with more hospitalizations and readmissions in SCD, suggesting the need for interventions targeted at improving patient-provider communication which could decrease hospitalizations for this population.

Outpatient care is critical in the management of chronic diseases, including sickle cell disease (SCD). Risk factors for poor adherence with clinic appointments in SCD are poorly defined. This exploratory study evaluated associations between modifying variables from the Health Belief Model and missed appointments.We surveyed adults with SCD (n = 211) and caregivers of children with SCD (n = 331) between October 2014 and March 2016 in six centres across the U.S. The survey tool utilized the framework of the Health Belief Model, and included: social determinants, psychosocial variables, social support, health literacy and spirituality.A majority of adults (87%) and caregivers of children (65%) reported they missed a clinic appointment. Children (as reported by caregivers) were less likely to miss appointments than adults (OR:0.22; 95% CI:(0.13,0.39)). In adults, financial insecurity (OR:4.49; 95% CI:(1.20, 20.7)), health literacy (OR:4.64; 95% CI:(1.33, 16.15)), and age (OR:0.95; 95% CI:(0.91,0.99)) were significantly associated with missed appointments. In all participants, lower spirituality was associated with missed appointments (OR:1.83; 95%CI:(1.13, 2.94)). The most common reason for missing an appointment was forgetfulness (adults: 31%, children: 26%). A majority thought reminders would help (adults: 83%, children: 71%) using phone calls (adults: 62%, children: 61%) or text messages (adults: 56%, children: 51%).Our findings demonstrate that modifying components of the Health Belief Model, including age, financial security, health literacy, spirituality, and lacking cues to action like reminders, are important in missed appointments and addressing these factors could improve appointment-keeping for adults and children with SCD.

Objective: Hospital admissions are significant events in the care of individuals with sickle cell disease (SCD) due to associated costs and potential for quality of life compromise. Methods: This cross-sectional cohort study evaluated risk factors for admissions and readmissions between October 2014 and March 2016 in adults with SCD (n = 201) and caregivers of children with SCD (n = 330) at six centres across the U.S. Survey items assessed social determinants of health (e.g. educational attainment, difficulty paying bills), depressive symptoms, social support, health literacy, spirituality, missed clinic appointments, and outcomes hospital admissions and 30-day readmissions in the previous year. Results: A majority of adults (64%) and almost half of children (reported by caregivers: 43%) were admitted, and fewer readmitted (adults: 28%; children: 9%). The most common reason for hospitalization was uncontrolled pain (admission: adults: 84%, children: 69%; readmissions: adults: 83%, children: 69%). Children were less likely to have admissions/readmissions than adults (Admissions: OR: 0.35, 95% CI: [0.23,0.52]); Readmissions: 0.23 [0.13,0.41]). For all participants, missing appointments were associated with admissions (1.66 [1.07, 2.58]) and readmissions (2.68 [1.28, 6.29]), as were depressive symptoms (admissions: 1.36 [1.16,1.59]; readmissions: 1.24 [1.04, 1.49]). In adults, difficulty paying bills was associated with more admissions, (3.11 [1.47,6.62]) readmissions (3.7 [1.76,7.79]), and higher spirituality was associated with fewer readmissions (0.39 [0.18,0.81]). Discussion: Missing appointments was significantly associated with admissions and readmissions. Findings confirm that age, mental health, financial insecurity, spirituality, and clinic attendance are all modifiable factors that are associated with admissions and readmissions; addressing them could reduce hospitalizations.

Silent cerebral infarcts are the most common neurologic injury in children with sickle cell anemia and are associated with the recurrence of an infarct (stroke or silent cerebral infarct). We tested the hypothesis that the incidence of the recurrence of an infarct would be lower among children who underwent regular blood-transfusion therapy than among those who received standard care.In this randomized, single-blind clinical trial, we randomly assigned children with sickle cell anemia to receive regular blood transfusions (transfusion group) or standard care (observation group). Participants were between 5 and 15 years of age, with no history of stroke and with one or more silent cerebral infarcts on magnetic resonance imaging and a neurologic examination showing no abnormalities corresponding to these lesions. The primary end point was the recurrence of an infarct, defined as a stroke or a new or enlarged silent cerebral infarct.A total of 196 children (mean age, 10 years) were randomly assigned to the observation or transfusion group and were followed for a median of 3 years. In the transfusion group, 6 of 99 children (6%) had an end-point event (1 had a stroke, and 5 had new or enlarged silent cerebral infarcts). In the observation group, 14 of 97 children (14%) had an end-point event (7 had strokes, and 7 had new or enlarged silent cerebral infarcts). The incidence of the primary end point in the transfusion and observation groups was 2.0 and 4.8 events, respectively, per 100 years at risk, corresponding to an incidence rate ratio of 0.41 (95% confidence interval, 0.12 to 0.99; P=0.04).Regular blood-transfusion therapy significantly reduced the incidence of the recurrence of cerebral infarct in children with sickle cell anemia. (Funded by the National Institute of Neurological Disorders and Stroke and others; Silent Cerebral Infarct Multi-Center Clinical Trial ClinicalTrials.gov number, NCT00072761, and Current Controlled Trials number, ISRCTN52713285.).

Background : Approximately 100,000 persons with sickle cell disease (SCD) live in the United States, including 15,000 in the Midwest. Unfortunately, many patients experience poor health outcomes due to limited access to primary care providers (PCPs) who are prepared to deliver evidence-based SCD care. Sickle Treatment and Outcomes Research in the Midwest (STORM) is a regional network established to improve care and outcomes for individuals with SCD living in Indiana, Illinois, Michigan, Minnesota, Ohio, and Wisconsin. Methods : STORM investigators hypothesized that Project ECHO ® methodology could be replicated to create a low-cost, high-impact intervention to train PCPs in evidence-based care for pediatric and young adult patients with SCD in the Midwest, called STORM TeleECHO. This approach utilizes video technology for monthly telementoring clinics consisting of didactic and case-based presentations focused on the National Heart, Lung and Blood Institute (NHLBI) evidence-based guidelines for SCD. Results : Network leads in each of the STORM states assisted with developing the curriculum and are recruiting providers for monthly clinics. To assess STORM TeleECHO feasibility and acceptability, monthly attendance and satisfaction data are collected. Changes in self-reported knowledge, comfort, and practice patterns will be compared with pre-participation, and 6 and 12 months after participation. Conclusions : STORM TeleECHO has the potential to increase implementation of the NHLBI evidence-based guidelines, especially increased use of hydroxyurea, resulting in improvements in the quality of care and outcomes for children and young adults with SCD. This model could be replicated in other pediatric chronic illness conditions to improve PCP knowledge and confidence in delivering evidence-based care. Keywords: provider education; telementoring; continuing education; sickle cell disease (Published: 24 November 2016) Citation: Med Educ Online 2016, 21: 33616 - http://dx.doi.org/10.3402/meo.v21.33616

The most common form of neurologic injury in sickle cell anemia (SCA) is silent cerebral infarction (SCI). In the Silent Cerebral Infarct Multi-Center Clinical Trial, we sought to identify risk factors associated with SCI. In this cross-sectional study, we evaluated the clinical history and baseline laboratory values and performed magnetic resonance imaging of the brain in participants with SCA (HbSS or HbSβ° thalassemia) between the ages of 5 and 15 years with no history of overt stroke or seizures. Neuroradiology and neurology committees adjudicated the presence of SCI. SCIs were diagnosed in 30.8% (251 of 814) participants who completed all evaluations and had valid data on all prespecified demographic and clinical covariates. The mean age of the participants was 9.1 years, with 413 males (50.7%). In a multivariable logistic regression analysis, lower baseline hemoglobin concentration (P < .001), higher baseline systolic blood pressure (P = .018), and male sex (P = .030) were statistically significantly associated with an increased risk of an SCI. Hemoglobin concentration and systolic blood pressure are risk factors for SCI in children with SCA and may be therapeutic targets for decreasing the risk of SCI. This study is registered at www.clinicaltrials.gov as #NCT00072761.

This article describes the planning, implementation, and evaluation of Research and Education Day, an event designed to promote education and awareness of sickle cell disease (SCD), while also fostering and increasing participation by families in research studies. Quantitative and qualitative evaluation data (tracking of participant attendance and participation in research studies; program evaluation surveys) indicate that program goals were achieved. Results show that event attendance, research participation and retention, and community volunteerism has increased exponentially over the 8-year history of the event. Parents report that their top-rated reasons for attending are learning about research studies and participating in SCD-related educational activities. Data also show that families learn about the event in multiple ways (i.e., flyers, clinic staff, word of mouth, etc.). Research and Education Day is a model for educating participants with sickle cell and their families, engaging them in research studies, and enhancing community awareness and support.

The purpose of this study was to examine perceived barriers to clinic attendance and strategies to overcome these barriers for adolescents with sickle cell disease.This was a 2-phased study, which used focus groups (n=13) and individual semistructured interviews (n=32) with adolescent patients (aged 13 to 21 y) from 3 pediatric sickle cell clinics in the Midwest.Adolescents identified competing activities, health status, patient-provider relationships, adverse clinic experiences, and forgetting as barriers to clinic attendance. Calendars/reminders and parent reminders were the most commonly reported strategies to facilitate clinic attendance. Adolescents also reported the need for flexible scheduling and improved patient-provider communication.Adolescents with sickle cell disease and their families may benefit from ongoing education about the importance of attending routine clinic visits. Adherence to clinic appointments for adolescents may be enhanced by developing interventions to decrease forgetting (eg, phone call reminders or text messaging) and increase patient satisfaction with clinic visits. Scheduling appointments to accommodate busy schedules/scheduling conflicts (eg, late clinic hours), providing teen-friendly clinic environments, and using technology may also facilitate attendance.

Keywords: sickle cell disease; atypical haemolytic uraemic syndrome; eculizumab; thrombotic microangiopathy; complement mutations

Iron-overload associated endocrinopathy is the most frequently reported complication of chronic transfusion therapy in patients with thalassaemia (Thal). This study compared iron-overloaded subjects with Thal (n = 142; 54%M; age 25.8 +/- 8.1 years) and transfused sickle-cell disease (Tx-SCD; n = 199; 43%M, 24.9 +/- 13.2 years) to non-transfused SCD subjects (non-Tx-SCD; n = 64, 50%M, 25.3 +/- 11.3 years), to explore whether the underlying haemoglobinopathy influences the development of endocrinopathy. Subjects were recruited from 31 centres in the USA, Canada and the UK. Subjects with Thal had more evidence of diabetes (13% vs. 2%, P < 0.001), hypogonadism (40% vs. 4%, P < 0.001), hypothyroidism (10% vs. 2%, P =

Objective To investigate the relationship between neuropsychological functioning and radiographic findings in children with sickle cell disease (SCD) with no history of clinical neurological events. Methods Thirty-one patients with SCD randomly selected from a regional treatment center underwent neuropsychological and disease severity assessments. Of these, 22 also had structural magnetic resonance imaging and magnetic resonance perfusion studies performed. Results Forty-five percent of the imaged subgroup showed imaging abnormalities that were found to be correlated with disease severity but not neuropsychological level of performance indices. A significant relationship, however, was found between imaging abnormalities and increased variability in neuropsychological performance. Conclusions These results corroborate the high rate of rheologic and vascular pathology in SCD and underscore the importance of representing neuropsychological functioning in multiple ways.

Objective To investigate the feasibility and efficacy of a randomized clinical pilot trial comparing routine services (RS) with a school intervention program (SIP) for children with sickle cell anemia (SCA). Method Twenty-four children (ages 8-12 years) with SCA were randomized to RS or SIP. General disease knowledge, consumer satisfaction, self-concept, and school absences were evaluated. Results Compared with children receiving RS, children and teachers receiving SIP evidenced more accurate information about their disease, and children with SCA had significantly fewer absences. Teachers receiving SIP reported higher consumer satisfaction. Conclusions A modest educational curriculum can increase knowledge of SCA, is associated with lower absence rates, and yields high consumer satisfaction ratings.

Elevated plasma homocysteine levels have been shown to be a risk factor for endothelial cell damage and thrombosis, which are implicated in sickle cell disease (SCD)-related vaso-occlusion. The aim of this study was to determine the prevalence of hyperhomocysteinemia in SCD. Fasting and postmethionine load (PML) homocysteine, red cell folate, and the MTHFR C677T mutation were determined in 77 patients with SCD and 110 African-American controls. Plasma methylmalonic acid and pyridoxine levels were determined in 54 patients and all controls. For analysis, the subjects were divided into two age groups (2-10 years and 10.1-21 years). In both age groups, median PML homocysteine levels were significantly elevated in patients with SCD compared with controls. Fasting homocysteine levels were elevated in patients with SCD versus controls only in those older than 10 years. Hyperhomocysteinemia was noted in 38% of patients versus 7% in controls. Folate levels were higher among patients than controls and showed a significant negative correlation with PML homocysteine levels in patients with SCD. Pyridoxine levels in patients with SCD were significantly lower than in controls and showed a negative correlation with PML homocysteine levels. Among patients with SCD, pyridoxine deficiency was more common (62%) among those with hyperhomocysteinemia compared with those with normal homocysteine levels (30%). Homozygosity for the MTHFR C677T mutation was rare. These data suggest that children with SCD have significant hyperhomocysteinemia, associated with pyridoxine and relative folate deficiencies.

Recently, a mild to moderate elevation in the plasma homocysteine (Hcy) level has been found to be an important risk factor for stroke. Homozygosity for a common mutation (C677T) in the gene encoding for the enzyme methylenetetrahydrofolate reductase (MTHFR) involved in Hcy metabolism has been associated with increased levels of Hcy. To determine the role of hyperhomocysteinemia in the pathogenesis of stroke in children with sickle cell disease (SCD), Hcy levels and C677T MTHFR genotype were determined in 40 patients homozygous for hemoglobin SS and compared with 197 healthy children. Eleven of 40 patients with SCD had a history of stroke. The prevalence of homozygosity for the C677T MTHFR variant was 5% in the patients with SCD. The median Hcy level was 5.8 micromol/L in the patients versus 5.4 micromol/L in the controls (Fisher's, P > 0.05). There was no correlation of Hcy levels with the MTHFR genotype in patients with SCD. In patients with SCD and stroke, the median Hcy level was 4.8 micromol/L versus 6.0 micromol/L in those without stroke (P = 0.44, Mann-Whitney rank sum test). There was no difference in the proportion of patients with SCD with or without stroke who were homozygous for the C677T MTHFR mutation (0/11 versus 2/29; Fisher's, P = 1.000). In conclusion, this study failed to demonstrate an elevation in plasma Hcy levels in children with SCD compared with normal controls. Furthermore, hyperhomocysteinemia did not seem to be a significant factor in the pathogenesis of stroke in children with SCD.

OBJECTIVES: (1) To determine serotype-specific IgG antibody responses to reimmunization with pneumococcal polysaccharide vaccine at age 5 years in children with sickle cell anemia and (2) to determine whether continued penicillin prophylaxis had any adverse effects on these responses. STUDY DESIGN: Children with sickle cell anemia, who had been treated with prophylactic penicillin for at least 2 years before their fifth birthday, were randomly selected at age 5 years to continue penicillin prophylaxis or to receive placebotreatment. These children had been immunized once or twice in early childhood with pneumococcal polysaccharide vaccine and were reimmunized at the time of randomization. RESULTS: Serotype-specific IgG antibody responses to reimmunization varied according to pneumococcal serotype but in general were mediocre or poor; the poorest response was to serotype 6B. The antibody responses were similar in subjects with continued penicillin prophylaxis or placebo treatment, and in subjects who received one or two pneumococcal vaccinations before reimmunization. The occurrence of pneumococcal bacteremia was associated with low IgG antibody concentrations to the infecting serotype. CONCLUSIONS: Reimmunization of children with sickle cell anemia who received pneumococcal polysaccharide vaccine at age 5 years induces limited production of serotype-specific IgG antibodies, regardless of previous pneumococcal vaccine history. Continued penicillin prophylaxis does not interfere with serotype-specific IgG antibody responses to reimmunization. (J Pediatr 1996;129:828-35)

Comparisons with measures of peer relationships and emotional well-being were made between youngsters with sickle cell disease (SCD) and same-classroom comparison peers. Relative to the comparison subjects, females with SCD were perceived by peers as being less sociable and less well accepted; males with SCD were perceived as being less aggressive than comparison peers. For both males and females with SCD, no other differences were identified on numerous measures of emotional well-being. None of the multiple measures of illness severity were significantly related to measures of psychological adjustment. The common side effects of SCD, chronic fatigue and small physical size, may divert males with the illness from manifesting difficulties related to aggressive behavior with peers. For females with the illness, the common side effects of the illness may hinder the development of normal social relationships. Despite chronic exposure to numerous stressful life events associated with SCD, the youngsters with the illness were remarkably similar to comparison peers, showing evidence of considerable hardiness.

Examined the peer relationships of siblings of children with SCA (N = 37), comparing them to a matched group of classroom comparison children who were the same race/gender, closest date of birth. Social reputation was examined from the perspective of teacher and peers; peer ratings and nominations of social acceptance were obtained; and information was obtained about the sibling's own view of their peer relationships. No differences were found between the two groups, suggesting that the overall functioning of this group of siblings was comparable to peers in their classrooms. Findings are discussed in terms of professional biases, the lack of empirical data relevant to the issue of sibling competence, and resilience.

Objective To assess the effects of chronic erythrocyte transfusions on prevalence of sonographic incidence of organ damage in children with sickle cell anemia (SCA). Study design Children (N = 148; mean age, 13.0 years) with SCA, receiving chronic transfusions (average, 7 years), underwent abdominal sonography at 25 institutions. After central imaging review, spleen, liver, and kidney measurements were compared with published normal values. Potential relations between ultrasound, clinical, and laboratory data were explored via analysis of variance, Student t test, and Cochran-Mantel-Haenzel tests of non-zero correlation. Results Average spleen length was similar to normal children, but over one-third had spleen volumes >300 mL, 15 had previous splenectomy for splenomegaly, and 24 had abnormal splenic echotexture. Two-thirds had hepatobiliary disease; 37 had prior cholecystectomy, 46 had gallstones, and 16 had gallbladder sludge. Gallbladder disease correlated with older age ( P = .002), longer liver length ( P P = .034), and higher total bilirubin ( P P P ≤ .005) were larger than published norms. Conclusions In children with SCA, long-term transfusion therapy may not prevent development or progression of abdominal organ dysfunction.

Chronic transfusions to maintain haemoglobin S (HbS) < or =30% are the mainstay of treatment for children with sickle cell anaemia (SCA) and previous stroke. This HbS target is often hard to maintain, however, and values achieved in current practice are unknown. In preparation for the Phase III Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) trial, we collected data on 295 children with SCA and stroke who received transfusions at 23 institutions. The overall average pre-transfusion %HbS was 35 +/- 11% (institutional range 22-51%). Receiving scheduled transfusions on time was the most predictive variable for maintaining HbS at the < or =30% goal.

Objective: To conduct a multidimensional assessment of pain in children with sickle cell disease (SCD). Variables included parent and child reports of pain location and intensity, qualitative descriptors of pain, perceptions regarding the seriousness and severity of SCD, and environmental and emotional factors associated with pain. Methods: We replicated previous SCD pain research and applied advanced assessment methodology and research design to a population of pediatric SCD patients and their caregivers. Results: Convergence of data supports the utility of multidimensional pain assessment with parents and children with SCD. SCD pain is experienced as intense and severe in home and hospital environments. Conclusions: Findings support including children as reporters in clinical assessment of SCD pain. Integration of assessment strategies into home-based pain management may improve health outcomes. Future research should target biobehavioral treatment for pediatric SCD pain.

This study was designed to determine the incidence of thoracic bone infarction in patients with sickle cell diseases who were hospitalized with acute chest or back pain above the diaphragm and to test the hypothesis that incentive spirometry can decrease the incidence of atelectasis and pulmonary infiltrates.We conducted a prospective, randomized trial in 29 patients between 8 and 21 years of age with sickle cell diseases who had 38 episodes of acute chest or back pain above the diaphragm and were hospitalized. Each episode of pain was considered to be an independent event. At each hospitalization, patients with normal or unchanged chest radiographs on admission were randomly assigned to treatment with spirometry or to a control nonspirometry group. Each patient in the spirometry group took 10 maximal inspirations using an incentive spirometer every two hours between 8 a.m. and 10 p.m. and while awake during the night until the chest pain subsided. A second radiograph was obtained three or more days after admission, or sooner if clinically necessary, to determine the incidence of pulmonary complications. Bone scanning was performed no sooner than two days after hospital admission to determine the incidence of thoracic bone infarction.The incidence of thoracic bone infarction was 39.5 percent (15 of 38 hospitalizations). Pulmonary complications (atelectasis or infiltrates) developed during only 1 of 19 hospitalizations of patients assigned to the spirometry group, as compared with 8 of 19 hospitalizations of patients in the nonspirometry group (P = 0.019). Among patients with thoracic bone infarction, no pulmonary complications developed in those assigned to the spirometry group during a total of seven hospitalizations, whereas they developed during five of eight hospitalizations in the nonspirometry group (P = 0.025).Thoracic bone infarction is common in patients with sickle cell diseases who are hospitalized with acute chest pain. Incentive spirometry can prevent the pulmonary complications (atelectasis and infiltrates) associated with the acute chest syndrome in patients with sickle cell diseases who are hospitalized with chest or back pain above the diaphragm.

Bone marrow transplantation (BMT) is the only curative therapy available for hemoglobinopathies. BMT was performed on a young child with sickle cell anemia (SCA) after approximately 9 months of transfusion therapy following her initial stroke. The patient received a matched sibling donor (sickle trait) BMT. The conditioning regimen consisted of busulfan 4 mg/kg/day x 4, cyclophosphamide 50 mg/kg/day x 4. Graft vs. host disease prophylaxis was daily cyclosporine for 6 months. There were no significant complications during BMT. Engraftment occurred on day +17 and the patient was transfusion independent since day +45. Pre-BMT cerebral arteriography showed multiple stenotic cerebral vessels and a moya-moya pattern. Perfusion MRI demonstrated reduced capillary perfusion. Approximately 170 days after BMT the patient experienced episodes of transient left-sided weakness and speech problems. Neuroimaging revealed progression of large vessel pathology by angiography despite significant improvement in cortical perfusion (MR perfusion scan). Molecular analysis by PCR and DNA fingerprinting confirmed absence of mixed mosaicism. Rheologic evaluation showed normal corrected bulk viscosity. It is possible that progression of large vessel pathology and return of clinical symptoms in the face of normal rheologic parameters may be due to worsening of the already damaged cerebral vessels by the BMT conditioning regimen. Further evaluations of patients with SCA undergoing BMT after a stroke are needed to answer this question.

PURPOSE The purpose of this case report is to (a) present a detailed description of a child with sickle cell anemia who developed sarcoidosis, (b) compare the clinical aspects of sickle cell anemia and sarcoidosis, and (c) review the literature with respect to the coincidence of these two diseases. Detailed clinical case history and laboratory findings are presented. A literature review of other case reports with children and adults who have both sickle cell anemia and sarcoidosis is given. RESULTS Based on limited data, there may be an increased incidence of sarcoidosis in adults with sickle cell diseases. No data are available in the pediatric population. CONCLUSIONS Sarcoidosis is difficult to diagnose in children with sickle cell anemia. The clinical manifestations of sickle cell anemia and sarcoidosis have overlapping features but represent distinct syndromes. Patients with sickle cell anemia who have unusual signs and symptoms need to be further evaluated for the possibility of other coincident diseases, including sarcoidosis.

Sickle cell anemia (SCA) is a chronic illness causing progressive deterioration in quality of life. Brain dysfunction may be the most important and least studied problem affecting individuals with this disease.To measure neurocognitive dysfunction in neurologically asymptomatic adults with SCA vs healthy control individuals.Cross-sectional study comparing neuropsychological function and neuroimaging findings in neurologically asymptomatic adults with SCA and controls from 12 SCA centers, conducted between December 2004 and May 2008. Participants were patients with SCA (hemoglobin [Hb] SS and hemoglobin levelor = 10 mg/dL) aged 19 to 55 years and of African descent (n = 149) or community controls (Hb AA and normal hemoglobin level) (n = 47). Participants were stratified on age, sex, and education.The primary outcome measure was nonverbal function assessed by the Wechsler Adult Intelligence Scale, third edition (WAIS-III) Performance IQ Index. Secondary exploratory outcomes included performance on neurocognitive tests of executive function, memory, attention, and language and magnetic resonance imaging measurement of total intracranial and hippocampal volume, cortical gray and white matter, and lacunae.The mean WAIS-III Performance IQ score of patients with SCA was significantly lower than that of controls (adjusted mean, 86.69 for patients with SCA vs 95.19 for controls [mean difference, -5.50; 95% confidence interval {CI}, -9.55 to -1.44]; P = .008), with 33% performing more than 1 SD (85) below the population mean. Among secondary measures, differences were observed in adjusted mean values for global cognitive function (full-scale IQ) (90.47 for patients with SCA vs 95.66 for controls [mean difference, -5.19; 95% CI, -9.24 to -1.13]; P = .01), working memory (90.75 vs 95.25 [mean difference, -4.50; 95% CI, -8.55 to -0.45]; P = .03), processing speed (86.50 vs 97.95 [mean difference, -11.46; 95% CI, -15.51 to -7.40]; P.001), and measures of executive function. Anemia was associated with poorer neurocognitive function in older patients. No differences in total gray matter or hippocampal volume were observed. Lacunae were more frequent in patients with SCA but not independently related to neurocognitive function.Compared with healthy controls, adults with SCA had poorer cognitive performance, which was associated with anemia and age.

Stroke, a devastating complication of sickle cell anemia (SCA), can cause irreversible brain injury with physical and cognitive deficits. Transcranial Doppler ultrasonography (TCD) is a non-invasive tool for identifying children with SCA at highest risk of stroke. National guidelines recommend that TCD screening begin at age 2 years, yet there is research to suggest less than half of young children undergo screening. The purpose of this project was to use quality improvement methods to improve the proportion of patients aged 24-27 months who successfully completed their initial TCD from 25% to 75% by December 31, 2013. Quality improvement methods (e.g., process mapping, simplified failure mode effect analysis, and plan-do-study-act cycles) were used to develop and test processes for identifying eligible patients, scheduling TCDs, preparing children and families for the first TCD, and monitoring outcomes (i.e., TCD protocol). Progress was tracked using a report of eligible patients and a chart showing the age in months for the first successful TCD (population metric). As of December 2013, 100% of eligible patients successfully completed their initial TCD screen; this improvement was maintained for the next 20 months. In November 2014, a Welch's one-way ANOVA was conducted. Results showed a statistically significant difference between the average age of first TCD for eligible patients born in 2009 and eligible patients born during the intervention period (2010-2013; F[1,11.712]=16.03, p=0.002). Use of quality improvement methods to implement a TCD protocol was associated with improved TCD screening rates in young children with SCA.

The lifespan of patients with sickle-cell disease (SCD) continues to increase, and most affected individuals in high-resource countries now live into adulthood. This necessitates a successful transition from pediatric to adult health care. Care for transitioning patients with SCD often falls to primary care providers who may not be fully aware of the many challenges and issues faced by patients and the current management strategies for SCD. In this review, we aim to close the knowledge gap between primary care providers and specialists who treat transitioning patients with SCD. We describe the challenges and issues encountered by these patients, and we propose a biopsychosocial multidisciplinary approach to the management of the identified issues. Examples of this approach, such as transition-focused integrated care models and quality improvement collaboratives, with the potential to improve health outcomes in adulthood are also described. Electronic supplementary material The online version of this article (doi:10.1007/s12325-015-0197-1) contains supplementary material, which is available to authorized users.

Foremost among the beneficial effects of screening umbilical cord blood is the optimized quality of care that can follow the immediate involvement of an infant with sickle cell disease and his or her family in an appropriate health care system. This is exemplified by the reduction in the case fatality rate of pneumococcal septicemia that has been achieved. Appropriate follow-up of screening also includes transmission of information about the diagnosis of a hemoglobinopathy trait or α-thalassemia to affected families and their physicians, with ready availability of education and counseling.