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OBJECTIVE: The objective of this study was to investigate the association between cognitive functioning, coping, and depressive symptoms in children and adolescents with sickle cell disease (SCD).   METHOD: Forty-four children (M age = 9.30, SD = 3.08; 56.8% male) with SCD completed cognitive assessments measuring working memory (Wechsler Intelligence Scale for Children-Fourth Edition) and verbal comprehension (Wechsler Abbreviated Scale of Intelligence-Second Edition). Participants' primary caregivers completed questionnaires assessing their child's coping and depressive symptoms.   RESULTS: Verbal comprehension was significantly positively associated with secondary control coping (cognitive reappraisal, acceptance, distraction), and both working memory and secondary control coping were negatively associated with depressive symptoms. In partial support of the primary study hypothesis, verbal comprehension had an indirect association with depressive symptoms through secondary control coping, whereas working memory had a direct association with depressive symptoms.   CONCLUSIONS: The results provide new evidence for the associations between cognitive function and coping, and the association of both of these processes with depressive symptoms in children with SCD. Findings provide potential implications for clinical practice, including interventions to improve children's cognitive functioning to attenuate depressive symptoms.   Abstract source

OBJECTIVES: Sickle cell disease (SCD) predominately affects Black Americans. This is the first study of its kind to describe the racial bias experiences of youth with SCD and their reactions to these experiences.   METHODS: Participants were 20 youth with SCD (ages 13-21 years) who were asked to describe any racial bias events they experienced, as recorded on the Perception of Racism in Children and Youth measure (PRaCY). Interviews were recorded, transcribed, and analyzed by two independent raters using a conventional content analysis approach.   RESULTS: All participants reported at least one incident of racial bias. Content analysis of racial bias events (n = 104) yielded 4 categories and 12 subcategories as follows: Perpetrator (Peers, Authority Figures, and General Public), Type of Racial Bias (Explicit, Implicit), Behavioral Reaction (Approach, Avoidant), and Emotional Response (Dysphoria, Anger, Unconcerned, Inferior, Anxious).   DISCUSSION: This study provides a description of racial bias experiences within community and medical settings and highlights the need for further evaluation of the impact of racial bias among youth with SCD.   Abstract source

BACKGROUND:Pregnant women with sickle cell disease (SCD) are at increased risk of maternal and fetal complications. There are limited data on the outcome of the treatment of VOCs with opioids in relation to neonatal complications during pregnancy. METHODS:This is a retrospective cohort study of women with SCD from January 1999 to December 2008. Women with SCD were identified by ICD-9 codes and matched 2:1 to a control group of women on methadone for opioid dependence. The primary outcome was the rate of neonatal abstinence syndrome (NAS). Secondary outcomes included the mean NAS score prior to treatment and the length of treatment. Statistical analysis was performed using SPSS. RESULTS:Twenty-one women with SCD who delivered a total of 23 neonates were included. The rate of NAS among infants born to women with SCD who were treated with opioids at any time was 22% compared to 54% in the methadone controls (p = .010). The rate of NAS was 27% among infants born to women taking opioids daily compared to 54% in the methadone control group (p = .062). CONCLUSIONS:Neonates born to women with SCD who are treated with daily opioids are at a similar risk for developing NAS as those born to mothers on methadone for opioid dependence. Neonates born to women with SCD treated with episodic opioids are at a significantly lower risk for developing NAS than those born to women on methadone for opioid dependence.  Abstract source

Guidelines recommend that African Americans know their sickle cell trait status to inform reproductive health decisions. Few studies have applied a behavioral theory to identify factors associated with sickle cell trait screening to inform intervention targets to increase this behavior. We applied a Sickle Cell Trait Screening Framework to identify factors associated with African Americans' intention to ask for sickle cell trait screening.Participants (N = 300), ages 18 to 35, completed a cross-sectional survey. A three-step sequential ordinary least squares regression analysis identified factors influencing intention. Results indicated socio-demographic factors (age, education), knowledge and fear beliefs (screening knowledge, perceived threat), and reasoned action approach (RAA) constructs were associated with intention.RAA constructs influenced intention over knowledge and fear beliefs with an increase in R2 of .468. Perceived behavioral control was more predictive of intention (β = .576, p < .001). Attitude and perceived norm also had significant weights (β = .325 and β = .192, both p < .001, respectively).Findings from this study can inform strategies (e.g., eliminating costs associated with screening, reducing fear of painful tests) to increase sickle cell trait screening among African Americans. Ultimately, more sickle cell carriers will become aware of their trait status and be able to make informed reproductive health decisions.  Abstract source

Objectives: To determine the effects of Cogmed, a home-based computerized Working Memory (WM) training intervention, in children with SCD using a randomized controlled trial design.   Results: Ninety-one participants (M age=10.43, SD=2.93; 59% female; 69% HbSS) enrolled in the study; 52% (n=47) exhibited WM deficits and were randomized to either begin Cogmed immediately or wait 5-8 weeks before starting Cogmed. Among those that have received the intervention and reached the end of their training period (n=42), 27 participants (59%) completed at least 5 Cogmed sessions, 19 (41%) finished at least 10 sessions, and 7 finished at least 20 sessions (15%). The mean number of completed Cogmed sessions was 9.10 (SD=7.77). Paired Samples t-tests revealed significant improvements on the Working Memory Index (t[38]=-2.44, p=0.020) and on the Digit Span (t[40]=-3.02, p=0.004), and Spatial Span-Backward (t[39]=-2.83, p=0.007) subtests. Improvements were especially pronounced for participants completing at least 10 sessions. Partial correlations controlling for respective baseline scores indicated that the number of Cogmed sessions completed was positively correlated with post-test scores on Digit Span (r=.38, p=.017) and Spatial Span-Backward (r=0.45, p=0.004) subtests. Among participants who completed at least 10 Cogmed sessions, 77% scored in the Average range or higher on the Working Memory Index at the post-intervention assessment, compared to 58% at baseline.   Conclusion: Results support the efficacy of Cogmed in producing significant improvements in WM. A dose-effect was observed such that participants who completed more Cogmed sessions had greater improvements in WM. Home-based cognitive training programs may ameliorate SCD-related WM deficits but methods for motivating and supporting patients as they complete home-based interventions are needed to enhance adherence and effectiveness.   Abstract source

  Sickle cell and beta thalassaemia are inherited recessive conditions. 'Recessive' means a person can only inherit the condition if both parents are carriers. Each child born to a couple who are both carriers has a 1 in 4 risk of having the condition and a 1 in 2 risk of being a carrier. If only one parent is a carrier, their children cannot inherit the condition itself, but they still have a 1 in 2 chance of inheriting carrier status. Being a carrier will not affect the child's health. If a woman discovers during pregnancy that she is a sickle cell or beta thalassaemia carrier, she will usually be advised to tell her partner and suggest he also has a screening test for sickle cell, thalassaemia and other variations (such as haemoglobin C, E, D or O Arab). For many couples, the test will show the male partner is not a carrier, and their baby will not inherit the condition.   Related story

Background: The 2014 National Heart Lung & Blood Institute(NHLBI) guidelines for acute management of VOE recommends rapid evaluation and treatment of pain, including administration of a parenteral opioid within 30-minutes of triage or 60-minutes from registration, pain reassessment & repeat opioid delivery within 15-30-minutes. INF use has been increasing in PEDs due to its rapid onset and ease of administration.   Objectives: To evaluate PED utilization of INF & its effect on intravenous (IV) opioid administration and pain control for the treatment of VOE.   Design/Method: A retrospective review of 250 EMR was performed on children with SCD±2years presenting to a PED with VOE (pain scores 6 on a 0-10 scale) from Jan-June 2017. Variables studied were median time (IQR, 95%CI) from PED arrival to first-parenteral-opioid-administration, time-to-first-IV-opioid, first & final pain score, disposition and readmission rate. Time-to-first-IV-opioid was also compared to historical data (Jan-Dec2012, n=231) prior to INF protocol initiation.   Results: Mean age was 13±4years, 48% male and majority had HbSS (66%). Admission rate was 60%. Of 250 VOE episodes, 183(73%) received INF & 204 (82%) received an IV opioid. Both INF+IV opioid were given to 141 patients, while 42 (17%) patients received INF only. Time-to-first-parenteral-opioid administration for those treated with INF vs. IV opioid alone was 26 minutes (19-33) versus 74 minutes (46-10), p<0.01. Pain scores at disposition were lower in patients who received INF. Time-to-IV-opioid was longer in patients receiving INF vs. IV opioid alone (115 vs 83 minutes<0.01) & compared to historical date (35±18minutes). Additionally, 15% patients received IV opioids within 60 minutes of ED arrival in the INF+IV opioid vs. 40% in the IV opioids alone group (p<0.01). No differences in 72-hour-return-rates were found in any of the groups, including INF alone group.   Conclusion: Use of INF in the PED for VOE is an excellent strategy to shorten time-to-first-parenteral-opioid-administration, improve pain scores & improve adherence to the NHLBI guidelines. However we had 2 distinct unexpected findings: (1) Delays in IV opioid delivery after INF use & (2) INF alone appeared to provide sufficient pain control without IV opioids for disposition home in 17% of VOE patients. Whether the latter reflects insufficient pain management or that there is a milder subgroup for which INF alone is sufficient, requires further investigation. This study illustrates our experience with a PED-based INF protocol in terms of unanticipated delays in IV opioids and also discharges after INF alone. Efforts are underway to further improve use of INF in VOE management.   Abstract source

Background: Sickle cell disease (SCD), a genetic disorder characterized by defective sickle hemoglobin (HbS), triggers red blood cell sickling, hemolysis, vaso-occlusion, and inflammation. Ischemic injury from SCD starts in infancy and accumulates over a lifetime, causing pain, fatigue, and progressive end-organ damage that culminates in early mortality. Voxelotor (GBT440) is an oral, once-daily therapy that modulates hemoglobin’s oxygen affinity, thereby inhibiting hemoglobin polymerization.   Objectives: To assess the safety, pharmacokinetics, and efficacy of voxelotor in pediatric patients with SCD.   Design/Method: This ongoing study is being conducted in 2 parts: Part A: a single dose of voxelotor 600 mg in pediatric and adolescent patients; Part B: multiple doses of voxelotor 900 mg/d or 1500 mg/d for 24 weeks in adolescents. Part B’s primary objective is to assess the effect of voxelotor on modifying anemia. Secondary objectives include measuring other markers of disease modification, such as hemolysis; daily SCD symptoms, using a patient-reported outcome (PRO) measure; and safety.   Results: As of November 6, 2017, 24 patients (10 females) had received voxelotor 900 mg and 12 patients (6 females) had received voxelotor for >=16 weeks. The median age for the 12 patients was 13 years, 92% were receiving hydroxyurea (HU), and 41% had >=1 painful crises in the past year. Data for hemolysis measures are available for 11 patients who received voxelotor for 16 weeks. Six of the 11 patients achieved a hemoglobin (Hb) response of >1 g/dL increase. Laboratory markers of hemolysis improved concordantly; the median reductions in reticulocytes and indirect bilirubin were 11% and 40%, respectively. Ten of 12 patients showed reduction in total symptom scores (TSS) at week 16, with a 94% median reduction in TSS from baseline. There were no treatment-related serious adverse events (AEs) or drug discontinuations due to AEs.   Conclusion: Voxelotor 900 mg for 16 weeks in adolescents with SCD, the majority receiving HU, demonstrated consistent, sustained efficacy on Hb levels and measures of hemolysis; >50% of patients showed a >1 g/dL improvement in Hb. Improvement in TSS in mildly symptomatic patients suggests that the PRO is sensitive to treatment effect and supports use in the ongoing HOPE phase 3 study. Voxelotor’s reassuring safety profile is consistent with results in adults. These interim results support ongoing clinical evaluation of voxelotor as a potential disease-modifying therapy for adults and children with SCD.   Abstract source