BACKGROUND: Sickle cell anemia (SCA) is the most common hemoglobinopathy in Brazil and worldwide and is part of a group of chronic genetic diseases resulting from abnormalities in the structure of hemoglobin.
OBJECTIVE: To evaluate the impact of oral health conditions on the quality of life (QoL) of children and adolescents with SCA.
METHODS: This is a cross-sectional study with a sample of 76 children and adolescents aged 8-14 years. For inclusion, they were required to have a diagnosis of HbSS SCA in their medical records, without a pain crisis or any dental emergency in the last three months. The children and adolescents with SCA were from Hematology and Hemotherapy Center of Maranhão. Demographic characteristics, socioeconomic status, oral hygiene, caries, malocclusion, and oral health-related quality of life (OHRQoL) were assessed. OHRQoL was assessed using the Child Perceptions Questionnaire. Descriptive statistics, Student\'s t and Mann-Whitney tests were performed (α = 5%).
RESULTS: Brown race was the most prevalent for both age groups (8-10 years-63.2% and 11-14 years-57.9%). Predominant monthly family income for both age groups was below $106. Visible plaque and gingival bleeding were higher in children aged 8-10 years. Dental caries significantly impacted the QoL of adolescents through the domain \"oral symptom\" (p = .031). Malocclusion significantly impacted the QoL of adolescents (\"total score,\" p = .026; \"social well-being\", p = .045).
CONCLUSIONS: Oral health impairment negatively affected the QoL of adolescents with SCA.

UNASSIGNED: The neurocognitive functions in Ugandan children aged 1-12 years with sickle cell anemia (SCA) were compared to their non-SCA siblings to identify risk factors for disease-associated impairment.
UNASSIGNED: This cross-sectional study of the neurocognitive functions in children with SCA (N = 242) and non-SCA siblings (N = 127) used age- and linguistically appropriate standardized tests of cognition, executive function, and attention for children ages 1-4 and 5-12. Test scores were converted to locally derived age-normalized z-scores. The SCA group underwent a standardized stroke examination for prior stroke and transcranial Doppler ultrasound to determine stroke risk by arterial flow velocity.
UNASSIGNED: The SCA group was younger than their siblings (mean ages 5.46 ± 3.0 vs. 7.11 ± 3.51 years, respectively; p < 0.001), with a lower hemoglobin concentration (7.32 ± 1.02 vs. 12.06 ± 1.42, p < 0.001). The overall cognitive SCA z-scores were lower, -0.73 ± 0.98, vs. siblings, -0.25 ± 1.12 (p < 0.001), with comparable findings for executive function of -1.09 ± 0.94 vs. -0.84 ± 1.26 (p = 0.045), respectively. The attention z-scores for ages 5-12 for the SCA group and control group were similar: -0.37 ± 1.4 vs. -0.11 ± 0.17 (p = 0.09). The overall differences in SCA status were largely driven by the older age group, as the z-scores in the younger subsample did not differ from controls. Analyses revealed the strongest predictors of poor neurocognitive outcomes among the SCA sample to be the disease, age, and prior stroke (each p < 0.001). The impacts of anemia and SCA were indistinguishable.
UNASSIGNED: Neurocognitive testing in children with SCA compared to non-SCA siblings revealed poorer SCA-associated functioning in children older than age 4. The results indicate the need for trials assessing the impact of disease modification on children with SCA.

A universal newborn screening program for sickle cell disease (uNS-SCD) was implemented in the province of Québec (Qc) in November 2013, close in time to the recommendation of early initiation of hydroxyurea (HU) therapy for children. This retrospective cohort study evaluated the impact of such a program on children first seen between January 2000 and December 2019. Cohorts pre-SCD-uNS in Qc (pre-QcNS) (n = 253) and post-QcNS (n = 157) for patients seen prior to or after Nov 2013 were compared. Kaplan-Meier curves, Poisson regression, and logistic regressions were used for statistical analysis, using Software R version 4.2.1. Median age at first visit decreased significantly from 14.4 [interquartile range: 2.4-72.0] to 1.2 months [1.2-57.6] (p < 0.001). The percentage of children born in Qc undiagnosed at birth and referred after a first SCD-related complication dropped from 42.6% to 0.0% (p < 0.0001). The median age of HU introduction for patients with SS/Sβ°-thalassemia decreased from 56.4 [31.2-96.0] to 9.0 months post-QcNS [8.0-12.1] (p < 0.001). Event-free survival improved significantly for any type of hospitalization as well as for vaso-occlusive crisis (VOC) (140-257 days (p < 0.001) and 1320 vs. 573 days (p < 0.002), respectively), resulting in a reduction from 2 [interquartile range: 1.0-3.0] to 1.0 hospitalizations/patient-year [0.6-1.4] (p < 0.001). Children with SS/Sβ°-thalassemia referred post-QcNS also had fewer emergency department visits for VOC (RR: 0.69, 95% confidence interval: 0.54-0.88). The Universal NS program allows early detection and referral of children with SCD to comprehensive care centers. Earlier access ensures that children benefit from essential preventive interventions, reducing disease burden. This cohort study highlights that uNS-SCD is an essential public health measure.

BACKGROUND: People with sickle cell anemia (SCA) may require frequent blood transfusions to treat acute and chronic complications. Hydroxyurea is a life-saving treatment for SCA that could also decrease the need for blood transfusions. Inadequate medication access and challenges in dose optimization limit the widespread use of hydroxyurea in Africa. If feasible, pharmacokinetic (PK) dosing might improve dose determination to minimize toxicities and maximize clinical benefits. The Alternative Dosing And Prevention of Transfusions (ADAPT, NCT05662098) trial will analyze the impact of hydroxyurea on transfusion rate and serve as a pilot study to evaluate the feasibility of PK-guided hydroxyurea dosing in Uganda.
METHODS: Herein we describe the rationale and design of ADAPT, a prospective cohort study of ∼100 children with SCA in Jinja, Uganda. The primary hypothesis is that hydroxyurea will decrease blood transfusion use by ≥ 50%, comparing the transfusion incidence rate ratio between a 3-month pretreatment and a 12-month treatment period. A key secondary hypothesis is that our PK-dosing approach will generate a suitable hydroxyurea dose for ≥80% of participants. Every ADAPT participant will undergo hydroxyurea PK testing, and if a dose is generated within 15-35 mg/kg/day, participants will start on their individualized dose. If not, they will start on a default dose of 20 mg/kg/day. Hydroxyurea dose optimization will occur with periodic dose adjustments.
CONCLUSIONS: Overall, demonstrating the reduction in blood transfusion utilization with hydroxyurea treatment would provide leverage to increase hydroxyurea access, and PK-guided hydroxyurea dosing should optimize the safe and effective treatment of SCA across sub-Saharan Africa.

UNASSIGNED: Sickle cell disease (SCD) is the most common hereditary hemoglobinopathy, which delays growth leading to an altered skeleton and craniofacial pattern. Palatal rugae patterning has been considered the regulator of the development of the palate. The purpose of the research work was to study the morphology of the palate, rugae pattern, and its dimensions in SCD children and compare them with healthy normal children, and to evaluate its role as minor physical anomalies (MPAs).
UNASSIGNED: A cross-sectional case-control study was designed as per STROBE guidelines. The sample comprised 50 children diagnosed with sickle cell disease (Group SCD) and 50 normal healthy children as control (Group C) belonging to the same age group (10-18 years). Dental impressions were made, followed by the pouring of dental casts. The length of the palatal rugae was measured and categorized into primary (>5 mm), secondary (3 mm-5 mm), and fragmentary rugae (<3 mm). The shape of each primary palatal rugae was identified and categorized as curved, wavy, straight, circular and non-specific. Linear and angular measurements of the palatal rugae patterns and palatal dimensions (width, height, area) were measured and recorded.
UNASSIGNED: The total number of palatal rugae and fragmentary rugae was lesser in Group SCD than in Group C (p < 0.05). The depth of the palate was significantly increased, whereas the area of the palate significantly decreased in Group SCD.
UNASSIGNED: The children with SCD showed distinctive palatal rugae patterns and dimensions when compared with normal healthy children that can be attributed as potential MPAs for sickle cell disease. Children with SCD had an under-developed palatal rugae pattern with a deep, narrow and small palate when compared to healthy children.The dimensions of the palatal rugae pattern in SCD showed reduced distance between the incisive papilla and the first and last rugae, indicating a further decrease in the anteroposterior dimensions of the palate. These findings may aid in the early diagnosis and prevention of malocclusion in children with SCD by appropriate interceptive orthodontic treatment.

UNASSIGNED: Children with sickle cell anemia (SCA) in Sub-Saharan Africa are at high risk of sickle cerebrovascular injury (SCVI). Hydroxyurea, a commonly used disease-modifying therapy, may prevent or decrease SCVI for reduced incident stroke, stroke risk and potentially cognitive dysfunction. We aim to test the impact of daily hydroxyurea therapy on these outcomes in Ugandan children with SCA. We hypothesize that hydroxyurea therapy over 36 months will prevent, stabilize or improve these complications of SCA.
UNASSIGNED: The BRAIN SAFE II study is an open-label, single-arm trial of daily hydroxyurea for 270 children with SCA (HbSS) in Uganda, ages 3-9 years. Following baseline assessments, participants began hydroxyurea therapy and clinically followed per local guidelines. Standard hydroxyurea dose is escalated to maximum tolerated dose (MTD). SCVI is assessed by cerebral arterial velocity using Doppler ultrasound, with cognitive function determined by formal neurocognitive testing (primary outcomes). Structural SCVI is assessed by magnetic resonance imaging (MRI) and angiography (MRA) in a sub-sample of 90 participants ages ≥5 years, along with biomarkers of anemia, inflammation and malnutrition (secondary outcomes). At trial midpoint (18 months) and completion (36 months), primary outcomes will be compared to participants\' baseline to determine hydroxyurea impact and relationships to secondary outcomes.
UNASSIGNED: This open-label, single-arm trial will examine the impact of hydroxyurea on preventing or ameliorating SCA SCVI in children, assessed by reducing incident stroke, stroke risk and neurocognitive dysfunction. Trial results will provide important insight into the role of hydroxyurea therapy on critical manifestations of SCVI in children with SCA.

OBJECTIVE: Sickle cell anemia (SCA) is a hematological condition that involves the synthesis of sickle-shaped, hemoglobin with a short lifespan. This study employed three-dimensional (3D) scanning to pinpoint the most common arch form in a sample of Iraqi adults with SCA.
METHODS: A cross-sectional study included maxillary and mandibular casts of 50 patients (25 male and 25 female) with SCA and 50 participants in a control group (25 male and 25 female). The facio-axial (FA) point was digitized on each tooth\'s labial or buccal surface using SolidWorks® 2020 software. The dimensions of the arch were calculated using two proportional measures and four linear measurements. The dental arch form was determined using 3M templates, and the arches were categorized as square, ovoid, or tapered to establish the most-common arch form. Chi-square was used to compare arch form distribution, and an independent t-test was used to calculate the difference between the control group and the SCA group.
RESULTS: The tapered arch was the predominant form in SCA males and females for the maxilla and the mandible. The molar vertical distance (upper and lower) was significantly higher in males in the SCA group than in males in the control group. There was no significant difference between females in the SCA group and females in the control group.
CONCLUSIONS: Most patients with SCA had tapered arches. Sagittal jaw dimensions in males with SCA were more affected by marrow hyperplasia. There was little if any to no effect on females with SCA.

BACKGROUND: Children with sickle cell anemia (SCA) are at high risk for stroke. Protocols for stroke prevention including blood transfusions, screening for abnormal non-imaging transcranial Doppler (TCD) measurements, and hydroxyurea therapy are difficult to implement in low-resource environments like Nigeria. This study aimed to examine the contextual factors around TCD screening in a community hospital in Nigeria using qualitative interviews and focus groups.
METHODS: We conducted a descriptive qualitative study in a community hospital in Kaduna, Nigeria, using focus groups and interviews. Interview guides and analysis were informed by the Consolidated Framework for Implementation Research (CFIR) framework and the Theory of Planned Behavior. Transcripts were coded and analyzed using an iterative deductive (CFIR)/Inductive (transcribed quotes) qualitative methodology.
RESULTS: We conducted two focus groups and five interviews with health care workers (nurses and doctors) and hospital administrators, respectively. Themes identified key elements of the inner setting (clinic characteristics, resource availability, implementation climate, and tension for change), characteristics of individuals (normative, control, and behavioral beliefs), and the implementation process (engage, implement, and adopt), as well as factors that were influenced by external context, caregiver needs, team function, and intervention characteristics. Task shifting, which is already being used, was viewed by providers and administrators as a necessary strategy to implement TCD screening in a clinic environment that is overstressed and under-resourced, a community stressed by poverty, and a nation with an underperforming health system.
CONCLUSIONS: Task shifting provides a viable option to improve health care by making more efficient use of already available human resources while rapidly expanding the human resource pool and building capacity for TCD screening of children with SCD that is more sustainable.
BACKGROUND: NCT05434000.

UNASSIGNED: Neurocognitive function in Ugandan children aged 1-12 years with sickle cell anemia (SCA) were compared to their non-SCA siblings to identify risk factors for disease-associated impairment.
UNASSIGNED: This cross-sectional neurocognitive function study of children with SCA (N=242) and non-SCA siblings (N=127) used age- and linguistically-appropriate standardized tests of cognition, executive function and attention for children ages 1-4 and 5-12 years. Test scores were converted to locally derived age-normalized z-scores. The SCA group underwent standardized stroke examination for prior stroke and transcranial doppler ultrasound (TCD) to determine stroke risk by arterial flow velocity.
UNASSIGNED: The SCA group was younger than siblings (mean ages 5.46±3.0 versus 7.11±3.51 years, respectively; p <.001), with lower hemoglobin concentration (7.32±1.02 vs. 12.06±1.42, p <.001). Overall cognitive SCA z-scores were lower: -0.73 ±0.98 vs. siblings -0.25 ±1.12 (p<.001), with comparable findings for executive function of -1.09±0.94 versus -0.84±1.26 (p=0.045), respectively. Attention z-scores for ages 5-12 for the SCA group and controls were similar: -0.37±1.4 vs. -0.11±0.17 (p=.09). Overall differences by SCA status were largely driven by the older age group, as z-scores in the younger sub-sample did not differ from controls. Analyses revealed the strongest predictors of poor neurocognitive outcomes among the SCA sample to be the disease, age and prior stroke (each p<.001). Impact from anemia and SCA were indistinguishable.
UNASSIGNED: Neurocognitive testing in children with SCA compared to non-SCA siblings revealed poorer SCA-associated functioning in children older than age 4. Results indicate need for trials assessing impact from disease modification for children with SCA.

OBJECTIVE: To explore the knowledge and attitude towards sickle cell disease (SCD) among care givers of paediatric sickle cell patients at Mbale regional referral hospital in Eastern Uganda.
METHODS: A cross sectional study was conducted at Mbale regional referral hospital. We used simple random sampling technique to recruit participants from among the care givers of pediatric sickle cell patients admitted at the hospital, administered questionnaires and conducted multivariable logistic regression to establish the association between the different factors.
RESULTS: 372 respondents participated in the study, 82.26% of which were female. 57.80% of the respondents had ever heard of SCD/SCA. 36.02% were willing to stay in a relationship with their partner despite the risk of having a child with SCD/SCA. A multivariate analysis revealed that variables; \"number of children\", \"children with sickle cell can cope with life\" and \"willing to stay in a relationship despite the risk of a having a child with sickle cell\" were statistically significant.
CONCLUSIONS: There was a high level of general awareness about SCD/SCA but comprehensive knowledge about its cause and prevention was low and the majority did not find a reason as to why it should influence their marital decisions. Inclusion of SCD/SCA in existing health education programs is highly recommended.