Burden and outcome of respiratory morbidities among children and adolescents with sickle cell disease-A retrospective review of emergency presentations in some Nigerian tertiary institutions.

BACKGROUND: Despite the huge burden of sickle cell disease (SCD) among Nigerian children, the burden and outcome of respiratory illnesses remain undocumented. Thus, we aimed to describe the spectrum and outcome of respiratory illnesses among SCD childrenand adolescentadmissions in ten Nigerian tertiary hospitals. METHOD: A retrospective review of the SCD admission records of children and adolescents with a confirmed diagnosis of respiratory illnesses from 2012 to 2021 in ten tertiary health facilities across five geopolitical zones in Nigeria was conducted. The data, collectedbetween March and June 2023, included the age, sex, diagnosis, complications, duration and outcome of hospitalization. RESULTS: Of the 72,333 paediatric admissions, 7,256 (10.0%) had SCD; the proportion of SCD from the total admission ranged from 2.1 to 16.3% in the facilities. Of the 7,256 children and adolescents with SCD, 1,213 (16.7%) had respiratory morbidities. Lower respiratory disease was the most common (70.0%) respiratory entity and the majority were pneumonia (40.1.0%), followed by acute chest syndrome (26.7%). Seventeen (1.4%) patients died; all had lower respiratory diseases [(acute chest syndrome ACS (11, 64.7%), pneumonia; 5, 29.4%, and asthma (1, 5.9%). Based on the proportion of deaths among overall SCD, the 17 death cases contributed 9.4% (95% CI 5.9 to 14.5). Factors associated with deaths included duration of hospitalization less than 72 hours and lower respiratory tract diseases. CONCLUSION: Sickle cell disease is a major contributor to hospitalization among Nigerian children and adolescents, with high respiratory morbidity and mortality. Pneumonia and acute chest syndrome were associated with mortality, andthe highest risk of death within the first 72 hours.

Association of hydroxyurea adherence with transcranial Doppler screenings in children with sickle cell disease.

BACKGROUND: National sickle cell disease (SCD) guidelines recommend oral hydroxyurea (HU) starting at 9 months of age, and annual transcranial Doppler (TCD) screenings to identify stroke risk in children aged 2-16 years. We examined prevalence and proportion of TCD screenings in North Carolina Medicaid enrollees to identify associations with sociodemographic factors and HU adherence over 3 years. STUDY DESIGN: We conducted a longitudinal study with children ages 2-16 years with SCD enrolled in NC Medicaid from years 2016-2019. Prevalence of TCD screening claims was calculated for 3 years, and proportion was calculated for 12, 24, and 36 months of Medicaid enrollment. Enrollee HU adherence was categorized using HU proportion of days covered. Multivariable Poisson regression assessed for TCD screening rates by HU adherence, controlling for age, sex, and rurality. RESULTS: The prevalence of annual TCD screening was between 39.5% and 40.1%. Of those with 12-month enrollment, 77.8% had no TCD claims, compared to 22.2% who had one or higher TCD claims. Inversely, in children with 36 months of enrollment, 36.7% had no TCD claims compared to 63.3% who had one or higher TCD claims. The proportion of children with two or higher TCD claims increased with longer enrollment (10.5% at 12 months, 33.7% at 24 months, and 52.6% at 36 months). Children with good HU adherence were 2.48 (p < .0001) times more likely to have TCD claims than children with poor HU adherence. CONCLUSION: While overall TCD screening prevalence was low, children with better HU adherence and longer Medicaid enrollment had more TCD screenings. Multilevel interventions are needed to engage healthcare providers and families to improve both evidence-based care and annual TCD screenings in children with SCD.

Assessment of hypoxemia among young adults with sickle cell anaemia in steady state in southwestern Nigeria: a crosssectional study.

OBJECTIVES: Hypoxia is a known feature of sickle cell anaemia (SCA) which results from chronic anaemia and recurrent vaso-occlusive crisis (VOC) which can cause tissue ischaemia that leads to an end organ damage. The hallmark of SCA is chronic anaemia and recurrent vaso-occlusive crisis. The aim of this study is to compare the oxygen saturation of sickle cell anaemic individuals with the normal haemoglobin type (Hb AA) control and also to determine the prevalence of hypoxemia among SCA. RESULTS: Two-hundred and twenty-two (136 Hb SS and 86 Hb AA) participated in the study. The mean ± SD of age (years), oxygen saturation (%) and pulse rate (bpm) of participants with sickle cell anaemia and Hb AA control were 21.85 ± 3.04 and 22.14 ± 3.18 (t = 0.701, p = 0.436), 95.21 ± 3.02 and 98.07 ± 0.81 (t=-8.598, p < 0.0001) and 77.10 ± 9.28 and 73.16 ± 8.52 (t = 3.173, p = 0.002) respectively. The prevalence of hypoxemia among SCA participants was 47.1%. Prevalence of hypoxemia in males with SCA was 60.9% while 39.1% of the females had hypoxemia.

Sickle Cell Health Awareness, Perspectives, and Experiences (SHAPE) survey: Perspectives of adolescent and adult patients, caregivers, and healthcare professionals on the burden of sickle cell disease.

OBJECTIVES: Sickle cell disease (SCD) is an inherited disorder that causes lifelong complications, substantially impacting the physical and emotional well-being of patients and their caregivers. Studies investigating the effects of SCD on quality of life (QOL) are often limited to individual countries, lack SCD-specific QOL questionnaires, and exclude the caregiver experience. The SHAPE survey aimed to broaden the understanding of the global burden of SCD on patients and their caregivers and to capture the viewpoint of healthcare providers (HCPs). METHODS: A total of 919 patients, 207 caregivers, and 219 HCPs from 10, 9, and 8 countries, respectively, answered a series of closed-ended questions about their experiences with SCD. RESULTS: The symptoms most frequently reported by patients were fatigue/tiredness (84%) and pain/vaso-occlusive crises (71%). Patients' fatigue/tiredness had one of the greatest impacts on both patients' and caregivers' QOL. On average, patients and caregivers reported missing 7.5 days and 5.0 days per month, respectively, of school or work. HCPs reported a need for effective tools to treat fatigue/tiredness and a desire for more support to educate patients on long-term SCD-related health risks. CONCLUSIONS: The multifaceted challenges identified using the SHAPE survey highlight the global need to improve both patient and caregiver QOL.

Disparities in pediatric hospital use during transition to adult healthcare for young adults with childhood-onset chronic conditions.

BACKGROUND: Young adults (YA) with childhood-onset chronic conditions-particularly YA with cystic fibrosis (CF), congenital heart disease (CHD), and sickle cell disease (SCD)-continue to have pediatric hospital admissions. Factors associated with this continued pediatric hospital use remain underexplored. OBJECTIVE: To determine if pediatric hospital use by YA differed (1) across condition and (2) within each condition by sociodemographic factors. METHODS: Conducted a cross-sectional analysis of admissions for YA 22-35 years with CF, CHD, and SCD from 2016 to 2020 in the National Inpatient Sample. Admissions for YA with CF, CHD, and SCD were identified by international classification of diseases, 10th revision-clinical modification diagnosis codes. To determine if conditions or sociodemographic factors were associated with YA pediatric hospital use, we used multivariable logistic regression with separate models for the different objectives. RESULTS: YA with SCD had lower odds of pediatric hospital use compared to YA with CF. Relationships between sociodemographic factors and pediatric hospital use varied. Black YA with both CF and CHD had lower odds of pediatric hospital use than white YA with CF and CHD. For YA with SCD, despite 17,810 (6.5%) having rural residence, zero (0) had pediatric hospital use; whereas YA with CF living in a rural area had greater odds of pediatric hospital use compared to urban residents. CONCLUSION: YA with SCD used pediatric hospitals less than YA with either CF or CHD. Coupled with our findings that Black YA with CF and CHD had less pediatric hospital use, these data may reflect systematic racial differences within pediatric to adult healthcare transition programs.

Sickle Cell Disease in an Older Adult Population: A Retrospective Review of Health Care Resource Utilization.

Sickle cell disease (SCD) has a history of health inequity, as patients with SCD are primarily Black and often marginalized from the health care system. Although recent health care and treatment advancements have prolonged life expectancy, it may be insufficient to support the complex needs of the growing population of older adults with SCD. This retrospective study used a cohort (N = 812) of Medicare Advantage beneficiaries 45 years and older (ages: 45-54, 55-64, 65-74, 75-89) with SCD to identify associations of SCD-related complications and comorbidities with emergency department (ED) visits, potentially avoidable ED visits, all-cause hospitalization, and potentially avoidable hospitalizations, 2018-2020. The 75-89 age group had lower odds of an ED visit (OR 0.56; 95% CI 0.32-1.00), 65-74 age group had lower odds of an ED visit (OR 0.49; 95% CI 0.31-0.78) and hospitalization (OR 0.50; 95% CI 0.31-0.79), compared with the 45-54 age group. Acute chest syndrome was associated with increased odds of an ED visit (OR 2.02; 95% CI 1.10-3.71), avoidable ED visit (OR 1.87; 95% CI 1.14-3.06), and hospitalization (OR 3.61; 95% CI 2.06-6.31). Pain was associated with increased odds of an ED visit (OR 2.64; 95% CI 1.85-3.76), an avoidable ED visit (OR 3.08; 95% CI 1.90-4.98), hospitalization (OR 1.51; 95% CI 1.02-2.24), and avoidable hospitalization (OR 6.42; 95% CI 1.74-23.74). Older adults with SCD have been living with SCD for decades, often while managing pain crises and complications associated increased incidence of an ED visit and hospitalization. The characteristics and needs of this population must continue to be examined to increase preventative care and reduce costly emergent health care resource utilization.

Cost-effectiveness of newborn screening for sickle cell disease: a systematic review protocol.

OBJECTIVE: The purpose of this systematic review is to assess the cost-effectiveness of targeted/selective newborn screening compared with universal screening for sickle cell disease across various countries and settings. INTRODUCTION: The incidence of sickle cell disease is a widespread and potentially fatal hematologic disorder that affects thousands of newborns worldwide. The cost of newborn screening creates a burden on households and the economy. INCLUSION CRITERIA: Studies will be eligible for inclusion in the review if they focus on the cost-effectiveness of newborn screening for sickle cell disease, comparing targeted/selective screening with universal screening. METHODS: A preliminary search of MEDLINE (PubMed) was undertaken using MeSH terms, such as sickle cell disease, newborn , and economic evaluations . Two reviewers will screen the titles, abstracts, and full text independently against the inclusion criteria. Disagreements will be resolved by discussion or with a third reviewer. To assess methodological quality, the JBI checklist for economic evaluation will be used. Data will be extracted by 2 reviewers using a modified JBI data extraction form. The JBI dominance ranking matrix for economic evaluations will be used to summarize and compare the results. Cost-effectiveness will be measured on the basis of cost per test/case detected, quality-adjusted life years gained, or disability-adjusted life years averted. The Grading of Recommendations, Assessment, Development and Evaluation (GRADE) assessment will be conducted to evaluate the certainty of economic evidence, such as use of resources and expenditures, and to incorporate the results into the decision-making process. REVIEW REGISTRATION: PROSPERO CRD42017057963.

Burden of employment loss and absenteeism in adults and caregivers of children with sickle cell disease.

ABSTRACT: Sickle cell disease (SCD) is a genetic disorder affecting 100 000 people with an estimated annual medical cost of $3 billion in the United States; however, the economic impact on patients is not well described. We aimed to examine the indirect economic burden and test the hypothesis that socioeconomic status and greater social vulnerability risks are associated with increased absenteeism and employment loss. We surveyed adults and caregivers of children with SCD at 5 US centers from 2014 to 2021. Logistic regression models were used to examine the associations of employment loss and missed days of work with demographics and social determinants. Indirect costs were estimated by multiplying the self-reported missed days of work and job loss by 2022 average wages by the state of the participating institution. Of the 244 participants, 10.3% reported employment loss in the last 5 years, and 17.5% reported missing 10 or more days of work. Adults had 3 times more employment loss compared with caregivers of children with SCD (OR, 3.18; 95% CI, 1.12-9.01) but fewer missed days of work (OR, 0.24; 95% CI, 0.11-0.0.51). Participants who did not live with a partner reported increased employment loss (OR, 4.70; 95% CI, 1.04-21.17) and more missed days of work (OR, 4.58; 95% CI, 1.04-20.15). The estimated annual indirect economic burden was $2 266 873 ($9290 per participant). Adults with SCD and caregivers of children with SCD commonly report employment loss and missed days of work as important risk factors. The high indirect economic burden suggests that future economic evaluations of SCD should include SCD-related indirect economic burden.

Prevalence of sickle cell trait and needs assessment for uptake of sickle cell screening among secondary school students in Kampala City, Uganda.

BACKGROUND: Sickle cell disease (SCD) is one of the most frequent and traumatizing genetic disease in Uganda, with the prevalence of the sickle cell trait (SCT) estimated at 13.3% leading to serious psycho-social and economic impact on the patients and their families. AIM: This study aimed to determine the burden of SCT and factors influencing the uptake of screening services among secondary school students in Uganda. METHODS: We used an analytical cross-sectional design with a multi-stage sampling approach. A total of 399 students from four secondary schools in Kampala City were enrolled in this study. Data were gathered using semi-structured questionnaires and blood screening. We used the sickling test to determine the presence of sickle cell alleles among the participants and hemoglobin electrophoresis as a confirmatory test. Data gathered using the questionnaire were analyzed using descriptive and inferential statistics. RESULTS: In total, 5.8% of participants who were tested during this study had SCT. Most (80.2%) participants were not in an intimate relationship at the time of data collection. The majority (60.4%) had moderate knowledge about SCT screening and obtained information about screening from the school. Only 29 (7.3%) participants knew of a family member with sickle cell. Overall, participants had a negative attitude toward SCT screening (67%), although 41.6% believed that most people who were sickle cell carriers did not live long and were often sick. Statistically significant associations were found between testing for SCT and knowing a partner's sickle cell status (odds ratio [OR] 2.112, p = 0.043) and Anglican religion (OR 2.075, p = 0.047). CONCLUSION: Despite the moderate level of knowledge and negative attitudes, a relatively large number of participants had SCT. This highlights the need for a comprehensive health education package targeting adolescents to promote SCD/SCT screening.

Assessment of the developmental defects of the enamel in children and adolescents with sickle cell disease: A comparative study.

BACKGROUND: Sickle cell disease (SCD) is the most important hemoglobinopathy in terms of frequency and social impact and can affect the stomatognathic system. AIM: To assess and compare the developmental defects of the enamel (DDE) in children and adolescents with and without SCD. DESIGN: This was a cross-sectional, analytical, and comparative study of 210 children and adolescents aged 5-18 years, who visited the Hematology and Hemotherapy Hospital of Pernambuco. RESULTS: Developmental defects of the enamel were observed in 55.2% of the SCD patients and 35.2% of the non-SCD patients (healthy group; p < .05). In the SCD group, DDE were more common in females than in males (69.1% vs. 40.0%; p < .05). The incidence of DDE in the permanent teeth was higher in the upper arch than in the lower arch (SCD group, 13.1% vs. 4.6%; healthy group, 8.9% vs. 3.6%; p < .05). CONCLUSION: Compared with the healthy group, the SCD patients were almost twice as likely to develop DDE, mostly affecting females and the permanent teeth. These findings suggest that individuals with SCD need early dental care to avoid future oral problems.

Prevalence and cost of sickle cell disease in France: real-world analysis using data from the Echantillon Généraliste des Bénéficiaires.

Sickle cell disease (SCD) is a genetic disorder of the hemoglobin resulting in chronic anemia, hemolysis, and vaso-occlusions. Its treatment mostly relies on hydroxycarbamide, transfusions, and stem cell transplantation. This study aimed at describing the epidemiology and management of SCD in adolescent and adult patients in France. This was a retrospective study performed among SCD patients aged ≥12 years between 2016 and 2018 and controls. SCD patients were matched on a 1:3 ratio with a group of individuals with no diagnosis of SCD, referred as control group. The matching of SCD patients and controls was a direct matching based on age, sex, CMU-c status (which corresponds to free-of-charge complementary coverage for people with low resources) and geographical region of residence. SCD patients and their matched controls were followed-up for the same amount of time by adjusting controls' follow-up period to that of the associated patients. This study used claims data from the French representative 1/97th sample of health data system. The main outcomes were the patients' characteristics and treatments received, healthcare consumptions and related costs among SCD cases and controls. Between 2016 and 2018, 151 patients with ≥6 months of follow-up were identified out of the total population of 732,164 individuals. SCD prevalence extrapolated to the entire population [95% CI] was 19,502 [19,230, 19,778] in 2018. The median (Q1-Q3) age at inclusion date was 37.0 (25.0-48.0) years, with 69.5% of patients being female. The mean (SD) reimbursed cost over follow-up was €24,310 (89,167), mostly represented by hospitalization costs accounting for €21,156 (86,402). A switch in SCD management was observed with age, as younger patients presented more frequent hospitalizations and acute procedures, while older ones had more frequent medical visits and paramedical care. Mean (SD) annual costs were €25,680 (91,843) and vs. €3,227 (23,372) for patients and controls, respectively (p < 0.001), representing an extra cost of almost €150 million over the entire SCD population. This study highlighted the important costs related to SCD and the related medical need with treatment alternatives, which could be filled by the emergence of new therapies.

Stigma of sickle cell disease among Indian tribal population: A multi-centric qualitative study.

BACKGROUND: Sickle Cell Disease (SCD) is the most prevalent hemoglobinopathy, impacting around 5% of the global population. The Indian tribal population, which has been a key focus of the Indian SCD program, can experience health-related stigma due to the multidimensional impact of the disease. This preliminary qualitative inquiry delves into the lived experiences of individuals and synthesizes domains to identify the sources of stigma. METHODOLOGY: The study's framework for developing the stigma tool was rooted in Bronfenbrenner's Ecology of Human Development. The study was implemented in five tribal-dominated districts of India and involved in-depth interviews with sickle cell disease (SCD) patients and their caregivers to explore their stigmatizing experiences. RESULTS: The analysis revealed four overarching themes and several subthemes explaining the type of stigma, its source, and factors contributing to stigmatization. First, the study focused on elements associated with perceived stigma, such as disclosure, self-isolation/refusal to participate, and self-judgment. The second theme pertained to the internalization of stigma. The third theme addressed experienced stigma concerning the disease's impact on day-to-day events, and the fourth theme explored the support system patients needed. The framework highlighted the varying degrees of stigmatizing components within different aspects of patients' ecology. CONCLUSION: Our study highlights the importance of addressing stigma at various levels. Policies, programs, and healthcare interventions must target stigma across these levels. Culturally adaptive tools for identifying stigma, implementing appropriate interventions, and improving healthcare participation are essential for enhancing the quality of life and reducing the disease burden.

Evaluation of treatment patterns, healthcare resource utilization and cost of illness for sickle cell disease in Ghana: a private medical insurance claims database study.

BACKGROUND: Sickle cell disease (SCD) is a major public health concern in sub-Saharan Africa, accounting for nearly 75% of the global disease burden. The current analysis evaluated patient characteristics, treatment patterns, healthcare resource utilization (HCRU) and associated costs in patients with SCD based on a Private Medical Insurance Database in Ghana. METHODS: This retrospective longitudinal cohort study was conducted using an e-claims database from Ghana (01 January 2015 to 31 March 2021). Patients were stratified by age (0 month to < 2 years, ≥ 2 years to ˂6 years, ≥ 6 years to < 12 years, ≥ 12 years to < 16 years; ≥16 years), vaso-occlusive crisis (VOC) (< 1, ≥ 1 to < 3, and ≥ 3 per year), and continuous enrolment. Study outcomes related to patient characteristics, comorbidities, treatment pattern, HCRU were evaluated for pre- and post-index period (index period was between July 2015 to March 2020). Descriptive analysis was used to analyse different study variables. RESULTS: The study included 2,863 patients (mean age: 20.1 years; Min age: 0; Max age: 83; females 56.1%). Overall, 52.2% (n = 1,495) of SCD patients were ≥ 16 years and 17.0% (n = 486) were in the ≥ 2 to ˂6-years age group. The majority of patients aged ≥ 16 years (62.5%) in the database did not have reported VOC episodes, 35.9% of patients had 1 to 3 VOCs per year and 1.5% had ≥ 3 VOCs per year during the follow-up period. Consultation-based prevalence of SCD was 0.5% [95% confidence interval (CI): 0-1.3%] - 1.4% [CI: 0.6-2.2%]. Malaria, upper respiratory tract infection (URTI) and sepsis were the common complications of SCD. Analgesics were the most frequently prescribed medications followed by anti-infectives, hematinics, and antimalarials. Hydroxyurea, a routine standard of care for SCD was under-utilized. SCD patients had median cost incurred for consultation/hospital services of $11.3 (Interquartile range [IQR] $6.2 - $27.2). For patients with VOC, maximum median cost was incurred for medications ($10.9 [IQR $5.0-$32.6]). Overall median healthcare cost was highest for individuals with ≥ 3 VOCs per year during the follow-up period ($166.8 [IQR $70.3-$223.5]). CONCLUSION: In this retrospective private insurance claims database analysis, SCD imposes a significant healthcare burden, especially in patients with VOC. There is a need for reimbursed treatment options that could reduce the long-term burden associated with SCD and VOC.

Pregnancy-related thromboembolism in women with sickle cell disease: An analysis of National Medicaid Data.

Pregnancy and sickle cell disease (SCD) both individually carry a risk of thromboembolism (TE). Pregnancy in people with SCD may further enhance the prothrombotic effect of the underlying disease. The objectives of this study were to determine the rate and risk factors for arterial and venous thrombosis in pregnant people with SCD. Administrative claims data from the United States Centers for Medicare and Medicaid Service Analytic eXtract from 2006 to 2018 were used. The study population included people with SCD from the start of their first identified pregnancy until 1 year postpartum and a control cohort of pregnant people without SCD of similar age and race. Outcomes of interest were identified with ICD-9 or 10 codes. Logistic regression analyses were used to analyze risk factors. We identified infant deliveries in 6388 unique people with SCD and 17 110 controls. A total of 720 venous thromboembolism (11.3%) and 335 arterial TE (5.2%) were observed in people with SCD compared to 202 (1.2%) and 95 (0.6%) in controls. People with SCD had an 8-11 times higher odds of TE compared to controls (p < .001). Within the SCD cohort, age, hemoglobin SS (HbSS) genotype, hypertension, and history of thrombosis were identified as independent risk factors for pregnancy-related TE. Pregnancy-specific factors (pre-eclampsia, eclampsia, multigestational pregnancy) were not associated with TE. In conclusion, the risk of pregnancy-related TE is considerably higher in people with SCD compared with controls without SCD. Hence, people with SCD, particularly those with multiple risk factors may be candidates for thromboprophylaxis during pregnancy and the postpartum period.

Comparison of thromboembolism outcomes in patients with sickle cell disease prescribed hormonal contraception.

Patients with sickle cell disease (SCD) are at a risk of thromboembolism (TE), and use of hormonal contraception can further increase that risk. This study aims to assess patterns of hormonal contraceptive use and compare risk of contraception-related TE between combined hormonal contraceptives (CHCs) and progestin-only contraceptives (POCs). Patients with SCD aged between 12 and 44 years with a new prescription of a hormonal contraceptive in the Centers for Medicare and Medicaid Services Medicaid Analytic eXtract database (2006-2018) were followed up to 1 year. We identified 7173 new users: 44.6% initiated CHC and 55.4% initiated POC. Combined oral contraceptive pills (OCPs; 36.5%) and progestin-only depot medroxyprogesterone acetate (33.9%) were the most frequently prescribed agents. A total of 1.8% of contraception users had a new diagnosis of TE within 1 year of the first identified contraception prescription. There were no significant differences in TE event rates between CHC and POC users (17.2 and 24.7 events per 1000 person-years, respectively). In patients prescribed OCP, there were no differences in TE event rates based on estrogen dose or progestin generation. Transdermal patch had a 2.4-fold increased risk of TE as compared with that of OCP. Although limited by the retrospective study design and use of administrative claims data, this study found no significant differences in TE rates between new users of CHC and POC in patients with SCD. Careful evaluation of underlying TE risk factors should be considered for each patient with SCD before initiation of hormonal contraception.

Recent national trends in outcomes and economic disparities among adult sickle cell disease-related admissions.

Sickle cell disease (SCD) is an inherited disorder caused secondary to a mutation in the hemoglobin beta subunit. There is sparse information regarding the trends in outcomes of SCD admissions in the past decade where rapid advances have been made in treatment. In this study, we wanted to analyze the trends and outcomes of SCD admissions in the United States from 2011 to 2019 and the influence of socio-economic status. Data were obtained from the National Inpatient Sample (NIS) database using the International Classification of Disease (ICD-9) and ICD-10 codes. Trends for primary in-hospital outcomes including mortality, length of stay (LOS), and total hospitalization charges (THC) were assessed. The impact of economic status on these outcomes was also studied. There was an annual percent change (APC) in the number of admissions for SCD of + 2.5% from 2010 to 2015 (95% CI: 1.3-3.8%, p = 0.003). However, there was no significant change in the number of admissions between 2015 and 2019 (95% CI - 1.8-0.7%, p = 0.323). The overall mortality across the years has decreased by about 4% yearly at the population level (p = 0.008, 95% CI 2-8%). However, the inpatient mortality for the high-income group had decreased significantly from 2010 to 2019, whereas there was no difference in the mortality rate for the low-income group across the decade. Despite the advances in the understanding of SCD and its treatment, its benefits have not reached all the people affected. Meaningful progress in healthcare is not achievable unless these economic disparities are addressed. Economic policies to address these inequities are the need of the hour.

Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000-2021: a systematic analysis from the Global Burden of Disease Study 2021.

BACKGROUND: Previous global analyses, with known underdiagnosis and single cause per death attribution systems, provide only a small insight into the suspected high population health effect of sickle cell disease. Completed as part of the Global Burden of Diseases, Injuries, and Risk Factors Study (GBD) 2021, this study delivers a comprehensive global assessment of prevalence of sickle cell disease and mortality burden by age and sex for 204 countries and territories from 2000 to 2021. METHODS: We estimated cause-specific sickle cell disease mortality using standardised GBD approaches, in which each death is assigned to a single underlying cause, to estimate mortality rates from the International Classification of Diseases (ICD)-coded vital registration, surveillance, and verbal autopsy data. In parallel, our goal was to estimate a more accurate account of sickle cell disease health burden using four types of epidemiological data on sickle cell disease: birth incidence, age-specific prevalence, with-condition mortality (total deaths), and excess mortality (excess deaths). Systematic reviews, supplemented with ICD-coded hospital discharge and insurance claims data, informed this modelling approach. We employed DisMod-MR 2.1 to triangulate between these measures-borrowing strength from predictive covariates and across age, time, and geography-and generated internally consistent estimates of incidence, prevalence, and mortality for three distinct genotypes of sickle cell disease: homozygous sickle cell disease and severe sickle cell ß-thalassaemia, sickle-haemoglobin C disease, and mild sickle cell ß-thalassaemia. Summing the three models yielded final estimates of incidence at birth, prevalence by age and sex, and total sickle cell disease mortality, the latter of which was compared directly against cause-specific mortality estimates to evaluate differences in mortality burden assessment and implications for the Sustainable Development Goals (SDGs). FINDINGS: Between 2000 and 2021, national incidence rates of sickle cell disease were relatively stable, but total births of babies with sickle cell disease increased globally by 13·7% (95% uncertainty interval 11·1-16·5), to 515 000 (425 000-614 000), primarily due to population growth in the Caribbean and western and central sub-Saharan Africa. The number of people living with sickle cell disease globally increased by 41·4% (38·3-44·9), from 5·46 million (4·62-6·45) in 2000 to 7·74 million (6·51-9·2) in 2021. We estimated 34 400 (25 000-45 200) cause-specific all-age deaths globally in 2021, but total sickle cell disease mortality burden was nearly 11-times higher at 376 000 (303 000-467 000). In children younger than 5 years, there were 81 100 (58 800-108 000) deaths, ranking total sickle cell disease mortality as 12th (compared to 40th for cause-specific sickle cell disease mortality) across all causes estimated by the GBD in 2021. INTERPRETATION: Our findings show a strikingly high contribution of sickle cell disease to all-cause mortality that is not apparent when each death is assigned to only a single cause. Sickle cell disease mortality burden is highest in children, especially in countries with the greatest under-5 mortality rates. Without comprehensive strategies to address morbidity and mortality associated with sickle cell disease, attainment of SDG 3.1, 3.2, and 3.4 is uncertain. Widespread data gaps and correspondingly high uncertainty in the estimates highlight the urgent need for routine and sustained surveillance efforts, further research to assess the contribution of conditions associated with sickle cell disease, and widespread deployment of evidence-based prevention and treatment for those with sickle cell disease. FUNDING: Bill & Melinda Gates Foundation.

Analysis of the 2007-2018 National Health Interview Survey (NHIS): Examining Neurological Complications among Children with Sickle Cell Disease in the United States.

This study compared neurological complications among a national sample of United States children with or without sickle cell disease (SCD) and evaluated health status, healthcare and special education utilization patterns, barriers to care, and association of SCD status and demographics/socioeconomic status (SES) on comorbidities and healthcare utilization. Data was acquired from the National Health Interview Survey (NHIS) Sample Child Core questionnaire 2007-2018 dataset that included 133,542 children. An affirmation from the guardian of the child determined the presence of SCD. Regression analysis was used to compare the associations between SCD and demographics/SES on neurological conditions at p < 0.05. Furthermore, adjusted odds ratios (AORs) were estimated for having various neurological conditions. Of the 133,481 children included in the NHIS, the mean age was 8.5 years (SD: 0.02) and 215 had SCD. Of the children with SCD, the sample composition included male (n = 110), and Black (n = 82%). The SCD sample had higher odds of having neuro-developmental conditions (p < 0.1). Families of Black children (55% weighted) reported household incomes < 100% of federal poverty level. Black children were more likely to experience longer wait times to see the doctor (AOR, 0.3; CI 0.1-1.1). Compared to children without SCD, those with SCD had a greater chance of seeing a medical specialist within 12 months (AOR 2.3; CI 1.5-3.7). This representative sample of US children with SCD shows higher odds of developing neurological complications, increased healthcare and special education services utilization, with Black children experiencing a disproportionate burden. This creates the urgency to address the health burden for children with SCD by implementing interventions in healthcare and increasing education assistance programs to combat neurocognitive impairments, especially among Black children.