UNASSIGNED: Sickle cell disease (SCD) is a severe hereditary form of anemia that contributes between 50% and 80% of under-five mortality in Africa. Eleven thousand babies are born with SCD annually in Tanzania, ranking 4th after Nigeria, the Democratic Republic of Congo and India. The absence of well-described SCD cohorts is a major barrier to health research in SCD in Africa.
UNASSIGNED: This paper describes the Sickle Pan African Consortium (SPARCO) database in Tanzania, from the development, design of the study instruments, data collection, analysis of data and management of data quality issues.
UNASSIGNED: The SPARCO registry used existing Muhimbili Sickle Cell Cohort (MSC) study case report forms (CRF) and later harmonized data elements from the SickleInAfrica consortium to develop Research Electronic Data Capture (REDCap) instruments. Patients were enrolled through various strategies, including mass screening following media sensitization and health education events during World Sickle Cell Day each June and the SCD awareness month in September. Additional patients were identified through active surveillance of previously participating patients in the MSC.
UNASSIGNED: Three thousand eight hundred patients were enrolled between October 2017 and May 2021. Of these, 1,946 (51.21%) were males and 1,864 (48.79%) were females. The hemoglobin phenotype distribution was 3,762 (99%) HbSS, 3 (0.08%) HbSC and 35 (0.92%) HbSb +thalassemia. Hemoglobin levels, admission history, blood transfusion and painful events were recorded from December 2017 to May 2021.
UNASSIGNED: The Tanzania SPARCO registry will improve healthcare for SCD in Africa through the facilitation of collaborative data-driven research for SCD.

UNASSIGNED: This study aimed to determine the health-related quality of life (HRQoL) of adult Omani patients with sickle cell disease (SCD). The quality of life of these patients in Oman is unknown.
UNASSIGNED: This cross-sectional study was conducted at the Sultan Qaboos University Hospital, Muscat, Oman, from June to October 2022 and included patients with SCD. A validated Arabic version of the 36-Item Short-Form Health Survey (SF-36) was used to assess HRQoL in 8 domains.
UNASSIGNED: A total of 235 patients with SCD were included in this study, the majority of whom were female (74.9%) and between 18 and 35 years of age (64.6%). The lowest HRQoL was reported for the domain of role limitations due to physical health (median score = 25.0, interquartile range [IQR] = 35.0), while the highest was seen for role limitations due to emotional problems (median score = 66.7, IQR = 100.0). Frequent SCD-related emergency department visits/hospital admissions and the adverse effect of SCD on patients\' social lives had a significant negative impact on SF-36 scores for all 8 HRQoL domains (P ≤0.05). Additionally, SCD\'s impact on academic performance and a history of having been bullied due to SCD had a significant negative impact on SF-36 scores for 7 domains (P ≤0.05).
UNASSIGNED: Omani adult patients with SCD reported relatively poor HRQoL in several domains, with various factors found to be significantly associated with this. Healthcare providers and policy makers should consider offering additional clinical, educational and financial support to these patients to enhance their HRQoL.

Tanzania is one of the countries with a high burden of sickle cell disease (SCD). Haemolytic anaemia is a clinical feature of SCD, and has been linked to major complications leading to morbidity and mortality. Treatment with hydroxyurea (HU) has shown to induce foetal haemoglobin (HbF) which in turn decreases haemolysis in patients. This study aimed to investigate the effects of HU on haemolysis in SCD patients attending Muhimbili National Hospital, Tanzania by comparing their haemolytic parameters before and after therapy. Patients meeting the criteria were initiated on HU therapy for 3 months. Two haemolytic biomarkers: unconjugated plasma bilirubin levels and absolute reticulocyte counts were measured from patients\' blood samples at baseline and after 3 months of HU therapy and compared. Both absolute reticulocyte counts and indirect plasma bilirubin levels significantly declined after HU therapy. Median (IQR) plasma unconjugated bilirubin levels dropped significantly from 20.3 (12.7-34.4) μmol/L to 14.5 (9.6-24.1) μmol/L (p < 0.001) and mean (SD) absolute reticulocyte counts dropped significantly from 0.29 (0.1) × 109/L to 0.17 (0.1) × 109/L (p < 0.001) after therapy, thus, a decline in both haemolytic biomarkers after treatment was observed. This study found a potential for use of HU therapy in managing SCD patients in our settings evidenced by improvements in their haemolytic parameters. Clinical trials with a lager sample size conducted for a longer time period would be beneficial in guiding towards the inclusion of HU in treatment protocols for the Tanzanian population.

People with sickle cell disease experience a high incidence of chronic kidney disease and end-stage kidney disease, secondary to tubular and glomerular effects of vaso-occlusion-induced hypoxia. Because of concerns of suboptimal kidney function, sickle cell donors are usually not considered for kidney donation, even if the rest of the parameters are acceptable for organ donation. A significant gap exists between the number of organ donors and the number of candidates waiting for a kidney transplant in the United States. To bridge the gap, we need to consider using nontraditional donors. We report kidney transplant outcomes in 6 recipients from 4 sickle cell kidney donors. Intracranial hemorrhage and sepsis were the causes of the death in donors, and no donor was in sickle cell crisis at the time of donation. None of the recipients experienced delayed graft function, and all recipients achieved excellent allograft function. The earliest allograft failure was at 27 months in a recipient who developed early acute rejection, while the longest follow-up was 10 years with adequate kidney function. In conclusion, given the shortage of kidneys for transplantation and demonstrated good outcomes, we propose that kidneys from sickle cell donors can be safely used.

Background/Objectives: Sickle cell disease (SCD) impacts about 100,000 people in the US. SCD increases the risk of cholelithiasis and microvascular ischemia, which could increase the risk of acute pancreatitis (AP). Abdominal pain is a common presenting symptom of AP and sickle cell vaso-occlusive crisis. The purpose of our systematic review is to estimate the prevalence and determine the severity of AP in individuals with SCD compared to the general population. Methods: Multiple electronic databases were searched. We included studies that included children and adults (population) and addressed the association of SCD (exposure) with AP (outcome) compared to the same population without SCD (control). Two authors screened titles and abstracts independently, and data were abstracted in duplication from included studies. We registered this protocol in PROSPERO-CRD42023422397. Results: Out of 296 studies screened from multiple electronic databases, we identified 33 studies. These studies included 17 case reports, one case series, and 15 retrospective cohort studies, and 18 studies included children. Eight of the AP case reports were in patients with HbSS genotype, two with sickle beta thalassemia, and one with HbSoArab, and in six case reports, a genotype was not specified. Complications were reported in 11 cases-respiratory complication (in at least four cases), splenic complications (three cases), pancreatic pseudocyst (two cases) and death from AP (one case). Of the four AP cases in the case series, three had HbSS genotype, and two cases had complications and severe pancreatitis. AP prevalence in SCD was estimated to be 2% and 7% in two retrospective studies, but they lacked a comparison group. In retrospective studies that evaluated the etiology of AP in children, biliary disease caused mostly by SCD was present in approximately 12% and 34%, respectively. Conclusions: Data on the prevalence of AP in individuals with SCD are limited. Prospectively designed studies aiming to proactively evaluate AP in individuals with SCD who present with abdominal pain are needed to improve timely diagnosis of AP in SCD and outcomes.

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are hereditary haemoglobinopathies characterized by a reduction in functional β-globin chains. Both conditions cause tiredness and increase susceptibility to infection, which can lead organ failure, significantly reducing life expectancy and typically requiring those affected to undergo regular erythrocyte transfusion. Recently, a novel therapeutic treatment for SCD and TDT was approved by the UK regulatory body (Medicines and Healthcare products Regulatory Agency; MHRA). Exagamglogene autotemcel (Casgevy) is the first licensed therapy globally to utilize CRIPSR/Cas9 technology and induces an increase in expression of γ-globin chains to compensate for the reduction in functional β-globin. Casgevy represents a first-in-class therapeutic, and numerous considerations were made by the MHRA throughout its assessment of the medicine. These include, but are not limited to, the risk of tumorigenicity and off-target editing, a limited cohort size, the validity of proposed dosing and the conduction of only single-arm studies. The MHRA\'s analyses of the data to support the proposed indications are presented and discussed throughout this manuscript. Overall, the sponsors claims were considered well supported by their data, and Casgevy was licensed for the treatment of TDT or SCD in patients 12 years of age and older for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen-matched related HSC donor is not available.

BACKGROUND: Sickle cell disease (SCD) is a common monogenic inheritable condition in sub-Saharan Africa. 30% of Ghanaians are estimated to be carriers creating a condition for stable SCD penetrance. Being inheritable, SCD is scientifically preventable through intentional spousal selection. This study therefore explored young adults\' knowledge, beliefs and prevention strategies regarding SCD.
METHODS: This exploratory study employed a mixed-methods approach (semi-structured questionnaires and focus group discussion [FGD]) to explore the SCD knowledge and beliefs of participants (15-49 years). The data collection was intentionally sequential; initial administration of 386 questionnaires and then followed by the FGD (16 participants). FGD was thematically analysed whereas quantitative data was explored using structural equation modeling (SEM); p < 0.05 was considered statistically significant under two-tailed assumptions.
RESULTS: Whereas 98.7% reported having heard about SCD, < 50% got the information through formal education. Overall, 49.7% knew their respective SCD status; the proportion increased with age (48.8% in < 30 years vs 64.4% in ≥ 30 years old), or higher degree status (48.8% in undergraduates vs 67.4% in postgraduates). Moreover, whereas nine-in-ten correctly identified that SCD is hereditable, three-in-ten believed that having a SCD child was a matter of fate. Our FGD revealed that whereas curses, and spiritual attack were misconceived as potential causes of SCD, stigmatization associated with SCD was a major concern. The SEM demonstrated that one\'s knowledge about SCD prevention is significantly positively associated with relationship choices (b = 0.757, p < 0.05). Also, a participant\'s knowledge about SCD preventive strategies was significantly associated with the individual\'s beliefs about SCD (b = 0.335; p < 0.05). However, a participant\'s SCD beliefs negatively mediated SCD preventive strategies-relationship choices association.
CONCLUSIONS: SCD beliefs likely transform linear quantitative associations into a complex non-linear interaction; public health campaigns ought to unearth and address SCD beliefs to maximize achieving the intended targets.

BACKGROUND: Sickle cell disease (SCD) affects approximately 100,000 people in the United States and millions worldwide, with the highest prevalence of 70% of SCD being found in individuals of African ethnicity. Delayed hemolytic, alloimmunization, and anamnestic transfusion reactions in multiple transfusion patients need to be investigated and managed to avoid a worsening of the patient\'s clinical status.
OBJECTIVE: This paper aims to investigate delayed transfusion reactions in SCD patients who were polytransfused in the Brazilian Amazon.
METHODS: The clinical and laboratory indicators of SCD patients with more than four transfusions were investigated. The patients were treated at the Fundação Hospitalar de Hematologia e Hemoterapia do Estado do Amazonas, Brazil.
RESULTS: A total of 44 polytransfused patients with SCD were followed. Regarding Rh phenotype, it was possible to observe a frequency of 26.6% (12) patients with the RZRZ (DCE/DCE) phenotype, in addition to 4.5% (two) patients with RH and RHCE variants. It was also possible to observe 20.5% (nine) patients with an alloimmunization reaction, who presented the following alloantibodies: anti-RhD, anti-E, anti-K, anti-Jkb, anti-N, anti-S, and anti-Dia, two of which are unidentified. Of these, four (44.4%) patients also presented autoantibodies, anti-e, and three unidentified antibodies, and four (44.4%) patients presented an anamnestic reaction, with anti-RhD, K, and Jkb antibodies. Of the 44 patients monitored, 54.4% (24) had clinical and laboratory indicators of a delayed hemolytic reaction.
CONCLUSIONS: Delayed transfusion reactions, often neglected, occur frequently. Therefore, transfusions need to be monitored for at least 28 days, with medical investigation of clinical and laboratory indicators to make greater use of this therapeutic resource.

Sickle cell disease often leads to avascular necrosis (AVN) of the hip joint, resulting in joint pain and restricted range of motion. In cases where traditional treatments like total hip arthroplasty or core decompression may not suffice, the Girdlestone procedure, involving the resection of the femoral head, is considered. This case study centers on a 19-year-old male nursing student with sickle cell disease who underwent a Girdlestone procedure at 16 years of age, seeking relief from hip pain and limited mobility. However, the procedure led to leg length discrepancy and reduced hip function. Subsequent total hip arthroplasty successfully converted the prior procedure into a stable joint, improving the patient\'s range of motion and eliminating pain. The comprehensive surgical approach, including soft tissue releases and postoperative rehabilitation, significantly enhanced the patient\'s quality of life, emphasizing the importance of total hip arthroplasty as a superior intervention post-Girdlestone procedure.

This study examined sickle cell disease (SCD) in Saudi Arabia. A systematic search of relevant databases was conducted to identify studies investigating SCD in the Saudi population. Studies were then screened based on predefined criteria and critically appraised for methodological quality. Data was extracted and synthesized to provide an overall picture of the SCD burden in Saudi Arabia. The most commonly reported complications were vaso-occlusive crises (VOC), acute chest syndrome (ACS), acute painful crisis, splenic sequestration, osteomyelitis, aplastic crisis, hemolytic crisis, serious bacterial infections, chronic vascular occlusion (CVO), depression, sickle cell nephropathy (SCN), obstructive sleep apnea (OSA), and renal complications. Reduced blood levels of antioxidant trace elements (Cu, Zn, and Se) may encourage oxidative stress, which in turn may contribute to the pathophysiology of SCD. Infections and ACS were common among young children (<7 years) while pain attacks were common in older children (>7 years). The high rate of hospitalizations among SCD patients highlights the need for better management strategies. Future research should focus on understanding the underlying causes of SCD complications and developing new ways to control them.