Painful Crises Research Articles

MEGALOBLASTIC CRISIS IN SICKLE CELL DISEASE–A RARE PHENOMENA WITH AVAILABLE REVIEW OF LITERATURE

Sickle cell disease (SCD) is an inherited disease where some red blood cells are shaped like sickles or crescent moons. This condition affects the blood and various organs of the body. These sickle cells also become rigid and sticky, which can slow or block blood flow, resulting in episodes of sickness that produce pain and other crisis. There are different types of crisis. SCD with a megaloblastic crisis is rare. The frequency of the carrier state determines the prevalence of sickle cell anemia. Here we describe a 20 y old female patient with sickle cell disease who experienced a megaloblastic crisis.

Association of Clinical and Paraclinical Factors with Pain Crisis Severity in Sickle-Thalassemia Patients in Iran

Objectives: This study aimed to evaluate the effects of several factors, including hospitalization frequency, inflammatory and hemolytic markers, and the numbers of blood transfusions and crises, on the severity of pain crises in sickle-thalassemia patients. Methods: Of all patients visiting hematologists in university-affiliated hospitals and clinics, only 75 sickle cell disease (SCD) and sickle-thalassemia patients met the inclusion criteria for this study. Disease severity was measured based on pain crises per year and laboratory markers, including hemolytic and inflammatory indicators, outpatient or inpatient status, the number of hospital admissions, and the number of blood units transfused over one year. Results: In sickle cell patients, a significant correlation was observed between hospital admission and WBC (0.01), ALP (0.001), MCHC (0.001), ferritin (0.021), and ESR (0.006). Additionally, pain crises were positively correlated with transferrin saturation (TS) (+0.28) and the number of transfused blood units (+0.49) and negatively correlated with Hb (-0.30) and total iron binding capacity (TIBC) (-0.23). Blood unit transfusions showed a positive correlation with serum iron (+0.44), RDW (+0.36), ferritin (+0.39), pain crises (+0.49), and TS (+0.49), along with a negative correlation with Hb (-0.75) and MCHC (-0.33). When categorizing pain crises into two groups, patients experiencing more frequent crises generally exhibited lower hemoglobin levels and a higher number of blood units transfused. Hemoglobin also showed a significant negative correlation with lactate dehydrogenase (LDH) level in both groups: -0.604 (P = 0.005) in patients with ≥ 3 crises per year and -0.368 (P = 0.006) in patients with < 3 crises per year. Patients with hemoglobin levels of 7.5 or higher tended to maintain an LDH level below 1000. Among patients referred with pain crises, hemoglobin was negatively correlated with hemolytic markers such as LDH (-0.41) and retic (-0.29). Conclusions: Hemoglobin and LDH can serve as follow-up markers, as they are routinely measured in all sickle cell patients and, in this study, hemoglobin levels of 7.5 or higher and LDH levels below 1000 were associated with fewer and less severe pain crises. Regular monitoring of hemolytic markers may help in pain crisis prevention, as this study found that hemolytic crises frequently coincided with pain crises. Further research is needed to provide conclusive evidence in this area.

Prophylactic red cell transfusions for sickle cell disease pregnancy: increased use of therapy could transform outcomes.

Pregnancy for people with sickle cell disease (SCD) is high risk with persistently high rates of severe maternal and fetal mortality and morbidity. Transfusion therapy is the best-studied treatment for SCD in pregnancy; hydroxyurea is not usually used because of teratogenicity concerns. In high-resource settings, red cell transfusions are likely underutilized, while in low-resource settings, they may be altogether unavailable. A randomized controlled trial and meta-analysis, two of the strongest forms of clinical research, show transfusion significantly reduces maternal and fetal death, painful crisis, thrombosis, and acute respiratory failure. Downstream benefits of treatment are less well measured and may include improving maternal anemia, reducing opioid exposure, and avoiding hospitalization, which presents risk for additional complications. Alloimmunization is a particular transfusion risk in SCD. However, many strategies can mitigate this risk. Accordingly, the American Society of Hematology classifies chronic transfusion in pregnancy as low risk. Given the low risk classification, lack of alternative therapies, dismal, stagnant pregnancy outcomes and the potential for profound treatment benefit, wider use of chronic transfusion therapy for SCD pregnancy is likely indicated. This review discusses the benefits and potential risks of prophylactic transfusions for SCD pregnancy. Use of chronic transfusions during pregnancy is indicated to help urgently transform outcomes.

Burden of vaso-occlusive crisis, its management and impact on quality of life of Indian sickle cell disease patients.

Sickle cell disease (SCD) with vaso-occlusive pain crisis (VOC) significantly impacts patient well-being and often results in extensive healthcare resource utilization. This study assessed the VOC burden, its management and its impact on patients' quality of life (QoL). A cross-sectional observational study was conducted between November 2021 and June 2022, including 1000 SCD patients from high-prevalence states in India. Data on demographics, clinical characteristics, VOC severity, management and QoL were collected. The study revealed that 33.5% of patients reported at least one VOC episode during the study period. In the year prior to their enrolment, 836 (83.60%) patients reported at least one VOC episode, with an equal proportion of 407/487 (83.6%) adults and 429/513 (83.6%) paediatric patients, reducing their QoL across all domains compared to patients without VOC. Of these, 469/1000 patients (46.9%) experienced ≥3 VOC episodes. Additionally, 764/1000 (76.40%) patients managed their VOCs at healthcare facilities, with 501/1000 (50.1%) requiring inpatient admissions. Further, 71.80% of patients received Hydroxyurea (HU) therapy. The study depicts the severity of the Arab-Indian haplotype in Indian SCD patients visiting healthcare settings based on high VOC burden. This highlights the urgent need for better management strategies and resource allocation for these patients.

Patient-reported pregnancy loss and maternal complications: Insights from the sickle cell disease implementation consortium.

Sickle cell disease (SCD) is associated with complications during pregnancy and can negatively influence maternal outcomes. Our study aimed to determine the prevalence and predictors of maternal morbidity among participants enrolled in an eight-site SCD Implementation Consortium (SCDIC) registry. We conducted a cross-sectional analysis of female registry participants, aged 15-45 years, with a confirmed diagnosis of SCD. Participants completed a survey of self-reported pregnancies and outcomes. Seven hundred and thirty-eight individuals had at least one pregnancy event, with 1076 live births. Twenty percent reported a pregnancy loss or fetal demise. Of the 1076 live births, 75% involved at least one complication. The most prevalent complications were pain crises (61.1%) and pregnancy requiring blood transfusion(s) (33.0%). Multiparous individuals with a prior occurrence of a complication in a previous pregnancy had higher odds of recurrence of the same complication in subsequent pregnancies (i.e., previous acute crisis was associated with subsequent acute pain events odds ratio [OR]: 3.13; 95% confidence interval [CI]: 2.06-4.76) and prior transfusion requiring another transfusion (OR: 3.22; 95% CI: 2.01-5.16). Individuals reported a high prevalence of pregnancy loss and maternal complications. Our findings underscore the importance of preconception counseling and early initiation of perinatal care in SCD.

The Current Role of Hydroxyurea in the Treatment of Sickle Cell Anemia.

Despite advancements in treatment of sickle cell disease (SCD), hydroxyurea, a ribonucleotide reductase inhibitor, remains the cornerstone of therapy. While its primary effect is the elevation of fetal hemoglobin (HbF), hydroxyurea's mechanisms of action are multifaceted. Hydroxyurea (HU) reduces leukocyte and platelet counts, decreases the expression of endothelial adhesion molecules CD36 and CD49d, and increases nitric oxide and cyclic nucleotide levels, which may facilitate vascular dilation and further HbF induction. Numerous studies have demonstrated that hydroxyurea therapy reduces the frequency of painful episodes, acute chest syndrome, and the need for erythrocyte transfusions and hospitalizations. Long-term use of hydroxyurea leads to reduced morbidity and mortality. Hydroxyurea should be initiated in children from 9 months of age, including asymptomatic individuals, and is recommended for adults experiencing pain crises that significantly interfere with daily activities or quality of life, as well as those with severe or recurrent vaso-occlusive crises, ACS, or severe symptomatic anemia. Hydroxyurea is not recommended during pregnancy or lactation due to potential teratogenic effects and transfer into breast milk. However, its use may be considered in high-risk patients, particularly during the second and third trimesters. Concerns about secondary tumor development have not been substantiated in long-term follow-up studies. Alternative therapies, including L-glutamine, crizanlizumab, and voxelotor, are not presently approved or available for clinical use in Europe.

Prescribing Hydroxyurea in Sickle Cell Disease Patients: The Pattern and Association with Co-Prescribed Medications Used to Manage the Disease Complications.

Background and Objectives: Hydroxyurea (HU) is an effective medication used to reduce the frequency of painful crises associated with sickle cell disease (SCD). However, data describing its prevalence among SCD patients in the Eastern Region of Saudi Arabia are scarce. This is a multi-center, retrospective, cross-sectional study that aims to investigate the pattern of prescribing HU in SCD patients and to determine the association between prescribing HU and other co-prescribed medications used to manage SCD complications. Methods: Data were collected from patients who visited the hematology clinics of Al-Qatif Central Hospital (QCH) and King Fahad Hospital in Hofuf (KFHH) between June 2021 to May 2023. The data included demographics, prescribed medications, and recent laboratory test results, all of which were collected from patients' medical records. Descriptive statistics were utilized to assess the difference between HU users vs. non-users. A binary logistic regression model was used to determine the association between prescribing HU and co-prescribed medications used to manage SCD complications. The results are presented as the odds ratio (OR) and 95% confidence interval (95% CI). Results: This study included 2816 SCD patients with a 56% prevalence of HU prescription. HU was prescribed for young age groups more often compared to old age group patients. Young males were more likely to be prescribed with HU compared to females, and it becomes dominant in females after the age of 36. HU users were more likely to have paracetamol (69% vs. 53%, OR = 1.9, 95% CI 1.6-2.2), NSAIDs (50% vs. 35%, OR = 1.7, 95% CI 1.5-2), and opioids (41% vs. 37%, OR = 1.3, 95% CI 1.1-1.6) co-prescribed, and less often to have laxatives (8% vs. 5%, OR = 0.66, 95% CI 0.48-0.9) and anticoagulants (22% vs. 15%, OR = 0.56, 95% CI 0.46-0.68) co-prescribed compared to non-users. Conclusions: The pattern of prescribing HU, supported by the association findings, raises concerns about patients' compliance and adherence to HU therapy. Early health education, specifically to young female SCD patients, is warranted to increase the success rate of HU therapy.

Navigating the nexus of personalized medicine and healthcare management: Advancing paediatric pain management modalities in sickle cell disease through thematic analysis for ages 1–12

ABSTRACT Sickle cell disease (SCD) affects millions of people and is a significant public health concern due to the severe pain crises it causes, often requiring emergency care and leading to hospitalization. Effective pain management strategies are crucial to improving the quality of life of children with SCD, particularly those aged 1 to 12 years, who are especially vulnerable. This study evaluates the effectiveness and suitability of various healthcare management strategies, with a specific focus on pain management modalities for this paediatric population. Through a thematic analysis of 36 articles from major healthcare databases, five key themes were identified: cognitive behavioral therapy (CBT) and complementary alternative medicine (CAM), digital and technological pain management methods, pain medications, individualized pain plans and enhancement strategies aimed at improving protocols. The findings highlight critical opportunities to advance pain management approaches and address current care gaps. The study underscores the importance of integrating parents and caregivers into the care process and addressing health inequalities that affect treatment outcomes. Evidence reveals that existing research on paediatric SCD pain management remains inadequate, and future studies are urgently needed to establish clear guidelines that improve treatment efficacy and translate into actionable evidence-based practices for this vulnerable population.

Liver and renal biochemical profiles of people with sickle cell disease in Africa: a systematic review and meta-analysis of case-control studies

BackgroundSickle cell disease (SCD) is a genetic blood disorder characterized by a painful vaso-occlusive crisis due to the sickling of red blood cells in capillaries. Complications often lead to liver and renal dysfunctions, contributing to morbidity and mortality, particularly for children under 5. This systematic review and meta-analysis aimed to evaluate the liver and renal functions of people with SCD (HbSS) compared to those without it (HbAA) in Africa.MethodsThe protocol was registered with PROSPERO (CRD42022346771). We searched PubMed, Embase, Web of Science, and Google Scholar using the keywords “liver function”, “renal function”, “sickle cell disease”, and “Africa” on 6th May 2023 for peer-reviewed articles with abstracts in English. We included case-control studies comparing SCD (HbSS) with controls without hemoglobinopathies (HbAA). We used the random-effect model to calculate the pooled average values for the blood tests of people with SCD in RStudio version 4.2.2.ResultsOverall, 17 articles were analyzed from five African countries involving 1312 people with SCD and 1558 controls. The pooled mean difference of liver enzymes aspartate transaminase (AST) was 8.62 (95% CI − 2.99–20.23, I2 = 97.0%, p < 0.01), alanine transaminase (ALT) 7.82 (95% CI − 0.16–15.80, I2 = 99%, p < 0.01) and alkaline phosphatase (ALP) − 2.54 (95% CI − 64.72 – 59.64, I2 = 99%, p < 0.01) compared to controls. The pooled mean difference for the renal biochemical profiles creatinine − 3.15 (95% CI − 15.02; 8.72, I2=99%, p < 0.01) with a funnel plot asymmetry of t = 1.09, df = 9, p = 0.3048 and sample estimates bias of 6.0409. The pooled mean difference for serum urea was − 0.57 (95% CI − 3.49; 2.36, I2 = 99%, p < 0.01), and the estimated glomerular filtration (eGFR) rate was 19.79 (95% CI 10.89–28.68 mL/min/1.73 m2, I2 = 87%, p < 0.01) compared to controls.ConclusionPeople with SCD have slightly elevated liver enzymes and estimated glomerular filtration rates compared to controls in Africa. With all the heterogeneity (I2) > 50%, there was substantial variation in the reported articles’ results.Systematic review registrationPROSPERO CRD42022346771

8194 Extramedullary Hematopoiesis Involving Bilateral Adrenal Glands In A Patient With Sickle Cell Anemia

Abstract Disclosure: V.T. Pham: None. L.N. Sendos: None. Title: Extramedullary hematopoiesis involving bilateral adrenal glands in a patient with sickle cell anemia Background: Extramedullary hematopoiesis refers to blood cell production occurring outside the bone marrow. It is a reactive process caused by inadequate production or excessive destruction of blood cells typically seen in patients with hereditary anemias or infiltrative bone marrow disorders. Extramedullary hematopoiesis usually occurs in the spleen, liver and lymph nodes, but it can also very rarely arise from the adrenal glands. Case Presentation:We present a case of a 42-year-old male with a history of sickle cell anemia who presented with chest pain and shortness of breath and had a chest CT angiography which incidentally showed a 5.6 x 3.8 cm left adrenal mass and a 2.9 x 2.1 cm right adrenal mass. Further evaluation with an MRI abdomen with and without contrast revealed a 5.5 cm lobulated left adrenal mass with patchy areas of microscopic fat and faint delayed heterogeneous enhancement as well as a 2.2 cm right adrenal nodule. The nodules were described as markedly T2 hypointense with heterogeneous T1 hyperintense/isointense signal on MRI with a broad differential diagnosis including extramedullary hematopoiesis in bilateral adrenal glands, lipid poor adenoma or neoplasm. Regarding his sickle cell disease history, the patient was diagnosed as an infant and had very rare episodes of sickle cell pain crisis as a child. However, once he entered adulthood, he began to have multiple episodes of sickle cell pain crisis requiring hospitalizations and transfusions. On further review of patient’s prior imaging scans during these previous hospitalizations, he was first noted to have bilateral sub centimeter adrenal nodules on a CT Abdomen without contrast done almost a decade prior to his current presentation. We ordered biochemical evaluation which did not show any evidence of adrenal hormonal hypersecretion. His case was discussed at our multidisciplinary conference with the radiology and surgery team and it was concluded that the imaging characteristics were most consistent with extramedullary hematopoiesis in the setting of bilateral adrenal gland involvement and patient’s history of sickle cell anemia. Therefore, no immediate surgical resection was recommended. Given the size of the left adrenal mass being greater than 4 cm in size, we advised follow-up imaging for surveillance. Discussion:There has been a rise in detection of adrenal incidentalomas due to the increased use of advanced diagnostic imaging in healthcare settings. When patients with hematologic diseases are found to have adrenal incidentalomas, it is important to consider extramedullary hematopoiesis in the differential diagnosis to avoid unnecessary invasive procedures and surgeries. Presentation: 6/2/2024

Getting Help During Active Pain Crises in Sickle Cell Disease: Patient and Caregiver Perspectives in Canada

Plain Language Summary What is this summary about? This is a plain language summary of an article originally published in Patient Preference and Adherence. This study aimed to identify the notable symptoms and impacts of sickle cell disease from the point of view of individuals living with sickle cell disease and their caregivers. The study also sought to understand the factors involved in individuals living with sickle cell disease’ and caregivers’ decisions and preferences if and when they seek care during a pain crisis. This summary describes: the symptoms experienced by adolescents and adults living with sickle cell disease and their severity; the treatments they and their caregivers prefer to use when they are experiencing a pain crisis; and the factors that go into deciding when and whether individuals living with sickle cell disease and their caregivers should seek outside help -- to go to a hospital, for example - during a pain crisis. What were the results? Individuals living with sickle cell disease undergoing an acute pain crisis and their caregivers consider many factors when deciding whether to seek care at a medical facility. These include: The intensity of their symptoms If the facility has a treatment plan for them already in place If the facility has long wait times If the facility has an understanding and compassionate staff The individual's age and ability to manage their daily responsibilities Racial bias, shown in the actions and lack of empathy of Emergency Department staff toward individuals living with sickle cell disease, frequently factored into the individuals' and caregivers’ decision-making and hinders their ability to get the treatment needed during a pain crisis. What do the results mean? There is a need for a unified care team at medical facilities that treats all individuals living with sickle cell disease with the same high level of empathy and treatment quality and for a strong support system for individuals living with sickle cell disease outside of the hospital or clinic. This is an abstract of the Plain Language Summary of Publication article. View the full Plain Language Summary PDF of this article to read the full-text Link to original article here

Potassium dynamics in sickle cell anemia: clinical implications and pathophysiological insights

Potassium dynamics are critical in the pathophysiology of sickle cell anemia (SCA), a genetic disorder characterized by the presence of abnormally shaped red blood cells that lead to various complications such as vaso-occlusive crises and hemolytic anemia. This review focuses on the clinical implications and pathophysiological insights of potassium regulation in SCA, highlighting its impact on disease progression and potential therapeutic strategies. The dysregulation of potassium transport in SCA leads to significant K+ efflux and cellular dehydration, exacerbating the sickling process. Dehydrated sickle cells, due to potassium loss, become more rigid and prone to causing blockages in small blood vessels, leading to painful vaso-occlusive crises and ischemia. Furthermore, chronic hemolysis in SCA, aggravated by potassium imbalance, contributes to severe anemia and systemic complications. These insights underscore the importance of maintaining potassium homeostasis to mitigate disease severity and improve patient outcomes. Therapeutic strategies targeting potassium regulation show promise in managing SCA. Inhibitors of the Gardos channel, such as senicapoc, have demonstrated potential in reducing sickling and hemolysis. Additionally, hydration therapy plays a crucial role in maintaining electrolyte balance and preventing RBC dehydration. A comprehensive approach that includes monitoring and correcting electrolyte imbalances, along with standard treatments like hydroxyurea and blood transfusions, is essential for effective disease management.

Impact of Etonogestrel-releasing contraceptive implant use in cisgender women with Sickle cell disease

BackgroundSickle cell disease is a hereditary hemolytic anemia that exposes women to increased health risks especially in pregnancy, with serious implications for the woman and fetus. Acute pain episodes can occur multiple times per month and result in reduction of quality of life and disruption of her life. ObjectiveTo assess the clinical, including pain and metabolic parameters of women with sickle cell disease using etonogestrel-releasing contraceptive implants. MethodsWomen with sickle cell disease, aged 18–40 years, with reports of pain crises in the preceding 3 months were included and followed up for 12 months. Blood samples were collected to evaluate the blood count, reticulocytes, liver profile (alkaline phosphatase, gamma-glutamyl transferase, alanine aminotransferase, aspartate aminotransferase, and total bilirubin and its fractions), lipid profile, and lactate dehydrogenase levels before and 6 and 12 months after implant insertion. The following clinical variables were analyzed every 3 months: bleeding pattern, blood pressure, weight, body mass index, pain intensity (assessed using a visual analogic scale (VAS) from 0 to 10), and frequency of pain crises. ResultsTwenty-three women completed the study. There were no differences in laboratory variables between baseline and 6 and 12 months after implant insertion. Similarly, clinical variables did not differ, except for pain intensity (VAS pre-insertion = 8 vs. VAS 12 months post insertion = 4; p = 0.005) and frequency of pain crises (pre-insertion = 6 vs. 12 days/month post insertion = 0; p = 0.000). ConclusionsEtonogestrel-releasing contraceptive implants were associated with a reduction in the intensity and frequency of pain crises in women with sickle cell disease. Moreover, it was safety among these population due to no changes in laboratory parameters during the first 12 months of use.

Impact of oral health conditions on the quality of life of children and adolescents with sickle cell anemia: A cross-sectional study.

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. To evaluate the impact of oral health conditions on the quality of life (QoL) of children and adolescents with SCA. 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%). 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). Oral health impairment negatively affected the QoL of adolescents with SCA.

Effects of Air Pollution on Respiratory Events and Pain Crises among Children with Sickle Cell Disease in New York City.

The disease burden of sickle cell disease (SCD) is highest among U.S. Black and Hispanic populations, who are often disproportionately represented in communities with poor air quality. There is limited data on the effects of air pollution exposure and social environmental factors on health outcomes in children with SCD. The objective of our study was to examine the associations between air pollution exposure and acute respiratory and vaso-occlusive pain crises (VOCs), and further study the associations stratifying by asthma status and neighborhood disadvantages. We conducted a retrospective study collecting data on outpatient sick and ED visits, and hospital admissions for respiratory events (i.e., respiratory tract infections, asthma exacerbation, acute chest syndrome), and hospitalizations for VOCs among children with SCD in a tertiary care center in New York City (NYC) from 2015-2018. Modeled data from the NYC Community Air Survey data using home addresses estimated street-level, annual-average exposure to air pollution (i.e., black carbon (BC), fine particulate matter (PM2.5), and nitrogen dioxide (NO2)). The area deprivation index (ADI): continuous national ranking percentile (1-100) was used, representing a composite index for neighborhood-level social disadvantage. We further dichotomized study participants at the upper tertile (high vs. low ADI). Multivariable Poisson regression in generalized estimating equation (GEE) models were used to estimate relative risks (RR), after adjusting for potential covariates. A total of 114 children with SCD were included in this study and had between 1-4 annual repeated measures of annual average air pollutants over a total of 425 visits. Overall, there were no significant associations between air pollution levels and acute respiratory and VOCs among children with SCD and when stratified by asthma status. We found significant interactions between air pollution levels and the continuous ADI variable on respiratory outpatient and frequent respiratory outpatient/ED visits (p<0.1). When stratified by high ADI, increased exposure to PM2.5 was significantly associated with more frequent respiratory outpatient/ED visits among children residing in higher ADI neighborhoods (RR (95% CI)= 1.13 (1.01, 1.27), p<0.05), but not among those in lower ADI neighborhoods. Increased exposure to NO2 was associated with more outpatient respiratory events for children in high ADI neighborhoods (RR (95%CI= 2.74 (1.24, 6.08), p<0.05), compared with low ADI neighborhoods. Air pollution exposures increased respiratory complications among children with SCD living in deprived neighborhoods.

Sex-disaggregated analysis of acute kidney injury in hospitalized children with sickle cell anemia in Uganda.

A growing body of research is categorizing sex differences in both sickle cell anemia (SCA) and acute kidney injury (AKI); however, most of this work is being conducted in high-resource settings. Here, we evaluated risk factors and clinical parameters associated with AKI and AKI severity, stratified by sex, in a cohort of children hospitalized with SCA and vaso-occlusive pain crisis (VOC). The purpose of this study was to explore sex disparities in a high-risk, vulnerable population. This study was a secondary analysis of data collected from a cohort of Ugandan children between 2 and 18 yr of age prospectively enrolled. A total of 185 children were enrolled in the primary study; 41.6% were female and 58.4% were male, with a median age of 8.9 yr. Incident or worsening AKI (P = 0.026) occurred more frequently in female compared with male children, despite no differences in AKI on admission. Female children also had altered markers of renal function including higher creatinine levels at admission (P = 0.03), higher peak creatinine (P = 0.006), and higher urine neutrophil gelatinase-associated lipocalin (NGAL) at admission (P = 0.003) compared with male children. Female children had elevated total (P = 0.045) and conjugated bilirubin at admission (P = 0.02) compared with male children and higher rates of hematuria at admission (P = 0.004). Here, we report sex differences in AKI in children with SCA and VOC, including increased incidence and worsening of AKI in female pediatric patients, in association with an increase in biological indicators of poor renal function including creatinine, estimated glomerular filtration rate, and NGAL.NEW & NOTEWORTHY In this study, we report an increased risk of developing acute kidney injury (AKI) during hospitalization, worsening AKI, and death among females with sickle cell anemia (SCA) hospitalized with an acute pain crisis compared with males. The sex differences in AKI were not explained by socioeconomic differences, severity of pain, or disease severity among females compared with males. Together, these data suggest that female children with SCA may be at increased risk of AKI.

Emergency department utilization before and during the COVID-19 pandemic among individuals with sickle cell disease

BackgroundThe emergency department (ED) is a vital source of healthcare for individuals living with sickle cell disease (SCD). Prior research indicates that during the COVID-19 pandemic some individuals with SCD avoided the ED for fear of acquiring COVID-19 or delayed visiting the ED by self-management of symptoms or pain crisis at home. The purpose of the current study was to understand ED utilization rates before and during the pandemic among individuals living with SCD.MethodsWe conducted a retrospective cohort study using population-based SCD surveillance systems in California, Georgia, Michigan, and Tennessee to assess the impact of the pandemic on ED utilization among people with SCD by (1) analyzing trends in monthly ED utilization from January 2019 - December 2020, with specific attention given to immediate changes at the onset of the pandemic; and (2) calculating changes in the volume of utilization by comparing the total ED visits made from March - December 2020 to the same period in 2019, both overall and by demographic characteristics.ResultsAcross all states, a decline in ED utilization during the onset of the pandemic was seen, with the largest decline seen in those under age 10. By December 2020, utilization rates were higher than their lowest observed month of April 2020, but had not fully returned to pre-COVID levels. During the pandemic, ED visits in each state decreased by as much as 25%, and the number of people with any ED utilization decreased by as much as 26%.ConclusionsThis study confirms and extends the existing literature related to the impact of the pandemic on healthcare utilization patterns in the US, in a unique population with increased healthcare needs.

Prevalence, Risk Factors, and Complications of Sickle Cell Disease in Saudi Arabia: A Systematic Review.

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.

Health Related Quality of Life and Psychosocial Profile of Patients with Sickle Cell Disease and Their Caregivers

Abstract Background Sickle cell disease (SCD) is a chronic, debilitating disorder can negatively affect health-related quality-of-life (HRQoL) outcomes in SCD. Aim of the Study Primary objective was to assess the prevalence of impaired psychosocial profile and poor HRQoL among SCD patients and their caregivers in comparison to age and sex matched controls, while secondary objective was to determine the association of such impairment if any with parameters of disease severity. Methods An observational, case control study recruited sixty-five children and adolescent with confirmed SCD, conducted at Paediatric Haematology Oncology clinic, Ain Shams University, Egyptfrom1st of August 2020 to 28th of February 2021. Demographic and clinical data were collected, and thorough clinical and psychiatric assessments were conducted. Quality-of-life (QoL) of both children and their caregivers were performed compared to 65 matched healthy control. Results sixty five children and adolescents with confirmed SCD were included; there were 34 (52.3%) boys and 31 (47.7%) girls and their mean age was 11.40 ± 3.55. Most patients 44(67.7%) had sickle HbSβ+-thalassemia, the most frequent manifestations were vasoooclusive crises in 24 (36.9%) patients, The most common causes for hospital admission were pain crisis in 14 (33.3%) patients and vasoocclusive crisis in 14 (33.3%). Results indicated that patients with SCD and their caregivers had depression and anxiety symptoms scores more than reported in control group.Sickle children had significantly less self-esteem and quality of life than control children, least scores in s ick le c hild re n Q O L wer e in co mmunica t io n do ma in. These were s ignific a nt ne ga t ive consequences, advanced age, duration of illness, number and duration of hospitalizations, disease severity score and occurrence of complications were associated with psychological disorders. Conclusion The QOoL of children suffered from SCD and their caregivers is adversely affected necessitate implementation of interventions which focus on reducing depressive symptoms, enhancing self-esteem and QoL.

P-361 Preserving fertility prior to hematopoietic stem cell transplant in post-pubertal women with sickle cell anaemia

Abstract Study question Can fertility preservation (FP) in women with sickle cell anemia (SCA) be linked to an increased risk of hematopoietic complications? Summary answer Only one participant on sixteen in our series had to discontinue the ovarian stimulation due to a complication related to a painful vaso-occlusive crisis (VOC). What is known already SCA is one of the most common severe monogenic diseases worldwide. It is a multisystemic disorder characterized by anaemia, increased haemolysis, and VOC. Despite advances in management, patients remain at a high risk of significant morbidity, especially increased thromboembolic events and early death. With the introduction of hematopoietic stem cell transplantation(HSCT) for non-malignant conditions, there is now a curative treatment for this debilitating condition. The myeloablative regimens for HSCT may impact ovarian reserve; therefore consideration of FP could be recommended. There is limited data on FP in this context and the risk of hematopoietic complications such as VOC is unclear. Study design, size, duration Cohort of women with SCA consulting at our center for FP before a HSCT project between 2016 and 2023. FP modalities were oocytes vitrification after ovarian stimulation and ovarian tissue cryopreservation (partial unilateral ovariectomy under laparoscopy). A protocol for the prevention of the risk of VOC, including hydration, oxygen therapy, and warming ± transfusion exchanges and for thromboembolic events was initiated before and during FP treatment, tailored on a case-by-case basis with the referring haematologist. Participants/materials, setting, methods The data related to hematopoietic complications (including VOC and thromboembolic event) [ps1] during FP were collected. Additionally, data on the follow-up of SCA, including received and planned treatments post-FP, were collected. This encompassed information on the completed FP treatments, preventive measures for SCA complications, and the outcomes of FP. Descriptive analysis was conducted. Main results and the role of chance During the study period, sixteen women affected with SCA aged 15–37 years have consulted for FP in our center. The mean age and BMI were 28.43±6.66 years and 21.97 ±3.46 respectively. The mean AMH level and antral follicle count were 1.77±1.70 and 15.83 ±7.12 respectively. Twelve patients underwent oocytes vitrification after mulfifollicular ovarian stimulation (OS), two had ovarian tissue cryopreservation, and 1 underwent both technics. One patient, initially planning for OS, cancelled after counselling. Among the 13 women that received an OS, 18 cycles were performed in total. The mean number of OS cycles per patient was 1.38±0.73. Protocols were antagonist (n = 16) or short agonist protocols (n = 2). The mean duration of gonadotropin treatment was 9.91±1.84 days, and the mean total gonadotropin dose was 3187.5±711.8 IU. Five OS were cancelled before oocyte retrieval because of an insufficient ovarian response. The mean number of oocytes retrieved was 8.83±5.61 per cycle. The mean number of cryopreserved oocytes was 12.00±5.66. One patient had to discontinue the OS due to a complication related to a painful VOC at 3 days of stimulation. As recommended, the hydroxycarbamide treatment was temporarily suspended to perform the FP. For patients who underwent unilateral partial ovariectomy, no complications were reported. Limitations, reasons for caution The main limitation is that this study is a small, retrospective case series; however, this represents the largest series to date. Wider implications of the findings Although limited, current data suggest that FP with specific conditioning does not pose a significant risk of complications in patients with SCA who are to undergo gonadotoxic treatment for HSCT. Trial registration number NA