Pediatric Sickle Cell Patients Research Articles

Neighborhood Disadvantage and Healthcare Utilization in Pediatric Sickle Cell Patients

Background: Sickle cell disease (SCD) is a complex heritable blood disorder associated with higher acute healthcare utilization further complicated by psychosocial and socioeconomic factors. Other studies have indicated a relationship between neighborhood disadvantage and increased healthcare utilization. We hypothesize that those with greater neighborhood disadvantage will have greater acute healthcare utilization.
 Methods: Data was collected retrospectively from the electronic medical record between 9/30/2021 and 7/11/2023 for patients followed at Riley Hospital for Children’s Pediatric SCD Clinic who completed a psychosocial needs assessment (PAT). Number of emergency department (ED) visits, hospitalizations, and missed clinic visits were collected from the time the PAT was completed up to 1 year after (or the end of the data collection period). Data regarding area deprivation index (ADI) and childhood opportunity index (COI) were collected utilizing patient home address. Descriptive statistics were performed on all data above, as well as t-tests and chi square tests for univariate analyses.
 Results: 142 patients completed a PAT during the study period. 88% had a primary care provider documented, and 73% had public insurance. The median state and national ADI (6, 76.5) and COI (2, 2) demonstrated greater neighborhood disadvantage and less childhood opportunity. There was no statistically significant difference between ADI or COI and number of ED encounters or hospitalizations. However, patients who missed 1 or more SCD visits had a higher median state ADI than those who did not miss a visit (7.5 vs 5, p=0.002). Median state COI was also lower in this group than those who did not miss a visit (1 vs 2, p=0.001).
 Conclusion: Those with higher ADI and lower COI could benefit from more directed support to improve access to preventative care visits. Further analysis accounting for comorbidities (seizures, asthma, depression) could disambiguate a relationship between ADI or COI and acute care utilization.

Improving Access to Comprehensive Care for Pediatric Sickle Cell Patients through the Creation of Multispecialty Clinics

There are many barriers to health care for those in underserved populations such as the pediatric sickle cell population. Due to limitations of transportation, clinical availability, and lack of care coordination, pediatric sickle cell patients are at an increased risk of not receiving the extensive multi-subspecialty health care recommended to prevent potential sickle cell related complications that affect every organ system. To improve access to this multidisciplinary care, UPMC Children's Hospital of Pittsburgh (CHP) pediatric sickle cell program developed a multi-subspecialty collaborative clinic model to offer coordinated sickle cell and pulmonary care, which is critical for sickle cell patients due to the increased risk of pulmonary complications associated with sickle cell disease. By considering health care barriers experienced by our pediatric sickle cell population, we proposed that this coordinated subspecialty clinic model will increase compliance by offering patients and families the opportunity to receive synchronized care from two essential subspecialty groups in just one medical center trip. This increased access to preventative health care for the pediatric sickle cell population should contribute to decreased acute medical events that require critical care and other extensive resources within the health care system. Methods: Two adjacent clinic appointments within the sickle cell and pulmonology clinics were designated for sickle cell patients also requiring preventative pulmonary care to be seen consecutively by both clinical teams during the same half day (one in sickle cell clinic and one in pulmonary clinic). The effect of improving completed referrals using this multi-specialty clinic model was assessed by retrospective electronic medical record review comparing the number of pulmonology referrals made by SCD team and pulmonary visits completed pre- and post- coordinated multi-subspecialty clinic initiative. The studied time period prior to the multispecialty clinic creation was chosen to be calendar year 2019 in order to eliminate the variable of the COVID-19 pandemic affecting outpatient clinic participation. Post-intervention data were collected during calendar year 2022 soon after the creation of this multi-subspecialty initiative. Prior to the creation of this multi-subspecialty clinic model, coordinating CHP sickle cell and pulmonology care, only 24% of pediatric sickle cell patients referred for outpatient pulmonology consultation completed the clinical evaluation. Our program referred 53 of 168 total families during calendar year 2019, with only 17 patients completing their visit. Since beginning this multi-subspecialty clinic initiative, pulmonology evaluations completed by referred sickle cell patients increased to 55%. Thus, CHP's multispecialty clinic offering coordinated sickle cell and pulmonology care increased completed pulmonology referrals by 129%. Using Pearson's chi-square test to compare the relationship between the two categorical variables of prior- and post- multispecialty clinic formation with a null hypothesis of multispecialty clinics will not increase completed referrals, we found a value of 10.76, which is greater than the applied critical value of 9.21 (p=0.01). Therefore, these results are significant, and the null hypothesis was rejected. In summary, by establishing this multi-subspecialty coordinated care at CHP for our pediatric sickle cell population, our goal of improving compliance with critical preventive care was achieved. We hope that this type of intervention will enhance the quality of life for children with sickle cell disease currently, as well as contribute to better health in the patients' futures. We also predict that this improved preventive care will also contribute to decreased emergency care and extensive treatments required for patients cared by CHP. This multi-subspecialty care model also has the potential to benefit other pediatric patients with chronic diseases from underserved populations who experience similar barriers, both in CHP's Western Pennsylvania region as well as underserved populations and health care organizations beyond our region. By applying an approach to care focused on reducing health care barriers to underserved populations, impactful improvements may be seen not only in the pediatric sickle cell population, but in the health care system at large.

Neighborhood Disadvantage Increases Risk of Adverse Clinical Events in Children with Sickle Cell Disease

Background: Hydroxyurea is the most widely used pharmacologic agent to treat patients with sickle cell disease (SCD). Hydroxyurea treatment has well established effects of inducing expression of fetal hemoglobin (HbF) and a beneficial impact on clinical outcomes such as reduced number of vaso-occlusive (VOC) events, transfusions, and hospitalizations. In non-SCD populations, living in socio-economic disadvantage is a well-established factor for poor health outcomes. However, there is a paucity of data to describe how social stressors impact children with SCD. We hypothesized that socio-economic disadvantage impacts risk of adverse clinical outcomes in pediatric SCD patients even while on hydroxyurea therapy. Methods: We performed a retrospective chart review on 121 pediatric patients with severe SCD genotypes (homozygous HbS and sickle-beta 0 thalassemia) seen at Texas Children's Hospital (TCH) between 2010-2021. All patients were treated with hydroxyurea for a minimum of five years. Adherence to hydroxyurea was assessed through lab markers including absolute neutrophil count and reticulocyte counts. Patients not adherent were excluded from analysis. We examined the incidence of all clinical events for a total of 1026 patient years, including the number of office visits, ER visits, hospitalizations, transfusions, VOC, acute chest syndrome episodes (ACS), acute splenic sequestration (ASS), and febrile illness. We collected laboratory data including absolute neutrophil count, hemoglobin level, and HbF levels for each patient per year they were on hydroxyurea. We used the area deprivation index (ADI) to determine a score of neighborhood disadvantage amongst our cohort of patients. The ADI is a factor-based index which uses 17 US Census variables including poverty, education, and employment. We used individual residential addresses during our observation period to assign a geocode specific to each individual and obtain an ADI score. The ADI scores are ranked from 1-10, with higher ADI rankings reflecting a higher level of disadvantage. Results: All individuals with SCD had a robust response to hydroxyurea within their first year of initiating the drug (>20% HbF). We stratified our cohort based on their ADI scores into low ADI (1-3; n=42); medium ADI (4-6; n=42); and high ADI groups (7-10; n=37), where high ADI is the group with highest levels of disadvantage. All three groups had a similar average duration of hydroxyurea treatment. There were no significant differences in age, sex, or reason given for starting hydroxyurea between the ADI groups. When we examined hydroxyurea response, there was no significant difference in HbF levels at 1 year (23.8 vs. 24.06 %HbF in the low vs. high ADI groups) or after 5 years (19.6 vs. 18.6 % HbF in the low and high ADI groups). Individuals with high ADI did started hydroxyurea at a younger age (2.9 years vs. 4.9 years, p=0.02). We found that any individual living in an area with higher ADI had an increased incidence of adverse clinical events. We calculated the incidence of each clinical event per 100 patient years in our 3 ADI groups and used a conditional maximum likelihood estimate of rate ratio method to compare incidences (Table 1). We found that among individuals living in an area with a higher ADI (more deprived region), there was increased frequency of ER visits, hospitalizations, transfusions, VOC, and ASS. In particular, patients with the highest ADI had a 3 and 5 times higher rate of VOC and ASS, respectively (Table 1). Discussion: While we did not observe an association between ADI and HbF expression, our results show that higher neighborhood disadvantage is associated with poorer clinical outcomes among hydroxyurea-adherent pediatric patients with SCD who maintained a robust response to the drug (>20% HbF). All individuals in our study had a similar number of routine office visits at our clinic but any patient living in an area of high ADI had significantly more ER visits and hospitalizations for VOC and ASS events. While hydroxyurea is shown to reduce hospitalizations and VOC events in pediatric sickle cell patients, we show that neighborhood disadvantage can influence clinical outcomes even among patients who respond to hydroxyurea. More analysis remains in order to better characterize this association, and to understand additional strategies that can be used to reduce clinical events in pediatric patients of lower socioeconomic status.

Establishing Mental Wellness Screening As Standard of Care in Pediatric Sickle Cell Patients

Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized with multi-organ involvement and frequent pain crises. Somatic complaints can give rise to neuropsychiatric disorders, among which anxiety and depression are the most common. Although mental health plays an important role in health outcomes, there is not a standardized approach to screen sickle cell patients for depression at most sickle cell centers across the country and at Texas Children's Hospital (TCH). The long-term goal of this project is to screen sickle cell patients over the age of 12 for depression across TCH hospitals: TCH-West Campus (TCH-WC), TCH-Woodlands (TCH-WL) and TCH-Medical Center (TCH-MC). Over the past two years, we successfully implemented depression screening at TCH-WC as standard of care through a series of PDSA cycles. The focus of this quality improvement project is to implement depression screening at TCH-WL. At TCH-WC, all sickle cell patients aged 12 years of greater are screened for depression using the Patient Health Questionnaire-9 modified for Adolescents (PHQ-A) . From October 2022 to July 2023, 71% of the patients that qualified for screening identified low risk, 16% identified mild risk and 12% identified moderate to severe risk of depression. Eleven percent endorsed suicidality. Psychology/Social Work (SW) saw 77.4% of patients with moderate to severe depression and 100% who screened positive for suicidality on the same day as screening. Now that our depression screening protocol is standard of care at TCH-WC, we extended this screening to TCH-WL in February 2023. Our goal is to increase the annual depression screening rate from 0 to 50% in English/Spanish speaking patients aged 12 years and older with sickle cell disease who present for follow-up care to the TCH-WL. During our first PDSA cycle at TCH-WL, one hematologist administered the PHQ-A to sickle cell patients > 12 years. No intervention was needed if patient scored 0-6 on the PHQ-A. Psychological resources were provided via handout if patient scored 7-9. SW referral was placed and patient was preferably seen same day if patient scored > 10 on the PHQ-A. If unable to be seen same day, SW followed-up via phone call. If patient screened positive for suicidality, an urgent SW referral was made and patient was seen in clinic the same day. Three sickle cell patients >12 years presented for follow up with the assigned provider. Two-thirds (67%) of patients were given the PHQ-A; half (50%) screened positive. During the second PDSA cycle at TCH-WL, all patients with SCD over the age of 12 who presented to TCH-WL Hematology Clinic were screened. The patients were given the PHQ-A by the Medical Assistant/nurse prior to the start of the appointment and then they followed the outlined and detailed algorithm listed above based on score.Forty-five patients were eligible for screening. Forty-two percent (19/45) were given the PHQ-A; 73% endorsed mild risk of depression; 26% endorsed moderate to severe risk; 22% endorsed suicidal ideation. 80% of patients with moderate to severe risk received same day SW assessment; 100% of patients with suicidal ideation received same day SW assessment. Over the course of our project, we have been able to maintain our goals at TCH-WC and came close to achieving our goal of screening 50% of eligible patients for depression at TCH-WL. We are working with staff and providers at TCH-WL to determine barriers and optimize clinic flow in order to achieve >50% screening. In the future, we plan to expand our project to our largest sickle cell clinic, TCH-Medical Center, and for depression screening to become standard of care for all sickle cell patients at Texas Children's Hospital.

Knowledge and attitude towards sickle cell anemia among care givers of paediatric sickle cell patients at a tertiary hospital in Eastern Uganda: a cross sectional study

ObjectiveTo explore the knowledge and attitude towards sickle cell disease (SCD) among care givers of paediatric sickle cell patients at Mbale regional referral hospital in Eastern Uganda.MethodsA cross sectional study was conducted at Mbale regional referral hospital. We used simple random sampling technique to recruit participants from among the care givers of pediatric sickle cell patients admitted at the hospital, administered questionnaires and conducted multivariable logistic regression to establish the association between the different factors.Results372 respondents participated in the study, 82.26% of which were female. 57.80% of the respondents had ever heard of SCD/SCA. 36.02% were willing to stay in a relationship with their partner despite the risk of having a child with SCD/SCA. A multivariate analysis revealed that variables; “number of children”, “children with sickle cell can cope with life” and “willing to stay in a relationship despite the risk of a having a child with sickle cell” were statistically significant.ConclusionThere was a high level of general awareness about SCD/SCA but comprehensive knowledge about its cause and prevention was low and the majority did not find a reason as to why it should influence their marital decisions. Inclusion of SCD/SCA in existing health education programs is highly recommended.

To transfuse or not to transfuse? A pilot study evaluating preoperative transfusion practices for pediatric sickle cell patients undergoing central venous access procedures

IntroductionPerioperative transfusion guidelines for pediatric sickle cell disease (SCD) patients are underdeveloped. The American Society of Hematology (ASH) provides transfusion recommendations for procedures requiring general anesthesia and lasting >1 hour. However, many pediatric SCD patients require long term central venous access and these short procedures do not fall within these guidelines. The objective of this study is to investigate our institutional preoperative transfusion practices in central venous access procedures. MethodsA retrospective chart review was performed from 2018 to 2022 of SCD patients who underwent a line placement or removal at a stand-alone quaternary care children's hospital. Clinical information including demographics, Hgb levels, number of transfusions, and postoperative complications were collected. Statistical analysis was performed using Fisher exact and Wilcoxon rank sum tests. ResultsA total of 30 patients underwent 34 venous access procedures (76 % line placement, 36 % line removal). Half of the patients (15) had an initial Hgb <9 g/dL. There were no significant differences in the demographics, complications, or readmission rates between patients with an initial Hgb <9 or >9 g/dL. Significantly more patients received a preoperative blood transfusion in patients with a Hgb <9 g/dL compared to those with a Hgb >9 g/dL (73% vs 20 %, p = 0.01) Length of stay, postoperative complications, intraoperative transfusion, and readmission rates were not significantly different in patients who received a preoperative transfusion. ConclusionWe encountered variability in the preoperative transfusion threshold; however, outcomes remained similar with no significant differences based on preoperative Hgb. Future studies focused on quality, safety, and resource allocation are needed for development of practice recommendations.

Vaccination Rates in Pediatric Sickle Cell Patients in Louisiana.

Vaccination Rates in Pediatric Sickle Cell Patients in Louisiana.

5613464 INTEGRATION OF PHARMACOKINETIC AND PHARMACODYNAMIC MODELS TO MULTI-CRITERIA DECISION ANALYSIS (MCDA) FOR PROSPECTIVE ASSESSMENT OF THE RISK-BENEFIT RATIO OF ORAL IRON CHELATORS IN PAEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT HAEMOGLOBINOPATHIES

Background: Current therapies for the treatment of thalassaemia and other hereditary rare haemoglobinopathies are symptomatic and aim at controlling or reducing iron overload by chelation of iron from a very early age. Iron chelation ultimately prevents complications or comorbidities that can significantly reduce patient survival. Given the processes underlying iron homeostasis and current understanding of the chelation mechanism of available chelating agents, it may be possible to implement alternative, more efficient approaches for evidence generation in support of the dose rationale and their use across different populations affected by transfusion-dependent haemoglobinopathies. Aim: This study aimed to characterise the benefit-risk balance (BRB) of deferiprone in different populations, including children < 2 years old and paediatric sickle cell patients, using a nonlinear mixed effects modelling approach in conjunction extrapolation concepts and multicriteria decision analysis (MCDA). Methods: Based on the underlying pharmacokinetic-pharmacodynamic relationships as well as on the incidence of adverse events, extrapolation concepts were applied to predict the BRB of treatment in young adults, paediatric patients younger than 2 years, and paediatric sickle cell disease patients receiving deferiprone. Using individual patient-level data from a randomised controlled clinical trial (DEEP-2, EudraCT 2012-000353-31), preference elicitation methods were applied to select and prioritise endpoints or outcomes to be used in the evaluation of the BRB across the populations of interest. Subsequently, model parameter estimates obtained from each model/endpoint were used in a set of clinical simulation scenarios in which the effect of chelation therapy was evaluated during 12 months in a virtual cohort of patients. Results: Five endpoints were selected and ranked by the experts involved in the preference elicitation process (Fig.1). ‘Absence or prevention of complications (diabetes/hypothiroidism)’ and ‘occurrence of agranulocytosis’ were the most important criteria, while ‘incidence of arthralgia’ and ‘occurrence of neutropenia’ were considered less important. Clinical trial simulations revealed that the overall benefit score of deferiprone was higher for the treatment of paediatric SCD patient compared with the reference population (i.e. DEEP-2 study). Small but statistically significant differences were observed for the use of DFP in children younger than 2 years old and adolescents or young adults. Table 1 summarises the clinical and demographic baseline characteristics of the DEEP-2 study and virtual cohort of patients included in the different simulation scenarios. Conclusion: This study has shown that MCDA can be utilised in combination with modelling and simulation principles to prospectively assess the effect of interventions in subgroups of patients for which clinical data generation is challenging or not feasible. It also offers an opportunity to evaluate the implications of dose adjustment as well as changes in the clinical management of these patients, providing a less empirical basis for dose selection and treatment choices in rare paediatric conditions such as transfusion-dependent haemoglobinopathies. Table 1 - Demographics of the populations in the MCDA analysis. Reference population reflects the actual patients in the DEEP-2 study, whilst scenarios reflect the baseline characteristics from the virtual patient population. Population Age (years) Weight (kg) Blood consumption (mL/kg/year) Baseline ferritin (ng/mL) Reference population (DEEP-2 study) 8.9 (2.7-17.2) 26.5 (12.1-53.5) 155.4 (17.6-404.3) 1991 (586.2-6488.7) Scenario 1: Adolescents and young adults 17.6 (11.3-33.8) 58 (29-83.6) 139.7 (28.5-347.6) 1933 (432-10106) Scenario 2: Children < 2 years old 1.3 (0.6-1.9) 6.3 (1.1-9.3) 162.2 (21-382.9) 726.3 (253.3-1757) Scenario 3: Paediatric sickle cell patients 14.5 (12.4-17.8) 39 (30-62.4) 139.5 (23.8-338.2) 1598.4 (476.3-8993.9)

The Prevalence of Cardiovascular Manifestations in Pediatric Sickle Cell Anemia Patients in a Large Tertiary Care Hospital in the Western Region of Saudi Arabia.

Background Sickle cell disease (SCD) is a common hematological disorder with a high prevalence in Saudi Arabia. Despite that, limited studies are available in our region regarding cardiovascular complications. Objectives The objective of the current study was to estimate the prevalence of cardiovascular complications among children with SCD. Design This was a cross-sectional study. Setting The study took place at a single tertiary-care center in Jeddah, Saudi Arabia. Materials and methods The study reviewed 126 electronic records of pediatric patients up to 16 years old diagnosed with SCD between January 2008 and December 2019 in King Abdulaziz Medical City (KAMC) in Jeddah, Saudi Arabia. Of these, 54 patients had a previous echocardiogram evaluation and were eligible for the study. Main outcomes measures The study identified cardiovascular complications in pediatric sickle cell patients. Sample size The study included a total of 54 pediatric sickle cell patients. Results The mean age was 11.9 (3.48) years, the male-to-female ratio was 2:1, the majority (94.4%) had the HbSS-HbSβ0 genotype, the mean baseline hemoglobin F (fetal hemoglobin) was 20.30 (9.03%), and the clinical severity score was severe in 19 (35.2%) and mild/moderate in 35 (64.8%) patients. Cardiovascular complications occurred in 32 (59.3%) patients. Increased systolic blood pressure was detected in 10 (18.5%) patients. Echocardiography showed left ventricular dilatation in nine (16.7%) patients, tricuspid valve insufficiency in six (11.1%) patients, mitral valve insufficiency in four (7.4%) patients, hyperdynamic left ventricle in one (1.9%) patient, and pulmonary hypertension in one (1.9%) patient. Long QTc interval was noticed in three (5.6%) and cardiomegaly was detected in 18 (33.3%) patients. Conclusion Cardiovascular complications occurred at a high frequency in our pediatric population despite high baseline hemoglobin F levels. Early evaluation and continuous monitoring are important for early intervention.

Outcomes of Pregnancy in Sickle Cell Disease Patients: Results from the Prospective ESCORT-HU Cohort Study.

Sickle cell disease (SCD) refers to a group of inherited hemoglobin disorders in which sickle red blood cells display altered deformability, leading to a significant burden of acute and chronic complications, such as vaso-occlusive pain crises (VOCs). Hydroxyurea is a major therapeutic agent in adult and pediatric sickle cell patients. This treatment is an alternative to transfusion in some complications. Indeed, it increases hemoglobin F and has an action on the endothelial adhesion of red blood cells, leukocytes, and platelets. Although the safety profile of hydroxyurea (HU) in patients with sickle cell disease has been well established, the existing literature on HU exposure during pregnancy is limited and incomplete. Pregnancy in women with SCD has been identified as a high risk for the mother and fetus due to the increased incidence of maternal and non-fetal complications in various studies and reports. For women on hydroxyurea at the time of pregnancy, transfusion therapy should probably be initiated after pregnancy. In addition, there is still a significant lack of knowledge about the incidence of pregnancy, fetal and maternal outcomes, and management of pregnant women with SCD, making it difficult to advise women or clinicians on outcomes and best practices. Therefore, the objective of this study was to describe pregnancy outcomes (n = 128) reported in the noninterventional European Sickle Cell Disease COhoRT-HydroxyUrea (ES-CORT-HU) study. We believe that our results are important and relevant enough to be shared with the scientific community.

Predictive Utility of Visual Evoked Potentials in Detection of Ocular Changes in Paediatric Sickle Cell Patients: A Cross-sectional Analytical Study

Introduction: Ocular manifestations are one of the complications of Sickle Cell Disease (SCD) that may occur in various segments of the eye. Optic nerve involvement is under-diagnosed though it can be involved as a sequela to ischemia. Prediction of disease at an early age aids in better diagnosis. Visual Evoked Potentials (VEP) helps to detect abnormalities (silent lesions) in patients with visual complaints who do not present with visible pathological ophthalmological changes. In literature search, no study has been undertaken to assess the predictive utility of VEP regarding subclinical ocular changes in paediatric age group of SCD patients. Aim: To evaluate predictive utility of VEP to identify subclinical ocular changes in paediatric patients of SCD and to record associated Visual Reaction Time (VRT). Materials and Methods: In this cross-sectional analytical study, 30 cases (SCD patients) and 30 normal children in age group 3-15 years were evaluated by ophthalmic examination followed by VRT and VEP using Light-emitting diode (LED) goggles (Flash). Statistical analysis included descriptive (percentages) and inferential statistics presented as unpaired t-test, linear regression curve, Pearson’s correlation coefficient, coefficient of determination (R2 ) and β (regression) coefficient. The analysis was done at 99% confidence interval with significance at p&lt;0.01. Results: There was statistically significant prolongation of P100 latency in both eyes in paediatric cases when compared to normal children (P&lt;0.01). N75-P100 amplitude, interocular difference showed no significant changes. When P100 latency was correlated with VRT, there was weak positive correlation (r=0.207, p=0.1278 for right eye, r=0.238, p=0.0801 for left eye). Though sensitivity of flash LED goggle VEP was 70%, specificity was high (96.66%). Positive predictive value was 95.45%. Conclusion: These findings show that VEP can be used as a predictive measure (tool) to detect subclinical changes in absence of ocular complaints and normal ophthalmological findings.

Breaking Down Barriers and Addressing Mental Wellness in Pediatric Sickle Cell Patients

Sickle cell disease is an inherited hemoglobinopathy that is characterized with multi-organ involvement and frequent pain crises. Over the last two decades, a growing body of clinical evidence has pointed out that these somatic complaints can give rise to neuropsychiatric disorders, among which anxiety and depression are the most common, that worsen the health-related quality of life of these patients. Mood disorders can in turn increase fatigue and pain through a psychosomatic component. Although mental health is deemed to play an important role in their health outcomes, at this time, we don't have standardized approach to screening sickle cell patients for depression at most sickle cell centers across the country and at Texas Children's Hospital(TCH). The objective of this Quality Improvement (QI) study is to increase the annual depression screening rate from 0 to 50% in English/Spanish speaking patients who are 12 years and older with an established diagnosis of Sickle Cell Disease (SCD) who present for follow-up care to the Texas Children's Hospital - West Campus (TCH-WC). Our QI project consisted of three distinct PDSA cycles. Prior to the start of the first PDSA cycle, we did not have a streamlined approach for depression screening. The physician team would verbally screen for depression and Social Work was consulted as needed. During our first PDSA cycle, a clinical psychologist was integrated into our monthly Hematology-Pulmonary Joint Clinic and all patients with SCD who presented to Joint Clinic were assessed for depression by the psychologist as part of the psychological evaluation. During the second PDSA cycle, all patients with SCD over the age of 12 who presented to Joint Clinic (with psychologist present) were given the Patient Health Questionnaire-9 modified for Adolescents (PHQ-A) by the Medical Assistant prior to the start of the medical appointment. After the physician team completed their visit, the psychologist addressed concerns raised on the PHQ-A, completed the psychological evaluation and provided appropriate intervention. During the third and final PDSA cycle, the PHQ-A was given to all patients with SCD above the age of 12 who presented to hematology clinic (including those outside of Joint Clinic during which the psychologist was not present). During this cycle, the MA gave the patient the PHQ-A at the start of the visit. The MA then followed an outlined and detailed algorithm. If patient scored 0-6 on the PHQ-A, no intervention was performed. If patient scored 7-9 on the PHQ-A, psychological resources were provided via handout. If patient scored > 10 on the PHQ-A, Social Work referral was placed and patient was preferably seen same day. If patient screened positive for suicide, an urgent Social Work referral was made and patient was seen same day prior to leaving clinic for further evaluation. During our first PDSA cycle, 19 patients above the age of 12 presented to Joint Clinic. Seventeen patients were seen by the psychologist. Twenty-four percent (4/17) of patients were given the PHQ-A; 50% of patients screened positive. Twenty-four percent (4/17) of patients were referred for outpatient follow-up with psychology. During our second PDSA cycle, 14 patients above the age of 12 presented to Joint Clinic. Ninety-three percent (13/14) of patients were given the PHQ-A; 7% (1/14) of patients screened positive for depression. Twenty-one percent (3/14) of patients were referred for outpatient follow-up with psychology. During our third PDSA cycle, 54 patients above the age of 12 presented to Hematology Clinic. Eighty percent (43/54) of eligible patients were given the PHQ-A. Fourteen percent of patients endorsed mild symptoms of depression; 23% of patients endorsed moderate to severe symptoms of depression; 23% of patients endorsed suicidal ideation. Eighty percent of patients with moderate to severe depression received same day Social Work Assessment. One hundred percent of patients with suicidal ideation received same day Social Work assessment. During the first three PDSA's cycle of our project, we achieved our goal of screening greater than 50% of eligible patients with SCD and providing same day intervention. In the future, we will need to examine if there are any downstream effects that negatively impact the clinic flow while administering the questionnaire. We also hope to expand screening for all patients with SCD over the age of 12 across Texas Children's Hospital campuses.

Impact of Social Determinants of Health on Disease-Related Outcomes in Pediatric Sickle Cell Patients

Background: Social Determinants of Health (SDoH) are socioeconomic factors that influence health and well-being and are largely responsible for health inequities. Research has shown that economic, housing, food and medical insecurities are associated with lower quality of life, poor mental health, and worse outcomes for people living with chronic illness. Individuals with Sickle Cell Disease (SCD) are at increased risk of mortality, poor health-related quality of life, functional disability and high health care utilization. Furthermore, SCD primarily affects populations of African descent, who also tend to be disproportionately burdened by poverty and systemic racism. Prior studies have shown that children with SCD from lower income families have worse health-related quality of life, and higher healthcare utilization rates. However, there is paucity of data investigating links between SDoHs with quality of life and disease-related outcomes in this population. Thus, it is important to gain a better understanding of how these factors affect the well-being of children living with SCD. In this study, we attempt to identify the unmet social needs of patients in one pediatric sickle cell clinic and their association with self-reported health care utilization and disease complications. Methods: We approached pediatric patients (aged 1-18 years) and their guardians attending routine visits at the Children's Hospital of Richmond Comprehensive Sickle Cell Clinic. Those coming for a newborn visit or having acute crises were excluded. After consenting, parents completed a demographic and patient-reported outcome questionnaire followed by the Boston Medical Center's WECARE survey that screens for SDoH, such as housing, food, utility, transportation and medical insecurities. We defined an unmet social need as any positive answer to the WECARE survey. Counts, percentages and exact tests were used to examine the relationship between unmet needs and demographic factors and disease outcome variables. Results: To date, 50 children with SCD aged 1-17 years and their parents were screened, consented, assented (children >7) and completed the surveys. There was incomplete data on social needs from 5 subjects, thus 45 total are included here. 53% of subjects indicated an unmet need in at least one of the categories in the WECARE survey: 41% had food insecurity, 4% had housing insecurity, 13% had problems paying for medication, 11% had problems with transportation, 24% had trouble paying utility bills, 9% had difficulty taking care of family members and 13% were unemployed. Children with at least one unmet social need were more likely (p<0.05) to have a lower household income (Table 1), report one or more hospitalizations in the past six months and report missing a scheduled activity or school in the previous month compared to children without an unmet need (Table 2). Those with an unmet need also reported higher Emergency Department visits, acute SCD complications, and lower grades than those without an unmet need, but these findings were not statistically significant. Conclusion: This preliminary data demonstrates that SDoH, represented by unmet social needs, have an association with increased healthcare utilization and disease-related complications for children with SCD. A larger sample size is needed to characterize these relationships more fully. Ongoing research includes investigating the relationships between SDoH with Quality of Life and disease indicators gathered from medical charts of children with SCD. By identifying which unmet socioeconomic needs exist in our clinics, we can provide more equitable and holistic care to children with SCD and ultimately improve their health outcomes. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Comparative effectiveness of adding Omega-3 or Vitamin D to standard therapy in preventing and treating episodes of painful crisis in pediatric sickle cell patients.

Sickle Cell Anemia (SCA), also called the Sickle Cell Disease (SCD), is an inherited hematological disorder characterized by a syndrome of acute anemia and a painful crisis. The sickling hemoglobin, Hgb-S causes viscosity and inflammation of blood vessels. Eventually, the red blood cells get eliminated from the circulation process, which leads to hemolytic anemia. This study examined the comparative effectiveness of supplementation of Omega-3 or vitamin-D to standard therapy (hydroxyurea + Ibuprofen) used for prevention and treatment of pain crises in pediatric patients living with SCD. 165 patients participated in this randomized, double-blind, standard therapy-controlled, parallel-design trial. The patients were randomly divided into three groups, receiving three capsules of either 1,000 mg Omega-3 fish oil (400 mg EPA and 300 mg DHA) or 1.5 mL vitamin-D (2,800 IU/7 ml) daily for 10 months plus the standard therapy. Lactate dehydrogenase, high-density lipoprotein (HDL), low-density lipoprotein (LDL), hematocrit, reticulocyte count, and white-blood-cell count were determined at baseline (month zero) and end of the 10th month. The pain severity was measured using the visual analog scale method (VAS). Therefore, a 10-cm ruler with a VAS design was used to determine the patient pain intensity. The baseline time point was defined as the time spot before starting to deliver the experimental medication to the patients (month zero). At that time, the biodata of the patient on the frequency of pain episodes and the rest of the variables were collected, and the baseline data were one-year retrospective data. Of 165 patients enrolled in the trial, 150 were included in the final analysis. At the end of the study, there was a significant increase in serum LDL and HDL in the Omega-3 group as compared with the control group (mean of 82 mg/dL vs. 57 mg/dL; p < 0.01 and mean of 47 mg/dL vs. 43 mg/dL; p < 0.028, respectively). Other laboratory parameters were significantly influenced. The number of painful crises and pain levels was significantly decreased in the Omega-3 group compared with the control group (mean of one-episode vs. mean of three episodes; p = 0.01, mean of three on pain scale vs. six on pain scale; p = 0.018). Results showed that Omega-3 was more effective than vitamin-D or standard treatment alone relative to pain crises and most of the other studied items. Vitamin-D was more effective than standard therapy alone. Clinicians should consider the addition of Omega-3 supplements to the standard therapy and a de-escalation dose plan for the hydroxyurea medication.

Long-term Use of Eculizumab for Prolonged Hemolysis Following a Delayed Hemolytic Transfusion Reaction in Pediatric Sickle Cell: A Case Report.

Complement activation has been implicated in delayed hemolytic transfusion reaction (DHTR) in patients with sickle cell disease (SCD), and eculizumab has been reported as an effective treatment for patients with DHTR. Previously reported patients with SCD and DHTR responded well after a few doses of eculizumab. We report on the long-term use of eculizumab in a pediatric sickle cell patient with prolonged hemolysis of unknown etiology after a DHTR who had a slow and less sustained response. Long-term use of eculizumab in this young patient with SCD was successful with limited adverse effects.

P110: CLINICAL CHARACTERISTICS OF COVID-19 IN SICKLE CELL DISEASE (SCD) PATIENTS

Introduction SCA is a common genetic disorder in Saudi Arabia with estimate of 1.4 % of population(1,2). The prevalence of SCD in Jazan region is around 24 per 10,000 which consider the second region with high prevalence of SCD after eastern province. COVID 19 infection outbreak has affected SCD with different outcome. Objectives: This research aims to determine the clinical picture of COVID-19 in patients with sickle cell disease SCD. The study represented the risk factors for severe COVID-19 presentation and predictors for poor prognosis in this group of patients. Methods: This study is a retrospective, descriptive, observational study of SCD adult and pediatric patients in Prince Mohammed Bin Nasser Hospital, Jazan, Saudi Arabia, who were diagnosed with COVID-19 virus infection at the study center from March 2020 to September 2021. To describe clinical presentation of SCD patients of different severities, who got COVID-19 during the study, socio-demographic data, clinical presentations, laboratory parameters, medications use, as well as COVID-19 symptoms and severity were extracted and analyzed from the medical record files. Results: 43 medical records for adult and pediatric sickle cell patients with COVID-19 were collected, in order to determine the impact of COVID-19 on the clinical presentation of SCD patients. (53%) were females, (47%) were males with a mean age of 24 years (±1.9). (37%) of the sample suffered from major comorbidities, out of which 44% had ACS. and 11.6% have pulmonary embolism. The most prevalent clinical symptoms were fever (56%) and Shortness of Breath (37%). (16%) of included patients were admitted to the ICU with an average length of stay equals to 3.9 days (±0.6). CBC showed normal averages of PLT with a mean equal to 327 K/uL (±21.6), Basophils 0.05 K/uL (±0.01), Lymphocytes 3.6 K/uL (±0.4). High averages were found for WBC 13.8 K/uL (±1.1), Neutrophils 8.8 K/uL (± 0.9) and PT 14.2 seconds (±0.3). The only laboratory parameter that showed low average reading was Hemoglobin, with a mean equal to 8 g/dl (±0.2). Out of the 41 patients who undergone CRP test, 85% were positive. The main CT chest finding were ground glass appearance in 30% of the patients 88% of the studied patients were on Hydroxyurea, 76% were on 1000 mg dose. For COVID-19 management, majority of patients were on Antibiotics 93%, 70% of patients started Anticoagulants, 51% of the patient has received antiviral treatment. 32.5% of the patient were treated with steroids. 70% of the patients have received blood transfusion. 5 patients 12% were managed at home. One patient had coinfection with falciparum malaria. Total deaths was 2 patients represented 4.6 % out of total SCD patients with COVID-19 who were included in the study. Conclusion: After exploring the impact of COVID-19 on SCD patients, the main presenting symptom were fever and shortness of breath. The major complication were acute chest syndrome 44% and pulmonary embolism in 12%. The overall prognosis was excellent and most of the patient have recovered. The death was reported in 2 patients only. Outpatient treatments were feasible in 12% of the patients.

S128: CONTINUOUS QUALITY IMPROVEMENT IN PAEDIATRIC SICKLE CELL DISEASE

Background: Sickle cell disease (SCD) is characterised by severe episodic pain requiring prompt and effective analgesia. However, SCD patients frequently experience poor quality of care due to lack of awareness of the condition among non- specialist staff, pre-conceived biases and unfounded allegation of drug-seeking behaviour. Several reports and surveys using Patient Reported Experience Measure (PREM) tools indicate widespread prevalence of ineffective pain relief in emergency department(ED), poor access to psychological therapies and poor funding for service development. Aims: To use a validated PREM tool to survey patients or carers of sickle cell disease within the paediatric service of a Specialist Haemoglobinopathy Team. To use an established Continuous Quality Improvement (CQI) methodology (5-D’s-define, describe, design, deliver, digest) to ensure sustained improvement in patient- reported areas of service deficit. Methods: The PREM survey was conducted as part of a network- wide initiative in 2018. The survey responses were analysed and problem scores created for specialist care, emergency care, ward-based care, information and support. These problem areas were categorised into domains where further improvement action was needed. CQI tools were used to map the patient journey within Emergency Department (ED) when presenting with pain followed by thematic analysis to identify potential improvements in the patient journey. Results: The PREM survey analysis identified three domains for improvement: Domain A: To improve the effectiveness of pain relief and quality of empathic and informed care to paediatric sickle cell patients and their families in ED. We developed multi-disciplinary teaching videos with patient involvement and patient information material based on ED/pain/sickle staff feedback. Excellent engagement from all staff groups obtained. Patient involvement in ED teaching video was well received. Thematic analysis of ED process mapping highlighted need for sustained staff education and re-writing ED pain protocol to align with current practice. Pre-conceived biases were identified among staff with respect to attributing drug-seeking behaviour to patients during severe sickle cell pain. Domain B: To improve the information presented to children regarding SCD and the care services available in our trust We developed child friendly Information leaflets targeting primary school age and secondary school age readers Domain C: To improve the availability of, and access to, mental health services for paediatric SCD patients. We developed virtual parent support groups and psychological health screening for adolescent patients in clinics. We organised a ‘Tree of Life’ (ToL) patient empowerment workshop. The parent support groups were well attended. Plans are in place to continue ToL workshops and support groups on a regular basis. Future work: A rapid improvement workshop is being planned to engage a wider multi-disciplinary team to engender a culture of empathy in line with our trust values. Further input from workshop is expected to identify and implement sustainable goals. We plan to undertake a repeat PREM survey following the end of this CQI project to understand the impact and the implemented improvements.

A Patient-Centered Approach to Gathering Information on Social Determinants of Health from Patients with Sickle Cell Disease in Preparation for Their Transition to Adult Care

Background:Transition from pediatric to adult care has been a recent focus of sickle cell programs across the country due to patients with sickle cell disease (SCD) to living longer. 1 Transition programs for other chronic conditions focus largely on patient knowledge of the disease, treatments, and appointment compliance but for patients with SCD, social determinants of health (SDOH) must also be considered when guiding patients toward adult care. 2 Historically, lack of acknowledgement of the importance of relationships and trust building when addressing SDOH has led to barriers in patient responsiveness. 3 To facilitate this aspect of transition, our recently hired Community Health Worker (CHW) developed a Community Health Transitional Questionnaire (CHTQ) and conducted a Quality Improvement (QI) project to address SDOH in a patient-centered manner during transition to adult care visits at the clinic.Aims: •Refine the CHTQ by gathering the “voice of the customer” to provide a more patient-centered approach to transition to adult care for patients with SCD.•Complete the CHTQ with 70% of transition-aged (12-25 years) patients by 7/1/21.Methods:The CHTQ consists of 32 questions about SDOH in 8 categories (Food, Housing, Money/Finances, Transportation, Education/Work, Family and Other Support, Stress Prior to/After COVID, and Sleep). Thirty pediatric sickle cell patients were identified as being within the established transitional age group. A series of Plan-Do-Study-Act (PDSA) cycles were used to refine the CHTQ. Through patient/caregiver feedback, small increments of change were used to establish a CHTQ that would improve patient care and be accepted by the patients/caregivers.PDSA #1: During regularly scheduled appointments, two patients and their caregivers met with the CHW who described the purpose of the CHTQ and then asked the patient to privately complete the CHTQ.PDSA #2: Because of feedback received from PDSA #1, the CHW allowed the next two patients to have caregivers present while answering the CHTQ, but explained that ideally the patient should be able to answer the CHTQ without input from their caregiver before transition to an adult environment.PDSA #3: Because of the feedback received from PDSA #2, the CHW accompanied the clinic RN to the exam room and worked the CHTQ into the nursing check-in questions in an informal manner making the questions seem more routine.Results:After 3 PDSA cycles, 17 additional patients/caregivers completed the CHTQ without issues or concerns. A total of 22/30 (73%) CHTQs met the goal of completion by 7/1/21. One additional CHTQ has been completed since.Using the information from the CHTQ, the team has been able to better address individualized SDOH for each patient. For example, one area of concern identified was transportation with 9/23 (39%) patients expressing concerns getting to & from appointments and/or school/work. Transportation needs were then discussed with the clinic social worker who is now working with those patients, giving them tools to arrange transportation.ConclusionFor SCD patients, it appears imperative that a relationship be established prior to inquires about SDOH. Using an informal patient-centered approach with the CHTQ was more likely to gain patient trust and allowed the CHW to obtain information needed to assist the patient with SDOH issues. Once SDOH issues were identified through the CHTQ, the CHW was able to find patient-driven, individualized resources addressing those concerns.

Determination of Red Cell Indices and Sero-prevalence of Transfusion Transmissible Infectious Diseases among Stable Paediatric Sickle Cell Patients

Red blood cell indices values were lower compared with control subjects, nature has it that SCD patients in the steady state have adapted their body system to this anaemic state and live healthy. Blood transfusion does not significantly increase the seroprevalence of transfusion transmissible infections in our clinically stable SCD participants.

A Standardized MRA Scanning Protocol Leading To Changes In Stroke Prevention Treatment Plan for Pediatric Sickle Cell Patients (4254)

A Standardized MRA Scanning Protocol Leading To Changes In Stroke Prevention Treatment Plan for Pediatric Sickle Cell Patients (4254)