Sickle Cell Center Research Articles

Tuberculosis in sickle cell disease patients

ObjectiveTuberculosis (TB) disease has rarely been reported in patients with sickle cell disease, but it is associated with an increased risk of bacterial infections. In France, sickle cell disease is frequent in populations with the highest prevalence of TB disease. We aimed to highlight clinical aspects of TB disease in patients with sickle cell disease. Patients and methodsOver a 10-year period, we retrospectively included all adults with sickle cell disease who had a positive culture for Mycobacterium tuberculosis managed in the adult sickle cell center of Henri-Mondor hospital. Sickle cell patients with TB disease were matched for comparison to adults without hemoglobinopathy and with documented TB disease in a 1:2 ratio. Logistic regression mixed models were performed. ResultsTwelve patients with sickle cell disease and documented TB disease (median age: 29years; IQR [25–34]) were compared to 24 non-sickle cell patients (median age: 33years; IQR [27.5–38.5]). Baseline characteristics were similar between groups except for sickle cell disease. Ten of the 12 patients with sickle cell disease had pulmonary TB. TB disease characteristics were similar between sickle cell and non-sickle cell patients although sickle cell patients had fewer positive sputum smears for acid-fast bacilli (P=0.003) and fewer lung cavitations (P=0.03). ConclusionsTB disease in sickle cell patients was globally similar to non-sickle cell patients, even though less infectious. Regular follow-up in specialized centers might allow for earlier TB disease diagnosis in sickle cell patients.

Nonmydriatic Ultra-Widefield Fundus Photography in a Hematology Clinic Shows Utility for Screening of Sickle Cell Retinopathy

To determine the feasibility and accuracy of nonmydriatic ultra-widefield (UWF) fundus photographs taken in a hematology clinic setting for screening of sickle cell retinopathy (SCR) DESIGN: Prospective cohort study. This single-site study took place at the Johns Hopkins Sickle Cell Center for Adults and the Wilmer Eye Institute. The study population was 90 eyes of 46 consecutive adults with sickle cell disease (SCD). Bilateral nonmydriatic fundus photos taken by clinic personnel during the participants' routine hematology appointment were graded by 2 masked retina specialists at the Wilmer Eye Institute for the presence of nonproliferative SCR (NPSR) and proliferative sickle retinopathy (PSR). A third retina specialist adjudicated in cases of grader disagreement. All participants underwent the standard dilated fundus examination (DFE) within 2 years of acquisition of UWF photographs. The main outcome measure was the sensitivity and specificity of nonmydriatic UWF images for the detection of NPSR and PSR RESULTS: PSR was noted in 19 of 90 eyes that underwent DFE and in 9 of 67 gradable UWF images. Interrater agreement between the 2 graders was moderate, with κ=0.65 (range 0.43-0.87) for PSR. For gradable UWF photos, the sensitivity and specificity of detecting SCR using the nonmydriatic photos compared with the DFE were 85.2% and 62.5% for NPSR, respectively, and 69.2% and 100% for detection of PSR, respectively. One participant required ophthalmic therapy in both eyes for active sea-fan neovascularization. UWF imaging shows utility in screening for SCR and may help identify patients with PSR who require a DFE and who may benefit from treatment.

Clinical Utility of the Addition of Molecular Genetic Testing to Newborn Screening for Sickle Cell Anemia.

Sickle cell disease (SCD) is a group of related yet genetically complex hemoglobinopathies. Universal newborn screening (NBS) for SCD is performed in the United States and many other nations. Classical, protein-based laboratory methods are often adequate for the diagnosis of SCD but have specific limitations in the context of NBS. A particular challenge is the differentiation of sickle cell anemia (SCA) from the benign condition, compound heterozygosity for HbS and gene-deletion hereditary persistence of fetal hemoglobin (HbS/HPFH). We describe a sequential cohort of 44 newborns identified over 4.5 years who had molecular genetic testing incorporated into NBS for presumed SCA (an “FS” pattern). The final diagnosis was something other than SCA in six newborns (12%). Three (7%) had HbS/HPFH. All had a final, correct diagnosis at the time of their first scheduled clinic visit in our center (median 8 weeks of age). None received initial counseling for an incorrect diagnosis. In summary, genetic testing as a component of NBS for SCD is necessary to provide correct genetic counseling and education for all newborns' families at their first visit to a sickle cell center. Genetic testing also permits the use of early, pre-symptomatic hydroxyurea therapy by preventing infants with HbS/HPFH from receiving unnecessary therapy. We argue that genetic testing should be incorporated into contemporary NBS for SCD.

COVID-19 Infection and Outcomes at a Comprehensive Sickle Cell Center

Introduction: The Grady Comprehensive Sickle Cell (SC) center is the largest adult sickle cell center in the United States (US) and has the first 24/7 acute care unit for management of sickle cell vaso-occlusive events (VOE). In 2019, the center provided 3077 sickle cell outpatient visits and 3695 acute care visits. When the COVID-19 pandemic reached the US, the center had a precipitous drop in the number of both outpatient clinic and acute care visits as state regulations for lockdown were passed. This report follows all the COVID-19 cases at a single adult center for sickle cell disease in one year.Methods: The clinical database has been tracking COVID-19 cases reported. Out of a total of 1343 patients, 55 patients contracted COVID-19 and were tracked in the clinical database with IRB approval.Results: Of the 55 patients with COVID-19, 28 were female and 27 were male. By genotype, 64% of patients were SS, 31% were SC and 5% were Sβ+ thalassemia. 35% of patient were on hydroxyurea for disease modification with the majority of them being of the SS genotype and 31% had elevated fetal hemoglobin determined by a percentage fetal hemoglobin above 5% by hemoglobin electrophoresis. Chronic pain (defined as patients experiencing daily pain episodes for more than 4 days a week for the last 3 months) and calculation of daily morphine equivalents were reported in clinic follow-up and the narcotic database utilization. 47% of the patients had chronic pain and the median morphine equivalents was 90 mg daily (45-225mg).The rate of emergency department (ED) visits or hospitalizations for the sickle cell patients with COVID-19 was 80%. 49% of the SC patients' visits were related to VOE and 27% related to COVID-19 primarily. 20% of SC patients with COVID-19 were not seen in any emergency setting or required any hospitalization.The COVID-19 signs and symptoms experienced by the patients were as follows: 58% had pain as the main presenting symptom, followed by cough and fever (40%), dyspnea (31%), and pneumonia with chest x-ray evidence (25%). 2 patients developed acute respiratory distress syndrome (ARDS) and were intubated, and 2 patients died. 29% of the patients had lung findings on imaging and 16 of 55 patients required treatment with the use of Remdesivir in 9, dexamethasone in 8 and red cell products in 7 of the 16 patients. The 2 patients who died had both presented with COVID-19 infection in June and July 2020 respectively. One patient had presented in June 2020 with VOE and was found to have bilateral lung opacities but was asymptomatic and was discharged home to return few days later with clinical picture of multi-organ failure for which a red cell erythrocytapheresis was attempted. The second patient had presented in July 2020 with COVID-19 pneumonia and was treated with Remdesivir and convalescent plasma with development of multi-organ failure and ARDS.Discussion:Several reports were published regarding the rate of COVID-19 related mortality and morbidity in sickle cell disease. The Grady comprehensive sickle cell center experience differs in the fact that 16 out of 55 patients who had contracted COVID-19 required treatment and 2 of those 16 had died. In fact, the deaths occurred early in the course of the pandemic in June and July 2020 when 20 total cases were diagnosed (from March to Septemeber 2020). The remaining 35 cases registered zero deaths (October 2020 to March 2021) with the rate of complicated COVID-19 hospitalizations decreasing with better treatment available. In addition, the timeline for the COVID-19 cases reported fits the population timeline of 2 peaks respectively happening in the summer of 2020 and the Winter of 2021. During the initial peak, we have noted a decrease in the number of clinic and acute care visits respectively. This was anticipated given the statewide lockdown that was implemented. To circumvent that, the center adopted virtual visits to deliver healthcare needs. This measure has aided in protecting patients against COVID-19. Additionally, it is interesting that despite the second peak in the winter of 2021, there were no reported deaths among the patients who developed COVID-19. This finding can suggest that despite the concern for morbidity and mortality of sickle cell patients, their diligence and awareness to stay home during the pandemic has proven crucial in reducing morbidity and mortality and the option of virtual visits for healthcare delivery was key and should be utilized further in sickle cell care. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Excess All-Cause Mortality in 2020 for People Living with Sickle Cell Disease in New York City

BackgroundThe COVID pandemic resulted in excess all-cause mortality in 2020 in the United States, especially among people who identify as Black and/or Latino. While many of these deaths were due to COVID-19 infection, others have been attributed to strain on the overburdened health care system and delays in patients seeking medical care due to the pandemic. We sought to examine the overall mortality in individuals with sickle cell disease (SCD) who received their care at our Sickle Cell Center in Bronx New York in 2020 and investigated whether the number and causes of death in 2020 differed from the previous 3 years.Methods:Death date was collected from electronic medical records (EMR) and death certificates. Clinical variables collected from EMR included age, gender, race, genotype, history of comorbidities, hydroxyurea use, and health care utilization in the 12 months prior to death date. To determine cause of death, two hematologists performed manual review of the EMR blinded from each other and deaths were categorized as due to: sudden death, cardiovascular causes, sepsis, stroke, acute organ failure (including multi organ failure syndrome), chronic organ failure, hemorrhage, and unknown when records of the death event were not available. A third hematologist served as a tie breaker in cases of disagreement. Patients were also categorized as having: 1) sepsis present or absent during the death event, 2) acute (new or newly worsened) renal or liver failure present or absent during the death event, and 3) COVID status at death (acute COVID, past COVID, no COVID, or unknown COVID status). Acute COVID infection was defined by a positive PCR swab during the hospitalization leading to death or in the prior two weeks. Past COVID infection was defined by a negative PCR swab during the hospitalization and a positive PCR swab and/or antibody test more than two weeks before the death event with documented resolution of symptoms. No COVID infection was defined by a negative PCR swab or antibody test documented in the EMR and no positive tests present or noted per patient report. Unknown COVID status was unknown if no testing was present in the EMR. To examine how 2020 decedents differed from decedents in the prior 3 years, we compared clinical variables using the Kruskal Wallis test for continuous variables and chi 2 tests for dichotomous variables. To examine differences between 2020 decedents between known COVID (acute or chronic) vs. no COVID, we repeated similar tests among the 2020 decedents only.Results:In the years 2017, 2018, 2019, and 2020, there were 9, 10, 8, and 22 patient deaths respectively. Compared with decedents in the prior 3 years, patients who died in 2020 were more likely to have acute liver failure during the hospitalization, were more likely to have history of stroke, and had more heme clinic visits in the prior 12 months. Otherwise, there was no difference in age, gender, genotype, hydroxyurea use, history of disease morbidity, hospital utilization in the prior 12 months, or sepsis before death (Table 1). Among the 22 who died in 2020, 3 had an acute COVID infection, five had past COVID, 8 had no COVID, and 6 had unknown COVID status. Among 2020 decedents, acute or past covid was associated with acute organ failure (p=0.02) and less hydroxyurea use (p=0.04). (Table 2).Conclusions:SCD deaths at our center more than doubled in 2020 compared to prior years, but fewer than half of the decedents had acute or prior COVID. Compared with prior years, 2020 decedents were more likely to have acute organ failure. This could be explained by COVID infection, delays in seeking care, or changes in care delivery during the COVID pandemic. This study emphasizes the need for further studies on the impact the pandemic had on the health of adults with SCD, as well as the need for prospective studies of patients with SCD who recover from COVID. [Display omitted] DisclosuresCurtis: GBT: Consultancy. Minniti: Forma: Consultancy; GBT: Consultancy; Novartis: Consultancy; Novo Nordisk: Consultancy; Chiesi: Consultancy; F. Hoffmann-La Roche: Consultancy; Bluebird Bio: Other: Endpoint adjudicator; CSL Behring: Other: Endpoint adjudicator.

Diagnostic Imaging Radiation in Severe Sickle Cell Disease: Cancer Risk Implications

Introduction: Patients with sickle cell disease (SCD) are repeatedly exposed to diagnostic radiation. Radiographs, computed tomography (CT) and nuclear medicine scans are often ordered for suspected complications caused by sickle cell disease that exposes patients with SCD to ionizing or another form of radiation. A few studies of low-dose cumulative radiation exposure (in people without SCD) suggest that 30 to 100 mSv over 30 decades is associated with higher excess risk of leukemia. New epidemiologic data of low quality suggests that individuals with sickle cell disease (SCD) accumulate “driver mutations” for acute myelogenous leukemia (AML) about 20-30 years earlier than the general population, and have higher risk of AML. In a gene therapy protocol with a few dozen patients, 2 cases of AML have occurred in sickle cell disease and none in thalassemia. It has been reported that children with SCD are frequently exposed to ionizing radiation in the form of plain radiographs, fluoroscopy, computed tomography (CT) scans, bone scans, and other tests. Exposure to ionizing radiation during childhood carries a risk of developing cancer that is directly related to the total radiation dose.Epidemiological data has demonstrated an increase both in diagnostic radiation and in actual or predicted resultant cancer diagnosis. Children are particularly vulnerable to radiation-induced cancer because they are still actively growing and thus are at greater risk of acquiring an oncogenic mutation in an actively dividing cell.Hypothesis: Frequent diagnostic imaging for children and adults with SCD can have significant cumulative radiation exposure that could add excess risk of AML.Methods:The study design was a retrospective chart review. The sample was selected to be enriched for the most severely-affected children and adults in the Sickle Cell Center at UI Health, which provides medical care for over 700 patients with SCD. The subgroup on chronic erythrocytapheresis blood transfusionswere selected as a sample of severe of SCD who are more likely to be exposed to repeated diagnostic radiation. Many have had stroke, which often leads to repeated head CT and cerebral angiograms. Others had pulmonary embolism or acute chest syndrome, which can lead to chest CT angiograms. Other SCD complications with high risk of morbidity or morbidity lead to similar likelihood that patients on chronic exchange transfusion therapy would have histories of multiple imaging studies. Medical records were reviewed for the type and number of all radiographic tests, especially CT scans, during the 10-year period 2011-2020. A second observer confirmed a subset of charts. Standard references were used to estimate radiation exposure in mSv for each type of test. The sum of mSv for each individual was a rough estimate of cumulative radiation in 10 years. The IRB approved the protocol.Results:Chart review on 39 patients (ages 16 - 60y) identified 1,030 radiographic tests with a mean of 26.4 tests/patient. Seven patients had > 50 tests, and one patient had > 100 tests over a 10-year period. Thirty-three patients had at least one CT scan. Eighteen patients had at least 3 CT scans. Twenty patients had cumulative radiation exposure > 30 mSv over a 10-year period, 4 patients had > 100 mSv, and one patient had > 200 mSv. Plain radiographs comprised 71% (736) of the studies and relatively low dose radiation exposure.Discussion:This retrospective study estimated that diagnostic radiography exposed 20 of 39 patients with severe SCD to the range of 30 to 200 mSv over 10 years, mostly from numerous CT scans. This range of cumulative radiation exposure has mixed evidence about possible heightened risk of AML and other cancers. The study is limited by the small sample at a single institution and a heavy bias toward patients with stroke and chest complications, but this severely-affected subgroup comprises many of those eligible for transplant and gene therapy in SCD. Cumulative exposure to diagnostic radiation might be one mechanism for the unexplained patterns of AML in SCD after gene therapy that led to a pause in SCD gene therapy studies for a few months in 2021. Further studies are needed. DisclosuresHsu: Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Aruvant: Consultancy, Membership on an entity's Board of Directors or advisory committees; Hoffman LaRoche: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Forma Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Cyclerion: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Imara: Research Funding; Eli Lilly: Research Funding; Baxalta / Shire / Takeda: Research Funding.

The Mortality of Adult Sickle Cell Patients at a Comprehensive Sickle Cell Center

Sickle cell disease (SCD) is the most common hemoglobinopathy affecting 100,000 people in the United States. Although without a national registry, the precise number remains an estimate and the mortality data remains unknown. The life expectancy of SCD has extended into adulthood with the advent of prophylactic penicillin use, antipneumococcal vaccines, newborn screenings, and use of hydroxyurea (HU). However as SCD patients age, comorbidities arise reducing the life expectancy.There is an evolving landscape regarding SCD therapies, with 3 FDA approved drugs since 2017 (L-glutamine, voxelotor, crizanlizumab) joining HU which was approved in 1998. It is paramount to understand the factors that lead to early mortality in SCD so that future therapies and cures can be accessible and benefit all SCD patients. We embarked a review of the mortality data in a healthcare system serving a significant number of SCD patients in Atlanta, GA.Methods:We conducted a retrospective chart review of the Georgia Comprehensive Sickle Cell Center at Grady Health system's electronic medical record for deceased sickle cell patients from 2013 to 2020. Patients were then grouped into 3 groups, those whom followed up at the Sickle Cell Center (defined as outpatient visit within 6 months prior to death-RF), those whom did not (defined as outpatient visit > 6 months-NF), and those with unknown status.Results:Overall, 72 patients were analyzed, with the following genotype distribution: SS n=49, SC n=14, Sbeta null thalassemia (Sβ0 thal) n=2, SBeta plus thalassemia (Sβ+ thal) n=7. Overall, the most common SCD complication was acute chest syndrome (ACS) (62.5%). The majority had hypertension (HTN) (52.8%), followed by reported heart failure (40.3%) and pulmonary HTN (pHTN) (40.3%), stroke (37.5%), and chronic kidney disease (CKD) (31.9%). 35% were current and/or former smokers. Median TR max velocity was 2.8 m/sec, median albumin/Cr ratio was 27.8 mg/g, and other characteristics are in Table 1.The median age of death was 44 (STD= 15.5) overall and by genotype it was: SS at age 38 (STD=14.6); Sβ0 thal at age 42.5 ( STD=2.12); Sβ+ thal at age 50 ( STD=14.7); SC at age 51 (STD=16.5). 46 patients had data of their final hospitalization available. 73.9% (n=34) received at least 1 simple transfusion with a only 17.4% (n=8) receiving an exchange transfusion. The majority were critically ill, with 84.8% in the ICU and 95.7% with multiorgan failure.50 patients had routine follow up (RF) while 14 patients did not (NF), and 8 patients with unknown outpatient visit status. The median age of death for RF and NF were the same 43.5 (STD=15.53) & 43 (STD=15.71) years, respectively. The genotype distribution, and other characteristics are listed in Table 2. About 44% (n=22) of the RF group were prescribed HU, with 31.8% (n=7) were prescribed a dose > 15mg/kg/dose. This was not a significant difference from the NF group, where 50% (n=7) were prescribed HU, with 42.9% at > 15mg/kg/dose. The median MCV, percentage of HbF were similar. Significant majority in RF and NF groups were prescribed chronic opiates (86% and 78.6%, respectively) with 105 and 95 median MME per day. 42.9% of patients in the NF group had alloimmunization compared to 26% in the RF group.Discussion:Most of the patients had at least 1 severe complications from SCD, and most had other comorbidities (Table 1). This is commensurate with SCD literature showing that the manifestations of chronic end-organ damage increase as patients age which increases the likelihood of early mortality. In our analysis, the median age of death was similar for those in the RF and NF groups across genotypes.HU is a disease-modifying therapy that impacts survival and reduces complications and was FDA approved in 1998, however less than 50% of patients had a prescription. Median HbF and MCV were similar between the 2 groups, suggesting HU adherence was not a factor. This raises concern about accessibility of HU for patients which was not within the scope of this review. Improvement of access and education for patients and clinicians of these therapies is needed to see reduction in mortality. As more of the adult population starts to include those with access to HU since early childhood, future studies can examine what impact that will have on HU adherence, and life expectancy. With the advent of new therapies, it is imperative to study these agents on the patients with SCD complications to evaluate their efficacy in reducing mortality. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Splenic Complications in Sickle Cell Disease: A Retrospective Cohort Review

▪Splenic dysfunction, particularly acute splenic sequestration crisis (ASSC), is a leading cause of death in the first decade of life of patients with sickle cell disease (SCD). Despite the severity of splenic disease in SCD, few large-scale studies have been performed to assess the natural progression of splenic complications, particularly in the less common sickle cell genotypes and with the onset of early use of hydroxyurea.We performed a retrospective chart review of patients with SCD born between January 2011 and December 2018 and treated at the Texas Children's Hospital Sickle Cell Center. We extracted data from our electronic medical record system to identify patients with documented splenic complications based on ICD9 and ICD10 codes. We then reviewed charts for these patients to confirm diagnoses and obtain additional information about these complications.We identified 688 patients treated for SCD at our center during the study period. Of these, 113 (16.4%) were diagnosed with one or more splenic complications. The overall prevalence of splenic complications was highest in patients with hemoglobin Sβ 0 thalassemia (Sβ 0) at 50.0%, compared to 20.7% in hemoglobin SS (SS) patients, 6.9% in hemoglobin SC (SC) patients, and 0% in hemoglobin Sβ + (Sβ +) patients and patients with other genotypes (Table 1). ASSC and splenectomy were likewise most prevalent in Sβ 0, less so in SS, and rare in SC, Sβ +, and other genotypes.To further delineate the incidence of ASSC, we performed an in-depth analysis of patients from our cohort for whom detailed information was available. Compared to patients with the SS genotype, the incidence rate ratio (IRR) for ASSC was significantly higher for Sβ 0 patients and significantly lower for SC patients (Table 2). The mean age of onset of ASSC was similar between SS, Sβ 0, and SC patients, but survival curves for time to the first event differed significantly between genotypes.We examined the association between early hydroxyurea therapy and ASSC in SS and Sβ 0 patients by comparing patients who initiated hydroxyurea therapy by the age of two years to those who initiated therapy after this age. ASSC were more frequent among patients initiated on hydroxyurea by the age of two years (Table 3, upper panel). We also compared patients on hydroxyurea to those not on this therapy and found a similar effect, with ASSC occurring more frequently among patients on hydroxyurea (Table 3, lower panel). Survival curves for time to first ASSC also diverged significantly between these groups of patients.Our data indicate that the prevalence and severity of splenic problems vary widely between different sickle cell genotypes in the first decade of life, with SC patients having relatively mild complications while Sβ 0 patients have the most severe and frequent complications. The association between hydroxyurea use and incidence of ASSC also suggests that hydroxyurea therapy may increase the risk of this complication of SCD, particularly when initiated before two years of age. [Display omitted] DisclosuresGeorge: Global Blood Therapeutics: Consultancy, Speakers Bureau; Wolters Kluwer: Patents & Royalties.

The Telemedicine Experience for Individuals with Sickle Cell Disease

IntroductionIndividuals with Sickle Cell Disease (SCD) require regular, and specialized treatment to manage their health. The COVID-19 pandemic disrupted in person medical visits for all individuals, with a rapid transition to telemedicine to provide medical care. Emerging data shows that the use of telemedicine may provide easier access to care and remove barriers to clinic attendance and improve access to appropriate medical care.ObjectiveThe purpose of this study was to use qualitative methods to understand the patients' experiences with telemedicine, identify patient preferences for type of appointment, and possible suggestions to improve telemedicine care.MethodsPatients from the Johns Hopkins Sickle Cell Center for Adults who had at least one telemedicine visit were invited to participate in a semi structured interview via zoom meeting or telephone. The interview asked participants about their satisfaction with telemedicine care, barriers to telemedicine, benefits and risks of telemedicine and possible telemedicine improvements. Interviews were recorded, transcribed and coded by two independent raters using thematic analyses to understand the experiences of telemedicine during the COVID-19 pandemic.ResultsOverall, 30 adults with SCD who had at least one telemedicine visit were invited to participate and completed their interview (mean age 41 years ± xx, 67% female, 93% Black/African American, 3% Multi-Race, 3% Other). “...I can't ignore the convenience of not having to worry about transportation ... that there's nothing to stop me from getting there.” During a SCD pain crisis it can it challenging to move and receive treatment as one participant reported “Maybe sometimes I might have pain...then moving around makes it difficult. So, getting in the car and finding somebody to drive you to a hospital or to whatever clinic would be difficult”. Being able to access specialized SCD care even while in pain is important. Having the option of either having telemedicine or in person visits was important to SCD patients “I could treat my crisis here at home. I don't have to go to the emergency room for it. So, if I can see my doctor in the tele-visit appointment and it's going to be constantly every day ... And when it's getting worse, then I could go to the emergency room more if needed. If it's not needed, I don't even need to go”. Another emerging theme amongst participants was despite the benefits from telemedicine, they also wanted to continue having in-person visits when they needed. SCD participants felt due to their SCD they still needed to see their doctor in person but it did not have to be for every visit “Well, I think telemedicine, for me, can be used in a setting where there's no such an emergency. Like if I'm having a routine exam, I don't mind having the telemedicine. But if ... I'm not feeling well ... I don't want to be having a telemedicine”. SCD participants felt they needed a physical exam periodically. “The only thing I didn't like about it was if I'm having some discomfort or some pain... there was no way for the physician to physically examine me”. Along with the lack of physical exam, there were concerns about the lack of vital signs “... the drawbacks would be the lack of the vitals beingtaken or there's not the personal touch and stuff”.ConclusionThe COVID-19 pandemic has presented many obstacles for patients to receive care. People living with SCD found telemedicine to be a positive tool to receive treatment. Patients reported the desire to continue with telemedicine even after the COVID -19 pandemic. Telemedicine allows for more accessibility for a group of individuals who already have numerous barriers to treatment. Future research can seek to identify the impact that telemedicine has on no-show rates, health care utilization, and the impact telemedicine has on patient reported quality of life. DisclosuresLanzkron: Teva: Current holder of individual stocks in a privately-held company; Shire: Research Funding; GBT: Research Funding; CSL Behring: Research Funding; Novo Nordisk: Consultancy; Bluebird Bio: Consultancy; Pfizer: Current holder of individual stocks in a privately-held company; Imara: Research Funding; Novartis: Research Funding.

Blood Conservation Strategies in Adult Sickle Cell Patients on Chronic Transfusion Therapy: A Single Center Experience

▪Background: Adult sickle cell patients on chronic transfusion therapy (CTT) garnered special concern during the COVID-19 pandemic, when mass cancellation of blood drives threatened the national blood supply. In response, the American Society of Hematology proposed several strategies to decrease blood utilization, while maintaining adequate disease control for sickle cell patients on CTT. These included targeting a higher end hemoglobin S%, switching patients to simple transfusions when appropriate, and transitioning to alternative disease modifying therapies. There is little evidence to support the safety of altering exchange therapy regimens. Accordingly, at the Johns Hopkins Sickle Cell Center for Adults, a multidisciplinary team of clinicians and transfusion medicine specialists evaluated patients on a case by case basis to determine how their exchanges could be modified to accommodate for anticipated blood supply shortages. We describe our blood conservation efforts during the COVID-19 pandemic and resulting clinical outcomes for adult patients with sickle cell anemia (SCA).Methods: For inclusion in this IRB-approved retrospective study, patients received at least 7 monthly exchange transfusions between March 2019 and February 2020 and continued care through March 2021. Decisions regarding CTT were made prior to data collection. Modifications to chronic exchanges included increasing the fraction of cells remaining (FCR), decreasing the end hematocrit, or switching to a hemodilution method. Additionally, select patients transitioned to monthly simple transfusions if they were clinically stable, had a hemoglobin ≤ 7 g/dL, and had a persistently suppressed hemoglobin S% (≤ 30).We collected basic demographics, pre-exchange laboratory studies, and exchange parameters before and after each transfusion encounter in the year before (March 2019 - February 2020) and during (March 2020 - February 2021) the COVID-19 pandemic. Lastly, we recorded presentations to urgent care and the emergency department; and hospital admissions during each time period. We reported descriptive statistics for the cohort and compared outcomes using the Wilcoxon signed-rank test and Mann Whitney U test. We analyzed data using Stata/SE Version 16.1.Results: We identified 58 patients with SCA who qualified for inclusion (Table 1). Fifty-three patients remained on chronic exchange transfusions during the pandemic, and five were switched to simple transfusions. For patients who remained on chronic exchanges, most received conventional automated red cell exchange (RCE) prior to (85%) and during the pandemic (77%). Use of hemodilution increased (15% to 23% of patients). Forty-three patients experienced an increase in mean FCR (33.6 (SD 11.6) vs 37.7 (4.7), p = .00). Of those, 22 patients saw a concomitant decrease in mean end hematocrit (30.8 (1.7) vs 29.6 (1.1), p = .00). These changes resulted in a decline in the average number of units per procedure (8.6 (1.9) vs 7.3 (1.7), p = .00), which corresponded to 890 units conserved. Mean pre-transfusion hemoglobin values declined (9.4 (1.3) vs 9.2 (1.3), p = 0.01), but hemoglobin S%, reticulocyte count, and ferritin values were unchanged (p > 0.05). Acute care presentations and hospital admissions declined, which were likely spurred by concerns about COVID-19 infection (Table 2, p < 0.05).During the pandemic, three patients died, one of whom had been switched to simple transfusions. This patient experienced a consistent rise in hemoglobin S% until death. Two of the remaining patients on simple transfusions were switched back to automated RCE due to an increase in hemoglobin S% above goal (Figure 1).Conclusions: During the COVID-19 pandemic, we conserved red blood cell units through expanded use of hemodilution, higher FCR values, and switching some patients to simple transfusions. Patients who remained on exchanges maintained hemoglobin S% values near a goal of 30% without increasing iron burden. In contrast, the majority of patients who were switched to simple transfusions were unable to maintain goal hematologic parameters, and one patient died. Our data suggest that in a blood shortage crisis, changing the exchange procedure itself may be the safest means of conserving blood in a population of adult sickle cell patients; however longer follow-up is needed to ensure that these changes are safe. [Display omitted] DisclosuresLanzkron: GBT: Research Funding; Shire: Research Funding; Novo Nordisk: Consultancy; CSL Behring: Research Funding; Pfizer: Current holder of individual stocks in a privately-held company; Teva: Current holder of individual stocks in a privately-held company; Novartis: Research Funding; Imara: Research Funding; Bluebird Bio: Consultancy.

Knowledge of Fertility and Perception of Fertility Treatment Among Adults with Sickle Cell Disease (KNOW FERTILITY)

BackgroundAdults with sickle cell disease (SCD) face unique fertility risks due to SCD and use of disease modifying therapies (DMTs). Concerns about compromising fertility may inform patients' therapy choices, but little is known about fertility knowledge in adults with SCD. The Cardiff Fertility Knowledge Scale (CFKS) and Fertility Treatment Perception Survey have been studied in international and national cohorts 1,2. The purpose of this study was to administer these surveys to adults with SCD and compare responses to previously studied populations.MethodsOur IRB approved this cross-sectional study of adults with SCD (≥18YO) cared for at our Sickle Cell Center for Adults. Due to the COVID-19 pandemic, eligible subjects were recruited during routine telemedicine clinic visits and by invitation via electronic medical record. We collected demographic information (sex, age (≥/< 31YO), educational attainment, and use of DMTs). The CFKS is a 13-question survey that measures knowledge of causes of reduced fertility, common misconceptions about fertility, and infertility facts. Questions are answered True/False/Don't know and equally weighted; the cumulative score is 0-100%. We compared the mean CFKS scores to the scores from two published cohorts 1,2. The fertility treatment perception survey consists of two positive and four negative statements about fertility treatment with responses given on a five-point Likert scale (1= strongly disagree to 5= strongly agree). Responses are calculated by number of respondents with an agreement score of 4 and/or 5 divided by total number of respondents per sub-group; higher scores indicate stronger agreement. Analysis included summary statistics with means and standard deviations and independent student's T-test to compare the mean fertility knowledge scores.ResultsWe contacted 435 subjects; 91 respondents were enrolled (21% response rate). Respondents were 77% female [median age 33 years (IQR 23, 50)]. 51% completed high school or less and 18% used one or more DMTs, with 65% taking hydroxyurea. Table 1 shows the CFKS results. The average CFKS score was 50%, lower than the international cohort (50% vs. 57%, p<0.001) and higher than a cohort of Black women in Atlanta, GA (50% vs. 38%, p<0.001). Respondents with higher educational attainment had a higher score (55% secondary education vs. 44% primary education, p=0.04). The questions most answered correctly addressed the lack of correlation between erectile function and fertility (79%) and smoking's risk to fertility in men (69%) and women (71%). The questions least answered correctly were about classifying infertility (32%) and the impact of age effect (34%), overweight effect (25%), and sexually transmitted infections' effect on fertility (36%). There was no difference in knowledge scores by age, sex, or SCD treatment. Table 2 shows fertility treatment perception survey results. Some respondents (34%) agreed that fertility treatments are safe. Almost half (46%) agreed that fertility treatments are effective. Over 60% of respondents agreed that fertility treatments are scary and/or cause emotional problems, while 48% agreed that fertility treatments may have short-term physical effects. There was no difference in responses by sex, age, or SCD treatment.ConclusionIn this study, we identify that higher educational attainment in adults with SCD is associated with better fertility knowledge. All subjects had low knowledge of sexually transmitted infections, weight gain, and older age as infertility risks. Although there is concern that hydroxyurea may compromise fertility, its use was not associated with greater fertility knowledge in this study. Given concerns about fertility in the SCD community, we identify an opportunity to support patients concerned about fertility by contextualizing real or theorized SCD-specific fertility risks within a broader set of established fertility risks.

Incidence of Stroke and Other Brain Imaging Findings in Adult Patients with Sickle Cell Disease: A Retrospective Study

Background: Vaso-occlusion and hemolytic anemia are the main features of sickle cell disease. Central Nervous system complications of sickle cell disease (SCD) can include silent cerebral infarcts (SCI), hemorrhagic or ischemic strokes, seizures and Moyamoya syndrome. SCI is the most common neurological complications in SCD. SCI patients have abnormal Magnetic Resonance Imaging (MRI) of the brain with normal neurological examination and no history of stroke. More precisely, Casella et al in the Silent Cerebral Infarct Multi-Center Clinical trial (SIT) defined infarct as MRI signal abnormality that was at least 3 mm in one dimension and that was visible in two planes on fluid-attenuated inversion recovery T2 -weighted images, as determined by agreement of two of the three study neuroradiologists. SCI in children is associated with lower Intelligence Quotient and increased risk of stroke.Methods: In this retrospective study we reviewed 100 adult patients at our Comprehensive Sickle Cell Center. We looked at the incidence of documented stroke and other neurological complications in SCD patients. We reviewed the hospital records of SCD patients for any incidental brain imaging (MRI brain or Computed Tomography(CT) brain) findings, done as inpatient or in the emergency room as part of hospital visits.Results: Out of the 100 patients, 14 patients had documented stroke. On the review of other 86 patients, 10 patients had MRI brain, 23 patients had CT brain, and 53 patients had no brain imaging record. Out of the 10 patients with MRI brain, 7 patient had significant findings- 3 patients had small vessel ischemic changes, 1 patient with chronic petechial hemorrhage of cerebellar vermis, 1 patient with ischemic changes and Chiari malformation type 1 (CTM 1), 1 patient with CTM 1 and non-specific white matter changes and 1 patient with CTM 1 alone. Out of the 22 CT of the brain and 1 CT angiogram of brain, 2 imaging showed ischemic changes and the angiogram showed large cerebral vessel narrowing. Therefore, on our review, 19 patients out of 100 were found to have stroke and 24 out of 100 had significant neurological findings. However, only 14 out of 19 patients had documented stroke. 7 out the 10 patients with MRI brain had significant incidental neurological findings. The number of adult SCD patients with neurological findings in our cohort could have been higher, if we had brain imaging record for the 53 other patients.Conclusion: This study shows that there is a heavy burden of neurological complications in SCD patients and SCI is under-diagnosed. MRI brain is superior to CT brain in detecting neurological complications in SCD patients. SIT showed that regular blood-transfusion significantly reduced the recurrence of cerebral infarct in children with SCI. Finding of SCI in adults also warrants consideration for follow up brain imaging, neurology referral and discussion of treatment modalities including chronic blood transfusion and hydroxyurea therapy. CTM 1 can also be associated with SCD. In CTM 1, tonsil herniates 5 millimeter or below foramen magnum. SCD may cause increased thickness of calvarium from extramedullary hematopoiesis in skull, which can result in cranio-cephalic disproportion with cerebellar ectopia and subsequent CTM 1. Because of high incidence of neurological complications in SCD patients, routine screening with MRI brain should be considered in adult SCD patients. DisclosuresIdowu: Forma Therapeutics, Inc.: Research Funding; Pfizer: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Ironwood: Research Funding.

Neurocognitive Assessment of Adults with Sickle Cell Disease: A Descriptive Study

Introduction: Sickle cell disease (SCD) causes devastating complications that can affect any organ in the body. Particularly, SCD can present with neurological complications of overt strokes, silent infarcts and cognitive impairment. As patients are living longer with SCD, cognitive functioning is an important aspect of their disease as deficits can impact education, employment, or adherence to medications. Most of the studies assessing cognitive impairment in this population have been in children with limited data on adults. This study explored the results of cognitive testing in adult patients with SCD when compared to normative data. This study also sought to determine any association between psychological factors (baseline anxiety and depression) as well as biological factors (i.e. hemoglobin levels).Methods: This was a cross-sectional study conducted at the Sickle Cell Center of Southern Louisiana in New Orleans.The study included adults with a diagnosis of sickle cell disease regardless of subtype who were over the age of 18. Patients were excluded if they were not able to physically complete the tasks. Executive function, memory, psychomotor speed, and memory were assessed using the following tasks from standardized pencil-and-paper cognitive tasks from the Cambridge Neuropsychological Automated Battery (CANTAB) test: Stocking of Cambridge (SOC),Delayed Matching to Sample (DMS), Paired Associates Learning (PAL), Motor Screening Task (MOT), Intra-Extra Dimensional Set Shift (IED), Spatial Working Memory (SWM). The Hospital Anxiety and Depression Subscale (HADS) clinical assessment tool was used to screen for anxiety and depression at the time of testing. A HADS score >8 denotes significant anxiety or depression.Results: A total of 22 patients (59% females, mean age 29.6±2.1 years) were included in this study. Attention and psychomotor speed were relatively preserved cognitive domains with 59.1% of patients scoring greater than 75 thpercentile relative to normative mean. Conversely, executive functioning often appeared impaired with 72.7% and 77.3% of patients scoring below 25 thpercentile in outcome measures of IED and SWM, respectively. Similarly, a significant percentage of patients scored below 25 thpercentile in outcome measures of visual memory, PAL and DMS, 63.6% and 45.4%, respectively. Fifteen participants (68%) screened positive for anxiety while two screened positive for depression. Patients with anxiety tended to perform worse on most cognitive tasks, although the differences in scores did not reach statistical significance. Additional analyses of association of biologic factors and neurocognitive functioning are currently underway.Discussion and Conclusion: Our results support that adults with sickle cell disease often suffer from cognitive deficits, which was expected based on pediatric studies demonstrating cognitive impairment in children with SCD. Interestingly, we observed a predominance of poor executive function over changes in attention and psychomotor speed in this study. This is in contrast with what providers familiar with “mini mental assessment” for dementia may expect. Typical dementia patients develop attention and memory changes before executive function is affected. Therefore, it is possible that cognitive impairment in SCD may go by unnoticed without proper testing. Additionally, in this cohort, anxiety was frequent and tended to associate with worse performance. Since this is a cross-sectional study, it is unclear whether cognition is progressively impaired over the years. Prospective studies are required to help determine whether and how fast progression occurs and what risk factors are implicated in the development and progression of cognitive impairment. Such deficits have been demonstrated to be associated with difficulties around employment and adherence to medication, which ultimately jeopardizes long term outcomes. Overall, we recommend neurocognitive and psychological evaluations as part of the routine care for adult SCD patients since abnormal findings seem common and may not be obvious without adequate testing for different domains. Treatment of anxiety disorder and cognitive rehabilitation may prove helpful to improve cognition in SCD patients. DisclosuresFertrin: Sanofi Genzyme: Consultancy, Membership on an entity's Board of Directors or advisory committees; Agios Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Kruse-Jarres: Genentech/Roche: Speakers Bureau; Pfizer: Consultancy; CSL Behring: Consultancy; CRISPR: Consultancy; Biomarin: Consultancy; Genentech: Consultancy, Research Funding.

The Effect of Individualized Pain Plans for Patients with Sickle Cell Disease on Emergency Provider Satisfaction: A Win for the Patient Can be a Win for the Provider

BackgroundSickle cell disease (SCD) is an inherited hematologic disorder that affects approximately 100,000 Americans and results in over 200,000 emergency departments visits annually, largely due to pain (Lanzkron et al). Delay in treatment in emergency room has been a significant barrier to patients with SCD, particularly adults. The objective of this study is to determine the effects of utilizing individualized pain plans for the treatment of vaso-occlusive crisis (VOC) on the satisfaction of healthcare providers in the emergency department (ED).MethodsThe Ohio State University has a comprehensive sickle cell center which creates individualized pain plans for patients who present to the ED with pain related to VOC. In January 2015, these pain plans were implemented into the electronic medical record listed in the overview of the problem sickle cell disease in each chart. In addition to creating pain plans, an interdisciplinary team was formed consisting of hematologists, pharmacists, and ED providers with the goal of education regarding SCD and the new implementation of pain plans. Surveys, using the secure web application, RedCAP, were distributed to the emergency department providers at the OSU ED. Questions included responders' role in the ED, prior experience with treating pain crisis, time to make treatment management decisions for VOC, satisfaction with treatment decision, and the providers view of their relationship with patients. Wilcoxon signed-rank test and Fisher's exact test were applied to evaluate the differences between pre and post survey numeric and categorical responses.ResultsSurveys were sent electronically to 170 ED providers. Sixty-nine responses, making up 40.5% of those surveyed, were obtained from 30 attending physicians, 2 fellows, 22 nurse practitioners or physician assistants, 1 registered nurse, and 14 residents. Of those who answered the survey, 14 had experience with treating pain crisis prior to the implementation of individualized pain plans. Implementation of individualized pain plans led to a reduction in median time to make treatment decisions from 5.5 to 2.5 minutes with a p-value of 0.0161. Provider satisfaction with treatment decisions improved as well (p = 0.0029) (Figure 2). In addition, ED providers felt more satisfied with their relationship with patients (p = 0.0078) (Figure 3). The majority of responders (91.2%) also rated their satisfaction with the treatment decision as either satisfied or very satisfied (Figure 1). Seventy eight percent of those answering the survey rated with relationship with patients as being good or very good (Figure 1). In terms of the ease of finding the pain plan in the electronic medical record, 91.3% of providers found them to be either very easy or easy to locate with 94.12% responding that implementing the plan was either easy or very easy (Figure 4). Regarding efficacy of the pain plans, 89.85% found the pain plans to be either effective or very effective (Figure 5). Finally, of the 36 providers who worked elsewhere, about half of the institutions from which they came did not have pain plans.DiscussionThe results of this study show the importance of utilizing individualized pain plans in the treatment of VOC in the ED. As shown in our prior studies, the implementation of individualized pain plans for patients with SCD resulted in a 48% decrease in time to first opioid in the ED, thereby signifying more prompt treatment (Della-Moretta et al). Not only does the data support an improvement in time to make treatment decisions, which benefit the patients, but providers also appear to view their use as an advantage. Pain plan utilization also leads to an increase in provider confidence in their treatment plans as well as a perceived improvement in patient-provider relationships. This is particularly significant as historically the relationship between emergency room staff and sickle cell patients has been seen as challenging by both patients as well emergency room providers (Haywood et al). Making patient centered individualized pain plans readily available, easily accessible, and simple to enact, can further enhance the relationship between the patient, emergency room, and the hematology team. Ongoing communication and education between all parties is beneficial. With the combination of patient and provider data, we show that a win for the patient can also be a win for the provider. [Display omitted] DisclosuresDesai: Pfizer: Other: Publication Fee, Research Funding; Novartis: Research Funding, Speakers Bureau; Global Blood Therapeutics: Honoraria, Research Funding; Foundation for Sickle Cell Research: Honoraria; Forma: Consultancy.

Quantitative Assessment of Physical Activity in Adults with Sickle Cell Disease

Cardiopulmonary disease is a leading cause of death for adults with sickle cell disease (SCD) and targeted therapies to modify cardiopulmonary disease currently do not exist. Physical fitness is an important predictor of cardiovascular morbidity and overall survival. More specifically, individuals who have lower fitness are at a higher risk for poor cardiovascular outcomes and premature death. Previous studies show that adults with SCD have lower physical fitness levels compared to the general population and deconditioning is one of the reasons cited for lower fitness in SCD. Fortunately, increasing physical activity can improve physical fitness and can result in better daily functioning, disease-related symptomatology, and cardiovascular outcomes in other chronic disease populations. Thus, evaluating the amount of physical activity in SCD provides an excellent opportunity to explore strategies to improve physical fitness which could have larger implications to cardiovascular health in SCD. The objective of this study is to determine physical activity levels in addition to identifying barriers, facilitators, and attitudes towards physical activity in an adult population with SCD.Methods: We obtained consent from 110 ambulatory adults with SCD (age=19-68 yrs; 69% female; 77% HbSS) during routine visits at an adult comprehensive sickle cell center. Participants completed validated quantitative assessments (International Physical Activity Questionnaire, CDC Barriers to Exercise Survey, and Determinants of Physical Activity) to determine weekly physical activity levels and to identify barriers, facilitators, and attitudes towards physical activity. High self-efficacy is a marker of resilience to sustaining physical activity, so we evaluated self-efficacy using a scale tailored to SCD, the Sickle Cell Self-Efficacy Scale. Information regarding genotype, gender, age, pain medication use, disease-modifying therapy, hospital admissions, and acute care visits were obtained from the electronic medical record. Physical activity levels were stratified into three groups: (1) sub-adequate( < 3 days of low to moderate-intensity exercise), (2) adequate(3 days of moderate-intensity activity or > 4 days of low-intensity activity), and (3) optimal ( ≥4 days of moderate-intensity activity). We compared proportions across the three groups using chi-square analysis. To compare scales across the three groups, we used the non-parametric Kruskal-Wallis test.Results: 94 completed entries and 16 partial entries were analyzed at the conclusion of the study. Only 21.8% (n=24) of the participants reported optimal levels of physical activity. There were more males in the optimal activity group (N=18/24, p=<0.0001) compared to the other groups, but there was not a significant difference between genotype and age among the three groups. Among the barriers evaluated, lacking energy was identified more often in the sub-adequate group compared to the other two (Table 1). Other important barriers to physical activity such as lack of time, social influence, and so forth were identified more often in the sub-adequate group than the optimal group but this was not statistically significant (Table 1). Facilitators to physical activity and self-efficacy scores did not differ tremendously between the three groups. Interestingly, participants in the optimal sub-category were more likely to use short-acting pain medications compared to the other groups (Table 1). However, there were no significant differences between use of long-acting pain medications, the use of disease-modifying therapy, and hospital admissions among the physical activity groups.Limitations: The quantitative surveys were validated in the general population which may not include the needs specific to SCD. Self-reported physical activity levels are subjective and consequently subjected to recall bias.Conclusion: This is the first report of physical activity levels in US adults with SCD. The majority of SCD participants reported sub-optimal levels of activity and currently do not meet the national guidelines of physical activity in the US. This study underscores the importance of implementing strategies such as regular exercise to increase physical activity in SCD. Gender-differences in physical activity, pain medication use, and fatigue as barriers and facilitators to physical activity will need to be studied further. [Display omitted] DisclosuresOgunsile: Global Blood Therapeutics: Consultancy, Other: Disease-Specific Presentations on SCD; Clearview Research Institute: Consultancy, Honoraria; Novartis: Consultancy, Other: Treatment-Specific Therapy on Crizanlizumab ; Forma: Consultancy, Other: Attended one Advisory Board Meeting . Lebensburger: Novartis: Consultancy; Bio Products Laboratory: Consultancy. Kanter: Fulcrum Therapeutics, Inc.: Consultancy; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Forma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Agios: Honoraria, Membership on an entity's Board of Directors or advisory committees; Beam: Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees; Graphite Bio: Consultancy; GuidePoint Global: Honoraria; Fulcrum Tx: Consultancy.

Telehealth acceptability and opioid prescribing patterns of providers of painful chronic diseases during the COVID-19 pandemic: A survey of sickle cell providers.

The coronavirus disease 2019 (COVID-19) has led to a rapid transition to telehealth services. It is unclear how subspecialists managing painful chronic diseases-such as sickle cell disease (SCD), an inherited hemoglobinopathy with significant disparities in access and outcomes-have viewed the transition to tele-health or altered their pain management practices. This study elicits the views of sickle cell providers regarding their transition to telehealth and their opioid prescribing patterns during the COVID-19 pandemic. An anonymous online survey was sent to eligible sickle cell providers. Comprehensive sickle cell centers and/or clinics across the United States. Physicians and advanced practice providers providing care to SCD patients. Respondents answered questions regarding their (1) views of telehealth compared to in-person encounters and (2) opioid prescribing practices during the early months of the pandemic. Of the 130 eligible participants, 53 respondents from 35 different sickle cell centers completed at least 90 percent of the survey. Respondents reported a significant increase in telehealth encounters for routine and acute appointments (mean difference and standard deviation: 57.6 ± 31.9 percent, p < 0.001 and 24.4 ± 34.1 percent, p < 0.001, respectively) since COVID-19. The overwhelming majority of respondents reported no changes in their opioid prescribing patterns since COVID-19, despite increased telehealth use. Only a minority copre-scribed naloxone as a risk mitigation strategy. The rapid uptake of telehealth has not suppressed ambulatory providers' prescribing of opioids for SCD. Studies assessing the impact of the COVID-19 pandemic and telehealth on opioid prescribing practices in other painful chronic diseases are needed to ensure health equity for vulnerable pain patients.

Pain Coping Strategies Used and Its Perceived Effectiveness Among Patients Attending Sickle Cell Center in Benin City, Edo State, Nigeria

Background: Sickle cell anemia is prevalent in the society with pain as the key factor affecting the life of the individuals with the disease. A large number of sickle cell patients visit the health care facilities due to episode of pain crisis. Aim: This study assesses pain coping strategies used and its perceived effectives among sickle cell patients attending sickle cell center in Benin City Edo State. Methods: A cross sectional descriptive survey design was used to conveniently assess a total of 140 participants who attends the sickle cell center using self-structured questionnaire as instrument for data collection. The instrument was subjected to face and content validity as well as reliability using split half reliability test. Data were analyzed using descriptive and inferential statistics such as T-test, and multivariate logistic regression tests at 5%level of significant. Statistical Package for Social Sciences version (SPSS) 21.0 was use in all analysis. Result: Findings from the study shows that use of pain reliefs, communication of pain to friends, prayers, hypnosis, guided imagery and Diversional therapy such as playing video games was the most commonly used coping strategies having a mean of &gt;2.5. Pain coping strategies such as prayer, relaxation and use of pain relievers were the most effective coping strategies used (mean &gt;2.5). Generally, about 66.4% of the entire population showed that coping strategies were effective to relief pain. Family and peer support were reported as the major factor influencing the use of coping strategies. Significant difference (p&lt;0.05) was found in the coping strategies used by male and female respondents. Respondents who strongly agreed that economic factor played a major role in effective pain coping strategies were 0.328 times more (B-1.114; OR 0.328; CI 0.142-0.759, p = 0.009) likely to believe that coping strategies were effective. Also, respondents who chose routine checkups as a factor affecting pain coping strategies were 3.035 times more (B 1.110, OR;0.3035, CI;1.301-7083, p = 0.010) likely to choose those coping strategies were effective. Conclusion: Considering the fact that pain is a lifelong issue for people with sickle cell disease, different coping strategies and how to effectively implement them should form the nucleus of health education given by nurses and other health care professionals to patients with sickle cell diseases. Patients should be taught on how to carry out these procedures themselves in order to manage pain in its prodromal phase as well as reduce frequent hospital visits and cost.

Health-related quality of life in infants, toddlers and young children with sickle cell disease.

Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0-1year) and Pediatric Quality of Life Inventory (PedsQL, 2-7years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0-7years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. The study enrolled 136 caregivers of 136 children. In children aged 0-5years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5-7years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

Low rates of transfusion-transmitted infection screening in chronically transfused adults with sickle cell disease.

Adults with sickle cell disease (SCD) on chronic transfusion therapy are exposed to a large volume of blood products, thus increasing their risk of transfusion-associated human immunodeficiency virus (HIV), hepatitis C (HCV), and hepatitis B (HBV). We performed a systematic chart review of chronically transfused SCD subjects at the Johns Hopkins Sickle Cell Center for Adults between October 2014 and September 2019 to determine our Center's adherence to the 2014 National Heart, Lung and Blood Institute (NHLBI) SCD guidelines for annual screening for Transfusion Transmitted infections (TTI) and assessed HBV immunity and HBV vaccination rates. The study included 85 subjects with a median age of 34 years (23-63); 52% were female. No subject received annual screening; 68 subjects (80%) were screened for HIV, 60 subjects (71%) for HCV and 53 subjects (62%) for HBV infections at least once in the study period. Of those screened, one patient was newly diagnosed with HCV infection, and none with HIV or HBV infection. Among 31 subjects tested for anti-Hepatitis B surface antibody, 16 subjects (52%) tested negative. Nineteen (20%) subjects had HBV vaccination documented. Low adherence to the NHLBI TTI screening guidelines, especially for HBV, highlights the resource intensiveness of this patient population. The low rates of anti-Hepatitis B surface antibody positivity highlight the need to confirm vaccination, provide boosters as indicated, and investigate the adults with SCD's immune response to HBV vaccination.

Assessment of Referral Patterns of Adult Patients to a Sickle Cell Disease Specialty Clinic within a Statewide Sickle Cell Network

Background: Sickle cell disease (SCD) is the most common inherited blood disorder in the United States (U.S.). Despite advances in the treatment for children with SCD, care for affected adults has not significantly improved and is often inaccessible. Difficulties with access to care for adults with SCD in the US is multifactorial and includes paucity of trained, specialized physicians, limited physical access to specialty centers, lack of health insurance/health care coverage, and lack of SCD-education by other medical providers. To address these issues, we have established a state-wide sickle cell disease network to improve access to care for affect adults in South Carolina. This network, (SC)2, Sickle Cell-South Carolina is working to enhance access to care and to examine ongoing barriers to care for adults with SCD. The “hub” of (SC)2 is located at the Medical University of South Carolina (MUSC); specific outside hospitals have been identified as “spokes” within the state to form the network. Clinics are either established or being established at each spoke and additional education is being given through tele-mentoring. Current knowledge of referral patterns for patients with SCD is limited. Information on the mechanism of referral and the coordination of the referral process will ultimately help to eliminate obstacles to appropriate and timely patient referrals and help develop patient engagement.Methods: This is retrospective review of patients >18 years of age referred as “new patients” to the Lifespan Comprehensive Sickle Cell Center at MUSC from October 2015-July 2017. Patients were identified through a chart review of the (SC)2 database including referral notes, telephone calls, and initial patient visits. Data collected included the age of the individual, location (within the state) in which the individual resides, presence or absence of SCD, SCD phenotype, referral source, hematologic care within the prior two years, and the consistency of follow-up after the initial visit to the Sickle Cell Center.Results: During this period, 130 new adult patients were referred to the Lifespan Sickle Cell Center at MUSC. Patients referred for care ranged in age from 18-67 (median 34.5 years) of age and 33% (34/104) were male. To be included in the results, these individuals could not have been seen be the Lifespan Sickle Cell Center within the prior three years so that patients transitioned within the center were not included. Of those patients referred during this period, 85% percent (110/130) were successfully seen at the Lifespan Sickle Cell Center for (at least) an initial visit. However, 40% of these individuals missed their first new patient visit at the Sickle Cell Center and required additional outreach in order to complete that first visit. Of those patients successfully seen for a new patient visit, 95% were confirmed to have SCD (6 individuals were diagnosed with sickle cell trait or did not have a hemoglobinopathy). Only 1/3 of the patients referred to the Sickle Cell Center had seen a hematologist in the last 2 years. The majority of the remaining affected patients had not seen a hematologist since they were children or had never seen a hematologist in their lifetime. Referral sources included primary care physicians, subspecialists, (SC)2 affiliated hospitals (spokes), non-affiliated emergency departments, community based organizations and also included family members and peers. Most of the new referrals were from outside of the Tricounty (local) area (75%) and region did not affect the likelihood of successfully establishing care.Conclusion: The development of a statewide network (SC)2 is an effective initial strategy to facilitate referrals to a SCD center for adults with SCD. Physician-mediated engagement and outreach was key for introducing the (SC)2 network to the state and enabling referrals. When patients missed their initial new visit appointments, aggressive follow-up and trouble-shooting was key to ensuring a successful visit. Additional examination of patient-specific barriers at each phase of the referral process will be key to understanding discrepancies in follow-up rates and obstacles in the referral process. The process through which patients are referred to the center and arrive for their first visit are determinants in their future medical care. Enhanced implementation of this referral process is key to the success of the (SC)2 network. DisclosuresKanter:GBT: Research Funding; American Society of Hematology (Sickle Cell Disease Guideline Panel): Membership on an entity's Board of Directors or advisory committees; MUSC: Other: The site PI for sponsored research conducted at MUSC who receives funds from: Novartis, bluebird bio, GBT, Sancillo, Apopharma, Pfizer; Pfizer: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; NHLBI (sickle cell disease research advisory committee): Membership on an entity's Board of Directors or advisory committees, Research Funding; Apopharma: Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees; Bluebird Bio: Membership on an entity's Board of Directors or advisory committees, Research Funding; Sancillo: Research Funding.