Outcomes of Fever in Infants and Young Children with SCD Presenting to the Emergency Room

Abstract The introduction of antibiotic prophylaxis and vaccinations has reduced the incidence of bacteremia and sepsis in pediatric patients with sickle cell disease (SCD). Due to concern of mortality from sepsis, SCD patients with fever require admission for IV antibiotics until bacteremia is ruled out. The 2014 NIH Evidence-Based Management of Sickle Cell Disease guidelines recommends hospitalization for patients with temperature ≥ 39.5 C and who appear ill. Prior pediatric research has defined "high risk patients" as those with temperature ≥ 40 C, WBC >30,000 or <5,000/mcl, appear ill, or have pulmonary infiltrates on chest x-ray (CXR). (Wilimas NEJM 1993) We reviewed all Emergency Room (ER) visits for febrile patients <6 years of age with HbSS or HbSB0 thalassemia to evaluate these predictive models for bacteremia. Methods: In a 16 year IRB approved cohort, we identified 609 ER visits to Children's of Alabama for fever among 169 children < 6 years old with HbSS or SB0 thalassemia. All patients receive standard of care penicillin prophylaxis and vaccination. We reviewed every blood culture obtained during their ER visit and admission to determine the incidence of bacteremia including pneumococcal bacteremia. We recorded vital signs, blood counts, and CXR findings during the ER visit. We compared differences in these variables among patients with and without bacteremia. We created categorical variables (yes/no) to evaluate NIH fever guidelines (Temp >39.5C and ill appearance) and "high risk patient" recommendations (Temp >40C, ill appearance, abnormal white blood cell count (>30,000 or <5,000//mcl), or pulmonary infiltrates on CXR). Descriptive statistics, t-test for normally distributed data and Wilcoxon for non-normally distributed data, and Fisher's exact test were performed in JMP12. Sensitivity and specificity were calculated from 2x2 tables. To examine the predictive models for bacteremia, we utilized multiple logistic regression to develop the receiving operating characteristics curves and area under the curve (AUC). Results: Among the 169 patients (0-5.99yrs) with HbSS or SB0 thalassemia that were evaluated in the Children's of Alabama ER for fever, 95 (56%) were female. Five hundred and twelve (84%) admissions were identified among the 609 ER visits including all patients with bacteremia. Fourteen patients (2.3%) evaluated in the ER for fever were subsequently diagnosed with bacteremia including 9 (1.5%) positive for pneumococcus. The incidence of bacteremia among young patients presenting to the ER for fever was 1.4 events per 100 patient years and the rate of pneumococcus was 0.9 events per 100 patient years. Patients with bacteremia had higher WBC (27.0 ±7.8 vs 17.2 ±8.5, p<0.0001) than patients without bacteremia. No statistical differences were noted for patients with and without bacteremia for temperature (p=0.06) or heart rate (p=0.3). The sensitivity and specificity of individual variables for bacteremia were: Temp ≥ 39.5 (Sen: 57%, Specificity: 65%), Temp ≥ 40 (sensitivity: 29%, specificity 90%), Ill appearing (sensitivity: 43%, specificity: 86%), abnormal WBC (sensitivity: 36%, specificity 91%) abnormal CXR (appearing (sensitivity: 57%, specificity: 72%). To evaluate models for bacteremia, the AUC for NIH admission guidelines and "high risk patients" were 0.68 and 0.80 respectively. Conclusion: While the incidence of bacteremia is low, young children with SCD are frequently admitted for IV antibiotics until bacteremia is ruled out. Our data suggests using the "high risk model" for admission criteria in febrile children with SCD. Developing models that can accurately predict bacteremia are limited due to the low incidence of bacteremia. Disclosures Lebensburger: NHLBI: Research Funding; American Society of Hematology, Scholar Award: Research Funding.

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Outcome of Severe Vaso-Occlusive Crisis in Sickle Cell Disease Adults Admitted to Referral Centers in Africa and Europe. Introduction of Machine Learning Methods to Improve the Presev Score

Blood ◽

10.1182/blood-2020-143059 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 14-15

Author(s):

Yamna Ouchtar ◽

Christian Kassasseya ◽

Kene Sekou ◽

Anne-Laure Pham Hung D'Alexandry D'Orengiani ◽

Mehdi Kellaf ◽

...

Keyword(s):

Machine Learning ◽

Sickle Cell Disease ◽

Decision Tree ◽

Predictive Value ◽

Research Funding ◽

Low Risk ◽

Cell Disease ◽

Risk Patients ◽

Decision Tree Method ◽

Tree Method

Introduction: Sickle Cell Disease (SCD) is one of the most common genetic disease worldwide. The Acute Chest Syndrome (ACS) is a leading cause of death for SCD patients. The PRESEV1 study was set to produce a predictive score to assess the risk of an ACS development (Bartolucci et al., 2016). PRESEV2 was an international, multicenter prospective confirmatory study to validate the PRESEV score. This study aims at improving these predictions with the addition of a machine learning (ML) method. Patients and methods: Included patients follow PRESEV1 and PRESEV2 studies 'rules. The dataset thus contains 97 patients who developed an ACS episode (18.3%) against 434 patients who did not (81.7%). To compute the PRESEV score, we firstly used the method developed previously with the following variables as input: leukocytes, reticulocytes, hemoglobin levels and cervical spine pain. This method is based on a decision tree with fixed rules and is referred to as the decision tree method throughout this abstract. Secondly we used a ML method using a combined sampling method named SMOTEENN to balance the data and a C-Support Vector Classification (SVC) with fixed parameters to predict the score. This method produces a probability, with a threshold of 0.2, under which the patient is predicted to declare an ACS. We considered the dataset composed of PRESEV1 dataset and 80 percent of PRESEV2 with a randomly choice. The test dataset is thus composed of the remaining 20 percent of PRESEV2. This technique of random choice allowed us to use a 50-cross-validation and compute with Python an average score and a standard deviation (std). In order to allow comparison of the developed score with or without the addition of the ML method, rates were calculated by adding the weight of ACS representation in the dataset. Results: Among all parameters analyzed, the SVC method considered the following variables for calculation of the score: leukocytes, LDH, urea, reticulocytes and hemoglobin levels. A hundred and two adult patients with a severe VOC requiring hospitalization were included. Out of this pool of patients, 26 (25.5%) were predicted with a low risk of developing an ACS episode (SVC method). Sensibility and specificity were of 94.7% and 26.8%, respectfully. The negative predictive value (NPV) was of 95.8% and the positive predictive value (PPV) of 22.4%. Results are resumed in table 1. When compared to the PRESEV score (decision tree method), 44 patients out of 372 were identified with a low risk score (11.8%), Discussion and Conclusion: While the addition of a ML method did not allow the improvement of the sensibility or the NPV of the PRESEV score, it improved both the specificity and the PPV. The addition of artificial intelligence thus provides a better prediction with a higher percentage of "low-risk" patients. As highlighted in the international PRESEV study, this score could represent a useful tool for physicians in hospital settings, with limited beds. While the PRESEV score could allow a better management of "low risk" patients on one side, the identification of "high-risk" patients could also represent a serious advantage to physicians, as it could improve the feasibility of clinical trials for the prevention of this lethal complication in SCD patients. Disclosures Bartolucci: Innovhem: Other; Novartis: Research Funding; Roche: Consultancy; Bluebird: Consultancy; Emmaus: Consultancy; Bluebird: Research Funding; Addmedica: Research Funding; AGIOS: Consultancy; Fabre Foundation: Research Funding; Novartis: Consultancy; ADDMEDICA: Consultancy; HEMANEXT: Consultancy; GBT: Consultancy.

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A Sickle Pickle

10.1093/med/9780190495541.003.0029 ◽

2016 ◽

Author(s):

Ji Y. Chong ◽

Michael P. Lerario

Keyword(s):

High Risk ◽

Sickle Cell Disease ◽

Sickle Cell ◽

Exchange Transfusion ◽

Stroke Risk ◽

Large Vessel ◽

Cell Disease ◽

Safety And Efficacy ◽

Risk Patients ◽

Iv Tpa

Sickle cell disease may result in large vessel intracranial stenoses, which cause high rates of stroke. Screening for elevated velocities on transcranial Dopplers is a good way to stratify stroke risk. Patients at high stroke risk should participate in an exchange transfusion program indefinitely to reduce the rate of subsequent stroke. Although there is a high risk of stroke in pediatric sickle cell patients, the use of IV tPA in this population is largely unstudied and not routinely recommended due to unclear safety and efficacy.

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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

Blood ◽

10.1182/blood-2021-151771 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 1889-1889

Author(s):

Sherraine Della-Moretta ◽

Rui Li ◽

David Way ◽

Michael G (MD) Purcell ◽

Melanie Heinlein ◽

...

Keyword(s):

Sickle Cell Disease ◽

Emergency Room ◽

Sickle Cell ◽

Research Funding ◽

Treatment Decision ◽

Treatment Decisions ◽

Cell Disease ◽

Satisfaction With Treatment ◽

Pain Crisis ◽

The Relationship

Abstract Background Sickle 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). Methods The 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. Results Surveys 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. Discussion The 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. Figure 1 Figure 1. Disclosures Desai: 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.

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Adherence to Iron Chelation Therapy and Associated Healthcare Resource Utilization and Costs in Medicaid Patients with Sickle Cell Disease

Blood ◽

10.1182/blood.v124.21.2177.2177 ◽

2014 ◽

Vol 124 (21) ◽

pp. 2177-2177

Author(s):

Francis Vekeman ◽

Medha Sasane ◽

Wendy Y Cheng ◽

Agnihotram V Ramanakumar ◽

Jonathan Fortier ◽

...

Keyword(s):

Sickle Cell Disease ◽

Emergency Room ◽

Resource Utilization ◽

Sickle Cell ◽

Research Funding ◽

Chelation Therapy ◽

Iron Chelation ◽

Cost Of Care ◽

Iron Chelation Therapy ◽

Cell Disease

Abstract Introduction: While adherence to iron chelation therapy (ICT) is critical for successful iron overload (IO) treatment among patients with sickle cell disease (SCD), published data indicate it is often suboptimal. Deferoxamine (DFO) and Deferasirox (DFX) are ICTs indicated for the treatment of chronic IO in patients with SCD. Lack of patient adherence may impact patient outcomes and increase cost of care. This study evaluated the economic burden of ICT non-adherence in patients with SCD from the state Medicaid program’s perspective. Methods: Patients with SCD were identified from Florida, Iowa, Kansas, Mississippi, Missouri, and New Jersey (1997-2013) state Medicaid programs. Patients were required to have ≥1 ICD-9 diagnosis code for SCD (282.6), ≥1 prescription for DFO or DFX, and ≥6 months of continuous enrollment prior to the 1st DFO/DFX prescription (index date), which was defined as the baseline period. Adherence was estimated using the medication possession ratio (MPR), defined as the sum of the days of medication supply divided by the number of days between 1st and last prescription fill plus the days of supply of the last fill; a threshold of ≥0.80 was used to define optimal adherence. All-cause and SCD-specific resource utilization per-patient-per-month (PPPM) was assessed using cumulative rates, accounting for all visits observed, and compared between adherent and non-adherent patients using cumulative rate ratios (CRR). All-cause and SCD-specific healthcare costs were computed using mean cost PPPM. Regression models adjusting for baseline characteristics were used to assess resource utilization and cost differences between adherent and non-adherent patients. Results: A total of 846 eligible patients with SCD were included with 77 in DFO-only, 686 in DFX-only), and 83 in DFO/DFX switch cohort. Mean (SD) MPR was 0.68 (0.27) for DFO-only patients and 0.75 (0.26) for DFX-only patients (p<0.05). Among all users of ICT, 409 (48.3%) were considered adherent. Adherent patients were slightly younger (19 vs. 21 years, p=0.003) than non-adherent patients. Rates of transfusions were comparable between the two groups (mean [SD] transfusions PPPM, adherent: 0.41 [0.47]; non-adherent: 0.40 [0.54], p=0.456) at baseline. The adjusted rate of all-cause IP visits PPPM was lower in adherent versus non-adherent patients (CRR=0.87 [95% CI: 0.83, 0.91]; p<0.001). The adjusted rates of all-cause outpatient (OP) visits (1.10 [1.08, 1.13], p<0.001) and ER visits (1.06 [1.01, 1.10], p=0.010) PPPM of adherent patients were higher in adherent patients than those in non-adherent patients. A similar trend was observed in SCD-specific resource utilization except for rates of ER visits, which were similar between cohorts. From cost perspective, total all-cause and SCD-specific costs were lower in adherent versus non-adherent patients primarily due to lower IP costs (Table 1). SCD-specific ER and OP costs were similar in both cohorts. All-cause pharmacy costs were higher in adherent versus non-adherent patients. Conclusion: Published studies have reported low adherence to ICT, and a similar trend was found in this study. Adherent patients were observed to have less frequent hospitalizations and lower overall and SCD-specific IP costs compared to non-adherent patients. It should be noted that the rate of OP visits was higher in the adherent patients compared to non-adherent patients suggesting that adherent patients may be more closely monitored potentially resulting in better overall patient management and fewer hospitalizations. Additional analyses are needed to explore differences between adherent and non-adherent patients. Table 1 Costs PPPM Adherent patients (N=409) [A] Non-adherent patients (N=437) [B] Adjusted cost difference[A] – [B] P -value All-cause, mean [SD] $4,766 [$4,388] $5,304 [$4,725] -$724 0.072 Inpatient $1,911 [$3,647] $2,996 [$4,439] -$947 0.016 Emergency room $27 [$87] $40 [$88] -$203 0.104 Outpatient $580 [$697] $485 [$617] $49 0.500 Pharmacy $2,248 [$1,949] $1,783 [$1,449] $432 0.004 Pharmacy without ICT $215 [$482] $274 [$544] -$50 0.192 SCD-specific, mean [SD] $2,237 [$3,679] $3,116 [$4,301] -$952 0.0160 Inpatient $1,776 [$3,546] $2,782 [$4,268] -$855 0.0160 Emergency room $18 [$63] $28 [$69] -$199 0.1200 Outpatient $443 [$658] $306 [$548] $105 0.1120 Disclosures Vekeman: Novartis Pharmaceuticals: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Cheng:Novartis Pharmaceuticals: Research Funding. Ramanakumar:Novartis Pharmaceuticals: Research Funding. Fortier:Novartis Pharmaceuticals: Research Funding. Duh:Novartis Pharmaceuticals: Research Funding. Paley:Novartis Pharma: Employment. Adams-Graves:Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau.

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The Telemedicine Experience for Individuals with Sickle Cell Disease

Blood ◽

10.1182/blood-2021-152870 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 1893-1893

Author(s):

Sara Weiss ◽

Sejean Yang ◽

Shu Zhang ◽

Mandy David ◽

Sophie M. Lanzkron ◽

...

Keyword(s):

Sickle Cell Disease ◽

Emergency Room ◽

Medical Care ◽

Sickle Cell ◽

Research Funding ◽

Physical Exam ◽

Cell Disease ◽

Receive Treatment ◽

The Impact ◽

To Receive

Abstract Introduction Individuals 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. Objective The 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. Methods Patients 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. Results Overall, 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 being taken or there's not the personal touch and stuff". Conclusion The 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. Disclosures Lanzkron: 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.

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A Pilot Study of Parent Education Intervention Improves Early Childhood Development Among Toddlers with Sickle Cell Disease

Blood ◽

10.1182/blood.v126.23.527.527 ◽

2015 ◽

Vol 126 (23) ◽

pp. 527-527 ◽

Cited By ~ 1

Author(s):

Melanie E. Fields ◽

Regina Abel ◽

Sara K. Vesely ◽

Catherine Hoyt Drazen ◽

Allison King

Keyword(s):

Pilot Study ◽

High Risk ◽

Sickle Cell Disease ◽

Young Children ◽

Parent Education ◽

Sickle Cell ◽

Expressive Language ◽

Developmental Outcomes ◽

Cell Disease ◽

Over Time

Abstract Background: Young children with sickle cell disease (SCD) are at high risk of cognitive delay. In a previous study, >50% of the infants and toddlers in our SCD clinic were significantly delayed (scaled scores 7 or below) in cognitive and language skills. These developmental delays were related to the home environment, but not to biologic risk factors associated with SCD. We hypothesized that a parent education program to encourage positive parent-child interactions would improve developmental outcomes. Methods: Children with SCD, ages 1 - 36 months, living in stable housing within 30 miles of the hospital and with caregivers who spoke fluent English were enrolled over a three-year period in a pilot study of a single-arm behavioral intervention. Caregivers consented to participate in an accredited Parents as Teachers (PAT) Born to Learn curriculum, and to complete Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and the Home Observation for Measurement of the Environment (HOME) assessments. An occupational therapist, certified as a PAT provider, completed all services and assessments. Home visits were scheduled monthly, but participants could cancel or reschedule at their discretion. Participation continued until the child was older than 3.5 years, or the parent chose to exit, or at the end of the three-year pilot period. SCD measures were extracted from medical records upon enrollment in the study. Analyses were conducted using IBM SPSS Statistics (Version 22, Chicago, IL). Continuous data were analyzed using the Wilcoxon signed ranks test for comparisons over time. Results: Thirty-five participants (20 male; 16 HbSS, 15 HbSC, 1 HbS beta thalassemia0, 1 HbS beta thalassemia+, 2 HbS with hereditary persistence of fetal hemoglobin) had at least 2 PAT visits with BSID-III assessments. The mean ages of children was 7.9 + 6 months (range, 1-34 months) and of primary caregivers was 25.2 + 5 years (range, 15-35). At baseline, the children's hematocrit was 28.0 + 3.5% (range, 22-39) and their pulse oximetry was 99 + 1.3% (range, 96-100). The median number of home visits was 12 (range, 2-41) over a median of 18 months (range, 2-32). None of the biological variables significantly correlated with the BSID-III domains, but the HOME scores correlated with cognition (r=.410, p=.014). While there was no significant difference in the pre/post HOME scores over time (pre: 29.2, post: 31.3, n=14; p=.161), statistically significant improvements occurred in the cognitive (p=.016) and expressive language domains (p=.002). Table I provides longitudinal BSID-III results. Table 1. BSID-III Scores of Toddlers with SCD at Entry and Exit to Parent Education Intervention. % with scaled scores < 7 Mean Mean change ± SD Gross Motor Time 1 32.6 8.7 0.7 + 3.0 Exit 11.4 9.5 Fine Motor Time 1 31.7 8.0 1.0 + 3.9 Exit 28.6 8.8 Cognitive Time 1 51.2 7.3 1.4 + 3.4* Exit 29.4 8.8 Receptive Language Time 1 44.2 7.6 0.9 + 3.9 Exit 38.2 8.6 Expressive Language Time 1 58.1 6.9 1.9 + 3.0* Exit 26.5 8.7 *= p < .05. Conclusion: Young children with SCD are at high risk for poor developmental outcomes. This is the first parenting intervention to specifically target parents of young children with SCD, and significant improvements occurred in the children's cognitive and expressive language domains. Further investigation is warranted to test the effect of implementation of this evidence-based intervention in a larger cohort of this vulnerable population. Disclosures Fields: Neurophage: Equity Ownership, Membership on an entity's Board of Directors or advisory committees.

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Five-Year Outcomes of Chronic Red Blood Cell Exchange Transfusion Program for Patients with Sickle Cell Disease, the Experience of University of Nebraska Medical Center

Blood ◽

10.1182/blood-2021-146718 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4988-4988

Author(s):

Omar Abughanimeh ◽

Steven Ebers ◽

Mahammed Khan Suheb ◽

Julie Eclov ◽

Robin High ◽

...

Keyword(s):

Sickle Cell Disease ◽

Blood Cell ◽

Emergency Room ◽

Sickle Cell ◽

Red Blood Cell ◽

Research Funding ◽

Medical Center ◽

Emergency Room Visits ◽

Cell Disease ◽

The Mean

Abstract Background: Red blood cell exchange (RBCX) is an effective therapy in treatment of acute and chronic complications of sickle cell disease (SCD). It involves exchanging patient's red blood cells (RBCs) with donor RBCs to significantly lower hemoglobin S concentration without subjecting the patient to the risk of iron overload. The University of Nebraska Medical Center (UNMC) established a chronic RBCX program in November 2015, which cared for patients with multiple hemoglobinopathies. In this study, we aim to evaluate some of the outcomes of patients with SCD who joined the program. Methods: This is a retrospective study based on review of medical records of patients with sickle cell disease. We reviewed the health records of patients with SCD who were enrolled in the chronic RBCX program between 11/2015-8/2020 at UNMC. We included patients with SCD, regardless of age, who underwent RBCX in the outpatient setting during the study period. Data were collected to assess if RBCX influenced the frequency of SCD crisis, emergency room visits, hospitalizations, and other sickle cell-related complications. Results: A total of 404 sessions of exchange transfusions were performed between November 2015 and August 2020 for 21 patients with SCD. The study included 9 adults (age ≥ 18 years) and 12 children with a median age of 12 years (2-31 years). During the study period, 3 adults left the program due to relocation out of state, patient's preference, or physician's decision. Table 1 summarizes the population demographic. The most common indication for enrollment in the RBCX program was recurrent sickle cell crisis (Figure 1). The mean number of emergency room visits before enrollment in the RBCX program was 22.5 visits (2-62 visits), which reduced after enrollment to 10.4 visits (0-65 visits), with a difference in mean of 12.1 visits (P=0.0021). The mean number of hospital admissions before enrollment in the RBCX program was 13.2 admissions(0-54 admissions), which also reduced to 6.7 admissions (0-50 admissions), with a significant difference in the means equal to 6. 6 admissions (P=0.0013) (Figure 2). Thirteen patients had a baseline ferritin > 500 ng/ml at enrollment; all of them had a decrease in their baseline ferritin during the study, with 4 of them achieving a new baseline < 500 ng/ml. Six patients had pre-existing antibodies at enrollment due to prior alloimmunization; however, no new alloantibodies were noticed after enrollment. The patients without preexisting antibodies were transfused with Rh and Kell matched blood. The patients with pre-existing antibodies were transfused with phenotypically matched blood. Three patients became pregnant during the study period, and their pregnancies were uncomplicated except for one patient with preeclampsia resulting in early delivery. There was no reportable death, acute chest syndrome, or stroke among the patients during the study period. Conclusion Outpatient chronic RBCX demonstrated safety and feasibility in both adults and children. It also showed promising outcomes in terms of reduction of sickle cell complications, number of emergency room visits and hospitalizations. These results can provide the basis for evaluating RBCX in a prospective study to better understand changes in quality of life and clinical outcomes of patients with SCD and limited therapeutic options. Figure 1 Figure 1. Disclosures Gundabolu: Pfizer: Research Funding; Samus Therapeutics: Research Funding; BioMarin Pharmaceuticals: Consultancy; Bristol-Myers Squibb Company: Consultancy; Blueprint Medicines: Consultancy.

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Hydroxyurea and Growth in Young Children With Sickle Cell Disease

PEDIATRICS ◽

10.1542/peds.2014-0917d ◽

2014 ◽

Vol 134 (3) ◽

pp. X5-X5

Keyword(s):

Sickle Cell Disease ◽

Young Children ◽

Sickle Cell ◽

Cell Disease

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Sickle Cell Anemia Presenting with Vaso-Occlusive Pain: Should We Screen for COVID-19?

Dubai Medical Journal ◽

10.1159/000513232 ◽

2021 ◽

pp. 1-4

Author(s):

Mohammad Ali ◽

Lina Okar ◽

Nabil E. Omar ◽

Jabeed Parengal ◽

Ashraf Soliman ◽

...

Keyword(s):

High Risk ◽

Sickle Cell Disease ◽

Sickle Cell ◽

Signs And Symptoms ◽

Risk Groups ◽

Position Statement ◽

Interesting Case ◽

International Federation ◽

Cell Disease ◽

The World

Despite the widespread of coronavirus disease-19 (COVID-19) infection around the world, there are very scarce reported literature about the care of patients with a known diagnosis of hemoglobin disorders such as sickle cell disease (SCD) or thalassemia and confirmed COVID-19 infection. Thalassemia International Federation issued a position statement to include patients with thalassemia and SCD among the high-risk groups of patients. Here, we present an interesting case of a 42-year-old patient know to have SCD presenting with Vaso-occlusive (VOC) pain episode in the absence of COVID-19 signs and symptoms, who tested positive for COVID-19 infection and had a smooth recovery. This case highlights the importance of screening SCD patients presenting with VOC-related events even in the absence of COVID-19 signs and symptoms.

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Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

Blood ◽

10.1182/blood.v126.23.68.68 ◽

2015 ◽

Vol 126 (23) ◽

pp. 68-68 ◽

Cited By ~ 3

Author(s):

Janet L. Kwiatkowski ◽

Julie Kanter ◽

Heather J. Fullerton ◽

Jenifer Voeks ◽

Ellen Debenham ◽

...

Keyword(s):

Ischemic Stroke ◽

High Risk ◽

Sickle Cell Disease ◽

Sickle Cell ◽

Sickle Cell Anemia ◽

Stroke Prevention ◽

Cell Disease ◽

Clinical Series ◽

The Mean

Abstract Background: The Stroke Prevention Trial in Sickle Cell Anemia (STOP) and Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) established routine transcranial Doppler ultrasound (TCD) screening with indefinite chronic red cell transfusions (CRCT) for children with abnormal TCD as standard of care. To identify children at high-risk of stroke, annual TCD screening is recommended from ages 2 to 16 years, with more frequent monitoring if the result is not normal. A reduction in stroke incidence in children with SCD has been reported in several clinical series and analyses utilizing large hospital databases when comparing rates before and after the publication of the STOP study in 1998. We sought to determine the rate of first ischemic stroke in a multicenter cohort of children who had previously participated in the STOP and/or STOP 2 trials and to determine whether these strokes were screening or treatment failures. Subjects and Methods: Between 1995 and 2005, STOP and STOP 2 (STOP/2) were conducted at 26 sites in the US and Canada. These studies included 3,835 children, ages 2 to 16 y with SCD type SS or S-beta-0-thalassemia. Participation in STOP/2 ranged from a single screening TCD to randomization. STOP 2 also had an observational arm for children on CRCT for abnormal TCD whose TCD had not reverted to normal. The Post-STOP study was designed to follow-up the outcomes of children who participated in one or both trials. 19 of the 26 original study sites participated in Post-STOP, contributing a total of 3,539 (92%) of the STOP/2 subjects. After exit from STOP/2, these children received TCD screening and treatment according to local practices. Data abstractors visited each clinical site and obtained retrospective data from STOP/2 study exit to 2012-2014 (depending on site) including follow-up TCD and brain imaging results, clinical information, and laboratory results. Two vascular neurologists, blinded to STOP/2 status and prior TCD and neuroimaging results, reviewed source records to confirm all ischemic strokes, defined as a symptomatic cerebral infarction; discordant opinions were resolved through discussion. For the first Post-STOP ischemic stroke, prior TCD result and treatment history subsequently were analyzed. Results: Of the 3,539 subjects, follow-up data were available for 2,850 (81%). Twelve children who had a stroke during STOP or STOP2 were excluded from these analyses resulting in data on 2,838 subjects. The mean age at the start of Post-STOP was 10.5 y and mean duration of follow-up after exiting STOP/2 was 9.1 y. A total of 69 first ischemic strokes occurred in the Post-STOP observation period (incidence 0.27 per 100 pt years). The mean age at time of stroke was 14.4±6.2 (median 13.8, range 3.5-28.9) y. Twenty-five of the 69 patients (36%) had documented abnormal TCD (STOP/2 or Post-STOP) prior to the stroke; 15 (60%) were receiving CRCT and 9 (36%) were not (treatment data not available for 1 subject). Among the 44 subjects without documented abnormal TCD, 29 (66%) had not had TCD re-screen in the Post-STOP period prior to the event; 7 of these 29 (24%) were 16 y or older at the start of Post-STOP, which is beyond the recommended screening age. Four of the 44 (9%) patients had inadequate TCD in Post-STOP (1 to 10.7 y prior to event). Six (14%) had normal TCD more than a year before the event (1.2 - 4 y); all but one of these children were younger than 16 y at the time of that TCD. Only 5 (11%) had a documented normal TCD less than 1 year prior to the event. Conclusions: In the Post-STOP era, the rate of first ischemic stroke was substantially lower than that reported in the Cooperative Study of Sickle Cell Disease, prior to implementation of TCD screening. Many (39%) of the Post-STOP ischemic strokes were associated with a failure to re-screen according to current guidelines, while only 11% occurred in children who had had recent low-risk TCD. Among those known to be at high risk prior to stroke, treatment refusal or inadequate treatment may have contributed. While TCD screening and treatment are effective at reducing ischemic stroke in clinical practice, significant gaps in screening and treatment, even at sites experienced in the STOP protocol, remain to be addressed. Closing these gaps should provide yet further reduction of ischemic stroke in SCD. Disclosures No relevant conflicts of interest to declare.

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