scholarly journals The Telemedicine Experience for Individuals with Sickle Cell Disease

Blood ◽  
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.

scholarly journals Implementation and Preliminary Effectiveness of mHealth Apps for Improving Sickle Cell Disease Care during COVID-19: A Mixed-Methods Evaluation

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3038-3038
Author(s):  
Sherif M. Badawy ◽  
Lisa DiMartino ◽  
Donald Brambilla ◽  
Ana Baumann ◽  
Ebony Burns ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Health Inequities ◽  
Cell Disease ◽  
Structured Interviews ◽  
Research Staff ◽  
Blood Disorder ◽  
The Impact ◽  
Mhealth Apps

Abstract Background: Sickle cell disease (SCD) is a chronic blood disorder, which disproportionately impacts Black individuals. Hydroxyurea therapy prevents SCD-related complications; yet it is underutilized, which contributes to further health inequities. Mobile health (mHealth) apps for patients and providers have the potential to improve hydroxyurea adherence. Our objective was to use the RE-AIM framework to evaluate the impact of the COVID-19 pandemic on implementation and effectiveness of patient and provider hydroxyurea (HU) mHealth apps (InCharge Health app and HU Toolbox app, respectively) in two large academic medical centers. Methods: Patient-level data (n=46) were collected between 11/2019-7/2020. Adherence was measured by calculating Percentage of Days Covered (PDC) from prescription records over 24 weeks before implementing the mHealth apps and during 12 and 24 weeks of implementation. As a response to the COVID-19 pandemic and to reduce virus spread, both sites temporarily suspended non-emergent clinical activities. During implementation, Site A clinics shut down for approximately 2 months (3/2020-5/2020) and Site B clinics for 7 months (3/2020-10/2020). To better understand contextual factors associated with mHealth implementation, we purposively sampled participants according to app use level (high vs low) and conducted semi-structured interviews with adult SCD patients, providers (physicians, nurse practitioners, and physician assistants), administrators, and research staff between 6/2020 and 3/2021. Results: A total of 46 patients participated and contributed to the PDC hydroxyurea adherence data. Mean change in PDC, adjusted for baseline PDC, was greater at Site A than at Site B (12-weeks: difference = 16.59%, p=.01, Figure 1A; 24-weeks: difference = 15.08%, p=.01, Figure 1B). Eleven patients (mean age 26.2 years old, 64% males, 100% Black, 73% HbSS, 45% low app users) and 11 providers (mean age 36.7 years old, 73% females, 36% Black, 54.5% physicians, average 8 years in practice, 36% low app users) completed the semi-structured interviews across the 2 sites. Site B was more affected during the COVID-19 pandemic where patients had difficulty obtaining hydroxyurea and other challenges related to reaching their providers and clinic setting for non-urgent or emergent reasons. In addition, participants with lower baseline hydroxyurea adherence level, as measured by PDC, had a more remarkable improvement in their PDC values at 12 weeks (Figure 1C) and 24 weeks (Figure 1D) Additional qualitative data focused on the implementation process were collected from 3 administrators, and 4 research staff. Among patients, both high and low app users reported the pandemic was a barrier to getting needed care (e.g., difficulty getting to hospital/clinic) and obtaining hydroxyurea; this was particularly a concern among low app users at Site B. Among providers, all but 2 high app users reported the pandemic did not impact app use, but nearly all low users perceived the pandemic to be a barrier to using the provider app because fewer patients came to clinic for maintenance SCD visits during the COVID-19 pandemic. Low users also reported the pandemic would negatively impact continued use of the app. Administrators and research staff also said reduced in-person clinic visits were a barrier to app implementation. Conclusions: mHealth apps are promising tools for improving hydroxyurea adherence among adolescents and adults with SCD. Contextual data show that some patients who experienced challenges accessing healthcare during COVID-19 pandemic have also experienced challenges navigating mHealth implementation. A focus on removing barriers to mobile apps use during care disruptions will likely improve patient and provider mHealth apps implementation, and ultimately, reduce health inequities for this vulnerable population. Our findings suggest that strategies such as including an mHealth facilitator has the potential to help addressing some of these implementation challenges. Note: Sherif Badawy and Lisa DiMartino are co-first authors with equal contribution Figure 1 Figure 1. Disclosures Badawy: Sanofi Genzyme: Consultancy; Bluebird Bio Inc: Consultancy; Vertex Pharmaceuticals Inc: Consultancy. Shah: Alexion: Speakers Bureau; Bluebird Bio: Consultancy; CSL Behring: Consultancy; Emmaus: Consultancy; GBT: Consultancy, Research Funding, Speakers Bureau; GLG: Consultancy; Guidepoint Global: Consultancy; Novartis: Research Funding, Speakers Bureau. Hankins: Bluebird Bio: Consultancy; UpToDate: Consultancy; Vindico Medical Education: Consultancy; Global Blood Therapeutics: Consultancy.

scholarly journals Using Project Echo Telementoring to Improve Sickle Cell Disease Care in the Midwest

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5923-5923
Author(s):  
Lisa Marie Shook ◽  
Christina Bennett Farrell ◽  
Karen A. Kalinyak ◽  
Stephen C Nelson ◽  
Brandon M. Hardesty ◽  
...  
Keyword(s):  
Primary Care ◽  
Sickle Cell Disease ◽  
Medical Care ◽  
Sickle Cell ◽  
Research Funding ◽  
Primary Care Providers ◽  
Healthcare Outcomes ◽  
Care Providers ◽  
Cell Disease ◽  
Project Echo

Abstract Sickle Treatment and Outcomes Research in the Midwest (STORM) is a regional sickle cell network, funded by the Health Resources and Services Administration Treatment Demonstration Project (HRSA U1EMC27863), established to improve outcomes for individuals with sickle cell disease (SCD) living in Indiana, Illinois, Michigan, Minnesota, Ohio and Wisconsin. The STORM network is led by pediatric and adult hematologists who coordinate network activities in each state, along with a Regional Coordinating Center that organizes efforts throughout the Midwest. The goal of the STORM network is to increase the number of pediatric and adult primary care providers (PCP) who are knowledgeable about the management and treatment of SCD, and who are willing to prescribe and manage hydroxyurea therapy as a means to improve medical care for the approximately 15,000 individuals living with SCD in the Midwest. One PCP engagement strategy that has been implemented to increase provider knowledge in the region is replication of the Project ECHOTM (Extension for Community Healthcare Outcomes) telementoring model. Project ECHO was developed by the University of New Mexico to utilize low-cost, high-impact video technology to link expert inter-disciplinary specialist teams with primary care providers to improve management of chronic diseases. This guided practice telementoring model delivers complex specialty medical care to underserved areas, reduces health disparities, and increases workforce capacity. Project ECHO's methodology is based on 1) using telehealth technology to build healthcare resources where they are scarce; 2) sharing best practices to reduce variation in clinical care; 3) utilizing practice-based learning to develop specialty expertise among providers; and 4) monitoring and evaluating provider outcomes. Project ECHO has demonstrated improved healthcare outcomes in Hepatitis C and several other chronic diseases, and is now being piloted by STORM to test its feasibility and applicability for SCD by using a regional approach with CME accreditation. STORM network site physician leads in each state are recruiting multi-disciplinary primary care teams to participate as "spokes" in monthly SCD TeleECHO clinics. The "hub" led by the STORM Regional Coordinating Center, located at Cincinnati Children's Hospital Medical Center, coordinates implementation and evaluation of the telementoring clinics, delivered through monthly teaching sessions. STORM TeleECHO participants log onto an internet-based virtual meeting site, using a webcam to interact during the session. STORM TeleECHO clinics include brief didactic presentations from nationally-recognized SCD content experts with topics and curriculum based on the National Heart Lung and Blood Institute Evidence-Based Management of Sickle Cell Disease guidelines released in 2014. TeleECHO teaching clinics also include 1-2 de-identified, HIPAA protected case discussions (pediatric and adult) presented by providers who would like medical and psychosocial feedback on management of challenging clinical scenarios. Providers participating in the STORM TeleECHO complete an initial survey assessing knowledge and comfort levels, practice behaviors (including hydroxyurea prescribing practices) and clinic demographics. Satisfaction surveys are sent to participants after each session as part of the CME-credit evaluation. Follow-up surveys at 6 months and 1 year will assess satisfaction, knowledge, comfort level and changes in practice. STORM's TeleECHO was launched in March 2016. Preliminary data indicate an interest in STORM TeleECHO teaching sessions by both pediatric and adult providers across the Midwest region. Future efforts will expand the network to more PCPs in the region, while improving the applicability and utility of STORM TeleECHO in SCD through provider assessment. Disclosures Ware: Global Blood Therapeutics: Consultancy; Biomedomics: Research Funding; Bristol Myers Squibb: Research Funding; Addmedica: Research Funding; Nova Laboratories: Consultancy; Bayer Pharmaceuticals: Consultancy.

Economic Burden of Sickle Cell Disease among Children and Adults.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 958-958 ◽  
Author(s):  
Abraham G. Hartzema ◽  
Teresa L. Kauf ◽  
Thomas D. Coates ◽  
Liu Huazhi ◽  
Nakita Mody-Patel
Keyword(s):  
Sickle Cell Disease ◽  
Emergency Room ◽  
Medical Care ◽  
Resource Utilization ◽  
Sickle Cell ◽  
Economic Burden ◽  
Medical Costs ◽  
Emergency Room Visits ◽  
Cell Disease ◽  
The Cost

Abstract Background: The economic burden of sickle cell disease (SCD) has not been determined. Cost-of-care estimates improve health care planning, inform research priorities, and contribute to an understanding of the cost-effectiveness of new SCD treatments. The objectives of this study were examine resource utilization in the Florida Medicaid population; to estimate the cost of medical care for SCD patients; and to examine the proportion of medical care costs attributable to SCD versus other morbidities. Methods: Pediatric (0 – 19 years; y) and adult (> 20 y) patients with a diagnosis of SCD between 1/2001–12/2005 were identified from Florida Medicaid claims. Patients with at least 1 inpatient or 2 outpatient SCD claims at least 30 days apart and ≥6 Medicaid-eligible months, not necessarily consecutive, were included in the analysis. Medicaid administrative data consist of medical and pharmacy utilization and reimbursement claims for medically-needy and low-income recipients. Resource utilization and costs were estimated using paid claims for hospitalizations, emergency room visits, outpatient visits, pharmacy claims, and other care (blood transfusions, hydroxyurea, iron chelation, serum ferritin tests). Claims containing a primary or secondary ICD-9 code indicating SCD were considered “SCD-related”; all other claims were classified as “non-SCD-related”. Individuals aged >65 y and/or dually eligible for Medicare or other health insurance were excluded. Annualized costs were estimated by multiplying mean cost per patient-month by 12. All costs were inflated to 2005 US$ using the medical care component of the Consumer Price Index. Results: A total of 4,294 individuals met study inclusion criteria. Mean age at first claim was 14.5 y (range: 0–64 y; SD 13.2); 55.8% were female; 79.1% African-American. Mean SCD-related costs for pediatric (0–9 y) and adolescent (10–19 y) patients were estimated at $5,292 and $11,508 per year, respectively. Overall, 66% of total medical costs were related to SCD. The distribution of SCD-related costs was bimodal, with the youngest (0–9 y) and oldest (50–64 y) having the lowest proportion of SCD-related costs (53.7% and 35.3%, respectively). Non-SCD-related costs increased with age, ranging from $4,548 annually for ages 0–9 to $16,656 for ages 50–64. While SCD accounted for the largest proportion of total medical costs for patients 10–19 y (77.27%; $11,508), absolute SCD costs were highest for patients 30–39 y (67.1%; $21,792). Cost distributions were similar for males and females. Hospitalizations accounted for 80.5% of all SCD-related costs; emergency room visits, 3.2%; office visits, 0.9%; outpatient drugs, 3.6%; other medical care, 11.7%. Total discounted medical costs (3%) for a SCD patient from birth through age 45 were estimated at $448,460, with 67% of costs related to SCD. Conclusion: Our results suggest that the burden of SCD is substantial. Moreover, non-SCD-related medical costs for SCD patients are considerably higher than expenditures reported for the general US population. While our results reflect the payer’s perspective, the clinical, humanistic, and economic burden of care on individual patients also is likely high. Interventions designed to prevent SCD complications and keep patients out of the hospital may reduce the significant economic burden of the disease.

scholarly journals 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 ◽  
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.

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

Blood ◽  
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.

scholarly journals 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 ◽  
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 &gt; 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 &lt; 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.

scholarly journals COVID Symptoms and COVID Anxiety in Sickle Cell Disease

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 16-17
Author(s):  
Wally R Smith ◽  
Benjamin Jaworowski ◽  
Shirley Johnson ◽  
Thokozeni Lipato ◽  
Daniel M Sop
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Telephone Survey ◽  
Cell Disease ◽  
Weighted Mean ◽  
The Us ◽  
Associated Symptoms ◽  
At Home

Background Even before the US upswing of the current COVID pandemic, the number of sickle cell disease (SCD) patients coming to hospitals and EDs appeared to fall drastically. This happened despite SCD patients having often been heavy utilizers of the ED and hospital for their iconic vaso-occlusive crises (VOC). Though ambulatory SCD clinics quick converted largely to telehealth in order to comply with stay-at-home orders designed to suppress person-to-person transmission, some SCD patients appeared to avoid care, delay care, or refuse doctors' invitations for care. Presumably patients did so out of COVID fears, but this has not been confirmed in the literature. Further, whether these patients had COVID symptoms but stayed at home has not been studied. As part of quality improvement (QI) to conduct COVID surveillance in an adult sickle cell program, we sought to explain and predict SCD health care utilization patterns we were observing, as well as to determine urgent physical and mental health needs of patients who appeared to be avoiding care. Methods Fifteen staff in the Adult Sickle Cell Medical Home at Virginia Commonwealth University, a large urban academic medical center, conducted a telephone survey ("wellness check"was used when we talked to patients) of all known adults with SCD over 19 days in 2020. A staff member confirmed the patient had SCD, asked permission to proceed, then asked about symptoms consistent with COVID-19. At the end of the telephone survey, respondents wer invited to complete an email survey of sickle cell and COVID-19 utilization attitudes (19-33 items, depending on the response pattern, either drawn from the National Health Interview Survey, from the Adult Sickle Cell Quality of Life Measurement quality of care survey, or drafted by the authors), the Sickle Cell Stress Survey-Adult (SCSS-A, a 10-item previously validated survey), and anxiety and depression (PHQ9 of the PRIME-MD). Results Of 622 adults approached by phone call, 353 responded to the following yes/no screening questions regarding the prior 14 days: fever over 100 F 0/353 (0.00%); cough 3/353(0.01%); difficulty breathing 0/353(0.00%); unexplained shortness of breath 2/353(0.01%); sore throat 2/353 (0.01%); unexplained muscle soreness 2/353(0.01%);contact with anyone who tested positive for COVID-19 2/353(0.01%); testing for COVID 19 6/353(0.02%). For QI purposes, we set a threshold of three or more COVID-associated symptoms or the presence of fever as criteria requiring intense telephone or in-person staff monitoring for the following week. Only three patients met criteria. A total of 219/353 had email surveys sent. Of 63 patients (28.8%) who returned email surveys by June 10, 2020, 35.9% had already managed a "pain attack" at home 4 or more times in the prior 12 months, and 45.5% of these said their bad ER experiences were very or somewhat important in that decision. In the prior 14 days, although 30/64 reported a crisis for at least one day, only 4/64 had visited the Emergency Department for pain. On a 0-10 scale, 21/61 patients endorsed "0" for worry that they would be COVID-infected by going for medical care (weighted mean 3.9), but 18/59 endorsed "10" for worry they were more at risk of COVID because of SCD (weighted mean 6.31), and 22/60 endorsed "10" for worry they would fare worse than others if COVID infected (weighted mean 6.97). Many patients forwent "needed" care (16/62) or delayed "needed" care by at least a day (36/61). Eleven patients met criteria for moderately severe to severe depression on the PHQ-9, and 28/63 somewhat or strongly agreed with the statement "death is always on the back of my mind" on the SCSS-A. Conclusions In adolescents and adults with SCD, many were already reticent to come to the ED for pain, but a significant portion reported delays or avoidance of needed care during the early stages of the US COVID pandemic, and few reported using the ED despite over half reporting at least one crisis day in 14. Patients nonetheless reported very few COVID-associated symptoms. Fears of COVID infection/susceptibility may limit visits for needed sickle cell care among adults. Acknowledgements: Mica Ferlis RN, FNP, Caitlin McManus, RN, FNP, Emily Sushko, RN, FNP, Justin West, RN, Kate Osborne, RN, Stefani Vaughan-Sams, Marla Brannon, BS, Nakeiya Williams, BS Disclosures Smith: GlycoMimetics, Inc.: Consultancy; Emmaeus Pharmaceuticals, Inc.: Consultancy; Novartis, Inc.: Consultancy, Other: Investigator, Research Funding; Global Blood Therapeutics, Inc.: Consultancy, Research Funding; Shire, Inc.: Other: Investigator, Research Funding; NHLBI: Research Funding; Patient-Centered Outcomes Research Institute: Other: Investigator, Research Funding; Health Resources and Services Administration: Other: Investigator, Research Funding; Incyte: Other: Investigator; Pfizer: Consultancy; Ironwood: Consultancy; Novo Nordisk: Consultancy; Imara: Research Funding; Shire: Research Funding.

scholarly journals Clinical and Biomarker Predictors for Avascular Necrosis in Sickle Cell Disease

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3091-3091
Author(s):  
Michael Rabaza ◽  
Maria Armila Ruiz ◽  
Liana Posch ◽  
Faiz Ahmed Hussain ◽  
Franklin Njoku ◽  
...  
Keyword(s):  
Risk Factors ◽  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Avascular Necrosis ◽  
Sickle Cell ◽  
Research Funding ◽  
Medical Attention ◽  
Cell Disease ◽  
Advisory Committees ◽  
Early Management

Abstract Introduction Sickle cell disease (SCD) affects 1 in 365 African Americans and approximately 25 million people world-wide. A common skeletal system complication is avascular necrosis (AVN), which can cause substantial pain and a reduced quality of life. While early management of AVN is focused on increasing range of motion with physical therapy and pain relief, there are no clear predictors for who is more likely to develop AVN and earlier institution of these preventive measure could help decrease disease progression. Vascular endothelial growth factor (VEGF) is a biomarker of endothelial injury and may indicate reduced vascular supply to the femoral or humeral head. Here we describe potential risk factors and biologic pathways for AVN in SCD, as understanding these may lead to improvements in future monitoring, early detection, and early intervention practices. Methods We investigated clinical and laboratory risk factors associated with AVN in a cohort of 435 SCD patients from our center. Blood samples, clinical, and laboratory data were collected at the time of enrollment during a clinic visit. Genotyping for alpha thalassemia was performed by PCR and the serum concentration of VEGF was measured by ELISA. AVN status was confirmed by review of the medical record and available imaging. We conducted a cross-sectional analysis comparing categorical and linear variables by AVN status using the chi-square and Kruskal-Wallis test, respectively. The independent association of the clinical and laboratory variables with AVN status was determined by logistic regression analysis. The initial model included variables with a P-value &lt; 0.1 on univariate analysis and the final model was ascertained by stepwise forward and backward selection. Median values and interquartile range (IQR) are provided. Results The median age of the cohort was 32 (IQR, 24 - 43) years, 57% (250/435) were female, and 46% (198/435) were on hydroxyurea. AVN was observed in 34% (149/435) of SCD patients. SCD patients with AVN were older, had more frequent vaso-occlusive crises requiring medical attention, and had a higher body mass index (Table I) (P ≤ 0.002). We measured VEGF in 241 of the SCD patients with serum samples available at the time of enrolment. Serum VEGF concentrations trended higher in SCD patients with versus without AVN (420 vs. 359 pg/mL, respectively; P = 0.078). In the multivariate analysis model, AVN was independently associated with increased number of vaso-occlusive crises (OR 1.1, 95% CI: 1.0 - 1.14; P = 0.02), AST concentration (natural log OR 0.5, 95% CI: 0.2 - 0.9; P = 0.03), VEGF concentration (natural log OR 1.4, 95% CI: 1.0 - 1.9; P = 0.047), and tobacco use (OR 1.9, 95% CI: 0.9 - 3.7; P = 0.078). Discussion In conclusion, we demonstrate a high prevalence of AVN in an adult cohort of SCD patients. The presence of AVN was independently associated with a greater frequency of vaso-occlusive pain episodes, which may demonstrate a shared pathophysiology between AVN and vaso-occlusion that merits further investigation. We demonstrate that serum VEGF concentrations are higher in SCD patients with AVN and may be a clinical tool to identify those at high-risk and for earlier intervention for this complication. Figure 1 Figure 1. Disclosures Gordeuk: Modus Therapeutics: Consultancy; Novartis: Research Funding; Incyte: Research Funding; Emmaus: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; CSL Behring: Consultancy. Saraf: Pfizer: Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding.

scholarly journals Alkaline Phosphatase Is Associated with Red Cell Alloimmunization in the Pulmonary Hypertension and Hypoxic Response (PUSH) Sickle Cell Disease Cohort

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 20-20
Author(s):  
Victoria Brooks ◽  
Oluwalonimi Adebowale ◽  
Victor R. Gordeuk ◽  
Sergei Nekhai ◽  
James G. Taylor
Keyword(s):  
Risk Factors ◽  
Alkaline Phosphatase ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Severe Disease ◽  
Case Control ◽  
Red Cell ◽  
Cell Disease ◽  
History Of

Background: Blood transfusion is a common therapy for sickle cell disease (SCD). Although, highly effective, a major limitation is development of alloantibodies to minor blood group antigens on donor red cells. Alloimmunization has a prevalence of 2-5% for transfusions in the general population, but it is significantly higher in SCD. Risk factors for alloimmunization have been poorly characterized, although number of lifetime transfusions is an important risk factor. Alloimmunization has been clinically observed in children with a prevalence of about 7%. With development of each antibody, blood donor matching becomes increasingly difficult and expensive with an increased risk for transfusion reactions and diminished availability of compatible red cell units for treatment of SCD. The ability to identify risk factors for developing alloantibodies would be beneficial for clinicians. To identify markers for alloimmunization in SCD, we have analyzed children and adults who developed this complication. Methods: We analyzed The Pulmonary Hypertension and Hypoxic Response in Sickle Cell Disease (PUSH) study, which enrolled n=468 pediatric and n=59 adult SCD subjects. In both children and adults, alloimmunization cases were defined as a history of at least 1 alloantibody. Controls in both cohorts were defined as subjects with no history of alloantibodies and receipt of more than 10 lifetime red cell transfusions. All others within the study who did not meet these criteria were assigned to a third comparison group. To identify differences between cases, controls and all others, we performed univariate analyses (using ANOVA or Kruskal Wallace where appropriate) for clinical parameters and laboratories. Case control comparisons were also performed for selected variables and plasma levels for 11 cytokines. Results were further analyzed using regression modeling. Results: The overall prevalence of alloimmunization was 7.3% among children (34/468 subjects; median age 12, range 3-20 years) compared to 28.8% in adults (17/59 subjects; median age 37, range 18-73 years). When only considering those with &gt;10 lifetime transfusions, the prevalence was considerably higher at 29.3% and 54.8% in children and adults, respectively. At the same time, 8 pediatric (23.5%) and 5 adult (29.4%) alloimmunization cases had received fewer than 10 transfusions. In a 3-way pediatric cohort comparison (cases, controls and all others), risk factors associated with alloimmunization included SS genotype, older age and markers of more severe disease (higher ferritin, WBCs, platelets and total bilirubin). Comparison of cases to controls showed alkaline phosphatase (P=0.05) was significantly lower in cases, whereas AST (P=0.02) was significantly higher even with adjustment for age. Levels of plasma cytokines MCP-1 (P=0.01) and IFNgamma (P=0.08) were lower in cases from a subset of the pediatric cohort. In adults, only 4/59 (6.8%) subjects had never received a lifetime transfusion (all non-SS). In the adult 3-way comparisons, only SS genotype and higher ferritin were associated with alloimmunization. The adult case control analysis showed higher absolute monocyte count (P=0.02), absolute eosinophil count (P=0.04) and absolute basophil count (P=0.008) in association with alloimmunization cases. In addition, alkaline phosphatase was again significantly lower among cases (P=0.02) as seen in the pediatric cohort. There were no significant differences in cytokine levels among adults. Conclusions: When considering only transfused SCD patients, the prevalence of alloimmunization is higher than 30%. As seen in prior studies, higher lifetime red cell transfusions are an important risk factor especially among adults where most patients have received transfusions. Children who develop alloantibodies appear to have laboratory markers of more severe disease, but this is not observed in adults. A novel association observed across both pediatric and adult subjects is a significantly lower serum alkaline phosphatase in those with alloantibodies. The results of this study suggest a need for improved tracking of red cell transfusion therapy in the US for SCD patients due to a high prevalence of alloimmunization. Further study is also needed to elucidate the significance of the alkaline phosphatase association. Disclosures Gordeuk: CSL Behring: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; Novartis: Consultancy; Ironwood: Research Funding; Imara: Research Funding.

scholarly journals Sickle Cell Disease Model Mice Lacking 2,3-Dpg Show Reduced RBC Sickling and Improvements in Markers of Hemolytic Anemia

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 27-28
Author(s):  
Kelly M. Knee ◽  
Amey Barakat ◽  
Lindsay Tomlinson ◽  
Lila Ramaiah ◽  
Zane Wenzel ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Blood Cell ◽  
Hemolytic Anemia ◽  
Sickle Cell ◽  
Red Blood Cell ◽  
Organ Damage ◽  
Complete Elimination ◽  
Cell Disease ◽  
The Impact ◽  
2,3 Dpg

Sickle cell disease (SCD) is a severe genetic disorder caused by a mutation in hemoglobin (b6Glu-Val), which allows the mutant hemoglobin to assemble into long polymers when deoxygenated. Over time, these polymers build up and deform red blood cells, leading to hemolytic anemia, vaso-occlusion, and end organ damage. A number of recent therapies for SCD have focused on modulating the mutant hemoglobin directly, however, reduction or elimination of 2,3-DPG to reduce Hb S polymerization and RBC sickling has recently been proposed as a therapeutic strategy for SCD. Current clinical studies focus on activation of pyruvate kinase to reduce 2,3-DPG, however, direct targeting of the enzyme which produces 2,3-DPG; Bisphosphoglycerate Mutase (BPGM) may also be possible. In this study we evaluate the impact of elimination of 2,3-DPG on SCD pathology by complete knockout of BPGM in Townes model mice. Animals with complete knockout of BPGM (BPGM -/-) have no detectable 2,3-DPG, while animals that are heterozygous for BPGM (BPGM -/+) have 2,3-DPG levels comparable to Townes mice. Western Blot analysis confirms that BPGM -/- animals completely lack BPGM, while BPGM -/+ animals have BPGM levels that are nearly equivalent to Townes mice. As expected from the lack of 2,3-DPG, BPGM -/- animals have increased oxygen affinity, observed as a 39% decrease in p50 relative to Townes mice. Complete elimination of 2,3-DPG has significant effects on markers of hemolytic anemia in BPGM -/- mice. Mice lacking 2,3-DPG have a 60% increase in hemoglobin (3.7 g/dL), a 53% increase in red blood cell count, and a 29% increase in hematocrit relative to Townes mice. The BPGM -/- mice also have a 57% decrease in reticulocytes, and a 61% decrease in spleen weight relative to Townes animals, consistent with decreased extramedullary hematopoiesis. Consistent with the reduction in hemolysis, BPGM -/- animals had a 59% reduction in red blood cell sickling under robust hypoxic conditions. BPGM -/+ animals had hemoglobin, RBC, and hematocrit levels that were similar to Townes animals, and a similar degree of RBC sickling to Townes mice. Liver phenotype was similar across all variants, with areas of random necrosis observed in BPGM -/-, BPGM -/+ and Townes mice. Higher percentages of microcytic and/or hyperchromic RBCs were observed in BPGM -/- animals relative to BPGM -/+ or Townes animals. These results suggest that modulation of 2,3-DPG has a positive effect on RBC sickling and hemolytic anemia, which may have therapeutic benefits for SCD patients. However, the lack of improvement in organ damage suggests that modulation of 2,3-DPG alone may not be sufficient for complete elimination of SCD phenotypes, and further investigation of this therapeutic avenue may be necessary. Disclosures No relevant conflicts of interest to declare.

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