Comprehensive Sickle Cell Disease Center Research Articles

Delayed Management of Sickle Cell Disease Vaso-Occlusive Episodes and the Impact on Prolonged Hospitalizations at a Single Academic Center

Introduction: Sickle Cell Disease (SCD) affects nearly 100,000 people in the United States (Centers for Disease Control and Prevention, 2023). Vaso-occlusive episode (VOE) is the most common reason for hospitalization in SCD. Many providers are unfamiliar with the pain management timing and dosages required to adequately treat these episodes (Jang et al., J Transl Med 2021). Nationally, areas with comprehensive sickle cell centers have shown decreased hospital utilization, fewer VOEs, and improved quality of life (Oyedeji et al. JAMA 2020). Even in areas without a comprehensive center, implementing individualized pain care plans for SCD has reduced admission rates of high-utilizing patients while not impacting their pain rating scores (Welch-Coltrane et al. Pain Medicine 2021). Our academic hospital, Oregon Health and Science University (OHSU), lacks a comprehensive SCD center and VOE treatment plans are not easily accessible for these patients. We conducted a retrospective analysis on a cohort of patients with SCD to investigate the factors influencing the length of emergency department (ED) and hospital stays for VOE. We aim to identify if implementing individualized pain plans via a standardized template within the electronic health record (EHR) for these patients will decrease hospital admission, length of stay and time to ED discharge. We also aim to integrate individualized pain plans into the EHR that would allow for direct exposure to the care team by creating plans that are easily accessible, reviewed, and routinely utilized for clinical decision-making. Methods: We performed a preliminary retrospective analysis of encounters (n=107) from 2008-2023 at OHSU, where the primary diagnosis was either a VOE or an equivalent diagnosis. Based on data demonstrating earlier maximum opioid doses leading to shortened hospital stays in frequent pain patients, we recorded the timing of the first opioid and morphine milliequivalents per hour in the first 24 hours (Payne et al. Pediatr Blood Cancer 2018). Additionally, we recorded the length of stay, admission rates, time to first IV fluid, and the type of IV fluid used. We performed a linear regression analysis on the collected data with the dependent variable as the length of stay across the independent variables of time to first opioid, time to first intravenous fluid, and morphine milliequivalents per hour during the first 24 hours after presentation. Results: Eighteen patients accounted for the 107 analyzed encounters. Thirteen patients (72%) had Hgb-SS disease, 2 (11%) had Hgb-SC disease, and 3 (17%) had Hgb-S-beta thalassemia. Sixty-two admissions (58%) were ED discharges, and 45 (42%) were ED to hospital admissions. The length of stay and time to the first opioid positively correlated with p=0.005 (Figure 1a). The length of stay and time to first intravenous fluid administration was also positively correlated with p=0.01. The length of stay and morphine milliequivalents per hour in the first 24 hours were negatively correlated with p<0.0001 (Figure 1b). Conclusions: Longer time to administration of opioids and intravenous fluids correlated with a longer length of stay in both the ED and the hospital. Additionally, lower morphine milliequivalents in the first 24 hours of presentation were associated with a prolonged hospital stay at our institution. We hypothesize that these three factors are secondary to delays in care and inappropriate initial treatment of VOE. To address these factors, we aim to implement standardized pain plans in the EHR of our institution. Implementation of individualized pain plans, particularly in the ED, has led to the earlier initiation of opioids (Della-Moretta et al. Ann Emerg Med 2020). We anticipate similar effectiveness for timely and appropriate fluid administration and adequate opioid dosing. We plan to continue retrospective analysis and compare this data to VOE encounters post-implementation. By implementing individualized plans that are integrated within the EHR and efficiently utilized by the care team, we hope to improve VOE management at our institution with decreased length of stay, less ED to hospital admissions, and improved patient satisfaction.

Sickle Cell Disease Management Practices across Nigeria: A Cross-Sectional Analysis

Introduction Of 300,000 children born with sickle cell disease (SCD) in the world, approximately 75% of these are born in Sub-Saharan Africa. Owing to the frequent painful vaso-occlusive and hemolytic episodes associated with this disease, irreversible multi-organ damage and premature mortality are common. For instance, in Nigeria, 490 deaths per 1000 births among children under 5 have been attributed to SCD; this is four times higher compared to mortality in children born without SCD. On the other hand, those with SCD who survive beyond childhood are faced with an estimated life expectancy of 21 years. To address these challenges, guidelines for SCD management were developed by the Nigerian Federal Health Ministry, National Heart, Lung, and Blood Institute (NHLBI), and the American Society of Hematology but their dissemination and implementation has been difficult. To better understand current management practices, we present a preliminary, descriptive analysis of hydroxyurea and blood transfusion practices for sickle cell disease management across Nigeria. Methods Using a cross-sectional design, we collected demographic and practice-related information from eligible clinicians that provide SCD care in Nigeria. The “SCD Knowledge, Attitudes, and Practices - Nigeria” survey was disseminated to pediatric and adult consultant physicians, resident doctors, and nurses through their respective professional communication platforms. SCD-related practices questions were based on the NHLBI recommendations for SCD management which informed the Nigerian Federal Ministry's National SCD Guideline. A 5-point Likert scale was used to answer each question on hydroxyurea use and blood transfusion. Descriptive analyses of current practices were used to characterize these results and bivariate analyses were conducted using Chi-square or Fisher's test as appropriate. Data analysis was performed using SAS 9.4 (SAS Institute; Cary, NC). Ethical approval was obtained from Duke University Institutional Review Board and the University of Nigeria, College of Medicine Health Research Ethics Committee. Results Analysis of SCD-related practices from the first 73 participants who have completed the survey, revealed most respondents to be female (62%), between 35-65 years old (77%), and adult hematologists or nurses (37% and 40% respectively). This group of clinicians provide care mainly to adults (47%), but 45% admitted providing care to both adults and children. Nearly half (45%) of this group provides care for 0-5 individuals with SCD weekly and 57 participants (78%) work in tertiary-level hospitals. Only 22 of those working in tertiary hospitals had a comprehensive SCD center in their hospital compared to those without an SCD center (39% vs. 61%, p=0.0148). SCD experts were also found in tertiary-level hospitals (75% vs. 25%, p<0.0001). General hematologist expertise was also common in tertiary care (88% vs. 12%, p=0.2424). Figures 1 and 2 report HU and blood transfusion practices. Although 73% confirmed hydroxyurea (HU) prescribing and 90% endorsed the use of simple blood transfusions, HU and blood transfusion practices varied greatly. Respondents indicated most educated patients about HU (frequently/always = 74%) and started HU for patients with >3 VOCs in past year (frequently/always = 62%), however, only 31% frequently/always begin at age 9 months (Figure 1). Most respondents reported prophylactic blood transfusions to reduce HbS below 30% (frequently/always = 71%) and 73% frequently/always performed transfusions for acute SCD complications, yet, 60% indicated they never/rarely match for RBC antigens. Conclusion The data highlights SCD-related expertise and specialty care are found more in tertiary-level hospitals. HU practices described may be attributed to the more conservative approach suggested by the Nigerian SCD Management Guideline compared to the NHLBI recommendations, however there is an important opportunity to expand HU prescribing. Blood transfusion, which has historically been considered difficult in this differently resourced setting, was common. However, chelation, leukoreduction/white blood cell filtration, and red blood cell antigen testing were not common. These preliminary findings represent an opportunity for a larger study and future studies to investigate strategies to increase uptake of existing SCD management recommendations.

Addressing Reproductive Education Among Young Adults with Sickle Cell Disease (SCD): Sickle Cell Reproductive Outreach and Education (ROE) Project

Background: As children with sickle cell disease (SCD) survive into adulthood, it is important to address emerging reproductive health concerns. This population faces unique fertility challenges, including balancing the desire for biological parenthood, risk of pregnancy-associated complications, unintended pregnancy, and fertility concerns with the use of SCD modifying therapies (DMTs) (Smith-Whitley, Blood, 2014, Pecker, Br J Haematol, 2021) . Recent research identified knowledge gaps that impact reproductive care for adults with SCD (Carrithers, Frontiers, 2023). To overcome these gaps, we need to evaluate the baseline knowledge of young adults with SCD regarding reproduction to understand the complex implications of reproductive decision-making. This study was done to gauge young adults' knowledge of reproduction, understanding of fertility, and current reproductive behaviors in order to develop educational material to support young adults' with SCD reproductive decisions. Methods: This IRB-approved, cross-sectional study included young adults living with SCD (ages 18-35) from University of Alabama-Birmingham's Lifespan Comprehensive SCD Center. Patients were recruited during routine clinic visits to complete an electronic 33-question survey to evaluate reproductive health topics adapted from the Sisters Informing Sisters About AIDS (SISTA) questionnaire (CDC, 2008), including demographics (age separated into younger age (18-25) and older age (26-35), sexual health behaviors (contraception, sexually transmitted infection (STI) prevention), family planning, patient advocacy, and attitudes towards reproductive education. Responses were recorded as “Yes, No, Don't Know”, unless questions specifically asked for participants to choose an option. Attitudes toward reproductive education were graded on 5-point Likert scale (1= strongly disagree to 5=strongly agree). Data analysis includes averages to evaluate subgroup comparisons and independent student's t-test to compare subgroups with statistical significance set at p<0.05. Results: We enrolled 47 participants (55% women and 51% were of older age). In this cohort, 87% completed high school and 60% were sexually active. Among those who were sexually active, 86% had previously undergone STI testing and 36% had prior history of STI (22% men vs. 77% women, 22% younger age vs. 77% older age, p<0.05). Thirty-eight percent identified personal or partner contraceptive use. Regarding children, 19% had birthed/fathered a child(ren) (55% men vs. 45% women, 22% younger age vs. 77% older age, p>0.005) among whom 11% knew their child had SCD, 55% knew their child had sickle cell trait (SCT), and 33% did not know if their child had SCD/SCT. Only 26% were aware of contraceptive options for people with SCD (17% men vs. 83% women, p<0.05). Importantly, 89% recognized high risk sexual activity as a risk factor for STIs but no one in the cohort recognized the risk of infertility due to STIs. When asking about their partners, 32% knew if their partner had SCD/SCT (20% men vs. 80% women, p<0.05). Notably, 79% desired biologic children. Among those who desired children, 41% felt that their SCD changed their decision to have children and 26% were previously told they could not have children due to SCD (12% men vs. 88% women, 25% younger age vs. 75% older age, p<0.05). Notably, 57% were interested in conceiving if pregnancy was planned (41% men vs. 59% women, 37% younger age vs. 63% older age, p<0.05). When asked if patients wanted to discuss their reproductive health, 87% wanted to discuss this with their hematologist but only 51% would discuss this with PCPs. Conclusion: In this study, we identified evaluated 4 tenets of reproductive health knowledge: contraceptive use, STI prevention, family planning, and patient advocacy. We identified gaps in knowledge in these areas that can be addressed to improve reproductive education in young adults with SCD. Young adults with SCD in our cohort have low contraceptive use and were often not aware of contraceptive options for people with SCD or their partner's SCD/SCT status. Our cohort also reported a significant history of STI but was unaware of the associated increasing risk for infertility. Importantly. young adults are especially interested in discussing reproductive health with their hematologists, which provides an opportunity to improve reproductive education within this unique population.

Implementation of a Multipronged Intervention to Improvement Inpatient Sickle Cell Disease Care

Introduction: Over the decades, many interventions have been used to improve care for patients with Sickle Cell Disease (SCD). There are 19 whole lifespan SCD centers, 30 pediatric SCD centers, and 28 adult SCD centers compared to 6,129 hospitals and over 220 children's hospitals in the US. The number of providers is insufficient to meet the needs of patients with SCD. Caring for patients without a dedicated Comprehensive SCD Center, such as our institution, can be challenging. Therefore, we developed a framework to leverage expertise in hematology, internal medicine, and emergency medicine to improve SCD patient care processes. In these settings, filling the gap in SCD care requires a multipronged creative approach that is implemented over time in a stepwise fashion. This allows time to adapt the intervention to the local medical system for more enduring change. This project looks at the longitudinal interventions that have occurred at the University of Chicago (UC) to address SCD care and discusses procedures to achieve successful implementation. Methods: In the first step, we identified the key stakeholders to determine the system's needs and goals (Table 1). This involved clinicians and staff who were interested in improving care for patients with SCD. The second step involved stakeholders creating a plan after analyzing the current care. Multipronged interventions were developed and implemented (see Figure 1 for a timeline and list of interventions). The final steps involved provider training. Furthermore, we tracked the progress of our interventions using the Electronic Medical Record (EMR). An EMR review was performed from 7/17/22 to 6/13/23 to evaluate our utilization. Descriptive statistics and Chi-Square were used to evaluate the data. This project was formally determined to be quality improvement and was therefore not overseen by the Institutional Review Board. Results: Regarding the first step, our task force comprises a large multidisciplinary group (see Table 1 for a full list). Next, it was important that we implement sustainable and useful systems (see Figure 1). In the review period, 154 patients were admitted 488 times. The SPP was used or viewed 134 times. Seventy-nine percent (122/154) of admitted patients had an IPP that was used, viewed, or created during their admission. Since the opening of the SCD Day clinic, there have been a total of 1045 SCD-related encounters, with an average of 36 patients a month or 1.7 patients a day. When comparing the review period to the same time in the prior year, the inpatient hydroxyurea protocol did not significantly improve the use of the medication. Hydroxyurea use changed from 64% to 65% of admissions and 65% to 68% of admitted patients. The inpatient SCD consult service received new consult requests in 44% of SCD admissions (214 total times). Lastly, over 100 hospitalists, resident physicians, inpatient teaching team physicians, and hematology physicians were educated along the way regarding changes in our system. Discussion: With time and a multipronged approach, achieving change in a large medical system is possible. The largest hurdle to overcome is funding. This is why our initial interventions focused on the implementation of EMR tools such as SPP, IPP, and educating providers. Though there was an up-front time cost, once completed, these interventions could be iterated on easily. It was not until 2022, that dedicated funding for a consult service could be secured. Having a large multidisciplinary team allows for creative ideas, dividing of tasks, accountability, and expertise in implementation. This does come with a limitation as it is harder to implement changes the larger the group becomes. Thus, a balance of members is vital. Frequent provider education is paramount in disseminating and improving the changes that are in the system. The plan, do, study, act model is a staple in quality improvement. It is important that there is foresight in developing a model to track the quality of care. The EMR's tools should be maximized to aid clinicians as efficiently as possible. Conclusion: Making large changes without funding or an institutional push can be challenging. Small incremental grassroots changes may be necessary until additional funding has been negotiated and secured. Meeting and exchanging ideas as a team and establishing pathways for improvement is a viable initial step.

Understanding caregiver burden with accessing sickle cell care in the Midwest and their perspective on telemedicine

BackgroundSurvival for children with sickle cell disease (SCD) has improved significantly. However, patients with SCD still encounter several impediments to accessing adequate healthcare. Rural and medically underserved areas, such as parts of the Midwest, can exacerbate these barriers, separating children with SCD from subspecialists even further. Telemedicine has been a means to close these gaps in care for children with other special healthcare needs, but few studies have discussed how caregivers of children with SCD perceive its use.MethodsThe objective of this study is to understand the experiences of caregivers of pediatric SCD patients in a geographically diverse area in the Midwest in accessing care, and their perspectives of telemedicine. Caregivers of children with SCD completed an 88-item survey via a secured REDCap link either in-person or via secure text. Descriptive statistics (means, medians, ranges, frequencies) were performed for all responses. Univariate chi square tests were performed to analyze associations, particularly with telemedicine responses.ResultsThe survey was completed by 101 caregivers. Nearly 20% of families traveled more than 1 hour to reach the comprehensive SCD center. Other than their SCD provider, caregivers reported their child having at least 2 other healthcare providers. Most barriers caregivers identified were financial or resource based. Almost a quarter of caregivers expressed feeling as though these barriers impacted their and/or their child’s mental health. Ease of access to team members, as well as scheduling, were common facilitators of care cited by caregivers. The majority were willing to participate in telemedicine visits, regardless of how far they lived from the SCD center, though many noted aspects requiring adaptation.ConclusionThis cross-sectional study describes barriers to care experienced by caregivers of children with SCD, regardless of proximity to an SCD center, as well as caregiver perceptions of the usefulness and acceptability of telemedicine for SCD care.

Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center.

Sickle cell disease (SCD) requires coordinated, specialized medical care for optimal outcomes. There are no United States (US) guidelines that define a pediatric comprehensive SCD program. We report a modified Delphi consensus-seeking process to determine essential, optimal, and suggested elements of a comprehensive pediatric SCD center. Nineteen pediatric SCD specialists participated from the US. Consensus was predefined as 2/3 agreement on each element's categorization. Twenty-six elements were considered essential (required for guideline-based SCD care), 10 were optimal (recommended but not required), and five were suggested. This work lays the foundation for a formal recognition process of pediatric comprehensive SCD centers.

Living Beyond Life Expectancy: Experience with Aging for Older Adults with Sickle Cell Disease

▪BackgroundOver the last five decades, advancements in management of sickle cell disease (SCD) have led to more individuals living beyond the median life expectancy, which has historically been 14 years based on autopsy data in 1973, to approximately 40 to 45 years in population-based studies and 61 years in recent cohorts from comprehensive programs. Despite the increasing life expectancy for people with SCD, there is a paucity of literature on the unique experiences of older adults with SCD (age > 50). This limits providers' understanding of how to address the unique needs of older adult patients with SCD as they age. Thus, the purpose of this study was to describe experiences with aging for older adults with SCD.MethodsWe recruited 19 older adults with SCD (age > 50) from a single academic comprehensive SCD center in the Southeastern United States. We conducted semi-structured qualitative interviews by phone and in person. Participants were asked open-ended questions about their experiences living with SCD, aging, and surviving beyond the SCD life expectancy. Audio-recorded interviews were transcribed verbatim. The transcripts were analyzed using conventional content analysis.ResultsThe mean age of participants was 58 years (range 50-71) and 47% (n=9) were female. The majority of participants were married or had a domestic partner (58%, n=11) and 47% (n=9) were working. Most participants had HbSS genotype (53%, n=10), 37% (n=7) had HbSC, and 11% (n=2) had HbSβ+-thalassemia. Most people reported current use of hydroxyurea (58%, n=11) and their most common chronic complications were avascular necrosis (79%, n=15), retinopathy (58%, n=11), and chronic kidney disease (47%, n=9).Participants described their experiences aging and living beyond the life expectancy for SCD, encompassed in three themes (Figure 1):Theme 1 was “challenges with SCD across the lifespan,” which focused on obstacles participates described from youth to adulthood, including experiencing pain and complications of SCD, but not being diagnosed with SCD until later in their childhood or until as late as mid-life. They described multiple obstacles as an older adult with SCD, including challenges maintaining employment, limitations in physical and social activities, and additional health problems such as kidney disease, increased fatigue, and pulmonary complications. They also reported perceiving healthcare providers were not knowledgeable enough about SCD to properly care for them.Theme 2 was “knowledge and self-management of SCD improves with age,” where participants described differences between being younger vs. older with SCD, including sharing lessons they learned over time about managing SCD, which made them feel more knowledgeable about how to care for themselves in their older age. They described strategies that helped prevent and manage pain crises, such as preparing for weather extremes, learning personal triggers for sickle cell crises, knowing when to rest and when to keep moving, hydration, and being proactive with their SCD care.Theme 3 was “surpassing life expectancy,” where participants discussed how their expectations for their life expectancy changed throughout their lives, and how their life expectancy was influenced by their family members, healthcare providers, and personal experiences and beliefs. Participants described being told by doctors at a young age that they would not live past age 18; however, when they made it to adulthood, some participants conveyed how they stopped allowing their prognosis to influence their thinking. Instead, they chose to trust in God that they would live long. Most participants said that their faith in God and healthy lifestyle choices were the main reasons why they surpassed the life expectancy for SCD.ConclusionThese findings described the experiences conveyed by older adults with SCD about living beyond their expected lifespan. These data provide direction for providers to address areas of care that are most important to this older population and inform development of interventions to increase the knowledge of providers about SCD and health self-efficacy. The information these participants shared will also provide guidance for younger adults with SCD as they navigate the aging process and implement strategies that can support successful aging with SCD. [Display omitted] DisclosuresStrouse: Takeda: Consultancy.

Barriers and Facilitators of Advance Care Planning for Older Adults with Sickle Cell Disease

Background Life expectancy for individuals with sickle cell disease (SCD) has improved significantly in the last 50 years, creating a new population of older adults with SCD; however, life expectancy of adults with SCD is still up to 30 years shorter than African Americans in the general population and much lower than whites. SCD complications cause significant morbidity, requiring patients to make complex decisions about end of life care. Yet, there is a paucity of literature on SCD advance care planning (ACP), to guide providers on how to address ACP in this population. Thus, the purpose of this study was to assess barriers and facilitators to ACP for older adults with SCD. Methods We recruited 19 older adults with SCD (age > 50) from a comprehensive SCD center in the Southeastern United States. We conducted semi-structured interviews by phone or in person. Interview questions addressed aging with SCD, living beyond SCD life expectancy, experience with health care, experience with end of life care, comfort discussing death and end of life care, presence and nature of prior ACP discussions, and preferences for future ACP discussions. Audio-recorded interviews were transcribed verbatim. The data were analyzed using conventional content analysis. Results The mean age of participants was 57 years (range 50-71) and 47% (n = 9) were female. Most participants were diagnosed with SCD several years after birth and were told that they were not expected to live past age 18. Four of 19 participants had written advanced directives. Most participants were comfortable and willing to discuss ACP and thought that SCD ACP discussions should ideally start in early adulthood. Barriers to ACP included lack of communication, inappropriate contexts for ACP discussions, lack of trust, difficulty navigating ACP documents, and spirituality (Figure 1). Lack of communication included limited to no communication from trusted healthcare providers about ACP preferences. Inappropriate contexts for ACP discussions included being approached at difficult times by unfamiliar providers, such as being asked to complete advance directives while sick in the hospital by a provider who did not know the patient well. Trust was a barrier, as several participants were concerned that being asked about ACP while sick meant providers were giving up on them; others were unsure if they could trust providers or family members to carry out their end of life wishes as written. They reported difficulty navigating ACP documents and several participants incorrectly assumed a lawyer was required to finalize ACP documents. Finally, spirituality was a barrier as some participants reported that end of life planning was inconsistent with their religious beliefs. Facilitators of ACP included discussion at the right time, provider familiarity and knowledge, presence of family, and assistance in completing ACP documents. Participants expressed a desire to have an opportunity to openly communicate their end of life wishes with their provider when they were not sick in the hospital. They preferred discussing ACP with a provider whom they had a good relationship with, was familiar with their history, and they perceived was knowledgeable about SCD. Participants identified their SCD provider, PCP, or pulmonologist as suitable providers to talk to about ACP. Most preferred their family to be present during the ACP discussion. Those that had already completed advance directives at the time of the study received assistance from someone outside the health system to do so, such as help from their church, a lawyer, or their family. Conclusion Older adults with SCD expressed a desire for communication about ACP from trusted providers who are knowledgeable about SCD and want to have ACP conversations when they are feeling well. In addition, having the patient's family present and giving them assistance may increase the patient's comfort in completing their advance directives. The lack of communication from trusted providers or communication from unfamiliar providers about ACP during hospitalizations for acute complications are major barriers to creating advance directives for older adults with SCD. Future research is needed to obtain providers' perspectives on barriers to initiating conversations about ACP with adults with SCD. We will use these results to inform development of patient-centered and culturally-sensitive interventions to improve ACP in adults with SCD. Disclosures Strouse: Takeda: Research Funding.

360o View of a Day Hospital Program Performing Exchange Transfusion and Outpatient Pain Management on Adults with Sickle Cell Disease

Purpose: Therapeutic erythrocytapheresis, known also as automated red blood cell exchange transfusion (RBC exchange), is standard of care for patients with sickle cell disease (SCD) presenting with severe complications and for primary prevention of CNS events in those with increased risks. There is limited data on the framework and experience of performing this procedure in a large adult population and even less on challenges and outcomes. Here, we present demographic and statistical data highlighting characteristics of the chronic exchange transfusion program at a comprehensive and multidisciplinary adult SCD center. Methods: Data were collected from adults (18 years and older) with a diagnosis of SCD that were receiving blood transfusions in the outpatient treatment facility from September 1st, 2017 to August 31st, 2018. Data gathered included age, gender, procedure indications and frequency, hemoglobin S percentage (Hb S%), target pre and post Hb S%, ferritin trend, and type of intravascular device used for procedure. Three hundred and forty-four (344) episodes of transfusions, were performed in 52 unique patients, corresponding to 21 % of the unique patients treated at our center during that period. Two hundred and sixty-two (262) of the transfusion procedures were RBC exchanges. These RBC exchanges were performed in 35 (14%) of the unique patients. Since a sizable proportion of our patients received pain treatment during RBC exchange, we explored how the pain visual analog score (VAS) changed while undergoing pain treatment during RBC exchange and in relation to multiple opiate doses. Descriptive and basic statistical analysis were performed on the data obtained for those receiving RBC exchange alone or in combination with pain management. Results: Thirty-five unique patients underwent RBC exchange during the study period. All but two (2) patients had the procedure via an implanted dual lumen Vortex port, one (1) by an arteriovenous fistula and one (1) via his native veins. Most of the RBC exchanges were performed as part of a chronic transfusion program for one of the following indications: primary prevention of stroke; secondary prevention of stroke; recurrent acute chest syndrome (ACS); intractable pain; presence of multiple comorbidities including pulmonary hypertension, chronic kidney disease and intractable leg ulcers. One patient received RBC exchange in preparation for bone marrow transplant. The three (3) other patients that only received one (1) RBC exchange during that period were previously on exchange transfusion but discontinued the procedure due lack of venous access or benefit from the procedure. The frequency of RBC exchange varied depending of the patient's specific indication for the procedure and the ability to reach a target Hb S% goal of < 30%. The RBC exchange occurred in patients as frequently as every four (4) to seven (7) weeks. The number of transfusions per patient/year ranged from one (1) to thirteen (13) with a mean and median of eight (8). Number of RBC units used per procedure ranged from four (4) to thirteen (13) with a mean of eight (8.6), with each unique patient usually receiving the same amount. Twenty-three (66%) unique patients received, at least once, acute pain treatment - oral, intravenous, or a combination during RBC exchange. The mean change of VAS between presentation and end of the pain management period was 1.9 (p<0.0001). Conclusion: Our data shows that therapeutic RBC exchange are performed on a regular basis on a small but significant subgroup of patients living with SCD and treated at a comprehensive SCD center. Intravenous opioid management of acute pain can be effectively performed while receiving RBC exchange which provides patients with multimodal interventions in the outpatient setting. The relationship between change in VAS and concurrent transfusion deserves further investigation. Although there are standard indications for RBC exchange, we and others use it for indications for which there is low level of evidence or no evidence of efficacy (i.e. leg ulcers). We found that there great variability on the frequency of the procedure, total number of RBC exchanges per year and number of RBC units used. Prospective studies are needed to better understand the short and long-term effects, complications and outcomes of chronic RBC exchange in adults living with SCD. Figure Disclosures Kato: Bayer: Research Funding; Novartis, Global Blood Therapeutics: Consultancy, Research Funding. De Castro:Pfizer: Consultancy; Novartis: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees.

Trajectories of Sickle Cell Disease Severity during Transition to Adult Care

BackgroundAdolescents and young adults (AYAs) with sickle cell disease (SCD) are at increased risk for disease complications and mortality, particularly during transition to adult care. Little is known about the disease course that might be contributing to the increase in morbidity and mortality during transition. This study provides a longitudinal description of the clinical characteristics and disease severity among AYAs with SCD during transition to adult care.MethodsWe conducted a longitudinal analysis of the electronic health records of 339 AYAs (ages 12-27 years) with SCD (97% black, 56% male, 69% hemoglobin SS) who received care at a comprehensive SCD center in the Southeast US between 1989 and 2015. Measures included sociodemographics, transfer, complications, comorbidities, and severity of SCD (using a modification of the pediatric SCD severity index).We used group-based trajectory modeling to identify subgroups of AYAs with distinct severity trajectories. AYAs who were in care for at least 10 years were included (n=133). We used chi-square and unpaired student t-test to explore subgroup differences among (1) AYAs who died compared to AYAs who were still alive during the study; (2) AYAs who were eligible to transfer (i.e., ≥ 19 years) (n=293) and transferred compared to AYAs who were eligible to transfer and did not transfer; and (3) AYAs with different severity trajectories. Statistical significance was set at p=0.05 and 95% CI.ResultsCommon complications among AYAs (n=339) included vaso-occlusive crises (80%), acute chest syndrome (41%), chronic pain (35%), and cerebral infarcts (22%). Comorbidities included depression (19%) and anxiety (14%). Most AYAs who were eligible to transfer transferred (n=220, 75%) at a mean age of 19 years (SD=1.3). Fourteen AYAs died, 10 (71%) after transfer at a mean of 7.3 years (SD=3.1) from transfer.SCD Severity Trajectories (Figure 1): Group-based trajectory modeling identified both stable and increasing severity trajectory groups: stable-low (n=31, 23%), stable-medium (n= 61, 46%), stable-high (n=6, 5%), low-increasing (n=13, 10%), and medium-increasing (n= 22,17%) severity trajectory groups. Most AYAs (74%) had stable severity over time, whereas 26% had increased severity with increasing age.Subgroup Differences in Care and Mortality: AYAs who died (n=14) spent less time in pediatric care (mean=4.6 years, 95%CI 3.7-5.6), however, had a higher mean number of pediatric annual visits (mean=9.3 visits; 95%CI 6.3-12.3) compared to those who were alive (n=324, mean=6 years in pediatric care, 95%CI 5.7-6.1; mean number of pediatric annual visits=5.4, 95%CI 4.8-6.1). There were no differences in demographics, SCD complications, or morbidity.Subgroup Differences in Transfer to Adult Care (Tables 1 and 2): Compared to AYAs who did not transfer, AYAs who transferred were older and lived closer to the SCD clinic. AYAs who transferred were at higher risk for SCD complications and comorbidities. They were more likely to be receiving hydroxyurea. They had higher mean annual pediatric SCD clinic visits, especially during older adolescence, and were in pediatric care for a longer duration.Subgroup Differences in Disease Severity (Tables 3 and 4). Compared to AYAs in stable severity trajectory groups (Figure 1: Groups 1, 3, and 5), AYAs in unstable disease severity trajectory groups (Figure 1: Groups 2 and 4) were more likely to be married, older, and lived closer to the SCD clinic. AYAs in unstable groups were also at higher risk for SCD complications and comorbidities. They were less likely to be receiving treatments and had less pediatric SCD clinic visits by age 18. However, they were more likely to transfer to adult care, have had more adult SCD visits overall, and remained longer in adult SCD care after transfer.ConclusionsWhereas most AYAs with SCD had stable disease severity, nearly a quarter had increasing disease severity, over time. AYAs with increasing severity lived closer to the SCD clinic, were more likely to transfer to adult care, and demonstrated higher and longer use of adult SCD care compared to AYAs with stable disease severity. Genotype was not associated with disease severity trajectory groups, underscoring the importance of clinical care for AYAs, over time. [Display omitted] DisclosuresKayle:Department of Health and Human Services, Administration for Community Living, NIDILRR Advanced Rehabilitation Research Training Health and Function Grant #90AR5019 (PI Heinemann): Other: post-doctoral fellowship; Department of Health and Human Services, Administration for Community Living, NIDILRR Advanced Rehabilitation Research Training Health and Function Grant # 90ARHF0003 (PI Heinemann): Other: Postdoctoral fellowship. Tanabe:Duke University: Employment; NIH and AHRQ: Research Funding; Alliant Health: Consultancy. Maslow:The Crohn's and Colitis Foundation and the American Gastroenterological Association, in collaboration with Pfizer, Inc: Research Funding. Holl:Before Brands, Inc: Research Funding; NIH: Research Funding; Branstad Family Foundation: Research Funding; AHRQ: Research Funding. Shah:Novartis: Research Funding, Speakers Bureau.

A telephonic mindfulness-based intervention for persons with sickle cell disease: study protocol for a randomized controlled trial

BackgroundOne of the most difficult symptoms for persons with sickle cell disease (SCD) to manage is chronic pain. Chronic pain impacts approximately one-third of persons with SCD and is associated with increased pain intensity, pain behavior, and frequency and duration of hospital visits. A promising category of nonpharmacological interventions for managing both physical and affective components of pain are mindfulness-based interventions (MBIs).Methods/designThe primary aim of this study is to conduct a randomized controlled study to evaluate the acceptability and feasibility, as well as to determine the preliminary efficacy, of a telephonic MBI for adults with SCD who have chronic pain. We will enroll 60 adult patients with SCD and chronic pain at an outpatient comprehensive SCD center in the southeastern United States. Patients will be randomized to either an MBI or a wait-listed control group. The MBI group will complete a six-session (60 minutes), telephonically delivered, group-based MBI program. The feasibility, acceptability, and efficacy of the MBI regarding pain catastrophizing will be assessed by administering questionnaires at baseline and weeks 1, 3, and 6. In addition, ten randomly selected MBI participants will complete semistructured interviews to help determine intervention acceptability.DiscussionIn this study protocol, we report detailed methods of the randomized controlled trial. Findings of this study will be useful to determine the acceptability, feasibility, and efficacy of an MBI for persons with SCD and chronic pain.Trial registrationClinicalTrials.gov identifier: NCT02394587. Registered on 9 February 2015.

Growing Pains – Determination of Transfer and Transition from Pediatrics to Adult Outpatient Clinics for Patients with Sickle Cell Disease (SCD)

Introduction. Advances in medicine in the last four decades have allowed adolescents with Sickle Cell Disease (SCD) to survive into adulthood. Consequently, there is a sizeable number of maturing young adults with SCD who require transition from pediatric to adult care. This transition can be difficult given the tumultuousness of outgrowing adolescence and entering adulthood, the burden of living with a chronic disease, and the self-advocacy and medical independence expected from patients by adult health care providers. To better understand utilization of care during this period in a comprehensive SCD center, we investigated the number of patients who transferred and successfully transitioned from pediatric outpatient hematology care to adult care.Methods. Following IRB approval, a retrospective chart review of patients with SCD aged 18-23 years in the Duke University Medical Center electronic medical record database was conducted. Included for each patient was a list of all clinic encounters at Duke through the December 2013. A total of 342 SCD patient records were retrieved, of which 88 (26%) were excluded because of missing data on outpatient SCD encounters. Transfer was defined as a clinic visit in pediatrics followed by a subsequent visit in the adult clinic. Successful transition was defined as at least 1 year of follow up care in adult hematology clinic post-transfer.Results. The 284 patient records fell into three utilization patterns: 182 whose last encounter was a pediatric visit (72%), 20 who started care in the adult clinic (8%), and 52 who transferred from pediatric to adult care (20%). Within the pediatric group, 36 patients were actively receiving care while 146 were lost to follow-up. Among patients that transferred, the average age of transfer was 18.6 years and the transfer occurred within 3 months of the last pediatric visit for 61% of patients. Although 71% of transferred patients continued care for at least 1 year post-transfer, the majority of 19-20 year old patients (61%) did not transition successfully.Discussion. Our findings show that SCD patients aged 18-23 are having difficulty transferring and successfully transitioning from pediatric to adult hematology care in the Duke Medical Center despite having a single medicine-pediatric provider. The majority of patients had their last visit in pediatric care which is consistent with Duke’s SCD clinical practice to keep 18-20 year old patients in pediatric care to better educate patients. The large number of patients who stopped care in pediatrics may be explained by care provided by non-Duke hematologists or primary care providers; relocation; or use of acute care clinics (emergency department, urgent care, and day hospital) as a substitution for regular care. As observed in this data, when transfer does occur among this younger adult group of 18-20 year olds, only a third of the patients have a successful transition. The low success rate may be accounted for by patient impression of health; avoidance of regular care; loss of insurance; and struggle with independence.Implications. The results underscore the need to not only improve successful transition rates for SCD patients but also to assist and track patients preparing to transfer to adult specialty clinics. Further research is needed to determine what is happening to patients who are lost to follow-up during their pediatric care and to determine the relationship between the amount of time to transfer and successful transition rates. Because our robust definition of a successful transition is more sensitive to identifying gaps in continuous care as compared to other success metrics based on a 50% show rate, we were able to identify 19-20 years as a critical age group for intervention. DisclosuresShah:Novartis: Speakers Bureau.