Maintenance of a High Influenza Vaccination Rate and Improvement in Health Outcomes in a Pediatric Sickle Cell Disease Clinic.

BACKGROUND: Children with sickle cell disease (SCD) are at high-risk of complications from influenza and should receive an influenza vaccination seasonally. Despite this recommendation, vaccination rates remain suboptimal. Boston Medical Center (BMC) previously achieved high influenza vaccination rates among its pediatric patients with SCD. The purpose of this study was to determine whether this high vaccination rate has been maintained and whether it has influenced outcome measures. PATIENTS AND METHODS: A retrospective chart review was conducted in the hematology clinic at an urban, academic medical center. Fisher's exact test and the independent samples t test were used to determine if there were any significant differences in characteristics between patients with influenza and patients without influenza, as well as between vaccinated and unvaccinated patients. Influenza vaccination rate, influenza-related hospitalization rate, and influenza-positive rate were collected and compared with reported rates. RESULTS: Data from 124 pediatric patients with SCD were examined. The influenza vaccination rate for pediatric patients with SCD at BMC (90.32%) was higher than previous studies that were not conducted at BMC, while BMC's influenza-related hospitalization rate (0) and influenza-positive rate (4.84%) were lower than other studies. Subjects who contracted influenza were younger than those who did not (4.67 vs. 10.03 y, P=0.005). CONCLUSIONS: BMC has maintained a high influenza vaccination rate among pediatric patients with SCD. BMC's vaccination strategy has been successful at improving outcome measures including rates of influenza and influenza hospitalizations without requiring additional staff. Such efforts should be replicated at other centers.

Measuring transition readiness in adolescents and young adults with sickle cell disease using the transition readiness assessment questionnaire.

BACKGROUND/OBJECTIVES: Adolescents and young adults (AYA) with sickle cell disease (SCD) face challenges related to the disease and its treatment. The Transition Readiness Assessment Questionnaire (TRAQ) is a self-report tool for assessing transition readiness for youth with special health care needs (YSHCN), including SCD. This study uses the TRAQ to understand transition readiness in patients with SCD treated at the Boston Medical Center and evaluates associations between TRAQ scores and transition outcomes (e.g., emergency department reliance [EDr] and emergency department utilization [EDu]). METHODS: We reviewed electronic medical records of AYA with SCD who completed the TRAQ in the pediatric hematology clinic between January 1, 2019, and March 1, 2020, and categorized healthcare encounters to calculate EDu and EDr.  We used t tests and ANOVA models to analyze mean TRAQ scores, sex, age, genotype, EDu, and EDr. RESULTS: The sample was 45 AYA patients with SCD between 13 and 22 years old. The mean TRAQ score for the overall patient sample was 3.67. Mean TRAQ scores did not significantly vary by sex or genotype but did significantly increase with age. TRAQ scores did not correlate to EDu or EDr. CONCLUSIONS: AYA patients with SCD have low transition readiness. The age of 18 may not be the most reliable attribute of readiness, though older patients do have higher readiness. The relationship between TRAQ scores, EDr, and EDu is not clear and requires further evaluation.

Two Sides of a Coin: Case Report of Unilateral Synangiosis and Contralateral Stroke Highlighting Consequences of Disease Progression and Efficacy of Revascularization in Sickle Cell Disease-Associated Moyamoya Syndrome.

Moyamoya syndrome increases the risk of stroke in sickle cell disease, but revascularization surgery can modify this risk. Collaborative management between hematology and neurosurgery offers effective strategies to reduce stroke risk in these patients. We describe a challenging case where a patient with sickle cell disease undergoing standard of care management as prescribed by the Stroke Prevention Trial in Sickle Cell Anemia and revascularization with pial synangiosis subsequently developed rapidly progressive disease in other cerebral vessels and suffered ischemic hemispheric stroke. This case not only demonstrates the success of management in accordance with the American Heart Association (AHA) and American Stroke Association (ASA) guidelines but also demonstrates critical areas where we lack understanding of disease progression.

Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial.

BACKGROUND: Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/ß0 thalassemia) aged as early as 9 months. However, the uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or tools, especially those that provide medical evidence and consider caregivers' preferences and values, to facilitate a shared discussion with caregivers. OBJECTIVE: The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences). METHODS: We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0-5 years) who were offered and prescribed hydroxyurea and the resultant health outcomes. RESULTS: The Ethics Committee of the Cincinnati Children's Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants. CONCLUSIONS: The long-term objective of this study is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA can make informed and shared decisions about their health. TRIAL REGISTRATION: ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/27650.

Household material hardships impact emergency department reliance in pediatric patients with sickle cell disease.

BACKGROUND: In sickle cell disease (SCD), high emergency department (ED) utilization is associated with worse outcomes and increased costs. A metric called ED reliance (EDr), the percentage of healthcare visits that occur in the ED, attempts to identify ED overutilization. It is unknown if household material hardships (HMH)-housing, utility, or food insecurity-impact reliance on the ED. As these may represent modifiable risk factors for high ED utilization, we aimed to estimate the association between HMH and EDr in pediatric patients with SCD. METHODS: We reviewed the electronic medical records of pediatric patients with SCD who received care in the Boston Medical Center network in Massachusetts, USA, to collect data on HMH and healthcare utilization. Using linear regression to control for potential confounders, we modeled the association between material hardships and EDr. RESULTS: Of 101 eligible patients, 60 (59%) reported one or more HMH. The mean EDr was 12% overall, with significant differences between those with and without HMH (15.9 vs 5.9, P = 0.0001). Each additional hardship experienced was associated with an increased average EDr of 7.7 percentage points (R2  = 0.34, P < 0.0001). Housing and utility hardships were each independently associated with increased EDr. CONCLUSION: HMH are associated with significantly increased EDr in children with SCD, independent of transportation hardship or insurance type. Through screening for HMH, providers and health systems could identify at-risk patients with modifiable risk factors for high EDr in order to provide them additional support.

Rethinking Rideshares: A Transportation Assistance Pilot for Pediatric Patients with Sickle Cell Disease.

OBJECTIVE: Transportation barriers can limit health care access. This is particularly problematic for patients with chronic medical conditions such as sickle cell disease (SCD) who require frequent medical visits. This study assesses the efficacy of health care-directed rideshare services for overcoming these barriers at an urban pediatric specialty clinic. METHODS: A pilot study was conducted at Boston Medical Center's Pediatric Hematology Clinic from January to April 2019. Patients whose caregivers reported transportation difficulties were offered rides. Primary outcomes were no-show rates and cost. Secondary outcomes included timeliness and patient experience. RESULTS: Implementation of rideshare services led to an 8.5% decrease in the no-show rate among patients with SCD. The intervention cost $2,175 over three months and generated $40,262 in charges. No adverse experiences were reported. CONCLUSIONS: In an urban, underserved pediatric hematology clinic, the use of rideshare services is a feasible and relatively low-cost strategy for improving health care access.

Universal screening for social determinants of health in pediatric sickle cell disease: A quality-improvement initiative.

BACKGROUND: Social determinants of health (SDoH) are socioeconomic factors that influence health outcomes. Guidelines recommend universal screening for SDoH at clinic visits; however, models that do not require additional resources are limited in subspecialty clinics. Individuals with sickle cell disease (SCD) face the burdens of chronic illness and often racial disparities, both of which may increase their vulnerability to adverse SDoH. Hematologists can impact both quality of life and clinical outcomes for their patients by implementing screening and referral programs addressing SDoH. METHODS: Through prospective, quality-improvement methods, we introduced universal screening for SDoH into our pediatric hematology clinic. The intervention was a paper screener followed by a referral to local community organizations for the specific needs endorsed. The aims of this study were to determine the feasibility of universal screening for SDoH in a busy subspeciality clinic using pre-existing resources to identify the needs of our patients and to facilitate referrals between our patients and community organizations via this low touch intervention. RESULTS: Between August 2017 and November 2018, 156 screens were completed. Sixty-six percent were positive for at least one unmet social need for which 80% were referred to a relevant community organization. Forty-five percent of patients available via follow-up phone call reached out to the community organization. CONCLUSIONS: There is a high burden of SDoH in families of children with SCD. Universal screening at a pediatric hematology clinic with the subsequent connection of patients with SCD to community resources is feasible using existing clinic resources.

Developing a Problem-solving Intervention to Improve Self-Management and Transition Readiness in Adolescents with Sickle Cell Disease.

PURPOSE: Adolescents and young adults (AYA) with sickle cell disease (SCD) are at risk for serious complications including increased morbidity and early mortality during their transition from pediatric to adult care. Self-management support may help improve transition outcomes in this vulnerable population. Interventions based on teaching problem solving skills have been shown to improve adherence to therapy for AYA with other chronic disease and is a promising intervention in SCD. We sought patient and parent perspectives on improving self-management and input on the development of a problem-solving education (PSE) intervention. DESIGN AND METHODS: We held focus groups with AYA with SCD and caregivers to discuss barriers and facilitators of self-management, acceptability of PSE and intervention content and delivery. RESULTS: Five focus groups were held with AYA (n = 17) and caregivers (n = 15). Groups participated jointly to discuss self-management and then separately to discuss PSE. Data were analyzed using grounded theory and double-coded until thematic saturation was achieved. CONCLUSIONS: Both groups endorsed PSE as an acceptable intervention. Barriers to self-management included wanting to fit in with peers (AYA) and trouble letting go (parents); facilitators included having a regular routine (AYA) and sharing responsibility (parents). Participants suggested meeting in small groups for PSE sessions rather than individually and adding group sessions for parents. PRACTICAL IMPLICATIONS: Understanding AYA and caregivers' perceptions of barriers and facilitators of transition in SCD can help us better prepare AYA for transition. The specifics both groups identified as helpful will guide intervention development.

A Mild Phenotype of Severe ß+ Thalassemia in a 16-Month-Old Boy.

ß thalassemia is characterized by a deficient production of functional ß-globin chains and a relative excess of α-globin chains. An extremely diverse clinical spectrum-asymptomatic to transfusion-dependent-is primarily due to homozygosity or compound heterozygosity for the very large number of ß-thalassemia-causing mutations, along with interacting mutations that affect the α-globin and γ-globin genes and their expression. We report a case of a 16-month-old boy who was initially diagnosed with iron deficiency anemia until he was later found to be homozygous for a severe ß-thalassemia genotype with a mild hematologic phenotype. This was likely as a result of his ability to produce high levels of fetal hemoglobin.

Development of quality indicators for transition from pediatric to adult care in sickle cell disease: A modified Delphi survey of adult providers.

BACKGROUND: Transition from pediatric to adult care is a vulnerable time for young adults with sickle cell disease (SCD); however, improvements in transition are limited by a lack of quality indicators. The purpose of this study was to establish quality indicators for transition in SCD and to determine the optimal timing between the final pediatric visit and the first adult provider visit. PROCEDURE: We conducted a modified Delphi survey to reach a consensus on which quality indicators are most important for a successful transition. Our expert panel consisted of members of the Sickle Cell Adult Provider Network. In the first round, the participants ranked a list of quality indicators by importance. In the second round, the participants chose their "top 5" quality indicators in terms of importance and also ranked them on feasibility. RESULTS: The response rates for the two rounds were 68 and 96%, respectively. Nine quality indicators were chosen as "top 5" by a majority of respondents, including communication between pediatric and adult providers, timing of first adult visit, patient self-efficacy, quality of life, and trust with their adult provider. Based on the comments from round 1, respondents were also asked for the optimal timing between leaving pediatric care and entering adult care. Most recommended a first adult visit within 2 months of the final pediatric visit. CONCLUSIONS: By using these quality indicators chosen by the majority of respondents, we can better develop and evaluate transition programs for young adults with SCD and improve health outcomes for these vulnerable patients.

APHON/ASPHO Policy Statement for the Transition of Patients With Sickle Cell Disease From Pediatric to Adult Health Care.

With advances in medical care, the majority of children with sickle cell disease are surviving to adulthood. Patients, families, and providers now face the need for this growing population to move from pediatric- to adult-focused care. In order to facilitate a successful transfer to adult health care, and prepare young adults for greater independence, it is recommended that all pediatric patients with sickle cell disease receive transition preparation. ASSOCIATION POSITION: As the professional voice of pediatric hematology/oncology healthcare practice, the Association of Pediatric Hematology/Oncology Nurses (APHON) and the American Society of Pediatric Hematology Oncology (ASPHO) recommends that the discussion of transition begin early and is presented as part of the natural process of becoming an adult; that patients, providers, and families are all involved in creating a transition plan and assessing transition preparedness annually; and that transfer of care involve direct communication between the pediatric team and the accepting adult provider.

Improving the Management of Vaso-Occlusive Episodes in the Pediatric Emergency Department.

OBJECTIVES: Vaso-occlusive episodes (VOEs) account for the majority of emergency department (ED) visits for children with sickle cell disease (SCD). We hypothesized that addressing key barriers to VOE care would improve receipt of analgesics and outcomes. METHODS: A quality improvement (QI) initiative was conducted from September 2010 to April 2014 to streamline VOE care in an urban pediatric ED. Four interventions were used: a standardized time-specific VOE protocol; intranasal fentanyl as the first parenteral pain medication; an SCD pain medication calculator; and provider and patient/family education. Data were collected for 3 outcome measures (mean time from triage to first parenteral opioid and admission/discharge decision, and proportion discharged from the ED); 1 process measure (mean time from triage to initiation of patient-controlled analgesia); and 4 balancing measures (mean time from triage to second intravenous opioid dose, 24-hour ED readmission, respiratory depression, and length of stay). RESULTS: There were 289 ED visits in the study period. Improvements were seen in mean time to: first dose of parenteral opioid (56 to 23 minutes); second opiate intravenous dose (106 to 83 minutes); admission and discharge decisions (163 to 109 minutes and 271 to 178 minutes, respectively); and initiation of patient-controlled analgesia (216 to 141 minutes). The proportion discharged from the ED increased from 32% to 48% (χ(2) = 6.5402, P = .01). No increase in 24-hour readmission, respiratory depression, or inpatient length of stay was observed. CONCLUSIONS: Using VOE-specific interventions, we significantly improved VOE care for children. Studies are needed to determine if these results can be replicated.

How we prevent and manage infection in sickle cell disease.

Sickle cell disease (SCD) affects approximately 100,000 people in the US, 12,500 in the UK, and millions worldwide. SCD is typified by painful vaso-occlusive episodes, haemolytic anaemia and organ damage. A secondary complication is infection, which can be bacterial, fungal or viral. Universal newborn screening, routine use of penicillin prophylaxis, availability of conjugated vaccines against S. pneumoniae and comprehensive care programmes instituted during the past few decades in industrialized countries have dramatically reduced childhood mortality and improved life expectancy. Yet patients with SCD remain at increased risk of infection. Unfortunately, the treatment of most bacterial infections that are common in SCD is not based on the results of randomized controlled clinical trials. In their absence, treatment decisions are based on consensus guidelines, clinical experience or adapting treatment applied in other diseases. This leads to wide variation in treatment among institutions and even between treating physicians in a single institution. Prevention of infection, when possible, is most important and we focus on prevention through targeted prophylaxis and vaccination. We will share our management strategies for managing the more common infections in SCD and provide the rationale for our recommendations.

Young Adult Perspectives on a Successful Transition from Pediatric to Adult Care in Sickle Cell Disease.

OBJECTIVE: This qualitative study sought to learn from young adults with sickle cell disease (SCD) about their experience leaving pediatric care and perspective on what makes a successful transition. METHODS: Fifteen young adults with SCD who had left pediatric care within the previous five years participated in focus groups led by a trained moderator. Transcripts were analyzed using grounded theory. RESULTS: Four main themes emerged from the analysis: facilitators of transition (meeting the adult provider prior to transfer, knowing what to expect, gradually taking over disease self-management and starting the process early), barriers to transition (negative perceived attitude of adult staff, lack of SCD specific knowledge by both patients and staff, and competing priorities interfering with transition preparation), what young adults wished for in a transition program (opportunities to meet more staff prior to transfer, more information about the differences between pediatric and adult care, learning from a peer who has been through the process, more SCD teaching, and flexibility in transition preparation) and how they define a successful transition (gradually assuming responsibility for self-management of their SCD). CONCLUSION: Our findings present unique opportunities to learn from young adults with SCD about ways to improve current transition programs.

Improvement in influenza vaccination rates in a pediatric sickle cell disease clinic.

BACKGROUND: Children with sickle cell disease (SCD) are at increased risk of complications from influenza. However, despite widespread recommendations that these patients receive an annual influenza immunization, reported vaccination rates remain very low at under 50%. PROCEDURE: Our aim was to increase the influenza vaccination rate among our pediatric patients with SCD aged 6 months to 21 years over two influenza seasons, 2012-2013 and 2013-2014, to 80%, consistent with the Health People 2020 goal. We used multiple quality improvement methods, based on the literature and our previous experience in other aspects of SCD care, including parent and provider education, enhancement of our EHR, use of a SCD patient registry and reminder and recall done by a patient navigator. RESULTS: We vaccinated 80% of our pediatric patients with SCD for influenza during the 2012-2013 season and 90% of patients in 2013-2014. Our early season vaccination rates were nearly double that of those for the general population. CONCLUSIONS: Use of quality improvement methods can increase rates of influenza vaccination for this high-risk population, suggesting that less health care utilization and lower cost might result.

Transition and transfer of adolescents and young adults with pediatric onset chronic disease: the patient and parent perspective.

PURPOSE: To determine patients' and parents' perceptions regarding the delivery of transition education and perceived barriers to transfer to adult oriented care. METHODS: A self-report survey was administered to a convenience sample of patients (16-25 years old) with various childhood onset chronic diseases. A similar survey was administered to their parents/guardians. RESULTS: A total of 155 patients and 104 parents participated in the study. The mean age of patients was 18.8 ± 2.3 years; 57% were female. Although most patients and parents reported receiving information and training about their medical condition, significant gaps in other aspects of transition education were identified. These included stated deficiencies in education regarding unprotected intercourse, health of future offspring, birth control, pregnancy, illicit drug use, and future career or vocation counseling. Commonly cited barriers to transfer were emotional attachments and lack of adult medicine specialty providers; however, the majority anticipated being ready to transfer to adult oriented care by age 25 years. CONCLUSION: There are significant gaps in the delivery of transition education as perceived by patients and their parents. Standardization of transition education may help ensure that patients acquire the knowledge and skills for health care self-management in adulthood and successful transfer to adult oriented care.

Relationship among chelator adherence, change in chelators, and quality of life in thalassemia.

PURPOSE: Thalassemia, a chronic blood disease, necessitates life-long adherence to blood transfusions and chelation therapy to reduce iron overload. We examine stability of health-related quality of life (HRQOL) in thalassemia and adherence to chelation therapy over time, especially after changes in chelator choice. METHODS: Thalassemia Longitudinal Cohort participants in the USA, UK, and Canada completed the SF-36v2 (ages 14+) and the PF-28 CHQ (parents of children <14 years). Chelation adherence was defined as self-reported percent of doses administered in the last 4 weeks. RESULTS: Two hundred and fifty-eight adults/adolescents (mean 29.7 years) and 133 children (mean 8.5 years) completed a mean of 2.8-years follow-up. Children made few chelator changes, whereas a mean of 2.2 changes was observed among the 37% of adults/adolescents who made chelator changes, mainly due to patient preference or medical necessity. Physical HRQOL improved among those with lower iron burden (better health status) at baseline who made a single change in chelator, but declined among participants with multiple changes and/or high iron burden (worse health status). Mental health improved among participants with lower iron burden, but iron overload was negatively associated with social functioning. Adherence did not significantly change over follow-up except for an increase after a change from deferoxamine (DFO) infusion to oral deferasirox (p = 0.03). Predictors of lower adherence for adults/adolescents at follow-up included side effects, smoking, younger age, problems preparing DFO, increased number of days per week DFO prescribed, and lower physical quality of life . CONCLUSIONS: Strategies to balance medical needs with family, work, and personal life may assist in adherence.

Self-reported transition readiness among young adults with sickle cell disease.

BACKGROUND: A growing body of literature addresses the need for transition programs for young adults with sickle cell disease (SCD); however, studies assessing transition readiness are limited and there are few validated instruments to use. OBJECTIVE: To conduct a pilot study to assess transition readiness of patients with SCD in our transition program and to evaluate a SCD-specific assessment tool that measures 5 knowledge skill sets (medical, educational/vocational, health benefits, social, and independent living), and 3 psychological assessments (feelings, stress, and self-efficacy). RESULTS: Of the 47 SCD patients between the ages of 18 and 22 seen in our facility, 33 completed the assessment tool. The majority of patients reported good medical knowledge of SCD and said they were motivated to pursue a career despite the burden of living with the disease. We identified knowledge gaps in the area of independent living and health benefits skills sets. A majority of patients reported being worried that their SCD would prevent them from doing things in their life; however, few respondents said they were worried or anxious about their transition to adult care. CONCLUSIONS: Adolescents beginning a transition intervention program reported a high level of knowledge of their disease and demonstrated a positive attitude toward transition with good self-efficacy.