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.

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.

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.

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.

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.

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.