Effects of the COVID-19 Pandemic on Caregivers of Young Children with Sickle Cell Disease Enrolled in the Engage-HU Trial

Background: Hydroxyurea (HU) is the primary medication used to prevent the significant medical and neurologic morbidities of pediatric sickle cell disease (SCD;HbSS or HbSB0 thalassemia). Despite the benefits of HU, it remains under-utilized likely due to lack of clinician knowledge/training and negative caregiver perceptions. Thus, we developed the Engage-HU randomized controlled trial (NCT03442114) as a novel approach to address HU utilization barriers. Engage-HU is designed to assess how clinicians can engage caregivers in a shared discussion that considers their values, preferences, and scientific evidence about HU. The COVID-19 pandemic has resulted in significant changes to healthcare delivery for children with SCD, as they are at increased risk of severe illness from COVID-19 infection. Given their risk status, it was recommended that patients with SCD complete telehealth visits when possible. Some families also chose to delay care because they feared their child would get infected at hospitals/healthcare clinics that care for COVID-19 positive patients. Since the lives of all families enrolled in the Engage-HU trial have been affected to some extent, we incorporated measures to capture the impact of the COVID-19 pandemic and the usability of telemedicine implementation and services. Methods: Engage-HU is a randomized control trial comparing two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD. Study outcomes include caregiver confidence in decision-making and perceptions of experiencing shared decision-making as well as HU uptake and child health outcomes. Eligible children are 0 to 5 years, candidates for HU, and their caregiver has not decided about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a unidirectional crossover design. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using the intent-to-treat principle, and all participants will remain in the arm to which they were randomized. A multiple group comparison analysis will be performed to assess significant response variable differences by group randomization. The Engage-HU study aims to recruit 174 caregivers who are considering initiating HU. The trial is being conducted at 9 sites in the United States. Data collection is ongoing, and 160 caregiver-participants have been enrolled to date. Since May 2020, caregiver-participants have completed the COVID-19 Exposure and Family Impact Scales (CEFIS), which contain 2 subscales (exposure to potentially traumatic aspects of the pandemic, impact on families), and the COVID-19 telemedicine use survey during a study visit. Results: Currently, 8 of the 9 sites have collected data from 48 caregivers (93.8% mothers), most of whom (93.8%) identify as African American/Black (see Figure 1). Correlations indicated that older caregivers experienced greater exposure (Mean = 7.0, SD = 4.1, range = 1-19) to potentially traumatic aspects of the pandemic (r =.31, p =.04). Distress related to COVID-19 varied widely across the sample, for both caregivers (Mean = 5.9, SD = 2.9, range = 1-10) and children (Mean = 4.1, SD = 3.4, range = 1-10). Scores on the telemedicine usability survey were generally high, indicating that caregivers are happy with the quality of care delivered via telehealth. However, caregivers (r =.30, p =.09) and children (r =.32, p =.07) experiencing more pandemic-related distress reported less satisfaction with telehealth. Conclusion: Although Engage-HU has resumed research operations, recruitment has not reached pre-pandemic targets, as fewer eligible patients are scheduled for routine care visits at SCD clinics. Our preliminary analyses suggest a significant continued impact of the pandemic on families and general satisfaction with the quality of healthcare delivered via telemedicine. These findings indicate that targeted screenings to identify and intervene for those who emonstrate more COVID-19 pandemic-related distress are needed. [Formula presented] Disclosures: Quinn: Forma Therapeutics: Consultancy;Aruvant: Research Funding;Novo Nordisk: Consultancy;Emmaus Medical: Research Funding. Yates: Agios Pharmaceuticals: Current Employment. Badawy: Sanofi Genzyme: Consultancy;Vertex Pharmaceuticals Inc: Consultancy;Bluebird Bio Inc: Consultancy. Thompson: bluebird bio, Inc.: Consultancy, Research Funding;Baxalta: Research Funding;Biomarin: Research Funding;Celgene/BMS: Consultancy, Research Funding;CRISPR Therapeutics: Research Funding;Vertex: Research Funding;Editas: Research Funding;Graphite Bio: Research Funding;Novartis: Research Funding;Agios: Consultancy;Beam: Consultancy;Global Blood Therapeutics: Current equity holder in publicly-traded company. Smith-Whitley: Global Blood Therapeutics: Current Employment. King: National Cancer Institute: Research Funding;National Heart, Lung, and Blood Institute: Research Funding;Health Resources and Services Administration: Research Funding;Global Blood Therapeutics: Research Funding. Meier: CVS Caremark: Consultancy;Forma Therapeutic: Membership on an entity's Board of Directors or advisory committees;NovoNordisk: Membership on an entity's Board of Directors or advisory committees;Novartis,: Other: Data Safety Monitoring Board membership;NHLBI: Other: Data Safety Monitoring Board membership;Global Blood Therapeutics: Other: Steering Committee membership, grant funding;CDC,: Other: grant funding;Indiana Department of Health: Other: grant funding. Tubman: Global Blood Therapeutics: Consultancy, Research Funding;Novartis Pharmaceuticals: Honoraria, Research Funding;Forma Pharmaceuticals: Consultancy;Perkin Elmer: Honoraria. Crosby: Forma Therapeutics: Honoraria;PCORI: Research Funding;HRSA: Research Funding;Global Blood Therapeutics Panel: Honoraria;Children's Hospital of Philadelphia: Honoraria;Professional Resource Exchange: Patents & Royalties: $30-$60 every other year;SCDAA: Honoraria;NHLBI: Other: Payment for review of LRP Proposals, Research Funding. OffLabel Disclosure: Hydroxyurea has been FDA approved for the treatment of sickle cell disease for patients ages 2 years and above but NHLBI and ASH Guidelines recommend it be offered to children as young as age 9 months.

Leveraging Project Echo Telementoring to Improve Sickle Cell Disease Care in the Midwest: Expanding Provider Education during a Pandemic

Introduction Approximately 15,000 individuals in the Midwest live with sickle cell disease (SCD), a lifelong, painful disorder with complications and comorbidities. Providers self-report limited knowledge and confidence in treating patients with SCD, leading to limited access to providers knowledgeable about evidence-based management and treatment guidelines and therefore poor health outcomes for patients. Sickle Treatment and Outcomes Research in the Midwest (STORM) is a regional sickle cell network, established to improve outcomes for individuals with SCD living in Indiana, Illinois, Michigan, Minnesota, North Dakota, Ohio, South Dakota and Wisconsin. One goal of STORM is to increase provider knowledge about evidence-based management of SCD. STORM TeleECHO, a replication of the Project ECHO (Extension for Community Healthcare Outcomes) telementoring model, was launched in March 2016 as an innovative, lifespan approach to provider education in the Midwest. In March 2020, in response to the COVID-19 public health pandemic, the STORM coordinating team quickly pivoted to host additional COVID-19 and SCD focused ECHO sessions. Methods STORM TeleECHO virtual clinics include didactic presentations with a curriculum based on the National Heart Lung and Blood Institute Evidence-Based Management of Sickle Cell Disease guidelines, as well as a de-identified case discussion presented by providers seeking feedback on the management of challenging clinical scenarios. Other additional medical and psychosocial issues have also been presented. Participants join the monthly hour-long sessions using Zoom© or telephone. COVID-19 and SCD sessions followed the same format and topics focused on emerging medical and psychosocial pandemic issues, such as: serology, multi-system inflammatory syndrome in children, the COVID and SCD surveillance registry, blood safety and transfusion practices, telemedicine, back to school issues, mental health, and Sickle Cell Disease Association of America patient and provider advisories. These sessions also provided an open forum for this hematology/primary care provider community of practice to share rapidly changing clinical practices and patient resources. Results Since March 2016, 58 sessions have been held with over 175 unique attendees. Evaluation data shows 100% of providers will continue participating and would recommend STORM TeleECHO to a colleague. Over 80% reported learning best practice guidelines for SCD;developing clinical expertise;and applying knowledge into practice. Data analysis has shown a statistically significant increase in provider confidence to identify eligible candidates and prescribing hydroxyurea. STORM has awarded over 750 CME credits (since 2016), over 300 MOC Part II from the American Board of Pediatrics and the American Board of Internal Medicine (since 2017) and 70 CNE credits (since 2019). Since March 2020, the 11 special COVID-19 sessions have averaged 42 attendees per session- nearly a 150% increase compared to the standing ECHO sessions in previous years (Table 1). Over 115 unique providers have attended at least one COVID-19 session, with 61 participants being new to STORM TeleECHO. Participants have cumulatively attended an average of 4 COVID-19 sessions and represented 17 states and Canada. On average, 93% of respondents rated the COVID-19 STORM TeleECHO sessions as “very good” or “excellent”. Discussion STORM TeleECHO has been instrumental in increasing knowledge and comfort of providers caring for patients with SCD. Data shows that STORM TeleECHO has the potential to improve outcomes and decrease health disparities in this underserved and medically vulnerable population. Moreover, STORM COVID-19 and SCD TeleECHO session participants have been highly satisfied with this educational forum for addressing emerging issues during the public health emergency. Our program quickly and successfully leveraged the existing STORM TeleECHO framework to expand educational forums for this community of practice. The continued increase in participants sustained throug out the COVID-19 ECHO's has demonstrated the need and interest for this educational forum to share practice changes, new clinical protocols, patient education and other resources. While the regular STORM TeleECHO sessions will continue, there are also plans to continue the additional COVID-ECHO sessions for the foreseeable future. [Formula presented] Disclosures: No relevant conflicts of interest to declare.