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

ABSTRACT

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.