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1891 Effects of the COVID-19 Pandemic on Caregivers of Young Children with Sickle Cell Disease Enrolled in the Engage-HU Trial

Program: Oral and Poster Abstracts
Session: 901. Health Services Research—Non-Malignant Conditions: Poster I
Hematology Disease Topics & Pathways:
Clinical Trials, Sickle Cell Disease, Workforce, Hemoglobinopathies, Patient-Reported Outcomes, Diseases, Pediatric, Study Population
Saturday, December 11, 2021, 5:30 PM-7:30 PM

Anna M Hood, PhD1*, Aimee K Hildenbrand, PhD2*, Joanna Rebitski, BA3*, Jasmine Stallworth4*, Yolanda Johnson, MLS5*, Stacey Gomes, MS4*, Catharine Whitacre, MS4*, Constance A Mara, PhD6*, Lisa M Shook, DHPE7,8*, Charles T. Quinn, MD, MS9,10, Jean L. Raphael, MD, MPH11, Amber M Yates, MD, FAAP12,13, Sherif M. Badawy, MD14,15, Alexis A. Thompson, MD, MPH15,16, Kim Smith-Whitley, MD17, Helge Hartung, MD18, Allison A. King, MD, MPH, PhD19, Susan E Creary, MD, MSc20,21, Neha Bhasin, MD22, Steven K Reader, PhD23*, Emily R. Meier, MD, MSHS24, Venee N. Tubman, MD25,26 and Lori E. Crosby, PhD27,28

1Developmental Neurosciences, University College London Institute of Child Health, London, OH, United Kingdom
2Center for Healthcare Delivery Science, Nemours Children’s Health System, Wilmington, DE
3College of Medicine, University of Cincinnati, Cincinnati, OH
4Cincinnati Children's Hospital Medical Center, Cincinnati, OH
53333 Burnet Ave, MLC 7039, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
6Division of Behavioral Medicine & Clinical Psychology, Cincinnati Children's Hospital Medical Center, CINCINNATI, OH
7Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
8Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
9Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
10Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Med. Ctr., Cincinnati, OH
11Center for Child Health Policy and Advocacy, Baylor College of Medicine, Houston, TX
12Texas Children's Hospital, Houston, TX
13Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, TX
14Department of Pediatrics, Division of Hematology, Oncology and Stem Cell Transplant, Ann %26 Robert Lurie Children's Hospital, Chicago, IL
15Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
16Division of Hematology, Oncology & Stem Cell Transplant, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
174th Floor Seashore House, Children's Hosp. of Phila. Division of Hematology, Philadelphia, PA
18The Children's Hospital of Philadelphia, Philadelphia, PA
19Program in Occupational Therapy, Washington University in St. Louis, School of Medicine, Clayton, MO
20Center for Innovation in Pediatric Practice, Division of Pediatric Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH
21Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH
22Center of Excellence in Sickle Cell Care, UCSF Benioff Children's Hospital Oakland, Oakland, CA
23Nemours Children’s Health System, Wilmington
24Indiana Hemophilia and Thrombosis Center, Indianapolis, IN
25Department of Pediatrics - Hematology/Oncology, Baylor College of Medicine, Houston, TX
26Cancer and Hematology Centers, Texas Children's Cancer and Hematology Centers, Houston, TX
27Children's Hospital Medical Center, Cincinnati, OH
28Cincinnati Children's Hospital, Cincinnati

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 demonstrate more COVID-19 pandemic-related distress are needed.

Disclosures: Quinn: Forma Therapeutics: Consultancy; Aruvant: Research Funding; Novo Nordisk: Consultancy; Emmaus Medical: Research Funding. Yates: Agios Pharmaceuticals: Current Employment. Badawy: PRECISIONscientia: Consultancy; Forma Therapeutics: Consultancy; Global Blood Therapeutics, Inc. (GBT): Consultancy; Vertex Pharmaceuticals Inc: Consultancy; ICON Plc: Consultancy; Sanofi Genzyme: 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: Forma Pharmaceuticals: Consultancy; Novartis Pharmaceuticals: Honoraria, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; 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.

*signifies non-member of ASH