-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

1574 Addressing Recruitment Challenges in the Engage-HU Trial in Young Children with Sickle Cell Disease

Program: Oral and Poster Abstracts
Session: 901. Health Services Research—Non-Malignant Conditions: Poster I
Hematology Disease Topics & Pathways:
sickle cell disease, Diseases, Pediatric, Hemoglobinopathies, Study Population
Saturday, December 5, 2020, 7:00 AM-3:30 PM

Anna M Hood, PhD1*, Heather Strong, PhD2*, Cara Nwankwo, BA3*, Yolanda Johnson, MLS4*, Constance A Mara, PhD2*, Lisa M Shook, DHPE5,6*, William Brinkman, MD, MEd7*, Francis J Real, MD, MEd7*, Melissa Klein, MD, MEd7*, Allison A. King, MD, MPH, PhD8, Cecelia Calhoun, MD9, Kim Smith-Whitley, MD10, Susan E Creary, MD11,12, Maria T Britto, PhD13*, Kay Linn Saving, MD14,15, Connie M. Piccone, MD16, Jean L. Raphael, MD, MPH17, Emmanuel Volanakis, MD18, Aimee K Hildenbrand, PhD19*, Steven K Reader, PhD20*, Sohail Rana, MD21*, Lynne D. Neumayr, MD22, Amber M Yates, MD23,24, Sherif M. Badawy, MD25,26, Alexis A. Thompson, MD27,28, Amy Sobota, MD, MPH29,30, Emily Riehm Meier, MD, MSHS31, Charles T. Quinn, MD6,32 and Lori E. Crosby, PhD33

1Great Ormond Street Institute of Child Health, University College London, Cincinnati, OH
2Division of Behavioral Medicine & Clinical Psychology, Cincinnati Children's Hospital Medical Center, CINCINNATI, OH
3Oklahoma State University, Stillwater, OK
43333 Burnet Ave, MLC 7039, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
5College of Medicine/Department of Pediatrics, University of Cincinnati, Cincinnati, OH
6Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
7Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH
8Washington University School of Medicine, St. Louis, MO
9Division of Pediatric Hematology/Oncology, Washington University School of Medicine in St Louis, St Louis, MO
104th Floor Seashore House, Children's Hosp. of Phila. Division of Hematology, Philadelphia, PA
11Center for Innovation in Pediatric Practice, Division of Pediatric Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH
12Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH
13Adolescent and Transition Medicine, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
14Children's Hosp. of Illinois St. Francis Medical Center, Peoria, IL
15University of Illinois College of Medicine- Peoria, Peoria, IL
16University Hospitals Rainbow Babies and Children's Hospital, Cleveland, OH
17Center for Child Health Policy and Advocacy, Baylor College of Medicine, Houston, TX
18Vanderbilt University, Nashville, TN
19Center for Healthcare Delivery Science, Nemours Children’s Health System, Wilmington, DE
20Nemours Children’s Health System, Wilmington
21Howard University, Washington DC, DC
22Division of Hematology/Oncology, University of California San Francisco, Oakland, CA
23Texas Children's Hospital, Houston, TX
24Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, TX
25Department of Pediatrics, Division of Hematology/Oncology/Stem Cell Transplant, Ann & Robert Lurie Children's Hospital, Chicago, IL
26Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago
27Northwestern University, Chicago, IL
28Division of Hematology, Oncology & Stem Cell Transplant, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
29Pediatric Hematology and Oncology, Boston Medical Center, Boston, MA
30Pediatric Hematology/Oncology, Boston Medical Center / Boston University, Boston, MA
31Indiana Hemophilia and Thrombosis Center, Indianapolis, IN
32Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH
33Cincinnati Children's Hospital, Cincinnati

Background: Sickle cell disease (SCD) is a genetic disorder that causes significant medical and neurologic morbidity in children. Hydroxyurea (HU) is the primary medication used to prevent these complications. National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering HU to children as young as 9 months of age with SCD (HbSS or HbSB0 thalassemia) using a shared decision-making approach. Although HU has proven efficacious it remains underutilized and caregivers report that they are not always actively involved in the decision to initiate this therapy. Reasons for limited HU uptake likely include lack of clinician knowledge and training and negative caregiver perceptions. Thus, we developed the Engage-HU trial as a novel approach to address HU utilization barriers. A critical consideration for this trial was that SCD primarily affects individuals of African and Hispanic/Latino descent. In these minority populations, intervention trials are sometimes terminated early because of recruitment difficulties related to mistrust of research, caregiver burden, and transportation issues. As such, the Engage-HU trial design included best-practice strategies for recruiting people of color in research. This study describes these strategies, the initial recruitment plan, preliminary recruitment outcomes and strategies, and our procedural adaptations.

Study Design and Methods: Engage-HU is a randomized control trial (NCT03442114) to assess how clinicians can engage caregivers in a shared discussion that considers their values and preferences and includes evidence that supports HU. Engage-HU compares two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD: 1) the American Society of Hematology Pocket Guide, and 2) the HU Shared-Decision Making (H-SDM) Toolkit. The study aims to recruit 174 caregivers and evaluate the effectiveness of the dissemination methods on patient-centered outcomes (caregiver confidence in decision-making and perceptions of experiencing shared decision-making) as well as HU uptake and child health outcomes. Eligible children are aged 0 to 5 years, candidates for HU, and their caregiver has not made a decision about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a stepped-wedge design. Data will be analyzed based on the intent-to-treat principle. All participants will remain in the arm of the study to which they were randomized, regardless of whether or not they receive the assigned dissemination method. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using a linear mixed effects regression model with a robust variance estimator and maximum likelihood estimation with observations clustered within site.

The Engage-HU trial includes adaptations to increase recruitment such as tailored messaging, a relational recruitment approach, streamlined data collection, and a Stakeholder Advisory Committee. However, even with these adaptations, the first 6-months of the trial yielded lower than anticipated recruitment. Rather than terminate the trial or accept low enrollment, the research team implemented a series of recruitment strategies to address barriers including helping to improve research coordinator knowledge of the study purpose and adjusting no-show and follow-up procedures (e.g., calls to families after missed appointments and reminder calls before appointments). Site clinicians and clinic staff were provided with additional training so they could give more context about Engage-HU to caregivers and the study principal investigator led monthly “all coordinator” calls to provide support by sharing updates and experiences about successful recruitment. Implementation of these strategies resulted in triple the number of enrollments over the next 7-months compared to the previous 6-months (Table 1). Our goal in sharing this information is to provide lessons learned that can be implemented in future trials with the systematically underserved SCD population. It is also anticipated that methods described here may also inform clinical approaches to better engage caregivers of young children around critical clinical conversations, such as initiating medications like HU.

Disclosures: King: Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Bioline: Consultancy; RiverVest: Consultancy; Novimmune: Research Funding; Celgene: Consultancy; Tioma Therapuetics: Consultancy; Amphivena Therapeutics: Research Funding; WUGEN: Current equity holder in private company; Cell Works: Consultancy; Incyte: Consultancy. Smith-Whitley: Prime: Other: Education material; Celgene: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Neumayr: Emmaus: Consultancy; Bayer: Consultancy; CTD Holdings: Consultancy; Pfizer: Consultancy; ApoPharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; Micelle: Other: Site principal investigator; GBT: Other: Site principal investigator; PCORI: Other: site principal investigator; Novartis: Other: co-investigator; Bluebird Bio: Other: co-investigator; Sangamo Therapeutics: Other; Silarus: Other; Celgene: Other; La Jolla Pharmaceuticals: Other; Forma: Other; Imara: Other; National Heart, Lung, and Blood Institute: Other; Health Resources and Services Administration: Other; Centers for Disease Control and Prevention: Other; Seattle Children's Research: Other. Yates: Novartis: Research Funding. Thompson: Beam: Consultancy; Novartis: Consultancy, Honoraria, Research Funding; Agios: Consultancy; BMS: Consultancy, Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; bluebird bio, Inc.: Consultancy, Research Funding.

*signifies non-member of ASH