Engaging Parents of Children With Sickle Cell Anemia and Their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)
Phase: NA
Conditions: Sickle Cell Anemia, Children, Only
Sponsor: Children's Hospital Medical Center, Cincinnati

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Title:
Engaging Parents of Children with Sickle Cell Disease and their 
Providers in Shared-Decision Making for Hydroxyurea (ENGAGE 
HU)
Principal Investigator:
Lori E. Crosby, PsyD.
Division of Behavioral Medicine and Clinical Psychology
Cincinnati Children’s Hospital Medical Center
Version: 
Version. 09
Date:
May 6, 2020

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1. ABSTRACT:
Sickle cell disease (SCD) is a genetic blood disorder that places children at risk for 
serious medical complications, early morbidity and mortality, and high healthcare utilization. In 
the U.S., SCD primarily affects African-American and Latino children. Hydroxyurea is the only 
disease-modifying treatment for this devastating and life-threatening disease. National 
Evidence-Based Guidelines recommend the use of a shared decision making approach to offer 
hydroxyurea to all children with SCD as early as nine months of age. Hydroxyurea uptake 
remains low because parents lack information about hydroxyurea and have concerns about its 
safety and potential long-term side effects (e.g. cancer, infertility, birth defects). Clinicians do not 
have the training or tools to facilitate a shared discussion with parents that provides medical 
evidence and considers parent preferences and values. The current study compares two 
methods for disseminating hydroxyurea guidelines and facilitating shared decision-making: the 
American Society of Hematology’s hydroxyurea clinician pocket guide (usual care method) and 
a clinician hydroxyurea shared decision-making toolkit (H-SDM toolkit). The specific aims of the 
study are to evaluate the effectiveness of the usual care dissemination method (clinician pocket 
guide) and the H-SDM clinician toolkit dissemination method on: parent report of decisional 
uncertainty (primary outcome chosen by parents of children with SCD), parent perception of 
experiencing shared decision-making, parent knowledge of hydroxyurea, the number of children 
offered hydroxyurea, hydroxyurea uptake (those with active prescriptions), and child health 
outcomes (pain, neurocognitive functioning, sickle cell related quality of life and healthcare 
utilization). Eligible children must be between the ages of 0 and 5.99 years and a candidate for 
hydroxyurea to participate. The trial will use a stepped-wedge design (clinic is the unit of 
randomization). The long-term objective of the research team is to improve the quality of care 
for children with SCD. We propose that suboptimal care for patients with SCD is preventable 
with the use of multicomponent dissemination methods if developed with key stakeholders and 
designed to address barriers to high quality care at multiple levels (patient, clinician, healthcare 
system, and community).
2. STUDY PURPOSE:
The purpose of the study is to answer the following research question: will use of the 
hydroxyurea shared decision making toolkit (H-SDM toolkit) dissemination method be more 
effective than the ASH clinician pocket guide dissemination method (usual care) at increasing 
clinicians’ ability to implement sickle cell specific evidence-based guidelines and 
improve/maintain patient-centered health outcomes?
3. BACKGROUND:
Sickle cell disease (SCD) is a genetic blood disorder that affects approximately 100,000 
individuals in the United States (US) (Hassell, 2010). Approximately 1 in 2500 babies born in the 
US has SCD, making it the most common disorder identified by newborn screening.(American 
Academy of Pediatrics, 2007) SCD is chronic and associated with early mortality (average 
lifespan of 40-50 years). A daily dose of oral hydroxyurea has been shown to significantly 
reduce the frequency of pain (two fold decrease) and serious medical complications: dactylitis, 
severe pain and swelling in the bones of the hands or feet (fivefold decrease), acute chest 
syndrome, a life-threatening condition where the lungs do not get the oxygen they need (three 

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fold decrease) (Wang et al., 2011). Children taking hydroxyurea require fewer transfusions and 
spend less time in the hospital for fewer dactylitis (50 fewer days), pain (100 fewer days), and 
acute chest syndrome (300 fewer days) (Wang et al., 2011). This medication may also protect 
organs from the effects of chronic sickling. 
Because the benefits of hydroxyurea outweigh the risks, in 2014, the National Heart, 
Lung, and Blood Institute (NHLBI) published Evidence-Based Guidelines for the Management of 
Sickle Cell Disease that recommended hydroxyurea therapy be offered to children with SCD as 
early as 9 months of age using a shared decision-making process. Prior to the release of the 
NHLBI guidelines, children with SCD were only offered hydroxyurea if they had frequent pain or 
other serious SCD-related complications.(National Heart, Lung and Blood Institute,2017)11,12 
In contrast, the new guidelines state that all children with the most severe genotypes (HbSS and 
HbS/β0thalassemia) be offered hydroxyurea. This has resulted in a change in practice, from a 
prevention model to an intervention model. It also means that the number of children eligible for 
hydroxyurea has significantly increased (about a 2-fold increase at our institution). 
Shared decision-making is one method of targeting the worries, fears and uncertainty 
noted by parents, which is one reason the NHLBI recommended its use. Currently, the only tool 
to assist providers with implementing hydroxyurea guidelines for SCD is a clinician pocket guide 
developed by the American Society of Hematology (ASH). Pocket guides are widely used and 
are usually rated as useful at the point of care by clinicians (Korn, Reichert, Simon, & Halm, 
2003; Siebens, Tucker, & Leander, 2004), but the majority of pocket guides only target clinician 
motivation. They do not provide training to increase motivation, guided practice in 
communication skills to elicit preferences that build clinician self-efficacy, audit and feedback to 
reinforce behavior change or decision support tools to help clinicians engage parents and 
support parents in decision-making.
We used Agency for Healthcare Research & Quality (AHRQ) funding to develop, test, 
and begin to disseminate a parent-centered, technology-enhanced decision-support toolkit, the 
Hydroxyurea Shared Decision Making Toolkit (H –SDM toolkit), to assist clinicians in 
implementing shared decision-making for hydroxyurea and parents in feeling more confident 
about their decision. This toolkit targets factors noted in the Cochrane reviews (Légaré et al., 
2010; Légaré et al., 2014) and the literature on behavior change (Godin, Bélanger-Gravel, 
Eccles, & Grimshaw, 2008), motivation, self-efficacy, and patient readiness; we hypothesize that 
this dissemination method will lead to increased shared decision-making, less parent 
uncertainty about the decision, increased offering of hydroxyurea, and ultimately improve 
hydroxyurea uptake. The current study proposes to address a critical gap in the literature by 
comparing two dissemination methods (ASH clinician pocket guide and the H-SDM toolkit) for 
improving adoption of the NHLBI guideline recommended practice of shared decision-making 
about hydroxyurea for children with SCD. We will also examine the impact of these 
dissemination methods on patient/parent-centered outcomes.
This project is significant because it will improve the quality of the evidence available for 
parents and clinicians to make an informed decision about hydroxyurea, thereby improving care 
and health outcomes for young children with SCD. There is high potential for study findings to 
be adopted into clinical practice and improve care delivery.  Furthermore, the project focuses on 
outcomes identified by parents and clinicians as important to a child’s health. 

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4.  STUDY DESIGN:
Overview
Cincinnati Children’s Hospital Medical Center (CCHMC) is the Coordinating Center (CC) 
for this multisite comparative effectiveness trial. As such, CCHMC will be overseeing the study 
conduct, regulatory and institutional review board (IRB) administration and compliance. CCHMC 
will not serve as a study site. 
We are proposing to use a Single (Central) IRB model to administer this project. Under 
this model, an ENGAGE HU site has the option to rely exclusively on the full review conducted 
by the CCHMC IRB provided a reliance agreement is in place.  The Site’s local IRB will not be 
involved in the approval of annual reviews and in any of the amendments to the 
study.  However, local sites and their IRB teams will be updated on study progress and 
amendments by the ENGAGE HU CC team.
Design
This study will be a stepped wedge cluster randomized controlled trial (RCT). In a 
stepped wedge design, each clinic begins to enroll patients using the usual care dissemination 
method. Then, each cluster, one-by-one, crosses over to using the H-SDM toolkit dissemination 
method (one cluster will crossover every 6 months).  Training for the H-SDM toolkit will begin 
during the last month of the Usual Care period for each cluster. Each cluster will enroll 
approximately 7-9 participants per time period. Enrollment will end 36 months after study 
initiation. 
A total of 174 participants (parents of young children with SCD) will be enrolled and 
complete measures at baseline, and 3 months later in-person or online via REDCap. Data on 
clinician offering of hydroxyurea, hydroxyurea uptake and healthcare utilization will be collected 
throughout the study. Fidelity will be assessed using a checklist and by reviewing audio 
recordings of clinic visits (20% of visits). 

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Study Flow -Chart:
Prior to 
Enrollment
Baseline &
Intervention
Final Visit
(3 months
 later)
36-39 months
Study Closeout
Total 174:  Obtain informed consent. Screen potential subjects by inclusion and exclusion 
criteria; obtain history, document.
Baseline assessments and administer study intervention
(SCD quality of life, ages & stages, health literacy, demographics, decisional conflict scale, 
dyadic option, hydroxyurea knowledge, COVID-19 attitudes, satisfaction with decision 
making)
Maintenance Assessment
Site follow-up survey
.
Enrollment
Arm 2 – HSDMT
87 subjects
Arm 1 - Usual Care
87 subjects
Final visit assessments of outcome measures and safety
(SCD quality of life, ages & stages, medication adherence, adverse events, COVID-19 
attitudes, use of decision aids)

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Specific Aim 1: Evaluate the effectiveness of the usual care dissemination method (clinician pocket 
guide) and the H-SDM toolkit dissemination method on: 1) parent report of decisional uncertainty for 
hydroxyurea (primary outcome chosen by parents of children with SCD - Effectiveness); and 2) parent 
report of experiencing shared decision making when talking with their clinician about hydroxyurea 
(Effectiveness), in a sample of children (0-5.99 years of age).   
Hypothesis 1: Compared to usual care (clinician pocket guide), the H-SDM toolkit 
dissemination method will result in an increase in the use of a shared decision making process 
for hydroxyurea, and parents feeling less uncertain in their decision about hydroxyurea.
Specific Aim 2: Evaluate the effectiveness of the usual care dissemination method (clinician pocket 
guide) and the H-SDM toolkit dissemination method on: 1) parent knowledge of hydroxyurea 
(Effectiveness); 2) children offered hydroxyurea (Reach); 3) children with an active hydroxyurea 
prescription (uptake – Effectiveness); and 4) child health outcomes: pain, neurocognitive functioning, 
sickle cell related quality of life and healthcare utilization (Effectiveness).
Hypothesis 2: Compared to usual care (clinician pocket guide), the H-SDM toolkit 
dissemination method will result in parents of children with SCD 0-5.99 years of age knowing 
more about hydroxyurea, more children offered and receiving hydroxyurea, and these children 
experiencing positive health outcomes.
Interventions
Usual Care: In this condition, sites will provide current guidelines for offering hydroxyurea and 
use the American Society of Hematology (ASH) pocket guide as a reference. ASH developed ‘The 
Hydroxyurea and Transfusion Therapy for the Treatment of Sickle Cell Disease’ clinician pocket guide 
based on the National Heart, Lung, and Blood Institute’s Evidence Based Management of Sickle Cell 
Disease: Expert Panel Report, 2014.’ The pocket guide recommends use of shared decision making. 
Hydroxyurea shared decision making toolkit (H-SDM toolkit): During the H-SDM toolkit 
condition, sites will develop methods for identifying Eligible Patients & Monitoring Progress, have the 
opportunity to use Implementation Tools, and will use the Visit Decision Aids. The H-SDM toolkit has 
four visit decision aids to support parents in their decision about hydroxyurea: pre-visit brochure, in-visit 
issue card, after-visit booklet and video narratives {videos of parents telling their story about how they 
made a decision about hydroxyurea). 
5. DURATION:
The project will begin in December 2017 and will last until November 2020. Each participant will 
be enrolled in the study for 3-7 months. Enrollment will be completed 36 months after the study begins. 
The final data report will be submitted for peer review to PCORI in February 2021.
6.  SELECTION AND RECRUITMENT OF PARTICIPANTS:
174 children diagnosed with sickle cell disease, 0-5.99 years of age, and their parents who 
receive care in one of the IRB-approved sickle cell sites will be enrolled in the study. Because the US 
SCD population consists of African Americans, African immigrants and Hispanic/Latinos, we anticipate 

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not all participants will have a social security or TIN number. We will assign a nine digit identification 
comprised of their participant ID and birthdate allowing us to track participant compensation.  
Inclusion Criteria
1.
Diagnosis: sickle cell disease
2.
Age: birth-5.99 years, inclusive
3.
Eligible for hydroxyurea (genotype SS, Sβ0Thal or other genotype + clinical complications)
4.
Child’s parent, legal guardian, or designated decision maker (caregiver) must participate in both 
study visits
5.
Child’s parent, legal guardian, or designated decision maker (caregiver) must able to read, 
understand, and speak English
Exclusion Criteria
1.
Parent/legal guardian has previously been approached OR made a decision about whether to 
initiate Hydroxyurea.
2.
Any and all other diagnoses or conditions which, in the opinion of the site investigator or 
hematologist, would prevent the patient from being a suitable candidate for the study.
3.
Sibling of participant actively enrolled in the study or enrolled in the past. 
Recruitment
Successful recruitment and retention of parents is essential to this study. To ensure successful 
recruitment, the Stakeholder Advisory Council will review and provide feedback on the 
recruitment/retention plan. 
We will consecutively enroll eligible young children with SCD who have been identified by 
provider referral or EMR review. Provider referral and EMR will be used to identify potentially eligible 
study participants (see informed consent section about partial waiver of authorization). The research 
team will approach the child’s provider to obtain approval before contacting the potential participant. 
Eligible patients will receive a letter, flyer by mail, or phone call. A research coordinator with 
experience recruiting patients with SCD will then either follow up by phone or approach families in 
clinic. Data will be collected on how families are recruited. Interested families will be consented in clinic 
or by phone (e-consent) and the baseline assessments scheduled. Study visits may be in-person or 
telehealth and will be scheduled at the family’s convenience (before/after clinic visits, evenings, and 
SCD events). If a parent/caregiver fails to complete baseline assessments within 30 days of consent, 
then the parent/caregiver may be rescreened, re-consented and asked to complete baseline 
assessments again. A participant may only be rescreened and re-consented one time.
Retention
Retention strategies will include: 1) scheduling visits at times convenient for the family (during 
clinic/telehealth visits); 2) “reminder” phone calls for visits or to complete assessments; 3) allowing 
questionnaires to be completed online, and 4) "check-in" calls to promote retention during the COVID-
19 pandemic . In addition, the rationale for two visits and the importance of follow-up will be reviewed 
with each family at baseline to engage them as partners in the research process. To prevent loss to 
follow up, we will also collect patient permission to contact two close family members or friends if the 
participant’s family is difficult to reach.

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7. PROCESS OF OBTAINING CONSENT:
The protocol includes a waiver of documentation of consent for clinicians participating in the 
trial. The protocol this protocol is eligible for waiver or alteration of required elements of the informed 
consent process for clinicians because the protocol meets all of the following criteria: 1) the research 
presents no more than “minimal risk” of harm to clinicians, 2) the research involves no procedures for 
which written consent is normally required outside of the research context. 
This protocol also includes a partial waiver of authorization for recruitment purposes. The 
purpose of this waiver is to allow the ongoing review of PHI for the purpose of identifying patients who 
need to be approached to obtain consent/authorization for research. This review will include all patients 
from birth to age 17 years 11 months old diagnosed with SCD receiving clinical care at the performance 
sites. Some of these patients may have discussed hydroxyurea with their provider in the past, but 
patients will only be considered eligible if they do not have an active hydroxyurea prescription (i.e. no 
filled prescriptions in the past six months). No identifying information will be re-used or disclosed prior 
to consent being obtained as noted below. Identifiers will be destroyed at the earliest opportunity 
consistent with the conduct of the research. 
The informed consent process will be initiated prior to the parent/caregiver agreeing to 
participate in the study and will continue throughout study participation. Discussion of risks and possible 
benefits of study participation will be provided to participants. Informed consent will be obtained by 
trained research staff and may be obtained in-person or via phone using the e-consent portal. Consent 
forms may be signed electronically using the e-consent portal, a secure web-based interface supported 
by the CCHMC Division of Biomedical Informatics in compliance with HIPAA designed to protect PHI in 
the electronic transfer and storage of the consent form. An IRB-approved consent form describing the 
study procedures and risks will be given or available to participants to view the from the e-consent 
portal. Participants are required to read and review the document or have the document read to her or 
him. Research staff will explain the research study to the participant and answer any questions that 
may arise. The parent/caregiver will sign or electronically sign the informed consent document prior to 
any study-related assessments. Parents/caregivers will be given the opportunity to discuss the study 
with family members or friends and think about it prior to agreeing to participate. They may withdraw 
consent at any time throughout the course of the study. A copy of the signed informed consent 
document will be mailed/e-mailed, given or available for download to parents/caregivers for their 
records. The rights and welfare of the family will be protected by emphasizing to parents/caregivers that 
the quality of their child’s clinical care will not be adversely affected if they decline to participate in this 
study. The consent process will be documented in the research and/or clinical record.  
8. STUDY PROCEDURES:
Clinics will either use the pocket guide or the H-SDM toolkit. The goal is to change care at the 
clinic level. In this way, all eligible patients will receive care consistent with the condition (usual care or 
intervention) regardless of whether they enroll in the study or not. Clinical teams will use tools from the 
dissemination methods during previsit planning, during the clinic visit where clinicians ask the parent to 
make a decision about initiating hydroxyurea, and afterwards to monitor progress (e.g., offering HU, 
documentation of parent decision, HU prescription, HU labs). 

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8a. Study Schedule
Screening

Total N=174 (n = 1-4 patients per month per site) 

Obtain informed consent 

Screen potential subjects by inclusion and exclusion criteria 

Obtain history, document 
Baseline period/Study intervention visit

Measures may be completed online or by phone before or after visit 

Administer clinician intervention in-person or via telehealth
Intervention visit follow-up

Mail/email/phone reminder for final visit (and to text pictures if have a 
hydroxyurea prescription)
Final Visit reminder telephone call (2 weeks prior to the visit)

Reminder call for final visit
 Final Visit (3 months after baseline; range 2-4 months)

Follow-up assessments of study endpoints and safety

Completed online or by phone
36 months – Site follow-up telephone call

Site staff complete follow-up survey with coordinating center
8b. For the usual care condition: 
All sites receive printed copies, the link to download copies, and the link to the app for the 
pocket guide to distribute to their clinicians. Site clinicians view a live or recorded didactic presentation 
reviewing the NHLBI guidelines for hydroxyurea. Sites will then develop or update their site-specific 
care guidelines for hydroxyurea, and a plan for implementing them.  A printed copy of the pocket guide 
will be made available in the clinic for reference.  Research staff will approach eligible patients/families 
prior to their clinic visit, provide informed consent (in-person or e-consent), and administer baseline 
assessments (15-30 minutes). If parents/caregivers prefer, some baseline assessments can be 
completed online. Clinicians discuss hydroxyurea.  Research staff administer remaining baseline 
assessments at end of the clinic visit (15-30 minutes) but parents/caregivers can complete these online 
if they prefer. After the baseline assessments are complete, clinicians/research staff document 
hydroxyurea offered and use of usual care (i.e. site-specific guidelines). The goal is to schedule the 
final visit to coincide with a routine clinic visit (4-6 months later); however, final visit assessments can 
be completed online (10-20 minutes).
8c. For the H-SDM toolkit condition:
All sites develop a systematic way of identifying eligible patients and documenting offering 
hydroxyurea. All sites receive didactic presentation on toolkit and virtual practice. Clinicians are trained 
on implementing visit decision aids and develop a process for systematically implementing them with 

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eligible patients. Research staff will approach eligible patients prior to their clinic visit, provide informed 
consent (in-person or e-consent), and administer baseline assessments (15-30 minutes). If 
parents/caregivers prefer, some baseline assessments can be completed online. Research/clinical staff 
give parents/caregivers previsit brochure and access to parent video narratives. Clinicians use in-visit 
and after visit booklet with eligible patients/families (10-20 minutes). Research staff administer 
remaining baseline assessments at end of the clinic visit (15-30 minutes) but parents/caregivers can 
complete these online if they prefer. After the baseline assessments are complete, clinicians/research 
staff document hydroxyurea offered and use of usual care (site-specific guidelines). The goal is to 
schedule the final visit to coincide with a routine clinic visit (3 months later); however, final visit 
assessments can be completed online (10-20 minutes).
8d. Baseline and Final Assessments
The following data will be collected from participants/families or clinical/research staff on their personal 
smart phones, tablets, or computers. Participants completing measures remotely will be encouraged to 
contact the study staff via phone or e-mail with any questions.
Assessment Strategy
Construct
Measure
Brief Description/ Psychometrics (GM-4)
Baseline 
Final Visit
Primary Outcomes
Parent reported 
decisional 
uncertainty 
Decisional Conflict 
Scale (DCS)(O’Connor)
 – E*
Measures uncertainty experienced when feeling uninformed 
about options, unclear about personal values, or unsupported in 
making a choice. Cronbach’s alpha of 0.96. 
X
Parent reported 
perception of 
shared decision-
making
Dyadic 
OPTION(Melbourne, 
Sinclair, Durand, 
Légaré, & Elwyn, 2010) 
– E*
Describes clinician behaviors to involve a patient/parent in 
decision-making. A total score is calculated which ranges from 0 
(no involvement) to 100 (maximal involvement). Dyadic OPTION 
scores correlate well with OPTION scale(Melbourne et al., 2011); 
1 item “My doctor and I made the decision together”(Légaré et 
al., 2010) - 
X
Secondary Outcomes
Parent reported 
Satisfaction with 
decision making
6 item survey – E*
3 items adapted from the empirical research related to the 
concept of procedural justice and 3 items assessing influence of 
faith on decision making.(Allen & Marshall, 2010; Cascardi, 
Poythress, & Hall, 2000) If the Cronbach’s alpha for these items 
is acceptable (≥ .70), ratings will be summed to obtain a total 
score; otherwise, items will be analyzed separately. 
X
Parent reported 
Hydroxyurea 
knowledge
8 item survey – E*
Hydroxyurea knowledge survey (8 items): developed based on 
the existing literature, the Ottawa Knowledge User Manual, 
parent and clinician stakeholders and used in our pilot 
work.(Walton et al., April 2016) If the Cronbach’s alpha for these 
items is acceptable (≥ .70), items will be summed to obtain a 
total score; otherwise, items will be analyzed separately. 
X
Hydroxyurea 
offered
1 item reported by 
research coordinator – 
R*
1 of 3 responses – completed by the research coordinator 
based on review of EMR data: hydroxyurea was not offered, 
offered, or previously prescribed. If not offered, coordinators will 
choose a reason why (i.e. not eligible because patient is on 
transfusions, not eligible because patient has comorbid 
condition, no time to offer, clinician forgot, ill visit, or an open 
field to enter another reason). This will be verified for recorded 
encounters using the audio files. 
X

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Assessment Strategy
Construct
Measure
Brief Description/ Psychometrics (GM-4)
Baseline 
Final Visit
Hydroxyurea 
uptake
Active hydroxyurea 
prescription –E*
1 item reported by the research coordinator. They will report 
whether patients enrolled in the study have an active 
prescription for hydroxyurea using the EMR (prescription in the 
last 6 months). 
X
X
Hydroxyurea 
adherence
Lab values & 
pharmacy refill records 
–E*
Labs reported by the research coordinator based on the EMR: 
1) fetal hemoglobin (HbF) level – fetal hemoglobin increases 
when taking hydroxyurea as prescribed; 2) absolute neutrophil 
count (ANC) – this lab decreases when taking hydroxyurea as 
prescribed; 3) MCV – this lab increased when taking 
hydroxyurea as prescribed.
X
 Parent report of        
Hydroxyurea   
adherence
Medical Adherence 
Measure (MAM) 
Subscale: Medication 
Adherence (Zelikovsky 
et al., 2008) –E*
9 item survey measuring adherence problems, extent of non-
adherence in pediatric populations. Nonadherence and late 
adherence are calculated as a percent (0% to 100%).
X
Parent report of 
SCD-specific 
quality of life and 
pain
Peds-QL SCD Module 
(Panepinto et al., 
2013) – E*
Measures several domains of health-related quality of life 
including pain impact, fatigue, pain management, emotions, 
communication and treatment adherence; Total Score; α = .95. 
X
X
Parent report of 
neurocognitive 
functioning
Ages & Stages 
Questionnaire(Squires, 
Bricker, & Potter, 
1997) – E*
Reliable, accurate developmental and social-emotional screener 
for children between birth and age 6. Cronbach’s alpha ranges 
from .60 to .85.
X
X
Healthcare 
utilization
Hospitalizations, 
emergency room 
visits, ill visits – E*
EMR data on the number of hospitalizations, ill visits, and 
emergency room visits in the 6 months prior to enrollment (if 
possible, some participants may be 9 months of age) and the 6 
months after enrollment.
X
X
Covariates
Demographics
Demographics survey
10 item survey assessing family demographics including patient 
and parent age, gender, race and ethnicity, socioeconomic 
status, insurance (public vs. private), and parent highest level of 
education completed.
X
Health Literacy
Newest Vital 
Sign(Weiss et al., 
2005)
Newest Vital Sign (3 minutes): tests literacy skills for both 
numbers and words and has been highly correlated with the 
REALM (Osborn et al., 2007). Cronbach’s alpha = >0.76
X
COVID-19 and 
telemedicine use 
survey
COVID-19 Exposure 
and Family Impact 
Survey (CEFIS) 
(Center for Pediatric 
Traumatic Stress, 
2020; Parmanto,, 
Lewis, Graham, & 
Bertolet, 2016 )
.
Various aspects of the COVID epidemic are likely to impact 
families and may influence the findings of research in pediatric 
health.  CEFIS was designed to be used in ongoing and new 
studies where COVID-19 may impact study outcomes.  It 
conceptualizes exposure to potentially traumatic aspects of 
COVID-19 and assesses the impact of the pandemic on the 
family. Items from the telemedicine usability questionnaire will 
assess the impact of telemedicine on care. 
X
X
Fidelity
Parent involvement 
in decision-making
Observed OPTION 
scale(Elwyn et al., 
2005) – I*
Observer quantifies clinician behaviors to involve a parent in 
decision-making. A total score is calculated which ranges from 0 
(no involvement) to 100 (maximal involvement). OPTION scores 
are reliable and valid(Elwyn et al., 2005). Each audiotaped clinic 
visit will be independently coded by two research coordinators 
X

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Assessment Strategy
Construct
Measure
Brief Description/ Psychometrics (GM-4)
Baseline 
Final Visit
to ensure high reliability [inter-observer agreement = 0.82 in Dr. 
Brinkman’s recent trial (Brinkman et al., 2013).
Intervention fidelity H-SDM toolkit fidelity – 
I*
Checklist to assess which components of the H-SDM toolkit 
used and to what extent.
X
Continued use of 
intervention
Follow-up survey - M
Survey to assess continued implementation of the guidelines 
and clinical characteristics of the sites to understand barriers 
and facilitators to maintaining implementation.
1-3 mo. 
after 
enrollment 
ends
*RE-AIM Model: R = Reach; E= Effectiveness, A = Adoption; I= Implementation; M = Maintenance
9. DATA/ANALYIS METHODS:
The data will be analyzed based on the intent-to-treat principle. All patients will remain in the arm 
of the study to which they were randomized, regardless of whether or not they receive the assigned 
dissemination method. 
9a. Randomization
This study will be a stepped wedge cluster randomized controlled trial (RCT). In a stepped 
wedge design, each clinic begins to enroll patients using the usual care dissemination method. Then, 
each cluster, one-by-one, crosses over to using the H-SDM toolkit dissemination method (one cluster 
will crossover every 7-9 months) (Légaré et al., 2010).  The order in which the clusters crossover is 
random (i.e., the cluster is the unit of randomization; see Figure 3) and will be managed by the study 
statisticians. Sites will be randomly assigned to 3 clusters (3-4 sites per cluster). The randomization will 
guarantee that each cluster has a site considered large, medium and small (<270 patients = small; 
>270 – 500 = medium; >501 = large). Training for the H-SDM toolkit will begin during the last month of 
the Usual Care period for each cluster. Each cluster will enroll approximately 7-9 participants per time 
period. 
Revised Study Design and Timeline
HSDMT = Hydroxyurea Shared Decision Making Toolkit; Usual Care = ASH clinician pocket guide

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9b. Missing Data
Missing data will be handled using multiple group multiple imputation (with M = 100 imputed 
datasets) (Cox, Smith, Brown, & Fitzpatrick, 2009) consistent with currently accepted methodological 
practice. (Godin, Bélanger-Gravel, Eccles, & Grimshaw, 2008).
9c. Aim 1 
Data from all participants at all time-points will be aggregated within treatment group 
assignment and analyzed in two steps. First, acknowledging that J = 3 sites (1 “small”, 1 “medium” and 
1 “large” site) each will be assigned to C = 3 clusters in a manner balanced by site size, we will assume 
response variable variance across the C = 3 clusters to be negligible, and response variable variation 
across the J = 11 total sites will be eliminated (i.e., the ICC = 0) following group mean centering of all 
analysis variables at their respective site means (Enders & Tofighi, 2007) (alternative methods, such as 
‘Type = Complex’ in Mplus, perform poorly if the number of nesting units, such as sites, is < 20). This 
will allow the effect of site-level clustering to be ignored and the effect of treatment group randomization 
to be assessed at the participant level without fear of inferential statistical test bias. Specifically, a 
multiple group comparison analysis will be performed in Mplus (Version 8) to assess significant 
response variable differences by group randomization. Further, missing data will be handled using 
multiple group multiple imputation (with M = 100 imputed datasets) (Graham, Olchowski, & Gilreath, 
2007) consistent with currently accepted methodological practice (Enders, 2011).  
9d. Aim 2
The secondary outcomes of hydroxyurea knowledge and child reported health outcomes will be 
evaluated using similar methods to eliminate site level variance prior to data analysis.  Specifically, we 
will analyze response variables (hydroxyurea offered, hydroxyurea uptake) as binary categorical and 
convert parameter estimates to odd ratios for interpretation. Specifically, for part one of Aim 2, simple 
logistic regression analyses will be performed on each of the M = 100 imputed datasets to determine 
significant differences between Toolkit and Usual Care after controlling for the covariates listed in Aim 
1. For part 2 of Aim 2, multiple-group SEM with covariates, as outlined in Aim 1 above, will be used to 
test for significant group differences in hydroxyurea knowledge, PEDS-QL sickle cell disease (SCD), 
Ages & Stages, Health Literacy, fetal hemoglobin levels and ANC. 
9e. Exploratory Analyses
For the third part of Aim 2, healthcare utilization variables (number of hospitalizations, ill visits, 
and emergency room visits) will be analyzed as count variables and examined in exploratory analyses. 
ER visits, hospitalizations, and ill visits count differences will be assessed with count variable analyses 
that first determine whether Poisson or negative binomial count distribution analyses are needed prior 
to testing for significant Toolkit versus Usual Care mean rate differences.
9f. Process Improvement
During the H-SDM toolkit period, data will be tracked on a monthly run charts (percent 
offered/percent eligible, percent with active prescription/percent eligible, and percent lab values in 
range, if H-SDM toolkit components were implemented). Run charts provide a graphic display of 
process performance over time to motivate and inform practice changes. We will convert run chart data 
to p-charts or control charts to determine if the process of offering hydroxyurea is under control 
(minimal variation in the data) and any special cause changes (i.e. factors that change the process 

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significantly). Upper and lower control limits will be calculated as 3 sigma from the mean (e.g. standard 
Shewhart chart method)(Langley et al., 2009). Any data point outside the control limits will be 
considered variation from a special cause. 
9g. Sensitivity Analyses
To better describe the study population and the effectiveness of the dissemination methods, the 
performance of the following subgroups will be compared with respect to outcome variables:
1.
Characteristics of participants who decide to enroll versus those who decline (Reach)
2.
Characteristics of drop-outs versus completers (Reach)
3.
Characteristics of clinicians who adopt shared decision-making versus those who do not 
(Adoption)
4.
Characteristics of sites who adopt the full H-SDM toolkit versus the core components (CRC will 
audit via a fidelity checklist. This will be a reported as a range [ e.g. 2-3 components]) 
(Adoption)
5.
Characteristics of settings that continue to implement guidelines versus those who do not as 
measured by the offering hydroxyurea measure (Maintenance)
In addition to the control covariates mentioned previously, we will examine whether certain 
characteristics affect the likelihood of treatment benefit (i.e., testing the interaction between subgroup 
variable and H-SDM toolkit group) using baseline subgroup variables that we believe may affect 
treatment outcomes. Primary subgroup variables will be selected based on evidence suggesting a 
moderating effect of these variables in the SDM literature (e.g., parent education, socioeconomic 
status) (W. B. Brinkman et al., 2011; Cox, Smith, Brown, & Fitzpatrick, 2009) or because we feel due to 
the nature of the H-SDM toolkit that these variables will impact effectiveness (e.g., parent health 
literacy, child health status). The outcomes assessed with HTE analyses will be the same as those 
assessed in the trial (e.g., decisional uncertainty etc.) (HT-4). 
Subgroup data will also be examined using advanced implementation science statistical 
approaches. We will use a combination of funnel charts, X-bar and S-charts to understand the 
implementation process and characteristics that may shift the process and to determine the relationship 
of these characteristics to outcomes. This will help us test and generate hypotheses that will inform 
future dissemination and implementation of the toolkit. For example, we will assess time to hydroxyurea 
prescription and possible predictors. We will also assess the relationship between COVID-19 impact 
and possible correlates (e.g. decisional conflict, satisfaction with decision making). 
9h. Fidelity Analysis
Fidelity checklist data will be analyzed via descriptive statistics. The criterion will be 80% 
meaning if a clinician or site does not complete 80% of the components required to implement the 
intervention, this indicates that the clinician or site needs to be retrained. A video conference call will be 
set up within one week of the determination (less than 80%) and members of the CC training team will 
meet with the clinician or site for up to 1 hour of training. The next 3 encounters will be reviewed for 
fidelity to make sure the clinician or site is now implementing 80% of the required components. Data 
from the site survey will also be summarized with descriptive statistics specifically, a description of 
which sites continue to use which intervention components and any differences between those 
sites/clinicians continuing to implement the intervention and those who do not.

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10. FACILITIES AND PERFORMANCE SITES:
The Performance centers will be added to the IRB protocol once the site has been determined 
as feasible for study participation. Performance sites will obtain Central or local IRB before any study 
activities are initiated. The Coordinating Center will be located at CCHMC.
11. POTENTIAL BENEFITS:
There is the potential for participants to receive a benefit from study involvement. First, parental 
knowledge about hydroxyurea may increase. Second, parents reviewing the video narratives may learn 
from other parents’ experiences and insights. Third, there is the potential for participants to feel more 
supported in the decision-making process thereby improving their relationship with their health care 
providers. 
There is also the potential for this study to impact future sickle cell clinical care and research. 
Due to the study design, all eligible patients at the sites will receive some level of intervention and this 
could be beneficial. It is also possible that information learned in this study may be used to promote 
shared decision making for other SCD treatments.  
12. POTENTIAL RISKS, DISCOMFORTS, INCONVENIENCES AND PRECAUTIONS:
Risks for participation in the study are minimal; however, a Data Safety and Monitoring Board 
(DSMB) will be convened to provide oversight of study conduct, study progress, and adverse event 
reports by the PI and/or site PIs. Participants might be slightly inconvenienced by participating in the 
study over 6 months. Questionnaires used in the protocol represent minimal risk to enrolled participants; 
however, participants will be advised that they can skip any items or stop at any time. Also, parents may 
not want to be audiotaped and can refuse participation in this part of the study but remain enrolled in the 
larger study. 
There is a minimal risk from inadvertent and unauthorized release of PHI to individuals outside 
the research team. Unanticipated problems will be documented and reported according to IRB and 
PCORI guidelines/standards. Unanticipated problems will be recorded in the data collection system 
throughout the study. 
An adverse event (AE) for the current study is an unfavorable medical occurrence in a 
participant (parent or child) temporally associated with the subject’s participation in the research, 
whether or not considered related to the subject’s participation in the research. A serious adverse event 
(SAE) may or may not result in death, be life threatening, or require hospitalization. The Medical 
Monitor and the Study PI will be responsible for determining whether an AE or SAE is expected or 
unexpected.  AE and SAE events with start dates occurring any time after informed consent is obtained 
until 7 (for non-serious AEs) or 30 days (for SAEs) after the last day of study participation will be 
reported according to IRB and PCORI guidelines.  Research staff will inquire about the occurrence of 
AE/SAEs since the last visit. Events will be followed for outcome information until resolution or 
stabilization.
13. RISK/BENEFIT ANALYSIS:
This study will subject participants to minimal risk, a risk level that is no greater than that 
encountered in a routine behavioral assessment and clinical care. The interventions and questionnaires 

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have been used in research without any reported negative effects. Knowledge gained in this study has 
the potential to improve treatment of SCD by enhancing shared decision making and hydroxyurea 
uptake that, consequently, would improve treatment outcomes in this population. 
14. DATA AND SAFETY MONITORING:
In addition to the PI’s responsibility for oversight, study oversight will be under the direction of a 
Data and Safety Monitoring Board (DSMB) composed of members with expertise in shared decision 
making, hematology, a behavioral psychologist, biostatistician, parent of a young child with SCD and an 
adult patient living with SCD. The DSMB will meet two times per year to assess safety and efficacy data 
(if applicable), study progress, and data integrity for the study.  If safety concerns arise, more frequent 
meetings may be held.  The DSMB will operate under the rules of a PCORI-approved charter that will 
be approved at the organizational meeting of the DSMB.  At this time, most data elements that the 
DSMB needs to assess will be clearly defined.  The DSMB will provide recommendations to PCORI 
and the PI.  
Interim Analyses and Stopping Rules
Given that this is a three year study using a stepped wedge design with new sites coming on 
throughout the three years, the primary concern for the interim analysis is futility or worsening of 
decisional outcomes. The statistician will conduct an interim analysis of futility once data on 
approximately n = 32 participants per arm (Toolkit vs. Usual Care) is available (given proper missing 
data handling). The O’Brien and Fleming (1979) group sequential method, as operationalized in SAS 
(version 9.2) PROC SEQDESIGN (SAS Institute, Inc., 2009) was used to calculate the standardized 
(Wald Z) test statistic values that quantify futility. The O’Brien-Fleming procedure was conducted 
assuming: 1) a mean DCS score difference between the Toolkit and Usual Care arms of 14-points 
quantifies a clinically meaningful difference (O’Connor, A.M., 1993, p.6) , 2) based on previous 
research (O’Connor, 1995, p. 28) a DCS total score standard deviation of 5.6 is anticipated in both 
arms, 3) data would be analyzed at two stages: midpoint and final analysis (i.e., nstages = 2), and 4) a 
two-sided test with (α = 0.05 & β = 0.10 [power = 0.90]) was appropriate for O’Brien-Fleming boundary 
calculations. Results showed the boundary for the mid-point interim analysis will be Z = + 2.79651 with 
p = .0026 (2-tail), and the boundary for the final analysis will be Z = 1.97743 with p = .024 (2-tail). Only 
if statistical evidence exists that the treatment group’s decision-making according to the DCS is 
significantly worse than the control condition at the interim assessment, a second informal assessment 
of futility using the same boundary value (Z = + 2.79651) will be undertaken. Specifically, assuming at 
the interim analysis, a mean DCS difference between Toolkit and Usual Care: 1) of 14 points or greater, 
2) with a Z test statistic > 2.79651, and 3) indicating the Usual Care group shows greater decision 
certainty and less delay, a second futility analysis will examine differences between the Toolkit and 
Usual Care groups on a binary indicator of receipt of a prescription for hydroxyurea (1=yes, 0=no). 
Results from both the psychological (DCS score differences) and behavioral (binary receipt of a 
hydroxyurea prescription) futility analyses, both using a boundary value of (Z > 2.79651) for rejecting 
the H0: of no group differences, will be reported to the Data Safety Monitoring Board (DSMB) who will 
recommend either that the trial continue or be terminated early due to both the psychological and 
behavioral futility criteria having been met.

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15. PRIVACY AND CONFIDENTIALITY:
Privacy
The informed consent process will address various aspects of participant privacy including the 
level of control over the circumstances and extent of sharing one’s personal information. As stated 
above, participants will only be approached by study personnel after permission from their provider. 
The nature of the intervention (e.g. clinic-based) leads to a loss of privacy as research and other clinical 
staff may know that they discussed hydroxyurea treatment with their provider. During the informed 
consent process, the study personnel will explain these risks. At that time, the potential participant may 
1) decline to participate or 2) agree to participate with the understanding that they may withdraw at any 
time. This will give the participant control over the circumstances of sharing their personal information. 
Study personnel will screen all questionnaires for completeness; participants are allowed to skip any 
survey questions they do not wish to answer, thereby providing additional control over the extent of 
sharing personal information.
Confidentiality
Individual data will not be available to anyone not directly associated with the study. All study 
personnel have been trained in data safety and monitoring, privacy and confidentiality, minimizing risks 
related to loss of privacy and confidentiality.  The performance of research personnel will be monitored 
to ensure the strictest standards. 
15.1 Data De-Identification
All data files will use unique study assigned identifier codes. Electronic data files (e.g. REDCap 
data) will be password protected with access limited to study personnel.
15.2     Data Storage
Informed consent documents and case report forms will be maintained in locked storage 
cabinets within the PI’s locked office space at each site. Consent and permission forms will be kept 
separate from participant’s data. Only the study staff will have access to the keys to the cabinets. 
Medical chart data will be collected by trained study staff under the supervision of the site PI. The full 
study database will be maintained on CCHMC’s web-based server. 
15.3 Data Quality
Source documents in this study will consist of Case Report Forms, parent measures (pdf or 
RedCap electronic data capture system, audio recordings of interventions, and Case Report Forms - 
CRFs). All parent data not collected directly from the parent in REDCap completed via pdf will be 
entered by trained research coordinators. Specific data structure routines will be utilized including 
double entry. Data quality will be monitored by random inspection by the CC independent monitor for 
the first 3-5 participants at each site and then quarterly. Inter-rater reliability among research 
coordinators will be computed for patient chart review. Any problems with reliability will be addressed 
with further training for the research coordinators. Discrepancies will be resolved by checking source 
data (pdf of measure or case report form) and if necessary, by returning to patient charts to correct any 
inaccuracies. 
16. COST OF PARTICIPATION
There are no costs for participants or third party payers for this study.  

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17. PAYMENT FOR PARTICIPATION
17a.Baseline Assessments
If parents/caregivers choose to complete some measures prior to the baseline visit, a ClinCard 
will be mailed to them with $20. The remainder of the compensation ($20) will be loaded onto their 
ClinCard after completion of the baseline visit.
Parents/caregivers completing all measures at the baseline visit will receive $40 compensation for 
their time and participation.
17b. Sending text messages/emails of prescription refills
Parents/caregivers will receive $5 compensation for sending pictures via text message or email 
of their prescription refills to a cell phone or an email address assigned to the study and managed by 
the Coordinating Center. 
17c. Final Visit Assessments
Parents/caregivers will receive $40 compensation for their time and participation for completing 
measures during the final visit (in-person or online).
17d.Transportation Hardship
In instances where parents/caregivers report financial hardship interfering with the ability to 
attend the Final visit (greater than 25 miles from the site, no bus route available, no insurance-related 
transportation available), the site CRC upon consultation with the Coordinating Center may make a 
determination to provide additional compensation to cover transportation to the study visit (e.g. cover 
the cost of a cab/Uber).  
17e. ClinCard Compensation
All compensation will be in the form of a ClinCard – a reloadable debit Master Card. The 
Coordinating Center will send clinical sites ClinCards with no money loaded. Site research staff will 
collect ClinCard paperwork and scan it to CCHMC. CCHMC will register all participants in the ClinCard 
system and be responsible for selecting visit payments to be loaded onto ClinCards and approving all 
payments. 
Per CCHMC ClinCard policy, for remote visits, the CRC will obtain and record the information 
on a W-9. 
–
In these instances, under Part II Certification of the W-9 the CRC will note “information 
obtained verbally [or electronically], no signature obtained.”
–
Initial and date the W-9 indicating you obtained the information verbally or electronically.
The CRC will record participant information directly onto electronic PDF form W9 and use the signature 
feature in Adobe Reader to add participant information. The CRC will be asked to complete the 
“ClinCard Payment Tracker for No In-Person Visits” to track payments and submit this to the study 
coordinating center each month. 

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