Children, Only, Sickle Cell Anemia
Conditions
Keywords
shared decision making, parent-provider communication, hydroxyurea, sickle cell disease
Brief summary
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent values and preferences). The study will compare two methods to help clinicians facilitate this-a clinician pocket guide and a clinician hydroxyurea shared decision making toolkit-in a group of parents of children ages 0-5 with sickle cell disease. The investigators hope that both methods lead to parents reaching a high-quality, well-informed decision. In addition, the team hopes to demonstrate that parents who experience a shared decision will have lower anxiety and decisional uncertainty. The researchers also expect these parents to be more likely to choose hydroxyurea and that their children will have less pain, fewer hospitalizations, better developmental outcomes, and higher quality of life. The project team hopes to show that the toolkit method is easy for clinicians to use and gives parents the support needed to make an informed decision.
Detailed description
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 one of 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 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. The investigators 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).
Interventions
BEHAVIORALHydroxyurea SDM Toolkit
Implementation tools and visit decision aids
BEHAVIORALClinician Pocket Guide
current hydroxyurea protocol and ASH pocket guide
Sponsors
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
NONE
Intervention model description
Stepped Wedge Randomized Clinical Trial
Eligibility
Sex/Gender
ALL
Age
1 Months to 5 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosis: sickle cell disease 2. Age: birth-5 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Decisional Conflict | Baseline - after shared discussion with clinician | Decisional Conflict Scale (DCS) is a 16-item parent-completed survey that measures uncertainty experienced when feeling uninformed about options, unclear about personal values, or unsupported in making a choice. Parents report their level of agreement with each item using a 5 point likert scale (0=strongly agree to 4=strongly disagree). For the total score, items are summed, divided by 16, and multiplied by 25. All subscores consist of 3 items except the Effective Decisions subcore (4 items) that are summed, divided by the number of items (3 or4) and multiplied by 25. Scores range from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice) on the total score and all subscores. Thus, a higher score indicates a high decisional conflict. |
| Dyadic OPTION | Baseline visit - after shared discussion with clinician | Dyadic OPTION 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). Higher scores indicate that the patient/parent has higher shared decision making competencies. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Ages & Stages Questionnaire - Gross Motor Subscale | After discussion with clinician | This questionnaire is a reliable, accurate developmental and social-emotional screener for children between birth and 6 years of age, with a Cronbach α = .60 to .85. Scores range from 0 to 60, with higher scores indicating that the child's development is on schedule. |
| Hydroxyurea Offered | From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months | 1 of 3 responses - completed by the research coordinator based on review of electronic medical record (EMR) data: hydroxyurea was not offered, offered, or previously prescribed. |
| Hydroxyurea Knowledge | After shared discussion with clinician | Eight-item survey developed based on the existing literature, the Ottawa Knowledge User Manual, and parent and clinician stakeholders and used in our pilot work. Items are summed to obtain a total score ranging from 0 to 9, with higher scores indicating more knowledge. |
| Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score | After shared discussion with clinician | Parent report of sickle cell disease (SCD)-specific quality of life (QOL) and pain, as measured by the Pediatric Quality of Life (PedsQL) SCD Module, which assesses several domains of health-related quality of life (HRQOL), including pain impact, fatigue, pain management, emotions, communication, and treatment adherence. Scores range from 0 to 100, with higher scores indicating higher HRQOL or higher functioning. |
| Satisfaction With Decision-Making | Baseline after the shared discussion | Eight-item survey adapted from the Satisfaction With Decision scale 41 (4 items) and the Agency for Healthcare Research and Quality's Consumer Assessment of Healthcare Providers and Systems survey related to patient experience of care (4 items). 42 Items are summed to obtain a total score ranging from 0 to 28, with higher scores indicating higher satisfaction. |
Countries
United States
Participant flow
Recruitment details
Since children were 0-5 years of age, parents completed all measures and were considered study participants. Study participants were recruited based on how they met inclusion criteria assessed by Clinical Research Coordinators and approved by participating physicians at 12 academic medical centers between July 2018 and November 2021.
Pre-assignment details
This study was originally designed as a stepped-wedge cluster randomized trial design. However, the study was amended to randomize participants instead of facilities, resulting in a parallel randomized controlled clinical trial. Of 176 enrolled participants,174 were allocated to either the Usual Care or Toolkit condition (quasi-experimental design), but only 137 completed the study. While parents completed the surveys and provided feedback, only children were enrolled in the study.
Participants by arm
| Arm | Count |
|---|---|
| Clinician Pocket Guide 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.'
Clinician Pocket Guide: current hydroxyurea protocol and ASH pocket guide | 166 |
| Hydroxyurea SDM Toolkit (H-SDM) 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).
Hydroxyurea SDM Toolkit: Implementation tools and visit decision aids | 126 |
| Total | 292 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Guardianship Change | 0 | 1 |
| Overall Study | Lost to Follow-up | 13 | 13 |
| Overall Study | Physician Decision | 2 | 2 |
| Overall Study | Protocol Violation | 0 | 2 |
| Overall Study | Withdrawal by Subject | 1 | 3 |
Baseline characteristics
| Characteristic | Clinician Pocket Guide | Hydroxyurea SDM Toolkit (H-SDM) | Total |
|---|---|---|---|
| Age, Continuous Child | 1.05 years STANDARD_DEVIATION 1.333 | 1.20 years STANDARD_DEVIATION 1.547 | 1.11 years STANDARD_DEVIATION 1.425 |
| Age, Continuous Parent/Guardian | 30.00 years STANDARD_DEVIATION 6.891 | 30.32 years STANDARD_DEVIATION 7.98 | 30.13 years STANDARD_DEVIATION 7.345 |
| Race (NIH/OMB) Child American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Child Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Child Black or African American | 79 Participants | 57 Participants | 136 Participants |
| Race (NIH/OMB) Child More than one race | 4 Participants | 4 Participants | 8 Participants |
| Race (NIH/OMB) Child Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Child Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Child White | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Parent/Guardian American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Parent/Guardian Asian | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Parent/Guardian Black or African American | 74 Participants | 56 Participants | 130 Participants |
| Race (NIH/OMB) Parent/Guardian More than one race | 6 Participants | 3 Participants | 9 Participants |
| Race (NIH/OMB) Parent/Guardian Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Parent/Guardian Unknown or Not Reported | 2 Participants | 2 Participants | 4 Participants |
| Race (NIH/OMB) Parent/Guardian White | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment United States | 166 participants | 126 participants | 292 participants |
| Sex: Female, Male Child Female | 45 Participants | 32 Participants | 77 Participants |
| Sex: Female, Male Child Male | 38 Participants | 30 Participants | 68 Participants |
| Sex: Female, Male Parent/Guardian Female | 76 Participants | 60 Participants | 136 Participants |
| Sex: Female, Male Parent/Guardian Male | 7 Participants | 2 Participants | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 94 | 0 / 80 |
| other Total, other adverse events | 3 / 94 | 2 / 80 |
| serious Total, serious adverse events | 5 / 94 | 1 / 80 |
Outcome results
Primary
Decisional Conflict
Decisional Conflict Scale (DCS) is a 16-item parent-completed survey that measures uncertainty experienced when feeling uninformed about options, unclear about personal values, or unsupported in making a choice. Parents report their level of agreement with each item using a 5 point likert scale (0=strongly agree to 4=strongly disagree). For the total score, items are summed, divided by 16, and multiplied by 25. All subscores consist of 3 items except the Effective Decisions subcore (4 items) that are summed, divided by the number of items (3 or4) and multiplied by 25. Scores range from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice) on the total score and all subscores. Thus, a higher score indicates a high decisional conflict.
Time frame: Baseline - after shared discussion with clinician
Population: All parents with data for baseline measures (146) were included in the analyses, whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Clinician Pocket Guide | Decisional Conflict | Support | 21.2 score on a scale | Standard Deviation 24.4 |
| Clinician Pocket Guide | Decisional Conflict | DCS Total Score | 22.9 score on a scale | Standard Deviation 20.3 |
| Clinician Pocket Guide | Decisional Conflict | Uncertainty | 26.9 score on a scale | Standard Deviation 25.4 |
| Clinician Pocket Guide | Decisional Conflict | Informed | 20.8 score on a scale | Standard Deviation 20.2 |
| Clinician Pocket Guide | Decisional Conflict | Values Clarity | 23.3 score on a scale | Standard Deviation 24.4 |
| Clinician Pocket Guide | Decisional Conflict | Effective Decisions | 22.3 score on a scale | Standard Deviation 21 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | Values Clarity | 21.0 score on a scale | Standard Deviation 21.1 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | Support | 17.9 score on a scale | Standard Deviation 16.8 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | Informed | 16.8 score on a scale | Standard Deviation 14.4 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | DCS Total Score | 19.8 score on a scale | Standard Deviation 17.1 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | Effective Decisions | 19.6 score on a scale | Standard Deviation 20.3 |
| Hydroxyurea SDM Toolkit (H-SDM) | Decisional Conflict | Uncertainty | 23.5 score on a scale | Standard Deviation 21.5 |
Primary
Dyadic OPTION
Dyadic OPTION 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). Higher scores indicate that the patient/parent has higher shared decision making competencies.
Time frame: Baseline visit - after shared discussion with clinician
Population: 145 parents with data for baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Clinician Pocket Guide | Dyadic OPTION | 39.8 score on a scale | Standard Deviation 6.7 |
| Hydroxyurea SDM Toolkit (H-SDM) | Dyadic OPTION | 40.3 score on a scale | Standard Deviation 5.8 |
Secondary
Ages & Stages Questionnaire - Gross Motor Subscale
This questionnaire is a reliable, accurate developmental and social-emotional screener for children between birth and 6 years of age, with a Cronbach α = .60 to .85. Scores range from 0 to 60, with higher scores indicating that the child's development is on schedule.
Time frame: After discussion with clinician
Population: The data of 120 children with baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Clinician Pocket Guide | Ages & Stages Questionnaire - Gross Motor Subscale | 50.9 score on a scale | Standard Deviation 14 |
| Hydroxyurea SDM Toolkit (H-SDM) | Ages & Stages Questionnaire - Gross Motor Subscale | 51.9 score on a scale | Standard Deviation 11 |
Secondary
Hydroxyurea Knowledge
Eight-item survey developed based on the existing literature, the Ottawa Knowledge User Manual, and parent and clinician stakeholders and used in our pilot work. Items are summed to obtain a total score ranging from 0 to 9, with higher scores indicating more knowledge.
Time frame: After shared discussion with clinician
Population: 145 parents with data for baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Clinician Pocket Guide | Hydroxyurea Knowledge | 6.3 score on a scale | Standard Deviation 1.7 |
| Hydroxyurea SDM Toolkit (H-SDM) | Hydroxyurea Knowledge | 6.1 score on a scale | Standard Deviation 1.5 |
Secondary
Hydroxyurea Offered
1 of 3 responses - completed by the research coordinator based on review of electronic medical record (EMR) data: hydroxyurea was not offered, offered, or previously prescribed.
Time frame: From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months
Population: 143 children with data for baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Clinician Pocket Guide | Hydroxyurea Offered | Offered | 61 Participants |
| Clinician Pocket Guide | Hydroxyurea Offered | Not Offered | 19 Participants |
| Clinician Pocket Guide | Hydroxyurea Offered | Previously Prescribed | 3 Participants |
| Hydroxyurea SDM Toolkit (H-SDM) | Hydroxyurea Offered | Offered | 32 Participants |
| Hydroxyurea SDM Toolkit (H-SDM) | Hydroxyurea Offered | Not Offered | 25 Participants |
| Hydroxyurea SDM Toolkit (H-SDM) | Hydroxyurea Offered | Previously Prescribed | 3 Participants |
Secondary
Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score
Parent report of sickle cell disease (SCD)-specific quality of life (QOL) and pain, as measured by the Pediatric Quality of Life (PedsQL) SCD Module, which assesses several domains of health-related quality of life (HRQOL), including pain impact, fatigue, pain management, emotions, communication, and treatment adherence. Scores range from 0 to 100, with higher scores indicating higher HRQOL or higher functioning.
Time frame: After shared discussion with clinician
Population: 121 children with data for baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Clinician Pocket Guide | Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score | 82.5 score on a scale | Standard Deviation 15.5 |
| Hydroxyurea SDM Toolkit (H-SDM) | Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score | 82.0 score on a scale | Standard Deviation 15.5 |
Secondary
Satisfaction With Decision-Making
Eight-item survey adapted from the Satisfaction With Decision scale 41 (4 items) and the Agency for Healthcare Research and Quality's Consumer Assessment of Healthcare Providers and Systems survey related to patient experience of care (4 items). 42 Items are summed to obtain a total score ranging from 0 to 28, with higher scores indicating higher satisfaction.
Time frame: Baseline after the shared discussion
Population: 145 parents with data for baseline measures were included in the analyses whether or not they attended the follow-up visit or completed additional measures.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Clinician Pocket Guide | Satisfaction With Decision-Making | 13.5 score on a scale | Standard Deviation 5.5 |
| Hydroxyurea SDM Toolkit (H-SDM) | Satisfaction With Decision-Making | 13.2 score on a scale | Standard Deviation 6.3 |