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The hemoglobinopathy newborn screen (NBS) performed on all neonates in the U.S. allows for early life-saving medical care for infants with sickle cell disease (SCD), an autosomal recessive genetic disorder. Because of its detection method, the NBS incidentally reveals hemoglobinopathy traits including sickle cell trait (SCT). In an effort to uphold the rights of the newborn to their medical data and preserve autonomy in medical decision making, pediatric and genetic society guidelines recommend disclosure and documentation of SCT results during infancy. Despite this guidance, a large guideline-to-practice gap exists: SCT status is grossly under-documented in the pediatric electronic health record and few adults report knowing their SCT status despite universal screening. We plan to evaluate the effect of a toolkit of SCT Documentation and Disclosure (SCT-DD) strategies on documentation and disclosure of SCT by pediatric primary care providers in a 2-arm randomized interrupted time series trial.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| "All-in" | Active Comparator | In the "all-in" arm, pediatric primary care physicians receive all toolkit components at once. |
|
| "Add-in" | Active Comparator | In the "add-in" arm, pediatric primary care physicians will have sequential addition of toolkit components in 6 week increments |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SCT Documentation and Disclosure Toolkit (SCT-DD) | Behavioral | A toolkit of implementation strategies |
|
| Measure | Description | Time Frame |
|---|---|---|
| Acceptability | Acceptability of toolkit components by pediatric primary care providers by survey at the end of the study (i.e. 18 weeks after initial roll-out of interventions). Reported as the number who answered Agree or Strongly Agree on a 4-point Likert scale that they liked the toolkit component. Likert scale options included strong disagree, disagree, agree, strongly agree. | At conclusion of study: 18 weeks after initial roll-out of interventions |
| Self-efficacy | Confidence to document/discuss SCT result by pediatric primary care providers by survey. Scale 1 to 10: 1 = not confident at all, 10 = extremely confident. | At conclusion of study (18 weeks after initial roll-out of interventions) |
| Feasibility of Using Toolkit Components | Number of pediatric primary care providers who used individual toolkit components in the last 6 weeks of the study (week 12-18) as indicated by answering "yes" to questions about whether individual toolkit components were used on a survey at the end of the study (18 weeks after roll-out of interventions). | Survey at end of study: 18 weeks after roll-out of interventions |
| Penetration | The number of newborns with newborn screen results visible in the electronic healthcare record and presence of documentation of abnormal newborn screen results. By chart review. | Chart review for retrospective patients was done before intervention roll-out. Chart review for prospective patients was done on a rolling basis at 2 months of age for any newborn patient seen during the 18 week intervention period |
| Measure | Description | Time Frame |
|---|---|---|
| Knowledge | Knowledge of newborn screen results by caregivers via survey. | Survey for retrospective patients was sent before intervention roll-out. Survey for prospective patients were sent on a rolling basis at 2 months of age for any newborn patient seen during the 18 week intervention period. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Corinna Schultz, MD, MSHP | Nemours | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Children's Hospital, Delware | Wilmington | Delaware | 19803 | United States |
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Invitations were sent to the "physician in charge" of 20 pediatric primary care sites within one hospital system in the mid-Atlantic region. Seven (7) sites were interested in participating in the pilot project and randomized to intervention arms. Clinicians and caregivers at each site were then recruited via email. Each group of participants is designated as a separate time period below: Clinicians, Caregivers from the retrospective period, caregivers from the prospective time.
| ID | Title | Description |
|---|---|---|
| FG000 | "All-in" | In the "all-in" arm, pediatric primary care physicians receive all toolkit components at once. |
| FG001 | "Add-in" | In the "add-in" arm, pediatric primary care physicians will have sequential addition of toolkit components in 6 week increments |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Clinician |
| |||||||||||||
| Caregiver Survey From Retrospective Time |
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| Caregiver Survey From Intervention Time |
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Two sets of participants included: 1) Physicians who were the primary target of the intervention, 2) Caregivers who consented and completed surveys. As physicians were the primary interest, demographic characteristics and the majority of study outcomes were based on physicians enrolled.
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| ID | Title | Description |
|---|---|---|
| BG000 | "All-in" | In the "all-in" arm, pediatric primary care physicians receive all toolkit components at once. |
| BG001 | "Add-in" | In the "add-in" arm, pediatric primary care physicians will have sequential addition of toolkit components in 6 week increments |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Two sets of participants included: 1) Physicians who were the primary target of the intervention, 2) Caregivers who consented and completed surveys. As physicians were the primary interest, demographic characteristics and the majority of study outcomes were based on physicians enrolled. |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Acceptability | Acceptability of toolkit components by pediatric primary care providers by survey at the end of the study (i.e. 18 weeks after initial roll-out of interventions). Reported as the number who answered Agree or Strongly Agree on a 4-point Likert scale that they liked the toolkit component. Likert scale options included strong disagree, disagree, agree, strongly agree. | At conclusion of study: 18 weeks after initial roll-out of interventions. | Posted | Count of Participants | Participants | At conclusion of study: 18 weeks after initial roll-out of interventions |
|
For Physicians: From enrollment until last survey completed (total 18 weeks). Adverse events not evaluated from chart review or caregiver surveys
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | "All-in" | In the "all-in" arm, pediatric primary care physicians receive all toolkit components at once. |
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Very small number of caregivers of children with sickle cell trait responded to survey requests.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Corinna Schultz | Nemours Children's Health | 302-651-5500 | corinna.schultz@nemours.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 21, 2025 | Oct 21, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D012805 | Sickle Cell Trait |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
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| NOT COMPLETED |
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| NOT COMPLETED |
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| BG002 | Total | Total of all reporting groups |
Physician population only |
| Count of Participants |
| Participants |
| No |
|
| Sex: Female, Male | Two sets of participants included: 1) Physicians who were the primary target of the intervention, 2) Caregivers who consented and completed surveys. As physicians were the primary interest, demographic characteristics and the majority of study outcomes were based on physicians enrolled. | Count of Participants | Participants | No |
|
| Race (NIH/OMB) | Race/Ethnicity not collected from Caregiver Survey Participants | Two sets of participants included: 1) Physicians who were the primary target of the intervention, 2) Caregivers who consented and completed surveys. As physicians were the primary interest, demographic characteristics and the majority of study outcomes were based on physicians enrolled. | Count of Participants | Participants | No |
|
| Confidence regarding hemoglobinopathies | On-line survey of confidence regarding the hemoglobinopathy newborn screen. Scale of 1-10. (1, least confident. 10, most confident). | Based on physicians enrolled (not caregivers) | Mean | Standard Deviation | units on a scale (1-10, 10 'extremely') |
|
In the "add-in" arm, pediatric primary care physicians will have sequential addition of toolkit components in 6 week increments |
|
|
| Primary | Self-efficacy | Confidence to document/discuss SCT result by pediatric primary care providers by survey. Scale 1 to 10: 1 = not confident at all, 10 = extremely confident. | Based on participants who answered final survey at 18 weeks post intervention | Posted | Mean | Standard Deviation | units on a scale (1-10) | At conclusion of study (18 weeks after initial roll-out of interventions) |
|
|
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| Primary | Feasibility of Using Toolkit Components | Number of pediatric primary care providers who used individual toolkit components in the last 6 weeks of the study (week 12-18) as indicated by answering "yes" to questions about whether individual toolkit components were used on a survey at the end of the study (18 weeks after roll-out of interventions). | Posted | Count of Participants | Participants | Survey at end of study: 18 weeks after roll-out of interventions |
|
|
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| Secondary | Knowledge | Knowledge of newborn screen results by caregivers via survey. | Surveys completed | Posted | Count of Participants | Participants | Survey for retrospective patients was sent before intervention roll-out. Survey for prospective patients were sent on a rolling basis at 2 months of age for any newborn patient seen during the 18 week intervention period. |
|
|
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| Primary | Penetration | The number of newborns with newborn screen results visible in the electronic healthcare record and presence of documentation of abnormal newborn screen results. By chart review. | Number of newborn patients in the all-in and add-in arms who were seen at sites enrolled in the trial during the retrospective and prospective periods. Chart reviews completed on all patients. Chart review for retrospective patients was done before intervention roll-out. Chart review for prospective patients was done at 2 months of age. | Posted | Count of Participants | Participants | Chart review for retrospective patients was done before intervention roll-out. Chart review for prospective patients was done on a rolling basis at 2 months of age for any newborn patient seen during the 18 week intervention period |
|
|
|
| 0 |
| 16 |
| 0 |
| 16 |
| 0 |
| 16 |
| EG001 | "Add-in" | In the "add-in" arm, pediatric primary care physicians will have sequential addition of toolkit components in 6 week increments | 0 | 12 | 0 | 12 | 0 | 12 |
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| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Male |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Confidence to discuss hemoglobinopathy results |
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| Confidence to discuss inheritance of hemoglobinopathies |
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| Confidence to discuss reproductive options of hemoglobinopathies |
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| Reference Materials |
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| Survey respondents who reported they received newborn screen results - Prospective |
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| Survey respondents who reported sickle cell trait on their child's newborn screen -Retrospective |
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| Survey respondents who reported sickle cell trait on their child's newborn screen -Prospective |
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| Newborn screen available in Careeverywhere - Retrospective |
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| Sickle cell trait on newborn screen - Retrospective |
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| Sickle cell trait on problem list - Retrospective |
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| Sickle cell trait documented as disclosed - Retrospective |
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| Newborn screen available in EPIC - Prospective |
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| Newborn screen available in Careeverywhere - Prospective |
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| Sickle cell trait on newborn screen - Prospective |
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| Sickle cell trait on problem list - Prospective |
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| Sickle cell trait documented as disclosed - Prospective |
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