Sickle Cell Disease
Conditions
Keywords
Sickle Cell Disease, Social Determinants of Health, Unmet Social Needs, WE CARE
Brief summary
This mixed-methods study aims to understand the implementation of a previously tested, efficacious social determinants of health (SDoH) screening and referral intervention in the outpatient pediatric hematology setting; qualitatively assess possible mechanisms for such interventions on improving child health; and obtain population-specific empirical estimates to plan a large-scale clinical trial.
Detailed description
Social determinants of health (SDoH)-the conditions in which people are born, grow, work, live, and age-are key drivers of health and health disparities. Children with medical complexity are particularly at-risk given their high healthcare need and utilization. Although the American Academy of Pediatrics and payers such as the Centers for Medicaid and Medicare Services are now recommending medical providers screen for SDoH at visits, studies have not yet demonstrated the impact of SDoH screening and referral interventions on improving child health and have fallen short of exploring potential mechanisms by which such interventions could improve health outcomes. Children with sickle cell anemia (SCA) are an ideal population in which to study the impact of SDoH interventions given the high prevalence of poverty and unmet material needs among this population and the disease's significant morbidity and mortality. This proposal addresses a timely clinically- and policy-relevant research gap by: (1) implementing a SDoH intervention in two outpatient pediatric hematology clinics and gathering preliminary data to assess its impact on child health; and (2) characterizing the potential mechanisms by which addressing SDoH may lead to improved health outcomes. The research team has developed, tested, and implemented a SDOH intervention (WE CARE) which relies on existing clinical processes to screen for unmet material needs and refer parents to community services; efficacy data demonstrates its positive impact on parental receipt of community resources. The investigators now propose conducting a pragmatic pilot cluster randomized controlled trial (RCT) to examine the implementation of WE CARE as standard of care in two of the four hematology clinics. To preliminarily examine outcomes,100 parents of children with SCA (25 per site) will be recruited and followed for one year in order to explore how addressing unmet social needs within the delivery of medical care may improve healthcare utilization and health outcomes. Given the limitations of applying existing theoretical frameworks to culturally diverse populations such as those with SCA, the investigators will also employ a mixed methods approach to characterizing how SDoH influences disease management processes. The specific aims are to: (1) Implement WE CARE in two pediatric hematology clinics in order to field test key study logistics and understand the facilitators and barriers to implementation and accelerate its adoption; (2) Obtain population-specific empirical estimates of study parameters to plan a large-scale multi-site cluster RCT of WE CARE that will definitely assess its impact on improving health outcomes for children with SCA; and (3) Qualitatively assess possible mechanisms linking SDoH interventions to improved health outcomes. It has significant implications for child health policy and is a critical step in potentially transforming the delivery of healthcare for medically complex children.
Interventions
BEHAVIORALWE CARE SDoH Screening Survey
The survey will be given at all visits by the front desk staff to all parents of SCA patients. It consists of 12 questions designed to: (1) briefly identify 6 unmet material needs (e.g., childcare, employment, food security, household heat, housing inadequate education) by self-report and (2) using a family-centered approach, determine whether parents would like assistance with each problem Parents wanting help will receive a resource referral. Clinical team members will be trained to review the WE CARE SDoH survey at visits and to provide community resource information sheets to parents with needs. Completed surveys will be scanned into the EHR
BEHAVIORALFamily Resource Book
The Family Resource book will contain one-page information sheets listing community resources (e.g., food pantries) and their contact information (i.e. telephone number) for each specific material need (e.g., food insecurity). Information sheets will be specific to each site and written at, or below, the 8th grade level. For parents with an identified need, providers will be instructed to give an information sheet. The book will contain six separate tabs, one for each unmet need, and will contain multiple copies of the information sheets. The Family Resource Book will be made available in each exam room. The investigators will work with each practice to create a Family Resource Book prior to study initiation.
OTHERStandard of care
Usual outpatient care for pediatric patients with sickle cell anemia will be provided.
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Boston Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SCREENING
Masking
NONE
Intervention model description
Pragmatic pilot cluster RCT to examine the implementation of WE CARE as standard of care in two of the four outpatient pediatric hematology clinics.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Adult parents of children with SCA (0-12 years of age) who take a daily medication (penicillin or hydroxyurea)to * English or Spanish speaking
Exclusion criteria
* Foster parents
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Emergency Department (ED)/Acute Care Visits | 12 months | Data on number of ED visits and acute care visits abstracted from the EHR. |
| Parental Enrollment in Community Resources | 12 months | Self-reported enrollment in a new community resource, where yes indicates enrollment in a new resource, and no indicates no enrollment in a new resource. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Vaso-occlusive Episodes | 12 months | Number of painful vaso-occlusive episodes (VOE) requiring an ED or acute care clinic visit |
| Prescriptions for Sickle Cell Disease | 12 months | Data on prescriptions written and filled for hydroxyurea and penicillin will be collected through EHR review. . Number of days covered by prescriptions will be reported |
| Personal Health Questionnaire Depression Scale (PHQ-8) | 12 months | Patient Health Questionnaire depression scale (PHQ-8) is an 8 item instrument with possible responses for each item of 0=Not at all, 1=Several days 2= More than half the days, 3=Nearly every day. The range of scores is 0 to 24. A score of 10 or greater is considered major depression, 20 or more is severe major depression. |
| White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence | 12 months | Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) including hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume. Each will be abstracted from medical records. |
| Mean Corpuscular Volume Values Related to Medication Adherence | 12 months | Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) including hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume (MCV) will be abstracted from medical records. MCV is a measure of the average volume of a red blood corpuscle (or red blood cell). |
| Hemoglobin Values Related to Medication Adherence | 12 months | Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) include hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume. Each wil be abstracted from the medical records. |
| Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | 12 months | Self-reported measure of effective and ineffective ways to cope with a stressful life event. 28 total items (list of coping behaviors) scored on a 4-point Likert scale from 1 (I haven't been doing this at all) to 4 (I've been doing this a lot). There are 14 coping behaviors for the 28 items and scores for each of the 14 can range from 1-8. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Intervention-WE CARE Received WE CARE screener and referral program | 41 |
| Control-Standard of Care Received standard of care | 49 |
| Total | 90 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| 12 Months Followup | Lost to Follow-up | 10 | 17 |
| Completed Baseline Assessment | Did not complete baseline assessment | 10 | 12 |
Baseline characteristics
| Characteristic | Intervention-WE CARE | Control-Standard of Care | Total |
|---|---|---|---|
| Age, Continuous | 37 years | 36 years | 37 years |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Acceptance | 5.5 scores on a scale | 5 scores on a scale | 5 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Active coping | 5 scores on a scale | 5 scores on a scale | 5 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Behavioral disengagement | 2 scores on a scale | 2 scores on a scale | 2 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Denial | 2 scores on a scale | 2 scores on a scale | 2 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Humor | 2 scores on a scale | 2 scores on a scale | 2 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Planning | 4 scores on a scale | 4 scores on a scale | 4 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Positive reframing | 5.5 scores on a scale | 5 scores on a scale | 5 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Religion | 6 scores on a scale | 6 scores on a scale | 6 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Self blame | 2 scores on a scale | 2 scores on a scale | 2 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Self distraction | 3 scores on a scale | 4 scores on a scale | 3.5 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Substance use | 2 scores on a scale | 2 scores on a scale | 2 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Use of emotional support | 4 scores on a scale | 4 scores on a scale | 4 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Use of instrumental support | 4 scores on a scale | 4 scores on a scale | 4 scores on a scale |
| Brief Coping Orientation to Problems Experienced (COPE) Inventory at Baseline Venting | 2 scores on a scale | 3 scores on a scale | 3 scores on a scale |
| Ethnicity (NIH/OMB) Hispanic or Latino | 5 Participants | 9 Participants | 14 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 30 Participants | 40 Participants | 70 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 6 Participants | 0 Participants | 6 Participants |
| Patient Health Questionnaire 8 (PHQ-8) Major (10-19 points) | 5 Participants | 5 Participants | 10 Participants |
| Patient Health Questionnaire 8 (PHQ-8) None (0-9 points) | 33 Participants | 42 Participants | 75 Participants |
| Patient Health Questionnaire 8 (PHQ-8) Severe (≥ 20 points) | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 29 Participants | 36 Participants | 65 Participants |
| Race (NIH/OMB) More than one race | 2 Participants | 8 Participants | 10 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 8 Participants | 4 Participants | 12 Participants |
| Race (NIH/OMB) White | 2 Participants | 1 Participants | 3 Participants |
| Region of Enrollment United States | 41 participants | 49 participants | 90 participants |
| Sex/Gender, Customized Female | 29 Participants | 43 Participants | 72 Participants |
| Sex/Gender, Customized Male | 7 Participants | 6 Participants | 13 Participants |
| Sex/Gender, Customized Unknown | 5 Participants | 0 Participants | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 41 | 0 / 49 |
| other Total, other adverse events | 0 / 41 | 0 / 49 |
| serious Total, serious adverse events | 0 / 41 | 0 / 49 |
Outcome results
Primary
Number of Emergency Department (ED)/Acute Care Visits
Data on number of ED visits and acute care visits abstracted from the EHR.
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Intervention-WE CARE | Number of Emergency Department (ED)/Acute Care Visits | 0 number of visits |
| Control-Standard of Care | Number of Emergency Department (ED)/Acute Care Visits | 1 number of visits |
Primary
Parental Enrollment in Community Resources
Self-reported enrollment in a new community resource, where yes indicates enrollment in a new resource, and no indicates no enrollment in a new resource.
Time frame: 12 months
Population: Data are missing for 3 participants in the control arm.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Intervention-WE CARE | Parental Enrollment in Community Resources | Employment | 1 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Food | 1 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Child care | 1 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Education | 1 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Housing | 1 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Utilities | 5 Participants |
| Intervention-WE CARE | Parental Enrollment in Community Resources | Any new resource | 9 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Utilities | 7 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Any new resource | 11 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Child care | 2 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Employment | 0 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Housing | 1 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Food | 1 Participants |
| Control-Standard of Care | Parental Enrollment in Community Resources | Education | 2 Participants |
Secondary
Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months
Self-reported measure of effective and ineffective ways to cope with a stressful life event. 28 total items (list of coping behaviors) scored on a 4-point Likert scale from 1 (I haven't been doing this at all) to 4 (I've been doing this a lot). There are 14 coping behaviors for the 28 items and scores for each of the 14 can range from 1-8.
Time frame: 12 months
Population: Data are missing for 1 participant in the intervention arm and 4 participants in the control arm
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Self-distraction | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Active copying | 4 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Denial | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Substance use | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Use of emotional support | 3 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Use of instrumental support | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Behavioral engagement | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Venting | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Positive reframing | 4.5 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Planning | 4 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Humor | 2 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Acceptance | 6 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Religion | 4.5 score on a scale |
| Intervention-WE CARE | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Self-blame | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Humor | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Self-distraction | 3.5 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Venting | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Active copying | 5 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Religion | 6.5 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Denial | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Positive reframing | 4 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Substance use | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Acceptance | 6 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Use of emotional support | 4 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Planning | 4 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Use of instrumental support | 4 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Self-blame | 2 score on a scale |
| Control-Standard of Care | Brief COPE (Coping Orientation to Problems Experienced Inventory) at 12 Months | Behavioral engagement | 2 score on a scale |
Secondary
Hemoglobin Values Related to Medication Adherence
Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) include hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume. Each wil be abstracted from the medical records.
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Intervention-WE CARE | Hemoglobin Values Related to Medication Adherence | 9.3 g/dL |
| Control-Standard of Care | Hemoglobin Values Related to Medication Adherence | 8.8 g/dL |
Secondary
Mean Corpuscular Volume Values Related to Medication Adherence
Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) including hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume (MCV) will be abstracted from medical records. MCV is a measure of the average volume of a red blood corpuscle (or red blood cell).
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Intervention-WE CARE | Mean Corpuscular Volume Values Related to Medication Adherence | 96 femtoliters (10^15; fl) |
| Control-Standard of Care | Mean Corpuscular Volume Values Related to Medication Adherence | 90.1 femtoliters (10^15; fl) |
Secondary
Personal Health Questionnaire Depression Scale (PHQ-8)
Patient Health Questionnaire depression scale (PHQ-8) is an 8 item instrument with possible responses for each item of 0=Not at all, 1=Several days 2= More than half the days, 3=Nearly every day. The range of scores is 0 to 24. A score of 10 or greater is considered major depression, 20 or more is severe major depression.
Time frame: 12 months
Population: Data are missing for 1 intervention participant and 4 control participants.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Intervention-WE CARE | Personal Health Questionnaire Depression Scale (PHQ-8) | None (0-9 points) | 28 Participants |
| Intervention-WE CARE | Personal Health Questionnaire Depression Scale (PHQ-8) | Major (10-19 points) | 1 Participants |
| Intervention-WE CARE | Personal Health Questionnaire Depression Scale (PHQ-8) | Severe (≥ 20 points) | 1 Participants |
| Control-Standard of Care | Personal Health Questionnaire Depression Scale (PHQ-8) | None (0-9 points) | 25 Participants |
| Control-Standard of Care | Personal Health Questionnaire Depression Scale (PHQ-8) | Major (10-19 points) | 3 Participants |
| Control-Standard of Care | Personal Health Questionnaire Depression Scale (PHQ-8) | Severe (≥ 20 points) | 0 Participants |
Secondary
Prescriptions for Sickle Cell Disease
Data on prescriptions written and filled for hydroxyurea and penicillin will be collected through EHR review. . Number of days covered by prescriptions will be reported
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment except for 1 participant in the intervention group and for 2 participants in the control group; for those 3 participants the data are missing.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Intervention-WE CARE | Prescriptions for Sickle Cell Disease | Penicillin | 365 days |
| Intervention-WE CARE | Prescriptions for Sickle Cell Disease | Hydroxurea | 365 days |
| Control-Standard of Care | Prescriptions for Sickle Cell Disease | Penicillin | 196 days |
| Control-Standard of Care | Prescriptions for Sickle Cell Disease | Hydroxurea | 258 days |
Secondary
Vaso-occlusive Episodes
Number of painful vaso-occlusive episodes (VOE) requiring an ED or acute care clinic visit
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Intervention-WE CARE | Vaso-occlusive Episodes | 1 number of episodes |
| Control-Standard of Care | Vaso-occlusive Episodes | 1 number of episodes |
Secondary
White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence
Laboratory markers commonly affected by hydroxyurea medication from the CBC (complete blood count) including hemoglobin and hemoglobin F levels, white blood cell and absolute neutrophil counts, and mean corpuscular volume. Each will be abstracted from medical records.
Time frame: 12 months
Population: EHR data abstraction was done at 12 months for all participants who completed the baseline assessment.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Intervention-WE CARE | White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence | White blood cell count (per dL) | 8.6 cells per dL |
| Intervention-WE CARE | White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence | Absolute neutrophil count (per dL) | 3.5 cells per dL |
| Control-Standard of Care | White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence | White blood cell count (per dL) | 9.0 cells per dL |
| Control-Standard of Care | White Blood Cell and Absolute Neutrophil Counts Related to Medication Adherence | Absolute neutrophil count (per dL) | 3.4 cells per dL |