Sickle Cell Disease
Conditions
Brief summary
The purpose of this proposal is to integrate family-centered self-management strategies with mobile health (mHealth) technology to improve reach, self-management behaviors, and child and caregiver physical and psychosocial symptoms and quality of life. Specifically, the investigators propose to conduct feasibility testing of SMYLS, which has been adapted based on user feedback in the first phase of this study. First the investigators will work with the Medical University of South Carolina (MUSC) Pediatric Sickle Cell Clinic to identify and recruit families with children with sickle cell disease (SCD) in the community, statewide. Next, the investigators will test the feasibility of the intervention with 30 dyads of children ages 8 - 17 with sickle cell disease and their parent or primary caregiver, (N=60)
Interventions
BEHAVIORALVoice Crisis Alert V2
Intervention consists of a mHealth app delivered via smartphone
Sponsors
Medical University of South Carolina
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* children ages 8 - 17 years and parent or primary caregiver 18 years or older * child with sickle cell disease, as reported by clinician at MUSC Pediatric Sickle Cell Clinic
Exclusion criteria
* Parent/caregiver or child with cognitive disability or delay that precludes ability to participate * Lack of wi-fi access
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rates of Recruitment | Assessed each week over a period of 6 months, cumulative data up to 6 months is reported. | Number of weeks required to recruit 30 participants. |
| Participant Adherence to Intervention | baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months | Number of participants who used the intervention (mHealth application) from baseline to mid-intervention, from mid-intervention to end-of-intervention, and from end-of-intervention to follow-up, assessed by number of participants who logged into and used the app, stored in the app's back end database. |
| Acceptability of Intervention | Assessed each week over a period of 6 months, cumulative data up to 6 months is reported. | Number of participants reporting problems with the intervention (mHealth app) per week. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention | baseline, 12 weeks | Pediatric Quality of Life Inventory (Peds QL): Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved |
| Difference in Mean Scores for Fatigue From Baseline to End-of-intervention | baseline, 12 weeks | Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Pediatric Short Form 10a: Raw Scores range from 0 - 40 with 0 being the least amount of fatigue and 40 the most fatigue. Difference in mean scores at 12 weeks minus baseline. Any decrease in rating = improved |
| Difference in Mean Scores for Anxiety From Baseline to End-of-intervention | baseline, 12 weeks | Patient Reported Outcomes Measurement System (PROMIS) Pediatric Short Form Anxiety 8a: Raw Scores range from 0 - 32, with 0 being the lowest anxiety rating and 32 the highest anxiety rating. Change in score at 12 weeks minus baseline. Any decrease difference in mean scores = improved |
| Home Medication Administration | 12 weeks | Adherence to home medications, measured by number of medications marked as taken in the app database. |
| Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention | baseline, 12 weeks | Patient Reported Outcome Measurement Information System (PROMIS) Pain Intensity Pediatric 3a. Raw scores range from 3 - 15, with 3 being the lowest pain intensity rating and 15 being the highest pain intensity rating. Change in score at 12 weeks minus baseline. Any decrease in mean difference between scores = improved |
| Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention | baseline, 12 weeks | Pediatric Quality of Life Inventory (Peds QL) with SCD module: Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved |
| Clinic Appointment Attendance | 12 weeks | Number of missed clinic appointments at baseline compared to 12 weeks |
| Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention | baseline,12 weeks | Patient Reported Outcomes Measurement System (PROMIS) Pediatric short form depression 8a: Raw Scores range from 0 - 32, with 0 being the lowest depression rating and 32 the highest depression rating. Change in score at 12 weeks minus baseline. Any decrease in differences in mean scores = improved |
| Difference in Mean Pain Score Rating From Baseline to End-of-intervention | baseline, 12 weeks | Patient Reported Outcome Measurement Information System (PROMIS) Pain Interference Pediatric Short Form 8a: Change in score at 12 weeks minus baseline. Raw scores range from 0 - 32, with 0 being the lowest pain rating and 32 the highest pain rating. Any decrease in mean difference between scores = improved |
| Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention | baseline, 12 weeks | Self-efficacy for Managing Chronic Disease 6-item scale. Scores for each item range from 1 - 10 with 1 indicating lower confidence/self-efficacy and 10 indicating higher confidence/self-efficacy. Scores for each item are averaged for the mean total score. Change in score at 12 weeks minus baseline. Any increase in difference in mean scores = improved |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Intervention Group This was a single-arm study; all participants received the intervention. | 30 |
| Total | 30 |
Baseline characteristics
| Characteristic | Intervention Group |
|---|---|
| Age, Continuous | 11.8 years STANDARD_DEVIATION 2.9 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 30 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| household size | 3.4 number of people STANDARD_DEVIATION 1.5 |
| Parent/caregiver education level 9 - 12 grade or less | 3 Participants |
| Parent/caregiver education level college graduate or above | 9 Participants |
| Parent/caregiver education level high school graduate or GED | 5 Participants |
| Parent/caregiver education level some college or certification course | 13 Participants |
| Parent/caregiver employment status caregiver/stay-at-home parent | 2 Participants |
| Parent/caregiver employment status disabled | 0 Participants |
| Parent/caregiver employment status employed | 21 Participants |
| Parent/caregiver employment status student | 0 Participants |
| Parent/caregiver employment status unemployed | 3 Participants |
| Parent/caregiver employment status unknown/no response | 4 Participants |
| Parent/caregiver marital status divorced | 3 Participants |
| Parent/caregiver marital status living with partner | 1 Participants |
| Parent/caregiver marital status married | 9 Participants |
| Parent/caregiver marital status single | 17 Participants |
| Parent/caregiver type father | 1 Participants |
| Parent/caregiver type grandmother | 2 Participants |
| Parent/caregiver type mother | 27 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 30 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment United States | 30 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 16 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 30 |
| other Total, other adverse events | 0 / 30 |
| serious Total, serious adverse events | 2 / 30 |
Outcome results
Primary
Acceptability of Intervention
Number of participants reporting problems with the intervention (mHealth app) per week.
Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Intervention Group | Acceptability of Intervention | 4 Participants |
Primary
Participant Adherence to Intervention
Number of participants who used the intervention (mHealth application) from baseline to mid-intervention, from mid-intervention to end-of-intervention, and from end-of-intervention to follow-up, assessed by number of participants who logged into and used the app, stored in the app's back end database.
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Intervention Group | Participant Adherence to Intervention | Number who used from baseline to 6 weeks | 30 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 6 weeks to 12 weeks | 15 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 12 weeks to 6 months | 10 participants |
Primary
Participant Adherence to Intervention
number of participants who accessed the educational component of intervention, assessed using back end app use database
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Intervention Group | Participant Adherence to Intervention | Number who used from baseline to 6 weeks | 28 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 6 weeks to 12 weeks | 7 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 12 weeks to 6 months | 4 participants |
Primary
Participant Adherence to Intervention
Number of participants who accessed the symptom monitoring component of the intervention, assessed using the app back end database
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Intervention Group | Participant Adherence to Intervention | Number who used from 6 weeks to 12 weeks | 15 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from baseline to 6 weeks | 29 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 12 weeks to 6 months | 10 participants |
Primary
Participant Adherence to Intervention
number of participants who sent messages to nurse practitioner
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Intervention Group | Participant Adherence to Intervention | Number who used from baseline to 6 weeks | 12 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 6 weeks to 12 weeks | 2 participants |
| Intervention Group | Participant Adherence to Intervention | Number who used from 12 weeks to 6 months | 1 participants |
Primary
Rates of Recruitment
Number of weeks required to recruit 30 participants.
Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Intervention Group | Rates of Recruitment | 14 weeks |
Secondary
Clinic Appointment Attendance
Number of missed clinic appointments at baseline compared to 12 weeks
Time frame: 12 weeks
Population: Limitations of this feasibility study included issues with the wording and participant interpretation of clinic appointment attendance questions, leading to an inability to accurately assess clinic appointment attendance.
Secondary
Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention
Self-efficacy for Managing Chronic Disease 6-item scale. Scores for each item range from 1 - 10 with 1 indicating lower confidence/self-efficacy and 10 indicating higher confidence/self-efficacy. Scores for each item are averaged for the mean total score. Change in score at 12 weeks minus baseline. Any increase in difference in mean scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to 12 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention | 0.2 scores on scale |
Secondary
Difference in Mean Pain Score Rating From Baseline to End-of-intervention
Patient Reported Outcome Measurement Information System (PROMIS) Pain Interference Pediatric Short Form 8a: Change in score at 12 weeks minus baseline. Raw scores range from 0 - 32, with 0 being the lowest pain rating and 32 the highest pain rating. Any decrease in mean difference between scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to 12 weeks (end-of-intervention) for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Difference in Mean Pain Score Rating From Baseline to End-of-intervention | -9.0 scores on scale |
Secondary
Difference in Mean Scores for Anxiety From Baseline to End-of-intervention
Patient Reported Outcomes Measurement System (PROMIS) Pediatric Short Form Anxiety 8a: Raw Scores range from 0 - 32, with 0 being the lowest anxiety rating and 32 the highest anxiety rating. Change in score at 12 weeks minus baseline. Any decrease difference in mean scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to end-of-intervention for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Difference in Mean Scores for Anxiety From Baseline to End-of-intervention | 1.6 scores on scale |
Secondary
Difference in Mean Scores for Fatigue From Baseline to End-of-intervention
Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Pediatric Short Form 10a: Raw Scores range from 0 - 40 with 0 being the least amount of fatigue and 40 the most fatigue. Difference in mean scores at 12 weeks minus baseline. Any decrease in rating = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to end-of-intervention for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Difference in Mean Scores for Fatigue From Baseline to End-of-intervention | -7.9 scores on scale |
Secondary
Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention
Pediatric Quality of Life Inventory (Peds QL) with SCD module: Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to end-of-intervention for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention | 12.5 scores on scale |
Secondary
Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention
Patient Reported Outcome Measurement Information System (PROMIS) Pain Intensity Pediatric 3a. Raw scores range from 3 - 15, with 3 being the lowest pain intensity rating and 15 being the highest pain intensity rating. Change in score at 12 weeks minus baseline. Any decrease in mean difference between scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to end-of-intervention
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention | -1.9 scores on scale |
Secondary
Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention
Pediatric Quality of Life Inventory (Peds QL): Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved
Time frame: baseline, 12 weeks
Population: 20 participants retained to end-of-intervention for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention | 10.9 scores on scale |
Secondary
Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention
Patient Reported Outcomes Measurement System (PROMIS) Pediatric short form depression 8a: Raw Scores range from 0 - 32, with 0 being the lowest depression rating and 32 the highest depression rating. Change in score at 12 weeks minus baseline. Any decrease in differences in mean scores = improved
Time frame: baseline,12 weeks
Population: 20 participants retained to end-of-intervention for analysis
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Intervention Group | Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention | -3.1 scores on scale |
Secondary
Home Medication Administration
Adherence to home medications, measured by number of medications marked as taken in the app database.
Time frame: 12 weeks
Population: Limitations of this feasibility study included technical difficulties with the app, leading to an inability to accurately assess home medication administration.