Trial Outcomes & Findings for Self-Management for Youth and Families Living With SCD - SMYLS (NCT NCT03585543)

NCT ID: NCT03585543

Last Updated: 2020-11-19

Results Overview

Number of weeks required to recruit 30 participants.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 30 participants
• Primary outcome timeframe: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
• Results posted on: 2020-11-19

Participant Flow

Participant milestones

Measure	Group Receiving mHealth Intervention This was a single-arm study; all participants received the intervention.
Overall Study STARTED	30
Overall Study Mid-intervention Assessment (6-weeks)	25
Overall Study End-of-intervention Assessment (12 Weeks	22
Overall Study 3-month Post-intervention Follow-up (6 m	15
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	15

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Self-Management for Youth and Families Living With SCD - SMYLS

Baseline characteristics by cohort

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Age, Continuous	11.8 years STANDARD_DEVIATION 2.9 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	30 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants
Parent/caregiver type mother	27 Participants n=5 Participants
Parent/caregiver type father	1 Participants n=5 Participants
Parent/caregiver type grandmother	2 Participants n=5 Participants
Parent/caregiver employment status employed	21 Participants n=5 Participants
Parent/caregiver employment status caregiver/stay-at-home parent	2 Participants n=5 Participants
Parent/caregiver employment status unemployed	3 Participants n=5 Participants
Parent/caregiver employment status student	0 Participants n=5 Participants
Parent/caregiver employment status disabled	0 Participants n=5 Participants
Parent/caregiver employment status unknown/no response	4 Participants n=5 Participants
Parent/caregiver education level 9 - 12 grade or less	3 Participants n=5 Participants
Parent/caregiver education level high school graduate or GED	5 Participants n=5 Participants
Parent/caregiver education level some college or certification course	13 Participants n=5 Participants
Parent/caregiver education level college graduate or above	9 Participants n=5 Participants
Parent/caregiver marital status married	9 Participants n=5 Participants
Parent/caregiver marital status living with partner	1 Participants n=5 Participants
Parent/caregiver marital status single	17 Participants n=5 Participants
Parent/caregiver marital status divorced	3 Participants n=5 Participants
household size	3.4 number of people STANDARD_DEVIATION 1.5 • n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Rates of Recruitment	14 weeks

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Participant Adherence to Intervention Number who used from baseline to 6 weeks	30 participants
Participant Adherence to Intervention Number who used from 6 weeks to 12 weeks	15 participants
Participant Adherence to Intervention Number who used from 12 weeks to 6 months	10 participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Acceptability of Intervention	4 Participants

PRIMARY outcome

Timeframe: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months

number of participants who accessed the educational component of intervention, assessed using back end app use database

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Participant Adherence to Intervention Number who used from baseline to 6 weeks	28 participants
Participant Adherence to Intervention Number who used from 6 weeks to 12 weeks	7 participants
Participant Adherence to Intervention Number who used from 12 weeks to 6 months	4 participants

PRIMARY outcome

Timeframe: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months

Number of participants who accessed the symptom monitoring component of the intervention, assessed using the app back end database

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Participant Adherence to Intervention Number who used from baseline to 6 weeks	29 participants
Participant Adherence to Intervention Number who used from 6 weeks to 12 weeks	15 participants
Participant Adherence to Intervention Number who used from 12 weeks to 6 months	10 participants

PRIMARY outcome

Timeframe: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months

number of participants who sent messages to nurse practitioner

Outcome measures

Measure	Intervention Group n=30 Participants This was a single-arm study; all participants received the intervention.
Participant Adherence to Intervention Number who used from baseline to 6 weeks	12 participants
Participant Adherence to Intervention Number who used from 6 weeks to 12 weeks	2 participants
Participant Adherence to Intervention Number who used from 12 weeks to 6 months	1 participants

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to 12 weeks (end-of-intervention) for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Difference in Mean Pain Score Rating From Baseline to End-of-intervention	-9.0 scores on scale Interval -15.0 to -3.0

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to 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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention	0.2 scores on scale Interval -0.5 to 0.9

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to end-of-intervention for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention	10.9 scores on scale Interval 4.3 to 17.4

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to end-of-intervention for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Difference in Mean Scores for Fatigue From Baseline to End-of-intervention	-7.9 scores on scale Interval -12.9 to -2.9

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to end-of-intervention for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Difference in Mean Scores for Anxiety From Baseline to End-of-intervention	1.6 scores on scale Interval -3.7 to 6.8

SECONDARY outcome

Timeframe: baseline,12 weeks

Population: 20 participants retained to end-of-intervention for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention	-3.1 scores on scale Interval -7.3 to 1.1

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained 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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention	-1.9 scores on scale Interval -4.4 to 0.6

SECONDARY outcome

Timeframe: baseline, 12 weeks

Population: 20 participants retained to end-of-intervention for analysis

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

Outcome measures

Measure	Intervention Group n=20 Participants This was a single-arm study; all participants received the intervention.
Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention	12.5 scores on scale Interval 7.1 to 17.9

SECONDARY outcome

Timeframe: 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.

Number of missed clinic appointments at baseline compared to 12 weeks

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 weeks

Population: Limitations of this feasibility study included technical difficulties with the app, leading to an inability to accurately assess home medication administration.

Adherence to home medications, measured by number of medications marked as taken in the app database.

Outcome measures

Outcome data not reported

Adverse Events

Intervention Group

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Intervention Group n=30 participants at risk This was a single-arm study; all participants received the intervention.
Blood and lymphatic system disorders Vaso-occlusive crisis	6.7% 2/30 • Number of events 2 • The duration of study participation; 6 months

Other adverse events

Adverse event data not reported

Additional Information

Shannon Phillips, PhD, RN, Associate Professor

Medical University of South Carolina College of Nursing

Phone: 843-792-9379

Email: phillipss@musc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place