Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

Home-based Assessment of Patient Reported Outcome (PROs) Measures in Sickle Cell Disease (SCD) Using A Smartphone App Platform: A Feasibility Study

The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease; Sickle B+ Thalassemia; Sickle Beta Zero Thalassemia; Sickle Cell Hemoglobin C
• Intervention / Treatment: Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app)

Detailed Description

Adherence to hydroxyurea is a multi-factorial dynamic process, and, to date, predictors for changes in adherence levels over time remain unclear. Changes in HRQOL data could serve as surrogate markers for changes in hydroxyurea adherence over time, and could suggest when patients might or might not need or benefit from an intervention based on worse or better HRQOL scores, respectively.

The main hypothesis of this study is that the assessment of patients' HRQOL at home using a SCD smartphone application platform (app) is feasible and acceptable, and that less frequent assessments of HRQOL at home will have an overall higher completion rate when compared to more frequent ones. We plan to assess HRQOL using the NIH-developed patient reported outcomes measurement information system (PROMIS®) with computerized adaptive testing (CAT) approach. PROMIS®-CAT approach is a reliable and valid PROs assessment platform that has been used in limited studies in SCD.

The study will include the following specific aims:

1. To evaluate the feasibility and acceptability of the assessment of patients HRQOL at home using smartphones with PROMIS®-CAT measures integrated into a SCD-app.
2. To examine the effect of the frequency of required HRQOL assessments on participants' completion rate over 24-week period with HRQOL evaluated every 2 weeks (Group 1) versus every 4 weeks (Group 2).
3. To explore participants' experience and preferences with the process and the frequency of HRQOL assessment at home using their smartphones with PROMIS®-CAT measures integrated into a SCD-app.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 25 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age 12 years or older at the time of study enrollment;
• SCD diagnosis (all genotypes) confirmed by hemoglobin electrophoresis;
• Own or have access to a smartphone or a tablet; and
• Speak and read English.

Exclusion Criteria:

• Patients or caregivers with cognitive impairment;
• Patients or caregivers physical impairment; and
• Patients or caregivers who will not be able to complete study assessments.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: SUPPORTIVE_CARE
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: Group A (PROs assessment every 2 weeks)	Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app); A smartphone app platform designed for patients with sickle cell disease. The SCD-app is programmed to send notification to patients to complete PROs assessment. The SCD-app is able to collect PROs data as patients complete the assessment using the app platform.
ACTIVE_COMPARATOR: Group A (PROs assessment every 4 weeks)	Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app); A smartphone app platform designed for patients with sickle cell disease. The SCD-app is programmed to send notification to patients to complete PROs assessment. The SCD-app is able to collect PROs data as patients complete the assessment using the app platform.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility of completing PRO assessments at home using SCD-app (HU-Go)	Having an overall completion rate of ≥ 50% for all study participants (Group A and B). This will be reported as a dichotomous outcome, either yes or no. The percentage (%) completion rate will be calculated as the number of PROs assessments completed divided by the number of PROs assessments expected over the study period.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Completion rate of PRO assessments at home using SCD-app (HU-Go)	Completion rate of different PRO assessments at home using PROMIS®-CAT measures among patients with SCD and/or their parents. PRO assessments are planned to be every 2 weeks in Group 1 and every 4 weeks in Group 2. This outcome will be calculated and reported as percent (%) completion rate.	6 months

Collaborators and Investigators

• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
• Investigators: Principal Investigator: Sherif M Badawy, MD, MS, Ann & Robert H Lurie Children's Hospital of Chicago

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 4, 2017
• Primary Completion (ACTUAL): July 21, 2019
• Study Completion (ACTUAL): July 21, 2019

Study Registration Dates

• First Submitted: December 3, 2020
• First Submitted That Met QC Criteria: December 17, 2020
• First Posted (ACTUAL): December 21, 2020

Study Record Updates

• Last Update Posted (ACTUAL): December 21, 2020
• Last Update Submitted That Met QC Criteria: December 17, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell; Thalassemia; Hemoglobin SC Disease
• Other Study ID Numbers: IRB 2017-757

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: All study data are de-identified and will be analysed as aggregate.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No