Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease (PF-Guide)

Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease (PF-Guide)

In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth application with booklets will decrease acute healthcare utilization and be cost-effective over booklets with the guidelines alone. Our team, head will test our hypotheses with the following aims: Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs. standard care in adults with SCD. is hybrid effectiveness-implementation trial design, according to the COM-B and RE-AIM frameworks with a mixed-methods approach, will give valuable insights into the effects, facilitators, and barriers to the implementation that will influence the effects of the patient-facing guidelines intervention.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Other: mHealth app + booklets

Detailed Description

The investigators propose a multi-center hybrid type I effectiveness-implementation trial to assess patient-facing evidence-based guidelines through a mHealth application plus (+) booklets in adults with sickle cell disease (SCD). Clinicians' care of SCD is informed by evidence-based guidelines, which can prevent morbidity and mortality. The National Heart, Lung, and Blood Institute (NHLBI) and the American Society of Hematology (ASH) published guidelines for evidence-based management of SCD for healthcare providers. However, patient, provider, or system barriers prohibit adequate reach within and across all three domains, leading to poor guideline adherence. Our prior work demonstrates 1) the patient's lack of knowledge of evidence-based guidelines, 2) the desire for guidelines to be patient-centered, accessible, and actionable in booklets and a mHealth app, 3) the development of patient-facing booklets with the guidelines and 4) the successful pilot of an engaging mHealth app with the guidelines that adults with SCD will use. Promising preliminary data from our multi-center feasibility randomized controlled trial support using a mHealth app + booklets with patient-facing guidelines for reducing acute healthcare utilization in adults with SCD. In the feasibility trial, the investigators screened 74 participants; 91% (67 of 74) agreed to be recruited and randomized to mHealth + booklet vs. booklets alone. A relative risk reduction of 44% occurred in acute healthcare utilization in the mHealth + booklet arm vs. the booklet alone arm. A total of 75% (50 of 67) of the participants agreed to be followed for six months to assess healthcare utilization and surveys of knowledge and patient-reported outcomes. Based on our preliminary trial results indicating a decrease of at least 44% in acute healthcare utilization (a decrease of 1.5 emergency room visits or hospital admissions per year) with mHealth + booklet, the investigators propose a three-center randomized controlled trial to test the following hypothesis: There will be a 44% relative risk reduction of acute healthcare utilization in adults with SCD (n=272), randomly allocated to mHealth app + booklets vs. standard care, defined as general guidance without patient-facing educational materials, for 12 months. The aims of this proposed study are Aim 1: evaluate the effectiveness of the patient-facing guidelines mHealth app + booklet intervention to decrease acute healthcare utilization (hospitalizations, emergency room visits, and day hospital visits) in adults with SCD over the standard care in a randomized controlled trial, Aim 2: evaluate the implementation outcomes of the mHealth app + booklet using the capability, opportunity, and motivation-behavior (COM-B) and reach, effectiveness, adoption, implementation, and maintenance (RE-AIM) frameworks, and Aim 3: evaluate the cost-effectiveness of patient-facing mHealth app + booklets vs. standard care in adults with SCD. This hybrid effectiveness-implementation trial design, according to the COM-B and RE-AIM frameworks with a mixed-methods approach, will give valuable insights into the effects, facilitators, and barriers to the implementation that will influence the effects of the patient-facing guidelines intervention.

• Study Type: Interventional
• Enrollment (Estimated): 287
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Robert M Cronin, MD, MS; Phone Number: 6146889220; Email: robert.cronin@osumc.edu

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60607
      • Recruiting
      • University of Illinois at Chicago
      • Contact: Saraf Santosh
      • Sub-Investigator: Victor Gordeuk
  • Missouri
    • St Louis, Missouri, United States, 63130
      • Recruiting
      • Washington University St. Louis
      • Contact: Allison King
  • Ohio
    • Columbus, Ohio, United States, 43212
      • Recruiting
      • The Ohio State University Medical Center
      • Contact: Robert Cronin; Email: robert.cronin@osumc.edu
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Recruiting
      • Vanderbilt University
      • Contact: Robert Cronin, MD, MS; Phone Number: 6159365097; Email: robert.cronin@vanderbilt.edu
      • Contact: Michael DeBaun, MD, MPH; Phone Number: (615) 875-3040; Email: m.debaun@vanderbilt.edu
      • Principal Investigator: Emmanuel Volanakis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion criteria for adults with SCD include

1. receiving care at the SCD clinic for the prior 12 months,
2. has a diagnosis of SCD (Hgb SS, SC, Sβ-thal),
3. able to speak and understand written English,
4. older than 18 years.

Inclusion criteria for the providers and staff members are

(1) to be involved in participants' clinical care

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: mHealth app + booklets; A mHealth app that has patient facing guidelines and engaging content and a booklet with the guidelines that are made to be patient-facing.	Other: mHealth app + booklets; A mHealth app that has patient facing guidelines and engaging content and a booklet with the guidelines that are made to be patient-facing.
No Intervention: Standard care; Group 1 will receive the control arm with standard care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Acute healthcare utilization	The investigators will measure a count of the # ER visits, # of hospitalizations, and # of day hospital visits (infusion center visits)	12 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Self-efficacy	sickle cell self-efficacy scale, as described in Edwards R, Telfair J, Cecil H, Lenoci J. Reliability and validity of a self-efficacy instrument specific to sickle cell disease. Behav Res Ther. 2000;38(9):951-63. Scale is 0-45, higher score is better Scale has no subscales and measures self-efficacy	12 months
SCD-specific knowledge	Knowledge about SCD guidelines. The knowledge part of the measure is a questionnaire that is multiple choice and asks questions about sickle cell disease with one correct answer and 3 incorrect answers. The investigators will sum up the number of correct answers to determine a score. This is not a scale.	12 months

Collaborators and Investigators

• Sponsor: Ohio State University
• Collaborators: Washington University School of Medicine; University of Illinois at Chicago; Vanderbilt University Medical Center
• Investigators: Principal Investigator: Robert M Cronin, MD, MS, Ohio State University

Study record dates

Study Major Dates

• Study Start (Actual): January 13, 2026
• Primary Completion (Estimated): March 1, 2031
• Study Completion (Estimated): March 1, 2032

Study Registration Dates

• First Submitted: April 22, 2024
• First Submitted That Met QC Criteria: April 2, 2025
• First Posted (Actual): April 9, 2025

Study Record Updates

• Last Update Posted (Actual): March 23, 2026
• Last Update Submitted That Met QC Criteria: March 19, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell
• Other Study ID Numbers: R01HL174590 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No