Clinical trial NCT05515497: BMT4me: Post-HSCT Medication Adherence mHealth App
Official title: BMT4me: Improving Adherence Through mHealth for Pediatric Stem Cell Transplant Patients (BMT4me 2.0)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Stem Cell Transplant
Adherence, Medication
Digital Health
Keywords: Adherence
mHealth
Stem Cell transplant
Immunosuppression
Digital Health
Pediatrics
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
Collaborators: name: National Institute of Nursing Research (NINR)
First posted: 2022-08-25
Enrollment: 50
Sex: ALL
Minimum age (years): 0
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: This is a mixed methods, prospective longitudinal pilot RCT to evaluate the 1) acceptability of a newly developed mHealth app (BMT4me), 2) the feasibility of enrolling and retaining caregivers of children in the acute phase post-HSCT, and 3) the potential efficacy of an mHealth app on adherence to immunosuppressants in post-HSCT children discharged during the acute phase.

Detailed description: In the United States, poor adherence accounts for up to 70% of all medication-related hospital admissions, resulting in $100 billion in healthcare costs annually Adherence rates have been reported as low as 0% in pediatric patients. Reasons for non-adherence are multifactorial. The most important determinants of non-adherence are consistently documented as complexity and duration of treatment regimens, as well as forgetfulness. Thus, children undergoing difficult hematopoietic stem cell transplants (HSCT) that require medication indefinitely are at high risk for medication non-adherence.

Only 4 published studies exist regarding adherence in pediatric HSCT. None address adherence to immunosuppressant medication, nor are they RCTs. Second, the complexity of most interventions for adherence is counter to the geographic, resource, and time constraints families of chronically ill children face. Adherence interventions based on conventional behavior theory have been cumbersome for families already stressed due to chronic illness. BE design is a significant paradigm shift to a simpler, less onerous approach that can engage those patients and families that would otherwise forego complicated adherence interventions. Although mHealth adherence apps are a widely available, simple, and innovative approach to addressing these problems, a third gap relates to poor usability. For example, a recent review of pediatric adherence apps found that none identified individual barriers to adherence, and nearly all were designed for adults. Thus, there is an urgent need to develop and evaluate innovative, accessible, and evidence-based approached to adherence among children receiving HSCT to prevent morbidity and mortality from GVHD.

The impact of non-adherence on clinical outcomes is largely unknown in pediatric HSCT. poor adherence is generally associated with adverse outcomes, including complications, hospital admissions, and even death. The societal burden of cancer care and HSCT is substantial and likely to increase based on the growing number of transplants each year. Clinicians and researchers have focused on GVHD prevention to minimize unnecessary treatment-related deaths. Acute GVHD develops in the first 100 days post-transplant. Children that develop acute GVHD have a 30% to 50% chance of survival. Morbidity and mortality due to GVHD can be decreased through prophylactic use of immunosuppressants. Although these medications are costly and produce unpleasant side effects, adherence is critical to decrease complications, reduce readmissions, and ultimately increase quality of life and survival.

Adherence is complex, but ultimately, the final common pathway to adherence is human behavior. In pediatrics, adherence is largely dependent on parents. As the primary caregivers, they are responsible for ensuring children receive the prescribed therapy correctly. In a high-risk HSCT population, caregivers are isolated with their child due to infection risk and must manage challenging treatment regimens at home, often with limited time and support. Complex behavioral interventions, typically employed to address adherence, are difficult to deliver and manage in the context of these daily tasks. Alternatively, behavioral economics (BE) theory suggests that small "nudges" can produce and sustain behavior change. A BE approach is a significant paradigm shift and assumes decision-making can be influenced through low-intensity interventions to lead patients to optimal choices. Improved adherence to medication and exercise programs using BE designed interventions in adults have been positive. Within pediatrics, BE has been successful in reducing childhood obesity, increasing vaccination rates, and improving adherence rates to infant HIV medications.

Eligibility criteria: Inclusion Criteria:

* Children of caregivers must be a) 0 to 21 years of age; b) receiving immunosuppression for an allogeneic transplant or anti-infective for an autologous transplant; c) discharged prior to Day 100 or completion of immunosuppression taper, and d) residing with the primary caregiver that enrolls on the study.
* Primary caregivers must be: d) English-speaking; and e) have an iOS or Android capable cellular device.

Exclusion Criteria:

* Adults unable to consent

Primary outcomes:
measure: Momentary Event Monitoring System (MEMS) Caps
timeFrame: Baseline to day 100
measure: Medication Adherence Measure (MAM)
timeFrame: Baseline to day 100
measure: System Usability Scale
timeFrame: Exit (Day 100)
measure: Posttransplant Perception Survey
timeFrame: Baseline (Week 0)
measure: Barrier Assessment Tool
timeFrame: Baseline (Week 0)
measure: Caregiver Satisfaction
timeFrame: Exit (Day 100)
measure: Pediatric Quality of Life Inventory (PedsQL) version 4.0
timeFrame: Week 3, Week 6, and Week 9
measure: Medy Remote Patient Management (RPM) medication box by Vaica
timeFrame: Baseline to day 100
Secondary outcomes:
measure: Demographic Data Form
timeFrame: Baseline (Week 0)
measure: Medication Possession Ratio (MPR)
timeFrame: Monthly until day 100
measure: Medication Level Variability Index (MLVI)
timeFrame: Weekly until day 100
measure: Graft vs. Host Disease (GVHD)
timeFrame: Weekly until day 100
measure: Readmissions
timeFrame: Weekly until day 100
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
References: