Clinical trial NCT05241847: Starzl Network Patient Reported Outcomes
Official title: Starzl Network for Excellence in Pediatric Transplantation (SNEPT) Implementation of a QoL Measure in Pediatric Transplant Recipients
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Liver Transplant; Complications
Quality of Life
Child Behavior
Adolescent Behavior
Keywords: quality of life
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
Collaborators: name: Icahn School of Medicine at Mount Sinai
name: The Hospital for Sick Children
name: Emory-Children's Center
name: Columbia University
name: Seattle Children's Hospital
name: University of San Francisco
First posted: 2022-02-16
Enrollment: 200
Sex: ALL
Minimum age (years): 8
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single arm interventional trial with use of the RTC mobile application representing the intervention of interest
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: This study uses a smartphone application/web interface (RealTime Clinic; RTC) to collect patient and parent reports of a pediatric liver transplant recipient's quality of life (QOL), and examines the extent to which QOL evaluations can be integrated into care with the help of the application. The QOL measure that is used in this study is the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire. Utilization, effectiveness, and efficiency data are evaluated.

Hypotheses are fully described in the protocol. The primary hypothesis is that 80% of recruited child-proxy dyads will have at least one RTC-enabled PeLTQL score at 12 months. Other hypotheses look at implementation metrics and patient outcomes.

Detailed description: Despite evidence supporting the benefits of QOL assessments and the availability of many QOL assessment instruments, the integration of these instruments into clinical practice has not yet become standard of care.

The Pediatric Liver Transplant Quality of Life (PeLTQL), the study measure, is a condition-specific 26-item questionnaire; the investigators will be evaluating both total scores as well as subdomain scores and, importantly, discrepancies between child and parent reports of the child's QOL.

The Electronic Platform: Real-Time Clinic (RTC). The platform will furnish providers with the total scores, subscale scores, thresholds ("met" vs. "not met"), discrepancy scores, and any question that has scores which may concern the clinician/clinical team.

This information will be available before the clinic visit and can guide and inform discussion and problem-solving between patient and clinical team. The study does not standardize the response to the results and will not suggest a preferred way of action. Interpretation of the results, as well as actions related to them are completely left to clinician's discretion.

The setting of this study - the Starzl Network for Excellence in Pediatric Transplantation (SNEPT Centers). SNEPT is a learning healthcare network that was established in 2018 to accelerate improvement in transplant outcomes by incorporating innovation, technology and the patient voice to address gaps in care that were identified by the collaborative transplant centers and family representatives.

Study Aims

The primary aim is successful implementation of the RTC app-based tool to obtain PeLTQL scores from pediatric liver transplant recipients and their parents or caregivers.

The investigators also aim to assess the usability, impact, and ease of use (both for clinicians and patients/families) of the RTC platform and app-based version of the PeLTQL. The investigators will evaluate clinicians' subjective views on the platforms' ease of use, their time spent evaluating results, both in clinic and before patient visits, as well as the app's impact on back-end clinic workflow.

Impact on patient outcomes will be evaluated using pre-post comparisons on adherence (determined by the objective medication level variability index, MLVI) as well as on the PeLTQL.

Eligibility criteria: Inclusion Criteria:

The patient must meet all of below criteria to be eligible for enrollment in the study:

1. The patient is \> 8 at enrollment and \< 20 years of age at 2 year post enrollment.
2. The patient received a liver transplantation at least 1 year prior to enrollment.
3. The patient and parent/ guardian have internet access either through a smartphone, tablet or computer.
4. The patient and at least one guardian speaks English or Spanish at a level that allows them to understand the study procedures and consent to the study.

Exclusion Criteria:

None of the following may be present if the patient is to be eligible for enrollment in the study:

1. The patient is expected to transition to another service (e.g., adult clinic, another hospital) in the year following enrollment.
2. The guardian or patient (in a developmentally-appropriate manner) do not understand the study procedures. This will be verified by asking both guardian and adolescent to repeat the study procedures.

Primary outcomes:
measure: Implementation metric
timeFrame: 12 months
Secondary outcomes:
measure: Patient baseline quality of life (child report on PeLTQL)
timeFrame: Time of enrollment
measure: Patient quality of life (child report on PeLTQL)
timeFrame: 12 months post-enrollment
measure: Proxy report of child's quality of life using PeLTQL
timeFrame: Time of enrollment
measure: Proxy report of child's quality of life using PeLTQL
timeFrame: 12 months post-enrollment
measure: Medication level variability index (MLVI)
timeFrame: Calculated for year following implementation (12 month period following enrollment)
measure: PeLTQL discrepancy score
timeFrame: First administration of PeLTQL in year 1
measure: PeLTQL discrepancy score
timeFrame: Second administration of PeLTQL within 24 months
measure: Implementation metric
timeFrame: 18 months
measure: Implementation metric
timeFrame: 24 months
Other outcomes:
measure: Rejection
timeFrame: 24 months
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Atlanta
country: United States
facility: Emory Children's
state: Georgia
city: New York
country: United States
facility: Columbia University Children's
state: New York
city: New York
country: United States
facility: Mt. Sinai
state: New York
city: Pittsburgh
country: United States
facility: UPMC Children's
state: Pennsylvania
city: Seattle
country: United States
facility: Seattle Children's
state: Washington
city: Toronto
country: Canada
facility: Toronto Sick Kids
state: Ontario
Study officials:
affiliation: Icahn School of Medicine at Mt. Sinai
name: Eyal Shemesh, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Pittsburgh
name: George Mazariegos, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ng V, Nicholas D, Dhawan A, Yazigi N, Ee L, Stormon M, Gilmour S, Schreiber R, Taylor R, Otley A; PeLTQL study group. Development and validation of the pediatric liver transplantation quality of life: a disease-specific quality of life measure for pediatric liver transplant recipients. J Pediatr. 2014 Sep;165(3):547-55.e7. doi: 10.1016/j.jpeds.2014.05.024. Epub 2014 Jun 26.