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| ID | Type | Description | Link |
|---|---|---|---|
| U18HS028380-01 | U.S. AHRQ Grant/Contract | View source |
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| Name | Class |
|---|---|
| Icahn School of Medicine at Mount Sinai | OTHER |
| The Hospital for Sick Children | OTHER |
| Emory-Children's Center | OTHER |
| Columbia University |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pediatric liver transplant recipients | Experimental | As this is a single arm trial, all eligible liver transplant recipients and their caregivers will be enrolled in this arm. Eligible participants are children who received a liver transplant at least 1 year prior to enrollment at a participating SNEPT center and continue to receive their post-transplant care at that center. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PeLTQL delivery via electronic means | Behavioral | Delivery of PeLTQL scoring information to clinicians via electronic means. |
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| Measure | Description | Time Frame |
|---|---|---|
| Implementation metric | The proportion of participants who complete the Pediatric Liver Transplant Quality of Life (PeLTQL) measure (either parent or child), at least once | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Patient baseline quality of life (child report on PeLTQL) | PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire. Range of scores (after transformation), range (0-100) Higher score = Better QoL | Time of enrollment |
| Patient quality of life (child report on PeLTQL) |
| Measure | Description | Time Frame |
|---|---|---|
| Rejection | Incidence of rejection of the transplanted liver | 24 months |
Inclusion Criteria:
The patient must meet all of below criteria to be eligible for enrollment in the study:
Exclusion Criteria:
None of the following may be present if the patient is to be eligible for enrollment in the study:
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| Name | Affiliation | Role |
|---|---|---|
| Eyal Shemesh, MD | Icahn School of Medicine at Mt. Sinai | Principal Investigator |
| George Mazariegos, MD | University of Pittsburgh | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States | ||
| Emory Children's |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24976329 | Background | 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. | |
| 42010801 | Derived | Ng VL, Kwan K, Logan S, Chessell J, Lobritto SJ, Hsu EK, Perito ER, Gupta NA, Dunphy C, Pieratt D, Kleinman LC, Shemesh E, Mazariegos GV. Minimal Clinically Important Difference for the PeLTQL in Pediatric Liver Transplantation: A Multicenter Analysis. Pediatr Transplant. 2026 May;30(4):e70320. doi: 10.1111/petr.70320. |
| Label | URL |
|---|---|
| Starzl Network Main Website | View source |
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| ID | Term |
|---|---|
| D002652 | Child Behavior |
| D000294 | Adolescent Behavior |
| ID | Term |
|---|---|
| D001519 | Behavior |
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| OTHER |
| Seattle Children's Hospital | OTHER |
| University of San Francisco | OTHER |
Single arm interventional trial with use of the RTC mobile application representing the intervention of interest
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PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire. Range of scores (after transformation), range (0-100) Higher score = Better QoL |
| 12 months post-enrollment |
| Proxy report of child's quality of life using PeLTQL | PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire. Range of scores (after transformation), range (0-100) Higher score = Better QoL | Time of enrollment |
| Proxy report of child's quality of life using PeLTQL | PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire. Range of scores (after transformation), range (0-100) Higher score = Better QoL | 12 months post-enrollment |
| Medication level variability index (MLVI) | Degree of fluctuation in immunosuppressant medication levels. Higher MLVI =worse adherence to medication. | Calculated for year following implementation (12 month period following enrollment) |
| PeLTQL discrepancy score | The discrepancy score is defined as the parent score minus the child's score on the PELTQL. PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire | First administration of PeLTQL in year 1 |
| PeLTQL discrepancy score | The discrepancy score is defined as the parent score minus the child's score on the PELTQL. PeLTQL = Pediatric Liver Transplant Quality of Life questionnaire | Second administration of PeLTQL within 24 months |
| Implementation metric | Percentage of patient-proxy dyads (amongst all enrolled) who completed the PeLTQL at least once. | 18 months |
| Implementation metric | Percentage of participating parent-child dyads (amongst all dyads) in which both the parent and the child completed one or more questionnaires during the study period. | 24 months |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| Columbia University Children's | New York | New York | 10027 | United States |
| Mt. Sinai | New York | New York | 10029 | United States |
| UPMC Children's | Pittsburgh | Pennsylvania | 15224 | United States |
| Seattle Children's | Seattle | Washington | 98105 | United States |
| Toronto Sick Kids | Toronto | Ontario | 10001 | Canada |