A multisite randomized controlled trial of an early palliative care intervention in children with advanced cancer: The PediQUEST Response Study Protocol

Veronica Dussel, Liliana Orellana, Rachel Holder, Rachel Porth, Madeline Avery, Joanne Wolfe, Veronica Dussel, Liliana Orellana, Rachel Holder, Rachel Porth, Madeline Avery, Joanne Wolfe

Abstract

Background: The Pediatric Quality of Life and Evaluation of Symptoms Technology Response to Pediatric Oncology Symptom Experience (PQ-Response) intervention aims to integrate specialized pediatric palliative care into the routine care of children, adolescents, and young adults (AYAs) with advanced cancer.

Aims: To evaluate whether PQ-Response, compared to usual care, improves patient's health related quality of life (HRQoL) and symptom burden (aim 1), parent psychological distress and symptom-related stress (aim 2), and family and symptom treatment activation (aim 3).

Design: Multisite, randomized (1:1), controlled, un-blinded, effectiveness trial comparing PediQUEST Response (intervention) vs usual cancer care (control).

Setting: Five US large, tertiary level pediatric cancer centers.

Participants: Children (≥2 years old)/AYAs who receive care at any of the participating sites because of advanced cancer or any progressive/recurrent solid or brain tumor and are palliative care "naïve." Target: 200 enrolled patient-parent dyads (minimum goal: 136 dyads randomized, N = 68/arm).

Interventions: PediQUEST Response: combines patient-mediated activation (weekly feedback of patient- and parent-reported symptoms and HRQoL to families and providers using the PediQUEST web system) with integration of the palliative care team. Usual Cancer Care: participants receive usual care, which can include palliative care consultation, and use PediQUEST web to answer surveys, with no feedback.

Methods: Following enrollment, patients (if ≥5 years) and one parent receive weekly PediQUEST-Surveys assessing HRQoL (Pediatric Quality of Life Inventory 4.0) and symptom burden (PediQUEST-Memorial Symptom Assessment Scale). After a 2-week run-in period, dyads who answer ≥2 PediQUEST surveys per participant (responders), are randomized (concealed allocation) and followed up for 16-weeks. Parents answer six additional surveys (parent outcomes).

Outcomes: Primary: mean patient HRQoL score over 16-weeks as reported by a) the parent; and b) the patient if ≥5 years-old. Secondary: patient's symptom burden; parent's anxiety, depressive symptoms, symptom-related stress; family activation; and symptom treatment activation.

Trial registration: ClinicalTrials.gov (NCT03408314) 1/24/18. https://ichgcp.net/clinical-trials-registry/NCT03408314.

Conflict of interest statement

The authors have declared that no competing interests exist.

Copyright: © 2022 Dussel et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Figures

Fig 1. PediQUEST response trial overview.
Fig 1. PediQUEST response trial overview.
*After consent there is a two-week run-in period to identify (and exclude) non-responders (i.e., dyads who answer Abbreviations: S-TAI: Spielberger’s State-Trait Anxiety Inventory (two components: State and trait); MOS-SS: Medical Outcomes Study-Social Support; CES-D-10: Center for epidemiologic Studies Short Depression Scale; UCT: Use of Complementary Therapies Checklist; aRSQ-Stress: Adapted Response to Stress Questionnaire (stress portion).
Fig 2. Main activities of the response…
Fig 2. Main activities of the response team integration component.
Abbreviations: RC: Research coordinator; RT: Response Team; POT: Primary Oncology Team; PQ e-PROMS: PediQUEST electronic patient reported outcome measures; Local PI: Site Principal Investigator; PQ ListServ: PediQUEST Listserv (expert listserv).

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