Patient-reported outcome measures in pediatric solid organ transplantation: Exploring stakeholder perspectives on clinical implementation through qualitative description

Patient-reported outcome measures (PROMs) are standardized instruments used to collect data about the subjective assessment of medical care from the patient perspective [1‐4]. In this context, PROMs represent “any report of the patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else” [5] and can comprise information about symptoms, functional status, satisfaction, adherence and quality of life (QOL) [6‐10]. When implemented effectively, PROMs can engage patients meaningfully and capture their varied experiences and attitudes [1‐4]. Further, findings show that PROM data can increase patients’ satisfaction with care and potentially improve patients’ symptom management, overall well-being, and QOL [7, 11‐13]. It is documented that the systematic collection of PROM data is more reflective of underlying health status than clinical objective reporting [7, 8, 11‐13].

Efforts to implement PROMs into clinical practice are in the early stages of development [9], and research that considers the clinical application of PROMs in pediatric care is scarce [1, 14, 15]. However, implementing PROMs within pediatric settings has gained increasing importance as health services prioritize patient-centred care [1‐4, 14, 16]. Research in adult clinical settings offers preliminary support for the role of PROMs in assessing the burden of disease and treatment and enhancing health outcomes through improved patient-provider communication, shared decision-making, and self-care management [7, 11‐13, 17]. Emerging research indicates that the systematic integration of PROMs in pediatric care may result in increased patient-provider communication about psychosocial issues, such as social and emotional functioning [15, 18, 19], increased referrals to psychosocial supports [18], and improved health-related QOL [19]. These are meaningful outcomes that support the use of PROMs in pediatric patient populations that require a preventative approach to address risks associated with health-related QOL [19].

Nonadherence to life-long medical follow-up is a well-documented concern in pediatric transplant care, leading to potential consequences such as graft loss, organ rejection and death [20‐22]. Pediatric transplant patients also have an increased risk of experiencing psychological distress, social isolation, impaired social functioning, cognitive difficulties, and behavioural problems [23‐29]. Using PROMs within this patient population could identify concerns regarding functional and emotional status, help healthcare practitioners (HCPs) to detect under- and unrecognized problems (e.g., depression and anxiety), and solicit support from a multi-disciplinary team when necessary [18].

Integrating PROMs into clinical pediatric transplant care settings requires a shared vision among patients, caregivers and HCPs [2, 4, 30, 31]. The need to ‘build collaborative action’ among stakeholders is considered necessary to create a capacity for change in the process of implementation [32, 33]. Yet to date, research has not explored stakeholder perspectives on integrating PROMs into pediatric transplant care. The purpose of the current qualitative study is to elicit perceptions from patients, caregivers, and HCPs about the potential role of PROMs in the clinical care of pediatric transplant patients to inform effective implementation in this setting.

Qualitative description is a research method that assumes a naturalistic approach [34]. It strives to depict a phenomenon by staying close to the ‘surface’ meaning of words and provides a ‘comprehensive summary’ of events that reflects their occurrence with minimal interpretation [35]. Qualitative description was used in the current study to elicit stakeholder perspectives of those directly involved in implementing PROMs into clinical pediatric transplant care.

Participants were recruited across five of the largest pediatric transplant centres in Canada, each receiving institutional research ethics approval: Alberta Children’s Hospital (REB18-0140), British Columbia Children’s Hospital (H18-00,391), Health Sciences Centre Winnipeg (HS21697 (H2018:146)), Stollery Children’s Hospital (Pro00079805), and The Hospital for Sick Children (REB1000057043). Eligible patients included any solid organ transplant recipient between eight and 17 years of age, who was ≥ three months post-transplant. Eligible caregivers included any individuals who acted as the primary caregiver(s) for an eligible patient, even if the patient chose not to participate in the study. All patients and caregivers were required to be English-speaking with the capacity to participate in an interview as determined by a HCP in their circle of care. Eligible HCPs included any member of the interdisciplinary healthcare team who has worked within pediatric solid organ transplantation for a minimum of one year. Purposive sampling was used to obtain maximum variation across age, gender, and transplant program for all participants, as well as discipline for HCPs. Written informed consent or assent was obtained from all participants; when patients provided assent, informed consent was obtained from their parents/legal guardians. Demographics were collected for all study participants and selected medical data was collected for patient participants.

A semi-structured interview guide was developed by the research team based on clinical and research experience [36]. Interviews were approximately 45 minutes in length and were conducted in-person by two facilitators (KY & SJA) trained in qualitative methods. Questions explored participant perspectives about QOL and their experiences around the use of PROMs in clinical care. Two PROMs were reviewed with participants – the PedsQL™ Generic Core Scales [37] and the PedsQL™ Transplant Module [38]; their selection was based on a systematic review identifying PROMs used in pediatric solid organ transplantation [36]. After reviewing the PROMs, participants were shown a short video of a Dutch electronic PROM (ePROM) platform [39, 40], which illustrated the implementation of ePROMs into clinical use in a pediatric hospital.

Interviews were audio-recorded, transcribed verbatim, de-identified, and subjected to content and thematic analysis as outlined by Elo and Kynga [41]. Two research team members (SJP & KY) coded the data independently, and categories were reviewed and refined until consensus and thematic saturation were reached [41, 42]. Trustworthiness was achieved through soliciting rich description and facilitating member checking within interviews, as well as hosting frequent team meetings to support in-depth, iterative analysis with reflexive discussion among team members. The qualitative software program, N-Vivo 12 [43], was used for qualitative data management.

A total of 61 interviews were conducted with 63 participants–20 patients (PTs), 22 caregivers (CGs), and 21 HCPs. Three eligible patients/caregivers declined to participate due to scheduling conflicts, one declined due to disinterest, and all consenting participants completed the study. Two interviews comprised the patient and their parent due to patient preference. Participants represented a diverse sample across several variables, including ethnicity, geographic location, organ transplant program and socio-demographic background. Demographic characteristics for the sample are summarized in Table 1.

Nearly all patients, caregivers and HCPs (60/63; 95%) endorsed the implementation of PROMs to enhance pediatric transplant clinical care. Three participants acknowledged the potential of PROMs to solicit essential patient information but were uncertain regarding their clinical use. Collectively, participants described three primary roles for PROMs that offered support for their use in pediatric clinical care: (1) to bring a transplant patient’s overall well-being into the clinical care conversation; (2) to improve patient communication and engagement; and, (3) to inform the practice of clinical pediatric transplant care. Insights gleaned for the effective implementation of PROMs are presented.

This study contributes to current research that supports the use of PROMs in clinical pediatric care and guides their effective implementation into practice. Participant stakeholders (patients, caregivers and HCPs) supported the use of PROMs in pediatric transplant care and described their role as encouraging discussions about patient well-being, improving patient engagement, and informing their clinical care. In particular, HCPs emphasized the potential role of PROMs to proactively address mental health, highlighting the potential negative psychological and social impacts associated with pediatric transplantation [23‐29]. Pediatric transplant patients noted their lack of engagement or direct communication with HCPs, offering almost unanimous support for the collection of PROMs to share their perspectives and safely disclose concerns during clinic visits.

Existing literature in both pediatric and adult chronic illness populations indicates improved patient-provider communication with the aid of PROMs, in particular, giving attention to patient psychosocial concerns that are often overlooked in clinic [12, 19]. HCPs in the current study reported a similar perspective and there was consensus among all stakeholders that capturing the patient’s voice should be a priority in clinical care.

Most participants preferred the use of a disease-specific PROM (PedsQL™ Transplant Module [38]) as a means to share patient experience. Further, participants indicated that eight to 10 years of age was ideal to initiate PROMs assessments; current research shows that most children are able to respond reliably to PROMs at this age [44]. While some participants acknowledged a role for caregivers to complete self- or proxy-reported PROMs (of their child’s experience), researchers have identified discrepancies between child self-report and parent proxy-report, supporting the view that proxy-reporting should not be used as a substitute for child self-report [45‐47].

The use of PROMs in clinical practice is quickly moving towards electronic administration in both pediatric and adult healthcare settings [15, 18, 39, 40]. An evaluation of the pediatric, Dutch ePROM system, KLIK, revealed that HCPs found ePROM data useful in 95%–100% of clinical consultations [18]. One benefit of using an ePROM platform in a pediatric setting is the cross-compatibility of electronic devices (e.g., laptop, smartphone, tablet) which could cater to the widespread Internet use among children and youth [48]. In the current study, all HCPs and a majority of patients and caregivers supported the use of an ePROM platform to collect and integrate PROM data in a pediatric transplant clinical care setting. Bennett, Jensen and Basch [49] reviewed five ePROM systems used in adult oncology and concluded that ePROM platforms provided instantly accessible data, eliminated manual data entry and scoring, created easily interpretable reports and saved time during clinic visits. In comparison, paper-based PROMs have been cited as a major barrier to integrating PROM data into clinical care with the increased time and human resource capacity required to collect, analyze, and report results [49].

In the current study, caregivers and HCPs believed that prompt feedback to patients and families to inform clinical pediatric transplant care was best facilitated by an ePROM system in comparison to paper-based PROM administration. Further to administrative advantages, eHealth technology provides innovative approaches to understand children’s perspectives of their illness experience [49‐52]. Our findings suggest that integrating ePROMs into clinical settings may support a system of care that proactively screens for psychosocial concerns in this patient population. Future research will need to explore if ePROMs are associated with improved health outcomes in clinical pediatric transplant care, such as QOL and medication adherence.

Limitations of this study include recruiting only English-speaking participants as well as those without severe cognitive impairment. It is possible that the perspectives of these individuals may be different from the current findings. Further, while PROM instruments and an e-platform were presented as exemplars, the specific instruments and platform reviewed may have influenced participants’ perceptions. Future studies will need to explore how to ensure PROMs are accessible in the face of language barriers and cognitive challenges to allow for accurate self-report. They will also need to incorporate various PROM measures to facilitate the cross-comparison of instruments. While the disease-specific PROM (PedsQL™ Transplant Module [38]) may be preferred to elicit patient experience, the benefit of using generic instruments (e.g., PedsQL™ Generic Core Scales [37]) to facilitate direct comparisons across disease groups, interventions, treatment regimens, and population norms cannot be overlooked when planning future research protocols [53, 54].

The best-practice methods to implement ePROMs into pediatric clinical practice are not universally established. Consensus will require further collaboration and support to address individual, organizational, community, and contextual factors of implementation [32, 33]. Most recently, the International Society for the Quality of Life (ISOQOL) published two reports highlighting important methodological and operational decisions for the clinical implementation of PROMs [55, 56]. The current study captured the perspectives of patients, caregivers and HCPs in a pediatric transplant setting about the role of PROMs in clinical care and specific considerations for its implementation. The diversity of clinical protocols across pediatric transplant programs and centres in Canada will need to be considered to move towards a consensus of best-practice for ePROMs in pediatric clinical care. Further, considerations for professional development, HCP training, and patient-family education are essential. Next steps should include the development, usability testing, and evaluation of a proposed ePROM platform which could inform future research initiatives.