Patient-reported outcome measures in pediatric palliative care—a protocol for a scoping review

The field of pediatric palliative care (PPC) includes all children and adolescents living with complex life-threatening or life-shortening conditions. Despite the previous lack of consensus when defining palliative care, and the often interchangeable use of palliative care, hospice care, end of life, and terminal care [1, 2], the definition of PPC seems to be more unified. According to the World Health Organization (WHO), PPC is “the active total care of the child’s body, mind and spirit, and [this] also involves giving support to the family” [3]. Due to life-sustaining technology of modern medicine, especially in high-income countries, an increasing number of children in PPC become young adults [4]. Thus, the patient population is often defined as children, adolescents, and young adults (CAYA; aged 0-25 years) [5]. The vast majority (> 97%) of children in need of PPC live in low- and middle-income countries. Worldwide, children with HIV and congenital malformations represent almost half of the population in need of PPC, followed by children with perinatal conditions, such as extreme prematurity and birth trauma [6]. The latter two also constitute the largest groups in Europe, followed by children with neurologic disease and cancer [7]. Children in PPC suffer a wide range of symptoms during the course of their disease. Although most of them have life-limiting conditions, most of these children become young adults, requiring complex care throughout their childhood and onwards [7].

Although adult palliative care and PPC share the overall aim of quality of life [8], PPC differs from adult palliative care as children move through different development stages while receiving care; they have different communication needs and a stronger dependency on their families [9]. Children often receive treatment that is more aggressive, and they are more frequently hospitalized compared to adults [10, 11]. This is contradictory to the evidence suggesting that homebased PPC is the best way to meet the aim of total care for the child and the family [9, 12‐17]. Hospitalization affects the family dynamics because the sick child cannot interact with the rest of the family, or participate in kindergarten, school, or activities as if they were at home [18].

Regardless of where the child receives PPC, the child and family rely on a close and continuous contact with healthcare personnel (HCP) to support their care needs. HCP need specialized knowledge on PPC to meet the complex needs of these children, and ensure quality, continuity, and coordination of care. A specialized palliative care service is associated with increased quality of life for the child [19]. PPC teams need tools for effective communication with each other, as well as with other parts of the healthcare services [20]. At the same time, communication between families and HCP is a core challenge in PPC [21], and systematic data collection (e.g., PROMs) could facilitate this communication and ensure that the relevant needs of the child are in focus.

To enhance quality of PPC and to ensure that the care meets the needs of the child and family, standardized measures for assessing symptoms and care needs can support HCP in their care provision. However, often the care needs are reported on behalf of the child, without directly asking children what they want or desire [22]. The subjective self-report, capturing the patients’ views of their own health, known as “Patient-Reported Outcome” (PRO), is defined as “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” [23]. Patient-reported outcome measures (PROM) is the measure or method to gather these patient reports. In addition, patient-reported experience measures (PREMs) are frequently being applied to capture the patient’s experience or satisfaction when being cared for; however, PRO, PROM, and PREM are often used interchangeably. PROM and PREM are complementary to the objective measures of anthropometrics like blood pressure, height, weight, or various blood analyses. In addition to the self-report of PRO, proxy measures conducted by family members or HCP are particularly relevant for children unable to self-report [24]. One obvious challenge with proxy measures is that PROMs often demand an assessment of subjective experiences, and when these are reported by a proxy, a level of uncertainty is added to the caregiver’s interpretation of the needs of the child [24].

Within PPC, standardized measures to assess the needs of children are not systematically applied [25]. The field with greatest focus has been within quality of life in PPC [26]. However, a systematic review on instruments to measure the impact of interventions on PPC [27] did not find any ideal outcome assessment tool for use in PPC. A systematic review from 2019 addressed the implementation of PRO for children with chronic illness in medical settings and found that in general, implementation of PRO increase attention to psychosocial factors [28]. We have previously assessed experiences with homebased PPC supported by eHealth, and found that systematic assessment of symptoms and needs was regarded important, but underdeveloped [29]. Previous research indicates promising results of implementing PROMs in children with various health challenges, but the relevance of PROMs in PPC has not been systematically assessed.

The main objective for the proposed scoping review is to investigate peer reviewed, published studies on PROMs in PPC to map the nature of current research, to identify and summarize existing knowledge, and to present current evidence gaps [30]. We anticipate that descriptions of PROMs in non-peer reviewed sources would lack a rigorous psychometric development; thus, we will limit our search to peer-reviewed literature.

We anticipate that the results of our scoping review will have implications for clinical practice, future primary research, and future systematic reviews based on the knowledge we identify. However, our findings must be cautiously interpreted, as a scoping review simply aims to map evidence in a field, while a stringent systematic review aims to clearly answer a research question with an appraisal of the risk of bias [30‐32].

The proposed scoping review will identify measures relevant to apply for researchers and HCP caring for a PPC population and will identify knowledge gaps of utmost importance for clinical care and future research to improve quality of PPC.

To the best of our knowledge, this scoping review will be the first to identify specific measures and experiences with these in a pediatric palliative population in order to enhance their care. The population demands various measures, depending on whether the child is able to complete any assessment, or if a proxy assessment by parents or HCP is required. Thus, the results will be extensive, covering aspects of interest for HCP in close contact with these children, their families, and care teams, as well as researchers striving to inform care and policy makers, as well as future research.