Patients In Palliative Care Setting Research Articles

How accurate is clinical prognostication by oncologists during routine practice in a general hospital and can it be improved by a specific prognosis training programme: a prospective interventional study

ObjectivesOncologists need competence in clinical prognostication to deliver appropriate care to patients with cancer. Most studies on prognostication have been restricted to patients in palliative care settings. This paper investigates...

Patients’ experiences with shared decision-making in home-based palliative care – navigation through major life decisions

BackgroundThis study addresses the issue of shared decision-making (SDM) in a Norwegian home-based palliative care setting. The significance of patient involvement in SDM is widely acknowledged, and many patients want to participate in decisions about care and treatment. Yet, it remains a need for more knowledge regarding the initiators and approaches of SDM in the context of home-based palliative care, particularly from the patients’ perspective. The aim of this study is to understand patients' experiences and preferences for SDM in home-based palliative care, seeking to enhance the quality of care and direct the planning of healthcare services.MethodsWe used a qualitative explorative design. A hermeneutic approach was employed, and data was collected through in-dept interviews with 13 patients.ResultsThe study uncovered an overarching theme of "Navigating to reach own decisions," comprising three sub-themes: “To be trapped in life without decisions to act on”; “To surrender to others and let others deal with decisions”; “To continue to be oneself without focusing on disease and decision-making”.ConclusionsThe findings underscore the need for flexible, person-centered approaches in SDM, tailored to the fluctuating health literacy and changing preferences of patients in palliative care settings. Our study contributes to the understanding of SDM in palliative care by highlighting how patients navigate the balance between autonomy and reliance on HCPs. Future research should explore how healthcare systems, including HCPs’ roles in the system, can adapt to the patients’ dynamic needs, to ensuring that SDM will remain a supportive and empowering process for patients at all stages of their disease.

Dental and nursing collaboration in oral healthcare research in palliative care inpatient settings.

Collaboration between palliative care nurses and oral health professionals has been frequently recommended to ensure optimal oral healthcare. However, the importance of this collaboration to the success of evidence-based protocols, involving oral healthcare interventions in the inpatient palliative care setting, is less discussed. Feasibility requires consideration during the planning, intervention and implementation phases of any clinical trial; all of which typically rely on good collaboration with nursing staff. Therefore, it is imperative that nursing staff appreciate the need for oral healthcare at the end of a person's life. Nursing and dental collaboration is critical to the feasibility of running research trials in this group and can help overcome some of the logistical and ethical challenges in this vulnerable population.

Racial/Ethnic and Language Concordance among Palliative Care Clinicians and Their Patients in Inpatient Palliative Care Settings in California (QI123)

Racial/Ethnic and Language Concordance among Palliative Care Clinicians and Their Patients in Inpatient Palliative Care Settings in California (QI123)

Neoliberally framed suffering in professionals and patients in palliative care settings – ’telling cases’ from an ethnographic multi-sited field study in Denmark

ABSTRACT In western countries, palliative care is a part of the formal healthcare system, ruled by neoliberal, organisational ideas. This Danish study illuminates how nurses and patients with non-western immigrant backgrounds (inter)act in relation to encounters in palliative care settings. Two ‘telling’ cases were selected, based on interviews and participating observations from a multi-sited ethnographic field study in Denmark. The results showed different care consequences of the structural framework, referring to, for example, political healthcare logics, strategies, and priorities, and illuminated how neoliberally organised healthcare systems inflicted suffering on patients and frustration amongst professionals. It did not seem that the immigrant background per se induced the challenges. The understandings of care and treatment conflicted as nurses often represented a medical, neoliberal logic of disease, treatment, and care, and patients often represented a non-medical logic of disease, formed by their lives. When palliative care took place in a general setting, (inter)actions were also influenced by a curative agenda of treatment and care. The study added to the evidence of the power of neo-liberal rationality across different forms of care and populations, regardless of ethnicity or location of that care.

Monitoring the clinical practice of palliative sedation (PALSED) in patients with advanced cancer: an international, multicentre, non-experimental prospective observational study protocol

BackgroundPalliative sedation involves the intentional lowering of consciousness at the end of life. It can be initiated to relieve a patient’s burden caused by refractory symptoms at the end of life. The impact of palliative sedation needs to be clinically monitored to adjust the proper dose and regimen of sedative medication to ensure that patients are at ease and comfortable at the end of their lives. Although there is consensus among health care professionals and within guidelines that efficacy of palliative sedation needs to be closely monitored, there is no agreement about how, when, and by whom, this monitoring should be performed. The aim of this study is to evaluate the effects of palliative sedation by measuring the discomfort levels and sedation/agitation levels of the patients at regular timepoints. In addition, the clinical trajectories of those patients receiving palliative sedation will be monitored and recorded.MethodsThe study is an international prospective non-experimental observational multicentre study. Patients are recruited from in-patient palliative care settings in Belgium, Germany, Italy, Spain and the Netherlands. Adult patients with advanced cancer are monitored by using proxy observations of discomfort (DS-DAT) and depth of sedation/agitation levels (RASS-PAL) during palliative sedation. After the palliative sedation period, the care for the specific participant case is evaluated by one of the attending health care professionals and one relative via a questionnaire.DiscussionThis study will be the first international prospective multicenter study evaluating the clinical practice of palliative sedation including observations of discomfort levels and levels of sedation. It will provide valuable information about the practice of palliative sedation in European countries in terminally ill cancer patients. Results from this study will facilitate the formulation of recommendations for clinical practice on how to improve monitoring and comfort in patients receiving palliative sedation.Trial registrationThis study is registered at Clinicaltrials.gov since January 22, 2021, registration number: NCT04719702.

Website information on visiting policies at specialist in-patient palliative care settings during COVID-19 pandemic across Central and Eastern Europe: a quantitative and qualitative study

Website information on visiting policies at specialist in-patient palliative care settings during COVID-19 pandemic across Central and Eastern Europe: a quantitative and qualitative study

Why patients in specialist palliative care in-patient settings are at high risk of falls and falls-related harm: A realist synthesis

Background: Falls are the third highest reported safety incident in Specialist Palliative Care in-patient settings and yet specific risk factors connected with falling and associated outcomes in this setting are poorly understood. Aim: To understand the key individualised risk factors leading to falls in specialist in-patient palliative care settings and understand the implications and outcomes for the patients who fall. Design: A realist synthesis of the literature, reported following the Realist And Meta-narrative Evidence Syntheses: Evolving Standards (RAMESES) standards. Data sources: An iterative literature search was conducted across three recognised health collections as well as grey literature from policy, practice and other relevant areas. Results: Falls taking place within in-patient specialist palliative care settings can cause significant harm to patients. The risk factors for these patients are multifaceted and often interlinked with underpinning complex realist mechanisms including a history of falls, the age of the person, impact of complex medications, improving functional status and the presence of delirium. Conclusion: In-patients in specialist palliative care settings are at risk of falling and this is multifactorial with complex reasoning mechanisms underpinning the identified risks. There is a significant impact of a fall in this cohort of patients with many sustaining serious harm, delayed discharge and both physical and psychological impacts.

The Impact of ASCITIC Paracentesis on Symptomatic Outcomes in Palliative Patients: 3-Year Experience of a Melbourne Inpatient Palliative Care Unit Using Palliative Care Outcomes Collaboration Assessments

Background Ascites formation is a common occurrence in patients receiving palliative care. This is associated with symptoms that may respond to drainage. Aim To review and quantify ascites-related symptoms pre- and post-paracentesis to evaluate its benefit in an inpatient palliative care setting. Methods A retrospective audit of patients admitted to an inpatient palliative care unit who underwent paracentesis between November 2016 to June 2020 was performed. The primary outcome was a symptomatic benefit of paracentesis. Secondary outcomes assessed the associated complications as well as the alterations to functional status. Data were analysed using a paired T-test. Results 43 instances of ascitic paracentesis were performed on a total of 26 patients. Three patients were excluded from the study due to the technical failure of their paracentesis procedure. There was a mean 0.16-point reduction in pain (95% confidence interval (CI) −0.33 to 0.65), a mean 0.03-point increase in dyspnoea (95% CI −0.51 to 0.46), and a mean 0.32-point reduction in nausea (95% CI −0.09 to 0.74). Conclusions Ascitic paracentesis in the palliative setting may demonstrate some benefit in managing symptoms associated with ascites. Although the findings of this study did not achieve statistical significance, these results may be substantiated by future studies with larger sample size.

A Systematic Review and Bayesian Network Meta-Analysis Investigating the Effectiveness of Psychological Short-Term Interventions in Inpatient Palliative Care Settings.

This paper reviews and summarises the evidence of short-term psychosocial interventions (up to 12 sessions delivered within less than eight weeks) on anxiety, depression, and emotional distress in palliative patients in inpatient settings. We screened publications from the following five databases, Embase, PubMed, PsycINFO, Web of Science, and CINAHL, from their inception to 10 September 2021. The eligible studies included controls receiving standard palliative care, actively treated controls, and wait-list controls. Nine studies met the eligibility criteria and reported the effects of five psychosocial interventions in a total of N = 543 patients. We followed PRISMA-guidelines for outcome reporting and the Cochrane Risk of Bias Assessment Tool for assessing study quality. This paper used the network meta-analysis to compare multiple treatments by providing greater statistical power and the cross-validation of observed treatment effects, using the R package BUGSnet. Compared to control groups, the following psychosocial interventions in inpatient settings showed to be superior: life review interventions were the best ranked treatment for improving anxiety and distress, while the top ranked treatment for reducing depression was outlook intervention. The short-term psychosocial interventions investigated in this meta-analysis, especially life review intervention, are feasible and can potentially improve anxiety, depression, and distress in palliative inpatients and should therefore be offered in inpatient settings.

Investigating the benefits and harms of hypodermoclysis of patients in palliative care: A consecutive cohort study

Background: Palliative populations are at risk for dehydration which can cause discomfort, distress and cognitive symptoms. Subcutaneous hydration (‘hypodermoclysis’) has been used as an alternative administration route to the more invasive intravenous route, but research is lacking on its net clinical effects (harms and benefits) for palliative populations, particularly in real world settings. Aim: To quantify prospectively the net clinical effects of hypodermoclysis in palliative patients with advanced disease who required supplementary fluids. Design: Multisite, multinational consecutive cohort study. Setting/participants: Patients receiving hypodermoclysis in an inpatient palliative care setting. Results: Twenty sites contributed data for 99 patients, of which 88 had complete benefits and harms data. The most common primary target symptom for infusion was generalised weakness (18.2%), and the most common non-symptom indication was supplemental hydration (31.8%). Benefits were experienced in 33% of patients in their primary target symptom, and in any symptom in 56.8%. Harms were experienced in 38.7% of patients (42% at Grade 1). Benefits increased with higher performance status, while harms were more frequent in patients with lower performance status (Australia-modified Karnofsky performance status ⩽40). Patients in the terminal phase of their illness experienced the least benefit (15.4% in any indication only) and had more frequent harms (38%). Conclusions: Hypodermoclysis may improve certain symptoms in patients in palliative care but frequency of harms and benefits may differ at certain timepoints in the illness trajectory. Further research is needed to better delineate which patients will derive the most net clinical benefit from hypodermoclysis.

Unintended Consequences of the 2016 CDC Guidelines for Prescribing Opioids for Chronic Pain for Patients in Palliative Care Settings in Hampton Roads: Findings from a Mixed Methods Study

Unintended Consequences of the 2016 CDC Guidelines for Prescribing Opioids for Chronic Pain for Patients in Palliative Care Settings in Hampton Roads: Findings from a Mixed Methods Study

Potentially inappropriate prescribing in older adults with cancer receiving specialist palliative care: a retrospective analysis of medical records

Abstract Introduction Older adults (≥65 years) with cancer often have existing comorbidities requiring multiple medications (1). Optimising medications in palliative care requires clinicians to consider whether each medication is appropriate in relation to patients’ context, treatment goals and life expectancy. The reported prevalence of potentially inappropriate prescribing (PIP) in general palliative care settings ranges from 15 to 92% (2). However, the application of tools that are specific to populations with limited life expectancy has been lacking in previous research (2). Aim To describe and assess the appropriateness of prescribing practices for older adults with cancer in the last seven days of life in an inpatient palliative care setting. Methods This was a retrospective observational study of medical records for older adults (≥65 years) with cancer who received inpatient specialist palliative care services in a hospice setting in Ireland in the final week of life over a two-year period. Data were extracted relating to patient demographics and prescribed medications using an electronic pro-forma. Medication appropriateness was assessed using the following tools: STOPPFrail (Version 2; consists of 25 deprescribing criteria for use in frail older adults with limited life expectancy), the OncPal deprescribing guideline (consists of eight medication classes for deprescribing in palliative patients with cancer) and criteria for identifying Potentially Inappropriate Prescribing in older adults with Cancer receiving Palliative Care (PIP-CPC; consists of 24 criteria for identifying potentially inappropriate prescribing of medications for symptomatic relief in older adults with cancer). These tools were retrospectively applied to the extracted data by the lead researcher. Data were analysed (Stata, Version 15) using descriptive statistics, including means (standard deviation, SD), medians (inter-quartile range, IQR) and frequency and percentage. Results One hundred and eighty older adults with cancer were included in this study. The majority were male (60.6%) and the median age was 74 years (range 65-94 years). The most common primary cancer diagnoses affected the digestive organs (31.7%), respiratory and intrathoracic organs (18.8%) and male genital organs (10%). Almost all patients (94.5%) had at least one comorbid condition (median 3, IQR 2-5). The median number of medications increased from five (IQR 3-7) seven days before death, to 11 medications on the day of death (IQR 9-15). The most prevalent drug classes were opioids, antipsychotics, antispasmodics, benzodiazepines and paracetamol. More than half of patients had at least one PIP identified by the tools (n=97, 53.9%). The identified prevalence of PIP per tool in the patient cohort was: STOPPFrail V2 (20.6%), OncPal (12.7%), PIP-CPC (32.8%). However, several criteria could not be applied due to the absence of clinical information. Conclusion This study highlights that the number of medications prescribed to older adults with cancer increased as time to death approached, and that more than half of patients received at least one medication which was considered potentially inappropriate during their last week of life. The absence of electronic prescribing records and retrospective nature of the study limited the applicability of some criteria. Interventions are needed to optimise medication prescribing and use in palliative care settings. References (1) Sharma M, Loh KP, Nightingale G, Mohile SG, Holmes HM. Polypharmacy and potentially inappropriate medication use in geriatric oncology. J Geriatr Oncol. 2016;7(5):346-53. (2) Cadogan CA, Murphy M, Boland M, Bennett K, McLean S, Hughes C. Prescribing practices, patterns, and potential harms in patients receiving palliative care: A systematic scoping review. Expl Res Clinic Soc Pharm. 2021;1(3):100050.

Patients Receiving Palliative Care and Their Experiences of Encounters With Healthcare Professionals.

The study aimed to explore experiences of encounters with health care professionals among patients receiving palliative cancer care in specialist palliative care inpatient units. A qualitative explorative study design was conducted in a specialist palliative care inpatient setting. Data collection was implemented using semi-structured individual interviews (20 palliative care cancer patients) and analysed with inductive content analysis. Palliative care patients experienced both meaningful and disrespectful encounters with healthcare professionals. The meaningful encounters encompassed authentic and supportive experiences, while the disrespectful encounters included indifferent and inadequate experiences. Caring for a patient receiving palliative care requires care beyond tending to a patient's physical needs. Patients should be encountered holistically and as equal human beings without highlighting their roles as patients. The healthcare professionals and the organisations should also acknowledge the importance of time and effort spent for encounters and conversations with the patients instead of concentrating resources mainly on physical care.

The Role of an Inpatient Hospice and Palliative Clinical Pharmacist in the Interdisciplinary Team.

Palliative care is a specialized health care service for individuals with serious illness at any stage and can be provided in any setting. Current national consensus developed by palliative care experts recommends the inclusion of pharmacists in an interdisciplinary team (IDT) to provide quality palliative care. However, national registry data report that less than 10% of inpatient palliative teams in the U.S. have a clinical pharmacist. Clinical pharmacists have an impactful role in palliative patients' quality of life by optimizing symptom management, deprescribing, and providing education to the palliative care team as well as patients and their families. In this report, we review the current literature on the role of a palliative pharmacist in an inpatient palliative care setting and compare and contrast this with our own clinical practice, providing case examples about the role of a palliative clinical pharmacist in an interdisciplinary inpatient palliative care setting. Future strategies are needed to increase post-graduate specialized pharmacy residency training in palliative care as well as education on palliative and hospice care in pharmacy schools to support the role of clinical pharmacists in palliative care.

Supportive care services (SCS) during curative-intent treatment with chemoradiation for patients with locally advanced head and neck cancer (HNC).

e18041 Background: SCS assist in managing symptoms and outcomes for patients in palliative care settings. But impact of services in curative intent settings is not well defined. Here we evaluate this for head and neck cancer patients. Methods: Following IRB approval, we retrospectively reviewed charts of patients with HNC treated from 2014-2017 at the University of Illinois Hospital. Of 260 patient charts reviewed, 67 patients fit the study criteria, which included completing treatment with curative intent at University of Illinois Hospitals, not living at a skilled nursing facility, and follow-up of at least 3 months. Demographic data and treatment course data were collected. SCS included opioid use, tracheostomy use, G-tube use, prophylaxis for mucositis, speech and swallow f/u, nutritionist visits, social worker visits, and anti-emetic prophylaxis. Outcomes included number of hydration visits, number of ER visits, and number of days in the hospital. We tested the significance of correlations between continuous variables using Pearson correlation and the significance of difference in a continuous variable between two groups using the Wilcoxon ranked sum test. We used Cox proportional hazards model for analyses of overall survival (OS). Analyses were univariable. Results: 20 patients had oropharyngeal cancer. 47 had non-oropharyngeal disease. 57 patients received cisplatin and 10 patients received cetuximab, concurrently with radiation therapy. Median weight loss was 6.0kg during treatment. Highest potency opioid at end of treatment was not associated with outcomes. Among the full cohort, having a G-tube was associated with increased number of ER visits (median 1.5 vs. 0.0, p < 0.01), hospitalizations (median 2.0 vs. 0.0, p < 0.01), and number of days in the hospital (median 7.0 vs. 0.0, p < 0.01). Increased hydration appointments, meanwhile, did not predict outcomes. In subgroup analysis of patients who received G-tubes, ER visits before or after G-tube placement did not predict OS (HR 0.64, p = 0.56 for ER visits before G-tube placement, HR 1.06, p = 0.75 for ER visits after G-tube placement). Number of hydration visits after starting treatment for cetuximab vs cisplatin was significant (median 8 for cisplatin and 2 for cetuximab, p = 0.02). Conclusions: G-tube placement, even after placement, was associated with medical care needs during treatment, suggesting an unmet need for this patient population. Further studies are needed to determine how to best provide support for this vulnerable patient population.

Providing a nurse-led complex nursing INtervention FOcused on quality of life assessment on advanced cancer patients: The INFO-QoL pilot trial

PURPOSE Unmet needs for advanced-disease cancer patients are fatigue, pain, and emotional support. Little information is available about the feasibility of interventions focused on patient-reported outcome measurement developed according to the Medical Research Council (MRC) Framework in advanced-disease cancer patients. We aimed to pilot a nurse-led complex intervention focused on QoL assessment in advanced-disease cancer patients. METHODS The INFO-QoL study was based on an exploratory, nonequivalent comparison group, pre-test-post-test design. Study sites received either the INFO-QoL intervention or usual care. Adult advanced-disease cancer patients admitted to hospice inpatient units that gave their informed consent were included in the study. Subjects were 187 patients and their families and 19 healthcare professionals. We evaluated feasibility, acceptability, and patients' outcomes using the Integrated Palliative Care Outcome Scale. RESULTS Nineteen healthcare professionals were included. The mean competence score increased significantly over time (p < 0.001) and the mean usefulness score was high 8.63 (±1.36). In the post-test phase, 54 patients were allocated to the experimental unit and 36 in the comparison unit. Compared to the comparison unit, in the experimental unit anxiety (R2 = 0.07; 95% CI = -0.06; 0.19), family anxiety (R2 = 0.22; 95% CI = -0.03; 0.41), depression (R2 = 0.31; 95% CI = -0.05; 0.56) and sharing feelings (R2 = 0.09; 95% CI = -0.05; 0.23), were improved between pre-test and post-test phase. CONCLUSIONS The INFO-QoL was feasible and potentially improved psychological outcomes. Despite the high attrition rate, the INFO-QoL improved the quality and safety culture for patients in palliative care settings.

Validation of the responding to urgency of need in palliative care (RUN-PC) triage tool

Background: The Responding to Urgency of Need in Palliative Care (RUN-PC) Triage Tool is a novel, evidence-based tool by which specialist palliative care services can manage waiting lists and workflow by prioritising access to care for those patients with the most pressing needs in an equitable, efficient and transparent manner. Aim: This study aimed to establish the intra- and inter-rater reliability, and convergent validity of the RUN-PC Triage Tool and generate recommended response times. Design: An online survey of palliative care intake officers applying the RUN-PC Triage Tool to a series of 49 real clinical vignettes was assessed against a reference standard: a postal survey of expert palliative care clinicians ranking the same vignettes in order of urgency. Setting/Participants: Intake officers (n = 28) with a minimum of 2 years palliative care experience and expert clinicians (n = 32) with a minimum of 10 years palliative care experience were recruited from inpatient, hospital consultation and community palliative care services across metropolitan and regional Victoria, Australia. Results: The RUN-PC Triage Tool has good intra- and inter-rater reliability in inpatient, hospital consultation and community palliative care settings (Intraclass Correlation Coefficients ranged from 0.61 to 0.74), and moderate to good correlation to expert opinion used as a reference standard (Kendall’s Tau rank correlation coefficients ranged from 0.68 to 0.83). Conclusion: The RUN-PC Triage Tool appears to be a reliable and valid tool for the prioritisation of patients referred to specialist inpatient, hospital consultation and community palliative care services.

Preferences on the Timing of Initiating Advance Care Planning and Withdrawing Life-Sustaining Treatment between Terminally-Ill Cancer Patients and Their Main Family Caregivers: A Prospective Study.

Background: The Western individualistic understanding of autonomy for advance care planning is considered not to reflect the Asian family-centered approach in medical decision-making. The study aim is to compare preferences on timing for advance care planning initiatives and life-sustaining treatment withdrawal between terminally-ill cancer patients and their family caregivers in Taiwan. Methods: A prospective study using questionnaire survey was conducted with both terminally-ill cancer patient and their family caregiver dyads independently in inpatient and outpatient palliative care settings in a tertiary hospital in Northern Taiwan. Self-reported questionnaire using clinical scenario of incurable lung cancer was employed. Descriptive analysis was used for data analysis. Results: Fifty-four patients and family dyads were recruited from 1 August 2019 to 15 January 2020. Nearly 80% of patients and caregivers agreed that advance care planning should be conducted when the patient was at a non-frail stage of disease. Patients’ frail stage of disease was considered the indicator for life-sustaining treatments withdrawal except for nutrition and fluid supplements, antibiotics or blood transfusions. Patient dyads considered that advance care planning discussions were meaningful without arousing emotional distress. Conclusion: Patient dyads’ preferences on the timing of initiating advance care planning and life-sustaining treatments withdrawal were found to be consistent. Taiwanese people’s medical decision-making is heavily influenced by cultural characteristics including relational autonomy and filial piety. The findings could inform the clinical practice and policy in the wider Asia–Pacific region.

"Standing Shoulder to Shoulder to Tell the Family What Was Really Going On": A Qualitative Study Exploring Palliative Care Clinicians' Perceptions of "Patient-Centered Family Meetings".

Background: Family meetings are often conducted in palliative care, but there is no universal agreed or accepted model. A new model of Patient-Centered Family Meetings is proposed whereby the patient sets the agenda. Aim: To seek palliative care clinicians' perceptions and experiences of Patient-Centered Family Meetings ("Meetings") and their acceptability and feasibility in the inpatient specialist palliative care setting. Design: A qualitative study used semistructured interviews. Theoretical and procedural direction was taken from grounded theory with thematic content analysis using the constant comparative method. Setting/Participants: Interviews were conducted with clinicians (n = 10) at the intervention site who had participated in a Meeting. Results: Four themes were identified: (1) a patient-set agenda gives patients a "voice"; (2) a patient-set agenda and the Meeting model enhances clinicians' understanding of patients and families; (3) the Meeting model was perceived to be acceptable; and (4) the Meeting model was perceived to be only feasible for selected patients. Conclusion: Clinicians perceived that a patient-set meeting agenda with defined questions enhanced their knowledge of the patient's issues and their understanding of the patient and their family's needs. The patients' most important issues often differed from the clinicians' expectations of what might be important to individual patients. There were contrasting views about the acceptability and feasibility of these Meetings as standard practice due to clinician time constraints and the Meeting not being required or relevant to all patients. Given the perceived benefits, the identification of patients and families who would most benefit is an important research priority.