Development of a Simple Patient-reported Outcome Measurement for Terminally Ill Cancer Patients Receiving Home-based Palliative Care

INTRODUCTION

Globally, the need for palliative care services is over 40 million individuals.^[1] The Ministry of Public Health of Thailand has been promoting palliative care since 2017^[2] with a focus on end-stage cancer patients. The national service plan aims to improve quality of life by encouraging home-based care, supported by primary care providers, rather than hospitalisation.^[3,4] However, there is a lack of an easy-to-use patient assessment tool for home-based care. Existing tools were mostly for clinical research and hospital-based applications; for example, ‘The Palliative Care Outcome Scale (POS)’^[5] and ‘The Edmonton Symptom Assessment System,’^[6] are primarily developed for clinical research and hospital-based applications.^[7,8] Patients might have trouble understanding the questions and need help in responding to them.^[9] Our communication with some Thai palliative care providers in hospitals and community settings found that POS was too complicated and had too many questions, while patients might give biased responses to the ESAS visual analogue scale, recalling their prior responses. The purpose of the study is therefore create a simple, valid, reliable and sensitive-to-change patient-reported outcome measurement for those who suffer from terminal cancer and are receiving palliative care.

MATERIALS AND METHODS

We applied three-step approaches mixed with qualitative and quantitative studies, running from February 2019 to February 2022.

Step 1: Item generation, using purposive review literature and focus groups.

Beginning in February 2019, we reviewed some key published literature based on the following selection criteria: (1) focused on cancer patients, (2) quality of life in palliative care and (3) published in Thai or English. Twenty studies were identified. They were then filtered by the exclusion criteria, including (1) not focusing on the terminal stage and (2) studying only specific areas of quality of life. Finally, twelve papers were selected.

Concurrently, we conducted two focus groups with (1) palliative medical experts and (2) highly experienced palliative care nurses. The expert group included ten palliative medicine specialists and family physicians from five leading medical schools and tertiary-care cancer centres in Thailand who had extensive experience in providing palliative care. They were asked to share ideas and experiences on the outcome of care for terminally ill patients, requirements for assessing end-stage cancer care outcomes, symptoms needing special care at home and assessment methods for advanced cancer patients receiving palliative care at home. The nurse group comprised ten nurses from a cancer hospital and general and community hospitals who had deep experience in the area of palliative care in hospitals, in-patient homes or community settings. The discussion focused on special care needs for end-of-life care at home. The nurses were also required to ask questions in a group of experts. One of the researchers acted as the facilitator, while the others joined as observers. The main ideas were captured to design the drafted version of patient-reported outcome measurement for terminally ill cancer patients (PROMs-TCP).

Step 2 Pilot-testing of the drafted PROMs-TCP for content validity and ease of use.

From October to December 2019, the drafted PROMs-TCP, originally with six questions, was tested for content validity. A panel of five experts, including the head of the palliative care centre of a university-affiliated medical centre, a family physician with a certificate in clinical fellowship (palliative care), a family physician with long-time experiences in providing palliative care at home and two palliative care nurses with hospital-based and home-based experiences, independently rated each question item using a 4-point rating scale (1 = not relevant, 2 = somewhat relevant, 3 = quite relevant and 4 = highly relevant).

The drafted PROMs-TCP was also given to 20 selected advanced cancer patients receiving palliative care in four tertiary-care and community hospitals – 12 in hospital settings and eight in home-based settings – for their feedback on relevance, ease of use, length, understandability of the items and response choices and other suggestions, if any. Collected feedback was reviewed and applied to make modifications to the question items, response choices and presentation format.

Step 3 Evaluation of the instrument properties of PROMsTCP, including validity, reliability and sensitivity to change. From January 2020 to March 2021, 169 pairs of advanced cancer patients and their caregivers were voluntarily recruited from palliative care centres of six participating tertiary-care hospitals – two in Bangkok, one in the north, one in the south and two in the central region. The inclusion criteria included (1) having been diagnosed with end-stage cancer; (2) receiving palliative care; (3) the patients and their caregivers both agreeing to participate; (4) being aged between 18 and 70 years; and (5) being able to communicate in Thai language. The number was slightly more than the calculated sample size for correlation based on α = 0.05, power = 0.9 and correlation coefficient rate of 0.25.^[10] All participating hospitals assigned palliative care nurses to be project coordinators. They received training from the research team on the protocols, consent procedures and application of POS and PROMs-TCP.

After the recruitment and consent process, demographic data of all patients were collected on day 0 (baseline). The nurses assessed each patient with POS. Each patient responded to a question on overall well-being (OWB) to indicate whether OWB on that day was ‘good’ or ‘terrible.’ A good day meant that the patient felt satisfied with their health status, given the underlying health conditions. By contrast, a terrible day implied that the patient needed immediate intervention to improve their condition or to alleviate the individual’s suffering. Finally, the patient, their caregiver and the nurse independently used PROMs-TCP to assess the patient’s status. After that, assessments using the OWB and PROMsTCP measurements were repeated daily, while the POS was repeated on day 5 and day 10. Each patient was monitored for 10 days unless the patient passed away during that period, regardless of whether the patient was hospitalised or continued to receive care at home.

RESULTS

Discriminant validity

The ROC analysis revealed that AUC was 0.91 (95% CI: 0.90, 0.93). The cut-off value that demonstrated the maximum accuracy for distinguishing patients’ ‘good day’ and ‘terrible day’ was four. With this cut-off value, PROMs-TCP correctly identified of the patients with good days of 76.91% and 88.67% of the patients with terrible days, respectively [Figure 1].

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Figure 1:

Different cut-off points for the PROMs-TCP (n = 1,709). CI: Confidence interval, ROC: Receiver operating characteristic, AUC: Areas under the curve, PPV: Positive predictive value, NPV: Negative predictive value. PROMs-TCP: Patient-reported outcome measurement for terminally ill cancer patients. The bold values in the cut points show how sensitive and specificity of the data.

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Sensitivity to change

Spearman’s rank correlation analysis of score changes for each period between day 0 and day 5 and between day 0 and day 10 of PROMs-TCP and POS revealed a moderately favourable association [r = 0.5–0.7, Figure 2]. The PROMsTCP and POS score variations over 10 days were small, however. The ES values were 0.31 and 0.30 for day 0–day 5 and 0.36 and 0.33 for day 0–day 10 for PROMs-TCP and POS, respectively.

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Figure 2:

The change score between PROMs-TCP and palliative care outcome scale assessment (n = 132). PROMs-TCP: Patient-reported outcome measurement for terminally ill cancer patients.

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DISCUSSION

The purpose of this study is to develop a patient-reported outcome measurement tool (PROMs-TCP) for assessing the well-being of terminally ill cancer patients who received palliative care at home. The ultimate purposes were to be able to differentiate between a ‘good day’ and a ‘terrible day’ and for the professional palliative care team to be alerted if the home-based patients needed urgent attention or care. The study findings demonstrate that PROMs-TCP was reasonably valid, reliable and sensitive to change.

PROMs-TCP covers the major determinants of well-being of terminally ill cancer patients receiving palliative care at home to indicate their urgent needs for our team attention. Although it was not designed to be a comprehensive tool or to capture broad areas of quality of life of palliative-care patients, the criterion-validity test demonstrated the PROMs-TCP scores correlated well to POS. PROMs-TCP addresses some items of POS, namely ‘pain’ and ‘feelings of hopelessness’^[5] and some items in ESAS including ‘tiredness,’ ‘shortness of breath,’ ‘drowsiness’ and ‘feeling sad.’^[33] Compared to POS, PROMs-TCP asks about feeling depressed and sleeping, instead of feeling anxious or worried about the illness or treatment and sharing feelings with family or friends. Other POS questions, such as feeling that time was wasted on health-care appointments or practical matters resulting from illnesses, seemed irrelevant to our aims. Moreover, using PROMs-TCP to determine ‘good day’ or ‘terrible day’ statuses corresponded well with the patients’ perception of their own statuses.

PROMs-TCP was satisfactorily reliable – considering the possibility or likelihood that patients might not always be able to use the tool by themselves and would need others, such as their personal caregivers, to perform the assessment. Inter-rater agreements between those of the patients and those of the caregivers, as well as between those of the patients and the nurses, were at a moderate-to-good level (Cohen’s weighted kappa = 0.69–0.87), whereas the POS achieved values of 0.31–0.35.^[5,34]

Comparing to the other assessment tools available in Thai such as POS (11 items), ESAS (10 items), Functional Assessment of cancer therapy-General scale (27 items)^[35] or the tool from The European Organization for Research and Treatment of Cancer (30 items),^[36] our six-question PROMsTCPs were more concise and user-friendly, making it better fit for home-based care. Most of the patients and caregivers were able to complete the assessment in <1 min although it took up to 2 min in some cases with reading difficulties. Moreover, with the cut-off point to differentiate the patients with terrible days from those with good days, PROMs-TCP allows the palliative care teams to focus their attentions to the patients who need more and urgent assistance from them. This enables allocation of limited palliative and primary care resources more effectively.

Given the promising findings, we recognise some limitations. First, asking patients directly to indicate a good day or a terrible day may not be a perfect choice for discriminant validity testing. However, the use of this overall self- indicating assessment was aligned with the purpose of the tool to be patient centric in identifying a home-based patient who needed assistance from professional palliative care teams with validity and reliability. It is also worth noting that the approach of using a single overall question for assessing quality of life has been used in many studies and found to correlate with multi-question tools.^[37-39] Second, the sensitivity to change of our tool was tested against POS based on specific timing, not against changes in patients’ clinical and health symptoms at exact moments of increase or relief of suffering. We encourage a study that addresses this issue in more detail. Finally, our study was conducted in a limited number of palliative cares patients and was limited to health-care settings in Thailand. This might impact some generalisability of PROMs-TCP. Further studies in different settings, or even different countries, are needed, it is crucial to develop electronic patient-reported outcome measures. These measures enable researchers and health-care professionals to collect valuable data on patients’ health outcomes and experiences in a more efficient and standardised manner.^[40]

CONCLUSION

PROMs-TCP was proven to be reasonably valid, reliable, sensitive to change and simple to use, making it suitable for supporting home-based palliative care by primary care or palliative care providers. It enables effective communication between care teams and their patients and families and allows for appropriate responses to the patients’ needs for medical attention as well as efficient allocation of resources.