Comparing the use of patient-reported outcomes in clinical studies in Europe in 2008 and 2018: a literature review.

PURPOSE: Several guidelines for the use of patient-reported outcomes (PROs) in clinical studies have been published in the past decade. This review primarily aimed to compare the number and compliance with selected PRO-specific criteria for reporting of clinical studies in Europe using PROs published in 2008 and 2018. Secondarily, to describe the study designs, PRO instruments used, patient groups studied, and countries where the clinical studies were conducted. METHODS: A literature search was conducted in MEDLINE to identify eligible publications. To assess the number of publications, all abstracts were screened for eligibility by pairs of reviewers. Compliance with PRO-specific criteria and other key characteristics was assessed in a random sample of 150 eligible full-text publications from each year. Randomized controlled trials (RCTs) were assessed according to the full CONSORT-PRO checklist. RESULTS: The search identified 1692 publications in 2008 and 4290 in 2018. After screening of abstracts, 1240 from 2008 and 2869 from 2018 were clinical studies using PROs. By full-text review, the proportion of studies discussing PRO-specific limitations and implications was higher in 2018 than in 2008, but there were no differences in the other selected PRO-specific criteria. In 2018, a higher proportion of studies were longitudinal/cohort studies, included ≥ 300 patients, and used electronic administration of PRO than in 2008. The most common patient groups studied were those with cancer or diseases of the musculoskeletal system or connective tissue. CONCLUSION: The number of clinical studies from Europe using PROs was higher in 2018 than in 2008, but there was little difference in compliance with the PRO-specific criteria. The studies varied in terms of study design and PRO instruments used in both publication years.

Cancer patients' diagnosis and symptoms and their family caregivers' self-efficacy and social support are associated with different caregiver reactions.

OBJECTIVE: To describe caregiver and patient characteristics that are associated with negative and positive reactions in family caregivers (FCs) of cancer outpatients. METHODS: A total of 194 FCs completed the Caregiver Reaction Assessment (CRA) scale 6 months after start of new treatment in patients with breast, ovarian, colorectal, or head and neck cancer. Linear regression models were used to examine which caregiver characteristics (i.e. demographic, self-efficacy and social support) and patient characteristics (i.e. clinical, symptoms) were associated with each of the CRA subscales (caregiver esteem, lack of family support, and impact on health, schedule and finances). RESULTS: Less social support was significantly associated with poorer scores on all subscales (B -0.01/0.01). Also, poorer scores on one or more of the CRA subscales were reported by FCs who had lower self-efficacy (B -0.02), a higher level of education (primary B 0.42, secondary B 0.22), more medical conditions (B 0.06), and were female (B 0.20), and by FCs of patients with colorectal (B 0.45) or head and neck cancer (B 0.27), and those who reported a higher symptom burden (B 0.28/0.49). CONCLUSION: Both caregiver and patient factors were associated with reactions in FCs of cancer outpatients. This information can be used by healthcare personnel to identify FCs who need additional support (e.g. counselling), and to increase focus on strengths and assets within the caregivers (e.g. support groups).

Patient factors and quality of life outcomes differ among four subgroups of oncology patients based on symptom occurrence.

CONTEXT: Reviews of the literature on symptoms in oncology patients undergoing curative treatment, as well as patients receiving palliative care, suggest that they experience multiple, co-occurring symptoms and side effects. OBJECTIVES: The purposes of this study were to determine if subgroups of oncology patients could be identified based on symptom occurrence rates and if these subgroups differed on a number of demographic and clinical characteristics, as well as on quality of life (QoL) outcomes. METHODS: Latent class analysis (LCA) was used to identify subgroups (i.e. latent classes) of patients with distinct symptom experiences based on the occurrence rates for the 13 most common symptoms from the Memorial Symptom Assessment Scale. RESULTS: In total, 534 patients with breast, head and neck, colorectal, or ovarian cancer participated. Four latent classes of patients were identified based on probability of symptom occurrence: all low class [i.e. low probability for all symptoms (n = 152)], all high class (n = 149), high psychological class (n = 121), and low psychological class (n = 112). Patients in the all high class were significantly younger compared with patients in the all low class. Furthermore, compared to the other three classes, patients in the all high class had lower functional status and higher comorbidity scores, and reported poorer QoL scores. Patients in the high and low psychological classes had a moderate probability of reporting physical symptoms. Patients in the low psychological class reported a higher number of symptoms, a lower functional status, and poorer physical and total QoL scores. CONCLUSION: Distinct subgroups of oncology patients can be identified based on symptom occurrence rates. Patient characteristics that are associated with these subgroups can be used to identify patients who are at greater risk for multiple co-occurring symptoms and diminished QoL, so that these patients can be offered appropriate symptom management interventions.

Radiation-induced long-term dysphagia in survivors of head and neck cancer and association with dose-volume parameters.

BACKGROUND: Although dysphagia is a common side effect after radiotherapy (RT) of head and neck cancer (HNC), data on long-term dysphagia is scarce. We aimed to 1) compare radiation dose parameters in HNC survivors with and without dysphagia, 2) investigate factors associated with long-term dysphagia and its possible impact on health-related quality of life (HRQoL), and 3) investigate how our data agree with existing NTCP models. METHODS: This cross-sectional study conducted in 2018-2020, included HNC survivors treated in 2007-2013. Participants attended a one-day examination in hospital and filled in patient questionnaires. Dysphagia was measured with the EORTC QLQ-H&N35 swallowing scale. Toxicity was scored with CTCAE v.4. We contoured swallowing organs at risk (SWOAR) on RT plans, calculated dose-volume histograms (DVHs), performed logistic regression analyses and tested our data in established NTCP models. RESULTS: Of the 239 participants, 75 (31%) reported dysphagia. Compared to survivors without dysphagia, this group had reduced HRQoL and the DVHs for infrahyoid SWOAR were significantly shifted to the right. Long-term dysphagia was associated with age (OR 1.07, 95% CI 1.03-1.10), female sex (OR 2.75, 95% CI 1.45-5.21), and mean dose to middle pharyngeal constrictor muscle (MD-MPCM) (OR 1.06, 95% CI 1.03-1.09). NTCP models overall underestimated the risk of long-term dysphagia. CONCLUSIONS: Long-term dysphagia was associated with higher age, being female, and high MD-MPCM. Doses to distally located SWOAR seemed to be risk factors. Existing NTCP models do not sufficiently predict long-term dysphagia. Further efforts are needed to reduce the prevalence and consequences of this late effect.

Chronic fatigue in long-term survivors of head and neck cancer treated with radiotherapy.

BACKGROUND: There is lack of evidence on chronic fatigue (CF) following radiotherapy (RT) in survivors of head and neck cancer (HNC). We aimed to compare CF in HNC survivors > 5 years post-RT with a reference population and investigate factors associated with CF and the possible impact of CF on health-related quality of life (HRQoL). MATERIAL AND METHODS: In this cross-sectional study we included HNC survivors treated in 2007-2013. Participants filled in patient-reported outcome measures and attended a one-day examination. CF was measured with the Fatigue Questionnaire and compared with a matched reference population using t-tests and Cohen's effect size. Associations between CF, clinical and RT-related factors were investigated using logistic regression. HRQoL was measured with the EORTC Quality of Life core questionnaire. RESULTS: The median age of the 227 HNC survivors was 65 years and median time to follow-up was 8.5 years post-RT. CF was twice more prevalent in HNC survivors compared to a reference population. In multivariable analyses, female sex (OR 3.39, 95 % CI 1.82-6.31), comorbidity (OR 2.17, 95 % CI 1.20-3.94) and treatment with intensity-modulated RT (OR 2.13, 95 % CI 1.16-3.91) were associated with CF, while RT dose parameters were not. Survivors with CF compared to those without, had significantly worse HRQoL. CONCLUSIONS: CF in HNC survivors is particularly important for female patients, while specific factors associated with RT appear not to play a role. The high CF prevalence in long-term HNC survivors associated with impaired HRQoL is important information beneficial for clinicians and patients to improve patient follow-up.

High symptom burden is associated with impaired quality of life in colorectal cancer patients during chemotherapy:A prospective longitudinal study.

PURPOSE: Multiple symptoms can have a negative impact on quality of life (QoL), but there is little information about the impact of multiple symptoms on QoL of patients with colorectal cancer (CRC) during outpatient chemotherapy. Therefore, the purpose was to assess the physical and mental QoL in CRC patients over six months of chemotherapy, to evaluate the association of QoL with the presence of multiple symptoms, and to determine which demographic and clinical characteristics are associated with physical and mental QoL scores. METHODS: Outpatients with CRC (N = 120) completed the Medical Outcomes Study Short Form (SF-12) and Memorial Symptom Assessment Scale (MSAS) at eight time points during six months of chemotherapy. Linear mixed models for repeated measures were used to analyse QoL over time; and its association with demographic and clinical characteristics; and with the presence of multiple symptoms (e.g., 'numbness/tingling' and 'problems with sexual interest'). RESULTS: The CRC patients had worse physical and mental QoL scores than the general population at all time points. Impaired physical QoL was significantly associated with psychological symptom burden (p < 0.001) and numbness/tingling (p < 0.027). Impaired mental QoL was associated with physical symptom burden (p < 0.001), with being female (p < 0.009), younger age (p < 0.024), and having problems with sexual interest (p < 0.009). CONCLUSIONS: Impaired QoL was associated with symptoms in CRC outpatients. This information about the symptoms and characteristics associated with worse QoL during chemotherapy may help clinicians identify and inform at-risk patients.

Symptom burden and patient characteristics: Association with quality of life in patients with head and neck cancer undergoing radiotherapy.

BACKGROUND: Patients with head and neck cancer (HNC) experience diminished quality of life (QOL) during and after treatment. This study examined which characteristics were associated with QOL over time. METHODS: One hundred thirty-three patients with HNC from a study of 534 oncology patients rated physical and mental QOL using the Short Form-12 5 times from the initiation of radiotherapy (RT) through the following 6 months. Linear mixed model analyses examined changes over time and associated characteristics. RESULTS: The QOL deteriorated during RT and gradually improved after completion. Less social support was negatively associated with both physical and mental QOL. Older age, more comorbidities, more psychological symptoms, and concomitant chemotherapy (CTX) were negatively associated with physical QOL. Male sex, less physical symptoms, surgery before RT, and concomitant chemotherapy were positively associated with mental QOL. CONCLUSION: Clinicians can use knowledge on time course and associated characteristics to identify and inform patients at higher risk for diminished QOL.

A Longitudinal Study of Depressive Symptoms in Patients With Head and Neck Cancer Undergoing Radiotherapy.

BACKGROUND: Although patients with head and neck cancer are at increased risk for depressive symptoms compared with other cancer patients, few longitudinal studies have evaluated changes in and predictors of this symptom over time. OBJECTIVE: The aim of this study was to determine whether levels of depressive symptoms changed over time and whether specific demographic, clinical, symptom, or psychosocial characteristics were associated with depressive symptoms. METHODS: In a longitudinal study of patients with head and neck cancer, depressive symptoms were assessed with the Center for Epidemiologic Studies-Depression scale, from the initiation of radiotherapy and for 6 months after. Hierarchical linear modeling was used to evaluate for changes in as well as for potential predictors of interindividual differences in depressive symptoms. RESULTS: The severity of depressive symptoms increased during radiotherapy and then decreased over time. The portion of patients who reported clinically meaningful levels of depressive symptoms at each assessment ranged from 29% to 42%. Several known predictors of pretreatment severity of depressive symptoms (ie, physical symptoms, less social support, dissatisfaction with looks) were corroborated. In addition, having surgery before radiotherapy was associated with lower levels of depressive symptoms at initiation of radiotherapy. CONCLUSION: A moderate proportion of patients with head and neck cancer reported levels of depressive symptoms that indicated the need for clinical evaluation. Several patient characteristics were associated with depressive symptoms. IMPLICATIONS FOR PRACTICE: Knowledge on prevalence, time course, and predictors of depressive symptoms from this study can be used to identify patients at higher risk for more severe depressive symptoms.

Changes in and predictors of pain characteristics in patients with head and neck cancer undergoing radiotherapy.

Pain is a common symptom in patients with head and neck cancer (HNC) that is associated with significant decrements in physical and psychological functioning. Only 4 studies have evaluated for changes in and predictors of different pain characteristics in these patients. In this longitudinal study of patients with HNC, changes in pain intensity (i.e., average pain, worst pain), pain interference with function, and pain relief were evaluated from the initiation of radiotherapy and through the following 6 months. Hierarchical linear modeling was used to evaluate for changes over time in these 4 pain characteristics, as well as to identify predictors of interindividual variability in each characteristic. Overall, pain intensity and interference with function scores were in the mild-to-moderate range, while pain relief scores were in the moderate range. The occurrence of pain, as well as scores for each pain characteristic, increased from the initiation to the completion of radiotherapy, followed by a gradual decrease to near pretreatment levels at 6 months. However, interindividual variability existed in patients' ratings of each pain characteristic. Predictors of more severe pain characteristic scores were more comorbidities, worse physical functioning, not having surgery before radiotherapy, difficulty swallowing, mouth sores, sleep disturbance, fatigue, more energy, and less social support. Patients with more depressive symptoms had better pain relief. Although some of the predictors cannot be modified (e.g., rrence of surgery), other predictors (e.g., symptoms) can be treated. Therefore, information about these predictors may result in decreased pain in patients with HNC.