scholarly journals Adapting preference-based utility measures to capture the impact of cancer treatment-related symptoms

2021 ◽  
Author(s):  
Koonal K. Shah ◽  
Bryan Bennett ◽  
Andrew Lenny ◽  
Louise Longworth ◽  
John E. Brazier ◽  
...  
Keyword(s):  
Cancer Treatment ◽  
De Novo ◽  
Patient Reported Outcome ◽  
Cancer Therapies ◽  
The Core ◽  
Transient Nature ◽  
Patient Reported ◽  
Value Sets ◽  
The Impact ◽  
Utility Measures

AbstractIt is important that patient-reported outcome (PRO) measures used to assess cancer therapies adequately capture the benefits and risks experienced by patients, particularly when adverse event profiles differ across therapies. This study explores the case for augmenting preference-based utility measures to capture the impact of cancer treatment-related symptoms. Additional cancer treatment-related items could be specific (e.g., rash) or global. While specific items are easier to describe and understand, their use may miss rarer symptoms and those that are currently unknown but will arise from future medical advancements. The appropriate number of additional items, the independence of those items, and their impact on the psychometric properties of the core instrument require consideration. Alternatively, a global item could encompass all potential treatment-related symptoms, of any treatments for any disease. However, such an item may not be well understood by general public respondents in valuation exercises. Further challenges include the decision about whether to generate de novo value sets for the modified instrument or to map to existing tariffs. The fluctuating and transient nature of treatment-related symptoms may be inconsistent with the methods used in conventional valuation exercises. Fluctuating symptoms could be missed by sub-optimal measure administration timing. The addition of items also poses double-counting risks. In summary, the addition of treatment-related symptom items could increase the sensitivity of existing utility measures to capture known and unknown treatment effects in oncology, while retaining the core domains. However, more research is needed to investigate the challenges, particularly regarding valuation.

scholarly journals Patient-Reported Outcome Measures Identify Impairment Better Than Clinician Documentation during Pediatric Cancer Treatment

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3291-3291 ◽  
Author(s):  
Sarah K. Dobrozsi ◽  
Julie A. Panepinto
Keyword(s):  
Depressive Symptoms ◽  
Standard Deviation ◽  
Cancer Treatment ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Impaired Function ◽  
Patient Reported ◽  
The Mean ◽  
The Impact ◽  
Clinic Visits

Introduction: Children receiving treatment for cancer commonly experience symptoms during their course that impact patient function. Understanding limitations to patient function caused by symptoms is critical to supporting children through their illness; however assessment of function is not standardized which can result in the under recognition of symptoms. Patient-reported outcome measures (PROs) can systematically measure and quantify the impact of symptoms on patient function. PROs have the potential to improve clinician recognition of symptoms and provide additional objective information about the impact of symptoms on patient function. However, to date there are minimal data using PROs in a clinical setting to detect symptoms and function in children receiving treatment for cancer. The objective of this study was to determine if symptoms causing significant impact on patient function as detected by PROs are recognized by standard clinician interview. We hypothesize that PROs identify symptoms causing significant burden to patients with greater sensitivity than clinician documentation. Methods: We conducted a prospective study of children age 8-21 years old that began treatment for cancer within the previous 4 weeks. Patients completed self-reported PROMIS questionnaires measuring impaired mobility, pain, fatigue, anxiety, and depressive symptoms monthly at outpatient clinic visits during the first 6 months of cancer treatment. PROMIS is a validated PRO that provides separate scores for each symptom measured with a mean score of 50 and standard deviation of 10. Patients with scores more than 1 standard deviation from the mean (T score ≥ 10 points from mean of 50) have significantly impaired function. Clinician documentation of symptoms at clinic visits were abstracted from the medical record in dichotomous fashion (present/absent) using pre-defined key words to define each symptom. The proportion of clinic visits with impaired function detected by PROMIS was compared to proportion of clinic visits with symptom documented by clinician using chi square tests. Results: Forty children participated in the study. The mean age of study participants was 11.7 years (SD 4.7). Fifty-eight percent of the patients were male and 60% of patients had leukemia or lymphoma. Paired PRO data/clinician documentation was available for 150 visits. The mean number of clinic visits per study patient was 3.75 (range 2-5). Patients demonstrated impaired function on PROMIS related to any symptom measured at 24% of clinic visits. For all clinic visits at which PROMIS detected impaired function, the symptom was documented by the clinician only 20% of the time (p<0.0001). Mobility: When impaired function was detected by PROMIS, concerns related to mobility were documented by the clinician only 20% of the time (p<0.0001) (figure 1). Pain: When impaired function due to pain was detected by PROMIS, pain was documented by the clinician 38% of the time (p<0.0001) (figure 1). Fatigue: When impaired function due to fatigue was detected by PROMIS, fatigue was documented by the clinician only 20% of the time (p<0.0001) (figure 1). Anxiety: When impaired function due to anxiety was detected by PROMIS, anxiety was documented by the clinician at 0% of the visits (p<0.0001) (figure 1). Depressive symptoms: When impaired function due to depressive symptoms were detected by PROMIS, depressive symptoms were documented by the clinician only 7% of the time (p<0.0001) (figure 1). Conclusion: Symptoms causing significant impact on patient function as detected by PROs are poorly detected and documented by standard clinician interview. These data demonstrate the natural limitations of clinician interview to illicit the true impact of symptoms on patients' lives and the likelihood that clinicians are unaware of the impact of disease and treatment on individuals and their function. The discrepancies in detection of this burden by current practice highlight opportunities to use PRO data to supplement clinical practice. Disclosures Panepinto: HRSA, NIH: Research Funding; NKT Therapeutics, Inc: Consultancy.

Variations in patient-reported outcome (PRO) collection and reporting in novel FDA approved anticancer therapies.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e18202-e18202
Author(s):  
Surbhi Singhal ◽  
Evan Thomas Hall ◽  
Brooke Peterson Gabster ◽  
James Dickerson ◽  
Lidia Schapira
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cancer Treatment ◽  
Treatment Options ◽  
Patient Reported Outcome ◽  
Cancer Therapies ◽  
Clinical Trial Registry ◽  
Fda Approval ◽  
Patient Reported ◽  
Anti Cancer

e18202 Background: Patient-reported outcomes (PROs) are increasingly valued as a key tool in patient-focused treatment decisions. However, a lack of standardization leads to significant variability in PRO collection and reporting in ground-breaking clinical trials of novel agents. We sought to characterize the mechanisms of assessment and variability by which PROs are reported for newly approved anti-cancer therapies. Methods: We reviewed the U.S. Food and Drug Administration (FDA) approvals between 2011 and 2017 for anti-cancer new molecular entities (NMEs) and new biologic approvals (BLAs). For each therapy, the pivotal clinical trial leading to FDA approval was identified using the national clinical trial (NCT) number and assessed for inclusion of PROs. A separate PubMed search was conducted to evaluate for PRO publication distinct from the original trial based on national clinical trial registry number. Results: From 2011 to 2017, the FDA approved 66 NMEs/BLAs based on 74 clinical trials for cancer treatment. Of the 74 clinical trial publications, 21 (28%) of the trials published PRO data in their original clinical publication, 18 (24%) published a separate PRO analysis, and 35 (47%) did not publish PRO data in either format. Among the 32 clinical trials (43%) that listed PROs as pre-specified outcomes, 72% published PROs (23/32). The separate PRO analyses (N = 18) were published considerably later following FDA approval (mean 605 days) than the original clinical trials (mean 20 days, N = 74, P < 0.001). Conclusions: As cancer treatment options expand, therapy decisions become increasingly nuanced. PROs assist decision-making by providing detailed information on important aspects of quality of life and tolerability. Our research has identified a significant lag in the publication of companion studies of PRO data associated with pivotal clinical trials, representing a meaningful gap in information critical to patients and oncologists in the process of making informed decisions.

Optimizing electronic capture of patient-reported outcome measures in oncology clinical trials: lessons learned from a qualitative study

2020 ◽  
Vol 9 (17) ◽  
pp. 1195-1204
Author(s):  
Florence D Mowlem ◽  
Brad Sanderson ◽  
Jill V Platko ◽  
Bill Byrom
Keyword(s):  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Lessons Learned ◽  
Structured Interviews ◽  
Patient Reported ◽  
Oncology Clinical Trials ◽  
Anticancer Treatment ◽  
Fit For Purpose ◽  
The Impact ◽  
Electronic Implementation

Aim: To understand the impact of anticancer treatment on oncology patients’ ability to use electronic solutions for completing patient-reported outcomes (ePRO). Materials & methods: Semi-structured interviews were conducted with seven individuals who had experienced a cancer diagnosis and treatment. Results: Participants reported that the following would impact the ability to interact with an ePRO solution: peripheral neuropathy of the hands (4/7), fatigue and/or concentration and memory issues (6/7), where they are in a treatment cycle (5/7). Approaches to improve usability included: larger, well-spaced buttons to deal with finger numbness, the ability to pause a survey and complete at a later point and presenting the recall period with every question to reduce reliance on memory. Conclusion: Symptoms associated with cancers and anticancer treatments can impact the use of technologies. The recommendations for optimizing the electronic implementation of patient-reported outcome instruments in this population provides the potential to improve data quality in oncology trials and places patient needs at the forefront to ensure ‘fit-for-purpose’ solutions.

scholarly journals Development of a patient-reported outcome measure for neck pain in military aircrew: qualitative interviews to inform design and content

BMJ Open ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. e039488
Author(s):  
Anna Dowling ◽  
Ellen Slungaard ◽  
Nicola R Heneghan
Keyword(s):  
Neck Pain ◽  
Outcome Measure ◽  
Physical Symptoms ◽  
Patient Reported Outcome ◽  
Direct Access ◽  
Specific Patient ◽  
Concept Elicitation ◽  
Patient Reported Outcome Measure ◽  
Patient Reported ◽  
The Impact

IntroductionThe prevalence of flight-related neck pain is 70% in UK fast jet pilots; much higher than the general population. The Aircrew Conditioning Programme and direct access physiotherapy exist to minimise the impact on military capability, but a population specific patient-reported outcome measure (PROM) is required to investigate the effectiveness of these. We aimed to explore the experiences of flight-related neck pain to inform the content validity and development of a population specific PROM.MethodsQualitative semistructured interviews combining phenomenological and grounded theory methods, reported using Consolidated criteria for Reporting Qualitative research guidelines. A purposive sample of 10 fast jet pilots with neck pain was recruited. Concept elicitation interviews were audio recorded, transcribed verbatim along with field notes. Data analysis involved subject and methodological expertise used a concept elicitation approach.ResultsParticipants included 10 male fast jet pilots, age 34.7 years. Identified themes included: (1) physical symptoms associated with flying activities; (2) occupational effects revealed modifications of flying, or ‘suboptimal’ performance owing to neck pain; (3) psychological effects revealed feelings or worry and (4) social and activity effects showed impact on out of work time.ConclusionPopulation-specific occupational, psychological and social factors should be considered alongside physical symptoms when managing neck pain in military aircrew. Findings support the development of a PROM specifically designed for military aircrew with neck pain.

Correlation between nasal mucosal temperature change and the perception of nasal patency: a literature review

2021 ◽  
Vol 135 (2) ◽  
pp. 104-109
Author(s):  
R Tjahjono ◽  
N Singh
Keyword(s):  
Temperature Change ◽  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Subjective Perception ◽  
Nasal Patency ◽  
Patient Reported Outcome Measure ◽  
Physical Measurements ◽  
Pubmed Database ◽  
Patient Reported ◽  
The Impact

AbstractBackgroundThe mechanism of nasal airflow sensation is poorly understood. This study aimed to examine the role of nasal mucosal temperature change in the subjective perception of nasal patency and the methods by which it can be quantified.MethodMedline and PubMed database searches were performed to retrieve literature relevant to the topic.ResultsThe primary mechanism producing the sensation of nasal patency is thought to be the activation of transient receptor potential melastatin family member 8 (‘TRPM8’), a thermoreceptor that is activated by nasal mucosal cooling. Computational fluid dynamics studies have demonstrated that increased airflow and heat flux are correlated with better patient-reported outcome measure scores. Similarly, physical measurements of the nasal cavity using temperature probes have shown a correlation between lower nasal mucosal temperatures and better patient-reported outcome measure scores.ConclusionNasal mucosal temperature change may be correlated with the perception of improved nasal patency. Future research should quantify the impact of mucosal cooling on the perception of nasal airway obstruction.

Nonunion Rates After Anterior Cervical Discectomy and Fusion: Comparison of Polyetheretherketone vs Structural Allograft Implants

Neurosurgery ◽  
2021 ◽  
Author(s):  
Won Hyung A Ryu ◽  
Dominick Richards ◽  
Mena G Kerolus ◽  
Adewale A Bakare ◽  
Ryan Khanna ◽  
...  
Keyword(s):  
Smoking Status ◽  
Posterior Instrumentation ◽  
Patient Reported Outcome ◽  
Anterior Cervical Discectomy ◽  
Successful Outcome ◽  
Cervical Discectomy ◽  
Structural Allograft ◽  
Modifiable Factors ◽  
Patient Reported ◽  
The Impact

Abstract BACKGROUND Although advances in implant materials, such as polyetheretherketone (PEEK), have been developed aimed to improve outcome after anterior cervical discectomy and fusion (ACDF), it is essential to confirm whether these changes translate into clinically important sustained benefits. OBJECTIVE To compare the radiographic and clinical outcomes of patients undergoing up to 3-level ACDF with PEEK vs structural allograft implants. METHODS In this cohort study, radiographic and symptomatic nonunion rates were compared in consecutive patients who underwent 1 to 3 level ACDF with allograft or PEEK implant. Prospectively collected clinical data and patient-reported outcome (PRO) scores were compared between the allograft and PEEK groups. Regression analysis was performed to determine the predictors of nonunion. RESULTS In total, 194 of 404 patients met the inclusion criteria (79% allograft vs 21% PEEK). Preoperative demographic variables were comparable between the 2 groups except for age. The rate of radiographic nonunion was higher with PEEK implants (39% vs 27%, P = .0035). However, a higher proportion of nonunion in the allograft cohort required posterior instrumentation (14% vs 3%, P = .039). Patients with multilevel procedures and PEEK implants had up to 5.8 times the risk of radiographic nonunion, whereas younger patients, active smokers, and multilevel procedures were at higher risk of symptomatic nonunion. CONCLUSION Along with implant material, factors such as younger age, active smoking status, and the number of operated levels were independent predictors of fusion failure. Given the impact of nonunion on PRO, perioperative optimization of modifiable factors and surgical planning are essential to ensure a successful outcome.

scholarly journals Digital patient-reported outcome measures (PROMs) for remote monitoring of patients on cancer treatment: a cross-sectional questionnaire feasibility study (Preprint)

2020 ◽  
Author(s):  
Mayuran Ananth Sivanandan ◽  
Catherine Sharma ◽  
Pippa Bullard ◽  
Judith Christian
Keyword(s):  
Cancer Treatment ◽  
Outcome Measures ◽  
Remote Monitoring ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Severe Toxicity ◽  
Cross Sectional ◽  
Treatment Pathways ◽  
Face To Face ◽  
Patient Reported

BACKGROUND Oncology has increasing outpatient activity related to increased cancer incidence, better survival rates and more treatments. Innovative technological solutions could help deal with this increasing demand and digital patient-reported outcome measures (PROMs) to identify those patients that need a face-to-face (FTF) appointment is one potential approach. OBJECTIVE Our study aimed to assess the feasibility of digital PROM questionnaires to enable remote symptom monitoring for patients on cancer treatment and their ability to determine the requirement for a FTF appointment. METHODS This study was performed at a tertiary oncology centre between December 2018 and February 2019. Target clinics covered both systemic therapy and radiotherapy cohorts. The Common Terminology Criteria for Adverse Events (CTCAE) helped form the basis for acute toxicity questionnaires which were adapted into patient-friendly language. Treatment-specific digital PROM questionnaires were answered by patients and their clinicians alongside face-to-face appointments. Patients and clinicians did not see each other’s results, which were not used for clinical decisions. Agreement between patients and clinicians was assessed through descriptive statistics. Patient and staff feedback was also obtained. RESULTS 90 patients took part in the study across 10 different treatment pathways. By comparing paired patient and clinician responses, the sensitivity of the patient-completed questionnaires in correctly determining the need for FTF review was 93.6% and no patients with severe toxicity would have been missed with the questionnaires. Digital PROMs revealed 28.9% of participating patients did not need FTF review based on their symptoms. Certain oncological treatment pathways, such as immunotherapy, were found to have a larger proportion of patients with minimal symptoms compared to others, such as conventional chemotherapy. Patient and staff feedback showed high approval with digital PROMs and their potential for use in remote monitoring. CONCLUSIONS Digital PROM questionnaires can feasibly determine the need for FTF review in ‘on treatment’ oncology clinics. Their use with specific treatments could safely reduce the requirement for FTF care and future work should evaluate their application in the remote monitoring of patients.

Do patient and proxy agree? Long-term changes in multiple sclerosis physical impact and walking ability on patient-reported outcome scales

2014 ◽  
Vol 20 (12) ◽  
pp. 1616-1623 ◽  
Author(s):  
Judith M Sonder ◽  
Lisanne J Balk ◽  
Libertje VAE Bosma ◽  
Chris H Polman ◽  
Bernard MJ Uitdehaag
Keyword(s):  
Multiple Sclerosis ◽  
Patient Reported Outcome ◽  
Study Data ◽  
Walking Ability ◽  
Impact Scale ◽  
Patient Reported ◽  
Proxy Responses ◽  
Physical Impact ◽  
The Impact

Background: Patient-reported outcome scales (PROs) are useful in monitoring changes in multiple sclerosis (MS) over time. Although these scales are reliable and valid measures in longitudinal studies in MS patients, it is unknown what the impact is when obtaining longitudinal data from proxies. Objective: The objective of this paper is to compare longitudinal changes in patient and proxy responses on PROs assessing physical impact of MS and walking ability. Methods: In a prospective observational study, data on the Multiple Sclerosis Impact Scale (MSIS-29 physical) and Multiple Sclerosis Walking Scale (MSWS-12) were obtained from 137 patient-proxy couples at baseline and at two-year follow-up. Demographic and disease-related variables explaining agreement or disagreement between patients and proxies were investigated using linear regression analyses. Results: Full agreement was found in 56% (MSIS) and 62% (MSWS) of the patient-proxy couples. Complete disagreement was very rare for both scales (2% MSIS, 5% MSWS). When patients were more positive than proxies, a higher age, longer disease duration, longer patient-proxy relationship and increased levels of depression, anxiety and caregiver burden in proxies were observed. Conclusion: In the majority of the patient-proxy couples there was agreement. Proxies can serve as a valuable source of information, but caution remains essential when using scores from proxies.

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