Patient-Reported Outcome Measures for Post-mastectomy Breast Reconstruction: A Systematic Review of Development and Measurement Properties.

BACKGROUND: Breast reconstruction (BR) is performed to improve outcomes for patients undergoing mastectomy. A recently developed core outcome set for BR includes six patient-reported outcomes that should be measured and reported in all future studies. It is vital that any instrument used to measure these outcomes as part of a core measurement set be robustly developed and validated so data are reliable and accurate. The aim of this systematic review is to evaluate the development and measurement properties of existing BR patient-reported outcome measures (PROMs) to inform instrument selection for future studies. METHODS: A PRISMA-compliant systematic review of development and validation studies of BR PROMs was conducted to assess their measurement properties. PROMs with adequate content validity were assessed using three steps: (1) the methodological quality of each identified study was assessed using the COSMIN Risk of Bias checklist; (2) criteria were applied for assessing good measurement properties; and (3) evidence was summarized and the quality of evidence assessed using a modified GRADE approach. RESULTS: Fourteen articles reported the development and measurement properties of six PROMs. Of these, only three (BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23) were considered to have adequate content validity and proceeded to full evaluation. This showed that all three PROMs had been robustly developed and validated and demonstrated adequate quality. CONCLUSIONS: BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23 have been well-developed and demonstrate adequate measurement properties. Work with key stakeholders is now needed to generate consensus regarding which PROM should be recommended for inclusion in a core measurement set.

Outcome selection, measurement and reporting for new surgical procedures and devices: a systematic review of IDEAL/IDEAL-D studies to inform development of a core outcome set.

BACKGROUND: Outcome selection, measurement and reporting for the evaluation of new surgical procedures and devices is inconsistent and lacks standardization. A core outcome set may promote the safe and transparent evaluation of surgical innovations. This systematic review examined outcome selection, measurement and reporting in studies conducted within the IDEAL (Idea, Development, Exploration, Assessment and Long-term monitoring) framework to examine current practice and inform the development of a core outcome set for early-phase studies of surgical procedures/devices. METHODS: Web of Science and Scopus citation searches were performed to identify author-reported IDEAL/IDEAL-D studies for any surgical procedure/device. Outcomes were extracted verbatim, including contextual information regarding outcome selection and measurement. Outcomes were categorized to inform a conceptual framework of outcome domains relevant to evaluating innovation. RESULTS: Some 48 studies were identified. Outcome selection, measurement and reporting varied widely across studies in different IDEAL stages. From 1737 outcomes extracted, 22 domains specific to evaluating innovation were conceptualized under seven broad categories: procedure completion success/failure; modifications; unanticipated events; surgeons' experiences; patients' experiences; resource use specific to the innovative procedure/device; and other innovation-specific outcomes. Most innovation-specific outcomes were measured and reported in only a small number of studies. CONCLUSION: This review highlighted the need for guidance and standardization in outcome selection and reporting in the evaluation of new surgical procedures/devices. Novel outcome domains specific to innovation have been identified to establish a core outcome set for future evaluations of surgical innovations.

ANTECEDENTES: La selección de resultados, mediciones y redacción de los informes para la evaluación de nuevos procedimientos y dispositivos quirúrgicos es inconsistente y carece de estandarización. Determinar un conjunto de resultados básicos (core outcome set, COS) podría contribuir a la transparencia y seguridad de las evaluaciones de las innovaciones quirúrgicas. Esta revisión sistemática analizó la selección de resultados, medición de los mismos e informes de estudios efectuados en el marco metodológico IDEAL, a fin de valorar la práctica actual y presentar el desarrollo de un COS para estudios en fase inicial de procedimientos/dispositivos quirúrgicos. MÉTODOS: Se realizaron búsquedas en las bases de datos Web of Science y Scopus para identificar estudios efectuados por los autores en el marco IDEAL/IDEAL-D referentes a cualquier procedimiento/dispositivo quirúrgico. Los resultados se extrajeron literalmente, incluida la información contextualizada referente a la selección y medición de los resultados. Los resultados se categorizaron para presentar información en un marco conceptual de dominios de resultados que fueran relevantes para poder evaluar la innovación. RESULTADOS: Se identificaron 48 estudios. Los estudios mostraban una amplia variedad en la selección de resultados, mediciones e información correspondientes a diferentes estadios IDEAL. A partir de 1.737 resultados extraídos, se conceptualizaron 22 dominios específicos para evaluar la innovación agrupados en 7 amplias categorías: éxito/fracaso para completar el procedimiento; modificaciones; eventos imprevistos; experiencias de los cirujanos; experiencias de los pacientes; uso de recursos específicos del procedimiento/dispositivo innovador y otros resultados específicos de la innovación. La mayoría de los resultados específicos de la innovación se midieron y notificaron solo en un pequeño número de estudios. CONCLUSIÓN: Esta revisión ha puesto de manifiesto la necesidad de orientación y estandarización en la selección de resultados y la notificación en la evaluación de nuevos procedimientos/dispositivos quirúrgicos. Se han identificado nuevos dominios de resultados específicos de innovación para establecer un COS para futuras evaluaciones de innovaciones quirúrgicas.

A systematic review and in-depth analysis of outcome reporting in early phase studies of colorectal cancer surgical innovation.

AIM: Early phase studies are essential to evaluate new technologies prior to randomized evaluation. Evaluation is limited, however, by inconsistent measurement and reporting of outcomes. This study examines outcome reporting in studies of innovative colorectal cancer surgery. METHODS: Systematic searches identified studies of invasive procedures treating primary colorectal adenocarcinoma. Included were a random sample of studies which authors reported as 'new' or 'modified'. Outcomes were extracted verbatim and categorized using an existing framework of 32 domains relevant to early phase studies. Outcomes were classified as 'measured' (where there was an explicit statement to that effect or evidence that data collection had occurred) or 'mentioned but not measured' (where outcomes were discussed but data collection was not evident). Patterns of identified outcomes are described. RESULTS: Of 8373 records, 816 were potentially eligible. Full-text review of a random sample of 218 studies identified 51 for inclusion of which 34 (66%) were 'new' and 17 (33%) were 'modified'. Some 2073 outcomes were identified, and all mapped to domains. 'Anticipated disadvantages' were most frequently identified [660 (32%) outcomes identified across 50 (98%) studies]. No domain was represented in all studies. Under half (944, 46%) of outcomes were 'measured'. 'Surgeon's/operator's experience of the innovation' was more frequently 'mentioned but not measured' [207 (18%) outcomes across 46 (90%) studies] than 'measured' [17 (2%) outcomes, 11 (22%) studies]. CONCLUSION: There is outcome reporting heterogeneity in studies of early phase colorectal cancer surgery. The adoption of core outcome sets may help to resolve these inconsistencies and enable efficient evaluation of surgical innovations.

Understanding and optimising patient and public involvement in trial oversight: an ethnographic study of eight clinical trials.

BACKGROUND: Trial oversight is important for trial governance and conduct. Patients and/or lay members of the public are increasingly included in trial oversight committees, influenced by international patient and public involvement (PPI) initiatives to improve the quality and relevance of research. However, there is a lack of guidance on how to undertake PPI in trial oversight and tokenistic PPI remains an issue. This paper explores how PPI functions in existing trial oversight committees and provides recommendations to optimise PPI in future trials. This was part of a larger study investigating the role and function of oversight committees in trials facing challenges. METHODS: Using an ethnographic study design, we observed oversight meetings of eight UK trials and conducted semi-structured interviews with members of their trial steering committees (TSCs) and trial management groups (TMGs) including public contributors, trial sponsors and funders. Thematic analysis of data was undertaken, with findings integrated to provide a multi-perspective account of how PPI functions in trial oversight. RESULTS: Eight TSC and six TMG meetings from eight trials were observed, and 66 semi-structured interviews conducted with 52 purposively sampled oversight group members, including three public contributors. PPI was reported as beneficial in trial oversight, with public members contributing a patient voice and fulfilling a patient advocacy role. However, public contributors were not always active at oversight meetings and were sometimes felt to have a tokenistic role, with trialists reporting a lack of understanding of how to undertake PPI in trial oversight. To optimise PPI in trial oversight, the following areas were highlighted: the importance of planning effective strategies to recruit public contributors; considering the level of oversight and stage(s) of trial to include PPI; support for public contributors by the trial team between and during oversight meetings. CONCLUSIONS: We present evidence-based recommendations to inform future PPI in trial oversight. Consideration should be given at trial design stage on how to recruit and involve public contributors within trial oversight, as well as support and mentorship for both public contributors and trialists (in how to undertake PPI effectively). Findings from this study further strengthen the evidence base on facilitating meaningful PPI within clinical trials.

Core information set for oesophageal cancer surgery.

BACKGROUND: Surgeons provide patients with information before surgery, although standards of information are lacking and practice varies. The development and use of a 'core information set' as baseline information before surgery may improve understanding. A core set is a minimum set of information to use in all consultations before a specific procedure. This study developed a core information set for oesophageal cancer surgery. METHODS: Information was identified from the literature, observations of clinical consultations and patient interviews. This was integrated to create a questionnaire survey. Stakeholders (patients and professionals) were surveyed twice to assess views on importance of information from 'not essential' to 'absolutely essential' using Delphi methods. Items not meeting predefined criteria were discarded after each survey and the final retained items were voted on, in separate patient and professional stakeholder meetings, to agree the core set. RESULTS: Some 67 information items were identified initially from multiple sources. Survey response rates were 76·5 per cent (185 of 242) and 54·8 per cent (126 of 230) for patients and professionals respectively (first round), and over 83 per cent in both groups thereafter. Health professionals rated short-term clinical outcomes most highly (technical complications), whereas patients prioritized information related to long-term benefits. The consensus meetings agreed the final set, which consisted of: in-hospital milestones to recovery, rates of open-and-close surgery, in-hospital mortality, major complications (reoperation), milestones in recovery after discharge, longer-term eating and drinking and overall quality of life, and chances of survival. CONCLUSION: This study has established a core information set for surgery for oesophageal cancer.

Delphi survey to identify topics to be addressed at the initial follow-up consultation after oesophageal cancer surgery.

BACKGROUND: There is no consensus among patients and healthcare professionals (HCPs) on the topics that need to be addressed after oesophageal cancer surgery. The aim of this study was to identify these topics, using a two-round Delphi survey. METHODS: In round 1, patients and HCPs (surgeons, dieticians, nurses) were invited to rate the importance of 49 topics. The proportion of panellists that considered a topic to be of low, moderate or high importance was then calculated for each of these two groups. Based on these proportions and the i.q.r., topics were categorized as: 'consensus to be included', 'consensus to be excluded' and 'no consensus'. Only topics in the first category were included in the second round. In round 2, panellists were provided with individual and group feedback. To be included in the final list, topics had to meet criteria for consensus and stability. RESULTS: There were 108 patients and 77 HCPs in the round 2 analyses. In general, patients and HCPs considered the same topics important. The final list included 23 topics and revealed that it was most important to address: cancer removed/lymph nodes, the new oesophagus, eating and drinking, surgery, alarming new complaints and the recovery period. CONCLUSION: The study provides surgeons with a list of topics selected by patients and HCPs that may be addressed systematically at the initial follow-up consultation after oesophageal cancer surgery.

Meta-analysis shows clinically relevant and long-lasting deterioration in health-related quality of life after esophageal cancer surgery.

PURPOSE: The purpose of the study is to (1) estimate the direction, clinical relevance, and duration of health-related quality of life (HRQL) change in the first year following esophageal cancer surgery and (2) to assess the robustness of the estimates by subgroup and sensitivity analyses, and an exploration of publication bias. METHODS: A systematic literature search in MEDLINE, EMBASE, CINAHL, PsychINFO, and CENTRAL to identify randomized and non-randomized studies was performed. We compared the baseline HRQL data with 3-, 6-, 9-, or 12-month follow-ups to estimate the magnitude and duration of HRQL change. These estimates were then classified as trivial, small, medium, or large. Primary outcomes were role functioning, eating, and fatigue. Secondary outcomes were physical and social functioning, dysphagia, pain, and coughing problems. We conducted subgroup analysis for open surgery, open surgery preceded by neo-adjuvant therapy, and minimally invasive surgery. Sensitivity analyses assessed the influence of study design, transformation/imputation of the data, and HRQL questionnaire used. RESULTS: We included data from 15 studies to estimate the change in 28 HRQL outcomes after esophageal cancer surgery. The main analysis showed that patients' social functioning deteriorated. Symptoms of fatigue, pain, and coughing problems increased. These changes lasted for 9-12 months, although some symptoms persisted beyond the first year after surgery. For many other HRQL outcomes, estimates were only robust after subgroup or sensitivity analyses (e.g., role and physical functioning), or remained too heterogeneous to interpret (e.g., eating and dysphagia). CONCLUSIONS: Patients will experience a clinically relevant and long-lasting deterioration in HRQL after esophageal cancer surgery. However, for many HRQL outcomes, more and better quality evidence is needed.

Meta-analysis shows clinically relevant and long-lasting deterioration in health-related quality of life after esophageal cancer surgery.

PURPOSE: The purpose of the study is to (1) estimate the direction, clinical relevance, and duration of health-related quality-of-life (HRQL) change in the first year following esophageal cancer surgery and (2) to assess the robustness of the estimates by subgroup and sensitivity analyses, and an exploration of publication bias. METHODS: A systematic literature search in MEDLINE, EMBASE, CINAHL, PsychINFO, and CENTRAL to identify randomized and non-randomized studies was performed. We compared the baseline HRQL data with 3-, 6-, 9-, or 12-month follow-ups to estimate the magnitude and duration of HRQL change. These estimates were then classified as trivial, small, medium, or large. Primary outcomes were role functioning, eating, and fatigue. Secondary outcomes were physical and social functioning, dysphagia, pain, and coughing problems. We conducted subgroup analysis for open surgery, open surgery preceded by neoadjuvant therapy, and minimally invasive surgery. Sensitivity analyses assessed the influence of study design, transformation/imputation of the data, and HRQL questionnaire used. RESULTS: We included the data from 15 studies to estimate the change in 28 HRQL outcomes after esophageal cancer surgery. The main analysis showed that patients' social functioning deteriorated. Symptoms of fatigue, pain, and coughing problems increased. These changes lasted for 9-12 months, although some symptoms persisted beyond the first year after surgery. For many other HRQL outcomes, estimates were only robust after subgroup or sensitivity analyses (e.g., role and physical functioning), or remained too heterogeneous to interpret (e.g., eating and dysphagia). CONCLUSIONS: Patients will experience a clinically relevant and long-lasting deterioration in HRQL after esophageal cancer surgery. However, for many HRQL outcomes, more and better quality evidence is needed.

A prospective study of patient reported outcomes in pancreatic and peri-ampullary malignancy.

BACKGROUND: The purpose of the present study was to describe the impact of treatment of pancreatic and peri-ampullary malignancy on patient reported outcomes (PRO). However, limited data are available describing the impact of curative or palliative therapy on pancreatic/peri-ampullary malignancy and quality of life. METHODS: Patients selected for pancreaticoduodenectomy (PD) completed the European Organisation for Research and Treatment of Cancer QLQ-C30 questionnaire pre-surgery and 6 weeks, 3, 6, 12, 18, and 24 months postoperatively. Patients selected for palliative treatments completed the same questionnaire before treatment and monthly thereafter. Mean scores and 95 % confidence intervals (CI) were calculated for functional scales. Symptom scales and single items were categorized as either minimal or severe, and they were reported as proportions of patients experiencing severe symptoms with 95 % CI. RESULTS: A total of 100 patients (53 planned PD, 47 palliative) were enrolled. Of the 53 patients planned for surgery, 12 had tumors that were unresectable and 41 underwent pancreatoduodenectomy (PD). Seven patients were excluded because of benign histology or concurrent malignancy. Baseline questionnaire compliance was 70 %. For those undergoing PD, there were 53 complications, 7 deaths at 1 year, and 14 deaths at 2 years. Post-surgery most functions and symptoms deteriorated. Recovery in global health and most symptoms occurred by 3 months, and functional scales recovered by 6 months. Recovery of PRO was maintained in the survivors at 2 years. Palliative patients had poorer function and more symptoms at baseline; however, poor follow-up questionnaire compliance prevented further analysis of this group. CONCLUSIONS: Pancreaticoduodenectomy has a short-term negative impact on PRO that recovers within 6 months and is maintained at 2 years in survivors. Further work evaluating palliative and curative treatment in larger patient groups with disease-specific questionnaires is necessary.

Integration of clinical and patient-reported outcomes in surgical oncology.

BACKGROUND: Patient-reported outcomes (PROs) provide information about the patient perspective and experience of undergoing surgery for cancer, but evidence suggests that they are not used widely to influence practice. This review considers key challenges and opportunities for using PROs effectively in gastrointestinal surgical oncology, drawing on principles learnt from surgical oncology in general. METHODS: Systematic reviews of randomized controlled trials (RCTs) in surgical oncology reporting PROs as primary or secondary outcomes, and studies examining methods to communicate PRO information, were identified. Common themes are summarized and the future of PRO studies considered. RESULTS: Reviews highlighted the need for improved design, conduct and reporting of PROs in RCTs in surgical oncology. Main issues related to the multiplicity of PRO measures hindering data synthesis and clinical understanding, problems with missing data risking bias, and limited integration of clinical and PRO data undermining the role of PRO data in practice. Reviews indicated that patients want PRO data to meet information needs and early work shows that graphically displayed PROs are understood by patients. CONCLUSION: PROs have a role in the evaluation of surgical oncology, but increased consensus and collaboration between surgeons and methodologists is needed to improve the design, conduct and reporting of PROs with clinical outcomes in trials. Possible solutions include investing more effort and systematic thought into the PRO rationale in RCTs, the development and use of 'core outcome sets' with PROs, and implementation of the extension to the Consolidated Standards of Reporting Trials (CONSORT) guidelines for reporting PROs in RCTs.

Impact of prostate cancer testing: an evaluation of the emotional consequences of a negative biopsy result.

BACKGROUND: When testing for prostate cancer, as many as 75% of men with a raised prostate-specific antigen (PSA) have a benign biopsy result. Little is known about the psychological effect of this result for these men. METHODS: In all, 330 men participating in the prostate testing for cancer and treatment (ProtecT) study were studied; aged 50-69 years with a PSA level of > or = 3 ng ml(-1) and a negative biopsy result. Distress and negative mood were measured at four time-points: two during diagnostic testing and two after a negative biopsy result. RESULTS: The majority of men were not greatly affected by testing or a negative biopsy result. The impact on psychological health was highest at the time of the biopsy, with around 20% reporting high distress (33 out of 171) and tense/anxious moods (35 out of 180). Longitudinal analysis on 195 men showed a significant increase in distress at the time of the biopsy compared with levels at the PSA test (difference in Impact of Events Scale (IES) score: 9.47; 95% confidence interval (CI) (6.97, 12.12); P<0.001). These levels remained elevated immediately after the negative biopsy result (difference in score: 7.32; 95% CI (5.51, 9.52); P<0.001) and 12 weeks later (difference in score: 2.42; 95% CI (0.50, 1.15); P=0.009). Psychological mood at the time of PSA testing predicted high levels of distress and anxiety at subsequent time-points. CONCLUSIONS: Most men coped well with the testing process, although a minority experienced elevated distress at the time of biopsy and after a negative result. Men should be informed of the risk of distress relating to diagnostic uncertainty before they consent to PSA testing.