Developing core outcomes sets: methods for identifying and including patient-reported outcomes (PROs)

BACKGROUND: Synthesis of patient-reported outcome (PRO) data is hindered by the range of available PRO measures (PROMs) composed of multiple scales and single items with differing terminology and content. The use of core outcome sets, an agreed minimum set of outcomes to be measured and reported in all trials of a specific condition, may improve this issue but methods to select core PRO domains from the many available PROMs are lacking. This study examines existing PROMs and describes methods to identify health domains to inform the development of a core outcome set, illustrated with an example. METHODS: Systematic literature searches identified validated PROMs from studies evaluating radical treatment for oesophageal cancer. PROM scale/single item names were recorded verbatim and the frequency of similar names/scales documented. PROM contents (scale components/single items) were examined for conceptual meaning by an expert clinician and methodologist and categorised into health domains. A patient advocate independently checked this categorisation. RESULTS: Searches identified 21 generic and disease-specific PROMs containing 116 scales and 32 single items with 94 different verbatim names. Identical names for scales were repeatedly used (for example, ‘physical function’ in six different measures) and others were similar (overlapping face validity) although component items were not always comparable. Based on methodological, clinical and patient expertise, 606 individual items were categorised into 32 health domains. CONCLUSION: This study outlines a methodology for identifying candidate PRO domains from existing PROMs to inform a core outcome set to use in clinical trials

Trial outcomes and information for clinical decision-making: a comparative study of opinions of health professionals

Background: Trials are robust sources of data for clinical practice, however, trial outcomes may not reflect what is important to communicate for decision making. The study compared clinicians’ views of outcomes to include in a core outcome set for colorectal cancer (CRC) surgery, with what clinicians considered important information for clinical practice (core information). Methods: Potential outcomes/information domains were identified through systematic literature reviews, reviews of hospital information leaflets and interviews with patients. These were organized into 6 categories, and used to design a questionnaire survey that asked surgeons and nurses from a sample of CRC centers to rate the importance of each domain as an outcome or as information on a 9-point Likert scale. Respondents were re-surveyed (round 2) following group feedback (Delphi methods). Comparisons were made by calculating the difference in mean scores between the outcomes and information domains, and paired t-tests were used to explore the difference between mean scores of the 6 outcome/information categories. Results: Data sources identified 1216 outcomes/information of CRC surgery that informed a 94 item questionnaire. First round questionnaires were returned from 63/81 (78%) of centers. Clinicians rated 76/94 (84%) domains of higher importance to measure in trials than to information to communicate to patients in Round 1. This was reduced to 24/47 (51%) in Round 2. The greatest difference was evident in domains about survival, which was rated much more highly as a trial outcome than important piece of information for decision-making (mean difference 2.3, 95% CI 1.9-2.8, p<0.0001). Specific complications and quality-of-life domains were rated similarly (mean difference 0.18, 95% CI -0.1-0.4, p=0.2 and 0.2, 95% CI -0.1-0.5, p=0.2 respectively). Conclusions: Whilst clinicians want to measure key outcomes in trials, they rate these as less important to communicate in decision-making with patients. This discrepancy needs to be explored and addressed to maximize the impact of trials on clinical practice

Core Outcomes for Colorectal Cancer Surgery: A Consensus Study

Background: Colorectal cancer (CRC) is a major cause of worldwide morbidity and mortality. Surgical treatment is common, and there is a great need to improve the delivery of such care. The gold standard for evaluating surgery is within well-designed randomized controlled trials (RCTs); however, the impact of RCTs is diminished by a lack of coordinated outcome measurement and reporting. A solution to these issues is to develop an agreed standard “core” set of outcomes to be measured in all trials to facilitate cross-study comparisons, meta-analysis, and minimize outcome reporting bias. This study defines a core outcome set for CRC surgery. Methods and Findings: The scope of this COS includes clinical effectiveness trials of surgical interventions for colorectal cancer. Excluded were nonsurgical oncological interventions. Potential outcomes of importance to patients and professionals were identified through systematic literature reviews and patient interviews. All outcomes were transcribed verbatim and categorized into domains by two independent researchers. This informed a questionnaire survey that asked stakeholders (patients and professionals) from United Kingdom CRC centers to rate the importance of each domain. Respondents were resurveyed following group feedback (Delphi methods). Outcomes rated as less important were discarded after each survey round according to predefined criteria, and remaining outcomes were considered at three consensus meetings; two involving international professionals and a separate one with patients. A modified nominal group technique was used to gain the final consensus. Data sources identified 1,216 outcomes of CRC surgery that informed a 91 domain questionnaire. First round questionnaires were returned from 63 out of 81 (78%) centers, including 90 professionals, and 97 out of 267 (35%) patients. Second round response rates were high for all stakeholders (>80%). Analysis of responses lead to 45 and 23 outcome domains being retained after the first and second surveys, respectively. Consensus meetings generated agreement on a 12 domain COS. This constituted five perioperative outcome domains (including anastomotic leak), four quality of life outcome domains (including fecal urgency and incontinence), and three oncological outcome domains (including long-term survival). Conclusion: This study used robust consensus methodology to develop a core outcome set for use in colorectal cancer surgical trials. It is now necessary to validate the use of this set in research practice

Valve-preserving surgery on the bicuspid aortic valve

Valve repair has emerged as an important intervention for the management of bicuspid aortic valve disease. This systematic review aims to assess the safety, efficacy and durability of bicuspid aortic valve repair. Initial searches yielded 682 abstracts, reduced by de-duplication to 370, of which 56 full papers were accessed and 30 met the inclusion criteria. Overall, 163 unique outcomes for bicuspid aortic valve-preserving surgery were reported on 280 occasions. Bicuspid aortic valve-preserving surgery exhibited low operative mortality (0.0-5.2%), excellent 5-year survival (82-100%) and 43-100% 5-year freedom from reoperation. Bicuspid aortic valve repair is safe and efficacious, but concerns regarding its durability necessitate further standardized outcome assessments

Effect of preoperative symptoms on outcomes after valve repair for degenerative aortic insufficiency

OBJECTIVES: This study evaluated the effect of preoperative symptoms on long-term survival and valve reintervention in patients undergoing aortic valve repair (AVr) for severe degenerative aortic insufficiency (AI). METHODS: Between March 1996 and June 2010, 274 patients underwent AVr for severe AI. Seventy-seven were performed in asymptomatic patients (Group I) and 197 were performed in symptomatic (Group II). Patients in Group I were younger (43.9 ± 15.0 vs 54.1 ± 15.5 years; P &lt; 0.001) with a higher proportion of bicuspid valves (n = 40, 51.9% vs n = 68, 30.4%; P = 0.008). Group II had more patients with impaired left ventricular (LV; n = 36, 18.3% vs n = 5, 6.4%; P = 0.013). The mean follow-ups for Group I and Group II were 43.0 ± 34.0 and 61.3 ± 39.5 months, respectively. RESULTS: There was no in-hospital mortality. Overall, leaflet repair and reimplantation were higher in Group I (P = &lt; 0.001 and P = 0.002, respectively), whereas subcommissural annuloplasty for tricuspid AV was higher in Group II (P = 0.002). Leaflet shaving and plication were performed in more patients in Group I (P = 0.002 for both). There was no difference in early complications, but during follow-up atrial fibrillation was more frequent in Group II (P = 0.03). There were 10 late cardiac deaths, all in Group II (P = 0.04). Overall, 10-year cardiac survival was better in Group I (100 vs 77.3 ± 8.6%) but not significant (P = 0.1). At 10 years, freedom from ≥AI2+ was 63.2 ± 8.1 vs 56.4 ± 4.5% (P = 0.4), and freedom from AV reintervention was 81.8 ± 7.1 vs 89.0 ± 2.8% (P = 0.36), in Groups I and II, respectively. In the whole cohort, 10-year freedom from AV reintervention was greater in patients with an end-diastolic diameter of &lt;60 mm (90.0 ± 7.6 vs 76.3 ± 5.5%; P = 0.003). Multivariate analysis identified increasing age as the only independent predictor of overall survival (P = 0.03). The incidence of valve-related complications was similar. CONCLUSION: In asymptomatic patients with severe degenerative AI, AVr is associated with excellent long-term outcomes, while symptoms are associated with reduced long-term survival. In dilated LV, freedom from reoperation is lower. If feasible, early AVr is recommended in experienced centres. © The Author 2013. Published by Oxford University Press on behalf of the European Association for Cardio-Thoracic Surgery. All rights reserved

Influence of higher valve gradient on long-term outcome after aortic valve repair.

To evaluate the effect of higher post-operative valve gradient on freedom from valve re-intervention and death in patients undergoing aortic valve repair (AVr)

Exploring the role of laparoscopic surgery in two-stage hepatectomy for bilobar colorectal liver metastases

BACKGROUND: The role of laparoscopy in two-stage hepatectomy for bilobar colorectal liver metastases (CRLMs) has not yet been extensively investigated.PATIENTS AND METHODS: We reviewed a prospectively collected database of 302 consecutive patients undergoing laparoscopic liver resection at our institution between 2003 and 2011.RESULTS: Eight patients undergoing laparoscopic first/second-stage hepatectomy for bilobar CRLMs (male/female 6:2; median age, 64 years) were analyzed. The first stage consisted of laparoscopic clearance of the left lobe in all patients with no postoperative morbidity and mortality. Seven patients underwent portal vein embolization or ligation. The median interval between first- and second-stage hepatic resections was 89 days (range, 36-123 days). Second-stage hepatectomy with right lobar clearance (open, n=5; laparoscopic, n=2; laparoscopic to open, n=1) was associated with no mortality and an operative morbidity rate of 50%. Adhesions were judged to be minimal or absent during the second-stage procedure. Complications included intra-abdominal collection (n=2), bleeding requiring re-operation (n=1), and bile leak (n=1). R0 resection was obtained in 7 of 8 cases after first-stage resection and in 8 of 8 cases after second-stage resection. Three patients (38%) died from disease recurrence. Of the remaining 5 patients, 4 are disease-free at a median follow-up of 24 months (range, 9-27 months).CONCLUSIONS: The well-recognized advantages of laparoscopy may play a favorable role in the management of patients with bilobar CRLMs candidate for a two-stage resection. The first-stage laparoscopic clearance of the left lobe could progressively become the "gold standard." Laparoscopic second-stage hepatectomy should be limited to selected cases