Assuring the patient centeredness of patient-reported outcomes: Content validity in medical product development and comparative effectiveness research

Not all patient-reported outcomes are patient-centered, and not all patient-centered outcomes are patient-reported. The essential characteristic of a patient-centered approach to outcome measurement is that it assesses concepts (i.e., health-related phenomena) that are considered most important by members of a given target population, based on direct input from representatives of that population. Concepts for measurement should not be selected based solely on convenience or interest to investigators. Patient-centered patient-reported outcome measures must meet this criterion and also be meaningful and comprehensible to members of a population when administered, including among those with diverse racial/cultural backgrounds and lower educational/literacy levels

Assuring the patient centeredness of patient-reported outcomes: Content validity in medical product development and comparative effectiveness research

Not all patient-reported outcomes are patient-centered, and not all patient-centered outcomes are patient-reported. The essential characteristic of a patient-centered approach to outcome measurement is that it assesses concepts (i.e., health-related phenomena) that are considered most important by members of a given target population, based on direct input from representatives of that population. Concepts for measurement should not be selected based solely on convenience or interest to investigators. Patient-centered patient-reported outcome measures must meet this criterion and also be meaningful and comprehensible to members of a population when administered, including among those with diverse racial/cultural backgrounds and lower educational/literacy levels

Control of bovine brucellosis from persistently infected holdings using RB51 vaccination with test-and-slaughter : a comparative case report from a high incidence area in Portugal

Bovine brucellosis due to Brucella abortus infection causes significant reproduc-tive and production losses in cattle and is a major zoonosis. Eradication of this disease has proved difficult to achieve in Portugal where it still occurs in some regions despite an ongoing national eradication programme. In 2004, the Alent-ejo region, a major cattle producing area, reported one of the highest levels of bovine brucellosis in the country, especially in one divisional area. In that area, bovine brucellosis was particularly problematic in a holding of ten herds, the largest extensive cattle unit in the country, which remained infected despite an extensive test-and-slaughter programme and depopulation of five herds. A 5-year programme of RB51 vaccination with biannual test-and-slaughter was thus implemented in 2004. The apparent animal seroprevalence decreased from 19% (646/3,400) to 3% (88/2930) on the third herd-level test and remained below 0.8% (27/3324) after the fourth test. After the tenth test, the holding had a prevalence of 0.1% (2/2332) and only one herd remained positive with a within-herd prevalence of 1.1% (2/177). The results were compared to all other herds (n = 10) in the divisional area that were also persistently infected but were subject only to test-and-slaughter before being depopulated. In these herds, the strategy of test-andslaughter did not reduce the prevalence, which remained significantly higher than the vaccinated group (median = 0.48% and 8.5% in vaccinated versus non-vaccinated herds; Wilcoxon rank sum test; P < 0.01). The success of this pilot programme in continental Portugal pro-vided a valuable case study to the official veterinary services by illustrating the value of RB51 vaccination with parallel testing and improved biosecurity as a comprehensive and sustainable strategy for bovine brucellosis control in persis-tently infected herds.http://onlinelibrary.wiley.com/journal/10.1111/(ISSN)1865-16822015-04-30hb201

Effect of nivolumab (NIVO) on quality of life (QoL) in patients (pts) with treatment-naïve advanced melanoma (MEL): results of a phase III study (CheckMate 066)

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Patient free text reporting of symptomatic adverse events in cancer clinical research using the National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)

Objective: The study sought to describe patient-entered supplemental information on symptomatic adverse events (AEs) in cancer clinical research reported via a National Cancer Institute software system and examine the feasibility of mapping these entries to established terminologies. Materials and Methods: Patients in 3 multicenter trials electronically completed surveys during cancer treatment. Each survey included a prespecified subset of items from the National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Upon completion of the survey items, patients could add supplemental symptomatic AE information in a free text box. As patients typed into the box, structured dropdown terms could be selected from the PRO-CTCAE item library or Medical Dictionary for Regulatory Activities (MedDRA), or patients could type unstructured free text for submission. Results: Data were pooled from 1760 participants (48% women; 78% White) who completed 8892 surveys, of which 2387 (26.8%) included supplemental symptomatic AE information. Overall, 1024 (58%) patients entered supplemental information at least once, with an average of 2.3 per patient per study. This encompassed 1474 of 8892 (16.6%) dropdowns and 913 of 8892 (10.3%) unstructured free text entries. One-third of the unstructured free text entries (32%) could be mapped post hoc to a PRO-CTCAE term and 68% to a MedDRA term. Discussion: Participants frequently added supplemental information beyond study-specific survey items. Almost half selected a structured dropdown term, although many opted to submit unstructured free text entries. Most free text entries could be mapped post hoc to PRO-CTCAE or MedDRA terms, suggesting opportunities to enhance the system to perform real-time mapping for AE reporting. Conclusions: Patient reporting of symptomatic AEs using a text box functionality with mapping to existing terminologies is both feasible and informative

Software for administering the National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events: Usability study

Background: The US National Cancer Institute (NCI) developed software to gather symptomatic adverse events directly from patients participating in clinical trials. The software administers surveys to patients using items from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) through Web-based or automated telephone interfaces and facilitates the management of survey administration and the resultant data by professionals (clinicians and research associates). Objective: The purpose of this study was to iteratively evaluate and improve the usability of the PRO-CTCAE software. Methods: Heuristic evaluation of the software functionality was followed by semiscripted, think-aloud protocols in two consecutive rounds of usability testing among patients with cancer, clinicians, and research associates at 3 cancer centers. We conducted testing with patients both in clinics and at home (remotely) for both Web-based and telephone interfaces. Furthermore, we refined the software between rounds and retested. Results: Heuristic evaluation identified deviations from the best practices across 10 standardized categories, which informed initial software improvement. Subsequently, we conducted user-based testing among 169 patients and 47 professionals. Software modifications between rounds addressed identified issues, including difficulty using radio buttons, absence of survey progress indicators, and login problems (for patients) as well as scheduling of patient surveys (for professionals). The initial System Usability Scale (SUS) score for the patient Web-based interface was 86 and 82 (P=.22) before and after modifications, respectively, whereas the task completion score was 4.47, which improved to 4.58 (P=.39) after modifications. Following modifications for professional users, the SUS scores improved from 71 to 75 (P=.47), and the mean task performance improved significantly (4.40 vs 4.02; P=.001). Conclusions: Software modifications, informed by rigorous assessment, rendered a usable system, which is currently used in multiple NCI-sponsored multicenter cancer clinical trials

Epidemiology of Escherichia coli bacteraemia in England: results of an enhanced sentinel surveillance programme

Background: Escherichia coli causes over one third of the bacteraemia cases in England each year, and the incidence of these infections is increasing. Aim: To determine the underlying risk factors associated with E. coli bacteraemia. Methods: A three month enhanced sentinel surveillance study involving 35 National Health Service hospitals was undertaken in the winter of 2012/13 to collect risk factor information and further details on the underlying source of infection to augment data already collected by the English national surveillance programme. Antimicrobial susceptibility results for E. coli isolated from blood and urine were also collected. Findings: A total of 1,731 cases of E. coli bacteraemia were included. The urogenital tract was the most commonly reported source of infection (51.2% of cases) with prior treatment for a urinary tract infection being the largest independent effect associated with this infection source. Half of all patients had prior healthcare exposure in the month prior to the bacteraemia with antimicrobial therapy and urinary catheterisation being reported in one third and one fifth of these patients. Prior healthcare exposure was associated with a higher proportion of antibiotic non-susceptibility in the blood culture isolates (P=0.001). Conclusion: Analysis of risk factors suggests potential community and hospital-related interventions particularly better use of urinary catheters and improved antibiotic management of urinary tract infections. As part of the latter strategy, antibiotic resistance profiles need to be closely monitored to ensure treatment guidelines are up to date to limit inappropriate empiric therapy

Risk Stratification, Treatment Selection, and Transplant Eligibility in Multiple Myeloma: A Qualitative Study of the Perspectives and Self-Reported Practices of Oncologists

Background Since the early 2000s, treatment options for multiple myeloma have rapidly expanded, adding significant complexity to the management of this disease. To our knowledge, no systematic qualitative research on clinical decision-making in multiple myeloma has been published. We sought to characterize how physicians view and implement guidelines and incorporate novel approaches into patient care. Methods We designed a semi-structured qualitative interview guide informed by literature review and an expert advisory panel. We conducted 60-minute interviews with a diverse sample of oncology physicians in the southeast United States. We used a constant comparative method to code and analyze interview transcripts. The research team and advisory panel discussed and validated emergent themes. Results Participants were 13 oncologists representing 5 academic and 4 community practices. Academic physicians reported using formal risk-stratification schemas; community physicians typically did not. Physicians also described differences in eligibility criteria for transplantation; community physicians emphasized distance, social support, and psychosocial capacity in making decisions about transplantation referral; the academic physicians reported using more specific clinical criteria. All physicians reported using a maintenance strategy both for post-transplant and for transplant-ineligible patients; however, determining the timing of maintenance therapy initiation and the response were reported as challenging, as was recognition or definition of relapse, especially in terms of when treatment re-initiation is indicated. Conclusions Practices reported by both academic and community physicians suggest opportunities for interventions to improve patient care and outcomes through optimal multiple myeloma management and therapy selection. Community physicians in particular might benefit from targeted education interventions about risk stratification, transplant eligibility, and novel therapies