Correction to: Psychometric properties of the FACT-M questionnaire in patients with Merkel cell carcinoma.

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When insulin degludec enhances quality of life in patients with type 2 diabetes: a qualitative investigation.

BACKGROUND: Anecdotal reports suggest that insulin degludec (IDeg) may offer unique health-related quality of life (HRQoL) benefits. As the nature of these benefits remain unclear, this study utilized qualitative research methods to investigate and elucidate the experience of "feeling better" after initiating IDeg. METHODS: Twenty adults with type 2 diabetes (T2D) who reported "feeling better" on IDeg for > 3 months participated in 90-min interviews. One focus group and nine telephone interviews were conducted at two sites in the United States (US) and one focus group was conducted in Switzerland. Patients were ≥ 18 years of age, did not take mealtime insulin, and had switched to IDeg from another basal insulin. Discussions were audio-recorded, transcribed and translated (Swiss German). Utilizing grounded theory, transcripts were analyzed by sorting quotes into concepts using thematic analysis. RESULTS: Participants' mean age was 66 years and the average duration of T2D was 17.6 years. Mean duration of IDeg use was 1.45 years. Four major factors were identified as key contributors to patients' sense of "feeling better": 1) reduced sense of diabetes as burdensome and requiring excessive attention; 2) enhanced feelings of adaptability and freedom; 3) heightened sense of security, especially regarding concerns about hypoglycemia; and 4) greater sense of physical well-being (greater energy/less fatigue). Content saturation was achieved. Generally, patients from the US sites were more focused on medical results than Swiss patients, who were more likely to identify IDeg's effect on overall HRQoL. A limitation of the study was that the population was primarily white, > 60 and otherwise healthy (no comorbid physical or mental condition). CONCLUSIONS: A group of patients with T2D, who had switched to IDeg from another basal insulin, reported HRQoL benefits which were attributed to both diabetes-specific improvements (feeling less burdened by day-to-day diabetes demands) and non-specific gains (greater energy). The conclusions may have limited transferability due to the characteristics of the sample population and further research is needed.

Psychometric properties of the FACT-M questionnaire in patients with Merkel cell carcinoma.

BACKGROUND: No validated disease-specific questionnaires exist to capture health-related quality of life (HRQoL) in patients with Merkel cell carcinoma (MCC). The Functional Assessment of Cancer Therapy - Melanoma (FACT-M) is validated in patients with melanoma, which shares many similarities with MCC. This paper reports the psychometric properties of the FACT-M in the metastatic MCC population. METHODS: Data were collected as part of a single-arm, open-label, multicenter trial involving patients with metastatic MCC who had failed at least one previous line of chemotherapy. FACT-M and EQ-5D were administered at baseline, Week 7, Week 13, and Week 25. An optional interview was administered at the same time points. MCC-specific FACT-M scores were derived following a combined quantitative and qualitative approach. Reliability and construct validity of original and additional MCC-specific FACT-M scores were assessed at baseline. Capacity to detect change in tumor size was assessed from baseline to Week 7. Minimally important differences (MIDs) were computed using distribution and anchor-based methods. RESULTS: Baseline assessments were available in 70 patients (mean age: 70 years; 74.3% male); 19 patients were interviewed at baseline. Additional MCC-specific scores were as follows: Physical Function score (six items), Psychological Impact score (six items), and MCC summary score (12 items). FACT-M original and additional MCC-specific scores both demonstrated acceptable psychometric properties: high reliability (Cronbach's alpha: 0.81-0.96), good convergent validity (correlations above 0.4 observed for 88% of items of the Melanoma surgery scale, 75% of items of the Melanoma scale, and 100% of items of the other FACT-M domains). Some evidence of floor/ceiling effects and poor discriminant ability was found. Higher scores (better HRQoL) on all FACT-M domains were observed in patients with better functioning (assessed by ECOG performance score), supporting clinical validity. Despite the small sample for responsiveness analysis (n = 37), the majority of FACT-M scores showed sensitivity to changes in tumor size at Week 7 with small to moderate effect sizes. MIDs were consistent with previously reported values in the literature for FACT-M domains. CONCLUSIONS: FACT-M is suitable to capture HRQoL in patients with metastatic MCC, thus making it a potential candidate for assessing HRQoL in MCC trials. TRIAL REGISTRATION: This study is a post-hoc analysis conducted on data collected in Part A of the JAVELIN Merkel 200 trial. This trial was registered on 2 June 2014 with ClinicalTrials.gov as NCT02155647 .

How do Clostridium difficile infections affect nurses' everyday hospital work: A qualitative study.

This qualitative study explored the impact of Clostridium difficile infections on nurses' everyday work in the hospital. Twelve nurses (six in France and six in the United States) were interviewed in depth using a semi-structured interview guide. Thematic analysis of the interviews was performed. Managing diarrhoea and taking precautionary measures for infection control were the two most inconvenient aspects nurses reported with C. difficile patient management. Precautions included contact isolation, hand hygiene and reorganization/coordination of nursing care and ward. Precautions were time consuming and significantly increased nurses' workload when combined with caring for patients with uncontrollable, frequent bouts of diarrhoea. Management of C. difficile infection is extremely burdensome for nurses in their everyday work and disruptive to hospital organizations as a whole. Prevention of C. difficile infections, together with coordinated team work and communication, would therefore contribute to decreasing nurses' workload and the burden to health-care facilities associated with caring for these patients.

Interviewing to develop Patient-Reported Outcome (PRO) measures for clinical research: eliciting patients' experience.

Patient-reported outcome (PRO) measures must provide evidence that their development followed a rigorous process for ensuring their content validity. To this end, the collection of data is performed through qualitative interviews that allow for the elicitation of in-depth spontaneous reports of the patients' experiences with their condition and/or its treatment. This paper provides a review of qualitative research applied to PRO measure development. A clear definition of what is a qualitative research interview is given as well as information about the form and content of qualitative interviews required for developing PRO measures. Particular attention is paid to the description of interviewing approaches (e.g., semi-structured and in-depth interviews, individual vs. focus group interviews). Information about how to get prepared for a qualitative interview is provided with the description of how to develop discussion guides for exploratory or cognitive interviews. Interviewing patients to obtain knowledge regarding their illness experience requires interpersonal and communication skills to facilitate patients' expression. Those skills are described in details, as well as the skills needed to facilitate focus groups and to interview children, adolescents and the elderly. Special attention is also given to quality assurance and interview training. The paper ends on ethical considerations since interviewing for the development of PROs is performed in a context of illness and vulnerability. Therefore, it is all the more important that, in addition to soliciting informed consent, respectful interactions be ensured throughout the interview process.

Patient and Caregiver Perceptions of Advanced Bladder Cancer Systemic Treatments: Infodemiology Study Based on Social Media Data.

BACKGROUND: In 2022, it was estimated that more than 80,000 new cases of bladder cancer (BC) were diagnosed in the United States, 12% of which were locally advanced or metastatic BC (advanced BC). These forms of cancer are aggressive and have a poor prognosis, with a 5-year survival rate of 7.7% for metastatic BC. Despite recent therapeutic advances for advanced BC, little is known about patient and caregiver perceptions of different systemic treatments. To further explore this topic, social media can be used to collect the perceptions of patients and caregivers when they discuss their experiences on forums and online communities. OBJECTIVE: The aim of this study was to assess patient and caregiver perceptions of chemotherapy and immunotherapy for treating advanced BC from social media-posted data. METHODS: Public posts on social media in the United States between January 2015 and April 2021 from patients with advanced BC and their caregivers were collected. The posts included in this analysis were geolocalized to the United States; collected from publicly available domains and sites, including social media sites such as Twitter and forums such as patient association forums; and were written in English. Posts mentioning any line of chemotherapy or immunotherapy were qualitatively analyzed by two researchers to classify perceptions of treatments (positive, negative, mixed, or without perception). RESULTS: A total of 80 posts by 69 patients and 142 posts by 127 caregivers mentioning chemotherapy, and 42 posts by 31 patients and 35 posts by 32 caregivers mentioning immunotherapy were included for analysis. These posts were retrieved from 39 public social media sites. Among patients with advanced BC and their caregivers, treatment perceptions of chemotherapy were more negative (36%) than positive (7%). Most of the patients' posts (71%) mentioned chemotherapy factually without expressing a perception of the treatment. The caregivers' perceptions of treatment were negative in 44%, mixed in 8%, and positive in 7% of posts. In combined patient and caregiver posts, immunotherapy was perceived positively in 47% of posts and negatively in 22% of posts. Caregivers also posted more negative perceptions (37%) of immunotherapy than patients (9%). Negative perceptions of both chemotherapy and immunotherapy were mainly due to side effects and perceived lack of effectiveness. CONCLUSIONS: Despite chemotherapy being standard first-line therapy for advanced BC, negative perceptions were identified on social media, particularly among caregivers. Addressing these negative perceptions of treatment may improve treatment adoption. Strengthening support for patients receiving chemotherapy and their caregivers to help them manage side effects and understand the role of chemotherapy in the treatment of advanced BC would potentially enable a more positive experience.

Integrating qualitative interviews in drug development and the use of qualitative evidence in product labelling and health technology assessments: a review.

Objective: Including qualitative research in clinical trial design is an innovative approach to understanding patients' perspective and incorporate the patient's voice in all stages of drug development and evaluation. This review aims to explore current practices, lessons learned from the literature, as well as how qualitative interviews are considered by health authorities for marketing authorization and reimbursement. Methods: A targeted literature review of Medline and Embase databases was conducted in February 2022 to identify publications on qualitative methods embedded in clinical trial of pharmaceutical products. An additional search of guidelines and labeling claims of approved products regarding qualitative research was performed in various sources of grey literature. Results: From the 24 publications and nine documents reviewed, we identified the research questions addressed with qualitative methods during clinical trials (e.g., change in quality of life, symptoms assessment, treatment benefit), preferred data collection methods (e.g., interviews), and data collection points (e.g., baseline and exit interviews). Moreover, the data from labels and HTAs demonstrate that qualitative data can play an important role in approval processes. Conclusion: The use of in-trial interviews is still emerging and is not yet common practice. Although the industry, scientific community, regulatory agencies and HTAs are showing an increasing interest in the use of evidence generated via in-trial interviews, guidance from regulators and HTAs would be helpful. Developing new methods and technologies to address the common challenges for such interviews is key to progress.

Lived experiences of patients with distal renal tubular acidosis treated with ADV7103 and of their caregivers: a qualitative study.

BACKGROUND: Consequences of distal renal tubular acidosis (dRTA) on growth, bone and kidney, sometimes associated with hearing loss, may significantly affect quality of life (QoL). This descriptive qualitative study explores QoL linked to dRTA and gathers the impressions of patients with this rare disease (and caregivers) 5 years after enrolment in a clinical study, during which patients were treated with ADV7103, a prolonged-release granule formulation combining potassium citrate and potassium bicarbonate. Semi-structured, one-hour interviews with 6 adult and 13 paediatric patients with a confirmed diagnosis of dRTA and with parents of paediatric patients were performed using an interview guide. Qualitative analysis of anonymized interview transcripts based on grounded theory was conducted. RESULTS: The main QoL domains impacted by dRTA and its treatment were education/work, social/family life, and emotional and physical well-being. ADV7103 (administered twice daily) was compared with the standard of care (SoC) taken before study entry (more than twice daily). Patients/parents reported that switching from previous SoC to ADV7103 had changed their lives: Difficulties at school due to burdensome administrative issues and need to explain disease and treatment affecting all families of paediatric patients (n = 13) disappeared, facilitating parents who had stopped working (to deal with their child's treatment) to return to work, Family functioning was improved (n = 18), as travel and holidays became easier to organise and patients/parents stopped thinking about managing treatment daily/nightly, reducing tension in the family or couple, The emotional burden of disease perceived was relieved (n = 12) in the absence of treatment-related invasive questions from others, Gastro-intestinal adverse events and taste problems improved with ADV7103 (n = 18) and better compliance led to milder physical impacts and less need to be hospitalised. The mean satisfaction score with ADV7103 compared to SoC was 9 out of 10 (10 = very satisfied). ADV7103 exceeded or met the expectations of 14 out of 17 patients that commented on that. CONCLUSIONS: Qualitative interviews show that dRTA and its treatment have a significant impact on QoL of patients and parents and that ADV7103 helps improve daily-life and reduces treatment burden, resulting in greater overall satisfaction of the patients and their families. Trial registration EU Clinical Trials Register, EudraCT 2013-003828-36 on the 3rd of September 2013.

Perceived Unmet Needs in Patients Living With Advanced Bladder Cancer and Their Caregivers: Infodemiology Study Using Data From Social Media in the United States.

BACKGROUND: Locally advanced or metastatic bladder cancer (BC), which is generally termed advanced BC (aBC), has a very poor prognosis, and in addition to its physical symptoms, it is associated with emotional and social challenges. However, few studies have assessed the unmet needs and burden of aBC from patient and caregiver perspectives. Infodemiology, that is, epidemiology based on internet health-related content, can help obtain more insights on patients' and caregivers' experiences with aBC. OBJECTIVE: The study aimed to identify the main discussion themes and the unmet needs of patients with aBC and their caregivers through a mixed methods analysis of social media posts. METHODS: Social media posts were collected between January 2015 and April 2021 from US geolocalized sites using specific keywords for aBC. Automatic natural language processing (regular expressions and machine learning) methods were used to filter out irrelevant content and identify verbatim posts from patients and caregivers. The verbatim posts were analyzed to identify main discussion themes using biterm topic modeling. Difficulties or unmet needs were further explored using qualitative research methods by 2 independent annotators until saturation of concepts. RESULTS: A total of 688 posts from 262 patients and 1214 posts from 679 caregivers discussing aBC were identified. Analysis of 340 randomly selected patient posts and 423 randomly selected caregiver posts uncovered 33 unique unmet need categories among patients and 36 among caregivers. The main unmet patient needs were related to challenges regarding adverse events (AEs; 28/95, 29%) and the psychological impact of aBC (20/95, 21%). Other patient unmet needs identified were prognosis or diagnosis errors (9/95, 9%) and the need for better management of aBC symptoms (9/95, 9%). The main unmet caregiver needs were related to the psychological impacts of aBC (46/177, 26.0%), the need for support groups and to share experiences between peers (28/177, 15.8%), and the fear and management of patient AEs (22/177, 12.4%). CONCLUSIONS: The combination of manual and automatic methods allowed the extraction and analysis of several hundreds of social media posts from patients with aBC and their caregivers. The results highlighted the emotional burden of cancer for both patients and caregivers. Additional studies on patients with aBC and their caregivers are required to quantitatively explore the impact of this disease on quality of life.

Validation of the Pediatric Narcolepsy Screening Questionnaire (PNSQ): A cross-sectional, observational study.

OBJECTIVE/BACKGROUND: This study evaluated psychometric properties of the Pediatric Narcolepsy Screening Questionnaire (PNSQ), developed in response to the difficulty of identifying pediatric narcolepsy. PATIENTS/METHODS: The initial PNSQ was updated following debriefing interviews with parents of children with suspected/diagnosed narcolepsy. Subsequently, newly recruited caregivers were categorized into groups: clinician-confirmed narcolepsy, other sleep problems (OSP), and no sleep problems (controls). Caregivers completed the 11-item PNSQ assessing narcolepsy symptomatology. PNSQ psychometric properties were evaluated; mean PNSQ Total Score (TS) was compared inter-group using analysis of variance. RESULTS: The analysis population (N = 158) included patients with narcolepsy (n = 49), OSP (n = 55), and controls (n = 54); mean ± SD age was 13.8 ± 2.8, 10.2 ± 4.3, and 10.0 ± 3.8 years, respectively. Inter-item Pearson correlations (range, 0.22-0.75) indicated good construct validity. Principal component analysis confirmed unidimensionality. Item discriminative power was high for narcolepsy vs control (range, 0.693-0.936) and lower for narcolepsy vs OSP (range, 0.584-0.729). The latent trait was well covered (separation index = 0.868). Item 7 (vivid dreams/nightmares), having low discriminative power and specificity, was removed. Cronbach's alpha (final PNSQ) indicated high internal consistency reliability (raw alpha = 0.88). Mean ± SD PNSQ TS (range, 0-50) in the narcolepsy, OSP, and control groups were 34.98 ± 7.98, 25.20 ± 9.43, and 9.54 ± 9.38, respectively (nominal P < 0.0001). Classification by PNSQ TS was defined: PNSQ+ (likely narcolepsy, TS ≥ 29), PNSQ 0 (likely OSP, TS 19-28), and PNSQ- (narcolepsy unlikely, TS ≤ 18); patients with narcolepsy were classified as PNSQ+ (79.6%), PNSQ 0 (18.4%), and PNSQ- (2.0%). CONCLUSIONS: The PNSQ demonstrated good psychometric properties and excellent performance discriminating narcolepsy, OSP, and control groups.

Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach.

BACKGROUND: MPS IIIA is a rare, degenerative pediatric genetic disease characterized by symptoms impacting cognition, mobility and behavior; the mean age of death is around 15 years of age. Currently, there are no approved therapies for MPS IIIA. METHODS: A two-year, multi-center, prospective, descriptive cohort study was conducted to document the natural history course of MPS IIIA. In the context of this study, semi-structured interviews were performed with parents of children at study entry and one year later. Interview transcripts were analyzed using thematic analysis methods to identity concepts of interest to children and parents, identify what factors impacted parents' burden the most, and develop qualitatively-derived disease severity stages. Children were sorted into these stages according to the symptoms their parents described at the entry interview. This sorting was compared quantitatively to the sorting of children at baseline according to the child's calendar age and their BSID development quotient (DQ). RESULTS: 22 parents in France, Germany, the Netherlands and the UK were interviewed. Children ranged in age from 28 to 105 months (mean 61.4 months). The conceptual models for children's symptoms and impacts and parents' impacts provided a detailed and comprehensive picture of what it is like for children of various ages and their parents to live with MPS IIIA. Four factors were identified as mediating the burden perceived by parents: state support, family support, time since diagnosis, and parent coping strategy. Four disease stages were developed, accounting for both the presence and the severity of MPS IIIA symptoms. The comparison of children's sorting into these stages with the BSID DQ and the child's calendar age showed strong statistical associations. CONCLUSIONS: The findings of this qualitative research embedded in a natural history study add to the current understanding of MPS IIIA as a complex disease that impacts every aspect of the lives of children and their families. This study demonstrates the unique potential of mixed methods research in rare diseases to address some of the current limitations of more traditional quantitative approaches by providing an individualized, detailed understanding of the patient experience.

Patient Experiences with Avelumab in Treatment-Naïve Metastatic Merkel Cell Carcinoma: Longitudinal Qualitative Interview Findings from JAVELIN Merkel 200, a Registrational Clinical Trial.

BACKGROUND AND OBJECTIVE: Avelumab is approved for the treatment of metastatic Merkel cell carcinoma, a rare aggressive skin cancer with a poor prognosis. The aim of this qualitative study embedded in a clinical trial was to explore patient experiences while receiving avelumab. METHODS: All treatment-naïve patients with metastatic Merkel cell carcinoma entering part B of the phase II, open-label, international, JAVELIN Merkel 200 trial (NCT02155647) were invited to participate in optional semi-structured phone interviews before avelumab administration (baseline) and at weeks 13 and 25. Interviews were conducted by trained professionals, audio-recorded, transcribed and analysed. Key concepts identified at baseline were assessed during follow-up interviews. RESULTS: Twenty-nine patients completed the baseline interview; 19 had at least one follow-up interview. Baseline interviews described the patients' challenging journeys before being correctly diagnosed with Merkel cell carcinoma, the negative psychological burden of living with a symptomless disease and the hope for avelumab to be a successful therapy. During the trial, most patients reported an increased or continued sense of hope and willingness to fight metastatic Merkel cell carcinoma. Patients who self-reported disease improvement (n = 12) also reported stability or improvement in physical well-being and ability to do daily activities, having more energy, worrying less and being optimistic. Six patients who reported their condition as stable (n = 4) or worsened (n = 3) reported a worsening of physical well-being. Nine patients reported fatigue/tiredness on the day of and after receiving avelumab. Baseline and longitudinal experiences were similar across countries. CONCLUSIONS: This study suggests that patients experience perceptible benefits in physical and psychological well-being following treatment success with first-line avelumab in metastatic Merkel cell carcinoma.

Multinational development of a questionnaire assessing patient satisfaction with anticoagulant treatment: the 'Perception of Anticoagulant Treatment Questionnaire' (PACT-Q).

BACKGROUND: The side effects and burden of anticoagulant treatments may contribute to poor compliance and consequently to treatment failure. A specific questionnaire is necessary to assess patients' needs and their perceptions of anticoagulant treatment. METHODS: A conceptual model of expectation and satisfaction with anticoagulant treatment was designed by an advisory board and used to guide patient (n = 31) and clinician (n = 17) interviews in French, US English and Dutch. Patients had either atrial fibrillation (AF), deep venous thrombosis (DVT), or pulmonary embolism (PE). Following interviews, three PACT-Q language versions were developed simultaneously and further pilot-tested by 19 patients. Linguistic validations were performed for additional language versions. RESULTS: Initial concepts were developed to cover three areas of interest: 'Treatment', 'Disease and Complications' and 'Information about disease and anticoagulant treatment'. After clinician and patient interviews, concepts were further refined into four domains and 17 concepts; test versions of the PACT-Q were then created simultaneously in three languages, each containing 27 items grouped into four domains: "Treatment Expectations" (7 items), "Convenience" (11 items), "Burden of Disease and Treatment" (2 items) and "Anticoagulant Treatment Satisfaction" (7 items). No item was deleted or added after pilot testing as patients found the PACT-Q easy to understand and appropriate in length in all languages. The PACT-Q was divided into two parts: the first part to measure the expectations and the second to measure the convenience, burden and treatment satisfaction, for evaluation prior to and after anticoagulant treatment, respectively. Eleven additional language versions were linguistically validated. CONCLUSION: The PACT-Q has been rigorously developed and linguistically validated. It is available in 14 languages for use with thromboembolic patients, including AF, PE and DVT patients. Its validation and psychometric properties have been tested and are presented in a separate manuscript.

Comparative effectiveness of avelumab versus chemotherapy in Merkel cell carcinoma: innovative use of patient insights.

AIM: To assess patient experience with chemotherapy and avelumab in metastatic Merkel cell carcinoma (mMCC). METHODS: In the JAVELIN Merkel 200 trial, chemotherapy-refractory mMCC patients could participate in optional qualitative interviews at baseline documenting recollection of previous chemotherapy experience, and at weeks 13/25 documenting current experience with avelumab. Functional Assessment of Cancer Therapy subscale for melanoma questionnaire (FACT-M) was administered in parallel. RESULTS: In our sample, chemotherapy was associated with an unpleasant experience. On selected FACT-M items addressing chemotherapy-impacted concepts, most patients receiving avelumab were improved or stable; few worsened. In addition, a few patients spontaneously reported experiencing less toxicity with avelumab than experienced during previous chemotherapy. CONCLUSION: This approach merging qualitative and quantitative data suggests that mMCC patients report a better experience with avelumab than with chemotherapy.

How to address the challenges of evaluating treatment benefits-risks in rare diseases? A convergent mixed methods approach applied within a Merkel cell carcinoma phase 2 clinical trial.

BACKGROUND: Demonstrating treatment benefits within clinical trials in the context of rare diseases is often methodologically and practically challenging. Mixed methods research offers an approach to overcome these challenges by combining quantitative and qualitative data, thus providing a better understanding of the research question. A convergent mixed methods design in the context of Merkel cell carcinoma, a rare skin cancer, was used during the JAVELIN Merkel 200 trial (NCT02155647). METHODS: Nine patients receiving avelumab in the JAVELIN Merkel 200 trial were interviewed at baseline prior to receiving study treatment, and at 13 weeks and 25 weeks after first avelumab administration. Key concepts of interest identified from the baseline interviews were physical functioning, fatigue/energy, and pain. Patient perceptions of the overall change in their cancer-related health status since starting study treatment were also recorded. During qualitative analysis, at each time-point, each concept of interest was assigned a category describing the trend in change (e.g. newly emerged, no change/stable, improved, worsened, ceased/disappeared). In parallel, patients' tumour status was determined by the clinical overall response status as per the clinical trial protocol. RESULTS: A high concordance between patient-reported qualitative data and assessed tumour response was observed. All eight patients who clinically improved had perceived a subjective improvement in their disease since the beginning of the study; the single patient whose disease worsened had a perceived deterioration. Patient perceived benefit in physical functioning, fatigue/energy and pain was subsequent to the measured change in clinical status as assessed by tumour response. This suggests that patient-reported assessment should be examined over the long term in order to optimally capture meaningful treatment effect. CONCLUSION: Embedding qualitative research in clinical trials to complement the quantitative data is an innovative approach to characterise meaningful treatment effect. This application of mixed methods research has the potential to overcome the hurdles associated with clinical outcomes assessment in rare diseases.

The community-acquired pneumonia symptom questionnaire: a new, patient-based outcome measure to evaluate symptoms in patients with community-acquired pneumonia.

STUDY OBJECTIVE: s: To develop and validate a patient-based outcome measure to evaluate symptoms in patients with community-acquired pneumonia (CAP). DESIGN: A psychometric study within an international, prospective, randomized, double-blind study. The CAP-symptom questionnaire (CAP-Sym) is a new, 18-item, patient-reported outcome measure that evaluates the bothersomeness of CAP-related symptoms during the past 24 h using a 6-point Likert scale. We used "gold standard" psychometric methods to comprehensively evaluate the acceptability, reliability, validity, and responsiveness of the CAP-Sym. SETTING: Sixty-four centers in 13 countries (France, Germany, Hungary, Israel, Italy, Norway, Poland, Portugal, South Africa, Spain, Sweden, Switzerland, United Kingdom). PATIENTS: Five hundred fifty-six patients with CAP, recruited from outpatient clinics, general practice, and hospital centers. INTERVENTIONS: Randomization 1:1 to moxifloxacin (400 mg once daily), oral or standard oral treatment (amoxicillin, 1 g tid, or clarithromycin, 500 mg bid), alone or in combination, for up to 14 days. RESULTS: Standard psychometric tests confirmed the acceptability (item nonresponse, item-endorsement frequencies, item/scale floor and ceiling effects), reliability (internal consistency, item-total and inter-item correlations, test-retest reliability), validity (content, construct, convergent, discriminant, known groups), and responsiveness of the CAP-Sym. CONCLUSIONS: The CAP-Sym is a practical and scientifically sound patient-based outcome measure of CAP-related symptoms that has been developed using "gold standard" methods. As the only fully validated measure of symptoms in patients with CAP, which is quick and easy to administer and is more responsive than the generic Medical Outcomes Study 36-Item Short-Form Health Survey, the CAP-Sym provides a practical and rigorous method for improving the evaluation of outcomes in clinical trials and audit.

Validation of the French version of the Fecal Incontinence Quality-of-Life (FIQL) scale.

INTRODUCTION: The aim of this multicenter study was to validate the French version of the fecal incontinence quality-of-life scale (FIQL scale) developed in the Unites States of America. PATIENTS AND METHODS: The FIQL scale has 29 items in four scales: lifestyle, coping/behavior, depression/self-perception and embarrassment. Each item is scored from 1 to 4, with poorest quality-of-life scored 1. An average is calculated for each scale. After linguistic validation of the questionnaire, the French version of the FIQL scale was tested twice, at day 0 and day 7, by 100 patients with fecal incontinence (FI). Construction validity, internal reliability, clinical validity and reproducibility were analysed. RESULTS: Analysis of convergent validity of the French version of the FIQL scale showed very good correlation between items and the corresponding scale for lifestyle (0.50-0.79) and depression/self-perception (0.44-0.74), good correlation for coping/behavior (0.31-0.70) and weak correlation for embarrassment (0.30-0.40). Valid discrimination was observed for 24 of the 29 items. Internal reliability was good for each scale (alpha Cronbach between 0.78 and 0.92). Scores determined with the FIQL scale were significantly correlated with Wexner FI scores, demonstrating the clinical validity of the instrument. Reproducibility, evaluated in patients whose FI was unchanged between day 0 and day 7, was good with intraclass correlation coefficients ranging from 0.80 (embarrassment) to 0.93 (lifestyle). CONCLUSIONS: The linguistic and psychometric evaluation demonstrated the validity of the French version of the FIQL scale. This standardized instrument is now available for clinical use in France for quality-of-life assessment in patients with FI.

Factors affecting adherence to asthma treatment: patient and physician perspectives.

AIMS: To identify important factors affecting treatment adherence of patients with asthma and to summarise this information as a guide for physicians. METHODS: Information from literature and interviews with 12 respiratory physicians (four each from France, Spain and UK) and 46 asthma patients was obtained. Factors affecting adherence to asthma treatment were identified, reviewed and a flow chart developed to indicate the relationship between key factors. RESULTS: Major factors influencing adherence included: the patient-physician relationship; the patient's understanding of the disease and its treatment; the patient's beliefs and perception of the disease and its treatment, and, importantly, the patient's willingness to take an active part in his/her asthma management. CONCLUSION: Patient adherence to asthma can be improved, and the likelihood of treatment success increased, by paying attention to the factors that influence patients' willingness to participate in their treatment.

Quality of life in patients with stomas: the Montreux Study.

Ostomy surgery profoundly affects a person's life. To determine the extent of the effect, the Stoma Care Quality of Life Index instrument was developed from a quality-of-life index. After ascertaining its validity and reliability, the instrument was used to measure patient quality of life in a European-wide study (16 countries). Six hundred, eighteen (618) stoma care nurses recruited 4,739 patients following stoma surgery. The self-administered questionnaire was completed immediately following surgery and after 3,6, 9, and 12 months. The mean age of patients was 61.6 years (+/- 13.4 years), 53.7% were men, and the majority (66.5%) had a colostomy. Stoma Care Quality of Life Index scores were fairly consistent in all patients throughout Europe immediately following surgery. While scores improved steadily over time, only the difference between the postoperative and 3-month scores was significant (P < 0.001). Stoma Care Quality of Life Index scores were significantly higher in patients who were satisfied with the care received than in those who were not satisfied. Similarly, patients who had a good relationship with the stoma care nurse and felt confident about changing the appliance had significantly higher Stoma Care Quality of Life Index scores than those who did not have a good relationship or feel confident. The results of this study suggest that stoma patient quality of life can be assessed, that it changes over time, and that patient access to specialist ostomy care nurses is particularly important during the first 3 to 6 months following surgery.

Patients' experience and perception of hospital-treated Clostridium difficile infections: a qualitative study.

BACKGROUND: Clostridium difficile is the leading cause of antibiotic-associated diarrhea and an important source of nosocomial infection. Clinical manifestations can range from mild diarrhea to lethal pseudomembranous colitis. Little is known about the burden of C. difficile infections (CDI) in patients. OBJECTIVE: This qualitative study explored the impact of hospital-treated CDI on patients' lives from the first occurrence of CDI symptoms, through their hospital stay, and after discharge. METHODS: Semi-structured interviews with 12 US and 12 French patients who had experienced CDI were conducted using an interview guide that was developed on the basis of a thorough literature review. Transcripts from these interviews were analyzed to identify concepts related to the research question. FINDINGS: CDI affected numerous aspects of patients' lives. Patients reported that the continuous, watery, and uncontrollable diarrhea characteristic of CDI had the most impact on their daily lives. Diarrhea prevented them from participating in usual daily activities; this caused the collapse of their social lives. Patients felt humiliated and embarrassed. Patients' emotional distress worsened once hospitalized; they reported feelings of loneliness and worry when placed in isolation. From discharge to the time of the interview, patients reported both psychological and physical improvement. However, despite continuing improvement, most patients reported persistent worry and fear of recurrent episodes, and they were thus more careful about their diet and hygiene. CONCLUSION: As one patient in this study explained, CDI is "the worst of everything that I've had." The emotional distress and extreme physical exhaustion associated with CDI result in a traumatic and frightening experience for patients. This trauma persists after recovery and includes lingering fears of a recurrent episode.