Applying a mixed methods design to test saturation for qualitative data in health outcomes research

Saturation, a core concept in qualitative research, suggests when data collection might end. It is reached when no new relevant information emerges with additional interviews. The aim of this research was to explore whether a mixed methods design could contribute to the demonstration of saturation. Firstly, saturation was conceptualized mathematically using set theory. Secondly, a conversion mixed design was conducted: a set of codes derived from qualitative interviews were quantitized and analyzed using partial least squares (PLS) regression to document whether saturation was reached. A qualitative study conducted by other researchers prior to this work (i.e. none of the present authors was involved in this study) was used to test saturation using PLS regression. This illustrative qualitative study aimed to investigate the impact of Clostridium difficile infection (CDI) on nurses’ work in the hospital and the results were published elsewhere (Guillemin et al. 2015). Semi-structured interviews were conducted with 12 nurses. Saturation was characterized by the cumulative percentage of variability accounted for by PLS factors. After 12 interviews, this percentage was 51% which suggests that saturation was achieved at least on main themes. Two main themes identifying similarities in the experience of nurses caring for patients with CDI were identified: Organization/Coordination of the working day and Time-consuming work. Although dependent on the coding of qualitative data, PLS regression of quantitized data from qualitative interviews generated useful information for the determination of saturation

comparative effectiveness of avelumab versus chemotherapy in merkel cell carcinoma innovative use of patient insights

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Surgical intervention on uterine fibromyoma in a country with limited resources: case of the gynaecology-obstetrics department of the Communal Medical Centre of Ratoma Conakry - Guinea

Background: In developing countries, treatment of uterine fibromyoma is confronted with numerous problems, namely: financial inaccessibility to the proposed treatments, fear of surgery and the weakness of the technical platform. The objectives of the study were to calculate the frequency of uterine fibromyomas, describe the socio-demographic characteristics of patients, identify the main clinical data and to describe the modalities of surgical management.Methods: It was a mixed descriptive study, cumulative over a period of 5 years (60 months) with data collection in two phases: a 4-year retrospective study from January 1, 2015 to December 31, 2018 and a 1-year prospective study from January 1, 2019 to December 31, 2019.Results: Authors collected 135 cases of uterine fibromyomas operated on out of a total of 260 cases of gynaecological pathologies, i.e. a frequency of 51.92%. Nulliparous women were the most concerned (45.18%), and women who attended school (60%) and those who did not attend school (40%). Women at home and housewives accounted for 42.20% and 54.07% respectively. Clinically, the circumstances of discovery were dominated by menometrorrhagia and menorrhagia respectively 77.77% and 68.14%. The large uterus was the most frequent physical sign found in 96.29% of cases. Uterine fibromyomas were recorded in 86.6% of cases in women with genital activity. The operative indications were dominated by the large polymyomatous uterus (64.44%), followed by hemorrhagic fibroma (18.52%) The surgical treatment was conservative in 92.60%. The total hysterectomy was performed in 7.40. Lethality was 1.4%.Conclusions: The surgical management of fibroids contrasts conservative treatment (myomectomy) with radical treatment (hysterectomy) with multiple possible approaches (hysteroscopy, vaginal surgery, laparoscopy or laparotomy). In this context, only laparotomy was possible due to lack of equipment. Laparoscopy and hysteroscopy equipment are necessary for less invasive surgery

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

Abstract 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

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

Abstract 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

Validation of the Peak Pruritus Numerical Rating Scale as a Patient-Reported Outcome Measure in Prurigo Nodularis

Abstract Introduction Validated patient report tools for quantifying patient experiences of itch in prurigo nodularis (PN) are limited. This study aimed to evaluate the validity of the 11-point peak pruritus numerical rating scale (PP NRS) as a single-item patient-reported outcome (PRO) measure for assessing itch severity in PN. Methods Content validity of the PP NRS was evaluated through qualitative interviews with adults with PN. The PP NRS was then psychometrically evaluated using data from a placebo-controlled trial of nemolizumab in adults with PN, during which patients completed the PP NRS daily. Meaningful within-patient change was estimated from the qualitative interviews and by anchor- and distribution-based analyses of trial data. Results The interview participants (N = 21) all understood the PP NRS and reported itch as their worst symptom overall. The PP NRS showed good test–retest reliability and demonstrated convergent validity and known-groups validity. PP NRS scores improved more in patients classified as “improved” on other clinical outcome measures than in those classified as “worsened/unchanged”. Triangulation of the different estimates identified a 2- to 5-point decrease in PP NRS score as a meaningful within-patient change threshold. Conclusion The PP NRS is a content-valid and reliable PRO measure for quantifying itch severity in adults with PN in clinical trials. Trial Registration Number NCT03181503

Scoring and psychometric validation of the ‘Determinants of Intentions to Vaccinate’ (DIVA©) questionnaire

International audienceBackground: Primary care physicians (PCPs) play a key role regarding vaccination in France. The aims of the present study were to define the scoring rules and to assess the measurement properties of the ‘Determinants of Intentions to Vaccinate’ (DIVA©) questionnaire that aims to assess PCPs’ attitudes and beliefs toward vaccination.Methods: The DIVA questionnaire was derived from a literature review and PCPs focus groups. Scoring and early validation of the DIVA questionnaire were determined during a cross-sectional study conducted in France. During the study, PCPs had to complete the DIVA questionnaire for any of the six vaccine-preventable diseases (VPDs) to which they were randomly assigned (measles, pertussis, pneumococcus infection, seasonal influenza, human papillomavirus -HPV- infection and tetanus). Descriptive analyses of items and the analysis of the grouping of items into domains were conducted. Internal consistency reliability and construct validity was assessed according to each VPD.Results: The DIVA questionnaire was completed by 1,069 PCPs and was well accepted. The ‘Commitment of the PCP to the vaccination approach’ score showed very good internal consistency reliability (Cronbach’s alpha >0.70 overall and for each VPD). The construct validity of the DIVA questionnaire was confirmed.Conclusions: The DIVA questionnaire is a valid and reliable measure of PCPs’ attitudes and beliefs toward vaccination