Toward a core outcome set for faecal incontinence-A systematic review of outcomes reported in the literature.

AIM: Faecal incontinence (FI) is a prevalent issue which can have a detrimental impact on the patient's quality of life. Current international guidelines lack strong treatment recommendations due to few studies in the field, in combination with the heterogeneity in outcome reporting. To address this, a core outcome set (COS) is proposed to standardize outcome reporting in FI studies, facilitating meta-analyses and enhancing therapeutic recommendations. Through several steps outlined by COMET 'what' to measure will be determined prior to determining 'how' to measure these outcomes. This systematic review aims to identify 'what' outcomes have been used in FI intervention studies so far in adult patients as a starting phase for the development of a future COS for FI. METHOD: Medline, Embase and the Cochrane library were searched to identify all outcomes reported in comparative effectiveness trials assessing one or more treatment option in adult patients suffering from FI. The outcomes were categorized according to the Core Outcome Measurement in Effectiveness Trials (COMET) taxonomy to standardize outcome terminology, assess completeness, and inform subsequent steps in COS development. RESULTS: A total of 109 studies were included, which revealed 51 unique outcomes classified into 38 domains within four core areas. On average four outcomes were reported per study (range 1-11). The most commonly reported outcomes were "severity of FI" (83%), "quality of life" (64%), "number of FI episodes" (40%), "anorectal motor function" (39%), and "frequency of bowel movements" (16%). CONCLUSION: This systematic review offers an overview of outcomes reported in FI studies, highlighting the heterogeneity between studies. This heterogeneity emphasizes the need for standardizing outcome reporting which can be established through the creation of a COS.

The N-LVA Study: effectiveness and cost-effectiveness of lymphaticovenous anastomosis (LVA) for patients with cancer who suffer from chronic peripheral lymphoedema - study protocol of a multicentre, randomised sham-controlled trial.

INTRODUCTION: Cancer-related lymphoedema is one of the most debilitating side-effects of cancer treatment with an overall incidence of 15.5%. Patients may suffer from a variety of symptoms, possibly resulting in a diminished health-related quality of life (HRQoL). A microsurgical technique known as lymphaticovenous anastomosis (LVA) might be a promising treatment option. The objective of this study is to evaluate whether LVA is effective and cost-effective compared with sham surgery in improving the HRQoL. METHODS AND ANALYSIS: A multicentre, double-blind, randomised sham-controlled trial conducted in three university hospitals in the Netherlands. The study population comprises 110 patients over the age of 18 years with unilateral, peripheral cancer-related lymphoedema, including 70 patients with upper limb lymphoedema and 40 patients with lower limb lymphoedema. A total of 55 patients will undergo the LVA operation, while the remaining 55 will undergo sham surgery. The follow-up will be at least 24 months. Patients are encouraged to complete the follow-up by explaining the importance of the study. Furthermore, patients may benefit from regular monitoring moments for their lymphoedema. The primary outcome is the HRQoL. The secondary outcomes are the limb circumference, excess limb volume, changes in conservative therapy, postoperative complications, patency of the LVA and incremental cost-effectiveness. ETHICS AND DISSEMINATION: The study was approved by the Medical Ethical Committee of Maastricht University Medical Center on 20 September 2023 (NL84169.068.23). The results will be presented at scientific conferences and published in peer-reviewed medical journals. TRIAL REGISTRATION NUMBER: NCT06082349.

Laparoscopic Versus Open Hemihepatectomy: The ORANGE II PLUS Multicenter Randomized Controlled Trial.

PURPOSE: To compare outcomes after laparoscopic versus open major liver resection (hemihepatectomy) mainly for primary or metastatic cancer. The primary outcome measure was time to functional recovery. Secondary outcomes included morbidity, quality of life (QoL), and for those with cancer, resection margin status and time to adjuvant systemic therapy. PATIENTS AND METHODS: This was a multicenter, randomized controlled, patient-blinded, superiority trial on adult patients undergoing hemihepatectomy. Patients were recruited from 16 hospitals in Europe between November 2013 and December 2018. RESULTS: Of the 352 randomly assigned patients, 332 patients (94.3%) underwent surgery (laparoscopic, n = 166 and open, n = 166) and comprised the analysis population. The median time to functional recovery was 4 days (IQR, 3-5; range, 1-30) for laparoscopic hemihepatectomy versus 5 days (IQR, 4-6; range, 1-33) for open hemihepatectomy (difference, -17.5% [96% CI, -25.6 to -8.4]; P < .001). There was no difference in major complications (laparoscopic 24/166 [14.5%] v open 28/166 [16.9%]; odds ratio [OR], 0.84; P = .58). Regarding QoL, both global health status (difference, 3.2 points; P < .001) and body image (difference, 0.9 points; P < .001) scored significantly higher in the laparoscopic group. For the 281 (84.6%) patients with cancer, R0 resection margin status was similar (laparoscopic 106 [77.9%] v open 122 patients [84.1%], OR, 0.60; P = .14) with a shorter time to adjuvant systemic therapy in the laparoscopic group (46.5 days v 62.8 days, hazard ratio, 2.20; P = .009). CONCLUSION: Among patients undergoing hemihepatectomy, the laparoscopic approach resulted in a shorter time to functional recovery compared with open surgery. In addition, it was associated with a better QoL, and in patients with cancer, a shorter time to adjuvant systemic therapy with no adverse impact on cancer outcomes observed.

The Assessment of Burden of ColoRectal Cancer (ABCRC)-tool; a validity and reliability study.

INTRODUCTION: Follow-up care after treatment for colorectal cancer (CRC) is increasingly focused on health-related quality of life (HRQoL) and functional outcomes. The Assessment of Burden of ColoRectal Cancer (ABCRC)-tool is developed to measure these outcomes and support patient-oriented care. The tool comprises items assessing burden of disease and lifestyle parameters. It consists of a generic module combined with one of the three CRC specific modules. The objective of this study is to assess the construct validity and reliability of the items of the ABCRC-tool. METHODS: Patients who were receiving follow-up care after surgical CRC treatment were invited to complete the ABCRC-tool together with other validated patient-reported outcome measures (PROMs). Construct validity was assessed by testing expected correlations between items of the ABCRC-tool and domains of other PROMs and by examining predefined hypotheses regarding differences in subgroups of patients. Patients completed the ABCRC-tool twice, with 8 days apart, to evaluate its reliability. RESULTS: In total, 177 patients participated (64% male) with a mean age of 67 years (range 33-88). The colon, rectum and stoma module were completed by subsequently 89, 53 and 35 patients. Most items correlated as expected with anticipated domains of the EORTC QLQ-C30 or EORTC QLQ-CR29 (all p-values <0.05). Furthermore, the ABCRC-tool could discriminate between subgroups of patients. The intraclass correlation coefficient (ICC) was good (>0.70) for most items, indicating good reliability. CONCLUSION: The ABCRC-tool is a valid and reliable instrument that is ready for use in a clinical setting to support personalized follow-up care after CRC treatment.

Cooled versus conventional radiofrequency treatment of the genicular nerves for chronic knee pain: 12-month and cost-effectiveness results from the multicenter COCOGEN trial.

BACKGROUND: Radiofrequency (RF) treatment of the genicular nerves reduces chronic knee pain in patients with osteoarthritis (OA) or persistent postsurgical pain (PPSP) after total knee arthroplasty (TKA). The objective of this study is to compare long-term outcomes of cooled and conventional RF and perform an economic evaluation. METHODS: The COCOGEN trial is a double-blinded, non-inferiority, pilot, randomized controlled trial that compared the effects up to 12 months of cooled and conventional RF in patients with chronic knee pain suffering from OA or PPSP after TKA following a 1:1 randomization rate. Outcomes were knee pain, functionality, quality of life, emotional health, medication use, and adverse events. A trial-based economic evaluation was performed with a 12-month societal perspective. Here, the primary outcome was the incremental costs per quality-adjusted life year (QALY). RESULTS: 41 of the 49 included patients completed the 12-month follow-up. One patient in the PPSP cooled RF group had substantial missing data at 12-month follow-up. The proportion of patients with ≥50% pain reduction at 12 months was 22.2% (4/18) in patients treated with conventional RF versus 22.7% (5/22) in patients treated with cooled RF (p>0.05). There was a statistically significant difference in the mean absolute numerical rating scale at 12 months after cooled RF and conventional RF in patients with PPSP (p=0.02). Differences between other outcomes were not statistically significant. The health economic analysis indicated that cooled RF resulted in lower costs and improved QALYs compared with conventional RF in PPSP but not in OA. There were no serious adverse events. CONCLUSIONS: Both RF treatments demonstrated in approximately 22% of patients a ≥50% pain reduction at 12 months. In patients with PPSP, contrary to OA, cooled RF seems to be more effective than conventional RF. Additionally, cooled RF has in patients with PPSP, as opposed to OA, greater effectiveness at lower costs compared with conventional RF. TRIAL REGISTRATION NUMBER: NCT03865849.

Construct validity and reliability of the Bilateral Vestibulopathy Questionnaire (BVQ).

Background: The Bilateral Vestibulopathy Questionnaire (BVQ) is a recently developed 54-item Patient Reported Outcome Measure (PROM) that evaluates the clinically important symptoms of bilateral vestibulopathy (BVP) and its impact on daily life. This study aimed to assess the construct validity and reliability of the BVQ in a large BVP cohort. Methods: Patients diagnosed with BVP were asked to complete a set of questionnaires, including the BVQ, the EuroQol-5D-5L, the Health Utilities Index, the Dizziness Handicap Inventory, the Hospital Anxiety and Depression Scale, and the Oscillopsia Severity Questionnaire. The construct validity of the BVQ was evaluated by confirmatory and exploratory factor analyses (CFA and EFA), followed by hypotheses testing and known groups validity. Structural properties were explored for each individual item. Reliability was assessed by testing the internal consistency of the BVQ constructs (Cronbach's alpha) and test-retest reliability [intraclass correlation coefficients (ICCs)]. Results: A total of 148 patients with BVP (50% women, mean age 66 years) completed the set of questionnaires. The CFA did not show a satisfactory model in the original BVQ. However, the EFA showed a four-factor solution with 20 Likert-scale items related to oscillopsia, imbalance, emotion, and cognition. The succeeding CFA provided evidence for construct validity and an acceptable model of fit. Hypothesis testing confirmed that this shortened version validly measures the constructs to be measured. Statistically significant differences in scores between known groups were found, providing further support for good construct validity. The structural properties were acceptable. Cronbach's alpha confirmed good internal consistency for the four constructs, ranging from 0.80 to 0.89. The ICCs of the 20 Likert-scale items and four visual analog scale (VAS) items were interpreted as good (range 0.76-0.93). Conclusion: This study showed evidence of good construct validity of the new shortened version of the BVQ, consisting of four constructs with a total of 20 Likert-scale items and four VAS items. The final 24-item BVQ proved to be a reliable and valid multi-item PROM that captures the clinically important symptoms of BVP and evaluates its impact on daily life. Consequently, the BVQ enables the gathering of high-level evidence of treatment effectiveness in a systematic and quantitative manner.

Comparison of conventional and cooled radiofrequency treatment of the genicular nerves versus sham procedure for patients with chronic knee pain: protocol for a multicentre, double-blind, randomised controlled trial (COGENIUS).

INTRODUCTION: The prevalence of chronic knee pain is increasing. Osteoarthritis (OA) and persistent postsurgical pain (PPSP) are two important causes of knee pain. Chronic knee pain is primarily treated with medications, physiotherapy, life-style changes and intra-articular infiltrations. A radiofrequency treatment (RF) of the genicular nerves is a therapeutical option for refractory knee pain. This study investigates the effectiveness and cost-effectiveness of conventional and cooled RF in patients suffering from chronic, therapy resistant, moderate to severe knee pain due to OA and PPSP. METHODS AND ANALYSIS: The COGENIUS trial is a double-blinded, randomised controlled trial with 2-year follow-up. Patients and outcome assessors are blinded. Patients will be recruited and treated in Belgium and the Netherlands. All PPSP after a total knee prothesis and OA patients (grades 2-4) will undergo a run-in period of 1-3 months where conservative treatment will be optimised. After the run-in period, 200 patient per group will be randomised to conventional RF, cooled RF or a sham procedure following a 2:2:1 ratio. The analysis will include a comparison of the effectiveness of each RF treatment with the sham procedure and secondarily between conventional and cooled RF. All comparisons will be made for each indication separately. The primary outcome is the Western Ontario and McMaster Universities Osteoarthritis Index score at 6 months. Other outcomes include knee pain, physical functionality, health-related quality of life, emotional health, medication use, healthcare and societal cost and adverse events up to 24 months postintervention. ETHICS AND DISSEMINATION: Ethics approval was obtained from the Ethics Committee of the University of Antwerp (Number Project ID 3069-Edge 002190-BUN B3002022000025), the Ethics committee of Maastricht University (Number NL80503.068.22-METC22-023) and the Ethics committee of all participating hospitals. Results of the study will be published in international peer-reviewed journals. TRIAL REGISTRATION NUMBER: NCT05407610.

Responsiveness of the Patient-Reported Outcome Measure-Haemorrhoidal Impact and Satisfaction Score in patients with haemorrhoidal disease.

AIM: In this study we aimed to assess the responsiveness of the symptom score of the recently developed Patient-Reported Outcome Measure-Haemorrhoidal Impact and Satisfaction Score (PROM-HISS). Furthermore, the minimally relevant difference (MRD) was determined. METHOD: The responsiveness of PROM-HISS was tested using a criterion-based (i.e. anchor) and construct-based (i.e. hypotheses testing) approach. Patients with haemorrhoidal disease (HD) completed the PROM-HISS before and 1 week after treatment in hospital. A global self-assessment of change question (SCQ) was administered 1-week after treatment and functioned as the criterion. The following analyses were performed: (1) correlation between the PROM-HISS symptom score and the criterion (SCQ) and (2) hypotheses testing. The MRD was determined as change in symptoms of the subgroup reporting 'somewhat fewer complaints' on the SCQ. RESULTS: Between February and August 2022, 94 patients with grade II-IV HD from three hospitals were included. The correlation between the SCQ and a change on the PROM-HISS symptom score was 0.595 indicating that an improvement on the SCQ corresponds to an improvement on the PROM-HISS symptom score. As hypothesized, the mean change in PROM-HISS scores was significantly different between subgroups of patients based on their SCQ responses. Patients reporting a small change in HD symptoms on the SCQ corresponded to a mean change of 0.3 on the PROM-HISS symptom score. CONCLUSION: The PROM-HISS symptom score is a responsive instrument as it identifies change in HD symptoms because of treatment. The estimated MRD of 0.3 can be used to inform clinical research and practice.

English translation and cross-cultural validation of the patient-reported outcome measurement-haemorrhoidal impact and satisfaction score (PROM-HISS).

AIM: The aim of this study was to translate the Dutch patient-reported outcome measure-haemorrhoidal impact and satisfaction score (PROM-HISS) to English and perform a cross-cultural validation. METHOD: The ISPOR good practice guidelines for the cross-cultural validation of PROMs were followed and included two steps: (1) Two forward and two backward translations. The forward translation concerned the translation from the source language (Dutch) to the target language (English), performed by two independent English speakers, one medical doctor and one nonmedical. Subsequently, a discussion about discrepancies in the reconciled version was performed by a stakeholder group. (2) Cognitive interviews were held with patients with haemorrhoidal disease (HD), probing the comprehensibility and comprehensiveness of the PROM-HISS. RESULTS: Discrepancies in the reconciled forward translation concerned the terminology of HD symptoms. Furthermore, special attention was paid to the response options, ranging from "not at all", indicating minor symptoms, to "a lot", implying many symptoms. Consensus among the stakeholder group about the final version of the translated PROM-HISS was reached. Interviews were conducted with 10 native English-speaking HD patients (30% female), with a mean age of 44 years (24-83) and primarily diagnosed with grade II HD (80%). The mean time to complete the PROM-HISS was 1 min 43 s. Patients showed a good understanding of the questions and response options, found all items relevant and did not miss important symptoms or topics. CONCLUSION: The translated English language PROM-HISS is a valid tool to assess symptoms of HD, its impact on daily activities and patient satisfaction with HD treatment.

Protocol for the development of a core outcome set for the optimisation of treatment and follow-up of patients with an anorectal malformation (ARM): The ARM and OUtcome Review (ARMOUR)-project.

BACKGROUND: Primary treatment of an anorectal malformation (ARM) is surgical restoration of the anatomy. These children can experience many problems later in life; therefore, a long-term follow-up by an experienced team is needed. The aim of the ARM and OUtcome Review (ARMOUR-study) is to identify the lifetime outcomes that are important from a medical and patients' perspective and develop a core outcome set (COS) that can be implemented in a care pathway to support individual ARM management decisions. METHODS: First, a systematic review will identify clinical and patient-reported outcomes described in studies conducted in patients with an ARM. Second, qualitative interviews with patients of different age categories and their caregivers will be held to ensure that the COS will include outcomes that are relevant from the patient's perspective. Finally, the outcomes will be taken forward to a Delphi consensus exercise. Using multiple web-based Delphi rounds, key stakeholders (medical experts, clinical researchers and patients) will prioritise outcomes. During a face-to-face consensus meeting, the final COS will be determined. These outcomes can be evaluated in a life-long care pathway for patients with ARM. DISCUSSION: The development of a COS for ARMs aims to reduce heterogeneity in outcome reporting between (clinical) studies, enhancing the availability of comparable data, which will facilitate evidence-based patient care. Assessment of the outcomes in the COS during individual care pathways for ARM can support shared decisions regarding management. The ARMOUR-project has ethical approval and is registered with the Core Outcome Measures in Effectiveness Trials (COMET) initiative. LEVEL OF EVIDENCE: Treatment study level II.

Filgotinib for Treating Moderately to Severely Active Ulcerative Colitis: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

The National Institute for Health and Care Excellence invited the manufacturer (Galapagos) of filgotinib (Jyseleca®), as part of the Single Technology Appraisal process, to submit evidence for the clinical effectiveness and cost effectiveness of filgotinib for treating moderately to severely active ulcerative colitis in adults who have had an inadequate response, loss of response or were intolerant to a previous biologic agent or conventional therapy. Kleijnen Systematic Reviews Ltd, in collaboration with Maastricht University Medical Centre+, was commissioned to act as the independent Evidence Review Group. This paper summarises the company submission, presents the Evidence Review Group's critical review on the clinical and cost-effectiveness evidence in the company submission, highlights the key methodological considerations and describes the development of the National Institute for Health and Care Excellence guidance by the Appraisal Committee. The company submission included one relevant study for the comparison of filgotinib versus placebo: the SELECTION trial. As there was no head-to-head evidence with any of the comparators, the company performed two separate network meta-analyses, one for the biologic-naïve population and one for the biologic-experienced population, and for both the induction and maintenance phases. The Evidence Review Group questioned the validity of the maintenance network meta-analysis because it assumed all active treatments to be comparators in this phase, which is not in line with clinical practice. The economic analysis used a number of assumptions that introduced substantial uncertainty, which could not be fully explored, for instance, the assumption that a risk of loss of response would be independent of health state and constant over time. Company and Evidence Review Group results indicate that at its current price, and disregarding confidential discounts for comparators and subsequent treatments, filgotinib dominates some comparators (golimumab and adalimumab in the company base case, all but intravenous and subcutaneous vedolizumab in the Evidence Review Group's base case) in the biologic-naïve population. In the biologic-experienced population, filgotinib dominates all comparators in both the company and the Evidence Review Group's base case. Results should be interpreted with caution as some important uncertainties were not included in the modelling. These uncertainties were mostly centred around the maintenance network meta-analysis, loss of response, health-related quality-of-life estimates and modelling of dose escalation. The National Institute for Health and Care Excellence recommended filgotinib within its marketing authorisation, as an option for treating moderately to severely active ulcerative colitis in adults when conventional or biological treatment cannot be tolerated, or if the disease has not responded well enough or has stopped responding to these treatments, and if the company provides filgotinib according to the commercial arrangement.

Picking Up the Threads: Long-Term Outcomes of the Sutured Haemorrhoidopexy: A Retrospective Single-Centre Cohort Study.

BACKGROUND: This study aimed to assess the short- and long-term safety and efficacy of the sutured haemorrhoidopexy (SH) in patients with haemorrhoidal disease (HD). METHODS: A retrospective study was performed, assessing the following treatment characteristics: number of sutures needed; operation time; perioperative complications; postoperative pain; hospital stay. The short- and long-term postoperative complications, HD recurrence and data on current HD symptoms were assessed according to the Core Outcome Set for HD. RESULTS: Between January 2009 and December 2021, 149 patients with HD underwent a SH. One-hundred and forty-five patients were included, with a mean age of 61 years (±12.8), of which 70 were women (48.3%). Patients were predominantly diagnosed with grade III (37.2%) HD and the median follow-up was nine years (5-11). Perioperative complications occurred in four cases (2.8%). In two patients (1.4%), short-term postoperative complications were reported, and in seven patients (6.2%), long-term complications were reported. The cumulative efficacy in terms of freedom of recurrence was 88.3% (95% CI, 83.1-93.5) at six months, 80.0% (95% CI, 73.5-86.5) at one year, and 67.7% (95% CI, 59.7-75.7) at five years. CONCLUSIONS: Sutured haemorrhoidopexy is a safe treatment for patients with HD and can be proposed as a minimally invasive surgical treatment if basic and outpatient procedures fail.

The effectiveness of health education interventions on cervical cancer prevention in Africa: A systematic review.

Low levels of knowledge and awareness on cervical cancer play a role in limiting cervical cancer prevention uptake. This systematic review aimed to identify effective educational interventions to increase cervical cancer awareness, knowledge, and subsequently screening or vaccination uptake in African women. A literature search was conducted in Medline and EMBASE databases. We examined original, peer-reviewed English literature published between 2005 and 2020. Nineteen studies examining health education interventions' impact on awareness, knowledge, and screening or vaccination uptake in African women were included. Ten studies were controlled trials, nine performed pre- and post-measurements in one group. Most studies were published between 2015 and 2020 (86%), many were from Nigeria (47%). Studies were mostly set up in communities and schools. The most frequently used intervention was lectures, alone or combined with videos and practical demonstrations. Sixteen studies evaluated knowledge or awareness, and all showed a statistically significant improvement following the intervention. Of the ten studies that evaluated screening uptake, either as the single outcome or combined with knowledge or awareness, six found a significant rise in screening uptake after intervention. Educational interventions increased knowledge and awareness in African women, some boosted uptake of cervical cancer screening, especially when using peer health educators and culturally tailored methods. Innovative approaches such as self-collected HPV testing and mHealth also demonstrated a potential to increase uptake of screening. More research is needed to identify and analyse barriers to screening uptake, which can still be present even after a successful educational intervention.

The patient perspective on the preoperative colorectal cancer care pathway and preparedness for surgery and postoperative recovery-a qualitative interview study.

BACKGROUND AND OBJECTIVES: This study aimed to explore colorectal cancer (CRC) patients' perspectives and experiences regarding the preoperative surgical care pathway and their subsequent preparedness for surgery and postoperative recovery. METHODS: CRC patients were recruited using purposive sampling and were interviewed three times (preoperatively, and 6 weeks and 3 months postoperatively) using semistructured telephone interviews. Interviews were audiotaped, transcribed verbatim and analysed independently by two researchers using thematic analysis with open coding. RESULTS: Data saturation was achieved after including 18 patients. Preoperative factors that contributed to a feeling of preparedness for surgery and recovery were patient-centred- and professional healthcare organization, sincere and personal guidance, and thorough information provision. Postoperatively, patients with complications or physical complaints experienced unmet information needs regarding the impact of complications and what to expect from postoperative recovery. CONCLUSIONS: The preoperative period is a vital period to prepare patients for surgery and recovery in which patients most value personalized information, personal guidance and professionalism. According to CRC patients, the feeling of preparedness for surgery and recovery can be improved by continually providing dosed information. This information should provide the patient with patient-tailored perspectives regarding the impact of (potential) complications and what to expect during recovery.

Development and content validation of the Assessment of Burden of ColoRectal Cancer (ABCRC)-tool.

BACKGROUND: It is essential to include patient-reported outcomes (PROs), such as health-related quality of life and symptom burden, in follow-up care of colorectal cancer (CRC) survivors. These outcomes are most valuable when they are discussed with patients and used to guide follow-up care. The purpose of this study was to develop and validate the Assessment of Burden of Colorectal cancer (ABCRC)-tool: a tool that includes a patient-reported questionnaire covering the experienced burden of colorectal cancer, assessment of lifestyle parameters, visualisation of the results, and treatment advice. METHODS: A 5-step method was used to develop the ABCRC-tool: (1) definition of the experienced burden of CRC, (2) determination of the components of experienced burden, (3) formulation of the instrument preconditions, (4) literature study on existing instruments, (5) development of an integrated instrument. Content validity was evaluated by think-aloud interviews with 11 patients and 22 healthcare professionals (HCPs). RESULTS: The ABCRC-tool consists of a generic module and three CRC specific modules with items related to experienced disease burden, and lifestyle. The CRC specific modules are available for both colon and rectal cancer patients with anastomosis, and patients with a stoma. An algorithm with cut-off points was developed to visualise outcomes and offer treatment advice based on (inter)national guidelines. The evaluation of content led to a few minor amendments. CONCLUSION: The ABCRC-tool is a product of close cooperation between patients and HCPs and has good face and content validity. It is aimed to incorporate PROs in treatment decisions in oncological care.

Development and Content Validity of the Bilateral Vestibulopathy Questionnaire.

Background: To date, the burden and severity of the full spectrum of bilateral vestibulopathy (BVP) symptoms has not yet been measured in a standardized manner. Since therapeutic interventions aiming to improve BVP symptoms are emerging, the need for a new standardized assessment tool that encompasses the specific aspects of BVP arises. Therefore, the aim of this study was to develop a multi-item Patient Reported Outcome Measure (PROM) that captures the clinically important symptoms of BVP and assesses its impact on daily life. Methods: The development of the Bilateral Vestibulopathy Questionnaire (BVQ) consisted of two phases: (I) initial item generation and (II) face and content validity testing. Items were derived from a literature review and individual semi-structured interviews focusing on the full spectrum of reported BVP symptoms (I). Subsequently (IIa), individual patient interviews were conducted using "thinking aloud" and concurrent verbal probing techniques to assess the comprehensibility of the instructions, questions and response options, and the relevance, missing domains, or missing items. Interviews continued until saturation of input was reached. Finally, international experts with experience in the field of the physical, emotional, and cognitive symptoms of BVP participated in an online focus group to assess the relevance and comprehensiveness of the BVQ (IIb). Results: The BVQ consisted of two sections. The first section included 50 items scored on a six-point Likert scale arranged into seven constructs (i.e., imbalance, oscillopsia, other physical symptoms, cognitive symptoms, emotional symptoms, limitations and behavioral changes and social life). The second section consisted of four items, scored on a visual analog scale from 0 to 100, to inquire about limitations in daily life, perceived health and expectations regarding future recovery. Interviews with BVP patients [n = 8, 50% female, mean age 56 years (range 24-88 years)] and the expert meeting confirmed face and content validity of the developed BVQ. Conclusion: The BVQ, which was developed to assess the spectrum of BVP symptoms and its impact on daily life, proved to have good face and content validity. It can be used to characterize current self-reported symptoms and disability and to evaluate symptom burden before and after therapeutic interventions in future research and clinical practice.

Patient reported outcome measure-haemorrhoidal impact and satisfaction score (PROM-HISS): Development, reliability and construct validity.

AIM: Haemorrhoidal disease (HD) is a frequently occurring disorder with a significant negative impact on a patient's quality of life. Here, we describe the development and validation of the Dutch patient reported outcome measure-haemorrhoidal impact and satisfaction score (PROM-HISS). METHODS: The development of the PROM-HISS followed recommended guidelines. Face and content validity, structural properties, reliability and construct validity were evaluated in a HD population. Reliability was tested by assessing the test-retest reliability, defined by the intraclass correlation coefficient (ICC), and internal consistency measured with Cronbach's alpha. Construct validity was evaluated using confirmatory factor analysis (CFA) and hypotheses testing. RESULTS: The PROM-HISS consists of three domains: (1) HD symptoms (blood loss; pain; prolapse; soiling; itching), (2) impact of symptoms on daily activities, and (3) satisfaction with treatment. The PROM-HISS showed good face and content validity. The PROM-HISS was completed by 102 patients (65% male), with a mean age of 58 years (23-81 years). The ICCs of the different items in the domain HD symptoms ranged between 0.56 and 0.79 and were interpreted as good. The Cronbach's alpha value was 0.80 and considered satisfactory. The CFA provided further evidence for construct validity with a good model fit. A high score on the symptoms of HD correlated with a high impact of HD on daily activities (Pearson's r = 0.632, p < 0.01) and a low degree of satisfaction (Pearson's r = 0.378, p < 0.01). CONCLUSION: The PROM-HISS is a reliable and valid instrument to evaluate symptoms of HD, impact on daily activities and satisfaction with treatment.

Living with cryptoglandular anal fistula: a qualitative investigation of the patient's experience through semi-structured patient interviews.

PURPOSE: Cryptoglandular anal fistula continues to be a subject of extensive surgical research due to the lack of effective and enduring treatments, some of which incur risks to continence and quality of life. However, the patient experience of disease has seldom been reported. The aims of this study are to understand the impact of living with a fistula and the treatment outcomes that are valued by patients. METHODS: Patients with cryptoglandular anal fistula were recruited using purposive sampling from two tertiary referral centres in the UK and the Netherlands. Patients underwent semi-structured interviews that were audio-recorded and transcribed verbatim. Dutch transcripts were translated into English and underwent independent, thematic analysis using open coding by two study team members to identify common themes and sub-themes. RESULTS: Twenty interviews were conducted before saturation was reached (11 male, median age 49, Interquartile range 39-55 years). Four broad themes emerged, covering the physical symptoms of fistula, the patient journey towards understanding the condition, life impact, and treatment. Several inter-related sub-themes were found, reflecting the extensive impact and adjustment that the disease entails. CONCLUSION: The impact of cryptoglandular anal fistula extends beyond the physical symptoms of pain and discharge, requires significant readjustment, and often negatively impacts psycho-social wellbeing. These aspects of disease should receive greater attention in future assessment of treatment and quality of life.

Out-of-pocket payments for complementary medicine following cancer and the effect on financial outcomes in middle-income countries in southeast Asia: a prospective cohort study.

BACKGROUND: Complementary medicine, which refers to therapies that are not part of conventional medicine, comprising both evidence-based and non-evidence-based interventions, is increasingly used following a diagnosis of cancer. We aimed to investigate out-of-pocket spending patterns on complementary medicine and its association with adverse financial outcomes following cancer in middle-income countries in southeast Asia. METHODS: In this prospective cohort study, data on newly diagnosed patients with cancer were derived from the ASEAN Costs in Oncology (ACTION) cohort study, a prospective longitudinal study in 47 centres located in eight countries in southeast Asia. The ACTION study measured household expenditures on complementary medicine in the immediate year after cancer diagnosis. Participants were given cost diaries at baseline to record illness-related payments that were directly incurred and not reimbursed by insurance over the 12-month period after study recruitment. We assessed incidence of financial catastrophe (out-of-pocket cancer-related costs ≥30% of annual household income), medical impoverishment (reduction in annual household income to below poverty line following subtraction of out-of-pocket cancer-related costs), and economic hardship (inability to make necessary household payments) at 1 year. FINDINGS: Between March, 2012, and September, 2013, 9513 participants were recruited into the ACTION cohort study, of whom 4754 (50·0%) participants were included in this analysis. Out-of-pocket expenditures on complementary medicine were reported by 1233 households. These payments constituted 8·6% of the annual total out-of-pocket health costs in lower-middle-income countries and 42·9% in upper-middle-income countries. Expenditures on complementary medicine significantly increased risks of financial catastrophe (adjusted odds ratio 1·52 [95% CI 1·23-1·88]) and medical impoverishment (1·75 [1·36-2·24]) at 12 months in upper-middle-income countries only. However, the risks were significantly higher for economically disadvantaged households, irrespective of country income group. INTERPRETATION: Integration of evidence-supported complementary therapies into mainstream cancer care, along with interventions to address use of non-evidence-based complementary medicine, might help alleviate any associated adverse financial impacts. FUNDING: None.