Economic evaluation of flap fixation techniques after mastectomy: Results of a double-blind randomized controlled trial (SAM-trial).

BACKGROUND: An economic evaluation was performed alongside an RCT investigating flap fixation in reducing seroma formation after mastectomy. The evaluation focused on the first year following mastectomy and assessed cost-effectiveness from a health care and societal perspective. METHODS: The economic evaluation was conducted between 2014 and 2018 in four Dutch breast clinics. Patients with an indication for mastectomy or modified radical mastectomy were randomly assigned to: conventional closure (CON), flap fixation with sutures (FFS) or flap fixation with tissue glue (FFG). Health care costs, patient and family costs and costs due to productivity losses were assessed. Outcomes were expressed in incremental cost-effectiveness ratios (ICERs): the incremental cost per quality-adjusted life year (QALY). Bootstrapping techniques, sensitivity and secondary analyses were employed to address uncertainty. RESULTS: The FFS-group yielded most QALYs (0.810; 95%-CI 0.755-0.856), but also incurred the highest mean costs at twelve months (€10.416; 95%-CI 8.231-12.930). CON was the next best alternative with 0.794 QALYs (95%-CI 0.733-0.841) and mean annual costs of €10.051 (95%-CI 8.255-12.044). FFG incurred fewer QALYs and higher costs, when compared to the CON group. The ICER of FFS compared to CON was €22.813/QALY. Applying a willingness to pay threshold in the Netherlands of €20.000/QALY, the probability that FFS was cost-effective was 42%, compared to 37% and 21% for CON and FFG, respectively. CONCLUSION: The cost-effectiveness of FFS following mastectomy, versus CON and FFG, is uncertain from a societal perspective. Yet, from a health care and hospital perspective FFS is likely to be the most cost-effective intervention.

Heterogeneity in outcome selection, definition and measurement in studies assessing the treatment of cryptoglandular anal fistula: findings from a systematic review.

BACKGROUND: Treatment for cryptoglandular anal fistula (AF) is challenging and a lack of uniform outcomes in the literature prevents direct comparison of treatments. This can be addressed by developing a core outcome set, a standardised set of outcomes reported in all interventional studies for a specific condition. The aim of this systematic review is to assess the range of outcomes, their definitions, and the measurement instruments currently utilised in interventional studies for adult patients with AF. This will inform the development of an AF core outcome set. METHODS: Medline, Embase and The Cochrane Library were searched to identify all patient- and clinician-reported outcomes in studies assessing medical, surgical or combination treatment of adult patients with AF published from January 2008 to May 2020. The resulting outcomes were categorized according to the Core Outcome Measurement in Effectiveness Trials (COMET) taxonomy to better understand their distribution. RESULTS: In total, 155 studies were included, 552 outcomes were extracted, with a median of three outcomes (interquartile range 2-5) per study. Only 25% of studies demonstrated high-quality outcome reporting. The outcomes were merged into 52 unique outcomes and structured into four core areas and 14 domains, with the majority in the domain of physiological or clinical (gastrointestinal) outcomes. The most commonly reported outcomes were healing (77%), incontinence (63%), and recurrence (40%), with no single outcome assessed across all studies. There was a wide variation in outcome definitions and measurement instruments used. CONCLUSIONS: There is substantial heterogeneity in outcomes, definitions, and measurement instruments reported in interventional studies for cryptoglandular anal fistula. This emphasises the need for standardised outcome reporting and measurement.

Patient-reported outcome measures in oncology: a qualitative study of the healthcare professional's perspective.

BACKGROUND: In the last decades, the number of cancer survivors has increased significantly due to improved treatment and better detection of recurrence. This increased survival redirects the scope from survival towards optimising functional outcomes and improving health-related quality of life (HRQol). Functional and HRQoL outcomes can be assessed with patient-reported outcome measures (PROMs). However, the use of PROMs in daily oncological care is not common. This qualitative study investigates the barriers and facilitators of PROM use in an oncological setting, from the perspective of the healthcare professionals (HCPs). METHODS: Individual semi-structured interviews were conducted among Dutch oncological HCPs. Barriers and facilitators of PROM implementation were identified on various levels of the healthcare system (i.e. level of the patient, individual professional, medical team, and healthcare organisation). Interviews were audio recorded and transcribed verbatim. Transcripts were manually analysed by two independent reviewers using a thematic approach. Identified barriers and facilitators were categorised into Grol and Wensing's framework for changing healthcare practice. RESULTS: Nineteen oncological HCPs working in academic and non-academic hospitals were interviewed. Barriers for PROM implementation were lack of good IT support, lack of knowledge on how to use PROMs, lack of time to complete and interpret PROMs, and a high administrative burden. PROM implementation can be facilitated by providing clear guidance regarding PROM interpretation, evidence that PROMs can save time, and stimulating multidisciplinary teamwork. CONCLUSION: From a HCP point of view, adequately functioning IT technology, sufficient knowledge on PROMs, and dedicated time during the consultation are essential for successful implementation of PROMs in oncological care. Additional local context-specific factors need to be thoroughly addressed.

Patient expectations, experiences and satisfaction with nintedanib and pirfenidone in idiopathic pulmonary fibrosis: a quantitative study.

BACKGROUND: Two antifibrotic drugs, nintedanib and pirfenidone, are available for treatment of idiopathic pulmonary fibrosis (IPF). Although efficacy and adverse events have been well studied, little is known about patient experiences with these drugs. We aimed to systematically and quantitatively evaluate patient expectations, experiences, and satisfaction with nintedanib and pirfenidone. Furthermore, we assessed which factors were associated with overall patient satisfaction with medication. METHODS: Outpatients with IPF prospectively completed the Patient Experiences and Satisfaction with Medication (PESaM) questionnaire before start, and after three and 6 months of antifibrotic treatment, as part of a randomized eHealth trial (NCT03420235). The PESaM questionnaire consists of an expectation module, a validated generic module evaluating patient experiences and satisfaction concerning the effectiveness, side-effects, and ease of use of a medication, and a disease-specific module about IPF. Satisfaction was scored on a scale from - 5 (very dissatisfied) to + 5 (very satisfied). RESULTS: In total, 90 patients were included, of whom 43% used nintedanib and 57% pirfenidone. After 6 months, the mean overall score for satisfaction with medication was 2.1 (SD 1.9). No differences were found in experiences and satisfaction with medication, and the number and severity of side-effects between nintedanib and pirfenidone. Perceived effectiveness of medication was rated as significantly more important than side-effects and ease of use (p = 0.001). Expectations of patients regarding effectiveness were higher than experiences after 6 months. Self-reported experience with effectiveness was the main factor associated with overall medication satisfaction. CONCLUSIONS: Patient experiences and satisfaction with antifibrotic treatment were fairly positive, and similar for nintedanib and pirfenidone. Systematic evaluation of patient expectations, experiences, and satisfaction with medication could enhance shared-decision making and guide drug treatment decisions in the future. TRIAL REGISTRATION: NCT03420235 .

The development of a cryptoglandular Anal Fistula Core Outcome Set (AFCOS): an international Delphi study protocol.

PURPOSE: Cryptoglandular anal fistula is a disorder with an incidence of around 1 per 5,000 people per year in European countries. Many studies have been conducted to evaluate the effectiveness of interventions for anal fistula. However, there is considerable heterogeneity in the outcomes assessed and reported in these studies. This limits research quality and complicates evidence synthesis. A solution for heterogeneity in outcome reporting is the development of a Core Outcome Set (COS). This paper describes the protocol for the development of a European COS for Anal Fistula (AFCOS). METHODS: The first step will be a systematic review of the literature to identify potential outcomes that may be included in the COS. Patient interviews will be conducted in The United Kingdom and The Netherlands to ensure that both clinician-important and patient-important outcomes are captured. The outcomes will be categorized using the COMET taxonomy and taken forward to a Delphi consensus exercise. In up to three web-based Delphi surveys the outcomes will be prioritized by patients, clinicians (surgeons, gastroenterologists, and radiologists), and (clinical) researchers. The responses will be summarized and reported anonymously in subsequent round(s) facilitating convergence to a consensus opinion. The final COS will be decided during a face-to-face consensus meeting with patients, clinicians, and (clinical) researchers. DISCUSSION: This study protocol describes the development of a European COS for anal fistula to improve research quality, evidence synthesis, and patient care.

European Society of Coloproctology Core Outcome Set for haemorrhoidal disease: an international Delphi study among healthcare professionals.

AIM: There is considerable heterogeneity in outcomes in studies reporting on the treatment of haemorrhoidal disease (HD). The aim of this study was to develop a Core Outcome Set (COS) for HD in cooperation with the European Society of Coloproctology. METHOD: A Delphi study was performed according to the Outcome Measures in Rheumatology (OMERACT) methodology. In total 38 healthcare professionals and 30 patients were invited to the panel. Previously, 10 outcome domains and 59 outcomes were identified through a systematic literature review. In this study, these domains and outcomes were formed into one questionnaire for healthcare professionals and a separate questionnaire for patients. Sequential questionnaire rounds prioritizing the domains and outcomes were conducted. Panel members were asked to rate the appropriateness of each domain and outcome on a nine-point Likert scale. During a face-to-face meeting, healthcare professionals agreed on the primary and secondary end-points of the COS for HD. Finally, a short survey was sent to the healthcare professionals in order to reach consensus on how the chosen end-points should be assessed and at which time points. RESULTS: The response rate in questionnaire round 1 for healthcare professionals was 44.7% (n = 17). Sixteen out of 17 healthcare professionals also completed the questionnaire in round 2. The response rate for the patient questionnaire was 60% (n = 18). Seventeen healthcare professionals participated in the face-to-face meeting. The questionnaire rounds did not result in a clear-cut selection of primary and secondary end-points. Most domains and outcomes were considered important, and only three outcomes were excluded. During the face-to-face meeting, agreement was reached to select the domain 'symptoms' as primary end-point, and 'complications', 'recurrence' and 'patient satisfaction' as secondary end-points in the COS for HD. Furthermore, consensus was reached that the domain 'symptoms' should be a patient reported outcome measure and should include the outcomes 'pain' and 'prolapse', 'itching', 'soiling' and 'blood loss'. The domain 'complications' should include the outcomes 'incontinence', 'abscess', 'urinary retention', 'anal stenosis' and 'fistula'. Consensus was reached to use 'reappearance of initial symptoms' as reported by the patient to define recurrence. During an additional short survey, consensus was reached that 'incontinence' should be assessed by the Wexner Fecal Incontinence Score, 'abscess' by physical examination, 'urinary retention' by ultrasonography, 'anal stenosis' by physical examination, and 'fistula' by physical examination and MR imaging if inconclusive. During follow-up, the outcome 'symptoms' should be assessed at baseline, 7 days, 6 weeks and 1 year post-procedure. The outcomes 'abscess' and 'urinary retention' should be assessed 7 days post-procedure and 'incontinence', 'anal stenosis' and 'fistula' 1 year post-procedure. CONCLUSIONS: We developed the first European Society of Coloproctology COS for HD based on an international Delphi study among healthcare professionals. The next step is to incorporate the patients' perspective in the COS. Use of this COS may improve the quality and uniformity of future research and enhance the analysis of evidence.

Effects and moderators of psychosocial interventions on quality of life, and emotional and social function in patients with cancer: An individual patient data meta-analysis of 22 RCTs.

OBJECTIVE: This individual patient data (IPD) meta-analysis aimed to evaluate the effects of psychosocial interventions (PSI) on quality of life (QoL), emotional function (EF), and social function (SF) in patients with cancer, and to study moderator effects of demographic, clinical, personal, and intervention-related characteristics. METHODS: Relevant studies were identified via literature searches in 4 databases. We pooled IPD from 22 (n = 4217) of 61 eligible randomized controlled trials. Linear mixed-effect model analyses were used to study intervention effects on the post-intervention values of QoL, EF, and SF (z-scores), adjusting for baseline values, age, and cancer type. We studied moderator effects by testing interactions with the intervention for demographic, clinical, personal, and intervention-related characteristics, and conducted subsequent stratified analyses for significant moderator variables. RESULTS: PSI significantly improved QoL (ß = 0.14,95%CI = 0.06;0.21), EF (ß = 0.13,95%CI = 0.05;0.20), and SF (ß = 0.10,95%CI = 0.03;0.18). Significant differences in effects of different types of PSI were found, with largest effects of psychotherapy. The effects of coping skills training were moderated by age, treatment type, and targeted interventions. Effects of psychotherapy on EF may be moderated by cancer type, but these analyses were based on 2 randomized controlled trials with small sample sizes of some cancer types. CONCLUSIONS: PSI significantly improved QoL, EF, and SF, with small overall effects. However, the effects differed by several demographic, clinical, personal, and intervention-related characteristics. Our study highlights the beneficial effects of coping skills training in patients treated with chemotherapy, the importance of targeted interventions, and the need of developing interventions tailored to the specific needs of elderly patients.

Economic evaluation of four follow-up strategies after curative treatment for breast cancer: results of an RCT.

BACKGROUND: An economic evaluation was performed alongside a randomised controlled trial (ISRCTN 74071417) investigating the cost-effectiveness of nurse-led telephone follow-up instead of hospital visits, and of a short educational group programme (EGP) in the first year after breast cancer treatment. METHOD: This economic evaluation (n = 299) compared the one-year costs and the effects of four follow-up strategies: (1) hospital follow-up; (2) nurse-led telephone follow-up; (3) hospital follow-up plus EGP; and (4) nurse-led telephone follow-up plus EGP. Costs were measured using cost diaries and hospital registrations. Quality-adjusted life years (QALYs) were measured using the EQ-5D. Outcomes were expressed in incremental cost-effectiveness ratios (ICERs) and cost-effectiveness acceptability curves. RESULTS: Hospital follow-up plus EGP yielded most QALYs (0.776), but also incurred the highest mean annual costs (€4914). The ICER of this strategy versus the next best alternative, nurse-led telephone follow-up plus EGP (0.772 QALYs and €3971), amounted to €235.750/QALY. Hospital and telephone follow-up without EGP both incurred higher costs and less QALYs than telephone follow-up plus EGP and were judged inferior. Hospital follow-up plus EGP was not considered cost-effective, therefore, telephone follow-up plus EGP was the preferred strategy. The probability of telephone follow-up plus EGP being cost-effective ranged from 49% to 62% for different QALY threshold values. Secondary and sensitivity analyses showed that results were robust. CONCLUSION: Nurse-led telephone follow-up plus EGP seems an appropriate and cost-effective alternative to hospital follow-up for breast cancer patients during their first year after treatment.

Nurse-led telephone follow-up and an educational group programme after breast cancer treatment: results of a 2 × 2 randomised controlled trial.

OBJECTIVE: To investigate whether frequent hospital follow-up in the first year after breast cancer treatment might partly be replaced by nurse-led telephone follow-up without deteriorating health-related quality of life (HRQoL), and whether a short educational group programme (EGP) would enhance HRQoL. PATIENTS AND METHODS: A multicentre pragmatic randomised controlled trial (RCT) with a 2×2 factorial design was performed among 320 breast cancer patients who were treated with curative intent. Participants were randomised to follow-up care as usual (3-monthly outpatient clinic visits), nurse-led telephone follow-up, or the former strategies combined with an educational group programme. The primary outcome for both interventions was HRQoL, measured by EORTC QLQ-C30. Secondary outcomes were role and emotional functioning and feelings of control and anxiety. RESULTS: Data of 299 patients were available for evaluation. There was no significant difference in HRQoL between nurse-led telephone and hospital follow-up at 12 months after treatment (p = 0.42; 95% confidence interval (CI) for difference: -1.93-4.64) and neither between follow-up with or without EGP (p = 0.86; 95% CI for difference: -3.59-3.00). Furthermore, no differences between the intervention groups and their corresponding control groups were found in role and emotional functioning, and feelings of control and anxiety (all p-values > 0.05). CONCLUSION: Replacement of most hospital follow-up visits in the first year after breast cancer treatment by nurse-led telephone follow-up does not impede patient outcomes. Hence, nurse-led telephone follow-up seems an appropriate way to reduce clinic visits and represents an accepted alternative strategy. An EGP does not unequivocally affect positive HRQoL outcomes.

Follow-up after curative treatment for breast cancer: why do we still adhere to frequent outpatient clinic visits?

Follow-up after curative treatment for breast cancer consists of frequent outpatient clinic visits, scheduled at regular intervals. Its aim is primarily to detect local disease recurrence, or a second primary breast cancer, but also to provide information and psychosocial support. The cost-effectiveness of these frequent visits is being questioned however, leading to a search for less intensive follow-up strategies, such as follow-up by the general practitioner, patient-initiated or nurse-led follow-up or contact by telephone. These strategies are generally considered to be safe, but they are not yet widely accepted in clinical practice. Since brief interventions based on self-education and information have been shown to be able to improve quality of life, we hypothesise that these interventions may lead to a better acceptance of reduced follow-up by both patients and professionals.