RESUMEN

BACKGROUND: Treatment with opioids is a mainstay in perioperative pain management. While the leading treatment paradigm has been procedure-specific pain management, efforts regarding personalized pain treatment are increasing. The OPI•AID project aims to develop personalized algorithms for perioperative pain management, taking demographic, surgical, and anaesthesiologic factors into account. We will undertake five parallel reviews to illuminate current evidence on different aspects of individual responses to perioperative opioid treatment. METHODS: Inclusion of adult populations in English-written studies. Review-specific searches are developed for the following databases: CENTRAL, MEDLINE, Embase, clinicaltrials.gov, and clinicaltrial.eu. Two authors will independently screen citations, extract data, and assess the risks of bias in each review (QUIPS, PROBAST and RoB2, as relevant). CONCLUSION: These reviews will evaluate various aspects of perioperative opioid treatment, including individualized treatment strategies, selection of specific opioids, and individual patient responses. These will guide future development of a personalized perioperative opioid treatment algorithm (OPI•AID) that will be validated and tested clinically against standard of care.

RESUMEN

BACKGROUND: Breast cancer is one of the most common cancers in women. Patient-reported outcome measures are used to evaluate patients' health-related quality of life in clinical breast cancer studies. This study evaluated the structure, validity, reliability, and responsiveness of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (NFBSI-16) subscales in a clinical trial featuring patients with advanced/metastatic breast cancer (aBC), and estimated NFBSI-16 meaningful change thresholds. METHODS: Data from 101 patients with aBC enrolled in a phase II trial (Xenera-1) were included for psychometric evaluation of the NFBSI-16. Subscale structure was evaluated by assessing inter-item correlations, item-total correlations, and internal consistency (cycles 2 and 5). Validity was assessed using scale-level convergent validity (cycles 2 and 5) and known-groups (Baseline). Reliability was analysed via test-retest at cycles 3-4, and responsiveness to improvement and worsening was evaluated at cycles 5, 7, and 9. Meaningful change thresholds were estimated using anchor-based methods (supported by distribution-based methods) at cycles 5, 7, and 9. RESULTS: NFBSI-16 internal consistency was acceptable, but item-total correlations suggested that its subscales and the GP5 item (side-effect of treatment) scores may be preferred over a total score. Convergent and known-groups evidence supported NFBSI-16 validity. Test-retest reliability was good to excellent for Total and DRS-P (disease-related symptoms: physical) scales, and moderate for the GP5 item. Responsiveness to worsening was generally demonstrated, but responsiveness to improvement could not be demonstrated due to limited observed improvement. Anchor-based meaningful change thresholds were estimated for DRS-P and Total scores. CONCLUSION: This study provides evidence that the NFBSI-16 has desirable psychometric properties for use in clinical studies in aBC. It also provides estimates of group- and individual-level meaningful change thresholds to facilitate score interpretation in future aBC research.

Asunto(s)

RESUMEN

To assess the minimal change in visual acuity perceived by patients as important in different eye-related interventions. PubMed was utilized to search articles on each of the four major interventions: cataract surgery, keratoplasty, different glaucoma treatments, and refractive eye surgery, each combined with quality of life (QoL) and visual acuity keywords. The search was narrowed to articles between 2000 and 2023. Seventy-four major articles were thus reviewed. Of these, 27 studies reviewed the results of cataract surgery, 20 studies discussed the effect of keratoplasty interventions on the vision-related QoL (VRQoL), most showing that VRQoL improved significantly after keratoplasty, 11 studies investigated the effect of different glaucoma interventions on patients' visual acuity and the QoL, 16 studies reviewed refractive surgery, where they showed an improved QoL in most of the cases, although some of the studies showed a slight superiority of one intervention over the other in the short term. The minimally important difference (MID) perceived in visual acuity depends mainly on the type of surgical intervention (keratoplasty, glaucoma, or refractive surgery), and the impact on QoL on improved visual acuity differs depending on the intervention.

RESUMEN

OBJECTIVE: The Berg Balance Scale (BBS), Performance-Oriented Mobility Assessment (POMA), and Dynamic Gait Index (DGI) are crucial standard tools used to evaluate functional balance. However, their minimal important difference (MID) threshold is yet to be established. The objective of this research was to explore the MID for BBS, POMA, and DGI in people suffering from chronic stroke. METHODS: A total of 130 chronic stroke patients were assessed using BBS, POMA and DGI before and after undergoing interventions. The treatment procedures included a series of task-oriented exercises focusing on motor re-learning. In anchor-based approach, the Clinical Global Impression of Improvement scale served as the anchor. The Receiver Operating Characteristic (ROC) curve was employed to identify the best MID cut-off point for BBS, POMA, and DGI. In the distribution-based approach, one-third standard deviation (SD), half SD, one standard error of measurement (SEM), and the total score of BBS, POMA and DGI were used to determine the MID. RESULTS: The MID from the ROC curve was greater than 2 points for BBS, greater than 1 point for POMA and DGI. For the distribution-based approach, the MIDs corresponding to one-third of the SD, half of the SD, and one SEM for BBS were 1.92, 3.20, and 0.90; for POMA were 1.47, 2.45, and 1.96; for DGI were 1.48, 2.47, and 1.16 points, respectively. CONCLUSION: BBS, POMA and DGI are suitable instruments for assessing functional balance in individuals with chronic stroke, with demonstrable MID. Additionally, the results indicated that DGI has higher sensitivity and specificity than the other examined scales for distinguishing between groups without change and those with minimal balance improvement after receiving interventions.

RESUMEN

INTRODUCTION: Through interviews with 148 older persons from four countries and in four languages, the content for a 17-item measure of active living was developed. The purpose of this paper is to present further evidence of the extent to which this new measure, Older Persons Active Living (OPAL), is "fit-for-purpose" for measuring the extent of active living at one point in time. METHODS: A cross-sectional study was carried out on a population aged 65 + and living independently, drawn from a participant panel, HostedinCanada, sampling people from Canada, United States, United Kingdom, and Netherlands. The survey instrument comprised the OPAL questionnaire rated on importance and frequency, sociodemographics, and information on physical and mental function. The argument-based approach to validity framed the analyses. Logistic regression, structural equation modeling, ordinary least-squares regression, and correlation were used to generate estimates for parameters underpinning validity evidence. RESULTS: A total of 1612 people completed the survey, 100 to 400 people across the 6 country-language strata. The proportion of people rating the items as extremely or quite important ranged from 60 to 90%, with no important differences between men and women and few differences between strata. A single-factor structure was supported. The ordinality of the response options justified an additive total score yielding a near normal distribution (mean: 33.1; SD: 11.5; range 0-51). Correlations with other measures of converging constructs were of moderate strength (~ 0.50), and differences across groups known to affect functioning and health were observed, suggesting a Miminal Important Difference (MID) of 6 out of 51. CONCLUSION: The results of this study provide evidence that the 17-item OPAL measure is fit for the purpose of estimating the extent to which older persons are living actively at one point in time.

RESUMEN

Background: The Minimal Important Difference (MID) and Smallest Detectable Change (SDC) are methods used to identify the smallest changes in Patient-Reported Outcome Measures (PROMs) that are of relevance to the patients. Data on these parameters is, however, limited for elbow conditions including traumatic injuries. The aim of this study was, therefore, to estimate the MID and SDC for three commonly used PROMs after elbow trauma; the Oxford Elbow Score (OES), Quick Disabilities of the Arm Shoulder and Hand (QuickDASH) and Single Assessment Numeric Evaluation (SANE). Methods: One hundred patients, 67 females, aged ≥18 years (mean age 52.4 years (standard deviation, 18.2)), who had sustained a fracture, tendon rupture or dislocation affecting the elbow, completed the OES, QuickDASH, and SANE 3-5 months after injury (T1) and again after a minimum of 3 weeks (T2). A transition item with a 7-level scale, enquiring about the situation with the elbow, was also completed at T1 and T2. The difference in scores between T1 and T2 was calculated (change scores). The MID was assessed using the mean change method; a response of "slightly better" or "slightly worse" was defined as being a clinically significant change. The SDC was estimated by calculating the standard error of measurement based on 2 administrations (1- to 3-week interval) of PROMs in a separate group of patients who had sustained an elbow injury 1- 2 years previously. Results: The most common diagnosis was fracture of the proximal radius (n = 33). Eighteen patients responded slightly better and 5 slightly worse on the transition item and had mean change scores of 7.9 (9.3) for the OES and -7.4 (11.4) for the QuickDASH. Assessment of SDC was based on 56 patients having sustained an elbow injury between September 2019 and October 2020. The SDC was: 12.1 for the OES, 11.4 for the QuickDASH, and 1.94 for the SANE. Conclusion: Change scores need to exceed 12.1 points for the OES, 11.4 points for the QuickDASH, and 1.94 points for the SANE in order to measure change with clinical relevance and not due to measurement errors.

RESUMEN

BODY-Q is a patient-reported outcome measure for comprehensive assessment of outcomes specific to patients undergoing bariatric surgery. The clinical utility of BODY-Q is hampered by the lack of guidance on score interpretation. This study aimed to determine minimal important difference (MID) for assessment of BODY-Q. Prospective BODY-Q data from Denmark and the Netherlands pre- and post-bariatric surgery were collected. Two distribution-based methods were used to estimate MID by 0.2 standard deviations of baseline scores and the mean standardized response change of scores from baseline to 3-years postoperatively. In total, 5476 assessments from 2253 participants were included of which 1628 (72.3%) underwent Roux-en-Y gastric bypass, 586 (26.0%) sleeve gastrectomy, 33 (1.5%) gastric banding, and 6 (0.03%) other surgeries. The mean age was 45.1 ± 10.9 with a mean BMI of 46.6 ± 9.6. Baseline MID ranged from 1 to 4 in health-related quality of life (HRQL) and from 2 to 8 in appearance scales. The mean change of scores ranged from 4 to 5 in HRQL and from 4 to 7 in the appearance scales. The estimated MID for the change in BODY-Q HRQL and appearance scales ranged from 3 to 8 and is recommended for use to interpret BODY-Q scores and assess treatment effects in bariatric surgery.

RESUMEN

BACKGROUND: The minimal important difference (MID) is defined as the smallest difference that the patient perceives as important. Furthermore, the smallest worthwhile effect (SWE) is the important change measured with the benefit-harm trade-off method. The aim of this study was to evaluate the MID in orthodontic treatment duration to inform the decision regarding seeking procedures to accelerate orthodontic tooth movement and reduce treatment duration. METHODS: We constructed a survey eliciting views of the MID from adult participants from four countries undergoing orthodontic treatment. Ten questions addressed reduction in the treatment duration for both durations 12 and 24 months, and four questions were related to the reduction in treatment duration that the patients would require to undergo surgical or non-surgical adjunctive procedures. We applied a univariable random effects logistic regression model to examine the association between the participants' characteristics and the MID. Then, we fitted a multivariable logistic random effects regression including significant predictors. RESULTS: Four hundred and fifty adults, with a median age of 21 (interquartile range: 19-24), undergoing orthodontic treatment participated in the survey. Of the respondents, 60% considered 15 days as a trivial reduction from 12 months duration of therapy and 70% considered 15 days a trivial reduction from 24 months. Of the respondents, 48% considered the period of 2 months a moderate reduction from 12 months, and 60% considered 2 months a moderate reduction from 24 months. From these results, we inferred that patients considered reductions of approximately 1 month as the MID in the treatment duration for both 12 and 24 months. However, SWE was considerably more than the MID for most of the participants to decide undergoing surgical adjunctive procedures to reduce the time of therapy. The participants required smaller SWE to undergo non-surgical procedures compared to surgical procedures. CONCLUSION: The MID in the treatment duration is one month for both treatment durations 12 and 24 months. Patients require a greater SWE than the MID to undergo adjunctive procedures to shorten the duration, particularly for surgical procedures.

Asunto(s)

RESUMEN

Managing clinical manifestations of cancer/treatment burden on functional status and quality of life remains paramount across the cancer trajectory, particularly for patients with cachexia who display reduced functional capacity. However, clinically relevant criteria for classifying functional impairment at a single point in time or for classifying meaningful functional changes subsequent to disease and/or treatment progression are lacking. This unmet clinical need remains a major obstacle to the development of therapies for cancer cachexia. This review aims to describe current literature-based evidence for clinically meaningful criteria for (1) functional impairment at a single timepoint between cancer patients with or without cachexia and (2) changes in physical function over time across interventional studies conducted in patients with cancer cachexia. The most common functional assessment in cross-sectional and interventional studies was hand grip strength (HGS). We observed suggestive evidence that an HGS deficit between 3 and 6 kg in cancer cachexia may display clinical relevance. In interventional studies, we observed that long-duration multimodal therapies with a focus on skeletal muscle may benefit HGS in patients with considerable weight loss. Future studies should derive cohort-specific clinically relevant criteria to confirm these observations in addition to other functional outcomes and investigate appropriate patient-reported anchors.

RESUMEN

BACKGROUND: The minimal important difference is a valuable metric in ascertaining the clinical relevance of a treatment, offering valuable guidance in patient management. There is a lack of available evidence concerning this metric in the context of outcomes related to female urinary incontinence, which might negatively impact clinical decision-making. OBJECTIVES: To summarize the minimal important difference of patient-reported outcome measures associated with urinary incontinence, calculated according to both distribution- and anchor-based methods. METHODS: This is a systematic review conducted according to the PRISMA guidelines. The search strategy including the main terms for urinary incontinence and minimal important difference were used in five different databases (Medline, Embase, CINAHL, Web of Science, and Scopus) in 09 June 2021 and were updated in January 09, 2024 with no limits for date, language or publication status. Studies that provided minimal important difference (distribution- or anchor-based methods) for patient-reported outcome measures related to female urinary incontinence outcomes were included. The study selection and data extraction were performed independently by two different researchers. Only studies that reported the minimal important difference according to anchor-based methods were assessed by credibility and certainty of the evidence. When possible, absolute minimal important differences were calculated for each study separately according to the mean change of the group of participants that slightly improved. RESULTS: Twelve studies were included. Thirteen questionnaires with their respective minimal important differences reported according to distribution (effect size, standard error of measurement, standardized response mean) and anchor-based methods were found. Most of the measures for anchor methods did not consider the smallest difference identified by the participants to calculate the minimal important difference. All reports related to anchor-based methods presented low credibility and very low certainty of the evidence. We pooled 20 different estimates of minimal important differences using data from primary studies, considering different anchors and questionnaires. CONCLUSIONS: There is a high variability around the minimal important difference related to patient-reported outcome measures for urinary incontinence outcomes according to the method of analysis, questionnaires, and anchors used, however, the credibility and certainty of the evidence to support these is still limited.

Asunto(s)

RESUMEN

OBJECTIVES: The EQ-5D-5L is a commonly used health-related quality of life instrument for evaluating interventions in patients receiving dialysis; however, the minimal important difference (MID) that constitutes a meaningful treatment effect for this population has not been established. This study aims to estimate the MID for the EQ-5D-5L utility index in dialysis patients. METHODS: 6-monthly EQ-5D-5L measurements were collected from adult dialysis patients between April 2017 and November 2020 at a renal network in Sydney, Australia. EQ-VAS and Integrated Palliative care Outcome Scale Renal symptom burden scores were collected simultaneously and used as anchors. MID estimates for the EQ-5D-5L utility index were derived using anchor-based and distribution-based methods. RESULTS: A total of 352 patients with ≥1 EQ-5D-5L observation were included, constituting 1127 observations. Mean EQ-5D-5L utility index at baseline was 0.719 (SD ± 0.267), and mean EQ-5D-5L utility decreased over time by -0.017 per year (95% CI -0.029 to -0.006, P = .004). Using cross-sectional anchor-based methods, MID estimates ranged from 0.073 to 0.107. Using longitudinal anchor-based methods, MID for improvement and deterioration ranged from 0.046 to 0.079 and -0.111 to -0.048, respectively. Using receiver operating characteristic curves, MID for improvement and deterioration ranged from 0.037 to 0.122 and -0.074 to -0.063, respectively. MID estimates from distribution-based methods were consistent with anchor-based estimates. CONCLUSIONS: Anchor-based and distribution-based approaches provided EQ-5D-5L utility index MID estimates ranging from 0.034 to 0.134. These estimates can inform the target difference or "effect size" for clinical trial design among dialysis populations.

Asunto(s)

RESUMEN

BACKGROUND: Morphine-sparing effects are often used to evaluate non-opioid analgesic interventions. The exact effect that would warrant the implementation of these interventions in clinical practice (a minimally important difference) remains unclear. We aimed to determine this with anchor-based methods. METHODS: This was a post hoc analysis of three studies investigating pain management after hip or knee arthroplasty (PANSAID [NCT02571361], DEX-2-TKA [NCT03506789] and Pain Map [NCT02340052]). The overall population was median aged 70, median ASA 2, 54% female. We examined the correlation between 0 and 24 h postoperative iv morphine equivalent consumption and the severity of nausea, vomiting, sedation and dizziness. The anchor was different severity degrees of these opioid-related adverse events. The primary outcome was the difference in morphine consumption between patients experiencing no versus only mild events. Secondary outcomes included the difference in morphine consumption between patients with mild versus moderate and moderate versus severe events. We used Hodges-Lehmann median differences, exact Wilcoxon-Mann-Whitney tests and quantile regression. RESULTS: The difference in iv morphine consumption was 6 mg (95% confidence interval: 4-8) between patients with no versus only mild events, 5 mg (2-8) between patients with mild versus moderate events and 0 mg (-4 to 4) between patients with moderate versus severe events. CONCLUSIONS: In populations comparable to this post-hoc analysis (orthopaedic surgery, median age 70 and ASA 2), we suggest a minimally important difference of 5 mg for 0-24 h postoperative iv morphine consumption.

Asunto(s)

RESUMEN

PURPOSE: Anchor-based studies are today the most popular approach to determine a minimal important difference value for an outcome variable. However, a variety of construction methods for such values do exist. This constitutes a challenge to the field. In order to distinguish between more or less adequate construction methods, meaningful minimal requirements can be helpful. For example, minimal important difference values should not reflect the intervention(s) the patients are exposed to in the study used for construction, as they should later allow to compare interventions. This requires that they are not sensitive to the distribution of the change score observed. This study aims at investigating to which degree established construction methods fulfil this minimal requirement. METHODS: Six constructions methods were considered, covering very popular and recently suggested methods. The sensitivity of MID values to the distribution of the change score was investigated in a simulation study for these six construction methods. RESULTS: Five out of six construction methods turned out to yield MID values which are sensitive to the distribution of the change score to a degree that questions their usefulness. Insensitivity can be obtained by using construction methods based solely on an estimate of the conditional distribution of the anchor variable given the change score. CONCLUSION: In future the computation of MID values should be based on construction methods avoiding sensitivity to the distribution of the change score.

Asunto(s)

RESUMEN

OBJECTIVES: The Oral and Systemic Health Impact Profile for Periodontal Disease (OSHIP-Perio) was developed to measure health-related quality of life (HRQoL) and oral health-related quality of life (OHRQoL). This study aimed to determine the responsiveness and minimal importance difference (MID) of the OSHIP-Perio. METHODS: Subjects with periodontal disease completed the OSHIP-Perio at baseline and six to ten weeks after non-surgical periodontal therapy. Comparisons of the clinical variables and the OSHIP-Perio scores before and after treatment were analysed using the Wilcoxon signed-rank test, together with the Oral Health Impact Profile (OHIP-49, OHIP-14 and OHIP-5) scores. The MIDs for all the instruments were calculated using distribution-based methods. RESULTS: Fifty-one case subjects who completed periodontal treatment and the OSHIP-Perio instrument were included for analysis. Significant improvement in all clinical variables after treatment (p < 0.001) was observed. The OSHIP-Perio total score as well as its four-dimensional scores (periodontal, oral function, orofacial pain and psychosocial impact) were significantly reduced after treatment (p < 0.001), indicating better OHRQoL. Findings were consistent with the other OHIP instruments. In determining the MID, the percentage point change of the OSHIP-Perio was found to be lower than the shorter OHIP-5 instrument when using both effect sizes (ES) (10.71% vs. 15.0% at 0.5SD) and standard error of measurement (SEM) (5.36% vs. 10.0% at 1SEM; 8.93% vs. 20.0% at 2SEM) calculations. CONCLUSIONS: The OSHIP-Perio demonstrated good responsiveness which was comparable to the OHIP-49 and its short-form derivatives. Its required percentage point change in determining its MID is smaller than the OHIP-5.

Asunto(s)

RESUMEN

OBJECTIVES: The acoustic voice quality index (AVQI) is a reliable tool that objectively assesses dysphonia levels using six acoustic parameters. Despite its high criterion-related concurrent validity, diagnostic accuracy, and minimal detectable change derived from test-retest reliability, the minimal important difference (MID) of the AVQI has not been tested before. This study aimed to estimate the MIDs of AVQI for improvement audibly perceived by clinicians and self-reported improvement by patients. METHODS: A retrospective study was conducted on 110 patients who received treatment for voice disorders. Patients completed AVQI and Voice Handicap Index-10 (VHI-10) questionnaires before and after the therapy. The MIDs of the AVQI were estimated using the anchor of either auditory-perceptual judgment of total dysphonia levels by clinicians or the VHI-10 questionnaire by patients. A distribution-based approach was also used to complement the results. RESULTS: First, using the auditory-perceptual anchor, a decrease of 0.95 in the AVQI was estimated as the MID for clinicians' perception, as a result of the receiver operating curve. Then, using the patient-reported anchor, an improvement of 1.36 in the AVQI was estimated as the MID for patients' voice-related disability. The distribution-based approach also ensured the anchor-based results of both the MIDs. CONCLUSIONS: The AVQI is a reliable and valid tool for evaluating voice quality, and a 0.95 decrease in the AVQI represents a meaningful improvement for clinicians' perception, whereas a 1.36 decrease in the AVQI influences patients' self-reported disability. This study contributes to understanding the minimal change necessary for clinicians to make informed decisions and ensure patient satisfaction. LEVEL OF EVIDENCE: 3 Laryngoscope, 134:2805-2811, 2024.

Asunto(s)

RESUMEN

BACKGROUND: The World Health Organization Disability Assessment Schedule 2.0 12-item survey (WHODAS-12) is a questionnaire developed by the WHO to measure functioning across health conditions, cultures, and settings. WHODAS-12 consists of a subset of the 36 items of WHODAS-2.0 36-item questionnaire. Little is known about the minimal important difference (MID) of WHODAS-12 in persons with chronic low back pain (LBP), which would be useful to determine whether rehabilitation improves functioning to an extent that is meaningful for people experiencing the condition. Our objective was to estimate an anchor-based MID for WHODAS-12 questionnaire in persons with chronic LBP. METHODS: We analyzed data from two cohort studies (identified in our previous systematic review) conducted in Europe that measured functioning using the WHODAS-36 in adults with chronic LBP. Eligible participants were adults with chronic LBP with scores on another measure as an anchor to indicate participants with small but important changes in functioning over time [Short-form-36 Physical Functioning (SF36-PF) or Oswestry Disability Index (ODI)] at baseline and follow-up (study 1: 3-months post-treatment; study 2: 1-month post-discharge from hospital). WHODAS-12 scores were constructed as sums of the 12 items (scored 0-4), with possible scores ranging from 0 to 48. We calculated the mean WHODAS-12 score in participants who achieved a small but meaningful improvement on SF36-PF or ODI at follow-up. A meaningful improvement was an MID of 4-16 on ODI or 5-16 on SF36-PF. RESULTS: Of 70 eligible participants in study 1 (mean age = 54.1 years, SD = 14.7; 69% female), 18 achieved a small meaningful improvement based on SF-36 PF. Corresponding mean WHODAS-12 change score was - 3.22/48 (95% CI -4.79 to -1.64). Of 89 eligible participants in study 2 (mean age = 65.5 years, SD = 11.5; 61% female), 50 achieved a small meaningful improvement based on ODI. Corresponding mean WHODAS-12 change score was - 5.99/48 (95% CI - 7.20 to -4.79). CONCLUSIONS: Using an anchor-based approach, the MID of WHODAS-12 is estimated at -3.22 (95% CI -4.79 to -1.64) or -5.99 (95% CI - 7.20 to -4.79) in adults with chronic LBP. These MID values inform the utility of WHODAS-12 in measuring functioning to determine whether rehabilitation or other health services achieve a minimal difference that is meaningful to patients with chronic LBP.

Asunto(s)

RESUMEN

Clinical relevance and statistical significance are different concepts, linked via the sample size calculation. Threshold values for detecting a minimal important change over time are frequently (mis)interpreted as a threshold for the clinical relevance of a difference between groups. The magnitude of a difference between groups that is considered clinically relevant directly impacts the sample size calculation, and thereby the statistical significance in clinical study outcomes. Especially in non-inferiority trials the threshold for clinical relevance, i.e. the predefined margin for non-inferiority, is a crucial choice. A truly inferior treatment will be accepted as non-inferior when this margin is chosen too large. The magnitude of a clinically relevant difference between groups should be carefully considered, by determining the smallest effect for each specific study that is considered worthwhile. This means taking into account the (dis)advantages of both study interventions in terms of benefits, harms, costs, and potential side effects. This article clarifies common sources of confusion, illustrates the implications for clinical research with an example and provides specific suggestions to improve the design and interpretation of clinical research.

Asunto(s)

RESUMEN

AIM: To establish, for the first time, the clinically important differences for the ICIQ-UI-SF and ICIQ-FLUTS questionnaires following surgical and conservative treatments for stress-predominant urinary incontinence in women. METHODS: Data from the SIMS and OPAL randomised controlled trials were analysed using an anchor-based method. Clinically important difference (CID; score change indicating a successful outcome) and minimal important difference (MID; score change indicating the smallest noticeable difference) were estimated using the PGI-I scale as the anchor. RESULTS: For ICIQ-UI-SF, following surgical management, CIDs were 5.0 (95%CI 4.3, 5.6) at 1 year and 4.9 points (95%CI 4.2, 5.5) at 3 years, while following conservative management, CIDs were 4.0 (95%CI 3.4, 4.5) at 1 year and 4.6 points (95%CI 4.0, 5.2) at 2 years. For ICIQ-FLUTS, the CID was 3.4 points (95%CI 2.9, 4.0) at 1 year for both surgical and conservative management. MIDs for ICIQ-UI-SF, after surgical treatment, were 4.7 (95% CI 3.2, 6.1) at 1 year and 1.6 points (95%CI -0.2, 3.0) at 3 years, and after conservative treatment they were 1.7 (95% CI 1.0, 2.5) at 1 year and 1.9 points (95%CI 1.1, 2.7) at 2 years. For ICIQ-FLUTS, MIDs were 1.8 (95% CI 0.6, 3.1) at 1 year and 3.2 points (95%CI 2.0, 4.4) at 2 years after surgical treatment, and 1.3 (95%CI 0.6, 1.9) at 1 year and 1.9 points (95%CI 1.1, 2.6) at 2 years after conservative treatment. CONCLUSION: Our study is the first to establish the CID for the ICIQ-UI-SF and ICIQ-FLUTS that women would associate with a successful outcome 3-years post-surgery and 2-years post-conservative treatment of stress-predominant urinary incontinence. The MID was lower following conservative compared to surgical treatment.

Asunto(s)

RESUMEN

PURPOSE: The 6-min walk test (6MWT) of allogenic hematopoietic stem cell transplantation (allo-HSCT) recipients has been gaining attention; however, minimal differences have not been reported. This study aimed to determine the minimal important difference (MID) in the 6MWT among hospitalized patients with allo-HSCT. MATERIALS AND METHODS: The MID of the 6MWT was calculated using three different methodologies based on an anchor-based method; basic anchor-based methods, linear regression analysis, and receiver operating characteristic (ROC) curve analysis. The decrease in the score of Question 2 of the European Organization for Research and Treatment of Cancer Quality of life questionnaire core-30 was included as an anchor question for calculating the MID. Both actual and percentage changes in 6MWT values from baseline and at discharge were used in the MID calculations. In the actual and percentage change of the 6MWT, the one with the larger the area under the curve in the ROC curve was recommended as the MID. RESULTS: Among the three methods using actual values, the largest MID of the 6MWT was -37.5 m (sensitivity: 54%, specificity: 88%). CONCLUSION: More careful follow-up after discharge is necessary for allo-HSCT patients who show a reduction of 37.5 m or more in the acute illness phase.

Advancements in allogeneic hematopoietic stem cell transplantation have improved the survival rates of individuals with malignant hematological disorders, and efforts should now be focused on enhancing their physical function and quality of life.There is a problem that the physical performance of the patients is reduced by the side effects of treatment.More careful follow-up after discharge is necessary for allo-HSCT patients who show a reduction of 37.5 m or more in the acute illness phase.