scholarly journals Estimates of the mean difference in orthopaedic randomized trials: obligatory yet obscure

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Lauri Raittio ◽  
Antti Launonen ◽  
Ville M. Mattila ◽  
Aleksi Reito
Keyword(s):  
Primary Outcome ◽  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Ceiling Effect ◽  
Mean Difference ◽  
Discussion Section ◽  
Power Calculations ◽  
Randomized Controlled ◽  
Patient Reported ◽  
The Mean

Abstract Background Randomized controlled trials in orthopaedics are powered to mainly find large effect sizes. A possible discrepancy between the estimated and the real mean difference is a challenge for statistical inference based on p-values. We explored the justifications of the mean difference estimates used in power calculations. The assessment of distribution of observations in the primary outcome and the possibility of ceiling effects were also assessed. Methods Systematic review of the randomized controlled trials with power calculations in eight clinical orthopaedic journals published between 2016 and 2019. Trials with one continuous primary outcome and 1:1 allocation were eligible. Rationales and references for the mean difference estimate were recorded from the Methods sections. The possibility of ceiling effect was addressed by the assessment of the weighted mean and standard deviation of the primary outcome and its elaboration in the Discussion section of each RCT where available. Results 264 trials were included in this study. Of these, 108 (41 %) trials provided some rationale or reference for the mean difference estimate. The most common rationales or references for the estimate of mean difference were minimal clinical important difference (16 %), observational studies on the same subject (8 %) and the ‘clinical relevance’ of the authors (6 %). In a third of the trials, the weighted mean plus 1 standard deviation of the primary outcome reached over the best value in the patient-reported outcome measure scale, indicating the possibility of ceiling effect in the outcome. Conclusions The chosen mean difference estimates in power calculations are rarely properly justified in orthopaedic trials. In general, trials with a patient-reported outcome measure as the primary outcome do not assess or report the possibility of the ceiling effect in the primary outcome or elaborate further in the Discussion section.

A Patient-Reported Outcome Measure for Symptoms and Symptom Burden of Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2094-2094 ◽  
Author(s):  
Loretta A. Williams ◽  
Araceli Garcia-Gonzalez ◽  
Hycienth O. Ahaneku ◽  
Jorge E. Cortes ◽  
Guillermo Garcia-Manero ◽  
...  
Keyword(s):  
Myeloid Leukemia ◽  
Research Funding ◽  
Outcome Measure ◽  
Symptom Burden ◽  
Patient Reported Outcome ◽  
Shortness Of Breath ◽  
Patient Reported Outcome Measure ◽  
Disturbed Sleep ◽  
Patient Reported ◽  
The Mean

Abstract Background: Patient report of disease- and treatment-related symptom burden in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) is scarce. Symptom burden is the combined impact of disease and treatment symptoms on daily functioning. Lack of recognition and monitoring of symptoms and symptom burden can lead to inadequate management and possible treatment non-adherence. Aims: Our aim is to develop a short, valid, reliable patient-reported outcome measure of symptoms and symptom burden experienced by AML and MDS patients and to determine the validity of a single measure for research and practice. Methods: After obtaining IRB approval, patients with AML (N=152) and MDS (N=97) were recruited to this cross-sectional study. Patients rated the 13 core symptom items (pain, fatigue, nausea, disturbed sleep, distress, shortness of breath, trouble remembering, lack of appetite, drowsiness, dry mouth, sadness, vomiting, and numbness and tingling), 6 proposed AML/MDS symptom items (muscle weakness, malaise, fever, headache, diarrhea, skin problems), and 6 interference items (general activities, mood, work, relations with others, walking, and enjoyment of life) of the MD Anderson Symptom Inventory (MDASI) on 0-to-10 scales (0 = not present or no interference; 10 = as bad as can be imagined or complete interference) twice 1-2 days apart. Clinical and demographic information was collected from medical records and analyzed using descriptive statistics. Means of the symptom and interference ratings for the AML and MDS patients were compared using T-tests. Standard psychometric techniques were used to determine the reliability, stability, and validity of the instrument in patients with AML and MDS. Results: All MDS patients were outpatients while 75 of the AML patients were inpatients and 77 were outpatients. The AML and MDS patients had been diagnosed a mean of 13.8 months (standard deviation [SD]=23.9) and 30.5 months (SD=32.4) respectively. The mean (Mn) symptom and interference ratings respectively for the AML inpatients (Mn=2.8, SD=1.6; Mn=4.0, SD=2.4) were significantly higher than for the AML outpatients (Mn=1.8, SD=1.4, p<0.01; Mn=2.7, SD=2.3, p<0.01) or MDS patients (Mn=1.9, SD=1.5, p<0.01; Mn=2.7, SD=2.5, p<0.01). The mean ratings for the 5 most severe symptom means for AML and MDS patients respectively were: fatigue (Mn=4.0, SD=2.8; Mn=4.0, SD=2.5; p =0.97), disturbed sleep (Mn=3.3, SD=3.2; Mn=2.7, SD=3.3; p=0.19), drowsiness (Mn=3.0, SD=2.8; Mn=2.8, SD=3.1; p=0.70), muscle weakness (Mn=2.9, SD=2.8; Mn=2.9, SD=3.0; p=0.91), dry mouth for AML patients (Mn=3.4, SD=3.2; Mn=2.2,SD=2.8; p<0.01), and shortness of breath for MDS patients (Mn=2.7, SD=2.8; 1.9, SD=2.3; p=0.02). Two of the 6 AML/MDS symptom items (fever and headache) were dropped because so few patients said they experienced the symptoms at more than a mild (0-4 rating) level (12% and 11% respectively). Both groups of patients endorsed similar symptoms, and none of the means of the 4 final AML/MDS symptoms were significantly different between the groups. Cronbach's reliability for all symptom items for AML and MDS respectively were 0.88 and 0.91 and for all interference items were 0.86 and 0.92. The test-retest reliability intra-class correlations were 0.85 for the core symptoms, 0.77 for AML/MDS symptoms, and 0.84 for the interference items. The MDASI-AML/MDS can be completed by patients in less than 5 minutes. Conclusion/Summary: Lack of recognition of symptoms experienced by patients with AML and MDS can lead to inadequate management of symptoms, interfere with the ability of patients to function and enjoy life, and impact the tolerability of and adherence to treatment regimens. While the symptoms experienced by the two groups had some variation in severity, a similar group of symptoms were the most common and relevant for both groups of patients, and the same measure was appropriate for both groups. The MDASI-AML/MDS is a brief, easily-completed, and validated measure of symptom burden for patients with AML and MDS that can be used for accurate and consistent monitoring of symptoms by clinicians and researchers. Disclosures Williams: Amgen: Consultancy; Novartis: Research Funding. Cortes:Pfizer: Consultancy, Research Funding; BMS: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; BerGenBio AS: Research Funding; Teva: Research Funding; Ariad: Consultancy, Research Funding; Astellas: Consultancy, Research Funding; Ambit: Consultancy, Research Funding; Arog: Research Funding; Celator: Research Funding; Jenssen: Consultancy. Mendoza:Amgen Inc.: Consultancy. Shi:Amgen Inc.: Consultancy. Cleeland:Amgen Inc.: Consultancy.

scholarly journals Application of the paediatric throat disorders outcome test (T-14) for tonsillectomy and adenotonsillectomy

2013 ◽  
Vol 95 (6) ◽  
pp. 410-414 ◽  
Author(s):  
K Konieczny ◽  
TC Biggs ◽  
S Caldera
Keyword(s):  
Patient Reported Outcome ◽  
Sleep Apnoea ◽  
Mean Difference ◽  
Clinical Value ◽  
Recurrent Tonsillitis ◽  
Obstructive Sleep ◽  
Patient Reported ◽  
The Mean ◽  
The Uk

Introduction The T-14 questionnaire is a validated patient reported outcome measure (PROM) used to assess the value of paediatric tonsillectomy from the patient’s perspective. Tonsillectomy is a procedure under threat. As such, this study aimed to provide further evidence supporting the role of tonsillectomy in the management of recurrent tonsillitis and obstructive sleep apnoea. Methods A prospective uncontrolled observational study was undertaken examining 54 paediatric patients undergoing tonsillectomy at our institution. Parents of children having surgery were invited to complete a T-14 questionnaire preoperatively as well as at three and six months postoperatively. Results The questionnaire was completed for 52 of the 54 patients preoperatively as well as at 3 and 6 months postoperatively (96% response rate). The mean difference between the preoperative and six-month T-14 score was 31.8 (p<0.0001). T-14 scores at three months were also significantly improved compared with those taken preoperatively (mean difference: 29.9, p<0.0001). Conclusions This is the first study in the literature to assess T-14 questionnaires at three and six months following paediatric tonsillectomy, providing evidence of the early benefit on PROMs. Tonsillectomy is the most common surgical procedure performed in the UK, and it is regarded highly by patients and otolaryngologists alike. This study provides significant evidence that tonsillectomy improves PROMs, thereby demonstrating its ongoing clinical value as a funded National Health Service procedure.

The Visual Analogue Scale for Pain: A Comparison of Scores Reported to Residents Versus an Attending Foot and Ankle Surgeon

2019 ◽  
Vol 13 (3) ◽  
pp. 207-210
Author(s):  
Calvin J. Rushing ◽  
Steven M. Spinner ◽  
Patrick Hardigan
Keyword(s):  
Visual Analogue Scale ◽  
Analogue Scale ◽  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Foot And Ankle ◽  
Point Scale ◽  
Patient Reported Outcome Measure ◽  
Patient Reported ◽  
The Mean ◽  
Patient Referrals

Background. Recent studies have raised concerns regarding the usefulness of the visual analogue scale (VAS) as an effective outpatient patient-reported outcome measure (PROM), with disparate scores reported during the same encounter to a nurse versus physician. The purpose of present study was to assess the VAS reported by new patient referrals to 2 different physicians of varying training levels (resident, attending), during the same initial outpatient encounter. Methods. One hundred and one patients treated by a single foot and ankle surgeon were included in the retrospective cohort. Each patient was asked to rate their pain intensity by a resident, and then by the attending surgeon using a standard horizontal VAS 0 to 10, from “no pain” to the “worst pain.” Differences in reported scores were analyzed. Results. Overall, the mean VAS reported to the residents (4.97 ± 2.75) and the attending surgeon (5.02 ± 2.71) were not significantly different ( P = .61). On the 11-point scale, the mean difference accounted for only 0.05 points. Conclusion. Taken into consideration with previous studies, the data suggest collection personnel may influence the reported VAS, possibly owing to patients’ preferences and perception of their care. Although the exact reasons remain unclear, our findings lend credence to the previous concerns expressed regarding the subjective nature of the VAS. Levels of Evidence: Level III: Comparative study

scholarly journals Randomized Controlled Trial of Paper-Based at a Hospital versus Continual Electronic Patient-Reported Outcomes at Home for Metastatic Cancer Patients: Does Electronic Measurement at Home Detect Patients’ Health Status in Greater Detail?

2021 ◽  
pp. 0272989X2110101
Author(s):  
Takeru Shiroiwa ◽  
Yasuhiro Hagiwara ◽  
Naruto Taira ◽  
Takuya Kawahara ◽  
Keiko Konomura ◽  
...  
Keyword(s):  
Randomized Controlled Trial ◽  
Health Status ◽  
Cancer Patients ◽  
Metastatic Cancer ◽  
Controlled Trial ◽  
Mean Difference ◽  
Randomized Controlled ◽  
Patient Reported ◽  
The Mean ◽  
At Home

Purpose This study aimed to determine whether continual electronic patient-reported outcome (ePRO) measurements at home can capture the fluctuations in health-related quality of life (HRQOL) scores between visits. Methods We performed a randomized controlled trial to compare the scores obtained by standard practice (paper-based measurements in the hospital) to scores by continuous measurement of ePRO at home. Metastatic cancer patients were randomly assigned to either the paper-based ( n = 50) or the ePRO group ( n = 52). EQ-5D-5L and EORTC QLQ C-30 scores were obtained on 3 different chemotherapy days in the paper-based group. Meanwhile, scores were obtained on the chemotherapy day and on days 3, 7, 10, and 14 in the ePRO group during 2 cycles. The first hypothesis of our study was that both scores at the same time points would be equivalent despite different measurement frequency, place, or mode of measurement. The second hypothesis was that PRO score–adjusted time would be different between the groups. For equivalence, the endpoint was the mean EQ-5D-5L index value on the chemotherapy day before the outpatient treatment. Only if equivalence was shown, quality-adjusted life-days (QALDs) were considered using all the data. Results The adjusted mean difference in the EQ-5D-5L index was determined to be −0.013 (95% confidence interval [CI]: −0.049 to 0.022); the 95% CI did not exceed the equivalence margin. Similarly, the mean difference in global health status (2.28 [95% CI: −2.55 to 7.11]) also showed equivalence. However, the QALD by EQ-5D-5L was significantly lower in the ePRO group by 1.36 per 30 d (95% CI: −2.22 to −0.51; P = 0.0021). Conclusions Continual measurements of the HRQOL at home by ePRO may yield more detailed profiles of the HRQOL.

Confirmatory Psychometric Evaluation of the Axillary Sweating Daily Diary: A Vaildated Patient-Reported Outcome Measure to Assess Axillary Hyperhidrosis Sweating Severity

2017 ◽  
Vol 1 ◽  
pp. s94 ◽  
Author(s):  
Dee Anna Glaser ◽  
Adelaide A Hebert ◽  
Sheri Fehnel ◽  
Dana DiBenedetti ◽  
Lauren Nelson ◽  
...  
Keyword(s):  
Outcome Measure ◽  
Daily Diary ◽  
Psychometric Evaluation ◽  
Patient Reported Outcome ◽  
Axillary Hyperhidrosis ◽  
Patient Reported Outcome Measure ◽  
Patient Reported

Abstract Not AvailableDisclosure: Study supported by Dermira.

scholarly journals Buccal mucosal graft urethroplasty for radiation-induced urethral strictures: an evaluation using the extended Urethral Stricture Surgery Patient-Reported Outcome Measure (USS PROM)

2020 ◽  
Vol 38 (11) ◽  
pp. 2863-2872 ◽  
Author(s):  
Malte W. Vetterlein ◽  
◽  
Luis A. Kluth ◽  
Valentin Zumstein ◽  
Christian P. Meyer ◽  
...  
Keyword(s):  
Radiation Therapy ◽  
Urethral Stricture ◽  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Patient Reported Outcome Measure ◽  
Urethral Strictures ◽  
Buccal Mucosal Graft ◽  
Surgery Patient ◽  
Patient Reported ◽  
Radiation Induced

Abstract Objectives To evaluate objective treatment success and subjective patient-reported outcomes in patients with radiation-induced urethral strictures undergoing single-stage urethroplasty. Patients and methods Monocentric study of patients who underwent single-stage ventral onlay buccal mucosal graft urethroplasty for a radiation-induced stricture between January 2009 and December 2016. Patients were characterized by descriptive analyses. Kaplan–Meier estimates were employed to plot recurrence-free survival. Recurrence was defined as any subsequent urethral instrumentation (dilation, urethrotomy, urethroplasty). Patient-reported functional outcomes were evaluated using the validated German extension of the Urethral Stricture Surgery Patient-Reported Outcome Measure (USS PROM). Results Overall, 47 patients were available for final analyses. Median age was 70 (IQR 65–74). Except for two, all patients had undergone pelvic radiation therapy for prostate cancer. Predominant modality was external beam radiation therapy in 70% of patients. Stricture recurrence rate was 33% at a median follow-up of 44 months (IQR 28–68). In 37 patients with available USS PROM data, mean six-item LUTS score was 7.2 (SD 4.3). Mean ICIQ sum score was 9.8 (SD 5.4). Overall, 53% of patients reported daily leaking and of all, 26% patients underwent subsequent artificial urinary sphincter implantation. Mean IIEF-EF score was 4.4 (SD 7.1), indicating severe erectile dysfunction. In 38 patients with data regarding the generic health status and treatment satisfaction, mean EQ-5D index score and EQ VAS score was 0.91 (SD 0.15) and 65 (SD 21), respectively. Overall, 71% of patients were satisfied with the outcome. Conclusion The success rate and functional outcome after BMGU for radiation-induced strictures were reasonable. However, compared to existing long-term data on non-irradiated patients, the outcome is impaired and patients should be counseled accordingly.

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