Commentary: Statistical significance and clinical significance - A call to consider patient reported outcome measures, effect size, confidence interval and minimal clinically important difference (MCID)

2019 ◽  
Vol 23 (4) ◽  
pp. 690-694 ◽  
Author(s):  
Michael Fleischmann ◽  
Brett Vaughan
Keyword(s):  
Confidence Interval ◽  
Clinical Significance ◽  
Outcome Measures ◽  
Effect Size ◽  
Minimal Clinically Important Difference ◽  
Statistical Significance ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Clinically Important Difference ◽  
Patient Reported

scholarly journals Minimal Clinically Important Difference of Patient Reported Outcome Measures of Lower Extremity Injuries in Orthopedics

2017 ◽  
Vol 5 (2_suppl2) ◽  
pp. 2325967117S0007 ◽  
Author(s):  
Derya Çelik ◽  
Özge Çoban ◽  
Önder Kılıçoğlu
Keyword(s):  
Lower Extremity ◽  
Outcome Measures ◽  
Minimal Clinically Important Difference ◽  
Rating Scale ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Foot And Ankle ◽  
Clinically Important Difference ◽  
Outcome Score ◽  
Patient Reported

Purpose: MCID scores for outcome measures are frequently used evidence-based guides to gage meaningful changes. To conduct a systematic review of the quality and content of the the minimal clinically important difference (MCID) relating to 16 patient-rated outcome measures (PROM) used in lower extremity. Methods: We conducted a systematic literature review on articles reporting MCID in lower extremity outcome measures and orthopedics from January 1, 1980, to May 10, 2016. We evaluated MCID of the 16 patient reported outcome measures (PROM) which were Harris Hip Score (HHS), Oxford Hip Score (OHS), Hip Outcome Score (HOS), Hip Disability and Osteoarthritis Outcome Score (HOOS), The International Knee Documentation Committee Subjective Knee Form (IKDC), The Lysholm Scale, The Western Ontario Meniscal Evaluation Tool (WOMET), The Anterior Cruciate Ligament Quality of Life Questionnaire (ACL-QOL), The Lower Extremity Functional Scale (LEFS), The Western Ontario and Mcmaster Universities Index (WOMAC), Knee İnjury And Osteoarthritis Outcome Score (KOOS), Oxford Knee Score (OKS), Kujala Anterior Knee Pain Scale, The Victorian Institute of Sports Assessment Patellar Tendinosis (Jumper’s Knee) (VİSA-P), Tegner Activity Rating Scale, Marx Activity Rating Scale, Foot And Ankle Outcome Score (FAOS), The Foot Function Index (FFI), Foot And Ankle Ability Measure (FAAM), The Foot And Ankle Disability Index Score and Sports Module, Achill Tendon Total Rupture Score(ATRS), The Victorian İnstitute Of Sports Assesment Achilles Questionnaire(VİSA-A), American Orthopaedic Foot and Ankle Society (AOFAS). A search of the PubMed/MEDLINE, PEDro and Cochrane Cen¬tral Register of Controlled Trials and Web of Science databases from the date of inception to May 1, 2016 was conducted. The terms “minimal clinically important difference,” “minimal clinically important change”, “minimal clinically important improvement” “were combined with one of the PROM as mentioned above. Results: A total of 223 abstracts were reviewed and 119 articles chosen for full text review. Thirty articles were included in the final evaluation. The MCID was mostly calculated for WOMAC and frequently reported in knee and hip osteoartritis, knee and hip atrhroplasties, femoraasetabular impingement syndrome and focal cartilage degeneration. In addition, Receiver Operating Characteristic (ROC) analysis was the most used method to report MCID. Conclusions: MCID is an important concept used to determine whether a medical intervention improves perceived outcomes in patients. Despite an abundance of methods reported in the literature, little work in MCID estimation has been done in the PRAM related to lower extremity. There is a need for future studies in this regard.

AB1255 AGREEMENT BETWEEN PATIENT-REPORTED OUTCOME MEASURES COLLECTED VIA A SMARTPHONE APPLICATION VS A TOUCHSCREEN SOLUTION IN AN OUTPATIENT CLINIC AMONG PATIENTS WITH INFLAMMATORY ARTHRITIS: A RANDOMISED, WITHIN-PARTICIPANT TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1919-1920
Author(s):  
L. Uhrenholt ◽  
R. Christensen ◽  
L. Dreyer ◽  
A. Mortensen ◽  
E. M. Hauge ◽  
...  
Keyword(s):  
Confidence Interval ◽  
Outcome Measures ◽  
Outpatient Clinic ◽  
Inflammatory Arthritis ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
University Hospital ◽  
Patient Reported ◽  
The Mean ◽  
Equivalence Margin

Background:Patient-reported outcome measures (PROMs) are essential to understand the patient’s perception of arthritis activity. In Demark, PROMs are registered on a touchscreen in the outpatient clinic. However, some patients find it inconvenient due to e.g. waiting in queue, lack of privacy, uncomfortable seating position, reduced upper limb strength and dexterity with seeing the touchscreen due to deformity of the cervical spine. The widespread use of smartphones makes it possible for patients to register PROMs via an application (app) on their own device.Objectives:The primary aim is to evaluate the agreement (i.e. similarity) between the two devices assessed by the Health Assessment Questionnaire Disability Index (HAQ-DI) status among patients with inflammatory arthritis.Methods:The study was a randomised, crossover, agreement trial (NCT03486613) conducted at Aalborg University Hospital, Denmark. Participants were recruited through an invitation on the touchscreen in the outpatient clinic. Patients with an established diagnosis (≥ 12 months) of rheumatoid arthritis (RA), psoriatic arthritis (PsA) or axial spondyloarthritis (axSpA) and experience with the PROM questionnaires (≥ 3 previous registrations) were enrolled and randomised in ratio 1:1 (stratified by diagnosis) to PROM registration through the DANBIO app and the touchscreen in random order. Figure 1A and 1B shows the two devices.The sample size calculation was based on a prespecified equivalence margin of ±0.11 HAQ-DI points (i.e. ≤ half of the minimal important difference of 0.22 points) yielding a power of 99.2% for 60 enrolled patients. There was a wash-out period of 1-2 days between the two device registrations to minimise the potential carryover effect.A paired t-test was used to calculate the mean HAQ-DI score for the two devices and the difference in HAQ-DI score with a 95% confidence interval (CI). A Bland-Altman plot was used to assess limits of agreement (LoA).Results:60 patients (20 with RA, 20 with PsA and 20 with axSpA) were randomised of whom 51.7% were male. Mean age was 53.7 years (range 22-77) and mean disease duration was 12.5 years (range 1.0-34.8).Mean HAQ-DI was 0.608 (95%CI 0.437;0.779) for the DANBIO app and 0.614 (95%CI 0.446;0.783) for the touchscreen (Table 1). Agreement between scores obtained with the two devices is illustrated with Bland-Altman plots in figure 2A and 2B. The paired mean difference of HAQ-DI between the two devices was -0.006 (95%CI -0.0424; 0.030); thus the 95% confidence interval for the mean difference was within the prespecified equivalence margin of ±0.11 HAQ-DI points.Table 1.HAQ-DI scores, difference and LoA for the two devices.App, mean (SD)Touchscreen, mean (SD)Difference, mean (95%CI)LoAMissing valuesHAQ-DI (0-3)0.608 (0.656)0.614 (0.646)-0.006 (-0.042;0.030)-0.277;0.2641Conclusion:The current study showed no statistical or clinically important difference in HAQ-DI measurement captured by a smartphone app or outpatient touchscreen. Therefore, we feel confident that the two devices perform similarly enough to be used interchangeably in patients with inflammatory arthritis.Disclosure of Interests:Line Uhrenholt Speakers bureau: Abbvie, Eli Lilly and Novartis (not related to the submitted work), Robin Christensen: None declared, Lene Dreyer: None declared, Annette Mortensen Speakers bureau: MSD and Eli Lilly (not related to the submitted work)., Ellen-Margrethe Hauge Speakers bureau: Fees for speaking/consulting: MSD, AbbVie, UCB and Sobi; research funding to Aarhus University Hospital: Roche and Novartis (not related to the submitted work)., Niels Steen Krogh: None declared, Mikkel Kramme Abildtoft: None declared, Peter C. Taylor Grant/research support from: Celgene, Eli Lilly and Company, Galapagos, and Gilead, Consultant of: AbbVie, Biogen, Eli Lilly and Company, Fresenius, Galapagos, Gilead, GlaxoSmithKline, Janssen, Nordic Pharma, Pfizer Roche, and UCB, Salome Kristensen: None declared

scholarly journals Minimal clinically important difference in idiopathic pulmonary fibrosis

Breathe ◽  
2021 ◽  
Vol 17 (2) ◽  
pp. 200345
Author(s):  
Mohleen Kang ◽  
Lucian Marts ◽  
Jordan A. Kempker ◽  
Srihari Veeraraghavan
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Outcome Measures ◽  
Minimal Clinically Important Difference ◽  
Pulmonary Function Tests ◽  
Threshold Value ◽  
Patient Reported Outcome Measures ◽  
List Type ◽  
Clinically Important Difference ◽  
Patient Reported

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive fibrosing lung disease with an estimated median survival of 2–5 years and a significant impact on quality of life (QoL). Current approved medications, pirfenidone and nintedanib, have shown a reduction in annual decline of forced vital capacity but no impact on QoL. The minimal clinically important difference (MCID) is a threshold value for a change in a parameter that is considered meaningful by the patient rather than solely relying on statistically significant change in the parameter. This review provides a brief overview of the MCID methodology along with detailed discussion of reported MCID values for commonly used physiological measures and patient-reported outcome measures in IPF. While there is no gold standard methodology for determining MCID, there are certain limitations in the MCID literature in IPF, mainly the choice of death, hospitalisation and pulmonary function tests as sole anchors, and pervasive use of distribution-based methods which do not take into account the patient's input. There is a critical need to identify accurate thresholds of outcome measures that reflect patient's QoL over time in order to more precisely design and evaluate future clinical trials and to develop algorithms for patient-oriented management of IPF in outpatient clinics.Educational aimsTo understand the concept of MCID and the methods used to determine these values.To understand the indications and limitations of MCID values in IPF.

Associations Between Patient-Reported Outcome Measures of Asthma Control and Psychosocial Symptoms

2018 ◽  
Vol 58 (3) ◽  
pp. 307-312
Author(s):  
ChangWon C. Lee ◽  
Faye F. Holder-Niles ◽  
Linda Haynes ◽  
Jenny Chan Yuen ◽  
Corinna J. Rea ◽  
...  
Keyword(s):  
Confidence Interval ◽  
Asthma Control ◽  
Outcome Measures ◽  
Odds Ratio ◽  
Adjusted Odds Ratio ◽  
Multivariable Analysis ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Primary Care Center ◽  
Patient Reported

There is growing emphasis on using patient-reported outcome measures to enhance clinical practice. This study was a retrospective review of scores on the Childhood Asthma Control Test (C-ACT) and the Pediatric Symptom Checklist-17 (PSC-17) at a pediatric primary care center in Boston, Massachusetts. A total of 218 patients were selected at random using billing codes for well-child (WC) care and asthma, excluding complex medical conditions. Cutoff scores were used to identify uncontrolled asthma (C-ACT ⩽19) and clinically significant psychosocial symptoms (+PSC-17). Multiple logistic regression was used to measure associations between C-ACT ⩽19 and +PSC-17, adjusting for covariates. In multivariable analysis, C-ACT ⩽19 at WC visits was associated with +PSC-17 at WC visits (adjusted odds ratio = 3.2 [95% confidence interval = 1.3-8.6]). C-ACT ⩽19 at non-WC visits was also associated with +PSC-17 at WC visits (adjusted odds ratio = 3.1 [95% confidence interval = 1.2-8.9]). Patient-reported outcome measures of asthma control and psychosocial symptoms were positively correlated in this sample.

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