7. Abstract Title: Engaging patients in clinical trial planning: Exploring the concept of a minimal clinically important difference among patients with obstetrical antiphospholipid syndrome

2018 ◽  
Vol 170 ◽  
pp. 6-7
Author(s):  
Leslie Skeith ◽  
Taryn Taylor ◽  
Shannon Bates ◽  
Lisa Duffett ◽  
Robert Silver ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Antiphospholipid Syndrome ◽  
Minimal Clinically Important Difference ◽  
Clinically Important Difference

scholarly journals Engaging Patients in Clinical Trial Planning: Exploring the Concept of a Minimal Clinically Important Difference Among Patients with Obstetrical Antiphospholipid Syndrome

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4723-4723
Author(s):  
Leslie Skeith ◽  
Taryn S. Taylor ◽  
Shannon M. Bates ◽  
Lisa D. Duffett ◽  
Robert M. Silver ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Antiphospholipid Syndrome ◽  
Live Birth ◽  
Research Funding ◽  
Pregnancy Loss ◽  
Minimal Clinically Important Difference ◽  
Perceived Safety ◽  
Physician Group ◽  
Clinically Important Difference ◽  
The Mean

Abstract INTRODUCTION: Women with antiphospholipid syndrome are at increased risk of pregnancy loss. A large randomized controlled trial is still needed to confirm or refute the efficacy of low-molecular-weight heparin (LMWH) and aspirin (ASA) prophylaxis during pregnancy in women with antiphospholipid syndrome (APS) and past pregnancy loss. The minimal clinically important difference (MCID) is a statistical concept that quantifies the smallest amount of benefit from a treatment that a patient would identify as important, and is used to adequately power clinical trials. When planning a clinical trial, the MCID is most often determined by specialists in the field. How patients make sense of MCID as a concept, and how patients' perceptions of MCID compare to physician responses is unknown. In a challenging and high stakes situation, understanding different patients' perspectives must be considered when planning a large clinical trial. METHODS: Consecutive patients were approached from a specialty Thrombosis clinic and from the TIPPS (Thrombophilia in Pregnancy Prophylaxis Study) screening logs between January 2017 and March 2018 in Ottawa, Canada to complete a survey and interview. Patients were eligible if they met the revised Sapporo/Sydney laboratory criteria for antiphospholipid syndrome and had at least 1 late pregnancy loss or 2 early losses (<10 weeks gestation). A similar survey was distributed to 350 specialists identified through 5 professional organizations between May-Sept 2016. Both patient and physician groups had access to an introductory video. The mean MCID between the patient group and physician group was compared using an independent t-test, with p<0.05 being significant. Patient interview data were collected and analysed iteratively, in keeping with constructivist grounded theory methodology. RESULTS: There were 22 (33%) patients who completed surveys and 10 patients who completed semi-structured interviews. Among the physician group, 72 (20.6%) responded to the survey, including obstetricians (34.7%), hematologists (26.4%), general internists (18%), maternal fetal medicine specialists (6.9%) and other specialties (12.6%). Assuming a live birth rate of 60% in the ASA alone arm, the mean MCID answered by physicians was 11.6% (range 5-25%, SD 5.2), compared to the mean MCID answered by patients was 10.0% (range 5-20%, SD 6.4) (p=0.28). The mean number needed to treat (NNT) answered by both physicians and patients was 16. In the majority of cases from both groups, the related concepts of MCID and NNT did not match statistically (i.e. an MCID of 15% is a NNT of 7). An overarching theme from the patient survey comments and interviews was that statistics were somewhat irrelevant. The majority of women thought that "any increase in a chance of live birth" was worth it. However, there was variation in patient responses where some women would only accept a certain success rate of LMWH to justify its use because of drug cost or side effects. Some women acknowledged that they would not use LMWH themselves, but still advocated for other patients to have the option of LMWH use for a chance of a live birth. CONCLUSION: The mean MCID and NNT were similar between patient and physician groups. The low mean MCID and NNT values likely reflect the importance of the outcome studied (avoiding pregnancy loss) and the perceived safety of the proposed interventions of LMWH and/or ASA, which is supported by qualitative data from patient interviews. While the MCID and NNT responses were similar among physicians and patients, the understanding of statistics was somewhat limited in both groups. By better understanding patients' rationale and beliefs about LMWH use, we can better engage them in clinical trial planning and patient care and ensure that we are performing research that is relevant to patients. Disclosures Skeith: Leo Pharma: Honoraria; CSL Behring: Research Funding. Rodger:Biomerieux: Research Funding.

scholarly journals A prospective study to establish the minimal clinically important difference of the Mini-BESTest in individuals with stroke

2021 ◽  
pp. 026921552110251
Author(s):  
Marla K Beauchamp ◽  
Rudy Niebuhr ◽  
Patricia Roche ◽  
Renata Kirkwood ◽  
Kathryn M Sibley
Keyword(s):  
Stroke Rehabilitation ◽  
Minimal Clinically Important Difference ◽  
Clinical Importance ◽  
Minimal Detectable Change ◽  
Global Rating ◽  
Detectable Change ◽  
Clinically Important Difference ◽  
Before And After ◽  
Small Change ◽  
A Prospective Study

Objective: To determine the minimal clinically important difference of the Mini-BESTest in individuals’ post-stroke. Design: Prospective cohort study. Setting: Outpatient stroke rehabilitation. Subjects: Fifty outpatients with stroke with a mean (SD) age of 60.8 (9.4). Intervention: Outpatients with stroke were assessed with the Mini-BESTest before and after a course of conventional rehabilitation. Rehabilitation sessions occurred one to two times/week for one hour and treatment duration was 1.3–42 weeks (mean (SD) = 17.4(10.6)). Main measures: We used a combination of anchor- and distribution-based approaches including a global rating of change in balance scale completed by physiotherapists and patients, the minimal detectable change with 95% confidence, and the optimal cut-point from receiver operating characteristic curves. Results: The average (SD) Mini-BESTest score at admission was 18.2 (6.5) and 22.4 (5.2) at discharge (effect size: 0.7) ( P = 0.001). Mean change scores on the Mini-BESTest for patient and physiotherapist ratings of small change were 4.2 and 4.3 points, and 4.7 and 5.3 points for substantial change, respectively. The minimal detectable change with 95% confidence for the Mini-BESTest was 3.2 points. The minimally clinical importance difference was determined to be 4 points for detecting small changes and 5 points for detecting substantial changes. Conclusions: A change of 4–5 points on the Mini-BEST is required to be perceptible to clinicians and patients, and beyond measurement error. These values can be used to interpret changes in balance in stroke rehabilitation research and practice.

Letter: Contemporary Analysis of Minimal Clinically Important Difference in the Neurosurgical Literature

Neurosurgery ◽  
2021 ◽  
Author(s):  
Sumit Raj ◽  
Pradeep Chouksey ◽  
Adesh Shrivastava ◽  
Rakesh Mishra ◽  
Amit Agrawal
Keyword(s):  
Minimal Clinically Important Difference ◽  
Clinically Important Difference

scholarly journals The ENDOPAIN 4D Questionnaire: A New Validated Tool for Assessing Pain in Endometriosis

2021 ◽  
Vol 10 (15) ◽  
pp. 3216
Author(s):  
Anne Puchar ◽  
Pierre Panel ◽  
Anne Oppenheimer ◽  
Joseph Du Cheyron ◽  
Xavier Fritel ◽  
...  
Keyword(s):  
Minimal Clinically Important Difference ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
Dimensional Structure ◽  
Clinically Important Difference ◽  
Urinary Tract Symptoms ◽  
New Instrument ◽  
Patient Reported ◽  
Good Internal Consistency ◽  
Pain Symptoms

Objectives: To study the measurement properties, the responsiveness and the minimal clinically important difference of the ENDOPAIN-4D: a new questionnaire for assessing pain in endometriosis. Methods: A prospective, observational, multicentre study was conducted including all women ≥18 years consulting for symptomatic proven endometriosis between 1 January 2017 and 30 June 2018 and volunteering to participate. Each patient had to answer a new self-administered patient-reported outcome (PRO) questionnaires (the ENDOPAIN-4D) at inclusion (T0) and 12 months after medical or surgical treatment (T1). Criteria defined by COSMIN were used to validate the questionnaire's measurement properties. The minimal clinically important difference was estimated by the anchor-based method. Results: The study included 199 women. The ENDOPAIN-4D score had a four dimensional structure with good internal consistency (measured by Cronbach α): I) pain-related disability (α = 0.79), II) painful bowel symptoms (α = 0.80), III) dyspareunia (α = 0.83), and IV) painful urinary tract symptoms (α = 0.77). They produced four subscores that can be summed to obtain a single score (α = 0.61). The ENDOPAIN-4D total score ranged from 0 to 94.00 (mean ± SD: 46.7 ± 22). The total score was significantly correlated with the PROs used in endometriosis. Sensitivity to change was good with large effect sizes (ES) (mean of the differences: 36.3 p = 1.8 10−7, ES 0.76). The minimal clinically important difference of the global score was determined to be 10.9. Conclusions: The ENDOPAIN-4D questionnaire is easy to use, valid, and effective in assessing patient reported pain symptoms in women treated for endometriosis. This new instrument can be used as the primary outcome for future clinical trials and as a tool for routine patient follow-up.

The Foot and Ankle Outcome Score (FAOS) During Early Recovery After Ankle Fracture

2021 ◽  
pp. 107110072110028
Author(s):  
Peter Larsen ◽  
Mohammed Al-Bayati ◽  
Rasmus Elsøe
Keyword(s):  
Cohort Study ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Ankle Fracture ◽  
Minimal Clinically Important Difference ◽  
Ankle Fractures ◽  
Foot And Ankle ◽  
Early Recovery ◽  
Clinically Important Difference ◽  
Test Retest Reliability

Background: Several patient-reported outcome measures (PROMs) are available for assessing the outcomes following ankle fractures. This study aimed to evaluate validity, reliability, and responsiveness and detect the minimal clinically important difference of the Foot and Ankle Outcome Score (FAOS) in patients with ankle fractures. Methods: The study design is a prospective cohort study, including all patients treated both conservatively and surgically following an ankle fracture (AO-43A/B/C). Content validity, test-retest reliability, responsiveness, and minimal clinically important difference were evaluated from 14 days to 3 months following the fracture. Results: The study population consisted of 52 females and 24 males. The mean age was 52.0 years (range, 15-75 years). The percentage of patients at 12 weeks reporting the 5 subscales at least somewhat relevant were pain, 77%; symptoms, 75%; activities of daily living (ADL), 64%; sport, 81%; and quality of life (QOL), 88%. High test-retest reliability of the FAOS questionnaire was observed. The interclass coefficients were 0.78, 0.77, 0.71, 0.73, and 0.74 for the pain, symptoms, ADL, sport, and QOL subscales, respectively. Responsiveness was evaluated with high effect size for the symptoms (0.83), ADL (1.19), sport (4.36), and QOL (2.12) subscales. The minimal clinically important difference of the FAOS was 14 (95% CI, 12-17). Conclusion: The FAOS during early recovery after ankle fracture has high reliability and validity. Level of Evidence: Level II, prospective cohort study

scholarly journals How much improvement can satisfy patients? Exploring patients’ satisfaction 3 years after total knee arthroplasty

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Xiao Yu Fan ◽  
Jin Hui Ma ◽  
Xinjie Wu ◽  
Xin Xu ◽  
Lijun Shi ◽  
...  
Keyword(s):  
Total Knee Arthroplasty ◽  
Knee Arthroplasty ◽  
Minimal Clinically Important Difference ◽  
Important Change ◽  
Score Change ◽  
Patients Satisfaction ◽  
Clinically Important Difference ◽  
Total Knee ◽  
Hss Score ◽  
Varus Angle

Abstract Background Despite the innovations in total knee arthroplasty (TKA), there is still a subset of patients who do not acquire significant relief or expected satisfaction after primary TKA. However, this subgroup of patients still gains improvements more or less in terms of objective or quantified assessments after the procedure. The purpose of our study is to explore the factors that correlate with patients’ satisfaction and identify minimal clinically important difference (MCID) and minimum important change (MIC) in clinical parameters. Methods We conducted a retrospective study of 161 patients diagnosed with osteoarthritis who underwent unilateral total knee arthroplasty from January 2017 to December 2017. We collected the following parameters: body mass index (BMI), duration of disease, education level, depression state, preoperative flexion contracture angle of knee, HSS scores, 11-point NRS scores, and radiological parameters (preoperative minimal joint space width and varus angle of knee). The satisfaction was graded by self-reported scores in percentage (0–100). Results We revealed that 80.8% of patients were satisfied 3 years overall after primary TKA. HSS score change, NRS-Walking score change, age, and pre-mJSW showed significant difference between satisfied and dissatisfied group. The varus angle change revealed statistical significance according to the levels of satisfaction. Simple linear regression identified the MCID for HSS score to be 5.41 and for the NRS-Walking to be 1.24. The receiver operating characteristics (ROC) curve identified the MIC for HSS score to be 25.5 and for the NRS-Walking score to be 6.5. Conclusions In summary, we identified several factors that correlated with patients’ satisfaction independently after TKA in a long term. In addition, we revealed the minimal clinically important difference (MCID) and minimum important change (MIC) for HSS and NRS score in these patients.

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