Are We Considering the Whole Patient? The Impact of Physical and Mental Health on the Outcomes of Spine Care.

STUDY DESIGN: Retrospective, observational. OBJECTIVE: To evaluate the influence of baseline health status on the physical and mental health (MH) outcomes of spine patients. SUMMARY OF BACKGROUND DATA: Spine conditions can have a significant burden on both the physical and MH of patients. To date, few studies have evaluated the outcomes of both dimensions of health, particularly in nonoperative populations. MATERIALS AND METHODS: At their first visit to a multidisciplinary spine clinic, 2668 nonoperative patients completed the Patient-reported Outcomes Measurement Information System-Global Health (PROMIS-GH) instrument and a questionnaire evaluating symptoms and goals of care. Patients were stratified by their baseline percentile score of the MH and physical health (PH) components of the PROMIS-GH. Four groups of patients were compared based on the presence or absence of bottom quartile PH or MH scores. The primary end point was the achievement of a minimal clinically important difference (MCID) on the MH or PH components at follow-up. Multivariate regression assessed the predictors of MCID achievement. RESULTS: After controlling for demographics, symptoms, and goals, each 1-point increase in baseline PROMIS-GH mental score reduced the odds of achieving MH MCID by 9.0% ( P <0.001). Conversely, each 1-point increase in baseline GH-physical score increased the odds of achieving MCID by 4.5% ( P =0.005). Each 1-point increase in baseline GH-physical score reduced the odds of achieving PH MCID by 12.5% ( P <0.001), whereas each 1-point increase in baseline GH-mental score increased the odds of achieving MCID by 5.0% ( P <0.001). CONCLUSIONS: Spine patients presenting with the lowest levels of physical or MH were most likely to experience clinically significant improvement in those domains. However, lower levels of physical or mental health made it less likely that patients would experience significant improvement in the alternative domain. Physicians should evaluate and address the complex spine population holistically to maximize improvement in both physical and mental health status.

[Minimal clinically important difference of the frequency of bowel movement for patients with chronic severe functional constipation treated with acupuncture].

OBJECTIVE: To estimate the minimal clinically important difference (MCID) of the frequency of bowel movement for the patients with chronic severe functional constipation treated with acupuncture so as to provide the evidence for the clinical decision. METHODS: In this study, 813 patients with chronic severe functional constipation treated with acupuncture in two previous randomized controlled trials were included. Through the anchor-based method (anchored by the item 28 "satisfaction with previous treatment" of the patient assessment of constipation-quality of life [PAC-QOL]) and the distribution-based method, the MCID of the weekly frequency of complete spontaneous bowel movement (CSBM) and spontaneous bowel movement (SBM) was analyzed statistically in the patients. RESULTS: The MCID of the mean weekly frequency of CSBM and SBM was 1.3 times and 1.6 times in patients with chronic severe functional constipation treated with acupuncture, respectively. CONCLUSION: The mean increase of the weekly CSMB is ≥ 1.3 times and that of SBM is ≥ 1.6 times after treatment when compared with the baseline respectively, suggesting the clinical significance.

Ascertaining minimal clinically meaningful changes in symptoms of depression rated by the 15-item Centre for Epidemiologic Studies Depression Scale.

RATIONALE, AIMS AND OBJECTIVES: In clinical practise and in clinical studies on depression it is important to estimate whether changes in symptomatology measured by self-rating instruments are, in fact, clinically relevant. Therefore, the aim of the study was to estimate the clinical relevance of changes on the 15-item version of the Centre for Epidemiologic Studies Depression Scale (CES-D-15) based on the concept of the minimal clinically important difference (MCID). METHODS: Data was acquired from 4781 patients with depression symptoms from a German psychosomatic hospital who have been assessed using the CES-D-15 before and after treatment. Threshold values representing the MCID were estimated on the basis of mean change scores and sensitivity/specificity analyses. Patients' global impression of change, clinical (therapists') global impression of change and change in impairment severity were used as external anchor criteria. RESULTS: On average, the MCID was represented by a reduction of approximately 11 points in the CES-D-15, irrespective of age, gender, type of treatment and first or secondary diagnosis. However, higher baseline scores in the CES-D-15 required larger changes of raw values to represent a clinically important difference. CONCLUSIONS: Anchor-based values are suggested here as an estimation of the clinical relevance of changes in the CES-D-15. Thus, instead of relying solely on effect sizes, the evaluation of treatment outcomes should be supplemented by reporting the percentage of patients who have reached the MCID. Further examinations to verify our results in other patient populations and with other types of anchor criteria will be needed.

Effect of a multicomponent nutritional supplement on functional outcomes for Duchenne muscular dystrophy: A randomized controlled trial.

BACKGROUND & AIMS: Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular condition causing progressive muscle weakness and premature death. Whilst effective treatments such as gene therapy are developed, families often seek complementary therapies such as nutrition supplements to help their son maintain function; however, there is limited evidence supporting the use of nutritional supplements in DMD. This study aimed to compare the effect of a Standard nutritional supplement with an Enhanced nutritional supplement combining three nutriceuticals on functional outcomes in ambulatory boys with Duchenne muscular dystrophy (DMD). DESIGN: A 50-week double blinded, randomized, controlled crossover trial was conducted in four Australian neuromuscular centres. Primary outcome measures were 6-min walk distance (6MWD) and community ambulation (StepWatch™ Activity Monitoring). Secondary outcome measures included body composition and quality of life. Serum 25-hydroxyvitamin D was measured. RESULTS: Twenty-seven boys completed the intervention. Traditional crossover analysis demonstrated the Enhanced supplement compared to the Standard supplement was associated with a difference of +12 (95% CI: -16, 40) metres in 6MWD, +0.5 (95% CI: -53, 54) inactive minutes per day and -95 (95% CI: -887, 696) steps per day. A mixed effect model indicated a potentially clinically important effect of the Enhanced supplement on the 6MWD of +31 (95% CI: -19, 81) metres. Mean serum 25 hydroxyvitamin D levels at week 50 was 94 (95% CI: 84, 104) nmol/L. There was no observable effect of either supplement regime on body composition or quality of life. CONCLUSIONS: Whilst a positive effect of the Enhanced supplement on functional outcomes was observed, this finding was inconclusive due to the small sample size. The results do not support the use of combined nutritional supplements to improve body composition or quality of life in DMD. A dose of 2000 IU vitamin D was an adequate dose to raise serum 25-hydroxyvitamin D over 50 weeks. CLINICAL TRIAL REGISTRY: Registry #: ACTRN12610000462088, http://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?ACTRN=12610000462088.

What is the minimal important difference of pain intensity, mandibular function, and headache impact in patients with temporomandibular disorders? Clinical significance analysis of a randomized controlled trial.

BACKGROUND: There are insufficient studies providing Minimal Clinically Important Difference (MCID) for outcomes related to temporomandibular disorders (TMD). OBJECTIVES: (1) To provide the MCID of outcomes related to TMD using the Global Rating of Change Scale (GRCS) as an anchor. (2) To verify which outcomes can predict a moderate or large response to the treatment. STUDY DESIGN: Secondary analysis of a randomized controlled trial in subjects with TMD. METHODS: Sixty-one women with TMD were divided into intervention and control groups. Visual Analogue Scale (VAS), Headache Impact Test (HIT-6), pressure pain thresholds (PPTs) of masticatory muscles, Mandibular Function Impairment Questionnaire (MFIQ), and Craniocervical Flexion Test (CCFT) were collected at baseline and 5-weeks follow-up. RESULTS: Participants were divided based on their response to the treatment, according to the GRCS. MCID values were provided for subjects that moderately or largely improved to the treatment. MCID was between 0 and 1.90 for orofacial pain, around 2 points for the MFIQ, between 3 and 6.26 points for the HIT-6, around 0.2 kg/cm2 for the PPTs on masticatory muscles, around 2.5 mm for MMO and between 60 and 68 points for CCFT. Orofacial pain and HIT-6 were the most discriminative variables at determining whether patients would largely/moderately improve or would not improve after treatment. CONCLUSIONS: The values of MCID could be used as guidance for both clinical practice and research. Pain intensity and headache impact were the most predictive outcomes for improvement of the general health status of women with TMD.

Characteristics Associated With Clinically Important Treatment Responses in Women Undergoing Nonsurgical Therapy for Fecal Incontinence.

OBJECTIVE: To identify baseline clinical and demographic characteristics associated with clinically important treatment responses in a randomized trial of nonsurgical therapies for fecal incontinence (FI). METHODS: Women (N = 296) with FI were randomized to loperamide or placebo- and manometry-assisted biofeedback exercises or educational pamphlet in a 2 × 2 factorial design. Treatment response was defined in 3 ways from baseline to 24 weeks: minimal clinically important difference (MID) of -5 points in St. Mark's score, ≥50% reduction in FI episodes, and combined St. Mark's MID and ≥50% reduction FI episodes. Multivariable logistic regression models included baseline characteristics and treatment groups with and without controlling for drug and exercise adherence. RESULTS: Treatment response defined by St. Mark's MID was associated with higher symptom severity (adjusted odds ratio [aOR] 1.20, 95% confidence interval [CI] 1.11-1.28) and being overweight vs normal/underweight (aOR 2.15, 95% CI 1.07-4.34); these predictors remained controlling for adherence. Fifty percent reduction in FI episodes was associated with the combined loperamide/biofeedback group compared with placebo/pamphlet (aOR 4.04, 95% CI 1.36-11.98), St. Mark's score in the placebo/pamphlet group (aOR 1.29, 95% CI 1.01-1.65), FI subtype of urge vs urge plus passive FI (aOR 2.39, 95% CI 1.09-5.25), and passive vs urge plus passive FI (aOR 3.26, 95% CI 1.48-7.17). Controlling for adherence, associations remained, except St. Mark's score. DISCUSSION: Higher severity of FI symptoms, being overweight, drug adherence, FI subtype, and combined biofeedback and medication treatment were associated with clinically important treatment responses. This information may assist in counseling patients, regarding efficacy and expectations of nonsurgical treatments of FI.

Minimal Clinically Important Difference of Four Commonly Used Balance Assessment Tools in Individuals after Total Knee Arthroplasty: A Prospective Cohort Study.

BACKGROUND: Although balance is commonly assessed during the recovery of total knee arthroplasty (TKA), the minimal clinically important difference (MCID) values of frequently used balance assessment tools have not been established previously in this population. OBJECTIVE: To determine the MCID of four balance tests-ie, the Balance Evaluation Systems Test (BESTest), Mini-BESTest, Brief-BESTest, and the Berg Balance Scale (BBS)-in individuals post-TKA. DESIGN: Prospective cohort. SETTING: Outpatient rehabilitation. PARTICIPANTS: Inclusion criteria: (1) first primary TKA with diagnosed knee osteoarthritis; (2) aged 50-85 years. EXCLUSION CRITERIA: (1) TKA due to rheumatoid arthritis of the knee or traumatic injury; (2) known medical conditions that influence balance ability. One hundred forty-six participants were recruited, and 134 of them with complete data were included in the analysis. INTERVENTIONS: Participants received individualized physiotherapy, consisting of electrotherapy for pain and edema control, mobilization and strengthening exercises, and gait and balance training, once or twice per week between assessments. MAIN OUTCOME MEASUREMENTS: Participants were assessed on the BESTest, Mini-BESTest, Brief-BESTest, BBS, and Functional Gait Assessment (FGA) 2 and 4 weeks after surgery. The FGA was used as the anchor reference measure to calculate the MCID of the other four balance tests. A distribution-based approach was also employed to derive the MCID (ie, standardized effect size of 0.5). RESULTS: The BESTest (area under curve [AUC] = 0.811, 95% confidence interval [CI] 0.739-0.883) had the highest accuracy in detecting clinically important improvements on the FGA (≥4 points), followed by the Mini-BESTest (AUC = 0.782, 95% CI 0.704-0.860), Brief-BESTest (AUC = 0.701, 95% CI 0.618-0.795), and BBS (AUC = 0.586, 95% CI 0.490-0.682). The anchor- and distribution-based MCIDs were 6-8 for the BESTest, 1-2 for the Mini-BESTest, and 2-3 for the Brief-BESTest. CONCLUSIONS: Improvements exceeding MCIDs established above are indicative of significant progress in balance function post-TKA. The BBS is not a recommended tool due to its low AUC value.

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

In healthcare research an intervention may be statistically significant based on quantitative analysis; however, simultaneously it may be relatively insignificant to the health or quality of life of patients affected by a particular condition or disease being treated by the intervention - thus may be interpreted as having low clinical significance. An understanding of statistics is fundamental for evidence informed healthcare. Patient-reported outcome measures (PROMs) direct patients to evaluate aspects of their own health, including quality of life, disability and function. Data obtained from PROMs can be used to demonstrate the impact of healthcare interventions on these elements of a person's quality of life. To interpret outcome measure data for clinical decision making, a clinician must understand the concepts of statistical significance and clinical significance. This commentary explores the concepts of patient reported outcome measures (PROMs), their statistical and clinical significance, and explores their relationship with a practical example for the clinician. Limitations of research that only reports p-values and the need to consider effect size, confidence intervals, and minimal clinically important difference are also discussed. Together, these concepts can assist the clinician to evaluate whether an intervention may be suitable for their clinical practice.

Patient-reported outcomes of adalimumab, phototherapy, and placebo in the Vascular Inflammation in Psoriasis Trial: A randomized controlled study.

BACKGROUND: There are limited data about the impact of narrowband ultraviolet B phototherapy on patient-reported measures of health-related quality of life. OBJECTIVE: To evaluate the impact of adalimumab and phototherapy on health-related quality of life. METHODS: We examined patient-reported outcomes from a multicenter, randomized, placebo-controlled trial (ClinicalTrials.gov no. NCT01553058). The Dermatology Life Quality Index and EQ-5D-3L were evaluated every 4 weeks. RESULTS: We enrolled 97 patients: 30.9% were female, mean age was 43.5 years (standard deviation, 14.0), and median Psoriasis Area and Severity Index score was 16.7 (interquartile range, 13.9-21.6). At week 12, patients being treated with adalimumab (odds ratio [OR], 2.88; 95% confidence interval [CI], 1.02-8.17) and phototherapy (OR, 8.83; 95% CI, 2.47-31.57) were more likely to achieve the minimal clinically important difference in the Dermatology Life Quality Index compared with those receiving placebo. There were higher odds of achieving the minimal clinically important difference for the EQ-5D-3L Index score when comparing phototherapy versus placebo (OR, 9.78; 95% CI, 2.99-31.95) and phototherapy versus adalimumab (OR, 4.07; 95% CI, 1.42-11.70). LIMITATIONS: Small sample size, secondary analysis, generalizability. CONCLUSION: Phototherapy and adalimumab both improve skin-related quality of life and overall health-related quality of life compared with placebo in patients with psoriasis; however, patients treated with phototherapy achieved more improvement in overall health-related quality of life compared with patients treated with adalimumab.

Directional preference constructs for patients' neck pain in the absence of centralization.

Objectives: We conducted a secondary analysis of a prospective, observational cohort study to (1) report the prevalence of Directional Preference (DP) constructs at first examination for patients with cervical spine challenges, and (2) determine the association between DP constructs and clinical outcomes at discharge from physical therapy. Methods: We analyzed data collected from 718 consecutive patients who presented to outpatient, private practice clinics with primary complaints of non-specific neck pain; 200 patients met the inclusion criteria and completed first examination and discharge data. Statistical analysis determined the association between DP constructs at first examination and clinical outcomes at discharge. Results: The findings in this investigation were that (1) the most prevalent DP constructs at first examination were related to ROM and pain intensity including Patient Reported Improvement in ROM (79.5%), Increase in Spine ROM (32.5%), and Pain Intensity Change (15.0%), (2) all DP groups improved and met the MCID for disability and pain intensity change at discharge except for the group that did not exhibit Increase in Spine ROM for pain intensity, (3) no clinically significant differences in pain intensity or disability existed between DP groups at discharge, and (4) 28.5% and 6.5% of patients exhibited a relative increase in cervical spine extension and flexion ROM, respectively, post-repeated movement testing on the first examination. Discussion: The most prevalent DP constructs at first examination were related to ROM and pain intensity, and each was associated with a comparable clinical trajectory in terms of pain and disability outcomes at discharge. The findings of this study help Mechanical Diagnosis and Therapy (MDT) providers understand the common DP constructs encountered in routine clinical practice and their relation to pain and disability outcomes for patients with cervical spine challenges. Level of Evidence: 2b.