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

Fleischmann, Michael; Vaughan, Brett

doi:10.1016/j.jbmt.2019.02.009

Cited by 26 publications

(24 citation statements)

References 40 publications

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“…Many of the participants improved in multiple outcome measures as measured by improvement above an MCID value. MCID is interpreted as an improvement that is relevant to individual participants [65] and is proposed as the "smallest difference in score in the domain of interest which patients perceive as beneficial and which would mandate, in the absence of troublesome side effects and excessive cost, a change in the patient's management" [66]. The use of a half standard deviation as a simple measure of MCID as proposed by Norman et al [37] is not universally accepted [67] and its use in our study with its small number of participants has resulted in a standard deviation that is higher than would be expected with a larger cohort, leading to imprecision in detecting an MCID change, with an MCID change more difficult to achieve, and an underestimate of the numbers of participants that show a substantial improvement.…”

Section: Discussionmentioning

confidence: 99%

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: a prospective proof-of-concept study

Liebert

Bicknell²,

Laakso

et al. 2021

BMC Neurol

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Background Parkinson’s disease (PD) is a progressive neurodegenerative disease with no cure and few treatment options. Its incidence is increasing due to aging populations, longer disease duration and potentially as a COVID-19 sequela. Photobiomodulation (PBM) has been successfully used in animal models to reduce the signs of PD and to protect dopaminergic neurons. Objective To assess the effectiveness of PBM to mitigate clinical signs of PD in a prospective proof-of-concept study, using a combination of transcranial and remote treatment, in order to inform on best practice for a larger randomized placebo-controlled trial (RCT). Methods Twelve participants with idiopathic PD were recruited. Six were randomly chosen to begin 12 weeks of transcranial, intranasal, neck and abdominal PBM. The remaining 6 were waitlisted for 14 weeks before commencing the same treatment. After the 12-week treatment period, all participants were supplied with PBM devices to continue home treatment. Participants were assessed for mobility, fine motor skills, balance and cognition before treatment began, after 4 weeks of treatment, after 12 weeks of treatment and the end of the home treatment period. A Wilcoxon Signed Ranks test was used to assess treatment effectiveness at a significance level of 5%. Results Measures of mobility, cognition, dynamic balance and fine motor skill were significantly improved (p < 0.05) with PBM treatment for 12 weeks and up to one year. Many individual improvements were above the minimal clinically important difference, the threshold judged to be meaningful for participants. Individual improvements varied but many continued for up to one year with sustained home treatment. There was a demonstrable Hawthorne Effect that was below the treatment effect. No side effects of the treatment were observed. Conclusions PBM was shown to be a safe and potentially effective treatment for a range of clinical signs and symptoms of PD. Improvements were maintained for as long as treatment continued, for up to one year in a neurodegenerative disease where decline is typically expected. Home treatment of PD by the person themselves or with the help of a carer might be an effective therapy option. The results of this study indicate that a large RCT is warranted. Trial registration Australian New Zealand Clinical Trials Registry, registration number: ACTRN12618000038291p, registered on 12/01/2018.

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Section: Discussionmentioning

confidence: 99%

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: a prospective proof-of-concept study

Liebert

Bicknell²,

Laakso

et al. 2021

BMC Neurol

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“…We started with a fully saturated model that included nativity, gestational groups, covariates, confounders, and interaction terms, allowing for the exploration of the simultaneous joint effects of nativitycovariate and covariate-covariate on PTB and early term birth, beyond the explanatory paradigm of the sum of the individual effect of nativity or maternal and obstetric characteristics; and 4) Action: We use knowledge generated from our study to disrupt the reliance of clinical outcomes as defined in administrative databases as the predominant approach to examining the causes of PTB inequities. Instead, we propose the use of a patient reported outcome or experience measure of PTB among Black women with GDM in clinical settings, particularly when clinical significance outweighs statistical significance [44].…”

Section: Introductionmentioning

confidence: 99%

Preterm birth and nativity among Black women with gestational diabetes in California, 2013–2017: a population-based retrospective cohort study

Scott

Chambers

Baer

et al. 2020

BMC Pregnancy Childbirth

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Background Despite the disproportionate prevalence of gestational diabetes (GDM) and preterm birth (PTB) and their associated adverse perinatal outcomes among Black women, little is known about PTB among Black women with GDM. Specifically, the relationship between PTB by subtype (defined as indicated PTB and spontaneous PT labor) and severity, GDM, and nativity has not been well characterized. Here we examine the risk of PTB by severity (early < 34 weeks, late 34 to 36 weeks) and early term birth (37 to 38 weeks) by nativity among Black women with GDM in California. Methods This retrospective cohort study used linked birth certificate and hospital discharge data for 8609 of the 100,691 self-identifying non-Hispanic Black women with GDM who had a singleton live birth between 20 and 44 weeks gestation in California in 2013–2017. Adjusted odds ratios (aOR) and 95% confidence intervals (CIs) were examine risks for PTB, by severity and subtype, and early term birth using multivariate regression modeling. Results Approximately, 83.9% of Black women with GDM were US-born and 16.1% were foreign-born. The overall prevalence of early PTB, late PTB, and early term birth was 3.8, 9.5, and 29.9%, respectively. Excluding history of prior PTB, preeclampsia was the greatest overall risk factor for early PTB (cOR = 6.7, 95%, CI 5.3 to 8.3), late PTB (cOR = 4.3, 95%, CI 3.8 to 5.0), and early term birth (cOR = 1.8, 95%, CI 1.6 to 2.0). There was no significant difference in the prevalence of PTB by subtypes and nativity (p = 0.5963). Overall, 14.2% of US- compared to 8.9% of foreign-born women had a PTB (early PTB: aOR = 0.56, 95%, CI 0.38 to 0.82; late PTB: aOR = 0.57, 95%, CI 0.45 to 0.73; early term birth: aOR = 0.67, 95%, CI 0.58 to 0.77). Conclusions Foreign-born status remained protective of PTB, irrespective of severity and subtype. Preeclampsia, PTB, and GDM share pathophysiologic mechanisms suggesting a need to better understand differences in perinatal stress, chronic disease, and vascular dysfunction based on nativity in future epidemiologic studies and health services research.

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“…Many of the participants improved in multiple outcome measures as measured by improvement above an MCID value. MCID is interpreted as an improvement that is relevant to individual participants (65) and is proposed as the "smallest difference in score in the domain of interest which patients perceive as beneficial and which would mandate, in the absence of troublesome side effects and excessive cost, a change in the patient's management" (66). The use of a half standard deviation as a simple measure of MCID as proposed by Norman et al ( 37) is not universally accepted (67) and its use in our study with its small number of participants has resulted in a standard deviation that is higher than would be expected with a larger cohort, leading to imprecision in detecting an MCID change, with an MCID change more difficult to achieve, and an underestimate of the numbers of participants that show a substantial improvement.…”

Section: Discussionmentioning

confidence: 99%

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: A prospective proof-of-concept study

Liebert

Bicknell

Laakso

et al. 2021

Preprint

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BACKGROUND: Parkinson's disease (PD) is a progressive neurodegenerative disease with no cure and few treatment options. Its incidence is increasing due to aging populations, longer disease duration and potentially as a COVID-19 sequela. Photobiomodulation (PBM) has been successfully used in animal models to reduce the signs of PD and to protect dopaminergic neurons. OBJECTIVE: To assess the effectiveness of PBM to mitigate clinical signs of PD in a prospective proof-of-concept study, using a combination of transcranial and remote treatment, in order to inform on best practice for a larger randomized placebo-controlled trial (RCT). METHODS: Twelve participants with idiopathic PD were recruited. Six were randomly chosen to begin 12 weeks of transcranial, intranasal, neck and abdominal PBM. The remaining 6 were waitlisted for 14 weeks before commencing treatment. After the 12-week treatment period, all participants were supplied with PBM devices to continue home treatment. Participants were assessed for mobility, fine motor skills, balance and cognition before treatment began, after 4 weeks of treatment, after 12 weeks of treatment and the end of the home treatment period. A Wilcoxon Signed Ranks test was used to assess treatment effectiveness at a significance level of 5%. RESULTS: Measures of mobility, cognition, dynamic balance and fine motor skill were significantly improved (p<0.05) with PBM treatment for 12 weeks and up to one year. Many individual improvements were above the minimal clinically important difference, the threshold judged to be meaningful for participants. Individual improvements varied but many continued for up to one year with sustained home treatment. There was a demonstrable Hawthorne Effect that was below the treatment effect. No side effects of the treatment were observed. CONCLUSIONS: PBM was shown to be a safe and potentially effective treatment for a range of clinical signs and symptoms of PD. Improvements were maintained for as long as treatment continued, for up to one year in a neurodegenerative disease where decline is typically expected. Home treatment of PD by the person themselves or with the help of a carer might be an effective therapy option. The results of this study indicate that a large RCT is warranted.

show abstract

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

Cited by 26 publications

References 40 publications

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: a prospective proof-of-concept study

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: a prospective proof-of-concept study

Preterm birth and nativity among Black women with gestational diabetes in California, 2013–2017: a population-based retrospective cohort study

Improvements in clinical signs of Parkinson’s disease using photobiomodulation: A prospective proof-of-concept study

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