scholarly journals Clinical outcome evaluations and CBT response prediction in Myotonic Dystrophy

Author(s):  
Daniel van As ◽  
Kees Okkersen ◽  
Guillaume Bassez ◽  
Benedikt Schoser ◽  
Hanns Lochmuller ◽  
...  

Background: The European OPTIMISTIC clinical trial has demonstrated a significant, yet heterogenous effect of Cognitive Behavioural Therapy (CBT) for Myotonic Dystrophy type 1 (DM1) patients. One of its remaining aims was the assessment of efficacy and adequacy of clinical outcome measures, including the relatively novel primary trial outcome, the DM1-Activ-c questionnaire. Objectives: Assessment of the relationship between the Rasch-built DM1-Activ-c questionnaire and 26 commonly used clinical outcome measurements. Identification of variables associated with CBT response in DM1 patients. Methods: Retrospective analysis of the to date largest clinical trial in DM1 (OPTIMISTIC), comprising of 255 genetically confirmed DM1 patients randomized to either standard care or CBT with optionally graded exercise therapy. Correlations of 27 different outcome measures were calculated at baseline (cross-sectional) and of their respective intervention induced changes (longitudinal). Bootstrap enhanced Elastic-Net (BeEN) regression was validated and implemented to select variables associated with CBT response. Results: In cross-sectional data, DM1-Activ-c correlated significantly with the majority of other outcome measures, including Six Minute Walk Test and Myotonic Dystrophy Health Index. Fewer and weaker significant longitudinal correlations were observed. Nine variables potentially associated with CBT response were identified, including measures of disease severity, executive cognitive functioning and perceived social support. Conclusions: The DM1-Activ-c questionnaire appears to be a well suited cross-sectional instrument to assess a variety of clinically relevant dimensions in DM1. Yet, apathy and experienced social support measures were less well captured. CBT response was heterogenous, requiring careful selection of outcome measures for different disease aspects.

2021 ◽  
pp. 1-16
Author(s):  
Daniël van As ◽  
Kees Okkersen ◽  
Guillaume Bassez ◽  
Benedikt Schoser ◽  
Hanns Lochmüller ◽  
...  

Background: The European OPTIMISTIC clinical trial has demonstrated a significant, yet heterogenous effect of Cognitive Behavioural Therapy (CBT) for Myotonic Dystrophy type 1 (DM1) patients. One of its remaining aims was the assessment of efficacy and adequacy of clinical outcome measures, including the relatively novel primary trial outcome, the DM1-Activ-c questionnaire. Objectives: Assessment of the relationship between the Rasch-built DM1-Activ-c questionnaire and 26 commonly used clinical outcome measurements. Identification of variables associated with CBT response in DM1 patients. Methods: Retrospective analysis of the to date largest clinical trial in DM1 (OPTIMISTIC), comprising of 255 genetically confirmed DM1 patients randomized to either standard care or CBT with optionally graded exercise therapy. Correlations of 27 different outcome measures were calculated at baseline (cross-sectional) and of their respective intervention induced changes (longitudinal). Bootstrap enhanced Elastic-Net (BeEN) regression was validated and implemented to select variables associated with CBT response. Results: In cross-sectional data, DM1-Activ-c correlated significantly with the majority of other outcome measures, including Six Minute Walk Test and Myotonic Dystrophy Health Index. Fewer and weaker significant longitudinal correlations were observed. Nine variables potentially associated with CBT response were identified, including measures of disease severity, executive cognitive functioning and perceived social support. Conclusions: The DM1-Activ-c questionnaire appears to be a well suited cross-sectional instrument to assess a variety of clinically relevant dimensions in DM1. Yet, apathy and experienced social support measures were less well captured. CBT response was heterogenous, requiring careful selection of outcome measures for different disease aspects.


2016 ◽  
Vol 45 (1) ◽  
pp. 79-84 ◽  
Author(s):  
David Hambrook ◽  
Tracey Taylor ◽  
Victoria Bream

Background: Paruresis, or “shy bladder syndrome”, is a relatively common anxiety disorder, yet little is known about the causes of, and effective treatments for this disabling condition. Aim: This report describes a case study in which a man (Peter) presenting with paruresis was treated using formulation-driven CBT, which aimed to address the idiosyncratic processes that were maintaining his anxiety and avoidance of urinating in public. Method: Peter attended 12 sessions of CBT including one follow-up session a month after treatment had ended. Treatment involved collaboratively developing an idiosyncratic case conceptualization (identifying longitudinal and cross-sectional factors involved in the development and maintenance of his difficulties), followed by a number of standard cognitive and behavioural interventions commonly used in evidence-based CBT protocols for other anxiety disorders. Peter completed sessional outcome measures of paruresis symptomatology, anxiety, depression, social anxiety and functional impairment. Results: Peter subjectively found the intervention helpful and his scores on all of the outcome measures reduced over the course of his therapy, and were maintained at one month follow-up. Conclusions: This report adds to the scarce literature regarding effective treatments for individuals suffering with paruresis. Limitations of the design are acknowledged and ideas for further research in this area are discussed.


2020 ◽  
Vol 29 (4) ◽  
Author(s):  
Annemarie I. Luik ◽  
Antonia Marsden ◽  
Richard Emsley ◽  
Alasdair L. Henry ◽  
Richard Stott ◽  
...  

2020 ◽  
pp. medhum-2019-011807
Author(s):  
Steven Lubet ◽  
David Tuller

In a recent article in Medical Humanities, Sharpe and Greco characterise myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) as an ‘illness without disease’, citing the absence of identified diagnostic markers. They attribute patients’ rejection of psychological and behavioural interventions, such as cognitive–behavioural therapy (CBT) and graded exercise therapy (GET), to a ‘paradox’ resulting from a supposed failure to acknowledge that ‘there is no good objective evidence of bodily disease’. In response, we explain that understandings about the causes of and treatments for medical complaints have shifted across centuries, and that conditions once thought to be ‘psychosomatic’ have later been determined to have physiological causes. We also note that Sharpe and Greco do not disclose that leading scientists and physicians believe that ME/CFS is a biomedical disease, and that numerous experts, not just patients, have rejected the research underlying the CBT/GET treatment approach. In conclusion, we remind investigators that medical classifications are always subject to revision based on subsequent research, and we therefore call for more humility before declaring categorically that patients are experiencing ‘illness without disease’.


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