Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06562686
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-12
Brief Title:
Assessment of Methods Used in Evaluating Balance Rehabilitation in Parkinsons Patients
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Assessment of Methods Used in Evaluating Balance Rehabilitation in Parkinsons Patients Determination of Responsiveness and Minimal Clinically Important Difference Values
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Balance rehabilitation holds an important place in the treatment of Parkinsons patients Before and after treatment patients are evaluated using various measurement methods In calculations of changes post-treatment although statistically significant changes are detected clinical differences are often not observed Jaeschke et al developed the concept of minimal clinically important difference MCID to address this They have worked on methods to determine the level of MCID These measurements help clinicians understand which results can be interpreted as clinically meaningful for the patient
Responsiveness refers to how sensitive a measurement tool is to changes whereas MCID focuses on determining whether these changes are clinically significant Both concepts are crucial for understanding and interpreting the performance of measurement tools
Identifying which patients have a high risk of balance problems and falls and screening those at risk is important for making treatment decisions Determining which change values are clinically significant MCID and identifying which tests are more sensitive in detecting changes responsiveness are essential in monitoring patients
Responsiveness refers to how sensitive a measurement tool is to changes whereas MCID focuses on determining whether these changes are clinically significant Both concepts are crucial for understanding and interpreting the performance of measurement tools
Identifying which patients have a high risk of balance problems and falls and screening those at risk is important for making treatment decisions Determining which change values are clinically significant MCID and identifying which tests are more sensitive in detecting changes responsiveness are essential in monitoring patients
Detailed Description:
Parkinsons disease PD is a chronic progressive neurodegenerative disease characterized by the degeneration or loss of dopaminergic neurons in the nigrostriatal and other neuronal systems along with the formation of Lewy bodies in dopaminergic neurons and it manifests with both motor and non-motor symptoms and findings It is the second most common neurodegenerative disease after Alzheimers disease The average onset age is between 60-80 years It is more commonly seen in men and the white race In Turkey the prevalence has been reported as 111100000 In societies where the average lifespan is increasing the frequency of PD and the associated economic burden are also expected to increase
The etiology of Parkinsons disease includes environmental genetic and aging-related processes These factors trigger mechanisms leading to neuron degeneration and loss causing PD
The clinical presentation of Parkinsons disease consists of motor and non-motor symptoms and findings The main motor symptoms are bradykinesia rigidity postural instability and resting tremor Other observed findings include akinesia freezing phenomenon postural and gait disorders reciprocal movement disorder masked facial expression impaired orofacial functions and speech swallowing disorder respiratory function impairment loss of hand dexterity and coordination Cognitive impairments depression sleep disorders autonomic dysfunction loss of smell fatigue and sialorrhea are among the non-motor symptoms
Patients with primary motor findings and accompanying symptoms are evaluated using the Parkinsons Disease United Kingdom Brain Bank Criteria and the National Institute of Neurological Disorders and Stroke NINDS diagnostic criteria A definitive diagnosis is made by pathologically demonstrating Lewy bodies in a biopsy The Hoehn-Yahr Scale is frequently used to determine the severity of the disease
The treatment of Parkinsons disease includes medical treatment surgical treatment and rehabilitation Rehabilitation plays an important role at all stages of PD In the early stages it helps maintain well-being and improve general conditioning in the later stages it is crucial for coping with motor and non-motor complications
Postural instability and balance disorders in Parkinsons patients lead to sudden falls progressive disability and immobility Patients fear of falling causes them to limit their activities which increases immobility and balance problems Preventing falls and maintaining mobility should involve a multidisciplinary treatment and rehabilitation program Various rehabilitation approaches are available including conventional therapy music therapy aquatic therapy dance therapy virtual reality therapy robot-assisted gait therapy and tai chi Many studies in the literature evaluate the effectiveness of rehabilitation approaches aimed at balance and mobility in Parkinsons disease
Balance disorders are a significant motor problem in individuals with PD It is necessary to identify at-risk patients through appropriate tests and to organize treatment accordingly Although there are many scales available to evaluate patients balance to determine their rehabilitation needs there is no clarity on which scales are more sensitive in assessing changes Additionally knowing which change scores in the scales the investigators use are clinically significant is important for evaluating treatment outcomes Knowing the sensitivity and minimal clinically important difference MCID values of tests for balance and fall concerns in Parkinsons patients can guide clinicians in selecting scales for screening patients before treatment and understanding which changes are clinically significant when evaluating outcomes after treatment
In 1989 Jaeschke et al developed the concept of minimal clinically important difference MCID Jaeschke and colleagues noted that while statistically significant changes were often detected in post-treatment change calculations clinically significant differences were not observed and attempted to determine the smallest clinically important change level This allowed patients to directly participate in the process of evaluating their condition These measurements help clinicians understand which treatments yield better results MCID is the smallest change that a patient would identify as important and that would indicate a difference in the patients management After R A Fisher defined the p-value and the 005 cutoff point all clinical studies began to evaluate whether results were statistically significant but the p-value despite being an objective result has two major issues One is failing to detect a difference due to insufficient sample size despite an important difference Type II error and the other is that the statistically significant difference does not actually represent a meaningful change for the patient Therefore statistical significance may not carry clinical importance MCID helps clinicians determine whether a treatment has a meaningful effect on the patient Additionally MCID is a valuable tool in planning scientific studies and calculating sample size Understanding the minimal clinically important difference helps researchers design studies that are both significant and applicable to clinical practice In summary MCID is a critical concept that bridges clinical practice and research emphasizing the impact of treatments from the patients perspective Understanding and applying MCID is essential for making correct treatment decisions and conducting meaningful scientific studies in healthcare It is important to remember that the concept of MCID is variable and can differ between health conditions In recent years clinicians and researchers have started to focus on patient-reported outcome assessments
The investigators also aim to determine the MCID values of measurement tools used to evaluate balance in Parkinsons disease in our study
There are two commonly used methods for calculating MCID
Anchor-based approach Distribution-based approach In the anchor-based approach a comparison is made between a result measure reflecting the patients condition eg post-treatment measurement value and an external criterion This external criterion can be the patients own perception or the clinicians assessment In this method changes between scores are compared with an anchor question that serves as a reference to determine whether the patient has improved compared to baseline To understand the perception of change a global rating scale much worse a little worse almost the same a little better and much better can be used The anchor question should be easily understandable and relevant to patients
The distribution-based approach attempts to estimate the likelihood that a detected difference in measurement tools is meaningful It uses statistical data such as fractions of standard deviation effect size and mean standard error It does not include the perceptions based on the experiences of experts or patients Since it does not include individual patient perceptions it should not be the first choice for determining MCID In this method as there is no anchor question to evaluate what is important for patients significant and clinically meaningful results cannot be defined In fact scores calculated with distribution-based methods are sometimes referred to as minimal detectable change instead of MCID Therefore this method is not recommended as the first choice for determining MCID
However some authors have suggested that a single MCID value can be misleading and that providing a range might be more appropriate For this reason in our study the investigators will use both anchor-based and distribution-based approaches to calculate MCID aiming to find a range of values In the anchor-based approach the investigators plan to use two anchor questions a patient-based and a clinician-based global assessment scale
Responsiveness refers to the ability of a measurement tool to detect true changes among individuals A sensitive scale responds accurately to the changes experienced Responsiveness is typically assessed using statistical analyses such as effect size and standard deviation and is used to evaluate the performance of measurement tools
While responsiveness indicates how sensitive a measurement tool is to changes MCID indicates whether these changes are meaningful from the patients or clinicians perspective In other words a responsive measurement tool has the potential to capture clinically significant changes Both concepts are important for understanding and interpreting the performance of measurement tools Determining the responsiveness and MCID of a measurement tool provides clinicians with a more comprehensive assessment in research studies and patient follow-up
The etiology of Parkinsons disease includes environmental genetic and aging-related processes These factors trigger mechanisms leading to neuron degeneration and loss causing PD
The clinical presentation of Parkinsons disease consists of motor and non-motor symptoms and findings The main motor symptoms are bradykinesia rigidity postural instability and resting tremor Other observed findings include akinesia freezing phenomenon postural and gait disorders reciprocal movement disorder masked facial expression impaired orofacial functions and speech swallowing disorder respiratory function impairment loss of hand dexterity and coordination Cognitive impairments depression sleep disorders autonomic dysfunction loss of smell fatigue and sialorrhea are among the non-motor symptoms
Patients with primary motor findings and accompanying symptoms are evaluated using the Parkinsons Disease United Kingdom Brain Bank Criteria and the National Institute of Neurological Disorders and Stroke NINDS diagnostic criteria A definitive diagnosis is made by pathologically demonstrating Lewy bodies in a biopsy The Hoehn-Yahr Scale is frequently used to determine the severity of the disease
The treatment of Parkinsons disease includes medical treatment surgical treatment and rehabilitation Rehabilitation plays an important role at all stages of PD In the early stages it helps maintain well-being and improve general conditioning in the later stages it is crucial for coping with motor and non-motor complications
Postural instability and balance disorders in Parkinsons patients lead to sudden falls progressive disability and immobility Patients fear of falling causes them to limit their activities which increases immobility and balance problems Preventing falls and maintaining mobility should involve a multidisciplinary treatment and rehabilitation program Various rehabilitation approaches are available including conventional therapy music therapy aquatic therapy dance therapy virtual reality therapy robot-assisted gait therapy and tai chi Many studies in the literature evaluate the effectiveness of rehabilitation approaches aimed at balance and mobility in Parkinsons disease
Balance disorders are a significant motor problem in individuals with PD It is necessary to identify at-risk patients through appropriate tests and to organize treatment accordingly Although there are many scales available to evaluate patients balance to determine their rehabilitation needs there is no clarity on which scales are more sensitive in assessing changes Additionally knowing which change scores in the scales the investigators use are clinically significant is important for evaluating treatment outcomes Knowing the sensitivity and minimal clinically important difference MCID values of tests for balance and fall concerns in Parkinsons patients can guide clinicians in selecting scales for screening patients before treatment and understanding which changes are clinically significant when evaluating outcomes after treatment
In 1989 Jaeschke et al developed the concept of minimal clinically important difference MCID Jaeschke and colleagues noted that while statistically significant changes were often detected in post-treatment change calculations clinically significant differences were not observed and attempted to determine the smallest clinically important change level This allowed patients to directly participate in the process of evaluating their condition These measurements help clinicians understand which treatments yield better results MCID is the smallest change that a patient would identify as important and that would indicate a difference in the patients management After R A Fisher defined the p-value and the 005 cutoff point all clinical studies began to evaluate whether results were statistically significant but the p-value despite being an objective result has two major issues One is failing to detect a difference due to insufficient sample size despite an important difference Type II error and the other is that the statistically significant difference does not actually represent a meaningful change for the patient Therefore statistical significance may not carry clinical importance MCID helps clinicians determine whether a treatment has a meaningful effect on the patient Additionally MCID is a valuable tool in planning scientific studies and calculating sample size Understanding the minimal clinically important difference helps researchers design studies that are both significant and applicable to clinical practice In summary MCID is a critical concept that bridges clinical practice and research emphasizing the impact of treatments from the patients perspective Understanding and applying MCID is essential for making correct treatment decisions and conducting meaningful scientific studies in healthcare It is important to remember that the concept of MCID is variable and can differ between health conditions In recent years clinicians and researchers have started to focus on patient-reported outcome assessments
The investigators also aim to determine the MCID values of measurement tools used to evaluate balance in Parkinsons disease in our study
There are two commonly used methods for calculating MCID
Anchor-based approach Distribution-based approach In the anchor-based approach a comparison is made between a result measure reflecting the patients condition eg post-treatment measurement value and an external criterion This external criterion can be the patients own perception or the clinicians assessment In this method changes between scores are compared with an anchor question that serves as a reference to determine whether the patient has improved compared to baseline To understand the perception of change a global rating scale much worse a little worse almost the same a little better and much better can be used The anchor question should be easily understandable and relevant to patients
The distribution-based approach attempts to estimate the likelihood that a detected difference in measurement tools is meaningful It uses statistical data such as fractions of standard deviation effect size and mean standard error It does not include the perceptions based on the experiences of experts or patients Since it does not include individual patient perceptions it should not be the first choice for determining MCID In this method as there is no anchor question to evaluate what is important for patients significant and clinically meaningful results cannot be defined In fact scores calculated with distribution-based methods are sometimes referred to as minimal detectable change instead of MCID Therefore this method is not recommended as the first choice for determining MCID
However some authors have suggested that a single MCID value can be misleading and that providing a range might be more appropriate For this reason in our study the investigators will use both anchor-based and distribution-based approaches to calculate MCID aiming to find a range of values In the anchor-based approach the investigators plan to use two anchor questions a patient-based and a clinician-based global assessment scale
Responsiveness refers to the ability of a measurement tool to detect true changes among individuals A sensitive scale responds accurately to the changes experienced Responsiveness is typically assessed using statistical analyses such as effect size and standard deviation and is used to evaluate the performance of measurement tools
While responsiveness indicates how sensitive a measurement tool is to changes MCID indicates whether these changes are meaningful from the patients or clinicians perspective In other words a responsive measurement tool has the potential to capture clinically significant changes Both concepts are important for understanding and interpreting the performance of measurement tools Determining the responsiveness and MCID of a measurement tool provides clinicians with a more comprehensive assessment in research studies and patient follow-up
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None