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25 June 2017
B&K Kommunikation
How effective is a treatment,
does it lead to the expected improvement and how can all this be
measured in an objective manner? Outcomes measurements are increasingly
important in neurology. Participants at the Congress of the European
Academy of Neurology in Amsterdam are discussing latest development in
the field of outcomes research and how patients benefit from these
activities.
Outcomes measurements are increasingly
important in neurology from the perspective of practitioners as well as
patients or the payers of health services. How can one objectively
determine and plausibly prove whether a given therapy brings about the
desired improvement, makes a useful clinical difference or delays the
progression of a disease? Is the treatment in reasonable proportion to
the patient benefits involved and to the financial cost? The 3rd
Congress of the European Academy of Neurology (EAN) in Amsterdam is
focused on this overarching focal theme, discussing it in several
scientific sessions.
For EAN Programme Committee Chairman Prof Paul Boon (Ghent University, Belgium and Kempenhaeghe, The Netherlands), the possible advantages for patients have priority when it comes to outcomes in neurology: “Of course, the clinical explanation of the disease mechanism revealed through such indicators is very important but patients obviously focus on other aspects.” After being diagnosed with epilepsy or migraine, they are usually keen to know the following: Will the therapy work? Will my seizures or headache attacks stop? Do the medications have undesirable side effects? How many pills do I need to take and when in order to achieve an optimum effect? “Outcomes measurements enable us to answer questions like these. In the process, we can find out how the disease will affect the patient in the future and see how our treatment is working,” explained Prof Dr Bernard Uitdehaag (VUmc MS Center Amsterdam), chair of the local organizing committee of the congress and chair of one of the EAN sessions on this topic.
Progress in measurement methods
Progress is being made in neurology in the development of assessment methods that are easy to handle, and can contribute to objective and precise outcomes measures. This is, however, not yet the case for all diseases. For slowly progressing neuromuscular diseases, for instance, existing outcomes measurements are still cumbersome and burdensome with regard to the patients’ restriction of motion and quality of life. By contrast, there have long been outcomes measurements for common diseases such as epilepsy and migraine. For example, the number of seizures or headache attacks is recorded. Prof Boon: “Recently, the measurements have become more differentiated. For example, we record days free of complaints. For patients, this metric is more meaningful than the total number of seizures.” With epilepsy, outcomes measurements are also assessed with EEG, which is then analysed using special software. Prof Boon: “This approach lets us detect certain indicators much more effectively than with customary methods.”
For instance, in order to measure a possible decline of the condition of multiple sclerosis patients, a simple test has been established as a standard procedure: patients are asked to insert pegs in a board with nine holes and subsequently take them out one by one. If they need 20 per cent longer than last time, this difference proves that the disease has deteriorated clinically.
Measuring outcomes for dementia
Researchers from Amsterdam have developed a questionnaire for Alzheimer’s patients which helps to detect dementia at an early stage. Further outcomes measurements for Alzheimer’s can be done with an amyloid PET scan. In this procedure, a low-radiation substance is injected, enabling amyloid plaques in the brain to be seen. This protein is a typical biomarker for Alzheimer’s. Examinations of cerebrospinal fluid can also allow conclusions to be drawn about the progression of neurodegeneration in dementia.
Combined methods yield more information
A combination of examination methods is increasingly relied upon in outcomes measurements. Prof Uitdehaag advised to use multidimensional outcomes measurements for heterogeneous diseases such as multiple sclerosis. “It is very revealing to harness the patient’s perspective together with e.g. imaging methods. An MRI scan can indicate a stable condition even though the patient is complaining about a change for the worse. Conversely, the patient can feel great while the MRI result indicates that problems are likely to occur in the near future.”
For EAN Programme Committee Chairman Prof Paul Boon (Ghent University, Belgium and Kempenhaeghe, The Netherlands), the possible advantages for patients have priority when it comes to outcomes in neurology: “Of course, the clinical explanation of the disease mechanism revealed through such indicators is very important but patients obviously focus on other aspects.” After being diagnosed with epilepsy or migraine, they are usually keen to know the following: Will the therapy work? Will my seizures or headache attacks stop? Do the medications have undesirable side effects? How many pills do I need to take and when in order to achieve an optimum effect? “Outcomes measurements enable us to answer questions like these. In the process, we can find out how the disease will affect the patient in the future and see how our treatment is working,” explained Prof Dr Bernard Uitdehaag (VUmc MS Center Amsterdam), chair of the local organizing committee of the congress and chair of one of the EAN sessions on this topic.
Progress in measurement methods
Progress is being made in neurology in the development of assessment methods that are easy to handle, and can contribute to objective and precise outcomes measures. This is, however, not yet the case for all diseases. For slowly progressing neuromuscular diseases, for instance, existing outcomes measurements are still cumbersome and burdensome with regard to the patients’ restriction of motion and quality of life. By contrast, there have long been outcomes measurements for common diseases such as epilepsy and migraine. For example, the number of seizures or headache attacks is recorded. Prof Boon: “Recently, the measurements have become more differentiated. For example, we record days free of complaints. For patients, this metric is more meaningful than the total number of seizures.” With epilepsy, outcomes measurements are also assessed with EEG, which is then analysed using special software. Prof Boon: “This approach lets us detect certain indicators much more effectively than with customary methods.”
For instance, in order to measure a possible decline of the condition of multiple sclerosis patients, a simple test has been established as a standard procedure: patients are asked to insert pegs in a board with nine holes and subsequently take them out one by one. If they need 20 per cent longer than last time, this difference proves that the disease has deteriorated clinically.
Measuring outcomes for dementia
Researchers from Amsterdam have developed a questionnaire for Alzheimer’s patients which helps to detect dementia at an early stage. Further outcomes measurements for Alzheimer’s can be done with an amyloid PET scan. In this procedure, a low-radiation substance is injected, enabling amyloid plaques in the brain to be seen. This protein is a typical biomarker for Alzheimer’s. Examinations of cerebrospinal fluid can also allow conclusions to be drawn about the progression of neurodegeneration in dementia.
Combined methods yield more information
A combination of examination methods is increasingly relied upon in outcomes measurements. Prof Uitdehaag advised to use multidimensional outcomes measurements for heterogeneous diseases such as multiple sclerosis. “It is very revealing to harness the patient’s perspective together with e.g. imaging methods. An MRI scan can indicate a stable condition even though the patient is complaining about a change for the worse. Conversely, the patient can feel great while the MRI result indicates that problems are likely to occur in the near future.”
Side effects count as part of the overall outcomes
In the treatment of neurological diseases, certain medications can provoke side effects such as difficulties in concentration or memory loss. Until a few years ago, these side effects were simply put up with, especially by older patients. Prof Boon emphasized that these attitudes are changing more and more, adding: “Side effects are recorded and taken seriously as part of outcomes measurements.” To an increasing extent, therapies are individually tailored to patients so outcomes, too, must be subject to a differentiated analysis. Prof Uitdehaag: “It no longer suffices to compare patient groups with each other in sweeping ways. Even if many people do respond to a certain treatment, it may not be effective for a given individual.” As therapies become more individualized, the challenge grows to record exactly what is being done, what benefits this action has and whether the treatment is beneficial for this specific patient.
Cost-benefit analysis
As cost pressure on public health care budgets mounts, the field of neurology is also increasingly confronted with demands to give evidence of therapy outcomes so the costs of a given treatment can be weighed against its benefits. Prof Boon: “Payers in the European healthcare systems are showing an increasing interest in outcomes-related reimbursement concepts. In other words, they are willing to assume costs if the effectiveness of a treatment can be verified. In the process, new therapies are compared against conventional ones to find out which ones are worth the price being charged. The more varied the possible treatment options for a disease, the more relevant outcomes measurement becomes,” the expert concluded.
Sources: van Munster CE, Uitdehaag BM. Outcome Measures in Clinical Trials for Multiple Sclerosis. CNS Drugs. 2017 Mar;31(3):217-236; Feys P, Lamers I, Francis G: The Nine-Hole Peg Test as a manual dexterity performance measure for multiple sclerosis, journals.sagepub.com/doi/full/10.1177/1352458517690824; Sikkes SA,de Lange-de Klerk ES, Pijnenburg YA, Gillissen F, Romkes R, Knol DL, Uitdehaag BM, Scheltens P. A new informant-based questionnaire for instrumental activities of daily living in dementia. Alzheimers Dement. 2012 Nov;8(6):536-43; Sikkes SA , Knol DL, Pijnenburg YA, de Lange-de Klerk ES, Uitdehaag BM, Scheltens P:Validation of the Amsterdam IADL Questionnaire©, a new tool to measure instrumental activities of daily living in dementia. Neuroepidemiology.2013;41(1):35-41.
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