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A Consensus Set of Outcomes for Parkinson’s Disease from the International Consortium for Health Outcomes Measurement

Authors:

Abstract

Background: Parkinson's disease (PD) is a progressive neurodegenerative condition that is expected to double in prevalence due to demographic shifts. Value-based healthcare is a proposed strategy to improve outcomes and decrease costs. To move towards an actual value-based health care system, condition-specific outcomes that are meaningful to patients are essential. Objective: Propose a global consensus standard set of outcome measures for PD. Methods: Established methods for outcome measure development were applied, as outlined and used previously by the International Consortium for Health Outcomes Measurement (ICHOM). An international group, representing both patients and experts from the fields of neurology, psychiatry, nursing, and existing outcome measurement efforts, was convened. The group participated in six teleconferences over a six-month period, reviewed existing data and practices, and ultimately proposed a standard set of measures by which patients should be tracked, and how often data should be collected. Results: The standard set applies to all cases of idiopathic PD, and includes assessments of motor and non-motor symptoms, ability to work, PD-related health status, and hospital admissions. Baseline demographic and clinical variables are included to enable case mix adjustment. Conclusions: The Standard Set is now ready for use and pilot testing in the clinical setting. Ultimately, we believe that using the set of outcomes proposed here will allow clinicians and scientists across the world to document, report, and compare PD-related outcomes in a standardized fashion. Such international benchmarks will improve our understanding of the disease course and allow for identification of 'best practices', ultimately leading to better informed treatment decisions.
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Journal of Parkinson’s Disease xx (20xx) x–xx
DOI 10.3233/JPD-161055
IOS Press
1
Research Report
1
A Consensus Set of Outcomes for
Parkinson’s Disease from the International
Consortium for Health Outcomes
Measurement
2
3
4
5
Paul de Roosa,c,, Bastiaan R. Bloemb, Thomas A. Kelleyc, Angelo Antoninid, Richard Dodele,
Peter Hagellf, Connie Marrasg, Pablo Martinez-Martinh, Shyamal H. Mehtai, Per Odinj,
Kallol Ray Chaudhurik, Daniel Weintraubl,m, Bil Wilsonnand Ryan J. Uittio
6
7
8
aDepartment of Neuroscience, Neurology, Uppsala University, Uppsala, Sweden9
bRadboud university medical center; Donders Institute for Brain, Cognition and Behavior; Department of
Neurology, Nijmegen, The Netherlands
10
11
cInternational Consortium for Health Outcomes Measurement, Cambridge, USA12
dParkinson and Movement Disorders Unit IRCS Hospital San Camillo, Venice, Italy13
ePhilipps-Universitat, Marburg, Germany14
fThe PRO-CARE Group, School of Health and Society, Kristianstad University, Kristianstad, Sweden15
gMorton and Gloria Shulman Movement Disorders Centre and the Edmond J. Safra Program in Parkinson’s
disease, University of Toronto, Toronto, Canada
16
17
hNational Center of Epidemiology and CIBERNED, Carlos III Institute of Health, Madrid, Spain18
iMayo Clinic, Scottsdale, USA19
jSk˚ane University Hospital, Lund, Sweden20
kKing’s College, London, UK21
lPerelman School of Medicine at the University of Pennsylvania, Philadelphia, USA22
mPhiladelphia Veterans Affairs Medical Center, Philadelphia, USA23
nICHOM Patient Representative, USA24
oMayo Clinic, Jacksonville, FL, USA25
Accepted 24 May 2017
Abstract.26
Background: Parkinson’s disease (PD) is a progressive neurodegenerative condition that is expected to double in prevalence
due to demographic shifts. Value-based healthcare is a proposed strategy to improve outcomes and decrease costs. To move
towards an actual value-based health care system, condition-specific outcomes that are meaningful to patients are essential.
27
28
29
Objective: Propose a global consensus standard set of outcome measures for PD.30
Methods: Established methods for outcome measure development were applied, as outlined and used previously by the
International Consortium for Health Outcomes Measurement (ICHOM). An international group, representing both patients
and experts from the fields of neurology, psychiatry, nursing, and existing outcome measurement efforts, was convened.
31
32
33
Correspondence to: Paul de Roos, Department of Neu-
roscience, Neurology, Uppsala University, 75185 Uppsala,
Sweden. Tel.: +46 186110000; Fax: +46 186115027; E-mail:
Paul.deroos@neuro.uu.se.
ISSN 1877-7171/17/$35.00 © 2017 IOS Press and the authors. All rights reserved
This article is published online with Open Access and distributed under the terms of the Creative Commons Attribution Non-Commercial License (CC BY-NC 4.0).
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2P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease
The group participated in six teleconferences over a six-month period, reviewed existing data and practices, and ultimately
proposed a standard set of measures by which patients should be tracked, and how often data should be collected.
34
35
Results: The standard set applies to all cases of idiopathic PD, and includes assessments of motor and non-motor symptoms,
ability to work, PD-related health status, and hospital admissions. Baseline demographic and clinical variables are included
to enable case mix adjustment.
36
37
38
Conclusions: The Standard Set is now ready for use and pilot testing in the clinical setting. Ultimately, we believe that using
the set of outcomes proposed here will allow clinicians and scientists across the world to document, report, and compare
PD-related outcomes in a standardized fashion. Such international benchmarks will improve our understanding of the disease
course and allow for identification of ‘best practices’, ultimately leading to better informed treatment decisions.
39
40
41
42
MESH terms: Delivery of Health Care/economics, Delivery of Health Care*/standards, Efficiency, Organizational, Inter-
national Cooperation, Health Care Costs Health Status, Health Surveys, Health Surveys/Health Status Indicators, Humans,
Outcome Assessment (Health Care), Quality of Health Care, Quality Indicators, Health Care/standards, Quality of Life, Aged,
Middle Aged, Disability Evaluation, Disease Progression, Female, Male, Parkinsonian Disorders, Parkinson Disease, Parkin-
son Disease/epidemiology, Parkinson Disease, Psychometrics, Activities of Daily Living, Outcome and Process Assessment
(Health Care)/standards, Parkinson Disease/therapy
43
44
45
46
47
48
INTRODUCTION34
Parkinson’s disease (PD) is a common and pro-
35
gressive neurodegenerative disease [1]. In the USA,
36
PD has an estimated prevalence of 0.3% and an esti-37
mated healthcare cost per patient of 10,000 USD/year38
[2]. Prevalence and costs are similar in Europe [3].
39
Due to the aging global population, the prevalence of
40
PD is expected to increase significantly [4], leading41
to greater disease-associated burden and higher care
42
expenditures. Optimizing the quality of PD care and43
minimizing the expense of care delivery are therefore
44
essential.45
Increasing value, defined as a patient’s outcomes46
divided by the cost to achieve those outcomes, has47
been proposed as a mechanism to improve the quality
48
of care [5]. A systematic measurement of outcomes49
can guide improvement and enable dissemination of
50
best practices. In order to move towards an actual51
value-based health care system, having condition-
52
specific outcomes that are meaningful to patients and53
their care providers is crucial. Transparency regard-
54
ing outcomes and costs is essential to help reduce
55
unwanted variations in healthcare delivery, and to56
increase the overall quality of care. This need has57
been recognized in the PD community for some time.58
Efforts to identify outcomes that are meaningful to59
patients and caregivers have led to the establishment
60
of various national assessment programs [6–9].
61
However, across the world, PD outcomes remain62
inconsistently defined, collected and reported. This
63
limits our ability to make reliable national and inter-
64
national comparisons, which in turn obscures our65
ability to learn from best practices, a necessary step66
to improve global healthcare.67
The International Consortium for Health Out- 68
comes Measurement (ICHOM) was formed to 69
develop global consensus sets of outcomes that 70
reflect patients’ concerns and experiences. ICHOM 71
has already developed international sets of outcomes 72
for 21 medical conditions [10]. We here report the 73
results of an ICHOM initiative to develop a simi- 74
lar set of outcomes for PD. To achieve this, ICHOM 75
brought together an International Working Group, 76
representing patients, neurology, psychiatry, nurs- 77
ing and existing outcome measurement efforts, to 78
develop a parsimonious standard set of outcome 79
indices for PD, with the aim of proposing the prod- 80
uct for international use. This paper describes the 81
development process and the resultant set. 82
METHODS 83
Working group 84
The formation of the Working Group was based 85
on the principles of previous ICHOM working 86
groups [11]. The PD Working Group consisted of 12 87
members from eight countries (USA, Canada, UK, 88
Spain, Italy, Germany, Netherlands, and Sweden) and 89
included expert neurologists (n= 9), a psychiatrist, 90
and a nurse specializing in PD, as well as an expe- 91
rienced patient advocate (Table 1). Working Group 92
members were identified by reviewing authors of 93
leading papers on PD care quality, and by identifying 94
members of international patient advocacy groups, 95
leading international PD scientific organizations, and 96
leading physicians in existing national and interna- 97
tional quality measurement efforts. 98
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P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease 3
Table 1
Working Group members
Working Group member Expertise
Bas Bloem Professor of Neurology, focusing on movement disorders.
Lead of National Parkinson’s Disease Registry in Netherlands.
Angelo Antonini Professor of Neurology, focusing on Parkinson’s disease and measurement of outcomes that matter to
patients.
Richard Dodel Professor of Neurology with interest in Parkinson’s disease and measurement of patient outcomes.
Member of MDS-UPDRS revision taskforce
Peter Hagell Professor of Neurological Caring Science, with a focus on outcomes measurement in Parkinson’s
disease.
Connie Marras Associate Professor of Neurology, focusing on Movement Disorders and the evaluation of clinical
assessment tools.
Pablo Martinez-Martin Neurologist, interest in Parkinson’s disease and development of clinical evaluation tools.
Shyamal Mehta Assistant Professor of Neurology, focusing on movement disorders and measuring outcomes in the
Parkinson’s disease clinic.
Per Odin Professor of Neurology, focusing on movement disorders.
Developed Swedish National Parkinson’s disease registry.
K Ray Chaudhuri Professor of Neurology, focusing on movement disorders.
Expertise in developing clinical evaluation tools.
Daniel Weintraub Professor of Psychiatry, with interest in psychiatric and cognitive complications of Parkinson’s
disease.
Bill Wilson Experienced Parkinson’s disease patient advocate. Part of the Parkinson’s Disease Foundation.
Ryan Uitti Professor of Neurology focusing on movement disorders with an academic interest in measuring
patient outcomes relative to cost.
Paul de Roos Neurology Resident. Research Fellow, providing literature review expertise.
Process
99
Following the process used in earlier ICHOM work100
[10, 11], a modified Delphi technique was employed101
to define the outcomes and case-mix variables. Case102
mix variables are defined as those variables that
103
capture the state of the patient independent of the104
medical condition for which they are being treated.
105
This includes demographic factors, health status (e.g.
106
co-morbidities) and treatments. The process is a
107
structured, consensus-driven approach, with telecon-
108
ferences and post-teleconference surveys to reach109
decisions. Proposals for each teleconference were110
generated in advance by a core ICHOM project team111
(RU, TAK, PdR). These were based on a literature112
review of existing guidelines and standards, as well113
as individual interviews with each Working Group
114
member.
115
The Working Group was officially announced in
116
December 2013 and launched with an in-person
117
meeting at the conference of the International118
Association of Parkinsonism and Related Disorders119
(IAPRD). This was followed by five 75-minute tele-
120
conferences, which took place every month between121
January and May 2014. All of these teleconferences122
were followed by a survey of the Working Group123
members to make decisions on key discussion areas.124
A 2/3 majority was required, being a commonly used125
threshold for Delphi and modified Delphi processes,126
on each survey question to reach consensus. Shifting 127
the threshold a bit did not have an impact on the selec- 128
tion process. When a 2/3 majority was not reached, 129
the topic was brought up for re-discussion at the fol- 130
lowing teleconference. The standard set of outcomes 131
was then launched at the International Parkinson and 132
Movement Disorder Society (MDS) Conference in 133
June 2014. 134
The process began with defining the scope of 135
the Working Group by deciding which causes of 136
parkinsonism to include in the set. Subsequently, 137
key outcome domains that are meaningful to patients 138
were identified based on relevant literature and out- 139
come measurement programmes [6–11]. These were 140
then reviewed with each Working Group member 141
individually to determine if additional domains, not 142
identified by the search, should be considered. The 143
resultant list of outcome domains was then organized 144
based on four criteria. Each criterion was rated on a 145
Likert scale of 1–4, where one was the lowest and 146
four was the highest score given: (1) Frequency of 147
the outcome domain in the patient population an 148
important consideration for a set that aims to be par- 149
simonious; (2) Impact of the outcome domain on the 150
patient an essential consideration for a set that aims 151
to reflect what is most meaningful to patients; (3) 152
Preventability/treatability of the outcome domain a 153
necessary consideration for a set that aims to be used 154
in the clinic to generate meaningful data on which 155
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4P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease
clinicians can act to modify their practice; and (4)156
Feasibility to capture the outcome domain in clinical
157
practice this is essential as the set is designed to158
be used in routine clinical practice. This formed the159
basis for the first teleconference discussion.160
Once the outcome domains were decided, the tools
161
for data collection were determined. Relevant scales162
or items were identified and prioritized using spe-163
cific criteria. Again, each criterion was rated on a164
Likert scale of 1–4, where one was the lowest and
165
four was the highest score given. The criteria were166
as follows: (1) Domain coverage this set aims to be167
of minimal burden and complexity. Thus, tools that
168
cover many domains were preferable; (2) Psychome-169
tric properties the data collected must be accurate,
170
and thus patient-reported tools were prioritized based171
on psychometric properties; (3) Feasibility to imple-172
ment the tool must be practical for day-to-day use
173
in the clinic; and (4) Clinical interpretability clini-174
cal teams must be able to understand the results. This175
formed the basis for the second teleconference dis-176
cussion. Finally, we sought to reach agreement on the177
frequency of data collection, balancing comprehen-178
siveness, practicalities for clinics, and what would be179
best for patients.
180
This was followed by identification of the base-
181
line case-mix variables, which are necessary to make182
meaningful comparisons between patients. Case-mix
183
variables to measure were prioritized based on three184
criteria. Each criterion was rated on a Likert scale of185
1–4, where one was the lowest score and four was
186
the highest score given. The criteria were as follows:187
(1) Relevance (strength of association between the
188
case-mix variable and the outcome) we aimed to
189
identify case-mix variables that could strongly affect190
the outcome; (2) Case-mix variable independency 191
given the aim to collect a minimum set of case-mix192
variables, the aim was to identify variables that would193
independently affect the outcome; (3) Feasibility to194
collect the set must be practical for use in the clinic.
195
This formed the basis for the third teleconference196
discussion.
197
The fourth teleconference focused on reaching198
agreement around internationally acceptable ways to199
measure case mix adjustment variables. The fifth tele-200
conference focused on reviewing the set prior to its201
launch to the international community.202
Literature search strategy203
The following PubMed MeSH terms and Boolean204
logic were used to perform a search to identify out-205
comes that matter to PD patients, as well as scales 206
to collect those outcomes: (“Parkinson’s disease” OR 207
“Parkinson disease” OR “Parkinsonism”) AND (“cri- 208
tique” OR “recommendation” OR “review”) AND 209
(“scale” OR “scales” OR “instrument” OR “instru- 210
ments” OR “questionnaire” OR “questionnaires”). 211
Limitations were applied, which included the need 212
to be review articles, written in the English language, 213
and published in the 10 years preceding January 2015. 214
From this search, article titles and abstracts were 215
reviewed to identify those that had a clear focus on 216
scales used in clinical practice. From these results, 217
references to scales were extracted and through tar- 218
geted searches, original validation studies and use of 219
the respective instruments were identified. 220
RESULTS 221
Scope 222
The set was designed to cover all cases of adult 223
(>18 years of age) idiopathic PD. Atypical parkin- 224
sonism was excluded, as the consensus was that 225
this would require different outcome measures. We 226
recommend that atypical causes of parkinsonism 227
be considered in future outcome sets. This set is 228
intended to be relevant to PD patients receiving all 229
common treatment options for motor and non-motor 230
symptoms, including pharmacotherapy (including 231
infusion or injection-based delivery), deep brain stim- 232
ulation, and rehabilitation-based therapy (including 233
allied health interventions, nursing, and behavioral 234
therapy). 235
Outcomes 236
A series of motor, non-motor and other outcomes 237
were agreed upon by the Working Group as essential 238
to collect. 239
Non-motor symptoms 240
Non-motor outcomes impact the ability of patients 241
with PD to carry out normal day-to-day activities 242
[12] and are key determinants of their perceived 243
health [13, 14]. Based on the current literature, non- 244
motor symptoms that are most important for PD 245
patients were listed [6, 7, 11, 15, 16]. As described 246
in Methods, the project team then prioritized this 247
list and suggested the following outcome domains 248
for inclusion in the standard set: depression, anxiety, 249
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P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease 5
cognitive function, urinary function, gastrointestinal250
function, pain, sleep, sexual function, treatment com-
251
plications (hemorrhage and behavior change). These252
were deemed frequent, of high impact on patients,253
treatable and feasible to capture in clinical practice.254
During the teleconference, the group agreed with
255
their inclusion but additionally felt that fatigue, hallu-256
cinations and sweating should also be included, due257
to their impact on patients. In the survey following258
the teleconference, the voting confirmed inclusion
259
of the aforementioned outcomes with the exception260
of treatment complications specifically, hemor-261
rhage, as it is very uncommon, and behavior change,
262
as this is captured under the cognitive and psy-263
chiatric domains. Additionally, the survey revealed
264
that psychosis, apathy, impulse control disorder and265
dizziness/syncope were further domains deemed nec-266
essary to be part of the standard set, again due to their
267
impact on patients. These were reviewed at the next268
teleconference and agreed by all WG members to be269
included in the Set. (See Table 2 for the full list of270
outcome domains and suggested scales).271
A range of tools for data collection were identified.272
These included the Scale for Outcomes of Parkin-273
son’s disease (SCOPA-AUT) [17], the Non-Motor
274
Symptom Questionnaire (NMSQuest) [18], the Non-
275
Motor Symptoms Scale (NMSS) [19], the Movement276
Disorder Society Unified Parkinson’s Disease Rat-
277
ing Scale (MDS-UPDRS) [20], as well as specific278
scales relating to depression [21, 22], anxiety [23],279
apathy [24], psychosis [25], fatigue [26], sleep [27]
280
and cognition [28, 29].281
It was felt that it would be simpler and less bur-
282
densome for patients and health systems to have a
283
single instrument rather than many individual patient-284
reported outcome measurements. A number of scales285
were considered, including NMSS [19], NMSQuest286
[19, 30], SCOPA-AUT [17] and MDS-UPDRS Part287
1 [31, 32]. Ultimately, the MDS-UPDRS part 1 was288
chosen, as it has the highest test-retest reliability
289
and internal consistency (as measured by Cronbach’s290
alpha), in comparison to the other tools, as well as
291
having acceptable construct validity. Additionally, it292
poses minimal burden on the health system, with the293
clinician-recorded component taking <10 minutes to294
complete and the rest being patient reported [32].295
Additionally, the MDS-UPDRS Part 2 (see below) is296
recommended for collection of the motor outcomes297
and thus it was felt simpler for clinics to use the MDS-
298
UPDRS for both motor and non-motor assessment.
299
Two of the selected domains (sweating and sex-
300
ual function) are not covered in the MDS-UPDRS
301
part 1 survey, so it was decided to use the questions 302
addressing these issues that are in the NMSQuest 303
[21]. While not a perfect solution, the Working Group 304
prioritized the selection of two simple, easy to admin- 305
ister, patient-reported questions. The Working Group 306
encourages the MDS to consider including questions 307
relating to sweating and sexual dysfunction in future 308
iterations of the MDS-UPDRS. 309
We initially considered using the MDS-UPDRS 310
part 1 as a screening tool for anxiety, depression 311
and cognitive symptoms, and to use domain specific 312
scales such as Beck Depression Inventory (depres- 313
sion) [33], State Trait Anxiety Inventory (anxiety) 314
[34] and Montreal Cognitive Assessment (cognition) 315
[35] to investigate these non-motor symptoms in 316
more detail. However, it was decided that this would 317
miss a key principle underpinning the work (i.e., to 318
produce a practical, minimum set of outcomes that is 319
of minimal burden to patients and staff). Therefore, 320
only the MDS-UPDRS part 1 was included as part of 321
the set. 322
Motor symptoms 323
Motor symptoms are an important problem in PD 324
and their presence is relied upon to make a clinical 325
diagnosis of PD. Motor features that were considered 326
to be most important to the PD patient were identi- 327
fied and listed [6, 7, 11]. The outcome domains that 328
the project team suggested including in the standard 329
set (following the process set out under the meth- 330
ods) included: mobility ability to walk; activities 331
of daily living living independently, handwriting 332
and keyboard capabilities; ability to self-care; tremor; 333
speech; swallowing; treatment complications (dysk- 334
inesia and dystonia). 335
During the teleconference (and confirmed by the 336
post-teleconference survey) it was agreed to include 337
these proposed outcome domains, and it was sug- 338
gested and agreed upon in the post-call survey to 339
include additional ones. The additional outcomes 340
included: leisure activities, saliva and drooling, and 341
ability to move in bed at night. These were agreed 342
upon as they are domains that can have a significant 343
impact on the patient’s quality of life. Ultimately, the 344
only outcome domains from the initial list not to be 345
included in the standard set were treatment compli- 346
cations specifically, dyskinesia and dystonia as it 347
was felt that we should focus on motor function, not 348
specific symptoms or side effects. 349
A wide variety of rating instruments were iden- 350
tified for different motor symptoms, including the 351
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Table 2
Summary of the Parkinson’s disease Standard Set. Full set can be found: http://www.ichom.org/wp-content/uploads/2014/08/PD-Reference-
Guide-6.11.14-KL.pdf
Category Domain Tool Data source
Cognitive and psychiatric
symptoms/functioning
Cognitive impairment MDS-UPDRS Part 1 Physician reported
Hallucinations & psychosis
Depressed mood
Anxious mood
Apathy
Features of dopamine
dysregulation syndrome
(including impulse control
disorders)
Non-motor functioning Sleep problems MDS-UPDRS Part 1 patient
questionnaire part 1
Patient and/or caregiver reported
Daytime sleepiness
Pain & other sensations
Urinary problems
Constipation problems
Light headedness on standing
Fatigue
Sexual function Non Motor Symptoms
Questionnaire
Patient and/or caregiver reported
Sweating
Motor functioning Speech MDS-UPDRS Part1–Patient
questionnaire part 2
Patient and/or caregiver reported
Saliva & drooling
Chewing & swallowing
Eating tasks
Dressing
Hygiene
Handwriting
Doing hobbies & other activities
Turning in bed
Tremor
Getting out of bed, a car, or a
deep chair
Walking & balance
Freezing
Additional health outcomes Ability to work Does your PD limit your ability
to work?
Patient reported
Hospital admissions 1. Admitted to hospital in last 12
months and how many times?
Patient and/or carer reported
2. Number of times related to
PD?
PD-related health status PDQ-8 Patient and/or carer reported
Falls Fall within last year and did it
cause a fracture?
Patient and/or carer reported
Case-mix variables Age In years Patient reported
Sex Male or female Patient reported
Level of education Defined using International
Standard Classification of
Education (ISCED)
Patient reported
Living status Who currently lives with you? Patient reported
Marital status Indication of marital status. Patient reported
Depression/anxiety/REM sleep
behavior disorder prior to PD?
Yes/No Patient reported
Age at PD diagnosis Age in years Patient reported
Age at onset of PD symptoms Age in years Patient reported
Comorbidities NHS comorbidity tool Patient reported
NB: All outcomes are collected annually.
Hoehn and Yahr staging [36, 37], the Schwab and352
England ADL scale [38], PD-related health sta-353
tus questionnaires [39] such as PDQ39 [40], the354
MDS-UPDRS, and scales which can be used to report 355
motor complications, such as “wearing off” [41], risk 356
of falling (including the Berg Balance Scale [42] 357
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P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease 7
and others [43, 44]) and mobility (Timed Get Up358
and Go Test) [45]. During the teleconference discus-
359
sions it was agreed that many domain-specific scales360
would be needed and that this would be too burden-361
some and complicated for patients and clinical teams.362
Therefore, the MDS-UPDRS and the PDQ-39 were
363
ultimately identified as the potential tools for data364
collection. The PDQ-39 is available in multiple lan-365
guages and is free to use, but only covers 6/10 motor366
domains that we identified as being important. In con-
367
trast, the MDS-UPDRS part 2 questionnaire is also368
available in multiple languages and is free to use clin-369
ically but covers 10/10 domains. MDS-UPDRS part
370
2 has excellent psychometric properties [20]. There-371
fore, the MDS-UPDRS part 2 was decided as the
372
motor tool of choice by the Working Group.373
Additional health outcomes
374
We identified four additional domains as impor-375
tant for patients with PD: ability to work, hospital
376
admissions, overall PD-related health status, and377
falls. These were selected by the group, particularly378
the patient representative, as important outcomes to
379
assess. To assess ability to work, hospital admissions,380
and falls, the questions currently used in the recently381
developed Dutch National Parkinson’s Disease Reg-382
istry (www.ParkinsonInzicht.nl), which cover these383
domains, were selected for use in the ICHOM set.
384
The Dutch registry uses the PDQ-39 to assess PD-385
related health status. The PDQ-8 and PDQ-39 are
386
comparable as health status indices, but the PDQ-8 is
387
significantly less burdensome to complete [46–48].
388
We recognize the value of having a single PD-related389
health status score and decided to include the PDQ-8.390
Finally, there was also a discussion around the391
assessment of cost of accessing care for the patient.392
While we agreed that cost is vitally important, it
393
was best included not as an outcome but rather394
the denominator of the value equation. Reporting
395
cost was therefore seen as out of the scope of this396
work.
397
Case-mix variables398
Patients with PD have a broad range of char-399
acteristics both related and unrelated to their
400
neurodegenerative disease that may influence their
401
outcomes. A parsimonious set of case-mix variables402
(Table 2) that were felt to strongly impact outcomes,403
based on existing literature [49, 50] and informal404
discussions, was proposed. For demographic vari-405
ables: age, gender, level of education, and living 406
status (i.e. whether the patient was living alone) were 407
proposed. Age and gender are associated with anxi- 408
ety, cognitive function, urinary function, GI function, 409
pain, sexual function and fatigue. Gender is associ- 410
ated with depression [51]. Level of education, gender 411
and living status are associated with cognitive func- 412
tion [49, 52]. For baseline health status: early age at 413
onset of PD, depression earlier in life, PD motor sub- 414
type, non-PD related cognitive dysfunction, non-PD 415
related co-morbidities, and non-PD related medica- 416
tion affecting sleep, sexual function, and dizziness 417
were proposed. During the teleconference it was sug- 418
gested and agreed upon to include marital status 419
as an additional demographic variable, as not being 420
married is known to be associated with the risk for 421
cognitive decline in the elderly general population 422
[53]. Other constructs such as loneliness and social 423
networks in late life also include marital status and 424
are known to be correlated to cognitive function [50]. 425
There was unanimous agreement to remove PD motor 426
subtype and all medication side effects due to the 427
difficulty of recording this information accurately. 428
There was agreement to change early age at diag- 429
nosis of PD to age at diagnosis of PD, as there are 430
conflicting views on the definition of “early”, while 431
age would provide a more specific time point assur- 432
ing less ambiguity in the data collected. For baseline 433
health status, the age of PD onset and diagnosis, the 434
diagnosis of depression, anxiety or rapid eye move- 435
ment (REM) sleep behavior disorder (RBD) before 436
PD diagnosis [53], and comorbidities were included. 437
We agreed on definitions for each of the case-mix 438
variables. For marital status and living status we 439
decided to use the widely accepted definitions devel- 440
oped by the European Social Survey [54]. For level of 441
education, the United Nations Educational, Scientific 442
and Cultural Organization (UNESCO) definitions of 443
education levels, which allow for international and 444
cross-cultural comparisons, were selected [55]. We 445
decided to change the term “tertiary” to “Univer- 446
sity or equivalent” as it was felt that this wording 447
would be easier for patients and care providers to 448
understand. For the case-mix variables, depression 449
and anxiety, we developed two new yes/no questions. 450
We agreed to include a single baseline patient- 451
reported question used to assess previous REM sleep 452
behavior disorder [53]. A validated patient-reported 453
Charlson Comorbidity Index currently in use by 454
the United Kingdom National Health Service [56] 455
was chosen to reduce data collection burden on 456
physicians.
Uncorrected Author Proof
8P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease
Data collection457
In order to be able to easily compare between
458
providers, centers and countries, the use of estab-
459
lished instruments with multiple translations was460
prioritized and data collection methods that can be461
applied across different countries and settings were
462
proposed. We aimed to reduce the reporting burden on463
clinicians and as such the vast majority of outcomes464
in the set are patient-reported, with the exception of465
the cognitive and mental health outcomes. We rec-
466
ommend all outcomes to be recorded annually.467
DISCUSSION
468
We have produced a standard set of outcomes,469
intended for international use to monitor the quality470
of clinical management of patients with PD. The set471
includes validated indicators of motor and non-motor
472
symptoms and health status. Additional case-mix
473
variables have been included to enable case mix474
adjustment so that inter-center and international com-475
parisons can be performed. It aims to build on existing
476
outcome measurement work [6–10] and additionally477
brings the perspective of leading clinicians and a478
patient advocate from around the world to ensure a479
global perspective.480
The aim was parsimony, so more detailed481
symptom-specific scales (e.g., the Beck Depression
482
Inventory and the Montreal Cognitive Assessment)483
were not selected. Additionally, not all possible out-
484
come domains were included, but rather a focus on
485
those essential outcomes that really reflect what mat-
486
ters to most people with Parkinson’s disease in most487
places. For example, driving is key component of the488
patient’s independence, and a frequently volunteered489
priority in clinical practice [57]. The fact that driving490
was not mentioned suggests that not all elements that
491
matter to patients came to light in this project, and492
consequently did not make it to the final instrument.
493
We therefore encourage teams to use this dataset as494
the basis on which other outcome domains can be
495
added.496
Ultimately, the MDS-UPDRS parts 1 and 2, three497
questions from the NMSQuest, the PDQ-8, and six498
questions from the Dutch National PD registry were499
chosen, as their questions represent all of the domains500
that the Working Group identified as being important.501
We realize that some health care providers currently
502
use different scales and that there may be challenges
503
in switching to the present recommendation, but we
504
feel that the prospective benefit of being able to
505
perform cross-provider comparisons and to collabo- 506
ratively learn and improve patient care will encourage 507
universal adoption of this set over time. We also 508
recognize that computer-adaptive patient-reported 509
outcome measures are currently under investiga- 510
tion, and that they may eventually replace the scales 511
included in this set. To ensure continuity of the set 512
over time, a subset of Working Group members has 513
formed a Steering Committee to review and update 514
the set on an annual basis. 515
This set aims to be used on a day-to-day basis in the 516
clinic, as a useful tool to help guide management deci- 517
sions for clinicians and patients. It is also hoped that 518
it will be used to compare the quality of care provided 519
by different centers around the world, stimulating dis- 520
cussion and learning from those centers with the best 521
outcomes. For the MDS-UPDRS, the NMSQuest and 522
the questions from the Dutch registry, it is envisaged 523
that the results of each individual question will be the 524
unit of comparison. For the PDQ-8, an overall score 525
can be calculated, which will be used for comparison. 526
We are recommending existing validated instru- 527
ments, and as such this dataset can be used 528
immediately by teams across the world in pilot exper- 529
iments. Specifically, before this ICHOM approach 530
to outcome measurement can be recommended fully 531
to international communities of clinicians, we rec- 532
ommend that pilot experiments should be performed 533
in a cohort of individuals with PD. The results of 534
such pilot studies should be evaluated using estab- 535
lished psychometric approaches to further optimize 536
the question set. Accordingly, we actively seek such 537
feedback from teams to ensure that the set remains 538
practical and relevant for people living with Parkin- 539
son’s disease. For most institutions, implementation 540
into routine clinical practice may be challenging, not 541
in the least because it may require new resource com- 542
mitments and infrastructure development. ICHOM 543
has developed an expert implementation team to 544
assist institutions in figuring out how to overcome 545
these challenges. While we recognize the challenges, 546
we are encouraged by the increasing availability of 547
electronic health records and communication tech- 548
nologies that enable outcome reporting directly into 549
the patient’s medical record. We hope that this set will 550
further spur development in this area. We also recog- 551
nize that in some languages, validated translations of 552
the proposed scales do not yet exist and will need to be 553
undertaken. Finally, we note that valid comparisons 554
of outcomes across countries are in their infancy and 555
will require further methodological development to 556
ensure validity [58]. 557
Uncorrected Author Proof
P. de Roos et al. / Consensus Set Outcomes for Parkinson’s Disease 9
A methodological draw back to the project was the558
absence of physiotherapy and rehabilitation expertise
559
in the Working Group, as well as absence of represen-560
tation from Asia, Oceania and South America. This561
will be addressed by identifying appropriate exper-562
tise to join the steering committee, which is charged
563
with monitoring and updating the set on an ongoing564
basis.565
In summary, we have developed a simple, rela-566
tively easy to implement, set of outcome indices that
567
we believe should, after piloting testing, be collected568
and tracked for all patients with PD. This is an ini-569
tial step towards driving meaningful and significant
570
improvements in the care of patients with PD around571
the world.
572
ACKNOWLEDGMENTS573
We would like to thank all authors for the time and574
effort contributed without financial compensation.
575
SOURCES OF SUPPORT576
This project was funded by the International Con-577
sortium for Health Outcome Measurement.578
CONFLICT OF INTEREST
579
No conflicts of interest to report: De Roos,580
Martinez Martin, Antonini, Ray Chaudhuri, Uitti,
581
Wilson, Hagell, Weintraub, Mehta, Marras, Kelley.582
583
Per Odin has given lectures with honorarium584
and/or had expert advisor role for the following
585
companies: Abbott/AbbVie, Britannia, Bayer, Lund-586
beck, Orion Pharma, UCB.587
588
Bas Bloem589
See separate file attached.
590
591
Richard Dodel
592
See separate file attached.593
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Med Care,42, I37-I48. 871
Article
Background and Purpose The article's significance lies in the substantial rise in the risk of developing Parkinson's disease (PD), necessitating the exploration of various approaches to rehabilitation and medical treatment. The purpose of the article is to detect the direct effect of physiotherapy for patients with PD and to identify how it helps in slowing down cardio‐pulmonary failure, improving the posture, balance, bradykinesia and tremor. Methods The research utilised clinical data from 407 PD patients aged 30–100 years at the Central Polyclinic of Durrës, spanning 2011–2022, and included a systematic literature review and statistical analysis comparing physiotherapy outcomes with European Union standards. Results The research demonstrates the efficiency of physiotherapy in the short and long term in the treatment of PD for patients and medical personnel. All information can be used to increase the functional abilities of patients and minimise complications after physiotherapy and to estimate the effectiveness of different exercises in delaying PD. Older adults, particularly those aged 71–80, are most affected by PD, with males more likely to be diagnosed. Physiotherapy rehabilitation improves motor symptoms, posture, and balance in 30‐80‐year‐olds, but its effectiveness declines with age. Advanced rehabilitation methods in Italy lead to better outcomes, suggesting the potential for improvement in Durres disease. Conclusions The study emphasises the need for improved rehabilitation strategies for older patients by recommending tailored programs, advanced methods, standardisation, training, and long‐term monitoring. Further research should concentrate on the long‐term sustainability of physiotherapy benefits, the development of targeted interventions for older patients, and the integration of innovative therapeutic approaches.
Article
Background: Physical exercise is effective in managing Parkinson's disease (PD), but the relative benefit of different exercise types remains unclear. Objectives: To compare the effects of different types of physical exercise in adults with PD on the severity of motor signs, quality of life (QoL), and the occurrence of adverse events, and to generate a clinically meaningful treatment ranking using network meta-analyses (NMAs). Search methods: An experienced information specialist performed a systematic search for relevant articles in CENTRAL, MEDLINE, Embase, and five other databases to 17 May 2021. We also searched trial registries, conference proceedings, and reference lists of identified studies up to this date. Selection criteria: We included randomized controlled trials (RCTs) comparing one type of physical exercise for adults with PD to another type of exercise, a control group, or both. Data collection and analysis: Two review authors independently extracted data. A third author was involved in case of disagreements. We categorized the interventions and analyzed their effects on the severity of motor signs, QoL, freezing of gait, and functional mobility and balance up to six weeks after the intervention using NMAs. Two review authors independently assessed the risk of bias using the risk of bias 2 (RoB 2) tool and rated the confidence in the evidence using the CINeMA approach for results on the severity of motor signs and QoL. We consulted a third review author to resolve any disagreements. Due to heterogeneous reporting of adverse events, we summarized safety data narratively and rated our confidence in the evidence using the GRADE approach. Main results: We included 154 RCTs with a total of 7837 participants with mostly mild to moderate disease and no major cognitive impairment. The number of participants per study was small (mean 51, range from 10 to 474). The NMAs on the severity of motor signs and QoL included data from 60 (2721 participants), and 48 (3029 participants) trials, respectively. Eighty-five studies (5192 participants) provided safety data. Here, we present the main results. We observed evidence of beneficial effects for most types of physical exercise included in our review compared to a passive control group. The effects on the severity of motor signs and QoL are expressed as scores on the motor scale of the Unified Parkinson's Disease Rating Scale (UPDRS-M) and the Parkinson's Disease Questionnaire 39 (PDQ-39), respectively. For both scales, higher scores denote higher symptom burden. Therefore, negative estimates reflect improvement (minimum clinically important difference: -2.5 for UPDRS-M and -4.72 for PDQ-39). Severity of motor signs The evidence from the NMA (60 studies; 2721 participants) suggests that dance and gait/balance/functional training probably have a moderate beneficial effect on the severity of motor signs (dance: mean difference (MD) -10.18, 95% confidence interval (CI) -14.87 to -5.36; gait/balance/functional training: MD -7.50, 95% CI -11.39 to -3.48; moderate confidence), and multi-domain training probably has a small beneficial effect on the severity of motor signs (MD -5.90, 95% CI -9.11 to -2.68; moderate confidence). The evidence also suggests that endurance, aqua-based, strength/resistance, and mind-body training might have a small beneficial effect on the severity of motor signs (endurance training: MD -5.76, 95% CI -9.78 to -1.74; aqua-based training: MD -5.09, 95% CI -10.45 to 0.40; strength/resistance training: MD -4.96, 95% CI -9.51 to -0.40; mind-body training: MD -3.62, 95% CI -7.24 to 0.00; low confidence). The evidence is very uncertain about the effects of "Lee Silverman Voice training BIG" (LSVT BIG) and flexibility training on the severity of motor signs (LSVT BIG: MD -6.70, 95% CI -16.48 to 3.08; flexibility training: MD 4.20, 95% CI -1.61 to 9.92; very low confidence). Quality of life The evidence from the NMA (48 studies; 3029 participants) suggests that aqua-based training probably has a large beneficial effect on QoL (MD -15.15, 95% CI -23.43 to -6.87; moderate confidence). The evidence also suggests that mind-body, gait/balance/functional, and multi-domain training and dance might have a small beneficial effect on QoL (mind-body training: MD -7.22, 95% CI -13.57 to -0.70; gait/balance/functional training: MD -6.17, 95% CI -10.75 to -1.59; multi-domain training: MD -5.29, 95% CI -9.51 to -1.06; dance: MD -3.88, 95% CI -10.92 to 3.00; low confidence). The evidence is very uncertain about the effects of gaming, strength/resistance, endurance, and flexibility training on QoL (gaming: MD -8.99, 95% CI -23.43 to 5.46; strength/resistance training: MD -6.70, 95% CI -12.86 to -0.35; endurance training: MD -6.52, 95% CI -13.74 to 0.88; flexibility training: MD 1.94, 95% CI -10.40 to 14.27; very low confidence). Adverse events Only 85 studies (5192 participants) provided some kind of safety data, mostly only for the intervention groups. No adverse events (AEs) occurred in 40 studies and no serious AEs occurred in four studies. AEs occurred in 28 studies. The most frequently reported events were falls (18 studies) and pain (10 studies). The evidence is very uncertain about the effect of physical exercise on the risk of adverse events (very low confidence). Across outcomes, we observed little evidence of differences between exercise types. Authors' conclusions: We found evidence of beneficial effects on the severity of motor signs and QoL for most types of physical exercise for people with PD included in this review, but little evidence of differences between these interventions. Thus, our review highlights the importance of physical exercise regarding our primary outcomes severity of motor signs and QoL, while the exact exercise type might be secondary. Notably, this conclusion is consistent with the possibility that specific motor symptoms may be treated most effectively by PD-specific programs. Although the evidence is very uncertain about the effect of exercise on the risk of adverse events, the interventions included in our review were described as relatively safe. Larger, well-conducted studies are needed to increase confidence in the evidence. Additional studies recruiting people with advanced disease severity and cognitive impairment might help extend the generalizability of our findings to a broader range of people with PD.
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Introduction Current clinical trials on swallowing disorders (dysphagia) in Parkinson’s disease (PD) apply a high variety of outcomes and different outcome measures making comparative effectiveness research challenging. Furthermore, views of patients and dysphagia clinicians when selecting trial outcomes have not been considered in the past, thus study results may have little importance to them. This study aims to develop an agreed standardised Core Outcome Set for Dysphagia Interventions in Parkinson’s disease (COS-DIP), systematically measured and reported as a minimum for all clinical trials. It will also comprise guidance on outcome definitions, outcome measures and time points of measurement. Methods and analysis The COS-DIP development will comprise five stages following established methodology: (1) a recent scoping review on all applied outcomes, their definitions, methods and time points of measurement in clinical trials in dysphagia in PD, (2) online surveys and focus groups with clinicians, patients, caregivers and family members to identify outcomes that are important to them, (3) an identified list of outcomes based on results of stage 1 and 2, (4) three round online Delphi survey with up to 200 key stakeholders to determine core outcomes and (5) two online consensus meetings with up to 40 representative key stakeholders to agree on all outcomes, definitions, methods and time points of measurement in the final COS-DIP. Ethics and dissemination Full ethical approval was obtained from the Research Ethics Committee, School of Linguistic, Speech and Communication Sciences, Trinity College Dublin, on 15 May 2023 (HT27). Dissemination of the COS-DIP will be enhanced through presentations at (inter-) national conferences and through peer-reviewed, open access publications of related manuscripts. Lay and professional information sheets and infographics will be circulated through relevant patient and professional organisations and networks. Trial registration number The COS-DIP study was registered prospectively with the Core Outcome Measures in Effectiveness Trials (COMET) database on 24 September 2021 (www.comet-initiative.org/Studies/Details/1942).
Article
Background: Dementia with Lewy bodies (DLB) is an important cause of dementia with a range of clinical manifestations, including motor, neuropsychiatric, and autonomic symptoms. Compared with more common forms of dementia such as Alzheimer’s disease, DLB has been the focus of significantly fewer treatment studies, often with diverse outcome measures, making comparison and clinical implementation difficult. A core outcome set (COS) can address this by ensuring that data are comparable, relevant, useful, and usable for making the best healthcare decisions. Methods: Using a multi-stage approach, development of the DLB-COS will include the following stages: (1) A systematic review, following PRISMA guidelines to create an initial long list of outcomes; (2) A two-round online Delphi including clinicians, scientists, policymakers, and individuals with lived experience of DLB and their representatives; (3) An online consensus meeting to agree on the final core list of outcomes (the final DLB-COS) for use in research and clinical practice; (4) A literature search to identify appropriate measurement instruments for the DLB-COS outcomes; (5) A final consensus meeting of the professional stakeholders who attended the online consensus meeting to agree on the instruments that should be used to measure the outcomes in the DLB-COS; and (6) Global dissemination. Discussion: This is a multi-stage project to develop a COS to be used in treatment trials for DLB. A DLB-COS will ensure the selection of relevant outcomes and will identify the instruments to be used to measure DLB globally.
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Background Value-based health care has been proposed as a unifying force to drive improved outcomes and cost containment. Objective To develop a standard set of multidimensional patient-centered health outcomes for tracking, comparing, and improving localized prostate cancer (PCa) treatment value. Design, setting, and participants We convened an international working group of patients, registry experts, urologists, and radiation oncologists to review existing data and practices. Outcome measurements and statistical analysis The group defined a recommended standard set representing who should be tracked, what should be measured and at what time points, and what data are necessary to make meaningful comparisons. Using a modified Delphi method over a series of teleconferences, the group reached consensus for the Standard Set. Results and limitations We recommend that the Standard Set apply to men with newly diagnosed localized PCa treated with active surveillance, surgery, radiation, or other methods. The Standard Set includes acute toxicities occurring within 6 mo of treatment as well as patient-reported outcomes tracked regularly out to 10 yr. Patient-reported domains of urinary incontinence and irritation, bowel symptoms, sexual symptoms, and hormonal symptoms are included, and the recommended measurement tool is the Expanded Prostate Cancer Index Composite Short Form. Disease control outcomes include overall, cause-specific, metastasis-free, and biochemical relapse-free survival. Baseline clinical, pathologic, and comorbidity information is included to improve the interpretability of comparisons. Conclusions We have defined a simple, easily implemented set of outcomes that we believe should be measured in all men with localized PCa as a crucial first step in improving the value of care. Patient summary Measuring, reporting, and comparing identical outcomes across treatments and treatment centers will provide patients and providers with information to make informed treatment decisions. We defined a set of outcomes that we recommend being tracked for every man being treated for localized prostate cancer.
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Many studies report an association of cognitive and social experiential factors and related traits with dementia risk. Further, many clinical-pathologic studies find a poor correspondence between levels of neuropathology and the presence of dementia and level of cognitive impairment. The poor correspondence suggests that other factors contribute to the maintenance or loss of cognitive function, with factors associated with the maintenance of function referred to as neural or cognitive reserve. This has led investigators to examine the associations of cognitive and social experiential factors with neuropathology as a first step in disentangling the complex associations between these experiential risk factors, neuropathology, and cognitive impairment. Despite the consistent associations of a range of cognitive and social lifestyle factors with cognitive decline and dementia risk, the extant clinical-pathologic data find only a single factor from one cohort, linguistic ability, related to AD pathology. Other factors, including education, harm avoidance, and emotional neglect, are associated with cerebrovascular disease. Overall, the associations are weak. Some factors, such as education, social networks, and purpose in life, modify the relation of neuropathology to cognition. Finally, some factors such as cognitive activity appear to bypass known pathologies altogether suggesting a more direct association with biologic indices that promote person-specific differences in reserve and resilience. Future work will first need to replicate findings across more studies to ensure the veracity of the existing data. Second, effort is needed to identify the molecular substrates of neural reserve as potential mediators of the association of lifestyle factors with cognition.
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An aging population brings increasing burdens and costs to individuals and society arising from late-life cognitive decline, the causes of which are unclear. We aimed to identify factors predicting late-life cognitive decline. Participants were 889 community-dwelling 70-90-year-olds from the Sydney Memory and Ageing Study with comprehensive neuropsychological assessments at baseline and a 2-year follow-up and initially without dementia. Cognitive decline was considered as incident mild cognitive impairment (MCI) or dementia, as well as decreases in attention/processing speed, executive function, memory, and global cognition. Associations with baseline demographic, lifestyle, health and medical factors were determined. All cognitive measures showed decline and 14% of participants developed incident MCI or dementia. Across all participants, risk factors for decline included older age and poorer smelling ability most prominently, but also more education, history of depression, being male, higher homocysteine, coronary artery disease, arthritis, low health status, and stroke. Protective factors included marriage, kidney disease, and antidepressant use. For some of these factors the association varied with age or differed between men and women. Additional risk and protective factors that were strictly age- and/or sex-dependent were also identified. We found salient population attributable risks (8.7-49.5%) for older age, being male or unmarried, poor smelling ability, coronary artery disease, arthritis, stroke, and high homocysteine. Preventing or treating conditions typically associated with aging might reduce population-wide late-life cognitive decline. Interventions tailored to particular age and sex groups may offer further benefits.
Article
Objectives: To validate the Fullerton Advanced Balance (FAB) Scale for patients with idiopathic Parkinson disease (PD); and to compare the FAB Scale with the Mini-Balance Evaluation Systems Test (Mini-BESTest) and Berg Balance Scale (BBS). Design: Observational study to assess concurrent validity, test-retest, and interrater reliability of the FAB Scale in patients with PD and to compare the distribution of the scale with the Mini-BESTest and BBS. Setting: University hospital in an urban community. Participants: Patients with idiopathic PD (N=85; Hoehn and Yahr stages 1-4). Interventions: Not applicable. Main outcome measures: FAB Scale, Mini-BESTest, BBS, timed Up and Go test, Unified Parkinson's Disease Rating Scale, and visual analog scale. Results: Interrater (3 raters) and test-retest (3±1 d) reliability were high for all scales (ICCs≥.95). The FAB Scale was highly correlated with the Mini-BESTest (Spearman ρ=.87) and timed Up and Go test item of the Mini-BESTest (Spearman ρ=.83). In contrast with the BBS, the FAB Scale and Mini-BESTest have only minimal ceiling effects. The FAB Scale demonstrated the most symmetric distribution when compared with the Mini-BESTest and BBS (skewness: FAB scale: -.54; Mini-BESTest: -1.07; BBS: -2.14). Conclusions: The FAB Scale is a valid and reliable tool to assess postural control in patients with PD. No ceiling effect was noted for the FAB Scale. Although the items of the FAB Scale are more detailed when compared with the Mini-BESTest, interrater and test-retest reliability were excellent. The scale is a promising tool to detect small changes of the postural control system in individuals with PD.
Article
The relative impact of motor- and non-motor symptoms on health-related quality of life in early Parkinson's disease is poorly documented. 188 patients with incident Parkinson's disease from a population-based study were examined at the time of diagnosis, before initiation of dopaminergic treatment, with follow-up of 166 patients three years later. Health-related quality of life was assessed by the 36-item Short-form Health Survey (SF-36). Motor and non-motor variables were derived from the Unified Parkinson's disease rating scale and other established scales. Multiple regression analyses showed that the non-motor symptoms strongest associated with reduced SF-36 scores at diagnosis and three years later were depression, fatigue and sensory complaints. The motor symptoms most related to impaired SF-36 scores were problems with gait and activities of daily living that cover personal needs. The variance of SF-36 mental summary scores was much better explained by non-motor vs. motor symptoms, both at baseline (R(2) = 0.384 vs. 0.095) and 3 years later (R(2) = 0.441 vs. 0.195). Also SF-36 physical summary scores were better explained by non-motor vs. motor symptoms with R(2) = 0.372 vs. 0.322 at baseline and R(2) = 0.468 vs. 0.315 after 3 years. In early PD, including the phase before dopaminergic treatment is initiated, non-motor symptoms are more important for reduced health-related quality of life than motor symptoms. Fatigue, depression, sensory complaints and gait disturbances emerge as the most relevant symptoms and should be given corresponding attention in the management of patients with early PD.
Article
Non-motor symptom (NMS) differences between male Parkinson's disease (PD) and female PD, and between early-onset PD (EOPD) and late-onset PD (LOPD) in Chinese populations remain largely unknown. A total of 522 PD patients from Southwest China were included. Patients were assessed using the Non-Motor Symptom Scale (NMSS) and Unified PD Rating Scale (UPDRS). More NMS and significantly higher NMSS score were found in LOPD patients than in EOPD patients (9.3 ± 5.9 vs. 7.7 ± 5.6, P = 0.005; 37.4 ± 32.2 vs. 30.5 ± 28.9, P = 0.018), while no such differences were found between male and female patients. The NMS of gastrointestinal and urinary domains were more common in LOPD patients than in EOPD patients, whereas sexual dysfunction was more common in EOPD than in LOPD. The sleep/fatigue domain, the mood/apathy domain and "pain" symptoms were more prevalent and severe in female patients than in male patients while urinary symptoms were more common and severe in male patients. Significant positive correlations were observed between disease duration, Hoehn & Yahr stage, UPDRS Ⅲ, and NMSS score in the total sample, subgroups of both male and female patients as well as both EOPD and LOPD patients. NMS are common in the Chinese PD population. LOPD patients are likely to present with more and severe NMS than EOPD patients. Males are subjected to urinary symptoms and females are subjected to mood/apathy, sleep and pain symptoms.
Article
Objectives: to briefly outline the development and validation of the Parkinson's Disease Questionnaire (PDQ-39) and then to provide evidence for the use of the measure as either a profile of health status scores or a single index figure. Design: the PDQ-39 was administered in two surveys: a postal survey of patients registered with local branches of the Parkinson's Disease Society of Great Britain (n = 405) and a survey of patients attending neurology clinics for treatment for Parkinson's disease (n = 146). Data from the eight dimensions of the PDQ-39 were factor-analysed. This produced a single factor on the data from both surveys. Outcome measures: the eight dimensions of the PDQ-39 and the new single index score—the Parkins's disease summary index (PDSI), together with clinical assessments (the Columbia rating scale and the Hoehn and Yahr staging score). Results: in the postal survey 227 patients returned questionnaires (58.2%). All 146 patients approached in the clinic sample agreed to take part. Higher-order principal-components factor analysis was undertaken on the eight dimensions of the PDQ-39 and produced one factor on both datasets. Consequently it was decided that the scores of the eight domains could be summed to produce a single index figure. The psychometric properties of this index were explored using reliability tests and tests of construct validity. The newly derived single index was found to be both internally reliable and valid. Discussion: data from the PDQ-39 can be presented either in profile form or as a single index figure. The profile should be of value in studies aimed at determining the impact of treatment regimes upon particular aspects of functioning and well-being in patients with Parkinson's disease, while the PDSI will provide a summary score of the impact of the illness on functioning and well-being and will be of use in the evaluation of the overall effect of different treatments. Furthermore, the PDSI reduces the number of statistical comparisons and hence the role of chance when exploring data from the PDQ-39.
Article
Parkinson's disease (PD), following Alzheimer's disease, is the second-most common neurodegenerative disorder in the United States. A lack of treatment options for changing the trajectory of disease progression, in combination with an increasing elderly population, portends a rising economic burden on patients and payers. This study combined information from nationally representative surveys to create a burden of PD model. The model estimates disease prevalence, excess healthcare use and medical costs, and nonmedical costs for each demographic group defined by age and sex. Estimated prevalence rates and costs were applied to the U.S. Census Bureau's 2010 to 2050 population data to estimate current and projected burden based on changing demographics. We estimate that approximately 630,000 people in the United States had diagnosed PD in 2010, with diagnosed prevalence likely to double by 2040. The national economic burden of PD exceeds $14.4 billion in 2010 (approximately $22,800 per patient). The population with PD incurred medical expenses of approximately $14 billion in 2010, $8.1 billion higher ($12,800 per capita) than expected for a similar population without PD. Indirect costs (e.g., reduced employment) are conservatively estimated at $6.3 billion (or close to $10,000 per person with PD). The burden of chronic conditions such as PD is projected to grow substantially over the next few decades as the size of the elderly population grows. Such projections give impetus to the need for innovative new treatments to prevent, delay onset, or alleviate symptoms of PD and other similar diseases. © 2013 Movement Disorder Society.