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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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... Interestingly, among the outcomes assessed in the included RCTs, only cognitive impairment, motor functioning and voice disorders are included in the core outcomes set for PD [79], with the MDS-UPDRS being the suggested tool for their assessment. Thus, most outcomes selected by the trialists were targeting problems that are not at the core of PD pathology, which induced great heterogeneity and difficulty in pooling outcomes together in the effect direction plot. ...
... Despite the empirical use of the KD since ancient times, research on ketogenic therapies is still in its infancy. Trials with state-of-the-art methodology, with measurable endpoints selected from the set of outcomes for PD research consensus [79], are required to improve our understanding of the effect of KD on the PD phenotype. In addition, trials on PD prodromal phase [85][86][87], are also required to improve our understanding on the effects of diet on PD pathology, rather than only symptomatology. ...
Article
Objective: The aim of the present systematic review was to assess the efficacy of ketogenic therapy in Parkinson's disease (PD), using all available data from randomized controlled trials (RCTs) on humans and animal studies with PD models. Design: Systematic review of in vivo studies. Methods: Studies related to the research question were identified through searches in PubMed, Cochrane Central Register of Controlled Trials (CENTRAL), Scopus, clinicaltrials.gov and the gray literature, from inception until November 2021. Rayyan was employed to screen and identify all studies fulfilling the inclusion criteria. Cochrane's revised Risk of Bias 2.0 and SYRCLE tools evaluated bias in RCTs and animal studies, respectively. An effect direction plot was developed to synthesize the evidence of the RCTs. Results: Twelve studies were identified and included in the qualitative synthesis (4 RCTs and 8 animal trials). Interventions included ketogenic diets (KDs), supplementation with medium-chain triglyceride (MCT) oil, caprylic acid administration and ketone ester drinks. The animal research used zebrafish and rodents, and PD was toxin-induced. Based on the available RCTs, ketogenic therapy does not improve motor coordination and functioning, cognitive impairment, anthropometrics, blood lipids and glycemic control, exercise performance or voice disorders in patients with PD. The evidence is scattered and heterogenous, with single trials assessing different outcomes; thus, a synthesis of the evidence cannot be conclusive regarding the efficacy of ketogenic therapy. On the other hand, animal studies tend to demonstrate more promising results, with marked improvements in locomotor activity, dopaminergic activity, redox status, and inflammatory markers. Conclusions: Although animal studies indicate promising results, research on the effect of ketogenic therapy in PD is still in its infancy, with RCTs conducted on humans being heterogeneous and lacking PD-specific outcomes. More studies are required to recommend or refute the use of ketogenic therapy in PD.
... Although, management of chronic conditions is becoming more effective, it is often costly, with a greater requirement to document and prioritize the most effective and least costly (treatment) approaches. Recently, national and international clinical guidelines for both assessment and interventions for people with chronic conditions have emerged [3][4][5][6], and there is a comprehensive evidence that exercise is effective for increasing and maintaining function in people with chronic conditions [7]. Given this, health practitioners advocating for exercise are critically important in the management of patients with chronic disease. ...
... Additional assessment options tailored to patient severity and diagnoses may also be important. For some patient populations there are already core outcomes sets and for other outcome sets are being developed [3][4][5][6]. This adaption will not only increase the relevance of the tool for the patients but it would also facilitate comparisons between specific patient groups, internationally. ...
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Background The Danish Physiotherapy Research Database for chronic patients receiving Free of Charge Physiotherapy (PhysDB-FCP) was piloted over a 1-year period. The purpose of the PhysDB-FCP is to provide a user friendly digital online structured tool that standardizes initial and follow up clinical assessments generating data that can be used for clinical decision making and support future research in physiotherapy for patients with chronic disease. Although initial assessments were completed, the attrition rate was 73% and 90% at 3- and 6- months, respectively, which suggests problems with the current tool. Objective To evaluate the perspectives of the physiotherapists that used the PhysDB-FCP and propose changes to the tool based on this feedback. Materials and methods Fifty of the 103 physiotherapists introduced to the PhysDB-FCP completed an anonymous online survey. Physiotherapists were asked Likert/categorical and yes/no questions on experiences with the PhysDB-FCP within their practice, perceptions of patient experiences, suitability of the resources and support provided by the PhysDB-FCP working group and the ideal administration frequency of the assessments within the PhysDB-FCP. Open ended feedback on possible improvements to the PhysDB-FCP was also collected. Results Physiotherapists agreed that the PhysDB-FCP was useful for taking a physiotherapy assessment (74%) and the patient survey was useful for goal setting (72%). Although physiotherapists felt the PhysDB-FCP was well-defined (82%), only 36% would like to use a similar tool again. Generally, the PhysDB-FCP was too time-consuming, administered too frequently and included irrelevant items. For example, 72% of physiotherapists took >45 min to administer the assessment in the first consultation which was performed over multiple sessions. Conclusions The perspectives of physiotherapists using The PhysDB-FCP suggest specific changes that will ensure better use of the tool in future practice. Changes will likely involve administering the assessment less frequently (every 6-months to 1-year), shortening the assessment, and using diagnosis-specific assessment items.
... A potential barrier for implementing ICHOM Standard Sets is that they are independently developed by different working groups. Although it is important that Standard Sets are developed by people who have expertise in the particular condition, collaboration and harmonization across Standard Sets is currently limited, which leads to large differences and inconsistencies in selected PROs, terminology used, and recommended Patient-Reported Outcome Measures (PROMs), even for the same PROs [4,[6][7][8][9][10][11][12][13][14][15][16][17]. This complicates the implementation and use of Standard Sets in clinical practice. ...
Article
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Background The International Consortium for Health Outcomes Measurement (ICHOM) develops condition-specific Standard Sets of outcomes to be measured in clinical practice for value-based healthcare evaluation. Standard Sets are developed by different working groups, which is inefficient and may lead to inconsistencies in selected PROs and PROMs. We aimed to identify common PROs across ICHOM Standard Sets and examined to what extend these PROs can be measured with a generic set of PROMs: the Patient-Reported Outcomes Measurement Information System (PROMIS®). Methods We extracted all PROs and recommended PROMs from 39 ICHOM Standard Sets. Similar PROs were categorized into unique PRO concepts. We examined which of these PRO concepts can be measured with PROMIS. Results A total of 307 PROs were identified in 39 ICHOM Standard Sets and 114 unique PROMs are recommended for measuring these PROs. The 307 PROs could be categorized into 22 unique PRO concepts. More than half (17/22) of these PRO concepts (covering about 75% of the PROs and 75% of the PROMs) can be measured with a PROMIS measure. Conclusion Considerable overlap was found in PROs across ICHOM Standard Sets, and large differences in terminology used and PROMs recommended, even for the same PROs. We recommend a more universal and standardized approach to the selection of PROs and PROMs. Such an approach, focusing on a set of core PROs for all patients, measured with a system like PROMIS, may provide more opportunities for patient-centered care and facilitate the uptake of Standard Sets in clinical practice.
... 12 The International Consortium for Health Outcomes Measurement has developed a consensus set of outcomes for PD, which include the MDS-Unified Parkinson's Disease Rating Scale parts 1 and 2, 3 questions from the NMSQuest, the Parkinson's Disease Questionnaire-8, and 6 questions from the Dutch National PD registry. 25 Aspects of the National Institute of Neurological Disorders and Stroke PD Common Data Elements Set could also be considered as outcome measurements. However, studies are necessary to validate the usefulness of these particular scales/instruments in this context. ...
... Clinical trials for therapies targeting cognition in PD may benefit from recent design improvements. More sensitive outcomes, including computerized cogni tive testing and wearables to measure motor and other functions, together with the development of an inter nationally recognized set of core outcomes, as has been done for idiopathic PD 276 , particularly focused on patients with cognitive impairment and on the effects of specific interventions (such as nonpharmacological interventions), will allow the reporting and comparison of research outcomes in a standardized manner. More targeted selection criteria using current diagnostic criteria 7,8 and recommended assessments 120 , combined with both biomarkers and genetic risk factors aiming to assign the right person to the right intervention at an early disease stage, as well as biomarkers demonstrating target involvement, will offer opportunities for improved statistical power and cheaper trials. ...
Article
Parkinson disease (PD) is the second most common neurodegenerative disorder, affecting >1% of the population ≥65 years of age and with a prevalence set to double by 2030. In addition to the defining motor symptoms of PD, multiple non-motor symptoms occur; among them, cognitive impairment is common and can potentially occur at any disease stage. Cognitive decline is usually slow and insidious, but rapid in some cases. Recently, the focus has been on the early cognitive changes, where executive and visuospatial impairments are typical and can be accompanied by memory impairment, increasing the risk for early progression to dementia. Other risk factors for early progression to dementia include visual hallucinations, older age and biomarker changes such as cortical atrophy, as well as Alzheimer-type changes on functional imaging and in cerebrospinal fluid, and slowing and frequency variation on EEG. However, the mechanisms underlying cognitive decline in PD remain largely unclear. Cortical involvement of Lewy body and Alzheimer-type pathologies are key features, but multiple mechanisms are likely involved. Cholinesterase inhibition is the only high-level evidence-based treatment available, but other pharmacological and non-pharmacological strategies are being tested. Challenges include the identification of disease-modifying therapies as well as finding biomarkers to better predict cognitive decline and identify patients at high risk for early and rapid cognitive impairment. Cognitive impairment is common in patients with Parkinson disease and ranges in severity. This Primer reviews the epidemiology, pathophysiology, diagnosis and treatment of cognitive impairment in Parkinson disease and describes the effects on patient quality of life and the future outlook for the field.
... Currently, clinical studies focused on MSC-mediated PD treatment have mainly used age and time from diagnosis as inclusion or exclusion criteria. These studies also vary in the outcome measures for the evaluation of MSC treatment success (Table 2), although strides have recently been made to propose a global consensus of outcome measures for PD 51 . Moreover, these scales, which include the Hoehn and Yahr scale 52 , the Unified Parkinson's Disease Rating Scale (UPDRS) 53 , the Movement Disorder Society-sponsored revision of the UPDRS 54 , NMS-Quest 55 , and the physician-assisted non-motor symptoms scale 56 , are not only used for selection of the patient but also for measuring the clinical improvements. ...
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Parkinson’s disease (PD) is the second most common neurodegenerative disease characterized by the progressive loss of dopaminergic neurons in the substantia nigra pars compacta and the presence of Lewy bodies, which gives rise to motor and non-motor symptoms. Unfortunately, current therapeutic strategies for PD merely treat the symptoms of the disease, only temporarily improve the patients’ quality of life, and are not sufficient for completely alleviating the symptoms. Therefore, cell-based therapies have emerged as a novel promising therapeutic approach in PD treatment. Mesenchymal stem/stromal cells (MSCs) have arisen as a leading contender for cell sources due to their regenerative and immunomodulatory capabilities, limited ethical concerns, and low risk of tumor formation. Although several studies have shown that MSCs have the potential to mitigate the neurodegenerative pathology of PD, variabilities in preclinical and clinical trials have resulted in inconsistent therapeutic outcomes. In this review, we strive to highlight the sources of variability in studies using MSCs in PD therapy, including MSC sources, the use of autologous or allogenic MSCs, dose, delivery methods, patient factors, and measures of clinical outcome. Available evidence indicates that while the use of MSCs in PD has largely been promising, conditions need to be standardized so that studies can be effectively compared with one another and experimental designs can be improved upon, such that this body of science can continue to move forward.
... Recently, the International Consortium for Health Outcomes Measurement published a consensus set of outcomes for PD and this may be a valuable tool for future trials. 29 Moreover, this meta-analysis highlights the variety of physiotherapy interventions used to treat PD. Specific trials are needed to directly compare different interventions with standardized characteristics regarding training regime, frequency, intensity, duration, and progression. ...
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Background: Physiotherapy is a commonly prescribed intervention for people with Parkinson's disease (PD). Conventional types of physiotherapy have been studied extensively, while novel modalities are being developed and evaluated. Objective: To evaluate the effectiveness of conventional and more recent physiotherapy interventions for people with PD. The meta-analysis performed as part of the 2014 European Physiotherapy Guideline for PD was used as the starting point and updated with the latest evidence. Methods: We performed a systematic search in PubMed, CINAHL, Embase, and Web of Science. Randomized controlled trials comparing any physiotherapy intervention with no intervention or sham treatment were included. Trials were classified into 12 categories: conventional physiotherapy, resistance training, treadmill training, strategy training, dance, martial arts, aerobic exercises, hydrotherapy, balance and gait training, dual tasking, exergaming, and Nordic walking. Outcomes included motor symptoms, balance, gait, and quality of life, and are presented as standardized mean differences. The GRADE (Grading of Recommendations, Assessment, Development and Evaluation) approach was used to systematically appraise methodological quality. Results: A total of 191 trials with 7998 participants were included. Conventional physiotherapy significantly improved motor symptoms, gait, and quality of life. Resistance training improved gait. Treadmill training improved gait. Strategy training improved balance and gait. Dance, Nordic walking, balance and gait training, and martial arts improved motor symptoms, balance, and gait. Exergaming improved balance and quality of life. Hydrotherapy improved balance. Finally, dual task training did not significantly improve any of the outcomes studied. Conclusions: This meta-analysis provides a comprehensive overview of the evidence for the effectiveness of different physiotherapy interventions in the management of PD, allowing clinicians and patients to make an evidence-based decision for specific treatment modalities. Further work is needed to directly compare the relative efficacy of the various treatments.
Article
Objective: This is the first study applying Clinimetric Patient-Reported Outcome Measures (CLIPROM) criteria to evaluate the construct validity, sensitivity, and clinical utility of the SCL-90-R in patients with Parkinson’s disease (PD). Methods: A Rasch analysis was conducted using a sample of 488 PD outpatients. Results: Testing for dimensionality revealed that less than 5% of t-tests were significant, indicating that the SCL-90-R subscales entailed the property of construct validity. As to the total score, a Person Separation Reliability Index of .96 was found. Conclusions: The SCL-90-R total score is a sensitive screening measure that can be used not only to differentiate healthy stress reactions from symptoms of psychological distress but also to detect PD patients with an increased risk for psychiatric complications. As to the subscales, the brief versions that did not include misfitting items should be used to assess the severity of specific symptoms of psychological distress affecting PD patients.
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Objectives A core outcome set (COS) describes a minimum set of outcomes to be reported by all clinical trials of one healthcare condition. Delphi surveys are frequently used to achieve consensus on core outcomes. International input is important to achieve global COS uptake. We aimed to investigate participant representation in international Delphi surveys, with reference to the inclusion of patients and participants from low and middle income countries as stakeholders (LMICs). Design Systematic review. Data sources EMBASE, Medline, Web of Science, COMET database and hand-searching. Eligibility criteria Protocols and studies describing Delphi surveys used to develop an international COS for trial reporting, published between 1 January 2017 and 6 June 2019. Data extraction and synthesis Delphi participants were grouped as patients or healthcare professionals (HCPs). Participants were considered international if their country of origin was different to that of the first or senior author. Data extraction included participant numbers, country of origin, country income group and whether Delphi surveys were translated. We analysed the impact these factors had on outcome prioritisation. Results Of 90 included studies, 69% (n=62) were completed and 31% (n=28) were protocols. Studies recruited more HCPs than patients (median 60 (IQR 30–113) vs 30 (IQR 14–66) participants, respectively). A higher percentage of HCPs was international compared with patients (57% (IQR 37–78) vs 20% (IQR 0–68)). Only 31% (n=28) studies recruited participants from LMICs. Regarding recruitment from LMICs, patients were under-represented (16% studies; n=8) compared with HCPs (22%; n=28). Few (7%; n=6) studies translated Delphi surveys. Only 3% studies (n=3) analysed Delphi responses by geographical location; all found differences in outcome prioritisation. Conclusions There is a disproportionately lower inclusion of international patients, compared with HCPs, in COS-development Delphi surveys, particularly within LMICs. Future international Delphi surveys should consider exploring for geographical and income-based differences in outcome prioritisation. PROSPERO registration number CRD42019138519.
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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.