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  1. Article ; Online: Qualitative interviews to support development of a patient-reported companion measure to the Glucocorticoid Toxicity Index.

    Howell, Timothy A / Matza, Louis S / Stone, John H / Gelinas, Deborah / Stone, Martha N / Rao, Vijayaraghava T S / Phillips, Glenn A

    Journal of the neurological sciences

    2024  Volume 458, Page(s) 122907

    Abstract: Introduction: Glucocorticoids (GCs) are associated with multiple toxicities that have substantial impact on patients. We conducted qualitative interviews with patients to identify the toxicities that are most relevant from their perspective, with the ... ...

    Abstract Introduction: Glucocorticoids (GCs) are associated with multiple toxicities that have substantial impact on patients. We conducted qualitative interviews with patients to identify the toxicities that are most relevant from their perspective, with the goal of creating a patient-reported companion measure to the Glucocorticoid Toxicity Index (GTI), a clinician-facing instrument.
    Methods: Thirty-one patients with recent or current GC use participated in concept elicitation interviews. Participants received GC treatment for myasthenia gravis, chronic inflammatory demyelinating polyradiculoneuropathy, vasculitis, or systemic lupus erythematosus. Transcripts were coded following a thematic analysis approach.
    Results: Participants reported more than 100 toxicities they believed to be associated with their GC medications. Common toxicities included weight gain (87%), increased appetite (84%), insomnia/sleep problems (77%), cognitive impairment/brain fog (71%), easy bruising (68%), anxiety (65%), irritability/short temper (65%), and osteoporosis (39%). These toxicities often centered on self-esteem, neuropsychiatric effects, skin toxicities, and musculoskeletal function. They can be categorized into domains such emphasizing neuropsychiatric, metabolic/endocrine, musculoskeletal, and dermatological effects, highlighting aspects of GC toxicity that patients are uniquely positioned to appreciate and report.
    Conclusion: Our results confirm that the toxicities associated with GCs are pervasive and diverse, with substantial impact on patients' lives. These data will be used to inform the development of a patient-reported outcome measure assessing GC toxicity. This patient-reported instrument will be designed to complement the clinician-reported GTI, facilitating a more detailed understanding of the nuances of change in GC toxicity.
    MeSH term(s) Humans ; Glucocorticoids/therapeutic use ; Lupus Erythematosus, Systemic ; Patient Reported Outcome Measures ; Vasculitis
    Chemical Substances Glucocorticoids
    Language English
    Publishing date 2024-01-30
    Publishing country Netherlands
    Document type Journal Article
    ZDB-ID 80160-4
    ISSN 1878-5883 ; 0022-510X ; 0374-8642
    ISSN (online) 1878-5883
    ISSN 0022-510X ; 0374-8642
    DOI 10.1016/j.jns.2024.122907
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: Utilization of MG-ADL in myasthenia gravis clinical research and care.

    Muppidi, Srikanth / Silvestri, Nicholas J / Tan, Robin / Riggs, Kimberly / Leighton, Trevor / Phillips, Glenn A

    Muscle & nerve

    2022  Volume 65, Issue 6, Page(s) 630–639

    Abstract: The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures myasthenia gravis (MG) symptoms and functional status. The objective of the current review is to summarize the psychometric properties of the MG- ... ...

    Abstract The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures myasthenia gravis (MG) symptoms and functional status. The objective of the current review is to summarize the psychometric properties of the MG-ADL and published evidence of MG-ADL use. A targeted literature review for published studies of the MG-ADL was conducted using a database and gray literature search. A total of 48 publications and 35 clinical trials were included. Studies indicated that the MG-ADL is a reliable and valid measure that has been used as an outcome in clinical trials and observational studies to measure MG symptoms and response to treatment. While most often used as a secondary endpoint in clinical trials, its use as a primary endpoint has increased in recent years. The most common MG-ADL endpoint is change in MG-ADL score from baseline, although there has been an increase in the analysis of a responder threshold using the MG-ADL. A new concept of minimal symptom expression (MSE) has emerged more recently. Duration of treatment effect is another important construct that is being increasingly evaluated using the MG-ADL. The use of the MG-ADL as a primary endpoint in clinical trials and in responder threshold analyses to indicate treatment improvement has increased in recent years. MSE using the MG-ADL shows promise in helping to determine success of treatment and may be the aspirational goal of MG treatment for the future once validated, particularly given the evolving treatment landscape in MG.
    MeSH term(s) Activities of Daily Living ; Humans ; Myasthenia Gravis ; Psychometrics
    Language English
    Publishing date 2022-01-06
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 438353-9
    ISSN 1097-4598 ; 0148-639X
    ISSN (online) 1097-4598
    ISSN 0148-639X
    DOI 10.1002/mus.27476
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  3. Article ; Online: Estimating health state utilities associated with a rare disease: familial chylomicronemia syndrome (FCS).

    Matza, Louis S / Phillips, Glenn A / Howell, Timothy A / Ciffone, Nicole / Ahmad, Zahid

    Journal of medical economics

    2020  Volume 23, Issue 9, Page(s) 978–984

    Abstract: Aims: ...

    Abstract Aims:
    MeSH term(s) Absenteeism ; Canada ; Cost-Benefit Analysis ; Female ; Health Status ; Humans ; Hyperlipoproteinemia Type I/complications ; Hyperlipoproteinemia Type I/physiopathology ; Hyperlipoproteinemia Type I/psychology ; Interviews as Topic ; Male ; Middle Aged ; Pancreatitis/etiology ; Patient Preference ; Quality of Life ; Rare Diseases ; Triglycerides/blood ; United Kingdom
    Chemical Substances Triglycerides
    Language English
    Publishing date 2020-07-01
    Publishing country England
    Document type Journal Article
    ZDB-ID 2270945-9
    ISSN 1941-837X ; 1369-6998
    ISSN (online) 1941-837X
    ISSN 1369-6998
    DOI 10.1080/13696998.2020.1776719
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  4. Article ; Online: Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT).

    Saccà, Francesco / Barnett, Carolina / Vu, Tuan / Peric, Stojan / Phillips, Glenn A / Zhao, Sihui / Qi, Cynthia Z / Gelinas, Deborah / Chiroli, Silvia / Verschuuren, Jan J G M

    Journal of neurology

    2023  Volume 270, Issue 4, Page(s) 2096–2105

    Abstract: There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as the Myasthenia Gravis Quality of ... ...

    Abstract There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as the Myasthenia Gravis Quality of Life 15-item revised (MG-QOL15r) and EuroQoL 5-Dimensions 5-Levels (EQ-5D-5L) are vital for evaluating the clinical benefit of therapeutic interventions in patients with MG, as they assess the burden of disease and the effectiveness of treatment, as perceived by patients. The phase 3 ADAPT study (NCT03669588) demonstrated that efgartigimod-a novel neonatal Fc receptor inhibitor-was well tolerated and that acetylcholine receptor antibody-positive (AChR-Ab+) participants who received efgartigimod had statistically significant improvements in MG-specific clinical scale scores. The ancillary data reported here, which cover an additional treatment cycle, show that these participants had similar significant improvements in HRQoL measures, the MG-QOL15r and EQ-5D-5L utility and visual analog scales, and that these improvements were maintained in the second treatment cycle. Positive effects on HRQoL were rapid, seen as early as the first week of treatment in both treatment cycles, and maintained for up to 4 weeks in the follow-up-only portion of treatment cycles. The pattern of improvements in HRQoL paralleled changes in immunoglobulin G level, and correlational analyses show that improvements were consistent across HRQoL measures and with clinical efficacy measures in the ADAPT study. The substantial and durable improvements in HRQoL end points in this study demonstrate the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.
    MeSH term(s) Infant, Newborn ; Humans ; Quality of Life ; Myasthenia Gravis/drug therapy ; Myasthenia Gravis/diagnosis ; Receptors, Cholinergic ; Treatment Outcome ; Autoantibodies
    Chemical Substances Receptors, Cholinergic ; Autoantibodies
    Language English
    Publishing date 2023-01-04
    Publishing country Germany
    Document type Randomized Controlled Trial ; Clinical Trial, Phase III ; Journal Article
    ZDB-ID 187050-6
    ISSN 1432-1459 ; 0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X
    ISSN (online) 1432-1459
    ISSN 0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X
    DOI 10.1007/s00415-022-11517-w
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  5. Article ; Online: Validation of an ambient measurement system (AMS) for walking speed.

    Varsanik, Jonathan S / Kimmel, Zebadiah M / de Moor, Carl / Gabel, Wendy / Phillips, Glenn A

    Journal of medical engineering & technology

    2017  Volume 41, Issue 5, Page(s) 362–374

    Abstract: Walking speed is an important indicator of worsening in a variety of neurological and neuromuscular diseases, yet typically is measured only infrequently and in a clinical setting. Passive measurement of walking speed at home could provide valuable ... ...

    Abstract Walking speed is an important indicator of worsening in a variety of neurological and neuromuscular diseases, yet typically is measured only infrequently and in a clinical setting. Passive measurement of walking speed at home could provide valuable information to track the progression of many neuromuscular conditions. The purpose of this study was to validate the measurement of walking speed by a shelf-top ambient measurement system (AMS) that can be placed in a patient's home. Twenty-eight healthy adults (16 male, 12 female) were asked to walk three pre-defined routes two times each (total of 168 traversals). For each traversal, walking speed was measured simultaneously by five sources: two independent AMSs and three human timers with stopwatches. Measurements across the five sources were compared by generalised estimating equations (GEE). Correlation coefficients compared pairwise for walking speeds across the two AMSs, three human timers, and three routes all exceeded 0.86 (p < .0001), and for AMS-to-AMS exceeded 0.92 (p < .0001). Aggregated across all routes, there was no significant difference in measured walking speeds between the two AMSs (p = .596). There was a statistically significant difference between the AMSs and human timers of 8.5 cm/s (p < .0001), which is comparable to differences reported for other non-worn sensors. The tested AMS demonstrated the ability to automatically measure walking speeds comparable to manual observation and recording, which is the current standard for assessing walking speed in a clinical setting. The AMS may be used to detect changes in walking speed in community settings.
    Language English
    Publishing date 2017-07
    Publishing country England
    Document type Journal Article
    ZDB-ID 243092-7
    ISSN 1464-522X ; 0309-1902
    ISSN (online) 1464-522X
    ISSN 0309-1902
    DOI 10.1080/03091902.2017.1308025
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  6. Article: Movement measurements at home for multiple sclerosis: walking speed measured by a novel ambient measurement system.

    Smith, Victoria Mj / Varsanik, Jonathan S / Walker, Rachel A / Russo, Andrew W / Patel, Kevin R / Gabel, Wendy / Phillips, Glenn A / Kimmel, Zebadiah M / Klawiter, Eric C

    Multiple sclerosis journal - experimental, translational and clinical

    2018  Volume 4, Issue 1, Page(s) 2055217317753465

    Abstract: Background: Gait disturbance is a major contributor to clinical disability in multiple sclerosis (MS). A sensor was developed to assess walking speed at home for people with MS using infrared technology in real-time without the use of wearables.: ... ...

    Abstract Background: Gait disturbance is a major contributor to clinical disability in multiple sclerosis (MS). A sensor was developed to assess walking speed at home for people with MS using infrared technology in real-time without the use of wearables.
    Objective: To develop continuous in-home outcome measures to assess gait in adults with MS.
    Methods: Movement measurements were collected continuously for 8 months from six people with MS. Average walking speed and peak walking speed were calculated from movement data, then analyzed for variability over time, by room (location), and over the course of the day. In-home continuous gait outcomes and variability were correlated with standard in-clinic gait outcomes.
    Results: Measured in-home average walking speed of participants ranged from 0.33 m/s to 0.96 m/s and peak walking speed ranged from 0.89 m/s to 1.51 m/s. Mean total within-participant coefficient of variation for daily average walking speed and peak walking speed were 10.75% and 10.93%, respectively. Average walking speed demonstrated a moderately strong correlation with baseline Timed 25-Foot Walk (r
    Conclusion: New non-wearable technology provides reliable and continuous in-home assessment of walking speed.
    Language English
    Publishing date 2018-01-23
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2841884-0
    ISSN 2055-2173 ; 2055-2173
    ISSN (online) 2055-2173
    ISSN 2055-2173
    DOI 10.1177/2055217317753465
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  7. Article ; Online: Qualitative Research on Fatigue Associated with Depression: Content Validity of the Fatigue Associated with Depression Questionnaire (FAsD-V2).

    Matza, Louis S / Murray, Lindsey T / Phillips, Glenn A / Konechnik, Thomas J / Dennehy, Ellen B / Bush, Elizabeth N / Revicki, Dennis A

    The patient

    2015  Volume 8, Issue 5, Page(s) 433–443

    Abstract: Background: Fatigue is one of the most common symptoms of major depressive disorder (MDD). The Fatigue Associated with Depression Questionnaire (FAsD) was developed to assess fatigue and its impact in patients with MDD. The current article presents the ... ...

    Abstract Background: Fatigue is one of the most common symptoms of major depressive disorder (MDD). The Fatigue Associated with Depression Questionnaire (FAsD) was developed to assess fatigue and its impact in patients with MDD. The current article presents the qualitative research conducted to develop and examine the content validity of the FAsD and FASD-Version 2 (FAsD-V2).
    Methods: Three phases of qualitative research were conducted with patients recruited from a geographically diverse range of clinics in the US. Phase I included concept elicitation focus groups, followed by cognitive interviews. Phase II employed similar techniques in a more targeted sample. Phase III included cognitive interviews to examine whether minor edits made after Phase II altered comprehensibility of the instrument. Concept elicitation focused on patients' perceptions of fatigue and its impact. Cognitive interviews focused on comprehension, clarity, relevance, and comprehensiveness of the instrument. Data were collected using semi-structured discussion guides. Thematic analyses were conducted and saturation was examined.
    Results: A total of 98 patients with MDD were included. Patients' statements during concept elicitation in phases I and II supported item development and content. Cognitive interviews supported the relevance of the instrument in the target population, and patients consistently demonstrated a good understanding of the instructions, items, response options, and recall period. Minor changes to instructions for the FAsD-V2 did not affect interpretation of the instrument.
    Conclusions: This qualitative research supports the content validity of the FAsD and FAsD-V2. These results add to previous quantitative psychometric analysis suggesting the FAsD-V2 is a useful tool for assessing fatigue and its impact in patients with MDD.
    MeSH term(s) Adult ; Depressive Disorder, Major/complications ; Depressive Disorder, Major/psychology ; Fatigue/diagnosis ; Fatigue/etiology ; Fatigue/psychology ; Female ; Focus Groups ; Humans ; Interviews as Topic ; Male ; Middle Aged ; Psychometrics ; Qualitative Research ; Reproducibility of Results ; Sickness Impact Profile ; Surveys and Questionnaires
    Language English
    Publishing date 2015-01-19
    Publishing country New Zealand
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Validation Studies
    ZDB-ID 2466680-4
    ISSN 1178-1661 ; 1178-1653
    ISSN (online) 1178-1661
    ISSN 1178-1653
    DOI 10.1007/s40271-014-0107-7
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  8. Article ; Online: Development and validation of a patient-report measure of fatigue associated with depression.

    Matza, Louis S / Phillips, Glenn A / Revicki, Dennis A / Murray, Lindsey / Malley, Karen G

    Journal of affective disorders

    2011  Volume 134, Issue 1-3, Page(s) 294–303

    Abstract: Introduction: Among researchers and clinicians who treat depression, there is growing interest in treatment of specific residual symptoms such as fatigue. However, there is no available measure that provides a detailed assessment of fatigue associated ... ...

    Abstract Introduction: Among researchers and clinicians who treat depression, there is growing interest in treatment of specific residual symptoms such as fatigue. However, there is no available measure that provides a detailed assessment of fatigue associated with depression. Thus, the purpose of the current study was to develop and validate a patient-reported outcome measure designed to assess depression-related fatigue and its impact.
    Methods: The Fatigue Associated with Depression Questionnaire (FAsD) was developed based on literature review, clinician interviews, and focus groups and cognitive debriefing interviews with patients. Then, a draft questionnaire was administered to a sample of patients with depression. Statistical analysis first focused on item reduction and subscale identification, followed by psychometric evaluation of the FAsD.
    Results: The per protocol sample (n = 317) was primarily female (68.1%), with a mean age of 47.0 years. Based on item performance and exploratory factor analysis, three items were dropped from the draft FAsD, yielding a final 13-item questionnaire with two subscales (experience and impact). The FAsD demonstrated good internal consistency reliability (Cronbach's alphas ≥ 0.88) and test-retest reliability (intraclass correlation coefficients ≥ 0.78). The FAsD demonstrated construct validity through strong correlations with measures assessing fatigue/energy and symptoms of depression. The FAsD also discriminated among groups of participants differentiated by clinician ratings of global severity of depression (CGI-S).
    Limitations: Limitations include heterogeneity of current treatments received by the sample, as well as lack of knowledge regarding the extent to which FAsD scores were influenced by patients' treatments rather than the depression itself.
    Conclusions: In this initial psychometric evaluation, the FAsD demonstrated good reliability, validity, and factor structure. This questionnaire may be a useful tool for evaluating treatment interventions that focus specifically on fatigue associated with depression.
    MeSH term(s) Adult ; Depression/complications ; Depressive Disorder ; Factor Analysis, Statistical ; Fatigue/complications ; Female ; Focus Groups ; Humans ; Male ; Middle Aged ; Psychometrics/instrumentation ; Psychometrics/statistics & numerical data ; Reproducibility of Results ; Research Design ; Research Report ; Surveys and Questionnaires ; Weights and Measures
    Language English
    Publishing date 2011-11
    Publishing country Netherlands
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Validation Studies
    ZDB-ID 135449-8
    ISSN 1573-2517 ; 0165-0327
    ISSN (online) 1573-2517
    ISSN 0165-0327
    DOI 10.1016/j.jad.2011.06.028
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  9. Article ; Online: Responder definition of the Multiple Sclerosis Impact Scale physical impact subscale for patients with physical worsening.

    Phillips, Glenn A / Wyrwich, Kathleen W / Guo, Shien / Medori, Rossella / Altincatal, Arman / Wagner, Linda / Elkins, Jacob

    Multiple sclerosis (Houndmills, Basingstoke, England)

    2014  Volume 20, Issue 13, Page(s) 1753–1760

    Abstract: Background: The 29-item Multiple Sclerosis Impact Scale (MSIS-29) was developed to examine the impact of multiple sclerosis (MS) on physical and psychological functioning from a patient's perspective.: Objective: To determine the responder definition ...

    Abstract Background: The 29-item Multiple Sclerosis Impact Scale (MSIS-29) was developed to examine the impact of multiple sclerosis (MS) on physical and psychological functioning from a patient's perspective.
    Objective: To determine the responder definition (RD) of the MSIS-29 physical impact subscale (PHYS) in a group of patients with relapsing-remitting MS (RRMS) participating in a clinical trial.
    Methods: Data from the SELECT trial comparing daclizumab high-yield process with placebo in patients with RRMS were used. Physical function was evaluated in SELECT using three patient-reported outcomes measures and the Expanded Disability Status Scale (EDSS). Anchor- and distribution-based methods were used to identify an RD for the MSIS-29.
    Results: Results across the anchor-based approach suggested MSIS-29 PHYS RD values of 6.91 (mean), 7.14 (median) and 7.50 (mode). Distribution-based RD estimates ranged from 6.24 to 10.40. An RD of 7.50 was selected as the most appropriate threshold for physical worsening based on corresponding changes in the EDSS (primary anchor of interest).
    Conclusion: These findings indicate that a ≥7.50 point worsening on the MSIS-29 PHYS is a reasonable and practical threshold for identifying patients with RRMS who have experienced a clinically significant change in the physical impact of MS.
    MeSH term(s) Adult ; Antibodies, Monoclonal, Humanized/administration & dosage ; Daclizumab ; Disability Evaluation ; Dose-Response Relationship, Drug ; Double-Blind Method ; Female ; Humans ; Immunosuppressive Agents/administration & dosage ; Male ; Multiple Sclerosis, Relapsing-Remitting/complications ; Multiple Sclerosis, Relapsing-Remitting/drug therapy ; Self Report ; Severity of Illness Index
    Chemical Substances Antibodies, Monoclonal, Humanized ; Immunosuppressive Agents ; daclizumab HYP ; Daclizumab (CUJ2MVI71Y)
    Language English
    Publishing date 2014-04-16
    Publishing country England
    Document type Journal Article ; Randomized Controlled Trial ; Research Support, Non-U.S. Gov't
    ZDB-ID 1290669-4
    ISSN 1477-0970 ; 1352-4585
    ISSN (online) 1477-0970
    ISSN 1352-4585
    DOI 10.1177/1352458514530489
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  10. Article ; Online: Antipsychotic adherence, switching, and health care service utilization among Medicaid recipients with schizophrenia.

    Noordsy, Douglas L / Phillips, Glenn A / Ball, Daniel E / Linde-Zwirble, Walter T

    Patient preference and adherence

    2010  Volume 4, Page(s) 263–271

    Abstract: Objective: To evaluate health care resource utilization in patients with schizophrenia who continued newly prescribed antipsychotic medications, compared with those switching to different treatments.: Methods: Adults with schizophrenia in the ... ...

    Abstract Objective: To evaluate health care resource utilization in patients with schizophrenia who continued newly prescribed antipsychotic medications, compared with those switching to different treatments.
    Methods: Adults with schizophrenia in the California Medicaid (MediCal) database who initiated treatment with index medications in 1998-2001, were classified as having: 1) abandoned antipsychotic medications; 2) switched to another medication; or 3) continued with the index antipsychotic, for up to 6 months after the index date.
    Results: Of 2300 patients meeting eligibility criteria, 1382 (60.1%) continued index medications, 480 (20.9%) switched, and 438 (19.0%) abandoned antipsychotic treatment. Utilization in several resource categories occurred significantly more frequently among patients whose regimens were switched (vs those continuing index medications). These included using psychiatric (24.2% vs 14.5%; P < 0.001) or nonpsychiatric (31.5% vs 24.3%; P < 0.05) emergency services; being admitted to a hospital (10.6% vs 7.4%; P < 0.05); making nonpsychiatric outpatient hospital visits (43.3% vs 36.4%; P < 0.05) or nonpsychiatric physician visits (62.7% vs 56.4%; P < 0.05); and using other outpatient psychiatric (53.3% vs 40.7%; P < 0.001) or nonpsychiatric (82.7% vs 74.6%; P < 0.001) services.
    Conclusions: Switching antipsychotic medications is associated with significantly increased health care resource utilization (vs continuing treatment).
    Language English
    Publishing date 2010-07-21
    Publishing country New Zealand
    Document type Journal Article
    ZDB-ID 2455848-5
    ISSN 1177-889X ; 1177-889X
    ISSN (online) 1177-889X
    ISSN 1177-889X
    DOI 10.2147/ppa.s6053
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