The validity, responsiveness, and score interpretation of the PROMISnq Physical Function – Multiple Sclerosis 15a short form in multiple sclerosis

Kamudoni, Paul; Amtmann, Dagmar; Johns, Jeffrey; Cook, Karon F.; Salem, Rana; Salek, Sam; Raab, Jana; Middleton, Rod; Repovic, Pavle; Alschuler, Kevin N.; Geldern, Gloria von; Wundes, Annette; Barrett, Amy; Olayinka-Amao, Oyebimpe; Henke, Christian

doi:10.1016/j.msard.2022.103753

Cited by 3 publications

(9 citation statements)

References 51 publications

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“…It is important for the development of welldefined and reliable PRO instruments that there is a detailed understanding of patients' experiences, and specific disease concepts, prior to the development of PRO instruments [29]. This study aimed to identify the symptoms and impacts that PwMS experience which affect their daily lives; the in-depth patient experience narratives supported the development of novel PRO measures of fatigue and physical function that are currently in use in MS trials [27,33].…”

Section: Discussionmentioning

confidence: 99%

“…The concepts identified in the CEIs and incorporated in the conceptual model are key to the development of targeted PRO instruments for MS. Assessment of fatigue by PROs is considered important by the EMA in providing a complete picture of disability [44]. Four MSspecific clinical outcome assessments have been submitted to the FDA Clinical Outcome Assessment qualification program, which will conclude whether the assessments are well defined and reliable for use in MS [45]: PROMIS Fatigue (MS) 8a [27], PROMIS Physical Function (MS) 15a [33], Symbol Digit Modality Test (processing speed) [46], and ActibeltÒ in MS (real-world walking speed) [47]. Concepts Fig.…”

Section: Implications For Outcomes Measurementmentioning

confidence: 99%

“…The specific objectives were (1) to explore the experiences of people with RRMS, including their symptoms and the impacts of the disease on their daily lives; (2) to develop a conceptual model of MS outcomes, outlining key disease symptoms and impact concepts, and their interrelationships to support the development of fit-for-purpose PRO instruments. The CEIs were conducted in the initial phase of a research program to develop novel, MS-specific PRO measures and the results ultimately supported the development of MSspecific PRO measures of physical function [33] and fatigue [27] that have been included in the phase 3 evolutionRMS 1 and 2 trials (NCT04338022, NCT04338061) of evobrutinib in people with relapsing MS (RRMS or secondary progressive MS [SPMS] with superimposed relapses).…”

Section: Introductionmentioning

confidence: 95%

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Understanding the Symptoms and Impacts Experienced by People with Relapsing–Remitting MS: A Qualitative Investigation Using Semi-Structured Interviews

Barrett,

Olayinka-Amao,

Ziemssen

et al. 2024

Neurol Ther

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Introduction Multiple sclerosis (MS) is a disabling disease with unpredictable clinical manifestations. As clinical assessments may not fully capture the impact of MS on quality of life, they can be complemented by patient-reported outcome (PRO) measures to provide a more comprehensive picture of MS disease state and impact. The objectives of this study were to explore the experiences of people with relapsing–remitting MS, including symptoms and impacts on daily life, and to provide a conceptual model of MS outcomes. Methods A literature review of studies that evaluated the experiences of people with MS was completed and combined with semi-structured concept elicitation interviews conducted with 14 people with relapsing–remitting MS in the USA. Results The average age of the 14 participants was 43.9 (range 25–64) years, most were White (78.6%) and female (78.6%), and the mean duration since diagnosis was 6.6 (2–10) years. The most bothersome symptoms identified included fatigue ( n = 9), cognitive dysfunction ( n = 5), mobility/difficulty with walking ( n = 3), and vision problems ( n = 3). The most commonly reported impacts on daily life were balance problems/instability ( n = 13), work life/productivity ( n = 12), difficulty walking ( n = 11), daily activities/household chores ( n = 11), and leisure activities ( n = 10). Conclusion There was a high frequency of concepts associated with physical function, fatigue, and sensory-motor actions. A conceptual model was developed that captures the disease symptoms, impairments, and impacts identified in the interviews as well as known processes and symptoms identified in the literature search. This model underpins the appropriateness of PRO instruments, such as the PROMIS Fatigue (MS) 8a and PROMIS Physical Function (MS) 15a, which evaluate symptoms and impacts that matter most to people with MS. Supplementary Information The online version contains supplementary material available at 10.1007/s40120-024-00584-y.

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Section: Discussionmentioning

confidence: 99%

Section: Implications For Outcomes Measurementmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 95%

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Understanding the Symptoms and Impacts Experienced by People with Relapsing–Remitting MS: A Qualitative Investigation Using Semi-Structured Interviews

Barrett,

Olayinka-Amao,

Ziemssen

et al. 2024

Neurol Ther

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“… Patients’ cognitive and emotional representations of their illness as assessed by the Brief Illness Perception Questionnaire ( 68 , 69 ). Physical function and fatigue severity assessment as determined by the Patient-Reported Outcomes Measurement Information System (PROMIS), Short Form (SF)-15, and SF-8 questionnaires ( 70 , 71 ). …”

Section: Methodsmentioning

confidence: 99%

“…Physical function and fatigue severity assessment as determined by the Patient-Reported Outcomes Measurement Information System (PROMIS), Short Form (SF)-15, and SF-8 questionnaires ( 70 , 71 ).…”

Section: Methodsmentioning

confidence: 99%

Prospective observational study to evaluate treatment satisfaction and effectiveness in patients with relapsing multiple sclerosis starting cladribine tablets (CLADREAL) in Italy

Filippi,

Ferrè,

Zanetta

et al. 2024

Front. Neurol.

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Disease-modifying therapies (DMTs) for multiple sclerosis (MS) reduce relapse frequency, magnetic resonance imaging (MRI) activity, and slow disability progression. Numerous DMTs are approved for relapsing forms of MS although real-world data on patient-reported outcomes (PROs) and quality of life (QoL) are needed to inform treatment choice. Immune reconstitution therapy with cladribine tablets is a highly effective treatment for relapsing MS (RMS). We present the protocol for an observational study to prospectively assess the effectiveness of cladribine tablets on clinical and MRI parameters as well as on PROs, including treatment satisfaction, QoL, sleep quality, self-perceived health, fatigue, and physical function. Enrolled patients at study sites in Italy will be adults with RMS (including relapsing–remitting and active secondary progressive MS) who are either treatment naïve or have received at least one first-line disease modifying DMT or no more than one second-line DMT. The primary objective will be change in global treatment satisfaction measured with the Treatment Satisfaction Questionnaire for Medication Version 1.4 approximately 24 months after initiating cladribine tablets in patients switching from previous DMTs. Secondary objectives will include global treatment satisfaction at earlier timepoints, will comprise treatment naïve patients, and will quantify treatment effectiveness and tolerability. We will also assess relapses, disability progression, MRI activity, and other PROs at approximately 12 and 24 months. The findings will provide insight from daily clinical practice into the patient’s experience to complement data from controlled trials and inform treatment choice. EU PAS Registration Number EUPAS49334 filed 17/10/2022.

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Evaluation of Patient-Reported Outcomes in Patients with Relapsing Multiple Sclerosis Treated with Cladribine Tablets in the CLAWIR Study: 12-Month Interim Analysis

Rau,

Müller,

Übler

2023

Adv Ther

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Introduction Patient-reported outcomes (PROs) provide an insightful method of assessing the subjective impact of therapies for those affected by multiple sclerosis (MS), a chronic neurologic disease notable for symptoms of fatigue and reduced physical function. The ongoing CLAWIR study aims to assess the effect of cladribine tablets (3.5 mg/kg cumulative dose over 2 years) in patients with highly active relapsing MS focusing on PROs of fatigue, physical function, treatment satisfaction, and work productivity. Here, we report on a pre-planned analysis at 12 months after treatment initiation with cladribine tablets. Methods CLAWIR is a 2-year, multicenter, prospective, observational study of patients with relapsing MS newly initiating cladribine tablets. The following PROs were analyzed: PRO Measurement Information System (PROMIS ® ) Fatigue MS (v1.0) and Physical Function MS (v2.1), Treatment Satisfaction Questionnaire for Medication (TSQM, v1.4), and Work Productivity and Activity Impairment MS Questionnaire (WPAI-MS). Data were analyzed descriptively. Results In total, 128 patients were eligible for analysis: 95 females (74.2%); median (range) age 34.5 (29, 44) years; 34 patients (26.6%) were treatment-naïve, and 89 (69.5%) were early switchers from platform therapies (the remaining 5 patients [3.9%] switched from a high-efficacy disease-modifying therapy). PROMIS ® Fatigue MS mean (± standard deviation [SD]) T-scores decreased from 54.6 (± 9.59) at baseline to 51.8 (± 10.30) at 12 months, indicating an alleviation of fatigue, whereas PROMIS ® Physical Function MS mean T-scores remained stable over time [baseline: 49.4 (± 10.69); 12 months: 50.3 (± 10.88)]. TSQM v1.4 mean scores indicated an improvement over time, increasing from 52.2 (± 27.79) at baseline to 81.4 (± 17.06) at 12 months for global satisfaction. WPAI-MS scores also showed an improvement across all four domains over 12 months. Conclusion This real-world study demonstrates the effect of cladribine tablets over 12 months on PROs of fatigue, physical function, treatment satisfaction, and work productivity. Trial Registration The CLAWIR study is registered at the German Federal Institute for Drugs and Medical Devices with the internal NIS number 7469.

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The validity, responsiveness, and score interpretation of the PROMISnq Physical Function – Multiple Sclerosis 15a short form in multiple sclerosis

Cited by 3 publications

References 51 publications

Understanding the Symptoms and Impacts Experienced by People with Relapsing–Remitting MS: A Qualitative Investigation Using Semi-Structured Interviews

Understanding the Symptoms and Impacts Experienced by People with Relapsing–Remitting MS: A Qualitative Investigation Using Semi-Structured Interviews

Prospective observational study to evaluate treatment satisfaction and effectiveness in patients with relapsing multiple sclerosis starting cladribine tablets (CLADREAL) in Italy

Evaluation of Patient-Reported Outcomes in Patients with Relapsing Multiple Sclerosis Treated with Cladribine Tablets in the CLAWIR Study: 12-Month Interim Analysis

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