Merck KGaA
Global Evidence & Value Development

Author Of 1 Presentation

Patient-Reported Outcomes and Quality of Life Poster Presentation

P1062 - The validity and applicability of a new PROMIS® physical function short form for use in relapsing and progressive multiple sclerosis (ID 1720)

Speakers
Presentation Number
P1062
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

There is need to respond to the challenges of developing a robust measurement technique for self-reporting of the level of physical functioning by patients with multiple sclerosis (MS) to help with identifying strategies for improving such outcome. The emergence of the NIH PROMIS item banks has opened new possibilities for developing instruments that are brief, optimally targeted and, potentially, have high precision. This holds promise for addressing unmet measurement needs in MS populations e.g. subtle physical disability changes.

Objectives

To describe the development, validity and applicability of a multiple sclerosis (MS)-specific PROMIS short form for use in relapsing and progressive MS types, the PROMIS SF v2.1 – Physical Function (MS) 15a.

Methods

A mixed-methods sequential design was followed in this research. Step (1) Concept elicitation (CE) interviews were carried out with MS patients (n=14). Step (2) results from the interviews were mapped to the PROMIS physical function item bank, to identify items relevant for MS patients. Subsequently, neurologists (n=6) rated the relevance of the item pool. Step (3) cognitive debriefing (CD) interviews were performed with MS patients to confirm the comprehensiveness, relevance and language clarity of the draft short form (n=48). Step (4) Further item reduction and psychometric evaluation was performed in two observational studies [cross-sectional study at two MS tertiary centers, n=296 (US); 96-week longitudinal study in UK MS Register cohort, n=558 (UK)].

Results

The initial item shortlist (48 items) from the NIH PROMIS item bank was revised in sequential steps, considering 1) optimization of coverage of the underlying concept, 2) results from CD interviews and 3) results from psychometric item-level analysis. Fifteen items were retained in the final short form.

Cronbach’s alpha (> 0.9) and ICC of test-retest scores (5 to 27 days) (> 0.9) indicated the short form’s strong reliability. Convergence validity was demonstrated by moderate-to-strong correlations with related PRO measures as well the EDSS (rho = ± 0.5 to 0.9). The short form discriminated between groups of patients according to levels of physical health and other criteria. A score banding system referencing responses on the individual items as anchors, was generated, to facilitate meaningful score interpretation.

Conclusions

The PROMIS PF (MS) 15a is a reliable and valid short form for assessing physical function with self-report in people living with MS. The inclusion of input from MS patients and neurologists during its qualitative phase of development, ensured comprehensiveness and relevance for both relapsing and progressive MS types.

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Presenter Of 2 Presentations

Patient-Reported Outcomes and Quality of Life Poster Presentation

P1061 - The interpretation and clinical application of the PROMIS® SF v1.0 - Fatigue (MS) 8b: a PROMIS short form for assessing fatigue in multiple sclerosis (ID 1727)

Speakers
Presentation Number
P1061
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

Fatigue is a very common and disabling symptom of multiple sclerosis (MS) that is challenging to characterize appropriately for both research and clinical practice. The emergence of the NIH PROMIS item banks provides new possibilities for the development of outcome measures that are brief and optimally targeted. Substantial evidence has accumulated regarding the use of the PROMIS SF 1.0 – Fatigue (MS) 8b short form to discriminate levels of fatigue among individuals who have MS. The short form was developed with input from MS patients and clinicians.

Objectives

To establish minimal important difference (MID) estimates and interpretation tools for the PROMIS Fatigue (MS) 8b in MS populations.

Methods

Two observational studies were performed in MS populations, a cross-sectional study at two tertiary MS centers in the US (n=296) [US sample] and a longitudinal study in the UK MS Register cohort (n=384) [UK sample]. The analysis sample included patients with relapsing- or progressive MS, and those with Patient-Reported Web EDSS <7. Minimal important difference (MID) of PROMIS Fatigue (MS) 8b T-score was analyzed based on score changes over a 52-week follow-up using an anchor-based approach [UK sample]. An interpretative guide for PROMIS scores was also developed [US sample].

Results

At baseline, study participants had a mean age of 44.5 – 49.9 years, and mean PROMIS Fatigue (MS) 8b T-score of 57.4 – 59.9. Three anchors met criteria and were used in the MID analysis [ i.e. PROMIS GHS fatigue question, GHS PHC global question, and the Fatigue Severity Scale]. The standard error of measurement [SD * √ (1 – reliability)] of baseline T-scores was 2.8. A score change of 3.4 – 4.0 points is proposed as MID criteria for minimal improvement or worsening in fatigue. A heatmap facilitating interpretation of scores based on fatigue concerns on individual items was developed. For example, a T-score of 60 represents a fatigue level characterized by (often) getting tired easily, (sometimes) being too tired to think clearly, and (some-) interference with physical functioning, in the last 7 days.

Conclusions

This research extends the evidence underpinning the applicability of the PROMIS Fatigue (MS) 8b in routine clinical practice and clinical research. The score interpretation guide may aid the integration of PROMIS scores into clinical decision-making as well as facilitate clinician-patient communication. MID estimates will be useful in evaluating fatigue over time.

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Patient-Reported Outcomes and Quality of Life Poster Presentation

P1062 - The validity and applicability of a new PROMIS® physical function short form for use in relapsing and progressive multiple sclerosis (ID 1720)

Speakers
Presentation Number
P1062
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

There is need to respond to the challenges of developing a robust measurement technique for self-reporting of the level of physical functioning by patients with multiple sclerosis (MS) to help with identifying strategies for improving such outcome. The emergence of the NIH PROMIS item banks has opened new possibilities for developing instruments that are brief, optimally targeted and, potentially, have high precision. This holds promise for addressing unmet measurement needs in MS populations e.g. subtle physical disability changes.

Objectives

To describe the development, validity and applicability of a multiple sclerosis (MS)-specific PROMIS short form for use in relapsing and progressive MS types, the PROMIS SF v2.1 – Physical Function (MS) 15a.

Methods

A mixed-methods sequential design was followed in this research. Step (1) Concept elicitation (CE) interviews were carried out with MS patients (n=14). Step (2) results from the interviews were mapped to the PROMIS physical function item bank, to identify items relevant for MS patients. Subsequently, neurologists (n=6) rated the relevance of the item pool. Step (3) cognitive debriefing (CD) interviews were performed with MS patients to confirm the comprehensiveness, relevance and language clarity of the draft short form (n=48). Step (4) Further item reduction and psychometric evaluation was performed in two observational studies [cross-sectional study at two MS tertiary centers, n=296 (US); 96-week longitudinal study in UK MS Register cohort, n=558 (UK)].

Results

The initial item shortlist (48 items) from the NIH PROMIS item bank was revised in sequential steps, considering 1) optimization of coverage of the underlying concept, 2) results from CD interviews and 3) results from psychometric item-level analysis. Fifteen items were retained in the final short form.

Cronbach’s alpha (> 0.9) and ICC of test-retest scores (5 to 27 days) (> 0.9) indicated the short form’s strong reliability. Convergence validity was demonstrated by moderate-to-strong correlations with related PRO measures as well the EDSS (rho = ± 0.5 to 0.9). The short form discriminated between groups of patients according to levels of physical health and other criteria. A score banding system referencing responses on the individual items as anchors, was generated, to facilitate meaningful score interpretation.

Conclusions

The PROMIS PF (MS) 15a is a reliable and valid short form for assessing physical function with self-report in people living with MS. The inclusion of input from MS patients and neurologists during its qualitative phase of development, ensured comprehensiveness and relevance for both relapsing and progressive MS types.

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