Patient-reported outcome measures for systemic lupus erythematosus clinical trials: a review of content validity, face validity and psychometric performance

Holloway, Laura; Humphrey, L.; Heron, Louise; Pilling, Claire; Kitchen, Helen; Højbjerre, Lise; Strandberg-Larsen, Martin; Hansen, Birgit Sehested

doi:10.1186/s12955-014-0116-1

Cited by 91 publications

(85 citation statements)

References 95 publications

(154 reference statements)

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“…PROMIS measures include domains commonly measured for systemic lupus erythematosus (SLE) studies (eg, pain, fatigue, and physical functioning) as well as domains that are relevant and important to patients with SLE (eg, social functioning and sleep) but are not often measured (1,2). The National Institutes of Health's (NIH's) Patient-Reported Outcomes Measurement Information System (PROMIS) measures represent the most comprehensive suite of patient-reported outcome (PRO) measures available.…”

Section: Introductionmentioning

confidence: 99%

Estimates of Responsiveness, Minimally Important Differences, and Patient Acceptable Symptom State in Five Patient‐Reported Outcomes Measurement Information System Short Forms in Systemic Lupus Erythematosus

Katz

Pedro

Alemao

et al. 2019

ACR Open Rheumatology

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Objective. Examinations of Patient-Reported Outcomes Measurement Information System (PROMIS) measures in adult systemic lupus erythematosus (SLE) have provided support for their cross-sectional validity in SLE. We estimated responsiveness to change, meaningful changes (minimally important differences [MIDs]), and the patient acceptable symptom state (PASS) for five PROMIS short forms to facilitate longitudinal use and interpretation of PROMIS scales in SLE.Methods. Data from five administrations of PROMIS short forms in the FORWARD SLE cohorts were used. Pearson correlation coefficients were used to assess associations between changes in PROMIS measures and changes in anchor measures for responsiveness analyses. Worse, same, or better groups were defined for each anchor. Differences in PROMIS scores were calculated for each consecutive PROMIS administration; mean changes in PROMIS scores of individuals in the worse, same, and better groups were calculated. Both anchor-based and distribution-based methods were used to estimate MIDs. PASS was defined as the 75th-percentile positive score among those who considered their health to be acceptable or who were somewhat or very satisfied with their health.Results. All PROMIS short forms showed adequate responsiveness to changes in related patient-reported outcomes. However, only the fatigue and pain interference scales were responsive to self-reported SLE activity. Taking into account all methods, we estimated MIDs for each scale to be approximately two points. All PASS values were better than the population mean T-score of 50.Conclusion. These results support use and further study of PROMIS short forms in SLE and should facilitate interpretation of PROMIS scores and changes.Funded by Bristol-Myers Squibb.

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Section: Introductionmentioning

confidence: 99%

Estimates of Responsiveness, Minimally Important Differences, and Patient Acceptable Symptom State in Five Patient‐Reported Outcomes Measurement Information System Short Forms in Systemic Lupus Erythematosus

Katz

Pedro

Alemao

et al. 2019

ACR Open Rheumatology

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“…Although diagnostic and therapeutic advances have improved 5-year to 10-year survival rates to more than 90%,2 3 there remain significant unmet needs in the management of SLE, particularly among patients with persistent, treatment-refractory disease 4. Furthermore, current treatments have limitations 5…”

Section: Introductionmentioning

confidence: 99%

Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study

et al. 2016

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ObjectiveTo evaluate the efficacy of a prolonged-release formulation of a porcine adrenocorticotropic hormone analogue (repository corticotropin injection (RCI)) added to standard of care in patients requiring moderate-dose corticosteroids for symptomatic SLE.MethodsThis prospective, randomised, double-blind, phase 4, pilot study (NCT01753401) enrolled 38 patients with persistently active SLE involving skin and/or joints. Enrolled patients received RCI, 40 U daily or 80 U every other day, or volume-matched placebo gel, for 8 weeks, with dose tapering to twice weekly during weeks 5–8. Efficacy endpoints included proportion of responders at week 4 based on a novel composite measure that included resolution of rash or arthritis measured using the hybrid SLE Disease Activity Index (hSLEDAI) without worsening British Isles Lupus Assessment Group (BILAG) scores in other organ systems at week 4 (primary), as well as improvements in total hSLEDAI and BILAG scores and other measures of skin and joint disease activity over the 8-week treatment period.ResultsResponse, as defined for the primary endpoint, did not differ significantly between the combined placebo and RCI-treated groups at week 4. At week 8, the proportion of responders was higher in RCI-treated patients but did not statistically differ between groups (RCI 40 U (53.8%), RCI 80 U (33.3%), combined placebo (27.3%)). However, RCI treatment was associated with statistically significant improvements in several secondary endpoints, including total hSLEDAI, total BILAG and Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity scores within 8 weeks. Treatment was well tolerated.ConclusionsAlthough the primary endpoint was not met in this pilot study, secondary and post hoc analyses suggested that RCI was associated with improvements in SLE disease activity in a select patient population with steroid-dependent persistent disease.Trial registration numberNCT01753401; results.

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“…There is a high level of patient acceptance with autoinjector devices in RA, including patients with severe hand disability [11]. Musculoskeletal and mucocutaneous manifestations, such as fatigue, joint pain, and swelling, are common in patients with active SLE, particularly in the fingers, hands, and wrists, and can impact the patient’s ability to carry out day-to-day tasks [12, 13, 14]. As patients with SLE have similar therapeutic needs to patients with RA, autoinjector usability should be studied in these patients.…”

Section: Introductionmentioning

confidence: 99%

Evaluation of a novel autoinjector for subcutaneous self-administration of belimumab in systemic lupus erythematosus

Sheikh¹,

Hammer²,

Fox³

et al. 2016

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Objective: To study self-administration and pharmacokinetics (PK) of subcutaneous (SC) belimumab in patients with systemic lupus erythematosus (SLE). Methods: Patients previously treated with belimumab self-administered belimumab 200 mg SC weekly for 8 weeks using an autoinjector. The primary endpoint was the proportion of patients able to self-administer their first and second dose (weeks 1 and 2) in the clinic. The proportion able to self-administer at weeks 4 and 8 (clinic) and weeks 3, 5, 6, and 7 (home) were secondary endpoints. Belimumab PK, safety, and injection-site pain were assessed. Results: 91/95 patients completed the study (withdrawals: adverse events (AEs): 3; lost to follow-up: 1). 93% were female, and mean (SD) age was 44.8 (12.50) years. The majority (99%, 89/90; no attempt, n = 5) successfully self-administered belimumab SC at weeks 1 and 2 (5 had clinic staff assistance), and 98% (85/87) successfully self-administered at weeks 4 and 8. Home-administration success rates were high (93%, (81/87) at weeks 3, 5, 6, and 7). Week 8 median trough concentration was 113 µg/mL. For patients with a ≤ 1.5-week interval between IV SC administration, week-1 concentrations were higher vs. week 8 (+ 51% median) but within a range observed with IV dosing; those with a ≥ 2.5-week interval had median differences close to 0. AEs and serious AEs were low, with no deaths; pain levels were low and decreased with subsequent injections. Conclusion: Patients with SLE successfully self-administered belimumab SC using a novel autoinjector; the PK profile was stable following a switch from IV with acceptable AE and pain levels. The recommended dosing interval between IV to SC dosing is 1 – 4 weeks.

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Patient-reported outcome measures for systemic lupus erythematosus clinical trials: a review of content validity, face validity and psychometric performance

Cited by 91 publications

References 95 publications

Estimates of Responsiveness, Minimally Important Differences, and Patient Acceptable Symptom State in Five Patient‐Reported Outcomes Measurement Information System Short Forms in Systemic Lupus Erythematosus

Estimates of Responsiveness, Minimally Important Differences, and Patient Acceptable Symptom State in Five Patient‐Reported Outcomes Measurement Information System Short Forms in Systemic Lupus Erythematosus

Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study

Evaluation of a novel autoinjector for subcutaneous self-administration of belimumab in systemic lupus erythematosus

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