Qualitative Research with Patients and Physicians to Assess Content Validity and Meaningful Change on ESSDAI and ESSPRI in Sjögren’s

Cooper, Carl; Wratten, Samantha; Williams-Hall, Rebecca; Bookman, Arthur; Ndife, Briana; Hueber, Wolfgang; Goswami, Pushpendra

doi:10.1007/s40744-022-00487-0

Cited by 3 publications

(9 citation statements)

References 34 publications

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“…In accordance with methodological guidelines, 34 42 46 this study incorporated the results of both anchor-based and distribution-based methods to propose a revised RD for ESSPRI: completing a trial with an ESSPRI score of ≤3, with an improvement in ESSPRI of ≥1.5 points compared with baseline. This revised RD not only increases the MCII threshold (ie, ≥1.5 points-previously ≥1 point or 15% improvement) and is supported by qualitative data from an interview study, 44 but also incorporates a low/ minimal symptom severity threshold that at ≤3 points was lower than the previously identified PASS of <5. Estimates for meaningful improvement ranged between 0.73 and 4.47 (including both 'minimal' and 'moderatemajor' improvement) and estimates for a cut-off point for 'low/minimal symptom severity' ranged between 3.0 and 4.3.…”

Section: Discussionmentioning

confidence: 69%

“…This was further supported by evidence from an interview study with individuals with Sjögren’s whereby a 1-point or 2-point improvement on ESSPRI was considered meaningful. 44 …”

Section: Discussionmentioning

confidence: 99%

“…This was further supported by evidence from an interview study with individuals with Sjögren's whereby a 1-point or 2-point improvement on ESSPRI was considered meaningful. 44 In an effort to develop a 'treat to target endpoint' (ie, a patient-relevant definition of a disease state that would improve their feeling and functioning), it was agreed that a cut-off point of ≤3 for 'low/minimal symptom severity' should be included in the revised RD. This was a fundamentally different goal than the original PASS, which was developed as a diagnostic criterion rather than to assess change, and the original PASS was not used as a RMD Open RMD Open RMD Open comparator during analyses.…”

Section: Discussionmentioning

confidence: 99%

“…As recommended by the FDA, 31 a separate interview study with individuals with Sjögren’s was also conducted in parallel to this quantitative study to help refine and contextualise the revised RD. 44 …”

Section: Methodsmentioning

confidence: 99%

“…However, data from the aforementioned interview study with individuals with Sjögren’s suggest that meaningful change thresholds are similar in both directions as most individuals thought a 1-point or 2-point worsening as well as improvement would be meaningful on ESSPRI. 44 …”

Section: Limitationsmentioning

confidence: 99%

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Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI)

et al. 2023

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ObjectivesDryness, fatigue and joint/muscle pain are typically assessed in Sjögren’s trials using European Alliance of Associations for Rheumatology Sjögren’s Syndrome Patient Reported Index (ESSPRI). A Patient Acceptable Symptom State of <5 and a Minimal Clinically Important Improvement (MCII)/responder definition (RD) of ≥1 point or 15% on ESSPRI have previously been defined. This study explored alternative RDs to better discriminate between active treatment and placebo in trials.MethodsAnchor-based and distribution-based methods were used to derive RD thresholds in blinded phase IIb trial data (N=190) and confirm these in blinded data pooled from three early phase II trials (N=126). The populations consisted of individuals with moderate-to-severe systemic primary Sjögren’s. Anchors were prioritised by ESSPRI correlations and used in similar conditions. Triangulated estimates were discussed with experts (N=3). The revised RD was compared with the original using unblinded data to assess placebo and treatment responder rates.ResultsPatients were predominantly female (>90%), white (90%), with mean age of 50 years. Receiver operating characteristic estimates supported an MCII threshold of 1.5–1.6 in the phase II data, whereas correlation-weighted mean change estimates supported a low/minimal symptom severity threshold of ≥2. A low/minimal symptom severity of ≤3 showed the greatest sensitivity/specificity balance. Analyses in the pooled data supported these thresholds (MCII: 1.5–2.1; low/minimal symptom severity: 2.7–3.7). Unblinded analyses confirmed the revised RD reduced placebo rates.ConclusionsCompleting a trial with an improvement of ≥1.5 points compared with baseline and an ESSPRI score of ≤3 points is a relevant RD for moderate-to-severe systemic Sjögren’s and reduces placebo rates.

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Section: Discussionmentioning

confidence: 69%

“…This was further supported by evidence from an interview study with individuals with Sjögren’s whereby a 1-point or 2-point improvement on ESSPRI was considered meaningful. 44 …”

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Limitationsmentioning

confidence: 99%

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Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI)

et al. 2023

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Effect of adipose tissue-derived stem cells therapy on clinical response in patients with primary Sjogren’s syndrome

Li,

Lu,

Shi

et al. 2023

Sci Rep

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The purpose of this trial was to clinically assess the effect and safety of Adipose Tissue-derived Stem Cells (ADSCs) treatment on primary Sjogren’s Syndrome (pSS). In this 6-month randomized, triple-blind, placebo-controlled clinical trial, pSS patients were randomly assigned to two groups. After demographic characteristics and clinical examination were achieved, local injection of ADSCs into bilateral glands was performed with patients in ADSCs group (n = 35) and placebo solution was used for another group (n = 39) at three time points. Patients were followed up at 1-, 3- and 6-month. At each visit, studies of clinical and laboratory outcomes, as well as subjective symptoms, were conducted. A total of 74 subjects who met the including criteria were allocated in two groups and eventually 64 subjects (86.5%) completed the treatments and the follow-up assessments. Secretion of salivary and lachrymal glands were significantly improved in 3-month (P < 0.05). A great improvement of European League Against Rheumatism Sjögren’s Syndrome Disease Activity Index (ESSDAI) was found after ADSCs treatment with intergroup comparison from baseline to follow-up (P < 0.05). There is also a significant difference of European Alliance of Associations for Rheumatology SS Patient Reported Index (ESSPRI) between the two groups in the follow-up (P < 0.05). A significant abatement of IgG, IgM, C3, C4 and ESR between two groups was observed in part of follow-up time points (P < 0.05). The ADSCs therapy can provide relief of oral and eye’s dryness in our trial in a short time and has potential improvement of subjective and systemic syndromes of pSS.

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Outcome Measures for Sjögren Disease—Novel Developments and Further Needs

Wilson,

Townsend,

Hawkins

et al. 2024

Br J Hosp Med

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This case report follows the events of a 36-year-old woman who presented to a hospital five days post-partum with an acute severe headache and vomiting. Despite a normal initial computed tomography (CT) head scan, a CT venogram was done due to neurological deterioration and revealed hydrocephalus secondary to subarachnoid haemorrhage (SAH). We discuss the role of CT imaging in the diagnosis of SAH, the risks of current guidelines for lumbar puncture (LP) and describe other important differential diagnoses for headache in the postpartum patient.

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Qualitative Research with Patients and Physicians to Assess Content Validity and Meaningful Change on ESSDAI and ESSPRI in Sjögren’s

Cited by 3 publications

References 34 publications

Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI)

Development and testing of an alternative responder definition for EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI)

Effect of adipose tissue-derived stem cells therapy on clinical response in patients with primary Sjogren’s syndrome

Outcome Measures for Sjögren Disease—Novel Developments and Further Needs

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