Patient Reported Outcome
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2021 ◽  
pp. 036354652110538
David A. Bloom ◽  
Daniel J. Kaplan ◽  
Edward Mojica ◽  
Eric J. Strauss ◽  
Guillem Gonzalez-Lomas ◽  

Background: The minimal clinically important difference (MCID) is a term synonymous with orthopaedic clinical research over the past decade. The term represents the smallest change in a patient-reported outcome measure that is of genuine clinical value to patients. It has been derived in a myriad of ways in existing orthopaedic literature. Purpose: To describe the various modalities for deriving the MCID. Study Design: Narrative review; Level of evidence, 4. Methods: The definitions of common MCID determinations were first identified. These were then evaluated by their clinical and statistical merits and limitations. Results: There are 3 primary ways for determining the MCID: anchor-based analysis, distribution-based analysis, and sensitivity- and specificity-based analysis. Each has unique strengths and weaknesses with respect to its ability to evaluate the patient’s clinical status change from baseline to posttreatment. Anchor-based analyses are inherently tied to clinical status yet lack standardization. Distribution-based analyses are the opposite, with strong foundations in statistics, yet they fail to adequately address the clinical status change. Sensitivity and specificity analyses offer a compromise of the other methodologies but still rely on a somewhat arbitrarily defined global transition question. Conclusion: This current concepts review demonstrates the need for (1) better standardization in the establishment of MCIDs for orthopaedic patient-reported outcome measures and (2) better study design—namely, until a universally accepted MCID derivation exists, studies attempting to derive the MCID should utilize the anchor-based within-cohort design based on Food and Drug Administration recommendations. Ideally, large studies reporting the MCID as an outcome will also derive the value for their populations. It is important to consider that there may be reasonable replacements for current derivations of the MCID. As such, future research should consider an alternative threshold score with a more universal method of derivation.

2021 ◽  
Jenna A. Chiang ◽  
Paulina T. Feghali ◽  
Anita Saavedra ◽  
Ashley M. Whitaker

Abstract Purpose While the effects of sleep on cognition in typically developing children are well established, there is a paucity of research in patients with pediatric brain tumor (PBT), despite their increased risk for sleep-related disturbances. The aim of this study was to examine the impact of sleep factors on patient-reported outcome (PRO) measures, including adaptive and executive functioning within this population.Methods 133 patients with PBT (52% male) ages 5-23 (x̄ = 12.8yrs; SD = 4.5yrs) underwent neuropsychological evaluation, including assessment of adaptive and executive functioning. Subjective sleep concerns, nocturnal sleep duration, and daytime sleep behavior were also collected and compared to age-based guidelines.Results Nearly 30% of patients reported subjective sleep concerns, while the sample as a whole presented with reduced nocturnal sleep duration (approximately one hour below age-based recommendations). Despite the expectation for monophasic sleep by age five, nearly half of the sample reported consistent daytime napping. With regard to functional outcomes, inadequate sleep predicted decreased adaptive functioning, F(1, 56) = 4.23, p < .05 (R2 = .07), as well as increased symptoms of executive dysfunction, F(1, 108) = 3.51, p < .05 (R2 = .03).Conclusion Patients with PBT demonstrate several aspects of abnormal sleep, which are associated with poorer long-term PROs. Further exploration of diagnostic, treatment-related, and demographic variables will be needed to better understand these relationships among patients with PBT in order to inform appropriate interventions.

2021 ◽  
Vol Publish Ahead of Print ◽  
Robin R. Whitebird ◽  
Leif I. Solberg ◽  
Jeanette Y. Ziegenfuss ◽  
Christine K. Norton ◽  
Ella A. Chrenka ◽  

Rebecca Heijke ◽  
Mathilda Björk ◽  
Ingrid Thyberg ◽  
Alf Kastbom ◽  
Laura McDonald ◽  

Abstract The onset of rheumatic disease affects each patient differently and may impact quality of life with progression. We investigated the relationship between patient-reported outcome measure (PROM) scores and organ damage in patients with recent-onset systemic lupus erythematosus (SLE) and those with early rheumatoid arthritis (RA). Patients with recent-onset SLE without prior organ damage from the Clinical Lupus Register in Northeastern Gothia and patients with early RA from the observational 2nd Timely Interventions in Early RA study, Sweden, were included. Systemic Lupus International Collaborating Clinics/American College of Rheumatology damage index (SDI) was used to assess organ damage. PROM (visual analog scale [VAS]: pain, fatigue, well-being, Health Assessment Questionnaire, and EQ-5D-3L) scores were captured at months 0, 6, 12, 24, 36, 48, and 60 after diagnosis. Statistical tests included Pearson correlation coefficients and t-tests. Forty-one patients with recent-onset SLE and 522 with early RA were included. Numerical differences were seen in age and sex. PROMs were worse for patients with RA versus SLE but improved by month 6 following diagnosis, while SLE PROMs remained stable. The incidence of organ damage in SLE was 13.6 per 100 patient-years. SDI significantly correlated with EQ-5D-3L (− 0.48, P = 0.003), VAS fatigue (0.44, P = 0.009), and well-being (0.41, P = 0.01) at month 24. As illustrated, the complexity of disease burden in patients with SLE is clear and may result from disease-related multiorgan system effects and slower symptom resolution compared with RA. This underscores the need for improved multiprofessional interventions to manage all aspects of SLE. Key Points• We observed an evident discrepancy in patient-reported outcome measures (PROMs) between patients with recent-onset SLE and early RA.• Despite differences in PROMs between patients with recent-onset SLE and early RA, both groups had prominent self-reported disability during the study period.• PROM scores for patients with RA were generally worse than those with SLE but improved by month 6, whereas PROM scores for patients with SLE remained stable over time.• Our findings underline the need of new therapeutic options and interventions for SLE disease management, including pharmacologic and multiprofessional aspects.

2021 ◽  
pp. annrheumdis-2021-220702
Mike O Becker ◽  
Rucsandra Dobrota ◽  
Alexandru Garaiman ◽  
Rudolf Debelak ◽  
Kim Fligelstone ◽  

ObjectivesPatient-reported outcome measures (PROMs) are important for clinical practice and research. Given the high unmet need, our aim was to develop a comprehensive PROM for systemic sclerosis (SSc), jointly with patient experts.MethodsThis European Alliance of Associations for Rheumatology (EULAR)-endorsed project involved 11 European SSc centres. Relevant health dimensions were chosen and prioritised by patients. The resulting Systemic Sclerosis Impact of Disease (ScleroID) questionnaire was subsequently weighted and validated by Outcome Measures in Rheumatology criteria in an observational cohort study, cross-sectionally and longitudinally. As comparators, SSc-Health Assessment Questionnaire (HAQ), EuroQol Five Dimensional (EQ-5D), Short Form-36 (SF-36) were included.ResultsInitially, 17 health dimensions were selected and prioritised. The top 10 health dimensions were selected for the ScleroID questionnaire. Importantly, Raynaud’s phenomenon, impaired hand function, pain and fatigue had the highest patient-reported disease impact. The validation cohort study included 472 patients with a baseline visit, from which 109 had a test–retest reliability visit and 113 had a follow-up visit (85% female, 38% diffuse SSc, mean age 58 years, mean disease duration 9 years). The total ScleroID score showed strong Pearson correlation coefficients with comparators (SSc-HAQ, 0.73; Patient’s global assessment, Visual Analogue Scale 0.77; HAQ-Disability Index, 0.62; SF-36 physical score, −0.62; each p<0.001). The internal consistency was strong: Cronbach’s alpha was 0.87, similar to SSc-HAQ (0.88) and higher than EQ-5D (0.77). The ScleroID had excellent reliability and good sensitivity to change, superior to all comparators (intraclass correlation coefficient 0.84; standardised response mean 0.57).ConclusionsWe have developed and validated the EULAR ScleroID, which is a novel, brief, disease-specific, patient-derived, disease impact PROM, suitable for research and clinical use in SSc.

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