Psychometric properties of the itch numeric rating scale, skin pain numeric rating scale, and atopic dermatitis sleep scale in adult patients with moderate-to-severe atopic dermatitis

Jonathan I Silverberg, Amy DeLozier, Luna Sun, Jacob P Thyssen, Brian Kim, Gil Yosipovitch, Fabio P Nunes, P Cristian Gugiu, Helen A Doll, Lawrence F Eichenfield, Jonathan I Silverberg, Amy DeLozier, Luna Sun, Jacob P Thyssen, Brian Kim, Gil Yosipovitch, Fabio P Nunes, P Cristian Gugiu, Helen A Doll, Lawrence F Eichenfield

Abstract

Background: The Itch Numeric Rating Scale (NRS), Skin Pain NRS, and Atopic Dermatitis Sleep Scale (ADSS) are self-administered patient-reported outcome (PRO) instruments developed to assess symptoms in patients with atopic dermatitis (AD). The objective of this study was to evaluate the psychometric properties (reliability, validity, and responsiveness) and interpretability thresholds of these PROs using data from three pivotal Phase 3 studies in adults.

Methods: BREEZE-AD1, BREEZE-AD2, and BREEZE-AD5 evaluated the safety and efficacy of baricitinib in adults with moderate-to-severe AD. Clinician-reported outcomes and other PROs commonly assessed in patients with AD were used to estimate meaningful changes and evaluate test-retest reliability, convergent and divergent validity, known-groups validity, responsiveness, and meaningful change thresholds (MCTs) of the Itch NRS, Skin Pain NRS, and ADSS.

Results: The test-retest reliability of the Itch NRS, Skin Pain NRS, and ADSS was evidenced by generally large intraclass correlation coefficients (> 0.7) in stable groups of patients between baseline and Week 1 and Weeks 4 and 8. Moderate-to-large correlations (r > 0.4) at baseline and Week 16 were generally observed between each measure and other PROs measuring the same concept, supporting convergent validity. Small-to-moderate correlations with clinician-reported outcomes demonstrated divergent validity. Each instrument was able to distinguish between known groups of disease severity as assessed using other indicators of AD severity. The responsiveness of the Itch NRS, Skin Pain NRS, and ADSS scales was demonstrated through significant differences in their change scores from baseline to Week 16 between categories of change in another PRO also from baseline to Week 16. Thresholds for interpreting meaningful change were estimated as - 4.0 for the 0-10 Itch and Skin Pain NRS items; - 1.25 for the 0-4 ADSS Items 1 and 3 and; - 1.50 for the 0-29 ADSS Item 2, these equivalent to moderate degrees of change.

Conclusions: Results of this study demonstrate that the psychometric properties of the Itch NRS, Skin Pain NRS, and ADSS are good to excellent. These findings support the use of these instruments in daily assessment of AD symptoms in adults with moderate-to-severe AD. Trial registration ClinicalTrials.gov numbers: NCT03334396, NCT03334422, and NCT03435081.

Keywords: Atopic dermatitis; Atopic dermatitis sleep scale; Convergent-divergent validity; Itch NRS; Numeric rating scale; Patient-reported outcome; Psychometric; Reliability; Responsiveness; Skin pain NRS; Validity.

Conflict of interest statement

JIS has received honoraria as a consultant and/or advisory board member for Abbvie, Afyx, Arena, Asana, Bluefin, Boehringer-Ingelheim, Celgene, Dermavant, Dermira, Eli Lilly, Galderma, GlaxoSmithKline, Incyte, Kiniksa, Leo, Luna, Menlo, Novartis, Pfizer, RAPT, Regeneron, Sanofi; speaker for Regeneron, Sanofi; institution received grants from Galderma. AD, LS and FN are employees of Eli Lilly and Company and may hold stock and/or stock options in the company. JPT reports personal fees from Pfizer, personal fees from Eli Lilly & Co, personal fees from Abbvie, personal fees from LEO Pharma, grants and personal fees from Regeneron, grants and personal fees from Sanofi-Genzyme, outside the submitted work. GY has been on advisory boards for and received honoraria from Sanofi and Regeneron Pharmaceuticals, Inc. TREVI, Pfizer, Novartis, Eli Lilly, Kiniksa, LEO, Galderma, Kiniksa, GSK, and his research has been funded by Pfizer, Galderma, Novartis, LEO, Kinksa, Sanofi Regeneron and Sun Pharma. LFE has received honoroaria for his work as a consultant for Abbvie, Dermavant, Dermira, Leo, Eli Lilly, Novartis, Regeneron, Sanofi-Genzyme and Ortho Dermatology, been an investigator/received grants for Abbvie, Galderma Laboratories, Ortho Dermatology and Pfizer. BK reports personal fees from AbbVie, personal fees from Almirall, personal fees from Boehringer Ingelheim, grants and personal fees from Cara Therapeutics, personal fees from AstraZeneca, personal fees from Menlo Therapeutics, personal fees from Regeneron, personal fees from Sanofi Genzyme, grants and personal fees from LEO Pharma, personal fees from Trevi Therapeutics, personal fees from Daewoong, personal fees from OM Pharma, personal fees from Incyte, personal fees from Amagma, personal fees from Maruho, outside the submitted work; In addition, BK has a patent on JAK inhibitors for chronic itch pending to None. PCG and HAD report no conflict of interest. Fabio P. Nunes was an employee of Eli Lilly and Company, Indianapolis, Indiana, USA at the time of conducting this study. Currently he is an employee of Janssen Pharmaceutical Companies of Johnson & Johnson, Raritan, New Jersey, USA.

© 2021. The Author(s).

References

    1. Lifschitz C. The impact of atopic dermatitis on quality of life. Ann Nutr Metab. 2015;66(Suppl 1):34–40. doi: 10.1159/000370226.
    1. Vakharia PP, Chopra R, Sacotte R, Patel KR, Singam V, Patel N, et al. Burden of skin pain in atopic dermatitis. Ann Allergy Asthma Immunol. 2017;119(6):548–552. doi: 10.1016/j.anai.2017.09.076.
    1. Hanifin JM, Thurston M, Omoto M, Cherill R, Tofte SJ, Graeber M. The eczema area and severity index (EASI): assessment of reliability in atopic dermatitis. EASI Evaluator Group Exp Dermatol. 2001;10(1):11–18. doi: 10.1034/j.1600-0625.2001.100102.x.
    1. Futamura M, Leshem YA, Thomas KS, Nankervis H, Williams HC, Simpson EL. A systematic review of Investigator Global Assessment (IGA) in atopic dermatitis (AD) trials: Many options, no standards. J Am Acad Dermatol. 2016;74(2):288–294. doi: 10.1016/j.jaad.2015.09.062.
    1. Simpson E, Bissonnette R, Eichenfield LF, Guttman-Yassky E, King B, Silverberg JI, et al. The Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD): The development and reliability testing of a novel clinical outcome measurement instrument for the severity of atopic dermatitis. J Am Acad Dermatol. 2020;83(3):839–846. doi: 10.1016/j.jaad.2020.04.104.
    1. US-FDA. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims 2009 [updated December 2009. .
    1. Newton L, DeLozier AM, Griffiths PC, Hill JN, Hudgens S, Symonds T, et al. Exploring content and psychometric validity of newly developed assessment tools for itch and skin pain in atopic dermatitis. J Patient Rep Outcomes. 2019;3(1):42. doi: 10.1186/s41687-019-0128-z.
    1. Simpson EL, Lacour JP, Spelman L, Galimberti R, Eichenfield LF, Bissonnette R, et al. Baricitinib in patients with moderate-to-severe atopic dermatitis and inadequate response to topical corticosteroids: results from two randomized monotherapy phase III trials. Br J Dermatol. 2020;183(2):242–255. doi: 10.1111/bjd.18898.
    1. Simpson EFS, Silverberg J, Zirwas E, Han G, Guttman-Yassky E, Marnell D, Bissonnette R, Waibel J, Nunes F, DeLozier A, Angle R, Holzwarth K, Goldblum O, Zhong J, Papp K. Efficacy and safety of baricitinib in moderate-to-severe atopic dermatitis: Results from a randomized, double-blinded, placebo-controlled phase 3 clinical trial (BREEZE-AD5). Revolutionizing Atopic Dermatitis, 5 April 2020. Br J Dermatol. 2020;183(4):e94–e121.
    1. Finlay AY, Khan GK. Dermatology Life Quality Index (DLQI)—a simple practical measure for routine clinical use. Clin Exp Dermatol. 1994;19(3):210–216. doi: 10.1111/j.1365-2230.1994.tb01167.x.
    1. Charman CR, Venn AJ, Ravenscroft JC, Williams HC. Translating Patient-Oriented Eczema Measure (POEM) scores into clinical practice by suggesting severity strata derived using anchor-based methods. Br J Dermatol. 2013;169(6):1326–1332. doi: 10.1111/bjd.12590.
    1. Outcomes and Psychometric Summit. Clinical Outcomes Solutions, C-Path PRO Consortium partner led Meeting Tucson, Arizona 2015.
    1. Litwin M. How to measure survey reliability and validity. 7. Thousand Oaks: Sage Publications; 1995.
    1. Nunnally J. The assessment of reliability. In: Bernstein I, editor. Psychometric theory. New York: McGraw Hill; 1994. pp. 248–292.
    1. Vaz S, Falkmer T, Passmore AE, Parsons R, Andreou P. The case for using the repeatability coefficient when calculating test-retest reliability. PLoS ONE. 2013;8(9):e73990. doi: 10.1371/journal.pone.0073990.
    1. Cohen J. Statistical power analysis for the behavioral sciences. 2. New York: Lawrence Erlbaum Associates; 1988.
    1. Cronbach LJ. Coefficient alpha and the internal structure of tests. Psychometrika. 1951;16(3):297–334. doi: 10.1007/BF02310555.
    1. Coon CD, Cook KF. Moving from significance to real-world meaning: methods for interpreting change in clinical outcome assessment scores. Qual Life Res. 2018;27(1):33–40. doi: 10.1007/s11136-017-1616-3.
    1. Revicki D, Hays RD, Cella D, Sloan J. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008;61(2):102–109. doi: 10.1016/j.jclinepi.2007.03.012.
    1. Kazis LE, Anderson JJ, Meenan RF. Effect sizes for interpreting changes in health status. Med Care. 1989;27(3 Suppl):S178–S189. doi: 10.1097/00005650-198903001-00015.
    1. Heeren T, D'Agostino R. Robustness of the two independent samples t-test when applied to ordinal scaled data. Stat Med. 1987;6(1):79–90. doi: 10.1002/sim.4780060110.
    1. McLeod LD, Coon CD, Martin SA, Fehnel SE, Hays RD. Interpreting patient-reported outcome results: US FDA guidance and emerging methods. Expert Rev Pharmacoecon Outcomes Res. 2011;11(2):163–169. doi: 10.1586/erp.11.12.
    1. Kimball AB, Naegeli AN, Edson-Heredia E, Lin CY, Gaich C, Nikai E, et al. Psychometric properties of the Itch Numeric Rating Scale in patients with moderate-to-severe plaque psoriasis. Br J Dermatol. 2016;175(1):157–162. doi: 10.1111/bjd.14464.
    1. Yosipovitch G, Reaney M, Mastey V, Eckert L, Abbe A, Nelson L, et al. Peak Pruritus Numerical Rating Scale: psychometric validation and responder definition for assessing itch in moderate-to-severe atopic dermatitis. Br J Dermatol. 2019;181(4):761–769. doi: 10.1111/bjd.17744.
    1. Silverberg JI. Associations between atopic dermatitis and other disorders. F1000Res. 2018;7:303. doi: 10.12688/f1000research.12975.1.
    1. Kunz B, Oranje AP, Labrèze L, Stalder JF, Ring J, Taïeb A. Clinical Validation and Guidelines for the SCORAD Index: Consensus Report of the European Task Force on Atopic Dermatitis. Dermatology. 1997;195(1):10–19. doi: 10.1159/000245677.
    1. Silverberg JI, Lai JS, Patel KR, Singam V, Vakharia PP, Chopra R, et al. Measurement properties of the Patient-Reported Outcomes Information System (PROMIS((R))) Itch Questionnaire: itch severity assessments in adults with atopic dermatitis. Br J Dermatol. 2020;183:891–898. doi: 10.1111/bjd.18978.

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