Treatment management of patients with systemic lupus erythematosus: a delphi consensus analysis
DOI:
https://doi.org/10.33393/grhta.2022.2470Keywords:
Anifrolumab, Consensus, Delphi, Efficacy, Likert scale, Systemic lupus erythematosusAbstract
Background: Systemic lupus erythematosus (SLE) is associated with clinical burden for the patient and organ damage. The development of therapies for SLE has been constrained by clinical and biologic heterogeneity. These represent challenges in clinical trial design and endpoint selection.
Objective: To identify the most relevant descriptors for efficacy, endpoints, disease activity, organ damage, quality of life (QoL), and Patient Reported Outcome Measures (PROMs) in the treatment of SLE.
Methods: A Delphi study was conducted using a national expert panel of clinicians in the treatment of SLE. A steering committee composed of 3 opinion leaders with deep expertise in SLE treatment was defined. The steering committee analyzed and appraised the evidence, designed the Delphi study, defined the statements, and analyzed the expert panel responses. A 2-round Delphi survey was conducted. Participants were asked to rate the statements using a five-point Likert scale.
Results: Nine experts participated in the Delphi survey. After the two rounds, the consensus was reached on 18 of the 23 statements: 2 statements were included in the “efficacy” domain, 2 in the “glucocorticoid-sparing” domain, 2 in the “endpoint evaluation” domain, 4 in the “score” domain, 1 in the “disease activity” domain, 1 in the “organ damage” domain, 1 in the “QoL” domain, 2 in the “PROMs” domain, 1 in the “AIFA monitoring” domain and 2 in the “extra” domain. No statements reached consensus within the “onset” domain.
Conclusion: In this Delphi study, 18 statements across 11 domains were agreed upon for the treatment of SLE.
Downloads
References
Justiz Vaillant AA, Goyal A, Varacallo M. Systemic Lupus Erythematosus. 2022 Jul 8. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022. PMID 30571026
Maidhof W, Hilas O. Lupus: an overview of the disease and management options. P&T. 2012;37(4):240-249. PMID:22593636
Sebastiani GD, Spinelli FR, Bartoloni E, et al. Baseline characteristics of systemic lupus erythematosus patients included in the Lupus Italian Registry of the Italian Society for Rheumatology. Lupus. 2021;30(8):1233-1243. PMID:33884900 https://doi.org/10.1177/09612033211012470 PMID:33884900 DOI: https://doi.org/10.1177/09612033211012470
Sebastiani GD, Prevete I, Iuliano A, et al. Early Lupus Project: one-year follow-up of an Italian cohort of patients with systemic lupus erythematosus of recent onset. Lupus. 2018;27(9):1479-1488. PMID:29779436 https://doi.org/10.1177/0961203318777112 PMID:29779436 DOI: https://doi.org/10.1177/0961203318777112
Sebastiani GD, Prevete I, Piga M, et al. Early Lupus Project - A multicentre Italian study on systemic lupus erythematosus of recent onset. Lupus. 2015;24(12):1276-1282. PMID:25979916 https://doi.org/10.1177/0961203315585817 PMID:25979916 DOI: https://doi.org/10.1177/0961203315585817
Prevete I, Espinosa G, Bellisai F, et al; Gruppo di Studio SIR sul Lupus Eritematoso Sistemico di recente insorgenza-Early Lupus and Registro Español de pacientes con Lupus Eritematoso Sistémico (RELES), Autoimmune Diseases Study Group (GEAS). Comparative study between two European inception cohorts of patients with early systemic lupus erythematosus. Clin Exp Rheumatol. 2020;38(5):925-932. PMID:31969225 PMID:31969225
Specchia ML, Cadeddu C, Di Pietro ML, et al. Health technology assessment of belimumab: a new monoclonal antibody for the treatment of systemic lupus erythematosus. BioMed Research International. 2014;2014:704207-704215. https://doi.org/10.1155/2014/704207 PMID: 25243173 DOI: https://doi.org/10.1155/2014/704207
Thong B, Olsen NJ. Systemic lupus erythematosus diagnosis and management. Rheumatology (Oxford). 2017;56(suppl 1):i3-i13. PMID:28013206
Lateef A, Petri M. Unmet medical needs in systemic lupus erythematosus. Arthritis Res Ther. 2012;14(suppl 4):S4. https://doi.org/10.1186/ar3919 PMID:23281889 DOI: https://doi.org/10.1186/ar3919
Gordon C, Amissah-Arthur MB, Gayed M, et al; British Society for Rheumatology Standards, Audit and Guidelines Working Group. The British Society for Rheumatology guideline for the management of systemic lupus erythematosus in adults. Rheumatology (Oxford). 2018;57(1):e1-e45. https://doi.org/10.1093/rheumatology/kex286 PMID:29029350 DOI: https://doi.org/10.1093/rheumatology/kex286
Tsioni V, Andreoli L, Meini A, et al. The prevalence and incidence of systemic lupus erythematosus in children and adults: a population-based study in a mountain community in northern Italy. Clin Exp Rheumatol. 2015;33(5):681-687. PMID:26089119
AstraZeneca. Ricerca di mercato 2021. Data on file.
Epidemiology of Systemic Lupus Erythematosus pages in Systemic Lupus Erythematosus. A Companion to Rheumatology. Mosby. 2007;1-18. Online https://www.sciencedirect.com/book/9780323044349/systemic-lupus-erythematosus
Zen M, Bassi N, Nalotto L, et al. Disease activity patterns in a monocentric cohort of SLE patients: a seven-year follow-up study. Clin Exp Rheumatol. 2012;30(6):856-863. PMID:22765883
Pons-Estel GJ, Alarcón GS, Scofield L, Reinlib L, Cooper GS. Understanding the epidemiology and progression of systemic lupus erythematosus. Semin Arthritis Rheum. 2010;39(4):257-268. https://doi.org/10.1016/j.semarthrit.2008.10.007 PMID:19136143 DOI: https://doi.org/10.1016/j.semarthrit.2008.10.007
Deijns SJ, Broen JCA, Kruyt ND et al. The immunologic etiology of psychiatric manifestations in systemic lupus erythematosus: A narrative review on the role of the blood brain barrier, antibodies, cytokines and chemokines. Autoimmun Rev. 2020 Aug;19(8):102592. https://doi.org/10.1016/j.autrev.2020.102592 PMID 32561462 DOI: https://doi.org/10.1016/j.autrev.2020.102592
Hess EV, Farhey Y. Etiology, environmental relationships, epidemiology, and genetics of systemic lupus erythematosus. Curr Opin Rheumatol. 1995;7(5):371-375. https://doi.org/10.1097/00002281-199509000-00002 PMID:8519608 DOI: https://doi.org/10.1097/00002281-199509000-00002
Aringer M, Costenbader K, Daikh D, et al. 2019 European League Against Rheumatism/American College of Rheumatology Classification Criteria for Systemic Lupus Erythematosus. Arthritis Rheumatol. 2019;71(9):1400-1412. https://doi.org/10.1002/art.40930PMID:31385462
Furie RA, Petri MA, Wallace DJ, et al. Novel evidence-based systemic lupus erythematosus responder index. Arthritis Rheum. 2009;61(9):1143-1151. https://doi.org/10.1002/art.24698PMID:19714615 DOI: https://doi.org/10.1002/art.24698
Mikdashi J, Nived O. Measuring disease activity in adults with systemic lupus erythematosus: the challenges of administrative burden and responsiveness to patient concerns in clinical research. Arthritis Res Ther. 2015;17(1):183. https://doi.org/10.1186/s13075-015-0702-6PMID:26189728 DOI: https://doi.org/10.1186/s13075-015-0702-6
Tanaka Y, Mizukami A, Kobayashi A, Ito C, Matsuki T. Disease severity and economic burden in Japanese patients with systemic lupus erythematosus: A retrospective, observational study. Int J Rheum Dis. 2018;21(8):1609-1618. https://doi.org/10.1111/1756-185X.13363PMID:30146745 DOI: https://doi.org/10.1111/1756-185X.13363
Navarra SV, Guzmán RM, Gallacher AE, et al; BLISS-52 Study Group. Efficacy and safety of belimumab in patients with active systemic lupus erythematosus: a randomised, placebo-controlled, phase 3 trial. Lancet. 2011;377(9767):721-731. https://doi.org/10.1016/S0140-6736(10)61354-2 PMID:21296403 DOI: https://doi.org/10.1016/S0140-6736(10)61354-2
Furie R, Petri M, Zamani O, et al; BLISS-76 Study Group. A phase III, randomized, placebo-controlled study of belimumab, a monoclonal antibody that inhibits B lymphocyte stimulator, in patients with systemic lupus erythematosus. Arthritis Rheum. 2011;63(12):3918-3930. https://doi.org/10.1002/art.30613 PMID:22127708 DOI: https://doi.org/10.1002/art.30613
Wallace DJ, Kalunian K, Petri MA, et al. Efficacy and safety of epratuzumab in patients with moderate/severe active systemic lupus erythematosus: results from EMBLEM, a phase IIb, randomised, double-blind, placebo-controlled, multicentre study. Ann Rheum Dis. 2014;73(1):183-190. https://doi.org/10.1136/annrheumdis-2012-202760 PMID:23313811 DOI: https://doi.org/10.1136/annrheumdis-2012-202760
Piga M, Floris A, Sebastiani GD, et al. Risk factors of damage in early diagnosed systemic lupus erythematosus: results of the Italian multicentre Early Lupus Project inception cohort. Rheumatology (Oxford). 2020;59(9):2272-2281. PMID:31840179 https://doi.org/10.1093/rheumatology/kez584 PMID:31840179 DOI: https://doi.org/10.1093/rheumatology/kez584
Polachek A, Gladman DD, Su J, Urowitz MB. Defining Low Disease Activity in Systemic Lupus Erythematosus. Arthritis Care Res (Hoboken). 2017;69(7):997-1003. https://doi.org/10.1002/acr.23109 PMID:27696791 DOI: https://doi.org/10.1002/acr.23109
Mahieu MA, Strand V, Simon LS, Lipsky PE, Ramsey-Goldman R. A critical review of clinical trials in systemic lupus erythematosus. Lupus. 2016;25(10):1122-1140. https://doi.org/10.1177/0961203316652492 PMID:27497257 DOI: https://doi.org/10.1177/0961203316652492
Lupus Research Alliance. Baricitinib Development as a Lupus Treatment Discontinued. Online https://lupustrials.org/lra-single/?id=10888 (Accessed January 2022).
Riggs JM, Hanna RN, Rajan B, et al. Characterisation of anifrolumab, a fully human anti-interferon receptor antagonist antibody for the treatment of systemic lupus erythematosus. Lupus Sci Med. 2018;5(1):e000261. https://doi.org/10.1136/lupus-2018-000261 PMID:29644082 DOI: https://doi.org/10.1136/lupus-2018-000261
Lichtman EI, Helfgott SM, Kriegel MA. Emerging therapies for systemic lupus erythematosus--focus on targeting interferon-alpha. Clin Immunol. 2012;143(3):210-221. https://doi.org/10.1016/j.clim.2012.03.005 PMID:22525889 DOI: https://doi.org/10.1016/j.clim.2012.03.005
Furie RA, Morand EF, Bruce IN et al. Type I interferon inhibitor anifrolumab in active systemic lupus erythematosus (TULIP-1): a randomised, controlled, phase 3 trial. The Lancet Rheumatology 2019;1(4):e219-e219. https://doi.org/10.1016/S2665-9913(19)30076-1 DOI: https://doi.org/10.1016/S2665-9913(19)30076-1
Morand EF, Furie R, Tanaka Y, et al; TULIP-2 Trial Investigators. Trial of Anifrolumab in Active Systemic Lupus Erythematosus. N Engl J Med. 2020;382(3):211-221. https://doi.org/10.1056/NEJMoa1912196 PMID:31851795 DOI: https://doi.org/10.1056/NEJMoa1912196
Preedy VR, Watson RR, eds. 5-Point Likert Scale. In: Handbook of Disease Burdens and Quality of Life Measures. Springer; 2010, https://doi.org/10.1007/978-0-387-78665-0_6363. DOI: https://doi.org/10.1007/978-0-387-78665-0
Humphrey-Murto S, Varpio L, Wood TJ, et al. The use of the Delphi and other consensus group methods in medical education research: a review. Acad Med. 2017;92(10):1491-1498. https://doi.org/10.1097/ACM.0000000000001812 PMID:28678098 DOI: https://doi.org/10.1097/ACM.0000000000001812
Schneider P, Evaniew N, Rendon JS, et al; PARITY Investigators. Moving forward through consensus: protocol for a modified Delphi approach to determine the top research priorities in the field of orthopaedic oncology. BMJ Open. 2016;6(5):e011780. https://doi.org/10.1136/bmjopen-2016-011780 PMID:27221129 DOI: https://doi.org/10.1136/bmjopen-2016-011780
Vogel C, Zwolinsky S, Griffiths C, Hobbs M, Henderson E, Wilkins E. A Delphi study to build consensus on the definition and use of big data in obesity research. Int J Obes. 2019;43(12):2573-2586. https://doi.org/10.1038/s41366-018-0313-9 PMID:30655580 DOI: https://doi.org/10.1038/s41366-018-0313-9
Ab Latif R, Dahlan A, Ab Mulud Z, Mat Nor MZ. The Delphi technique as a method to obtain consensus in health care education research. Educ Med J. 2017;9(3):89-102. https://doi.org/10.21315/eimj2017.9.3.10 DOI: https://doi.org/10.21315/eimj2017.9.3.10
De Meyer D, Kottner J, Beele H, et al. Delphi procedure in core outcome set development: rating scale and consensus criteria determined outcome selection. J Clin Epidemiol. 2019;111:23-31. https://doi.org/10.1016/j.jclinepi.2019.03.011 PMID:30922885 DOI: https://doi.org/10.1016/j.jclinepi.2019.03.011
Avella JR. Delphi panels: research design, procedures, advantages, and challenges. Int J Dr Stud. 2016;11:305-321. https://doi.org/10.28945/3561 DOI: https://doi.org/10.28945/3561
Akins RB, Tolson H, Cole BR. Stability of response characteristics of a Delphi panel: application of bootstrap data expansion. BMC Med Res Methodol. 2005;5(1):37. https://doi.org/10.1186/1471-2288-5-37 PMID:16321161 DOI: https://doi.org/10.1186/1471-2288-5-37
Iqbal S, Pipon-Young L. The Delphi method. Psychologist. 2009;22(7):598-601.
Downloads
Published
How to Cite
Issue
Section
Categories
License
Copyright (c) 2022 The authors
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Authors contributing to Global & Regional Health Technology Assessment agree to publish their articles under the CC-BY-NC 4.0 license, which allows third parties to re-use the work without permission as long as the work is properly referenced and the use is non-commercial.
Accepted 2022-08-30
Published 2022-10-10
